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Item 1A. Risk Factors

2021-03-30 compared with 2020-02-14 · 1412 added, 1389 removed, 691 unchanged (80% of the section changed)

An investment in our common shares involves

a significant risk of loss.

You should carefully read this entire Annual Report and should give particular attention to the

following risk factors.

You should recognize that other significant risks may arise in the future, which we cannot reasonably

foresee at this time.

Also, the risks that we now foresee might affect us to a greater or different degree than currently

There are a number of important factors that could cause our actual results to differ materially from those expressed

or implied by any of our forward-looking statements in this Annual Report.

These factors include, without limitation, the

risk factors listed below, and other factors presented throughout this Annual Report and any other documents filed by us with the

SEC and the Canadian securities regulators on SEDAR.

An investment in our common shares involves a significant risk

You should carefully read this entire Annual Report and should give particular attention to the following risk factors.

should recognize that other significant risks may arise in the future, which we cannot reasonably foresee at this time.

the risks that we now foresee might affect us to a greater or different degree than currently expected.

There are a number

of important factors that could cause our actual results to differ materially from those expressed or implied by any of our forward-looking

statements in this Annual Report.

These factors include, without limitation, the risk factors listed below, and other factors

presented throughout this Annual Report and any other documents filed by us with the SEC and the Canadian securities regulators

Risk Factors Summary

The following is a summary of the principal

risks that could adversely affect our business, operations, and financial results.

A more thorough discussion of these and other

risks follows this summary.

Risks Related to Our Business

We have a history of significant losses

and have had no revenues to date through the sale of our products.

If we do not generate significant revenues, we will not achieve

profitability.

To date, we have been engaged primarily

in research and development activities.

We have had no revenues through the sale of our products, and we do not expect to

have significant revenues until we are able to either sell our product candidate after obtaining applicable regulatory

approvals or we establish collaborations that provide us with up-front payments, licensing fees, milestone payments,

royalties or other revenue.

We have a history of significant losses and have generated de minimis licensing revenue and no

revenue from the sale of products since our inception.

PEDMARK TM is currently our only product candidate.

PEDMARK TM is still in

development and there is no assurance that it will receive regulatory approval or that we will successfully develop it into a commercially

viable product.

We may be required to conduct additional clinical trials for PEDMARK TM , which would

be costly and time-consuming to complete.

We may require additional financing to obtain regulatory approval for and commercialize PEDMARK TM ,

and a failure to obtain this capital when needed on acceptable terms, or at all, could force us to delay, limit, reduce, or terminate

our product development, other operations, or commercialization efforts.

We are currently and may in the future be the target of securities litigation, which may be costly

and time-consuming to defend.

Our business may be adversely affected by the ongoing COVID-19 pandemic.

Our business involves environmental risks and potential exposure to environmental liabilities.

Risks Related to the Clinical Development and Marketing Approval

of Our Product Candidate

PEDMARK TM has not received marketing approval from the FDA or any comparable foreign

These approval processes are costly, time-consuming, and inherently unpredictable, and it is possible that our applications

for marketing approval will be denied.

Our NDA for PEDMARK TM received a CRL from the FDA in August 2020, which identified

deficiencies in the third-party manufacturing facility that manufactures PEDMARK TM on our behalf.

Risks Related to Commercialization of Our Product Candidate

Even if we receive regulatory approvals for PEDMARK TM , it will still be subject to continued

regulatory review and could be subject to labeling and other restrictions.

Sales of PEDMARK TM , assuming it obtains regulatory approval, will depend on reimbursement

by payers and these payers are subject to pressures to contain costs.

In addition, coverage and reimbursement for PEDMARK TM

may be limited or unavailable in certain market segments.

PEDMARK TM targets diseases with small patient populations and we may not be effective

at identifying patients.

We may not be able to gain or maintain market acceptance of PEDMARK TM among the medical

community, patients, or payers.

If we fail to comply with applicable healthcare laws and regulations, we may be subject to investigations

and civil or criminal penalties and could lose any regulatory approvals that we obtain for PEDMARK TM .

We have just started to build our commercial team and have limited experience in commercializing

pharmaceutical products.

Changes in healthcare laws and regulations, as well as changes in healthcare policy, could adversely

affect our business.

PEDMARK TM may be subject to product liability claims.

Risks Related to Third Parties

We rely on third parties to supply raw materials, to conduct clinical trials, and to manufacture

If these third parties fail to satisfactorily perform for us, or if they fail to comply with applicable legal

and regulatory requirements, it could have a material adverse effect on our business.

The above-referenced CRL resulted from deficiencies

identified by the FDA in the third-party manufacturing facility that manufactures PEDMARK TM on our behalf.

Risks Related to Our Intellectual Property

PEDMARK TM is based on intellectual property exclusively licensed to us by OHSU, and

the license is terminable in the event of a breach by us under the license agreement.

If we were to lose our license from OHSU,

we may be required to terminate any development and commercialization of PEDMARK TM .

If we cannot obtain new patents, maintain our existing patents, and protect our trade secrets and

other intellectual property, our business and competitive position may be harmed.

Patent protection for PEDMARK TM may expire before we are able to realize its commercial

Changes in United States patent law could diminish the value of patents in general, thereby impairing

our ability to protect PEDMARK TM .

If we are found to be infringing third-party patents, we may be forced to pay damages and/or obtain

If we cannot obtain a license, we may be prevented from the development, manufacture, or commercialization of PEDMARK TM .

It is possible that we could lose market exclusivity for PEDMARK TM earlier than expected.

Risks Related to Our Industry

Drug development involves a lengthy and expensive process with an uncertain outcome, and results

of earlier studies and trials may not be predictive of future trial results.

The biotechnology and pharmaceutical industry, and in particular the field of cancer therapeutics

where we are focused, is highly competitive.

We face significant competition from other pharmaceutical, biopharmaceutical, and

biotechnology companies, many of which have significantly greater financial, technical, and human resources than we do and may

be better equipped to develop, manufacture, and market products.

Risks Related to Owning Our Common Shares

We may be unable to maintain the listing of our common shares on the Nasdaq Capital Market or the

TSX, which would make it more difficult for shareholders to dispose of our common shares.

The market price of our common shares is highly volatile and could cause the value of your investment

to significantly decline.

Our existing principal shareholders hold a substantial number of our common shares and may be able

to exercise influence in matters requiring approval of our shareholders.

There are a large number of our common shares underlying outstanding options, and reserved for

issuance under our stock option plan, that may be sold in the market, which could depress the market price of our shares and result

in substantial dilution to our shareholders.

We may need to raise additional funds in the future to continue our operations.

Any equity offering

could result in significant dilution to our shareholders and decrease the value of our common shares.

Any debt offering will increase

our financial risk.

We have not paid any dividends and do not anticipate declaring any dividends in the foreseeable

We may be a passive foreign investment company, which could result in adverse United States federal

income tax consequences to U.S.

Risks Related to Our Business

We have a history of significant losses and have had no revenues

to date through the sale of our products.

If we do not generate significant revenues, we will not achieve profitability.

date, we have been engaged primarily in research and development activities.

We have had no revenues through the commercial sale

of products, and we do not expect to have significant revenues until we are able to either sell our product candidate after obtaining

applicable regulatory approvals or we establish collaborations that provide us with up-front payments, licensing fees, milestone

payments, royalties or other revenue.

We have incurred significant operating losses every year since our inception on September 3,

We reported a loss of approximately $12.8 million for the year ended December 31, 2019 and reported a net loss of

approximately $9.9 million (which included a non-cash loss on derivative liabilities of $0.2 million) for the year ended

December 31, 2018.

At December 31, 2019, we had an accumulated deficit of approximately $144.0 million.

We anticipate

incurring substantial additional losses due to the need to spend substantial amounts on activities required for regulatory

approval of PEDMARK TM , commercial launch preparation of PEDMARK TM , anticipated research and development

activities, and general and administrative expenses, among other factors.

We have not commercially introduced any products.

Our ability to attain profitability will depend upon our ability to fund and develop products that are safe, effective and

commercially viable, to obtain regulatory approval for the manufacture and sale of our product candidate and to license or

otherwise market our product candidate successfully.

We reported a net loss of approximately $18.1 million for the year ended December 31, 2020 and reported a net

loss of approximately $12.8 million for the year ended December 31, 2019.

At December 31, 2020, we had an accumulated

deficit of approximately $162.1 million.

We anticipate incurring substantial additional losses due to the need to spend substantial

amounts on activities required for regulatory approval of PEDMARK TM , commercial launch preparation of PEDMARK TM ,

anticipated research and development activities, and general and administrative expenses, among other factors.

We have not commercially

introduced any products.

Our ability to attain profitability will depend upon our ability to fund and develop products that are

safe, effective and commercially viable, to obtain regulatory approval for the manufacture and sale of our product candidate and

to license or otherwise market our product candidate successfully.

Any revenues generated from such product, assuming it is successfully

developed, marketed and sold, may not be realized for a number of years.

We may never achieve or sustain profitability on an

ongoing basis.

PEDMARK TM is currently our only

product candidate and there is no assurance that we will successfully develop PEDMARK TM into a commercially viable product.

Since our formation in September 1996, we

have engaged in research and development programs.

We have generated no revenue from product sales, do not have any products currently

available for sale, and none are expected to be commercially available for sale until we have completed regulatory approval of

We may never achieve or sustain profitability on an ongoing

is currently our only product candidate and there is no assurance that we will successfully develop PEDMARK TM into a

commercially viable product.

Since our formation in September 1996, we have engaged

in research and development programs.

We have generated no revenue from product sales, do not have any products currently available

for sale, and none are expected to be commercially available for sale until we have completed regulatory approval of PEDMARK TM .

PEDMARK TM is currently our only product candidate.

There can be no assurance that the research

we fund and manage will lead PEDMARK TM or any future product candidate to become a commercially viable product.

completed two-Phase 3 studies for PEDMARK TM and completed submission of our NDA in the U.S.

We anticipate substantial

regulatory review prior to the commercialization of PEDMARK TM .

We may require additional financing to

obtain marketing approval of PEDMARK TM and commercialize PEDMARK TM and a failure to obtain this capital when

needed on acceptable terms, or at all, could force us to delay, limit, reduce or terminate our product development, other operations

or commercialization efforts.

Based on available resources, we believe that

our cash and cash equivalents of $13.7 million available and the $12.5 million debt facility announced in February 2019 (under

which we had not yet made an borrowings) are sufficient to fund our anticipated operating and capital requirements to NDA approval

and the commencement of commercialization efforts expected to occur later in 2020, subject to approval of our NDA.

expect to continue to incur losses for the foreseeable future as we continue our development of and seek marketing approvals for

Further, we may not be able to secure NDA approval prior to the expiration of our debt facility in September

There can be no assurance that the research we fund and manage will

lead PEDMARK TM or any future product candidate to become a commercially viable product.

We have completed two-Phase

3 studies for PEDMARK TM .

We anticipate substantial regulatory review prior to the commercialization of PEDMARK TM .

may require additional financing to obtain marketing approval of PEDMARK TM and commercialize PEDMARK TM

and a failure to obtain this capital when needed on acceptable terms, or at all, could force us to delay, limit, reduce or terminate

our product development, other operations or commercialization efforts.

Based on available resources, we believe that our cash and cash

equivalents of $30.3 million available as of December 31, 2020 are sufficient to fund our anticipated operating and capital

requirements to NDA approval and the commencement of commercialization efforts, subject to approval of our NDA.

Moreover, we expect

to continue to incur losses for the foreseeable future as we continue our development of and seek marketing approvals for PEDMARK TM .

We may not be able to obtain additional financing in sufficient amounts or on acceptable terms when needed.

If we fail to

arrange for sufficient capital on a timely basis, we may be required to curtail our business activities until we can obtain adequate

If we fail to arrange

for sufficient capital on a timely basis, we may be required to curtail our business activities until we can obtain adequate financing.

Debt financing must be repaid regardless of whether or not we generate profits or cash flows from our business activities.

Equity financing may result in dilution to existing shareholders and may involve securities that have rights, preferences, or privileges

financing may result in dilution to existing shareholders and may involve securities that have rights, preferences, or privileges

that are senior to our common shares or other securities.

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have to curtail operations and you may lose part or all of your investment.

If we do not maintain current or enter

into new collaborations with other companies, we might not successfully develop our product candidate or generate sufficient revenues

to expand our business.

We currently rely on scientific and research

and development collaboration arrangements with academic institutions and other third-party collaborators, including an exclusive

worldwide license from OHSU for PEDMARK TM .

We also rely on collaborators for testing PEDMARK TM , including

SIOPEL and the Children’s Oncology Group.

The agreements with OHSU are terminable by

either party in the event of an uncured breach by the other party.

We may also terminate our agreement with OHSU at any time upon

prior written notice of specified durations to OHSU.

Termination of any of our collaborative arrangements could materially adversely

affect our business.

For example, if we are unable to make the necessary payments under these agreements, the licensor might terminate

the agreement which might have a material adverse impact.

If we do not maintain current or enter into new collaborations

with other companies, we might not successfully develop our product candidate or generate sufficient revenues to expand our business.

currently rely on scientific and research and development collaboration arrangements with academic institutions and other third-party

collaborators, including an exclusive worldwide license from OHSU for PEDMARK TM .

We also rely on collaborators

for testing PEDMARK TM , including SIOPEL and the Children’s Oncology Group.

The agreements with OHSU are terminable by either party in the

event of an uncured breach by the other party.

We may also terminate our agreement with OHSU at any time upon prior written notice

of specified durations to OHSU.

Termination of any of our collaborative arrangements could materially adversely affect our business.

For example, if we are unable to make the necessary payments under these agreements, the licensor might terminate the agreement

which might have a material adverse impact.

In addition, our collaborators might not perform as agreed in the future.

Since we conduct a significant portion of

our research and development through collaborations, our success may depend significantly on the performance of such collaborators,

as well as any future collaborators.

Collaborators might not commit sufficient resources to the research and development or commercialization

of our product candidate.

Economic or technological advantages of products being developed by others, among other factors, could

lead our collaborators to pursue other product candidates or technologies in preference to those being developed in collaboration

The commercial potential of, development stage of and projected resources required to develop our drug candidate will

affect our ability to maintain current collaborations or establish new collaborators.

There is a risk of dispute with respect to

ownership of technology developed under any collaboration.

Our management of any collaboration will require significant time and

effort as well as an effective allocation of resources.

We may not be able to simultaneously manage a large number of collaborations.

we conduct a significant portion of our research and development through collaborations, our success may depend significantly on

the performance of such collaborators, as well as any future collaborators.

Collaborators might not commit sufficient resources

to the research and development or commercialization of our product candidate.

Economic or technological advantages of products

being developed by others, among other factors, could lead our collaborators to pursue other product candidates or technologies

in preference to those being developed in collaboration with us.

The commercial potential of, development stage of and projected

resources required to develop our drug candidate will affect our ability to maintain current collaborations or establish new collaborators.

There is a risk of dispute with respect to ownership of technology developed under any collaboration.

Our management of any collaboration

will require significant time and effort as well as an effective allocation of resources.

We may not be able to simultaneously

manage a large number of collaborations .

Any of these negative impacts on our current or future collaborations could have

a material adverse effect on our business and results of operations.

Our product candidate is still in development.

Due to the long, expensive and unpredictable drug development process, we might not ever successfully develop and commercialize

our product candidate.

In order to achieve profitable operations,

we, alone or in collaboration with others, must successfully fund, develop, manufacture, introduce and market our product candidate.

The time necessary to achieve market success for any individual product is long and uncertain.

Our product candidate and research

programs are in clinical development and require significant, time-consuming and costly research, testing and regulatory clearances.

In developing our product candidate, we are subject to risks of failure that are inherent in the development of therapeutic products

based on innovative technologies.

The results of preclinical and initial clinical trials are not necessarily predictive of future

Our product candidate might not be economical to manufacture or market or

might not achieve market acceptance.

third parties might hold proprietary rights that preclude us from marketing our product candidate or others might market equivalent

or superior products.

We may need to conduct additional human

clinical trials to assess our product candidate.

If these trials are delayed or are unsuccessful, our development costs will significantly

increase, and our business prospects may suffer.

Before obtaining regulatory approvals for

the commercial sale of our product candidate, we must demonstrate, through preclinical studies with animals and clinical trials

with humans, that our product candidate is safe and effective for use in each target indication.

To date, we have performed only

limited clinical trials.

Much of our testing has been conducted on animals or on human cells in the laboratory, and the benefits

of treatment seen in animals or on human cells in a laboratory setting may not ultimately be obtained in human clinical trials.

As a result, we may need to perform significant additional research and development activities and conduct extensive preclinical

and clinical testing prior to any application for commercial use.

We may suffer significant setbacks in additional clinical trials,

and the trials may demonstrate our product candidate to be unsafe or ineffective.

We may also encounter problems in our clinical

trials that will cause us to delay, suspend or terminate those clinical trials, which would increase our development costs and

harm our financial results and commercial prospects.

Identifying and qualifying patients to participate in clinical trials of our

potential products is critically important to our success.

The timing of our clinical trials depends on, among other things, the

speed at which we can recruit patients to participate in testing our product candidate.

We have experienced delays in some of our

clinical trials and we may experience significant delays in the future.

If patients are unwilling to participate in our trials

because of competing clinical trials for similar patient populations, perceived risk or any other reason, the timeline for recruiting

patients, conducting trials and obtaining regulatory approval of potential products will be delayed.

Other factors that may result

in significant delays include obtaining regulatory or ethics review board approvals for proposed trials, reaching agreement on

acceptable terms with prospective clinical trial sites, and obtaining sufficient quantities of drugs for use in the clinical trials.

Such delays could result in the termination of the clinical trials altogether.

Regulatory approval of our product candidate

is time-consuming, expensive and uncertain, and could result in unexpectedly high expenses and delay our ability to sell our product.

Development, manufacture and marketing of

our product is subject to extensive regulation by governmental authorities in the United States and other countries.

This regulation

could require us to incur significant unexpected expenses or delay or limit our ability to sell our product candidate.

studies might be delayed or halted, or additional studies might be required, for various reasons, including:

long, expensive and unpredictable drug development process, we might not ever successfully develop and commercialize our product

order to achieve profitable operations, we, alone or in collaboration with others, must successfully fund, develop, manufacture,

introduce and market our product candidate.

The time necessary to achieve market success for any individual product is long and

Our product candidate and research programs are in clinical development and require significant, time-consuming and

costly research, testing and regulatory clearances.

In developing our product candidate, we are subject to risks of failure that

are inherent in the development of therapeutic products based on innovative technologies.

The results of preclinical and initial

clinical trials are not necessarily predictive of future results .

Our product candidate might not be economical to manufacture

or market or might not achieve market acceptance.

In addition, third parties might hold proprietary rights that preclude us from

marketing our product candidate or others might market equivalent or superior products.

We may need to conduct additional human clinical trials to

assess our product candidate.

If these trials are delayed or are unsuccessful, our development costs will significantly increase,

and our business prospects may suffer.

Before obtaining regulatory approvals for the commercial sale

of our product candidate, we must demonstrate, through preclinical studies with animals and clinical trials with humans, that our

product candidate is safe and effective for use in each target indication.

To date, we have performed only limited clinical trials.

Much of our testing has been conducted on animals or on human cells in the laboratory, and the benefits of treatment seen in animals

or on human cells in a laboratory setting may not ultimately be obtained in human clinical trials.

As a result, we may need to

perform significant additional research and development activities and conduct extensive preclinical and clinical testing prior

to any application for commercial use.

We may suffer significant setbacks in additional clinical trials, and the trials may demonstrate

our product candidate to be unsafe or ineffective.

We may also encounter problems in our clinical trials that will cause us to

delay, suspend or terminate those clinical trials, which would increase our development costs and harm our financial results and

commercial prospects.

Identifying and qualifying patients to participate in clinical trials of our potential products is critically

important to our success.

The timing of our clinical trials depends on, among other things, the speed at which we can recruit patients

to participate in testing our product candidate.

We have experienced delays in some of our clinical trials and we may experience

significant delays in the future.

If patients are unwilling to participate in our trials because of competing clinical trials for

similar patient populations, perceived risk or any other reason, the timeline for recruiting patients, conducting trials and obtaining

regulatory approval of potential products will be delayed.

Other factors that may result in significant delays include obtaining

regulatory or ethics review board approvals for proposed trials, reaching agreement on acceptable terms with prospective clinical

trial sites, and obtaining sufficient quantities of drugs for use in the clinical trials.

Such delays could result in the termination

of the clinical trials altogether.

Regulatory approval of our product candidate is time-consuming,

expensive and uncertain, and could result in unexpectedly high expenses and delay our ability to sell our product.

Development, manufacture and marketing of our product is subject

to extensive regulation by governmental authorities in the United States and other countries.

This regulation could require us

to incur significant unexpected expenses or delay or limit our ability to sell our product candidate.

Our clinical studies might

be delayed or halted, or additional studies might be required, for various reasons, including:

there is a lack of sufficient funding;

the drug is not effective;

patients experience severe side effects during

appropriate patients do not enroll in the

studies at the rate expected;

drug supplies are not sufficient to treat

the patients in the studies;

patients experience severe side effects during treatment;

appropriate patients do not enroll in the studies at the rate expected;

drug supplies are not sufficient to treat the patients in the studies;

we decide to modify the drug during testing.

If regulatory approval of our product is granted,

it will be limited to those indications for which the product has been shown to be safe and effective, as demonstrated to the satisfaction

If regulatory approval of our product is granted, it will be

limited to those indications for which the product has been shown to be safe and effective, as demonstrated to the satisfaction

of the FDA and foreign regulators through clinical studies.

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of them might impair our ability to effectively market our product.

We and our third-party manufacturers are also

required to comply with the applicable current FDA Good Manufacturing Practices regulations, which include requirements relating

to quality control and quality assurance, as well as the corresponding maintenance of records and documentation.

Further, manufacturing

facilities, which we outsource to third parties, must be approved by the FDA before they can be used to manufacture our product,

and they are subject to additional FDA inspection.

If we fail to comply with any of the FDA’s continuing regulations, we

could be subject to reputational harm and sanctions, including:

We and our third-party manufacturers are also required to comply

with the applicable current FDA Good Manufacturing Practices regulations, which include requirements relating to quality control

and quality assurance, as well as the corresponding maintenance of records and documentation.

Further, manufacturing facilities,

which we outsource to third parties, must be approved by the FDA before they can be used to manufacture our product, and they are

subject to additional FDA inspection.

The CRL that we received from the FDA in August, 2020 as a result of deficiencies in the

third-party manufacturing facility that manufactures PEDMARK TM on our behalf is a specific example of the risks associated

with our third-party manufacturers.

If we fail to comply with any of the FDA’s continuing

regulations, we could be subject to reputational harm and sanctions, including:

delays, warning letters and fines;

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total or partial suspension of production;

withdrawals of previously approved marketing

applications;

withdrawals of previously approved marketing applications;

civil penalties and criminal prosecutions.

In addition, identification of side effects

after a drug is on the market or the occurrence of manufacturing problems could cause subsequent withdrawal of approval, reformulation

of the drug, additional testing or changes in labeling of the product.

We may be unable to effectively deploy

the proceeds from our recent financings for the development of PEDMARK TM .

In February 2019,

the Company announced a $12.5 million debt facility available to the Company upon approval of PEDMARK TM .

Any inability

on our part to manage effectively the deployment of this capital could limit our ability to successfully develop PEDMARK TM .

Further, under the terms of the debt facility to be issued we must obtain NDA approval prior to September 30, 2020.

If our licenses to proprietary technology

owned by others are terminated or expire, we may suffer increased development costs and delays, and we may not be able to successfully

develop our product candidate.

The development of our drug candidate and

the manufacture and sale of any products that we develop will involve the use of processes, products and information, some of the

rights to which are owned by others.

STS is licensed under agreements with OHSU.

In addition, identification of side effects after a drug is

on the market or the occurrence of manufacturing problems could cause subsequent withdrawal of approval, reformulation of the drug,

additional testing or changes in labeling of the product.

may be unable to effectively deploy the proceeds from our recent financings for the development of PEDMARK TM .

May 5, 2020, we announced the completion of an underwritten public offering of 4.8 million of our common shares at a public

offering price of $6.25 per share.

In addition, we issued an additional 0.66 million common shares in connection with the partial

exercise of the underwriters’

over-allotment option.

The approximate total gross proceeds from the offering were $34.1 million

($32.2 million net of commissions, fees and issue costs).

Any inability on our part to manage effectively the deployment

of this capital could limit our ability to successfully develop PEDMARK TM .

If our licenses to proprietary technology owned by others

are terminated or expire, we may suffer increased development costs and delays, and we may not be able to successfully develop

our product candidate.

The development of our drug candidate and the manufacture and

sale of any products that we develop will involve the use of processes, products and information, some of the rights to which are

owned by others.

PEDMARK TM is licensed under agreements with OHSU.

Although we have obtained licenses or rights with

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not be granted.

If we are unable to adequately protect

or maintain our patents and licenses related to our product candidate, or if we infringe upon the intellectual property rights

of others, we may not be able to successfully develop and commercialize our product candidate.

The value of our technology will depend in

part upon our ability, and those of our collaborators, to obtain patent protection or licenses to patents, maintain trade secret

protection and operate without infringing on the rights of third parties.

Although we have successfully pursued patent applications

in the past, it is possible that:

some or all of our pending patent applications,

or those we have licensed, may not be allowed;

proprietary products or processes that we

develop in the future may not be patentable;

any issued patents that we own or license

may not provide us with any competitive advantages or may be successfully challenged by third parties;

the patents of others may have an adverse

effect on our ability to do business.

It is not possible for us to be certain that

we are the original and first creator of inventions encompassed by our pending patent applications or that we were the first to

file patent applications for any such inventions.

Further, any of our patents, once issued, may be declared by a court to be invalid

or unenforceable.

STS is currently protected by methods of use

patents that we exclusively licensed from OHSU that expire in Europe and the United States in 2021 and additional patents that

are currently pending in the United States.

In addition, periods of marketing exclusivity for STS may also be possible in the United

States under orphan drug status.

We obtained Orphan Drug Designation in the United States for the use of STS in the prevention

of platinum-induced ototoxicity in pediatric patients in 2004;

if it is subsequently approved, we will have seven and a half years

of pediatric exclusivity in the United States from the approval date.

Refer to the “Description of Business”

of this Annual Report for a further description of the United States Orphan Drug Designation.

We may be required to obtain licenses under

patents or other proprietary rights of third parties, but the extent to which we may wish or need to do so is unknown.

licenses may not be available on terms acceptable to us or at all.

If such licenses are obtained, it is likely they would be royalty

bearing, which would reduce any future income.

If licenses cannot be obtained on an economical basis, we could suffer delays in

market introduction of planned products or their introduction could be prevented, in some cases after the expenditure of substantial

If we do not obtain such licenses, we would have to design around patents of third parties, potentially causing increased

If we are unable to adequately protect or maintain our patents

and licenses related to our product candidate, or if we infringe upon the intellectual property rights of others, we may not be

able to successfully develop and commercialize our product candidate.

The value of our technology will depend in part upon our ability,

and those of our collaborators, to obtain patent protection or licenses to patents, maintain trade secret protection and operate

without infringing on the rights of third parties.

Although we have successfully pursued patent applications in the past, it is

possible that:

some or all of our pending patent applications, or those we have licensed, may not be allowed;

proprietary products or processes that we develop in the future may not be patentable;

any issued patents that we own or license may not provide us with any competitive advantages or may be successfully challenged

by third parties;

the patents of others may have an adverse effect on our ability to do business.

It is not possible for us to be certain that we are the original

and first creator of inventions encompassed by our pending patent applications or that we were the first to file patent applications

for any such inventions.

Further, any of our patents, once issued, may be declared by a court to be invalid or unenforceable.

PEMARK TM is currently protected by methods of use

patents that we exclusively licensed from OHSU that expire in Europe in 2021 and the United States in 2038 and a patent owned by

us that expires in 2039.

Further, patents are currently pending in the United States and other territories.

In addition, periods

of marketing exclusivity for PEDMARK TM may also be possible in the United States under orphan drug status.

We may be required to obtain licenses under patents or other

proprietary rights of third parties, but the extent to which we may wish or need to do so is unknown.

Any such licenses may not

be available on terms acceptable to us or at all.

If such licenses are obtained, it is likely they would be royalty bearing, which

would reduce our future income, if any.

If licenses cannot be obtained on an economical basis, we could suffer delays in market

introduction of planned products or their introduction could be prevented, in some cases after the expenditure of substantial funds.

If we do not obtain such licenses, we would have to attempt to design around patents of third parties, potentially causing increased

costs and delays in product development and introduction or precluding us from developing, manufacturing or selling our planned

products, or our ability to develop, manufacture or sell products requiring such licenses could be foreclosed.

Litigation may also be necessary to enforce

or defend patents issued or licensed to us or our collaborators or to determine the scope and validity of a third party’s

proprietary rights.

We could incur substantial costs if litigation is required to defend ourselves in patent suits brought by third

parties, if we participate in patent suits brought against or initiated by our collaborators, or if we initiate such suits.

might not prevail in any such action.

An adverse outcome in litigation or an interference to determine priority or other proceeding

in a court or patent office could subject us to significant liabilities, require disputed rights to be licensed from other parties

or require us or our collaborators to cease using certain technology or products.

Any of these events would likely have a material

adverse effect on our business, financial condition and results of operations.

Much of our technological know-how that is

not patentable may constitute trade secrets.

Our confidentiality agreements might not provide for meaningful protection of our

trade secrets, know-how or other proprietary information in the event of any unauthorized use or disclosure of information.

addition, others may independently develop or obtain similar technology and may be able to market competing products and obtain

regulatory approval through a showing of equivalency to our product that has obtained regulatory approvals, without being required

to undertake the same lengthy and expensive clinical studies that we would have already completed.

The vulnerability to off-label use or sale

of our product candidate that are covered only by “method of use”

patents may cause downward pricing pressure on the

product candidate if they are ever commercialized and may make it more difficult for us to enter into collaboration or partnering

arrangements for the development of this product candidate.

STS is currently only covered by “method

of use”

patents, which covers the use of certain compounds to treat specific conditions and are not covered by “composition

of matter”

patents, which would cover the chemical composition of the compound.

Method of use patents provide less protection

than composition of matter patents because of the possibility of off-label competition if other companies develop or market the

compound for other uses.

If another company markets a drug that we expect to market under the protection of a method of use patent,

physicians may prescribe the other company’s drug for use in the indication for which we obtain approval and have a patent,

even if the other company’s drug is not approved for such an indication.

Off-label use and sales could limit our sales and

exert pricing pressure on any product we develop covered only by method of use patents.

Also, it may be more difficult to find

a collaborator to license or support the development of our product candidate that is only covered by method of use patents.

If our third-party manufacturers breach

or terminate their agreements with us, or if we are unable to secure arrangements with third party manufacturers on acceptable

terms as needed in the future, we may suffer significant delays and additional costs.

We have no experience manufacturing products

and do not currently have the resources to manufacture any products that we may develop.

We currently have agreements with contract

manufacturers for clinical supplies of PEDMARK TM , including drug substance providers and drug product suppliers, but

they might not perform as agreed in the future or may terminate our agreements with them before the end of the required term.

additional time and expense would be required to effect a transition to a new contract manufacturer.

We plan to continue to rely on contract manufacturers

for the foreseeable future to produce quantities of products and substances necessary for research and development, preclinical

trials, human clinical trials and product commercialization, and to perform their obligations in a timely manner and in accordance

with applicable government regulations.

Litigation may also be necessary to enforce or defend patents

issued or licensed to us or our collaborators or to determine the scope and validity of a third party’s proprietary rights.

We could incur substantial costs if litigation is required to defend ourselves in patent suits brought by third parties, if we

participate in patent suits brought against or initiated by our collaborators, or if we initiate such suits.

We might not prevail

in any such action.

An adverse outcome in litigation or an interference to determine priority or other proceeding in a court or

patent office could subject us to significant liabilities, require disputed rights to be licensed from other parties or require

us or our collaborators to cease using certain technology or products.

Any of these events would likely have a material adverse

effect on our business, financial condition and results of operations.

Much of our technological know-how that is not patentable may

constitute trade secrets.

Our confidentiality agreements might not provide for meaningful protection of our trade secrets, know-how

or other proprietary information in the event of any unauthorized use or disclosure of information.

In addition, others may independently

develop or obtain similar technology and may be able to market competing products and obtain regulatory approval through a showing

of equivalency to our product that has obtained regulatory approvals, without being required to undertake the same lengthy and

expensive clinical studies that we would have already completed.

The vulnerability to off-label use or sale of our product

candidate that are covered only by “method of use”

patents may cause downward pricing pressure on the product candidate

if they are ever commercialized and may make it more difficult for us to enter into collaboration or partnering arrangements for

the development of this product candidate.

PEDMARK TM is currently covered by “method of

patents and “composition of matter”

“Method of use”

patents cover the use of certain

compounds to treat specific conditions and “composition of matter”

patents cover the chemical composition of the compound.

Method of use patents provide less protection than composition of matter patents because of the possibility of off-label competition

if other companies develop or market the compound for other uses.

If another company markets a drug that we expect to market under

the protection of a method of use patent, physicians may prescribe the other company’s drug for use in the indication for

which we obtain approval and have a patent, even if the other company’s drug is not approved for such an indication.

use and sales could limit our sales and exert pricing pressure on any product we develop covered only by method of use patents.

Also, it may be more difficult to find a collaborator to license or support the development of our product candidate that is only

covered by method of use patents.

If our third-party manufacturers breach or terminate their

agreements with us, or if we are unable to secure arrangements with third party manufacturers on acceptable terms as needed in

the future, we may suffer significant delays and additional costs.

We have no experience manufacturing products and do not currently

have the resources to manufacture any products that we may develop.

We currently have agreements with contract manufacturers for

clinical supplies of PEDMARK TM , including drug substance providers and drug product suppliers, but they might not perform

as agreed in the future or may terminate our agreements with them before the end of the required term.

Significant additional time

and expense would be required to effect a transition to a new contract manufacturer.

We plan to continue to rely on contract manufacturers for the

foreseeable future to produce quantities of products and substances necessary for research and development, preclinical trials,

human clinical trials and product commercialization, and to perform their obligations in a timely manner and in accordance with

applicable government regulations.

If we develop any product with commercial potential, we will need to develop the facilities

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withdrawals and other problems that could significantly hurt our business.

We intend to maintain a second source for back-up commercial

manufacturing, wherever feasible.

However, if a replacement to our future internal or contract manufacturers were required, the

ability to establish second-sourcing or find a replacement manufacturer may be difficult due to the lead times generally required

The CRL that we received from the FDA in August, 2020

as a result of deficiencies in the third-party manufacturing facility that manufactures PEDMARK TM on our behalf is a

specific example of the risks associated with our third-party manufacturers.

We intend to maintain a second source for back-up

commercial manufacturing, wherever feasible.

However, if a replacement to our future internal or contract manufacturers were required,

the ability to establish second-sourcing or find a replacement manufacturer may be difficult due to the lead times generally required

to manufacture drugs and the need for FDA compliance inspections and approvals of any replacement manufacturer, all of which factors

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might be twelve months or longer.

We may lack the resources necessary to

effectively market our product candidate, and we may need to rely on third parties over whom we have little or no control and who

may not perform as expected.

We may not have the necessary resources to

market our product candidate.

If we develop any products with commercial potential, we will either have to develop a marketing

capability, including a sales force, which is difficult and expensive to implement successfully, or attempt to enter into a collaboration,

merger, joint venture, license or other arrangement with third parties to provide a substantial portion of the financial and other

resources needed to market such products.

We may lack the resources necessary to effectively market

our product candidate, and we may need to rely on third parties over whom we have little or no control and who may not perform

We may not have the necessary resources to market our product

If we develop any products with commercial potential, we will either have to develop a marketing capability, including

a sales force, which is difficult and expensive to implement successfully, or attempt to enter into a collaboration, merger, joint

venture, license or other arrangement with third parties to provide a substantial portion of the financial and other resources

needed to market such products.

We may not be able to do so on acceptable terms, if at all.

If we rely extensively on

third parties to market our products, the commercial success of such products may be largely outside of our control.

We conduct our business internationally

and are subject to laws and regulations of several countries which may affect our ability to access regulatory agencies and may

affect the enforceability and value of our licenses.

We have conducted clinical trials in the United

States, Canada, Europe and the Pacific Rim and intend to, or may, conduct future clinical trials in these and other jurisdictions.

There can be no assurance that any sovereign government will not establish laws or regulations that will be deleterious to our

There is no assurance that we, as a British Columbia corporation, will continue to have access to the regulatory agencies

in any jurisdiction where we might want to conduct clinical trials or obtain regulatory approval, and we might not be able to enforce

our licenses or patent rights in foreign jurisdictions.

Foreign exchange controls may have a material adverse effect on our business

and financial condition, since such controls may limit our ability to flow funds into or out of a particular country to meet obligations

under licenses, clinical trial agreements or other collaborations.

Our cash invested in money market funds

might be subject to loss.

Even though we believe we take a conservative

approach to investing our funds, the nature of financial markets exposes us to investment risk, including the risks that the value

and liquidity of our money market investments could deteriorate significantly and the issuers of the investments we hold could

be subject to credit rating downgrades.

While we have not experienced any loss or write down of our money market investments in

the past, we cannot guarantee that such losses will not occur in future periods.

Risks Related to the Clinical Development

and Marketing Approval of Our Product Candidate

The marketing approval processes of the

FDA and comparable foreign authorities are lengthy, time-consuming and inherently unpredictable, and if we are ultimately unable

If we rely extensively on third parties

to market our products, the commercial success of such products may be largely outside of our control.

We conduct our business internationally and are subject to

laws and regulations of several countries which may affect our ability to access regulatory agencies and may affect the enforceability

and value of our licenses.

We have conducted clinical trials in the United States, Canada,

Europe and the Pacific Rim and intend to, or may, conduct future clinical trials in these and other jurisdictions.

no assurance that any sovereign government will not establish laws or regulations that will be deleterious to our interests.

is no assurance that we, as a British Columbia corporation, will continue to have access to the regulatory agencies in any jurisdiction

where we might want to conduct clinical trials or obtain regulatory approval, and we might not be able to enforce our licenses

or patent rights in foreign jurisdictions.

Foreign exchange controls may have a material adverse effect on our business and financial

condition, since such controls may limit our ability to flow funds into or out of a particular country to meet obligations under

licenses, clinical trial agreements or other collaborations.

Our cash invested in money market funds might be subject

Even though we believe we take a conservative approach to investing

our funds, the nature of financial markets exposes us to investment risk, including the risks that the value and liquidity of our

money market investments (the amounts of which substantially exceed the $250,000 amount insured by the FDIC) could deteriorate

significantly and the issuers of the investments we hold could be subject to credit rating downgrades.

While we have not experienced

any loss or write down of our money market investments in the past, we cannot guarantee that such losses will not occur in future

clinical development process be successfully completed, our ability to derive revenues from the sale of therapeutics will depend

upon our first obtaining FDA as well as foreign regulatory approvals, all of which are subject to a number of unique risks and

uncertainties.

Even if we are

able to demonstrate the safety and efficacy of our product candidate in clinical trials, if we fail to gain timely approval to

commercialize PEDMARK TM from the FDA and other foreign regulatory authorities, we will be unable to generate the revenues

we will need to build our business.

The FDA or comparable regulatory authorities in other countries may delay, limit or deny approval

of PEDMARK TM for various reasons.

For example, such authorities may disagree with the design, scope or implementation

of our clinical trials;

or with our interpretation of data from our preclinical studies or clinical trials;

or may otherwise take

the position that PEDMARK TM fails to meet the requirements and standards for regulatory approval.

There are numerous

FDA personnel assigned to review different aspects of a NDA, and uncertainties can be presented by their ability to exercise judgment

and discretion during the review process.

During the course of review, the FDA may request or require additional preclinical, clinical,

chemistry, manufacturing, and control (“CMC”), or other data and information, and the development and provision of

these data and information may be time consuming and expensive.

Regulatory approvals may not be granted on a timely basis, if at

all, and even if and when they are granted, they may not cover all the indications for which we seek approval.

On February 10,

2020, we submitted an NDA to the FDA for PEDMARK TM for the prevention of ototoxicity associated with cisplatin chemotherapy

in pediatric patients ≥1 month to 18 years of age with localized, non-metastatic, solid tumors.

On April 10, 2020, we received

notification from the FDA that the NDA was accepted for filing and the original application was granted Priority Review with a

PDUFA target action date of August 10, 2020.

On August 10, 2020, we received a CRL from the FDA.

According to the CRL,

after recent completion of a pre-approval inspection of the manufacturing facility of our drug product manufacturer, the FDA identified

deficiencies resulting in a Form 483, which is a list of conditions or practices that are required to be resolved prior to

the approval of PEDMARK™.

We have developed a detailed plan and have dedicated, and continue to commit, significant

resources to addressing the CRL, while, in parallel, working with our third-party drug product manufacturer to be ready for re-inspection

If the FDA determines that these actions were not sufficient, or based on the re-inspection FDA officials do not recommend

approval relative to the drug product manufacturing facility, or if information deemed necessary by the FDA cannot be provided

as part of our NDA submission or during the review period as deemed appropriate on a timely basis, such events could further delay

the progress of our NDA and could require additional actions that we cannot complete during the review period, which may adversely

impact our business.

Further, while

we may develop a product candidate with the intention of addressing a large, unmet medical need, the FDA may only approve the use

of the drug for indications affecting a relatively small number of patients, thus greatly reducing the market size and our potential

The approvals may also contain significant limitations in the form of warnings, precautions or contraindications with

respect to conditions of use, which could further narrow the size of the market.

In certain countries, even if the health regulatory

authorities approve a drug, it cannot be marketed until pricing for the drug is also approved.

Finally, even after approval can

be obtained, we may be required to recall or withdraw a product as a result of newly discovered safety or efficacy concerns, either

of which would have a materially adverse effect on our business and results of operations.

We are currently and may in the future

be the target of securities litigation, which may be costly and time-consuming to defend.

Following periods of market volatility

in the price of a company’s securities or the reporting of unfavorable news, security purchasers have often instituted class

action litigation.

This risk is especially relevant for us because pharmaceutical companies like us have experienced significant

stock price volatility in recent years.

Specifically, we were named in a putative securities class action complaint as a result

of the decline in our stock price following the August 10, 2020 announcement that we had received a CRL from the FDA regarding

our NDA for PEDMARK™.

Regardless of the outcome of this or future litigation, we could incur substantial legal costs and

our management’s attention could be diverted from the operation of our business, causing our business to suffer.

Our insurance

coverage may be insufficient to cover all legal fees, judgments or settlements.

If the outcome of any such litigation is unfavorable,

it could result in us paying significant damages or settlement payments, which could have a material adverse effect on our financial

There are limitations

on the liability of our directors, and we may have to indemnify our officers and directors in certain instances.

Our articles limit,

to the maximum extent permitted under British Columbia law, the personal liability of our directors for monetary damages for breach

of their fiduciary duties as directors.

Our articles provide that we will indemnify our officers and directors and may indemnify

our employees and other agents to the fullest extent permitted by law.

These provisions may be in some respects broader than the

specific indemnification provisions under British Columbia law.

The indemnification provisions may require us, among other things,

to indemnify such officers and directors against certain liabilities that may arise by reason of their status or service as directors

or officers (other than liabilities arising from willful misconduct of a culpable nature), to advance their expenses incurred as

a result of certain proceedings against them as to which they could be indemnified and to obtain directors’

and officers’

We believe that

our limitation of officer and director liability assists us to attract and retain qualified employees and directors.

the event an officer, a director or the board of directors commits an act that may legally be indemnified under British Columbia

law, we will be responsible to pay for such officer(s) or director(s) legal defense and potentially any damages resulting

Furthermore, the limitation on director liability may reduce the likelihood of derivative litigation against directors

and may discourage or deter stockholders from instituting litigation against directors for breach of their fiduciary duties, even

though such an action, if successful, might benefit our stockholders and us.

Given the difficult environment and potential for

incurring liabilities currently facing directors of publicly-held corporations, we believe that director indemnification is in

our and our stockholders’

best interests because it enhances our ability to attract and retain highly qualified directors

and reduce a possible deterrent to entrepreneurial decision-making.

Nevertheless,

limitations of director liability may be viewed as limiting the rights of stockholders, and the broad scope of the indemnification

provisions contained in our certificate of incorporation and bylaws could result in increased expenses.

Our board of directors

believes, however, that these provisions will provide a better balancing of the legal obligations of, and protections for, directors

and will contribute positively to the quality and stability of our corporate governance.

Our board of directors has concluded that

the benefit to stockholders of improved corporate governance outweighs any possible adverse effects on stockholders of reducing

the exposure of directors to liability and broadened indemnification rights.

business and operations could be adversely affected by the effects of health epidemics, including the ongoing COVID-19 pandemic.

operational and financial performance have already been affected by the impact of the COVID-19 pandemic.

The COVID-19 pandemic

is also affecting the operations of government entities, such as the FDA, as well as contract research organizations, third-party

manufacturers, and other third-parties upon whom we rely.

As a result of “shelter-in-place”

orders, quarantines or

similar orders or restrictions to control the spread of COVID-19, many companies, including our own, have implemented work-from-home

policies for their employees.

The effects of these stay at home orders and work-from-home policies may negatively impact productivity,

resulting in delays in our Product launch.

The extent of the impact on our operations depends in part on the time these restrictions

remain in place, and whether restrictions are reinstated as a result of a rising surge in COVID-19 cases.

These and similar disruptions

in our operations could negatively impact our business, operating results and financial condition.

spread of COVID-19 has also led to disruption and volatility in the global capital markets, which increases the cost of, and

adversely impacts access to capital and increases economic uncertainty.

To the extent the COVID-19 pandemic adversely affects

our business, financial results and value of our common stock, it may also affect our ability to access capital and obtain financing,

which could in the future negatively affect our liquidity and ability to continue as a going concern.

global pandemic of COVID-19 continues to evolve rapidly, and the ultimate impact of the COVID-19 pandemic or a similar health epidemic

is highly uncertain and subject to change.

We do not yet know the full impact of potential delays or effects on our business, our

clinical trials, our ability to access the capital markets, or supply chains or on the global economy as a whole.

However, these

effects could have a material impact on our operations, and we will continue to monitor the COVID-19 situation closely.

Natural disasters, epidemic or pandemic

disease outbreaks, trade wars, political unrest or other events could disrupt our business or operations or those of our development

partners, manufacturers, regulators or other third parties with whom we conduct business now or in the future.

A wide variety of events beyond our control,

including natural disasters, epidemic or pandemic disease outbreaks (such as the COVID-19 pandemic), trade wars, political unrest

or other events, could disrupt our business or operations or those of our manufacturers, regulatory authorities, or other third

parties with whom we conduct business.

These events may cause businesses and government agencies to be shut down, supply chains

to be interrupted, slowed, or rendered inoperable, and individuals to become ill, quarantined, or otherwise unable to work and/or

travel due to health reasons or governmental restrictions.

These limitations could negatively affect our business operations and

continuity, and could negatively impact our development timelines and ability to timely perform basic business functions, including,

without limitation, making SEC filings and preparing financial reports.

If our operations or those of third parties with whom we

conduct business are impaired or curtailed as a result of these events, the development and commercialization of our products and

product candidate could be impaired or halted, which could have a material adverse impact on our business.

Risks Related to the Clinical Development and Marketing Approval

of Our Product Candidate

The marketing approval processes of

the FDA and comparable foreign authorities are lengthy, time-consuming and inherently unpredictable, and if we are ultimately unable

to obtain marketing approval for our product candidate, our business will be substantially harmed.

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We cannot commercialize our product candidate in the United

States without first obtaining approval from the FDA to market each product candidate.

States without first obtaining approval from the FDA to market our product candidate.

Similarly, we cannot commercialize our product

1 unchanged sentence

product candidate could fail to receive marketing approval for many reasons, including the following:

the FDA or comparable foreign regulatory

authorities may disagree with the design or implementation of our clinical trials;

the FDA or comparable foreign regulatory

authorities may find the human subject protections for our clinical trials inadequate and place a clinical hold on an Investigational

New Drug Application, or IND, at the time of its submission precluding commencement of any trials or a clinical hold on one or

more clinical trials at any time during the conduct of our clinical trials;

the FDA could determine that we cannot rely

on Section 505(b)(2) of the Federal Food, Drug and Cosmetic Act, or FFDCA, for our product candidate;

we may be unable to demonstrate to the satisfaction

of the FDA or comparable foreign regulatory authorities that a product candidate is safe and effective for its proposed indication;

the results of clinical trials may not meet

the level of statistical significance required by the FDA or comparable foreign regulatory authorities for approval;

we may be unable to demonstrate that a product

candidate’s clinical and other benefits outweigh its safety risks;

the FDA or comparable foreign regulatory

authorities may disagree with our interpretation of data from preclinical studies or clinical trials;

the FDA could determine that our application

relies, or must rely, upon a listed drug or drugs that we a failed to identify or that approval of our 505(b)(2) application for

our product candidate is blocked by patent or non-patent exclusivity of the listed drug or drugs;

the data collected from clinical trials of

our product candidate may not be sufficient to support the submission of an application to obtain marketing approval in the United

States or elsewhere;

the FDA or comparable foreign regulatory

authorities may find inadequate the manufacturing processes or facilities of third-party manufacturers with which we contract for

clinical and commercial supplies;

the approval policies or regulations of the

FDA or comparable foreign regulatory authorities may significantly change in a manner that would delay marketing approval.

Before obtaining marketing approval for the

commercial sale of any drug product for a target indication, we must demonstrate in preclinical studies and well-controlled clinical

trials and, with respect to approval in the United States, to the satisfaction of the FDA, that the product is safe and effective

for its intended use and that the manufacturing facilities, processes, and controls are adequate to preserve the drug’s identity,

strength, quality and purity.

In the United States, it is necessary to submit and obtain approval of a New Drug Application, or

NDA, from the FDA.

An NDA must include extensive preclinical and clinical data and supporting information to establish the product’s

safety and efficacy for each desired indication.

The NDA must also include significant information regarding the chemistry, manufacturing,

and controls for the product.

After the submission of an NDA, but before approval of the NDA, the manufacturing facilities used

to manufacture a product candidate generally must be inspected by the FDA to ensure compliance with the applicable Current Good

Manufacturing Practice, or cGMP, requirements.

The FDA and the Competent Authorities of the Member States of the European Economic

Area, or EEA, and comparable foreign regulatory authorities, may also inspect our clinical trial sites and audit clinical study

data to ensure that our studies are properly conducted in accordance with the IND regulations, human subject protection regulations,

and good clinical practice, or cGCP.

Obtaining approval of an NDA is a

lengthy, expensive and uncertain process, and approval may not be obtained.

Upon submission of an NDA, the FDA must make an

initial determination that the application is sufficiently complete to accept the submission for filing.

We cannot be certain

that any submissions will be accepted for filing and reviewed by the FDA, or ultimately be approved.

If the application is

not accepted for review, the FDA may require that we conduct additional clinical studies or preclinical testing, or take

other actions before it will reconsider our application.

If the FDA requires additional studies or data, we would incur

increased costs and delays in the marketing approval process, which may require us to expend more resources than we have

In addition, the FDA might not consider any additional information to be complete or sufficient to support the

filing or approval of the NDA.

the FDA or comparable foreign regulatory authorities may disagree with the design or implementation

of our clinical trials;

the FDA or comparable foreign regulatory authorities may find the human subject protections for

our clinical trials inadequate and place a clinical hold on an Investigational New Drug Application, or IND, at the time of its

submission precluding commencement of any trials or a clinical hold on one or more clinical trials at any time during the conduct

of our clinical trials;

the FDA could determine that we cannot rely on Section 505(b)(2) of the Federal Food,

Drug and Cosmetic Act, or FFDCA, for our product candidate;

we may be unable to demonstrate to the satisfaction of the FDA or comparable foreign regulatory

authorities that a product candidate is safe and effective for its proposed indication;

the results of clinical trials may not meet the level of statistical significance required by the

FDA or comparable foreign regulatory authorities for approval;

we may be unable to demonstrate that a product candidate’s clinical and other benefits outweigh

its safety risks;

the FDA or comparable foreign regulatory authorities may disagree with our interpretation of data

from preclinical studies or clinical trials;

the FDA could determine that our application relies, or must rely, upon a listed drug or drugs

that we a failed to identify or that approval of our 505(b)(2) application for our product candidate is blocked by patent

or non-patent exclusivity of the listed drug or drugs;

the data collected from clinical trials of our product candidate may not be sufficient to obtain

marketing approval in the United States or elsewhere;

the FDA or comparable foreign regulatory authorities may find inadequate the manufacturing processes

or facilities of third-party manufacturers with which we contract for clinical and commercial supplies (for example, see the discussion

elsewhere concerning the CRL we received from the FDA in August, 2020);

the approval policies or regulations of the FDA or comparable foreign regulatory authorities may

significantly change in a manner that would delay marketing approval.

obtaining marketing approval for the commercial sale of any drug product for a target indication, we must demonstrate in preclinical

studies and well-controlled clinical trials and, with respect to approval in the United States, to the satisfaction of the FDA,

that the product is safe and effective for its intended use and that the manufacturing facilities, processes, and controls are

adequate to preserve the drug’s identity, strength, quality and purity.

In the United States, it is necessary to submit and

obtain approval of an NDA from the FDA.

An NDA must include extensive preclinical and clinical data and supporting information

to establish the product’s safety and efficacy for each desired indication.

The NDA must also include significant information

regarding the chemistry, manufacturing, and controls for the product.

After the submission of an NDA, but before approval of the

NDA, the manufacturing facilities used to manufacture a product candidate generally must be inspected by the FDA to ensure compliance

with the applicable Current Good Manufacturing Practice, or cGMP, requirements (for example, see the discussion elsewhere

concerning the CRL we received from the FDA in August, 2020).

The FDA and the Competent Authorities of the Member States of the

European Economic Area, or EEA, and comparable foreign regulatory authorities, may also inspect our clinical trial sites and audit

clinical study data to ensure that our studies are properly conducted in accordance with the IND regulations, human subject protection

regulations, and good clinical practice, or cGCP.

Obtaining approval of an NDA is a lengthy,

expensive and uncertain process, and approval may not be obtained.

Upon submission of an NDA, the FDA must make an initial determination

that the application is sufficiently complete to accept the submission for filing.

We cannot be certain that any submissions will

be accepted for filing and reviewed by the FDA, or ultimately be approved.

If the application is not accepted for review, the FDA

may require that we conduct additional clinical studies or preclinical testing, or take other actions before it will reconsider

our application.

If the FDA requires additional studies or data, we would incur increased costs and delays in the marketing approval

process, which may require us to expend more resources than we have available.

In addition, the FDA might not consider any additional

information to be complete or sufficient to support the filing or approval of the NDA.

Regulatory authorities outside of the United

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The process to develop, obtain marketing

approval for, and commercialize product candidates is long, complex and costly, both inside and outside of the United States,

and approval is never guaranteed.

approval for, and commercialize product candidates is long, complex and costly, both inside and outside of the United States, and

approval is never guaranteed.

The time required to obtain approval by the FDA and comparable foreign authorities is unpredictable

but typically takes many years following the commencement of clinical trials and depends upon numerous factors, including the

substantial discretion of the regulatory authorities.

In addition, approval policies, regulations, or the type and amount of clinical

data necessary to gain approval may change during the course of a product candidate’s clinical development and may vary

among jurisdictions.

Even if our product candidate were to successfully obtain approval from regulatory authorities, any such

approval might significantly limit the approved indications for use, including more limited patient populations, require that

precautions, warnings or contraindications be included on the product labeling, including black box warnings, require expensive

and time-consuming post-approval clinical studies, risk evaluation and mitigation strategies or surveillance as conditions of

approval, or, through the product label, the approval may limit the claims that we may make, which may impede the successful commercialization

of our product candidate.

Following any approval for commercial sale of our product candidate, certain changes to the product,

such as changes in manufacturing processes and additional labeling claims, as well as new safety information, may require new

studies and will be subject to additional FDA notification, or review and approval.

Also, marketing approval for any of our product

candidate may be withdrawn.

If we are unable to obtain marketing approval for our product candidate in one or more jurisdictions,

or any approval contains significant limitations, our ability to market to our full target market will be reduced and our ability

to realize the full market potential of our product candidate will be impaired.

Furthermore, we may not be able to obtain sufficient

funding or generate sufficient revenue and cash flows to continue or complete the development of any of our current or future

product candidates.

but typically takes many years following the commencement of clinical trials and depends upon numerous factors, including the substantial

discretion of the regulatory authorities.

In addition, approval policies, regulations, or the type and amount of clinical data

necessary to gain approval may change during the course of a product candidate’s clinical development and may vary among

jurisdictions.

Even if our product candidate were to successfully obtain approval from regulatory authorities, any such approval

might significantly limit the approved indications for use, including more limited patient populations, require that precautions,

warnings or contraindications be included on the product labeling, including black box warnings, require expensive and time-consuming

post-approval clinical studies, risk evaluation and mitigation strategies or surveillance as conditions of approval, or, through

the product label, the approval may limit the claims that we may make, which may impede the successful commercialization of our

product candidate.

Following any approval for commercial sale of our product candidate, certain changes to the product, such as

changes in manufacturing processes and additional labeling claims, as well as new safety information, may require new studies and

will be subject to additional FDA notification, or review and approval.

Also, marketing approval for any of our product candidate

may be withdrawn.

If we are unable to obtain marketing approval for our product candidate in one or more jurisdictions, or any

approval contains significant limitations, our ability to market to our full target market will be reduced and our ability to realize

the full market potential of our product candidate will be impaired.

Furthermore, we may not be able to obtain sufficient funding

or generate sufficient revenue and cash flows to continue or complete the development of any of our current or future product candidates.

Our risk of delay in product approvals

is increased if the United States government is fully or partially shut down due to lack of continuity in funding.

Our business operations, and particularly

the timing of the outcome of review of our NDA filing for marketing approval of PEDMARK TM , are directly and indirectly

affected by the operations of the United States government, including but not limited to the FDA.

Any interruption in the continuity

of funding of all or a part of government activities could have a significant negative effect on our business, including the timing

of that review decision.

For example, over the last several years, including beginning on December 22, 2018 and ending on January

25, 2019, the United States government has had shut downs.

We cannot predict the likelihood, duration, impact, or timing of any

future shutdown.

business operations, and particularly the timing of the outcome of review of our NDA filing for marketing approval of PEDMARK TM ,

are directly and indirectly affected by the operations of the United States government, including but not limited to the FDA.

interruption in the continuity of funding of all or a part of government activities could have a significant negative effect on

our business, including the timing of that review decision.

The United States government shuts down from time to time, most recently

from December 22, 2018 to January 25, 2019.

We cannot predict the likelihood, duration, impact, or timing of any future

There can be no assurance that if such shutdown(s) were to occur in the future, adequate funds would be available

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If we are unable to submit an application

for approval under Section 505(b)(2) of the FFDCA or if we are required to generate additional data related to safety and efficacy

in order to obtain approval under Section 505(b)(2), we may be unable to meet our anticipated development and commercialization

for approval under Section 505(b)(2) of the FFDCA or if we are required to generate additional data related to safety

and efficacy in order to obtain approval under Section 505(b)(2), we may be unable to meet our anticipated development and

commercialization timelines.

Our current strategy for seeking marketing

authorization in the United States for our product candidate relies primarily on Section 505(b)(2) of the FFDCA, which permits

use of a marketing application, referred to as a 505(b)(2) application, where at least some of the information required for approval

comes from studies not conducted by or for the applicant and for which the applicant has not obtained a right of reference or use.

The FDA interprets this to mean that an applicant may rely for approval on such data as that found in published literature or the

FDA’s finding of safety or effectiveness, or both, of a previously approved drug product owned by a third party.

no assurance that the FDA would find the published literature or third-party data relied upon by us in a 505(b)(2) application

authorization in the United States for our product candidate relies primarily on Section 505(b)(2) of the FFDCA, which

permits use of a marketing application, referred to as a 505(b)(2) application, where at least some of the information required

for approval comes from studies not conducted by or for the applicant and for which the applicant has not obtained a right of reference

The FDA interprets this to mean that an applicant may rely for approval on such data as that found in published literature

or the FDA’s finding of safety or effectiveness, or both, of a previously approved drug product owned by a third party.

is no assurance that the FDA would find the published literature or third-party data relied upon by us in a 505(b)(2) application

sufficient or adequate to support approval, and the FDA may require us to generate additional data to support the safety and efficacy

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is no assurance that such data generated from such additional activities would be sufficient to obtain approval.

If the data to be relied upon in a

505(b)(2) application are related to drug products previously approved by the FDA and covered by patents that are listed in

the FDA’s Orange Book, we would be required to submit with our 505(b)(2) application an appropriate patent

certification or statement.

The type of patent certification that would enable us to obtain approval of our application

before a listed patent expires, known as a Paragraph IV Certification, would require us to certify that we do not infringe

the listed patent or that such patent is invalid or unenforceable.

We would be required to provide timely notice to the

patent owner and the holder of the approved NDA.

If a patent infringement action is initiated against us within 45 days from

receipt of our Paragraph IV Certification, the approval of our NDA would be subject to a stay of up to 30 months or more

while we defend against such a suit.

Approval of our product candidate under Section 505(b)(2) may, therefore, be delayed

until patent exclusivity expires or until we successfully challenge those patents.

Alternatively, we may elect to generate

sufficient clinical data so that we would no longer need to rely on third-party data, which would be costly and

time-consuming and there would be no assurance that such data generated from such additional activities would be sufficient

to obtain approval.

We may not be able to obtain shortened review

of our applications, and the FDA may not agree that our product candidate qualifies for marketing approval.

If the data to be relied upon in a 505(b)(2) application

are related to drug products previously approved by the FDA and covered by patents that are listed in the FDA’s Orange Book,

we would be required to submit with our 505(b)(2) application an appropriate patent certification or statement.

patent certification that would enable us to obtain approval of our application before a listed patent expires, known as a Paragraph

IV Certification, would require us to certify that we do not infringe the listed patent or that such patent is invalid or unenforceable.

We would be required to provide timely notice to the patent owner and the holder of the approved NDA.

If a patent infringement

action is initiated against us within 45 days from receipt of our Paragraph IV Certification, the approval of our NDA would be

subject to a stay of up to 30 months or more while we defend against such a suit.

Approval of our product candidate under Section 505(b)(2) may,

therefore, be delayed until patent exclusivity expires or until we successfully challenge those patents.

Alternatively, we may

elect to generate sufficient clinical data so that we would no longer need to rely on third-party data, which would be costly and

time-consuming and there would be no assurance that such data generated from such additional activities would be sufficient to

obtain approval.

We may not be able to obtain shortened

review of our applications, and the FDA may not agree that our product candidate qualifies for marketing approval.

If we are required

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approval of our product candidate.

If the FDA changes its interpretation of Section 505(b)(2) allowing reliance on data in published

literature or a previously approved drug application owned by a third party, or there is a change in the law affecting Section

505(b)(2), this could delay or even prevent the FDA from approving any Section 505(b)(2) application that we submit.

Even if we receive marketing approval for

our product candidate, such approved products will be subject to ongoing obligations and continued regulatory review, which may

result in significant additional expense.

Additionally, our product candidate, if approved, could be subject to labeling and other

restrictions, and we may be subject to penalties and legal sanctions if we fail to comply with regulatory requirements or experience

unanticipated problems with our approved products.

If the FDA approves any of our product candidate,

If the FDA changes its interpretation of Section 505(b)(2) allowing reliance on data

in published literature or a previously approved drug application owned by a third party, or there is a change in the law affecting

Section 505(b)(2), this could delay or even prevent the FDA from approving any Section 505(b)(2) application that

Even if we receive marketing approval

for our product candidate, such approved products will be subject to ongoing obligations and continued regulatory review, which

may result in significant additional expense.

Additionally, our product candidate, if approved, could be subject to labeling and

other restrictions, and we may be subject to penalties and legal sanctions if we fail to comply with regulatory requirements or

experience unanticipated problems with our approved products.

If the FDA approves our product candidate,

the manufacturing processes, labeling, packaging, distribution, adverse event reporting, storage, advertising, promotion and recordkeeping

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supporting the product approval, may result in, among other things:

restrictions on the marketing or manufacturing

of the product, withdrawal of the product from the market, or voluntary or mandatory product recalls;

fines, warning letters, or holds on clinical

refusal by the FDA to approve pending applications

or supplements to approved applications filed by us, or suspension or revocation of product approvals;

product seizure or detention, or refusal

to permit the import or export of products;

injunctions or the imposition of civil or

criminal penalties.

The FDA’s policies may change, and additional

government regulations may be enacted that could prevent, limit or delay marketing approval, manufacturing or commercialization

restrictions on the marketing or manufacturing of the product, withdrawal of the product from the

market, or voluntary or mandatory product recalls;

fines, warning letters, or holds on clinical trials;

refusal by the FDA to approve pending applications or supplements to approved applications filed

by us, or suspension or revocation of product approvals;

product seizure or detention, or refusal to permit the import or export of products;

injunctions or the imposition of civil or criminal penalties.

The FDA’s policies may change, and

additional government regulations may be enacted that could prevent, limit or delay marketing approval, manufacturing or commercialization

of our product candidate.

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subject to significant liability.

Regulatory authorities like the FDA and national

competition laws in Europe strictly regulate the promotional claims that may be made about prescription products, such as PEDMARK TM ,

In particular, a product may not be promoted for uses that are not approved by the FDA or comparable foreign regulatory

authorities as reflected in the product’s approved labeling, known as “off-label”

use, nor may it be promoted

prior to obtaining marketing approval.

If we receive marketing approval for our product candidate for our proposed indications,

physicians may nevertheless use our products for their patients in a manner that is inconsistent with the approved label if the

physicians personally believe in their professional medical judgment it could be used in such manner.

Although physicians may prescribe

legally available drugs for off-label uses, manufacturers may not market or promote such off-label uses.

Regulatory authorities like the FDA and

national competition laws in Europe strictly regulate the promotional claims that may be made about prescription products, such

as PEDMARK TM , if approved.

In particular, a product may not be promoted for uses that are not approved by the FDA or

comparable foreign regulatory authorities as reflected in the product’s approved labeling, known as “off-label”

use, nor may it be promoted prior to obtaining marketing approval.

If we receive marketing approval for our product candidate for

our proposed indications, physicians may nevertheless use our products for their patients in a manner that is inconsistent with

the approved label if the physicians personally believe in their professional medical judgment it could be used in such manner.

Although physicians may prescribe legally available drugs for off-label uses, manufacturers may not market or promote such off-label

In addition, the FDA requires that promotional

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The FDA could also seek permanent injunctions under which specified promotional conduct is monitored, changed or curtailed.

Our current and future relationships with

healthcare professionals, investigators, consultants, collaborators, actual customers, potential customers and third-party payors

in the United States and elsewhere may be subject, directly or indirectly, to applicable anti-kickback, fraud and abuse, false

claims, physician payment transparency, health information privacy and security and other healthcare laws and regulations, which

could expose us to sanctions.

Our current and future relationships

with healthcare professionals, investigators, consultants, collaborators, actual customers, potential customers and third-party

payors in the United States and elsewhere may be subject, directly or indirectly, to applicable anti-kickback, fraud and abuse,

false claims, physician payment transparency, health information privacy and security and other healthcare laws and regulations,

which could expose us to sanctions.

Healthcare providers, physicians and third-party

payors in the United States and elsewhere will play a primary role in the recommendation and prescription of any drug candidates

for which we obtain marketing approval.

payors in the United States and elsewhere will play a primary role in the recommendation and prescription of our drug candidates

if it obtains marketing approval.

Our current and future arrangements with healthcare professionals, investigators, consultants,

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The applicable federal, state and foreign healthcare laws that may affect our ability to operate include the following:

the federal Anti-Kickback Statute, which

prohibits, among other things, persons from knowingly and willfully soliciting, offering, receiving or providing remuneration,

directly or indirectly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or

the purchase, lease, order or recommendation of, any good, facility, item or service, for which payment may be made, in whole or

in part, under federal and state healthcare programs such as Medicare and Medicaid;

federal civil and criminal false claims laws

and civil monetary penalty laws, including the federal False Claims Act, which impose criminal and civil penalties, including civil

whistleblower or qui tam actions, against individuals or entities for, among other things, knowingly presenting, or causing to

be presented, to the federal government, including the Medicare and Medicaid programs, claims for payment that are false or fraudulent

or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government;

the civil monetary penalties statute, which

imposes penalties against any person or entity who, among other things, is determined to have presented or caused to be presented

a claim to a federal health program that the person knows or should know is for an item or service that was not provided as claimed

or is false or fraudulent;

the federal Health Insurance Portability

and Accountability Act of 1996, or HIPAA, which created new federal criminal statutes that prohibit knowingly and willfully executing,

or attempting to execute, a scheme to defraud any healthcare benefit program or obtain, by means of false or fraudulent pretenses,

representations or promises, any of the money or property owned by, or under the custody or control of, any healthcare benefit

program, regardless of the payor (e.g., public or private), knowingly and willfully embezzling or stealing from a healthcare benefit

program, willfully obstructing a criminal investigation of a healthcare offense and knowingly and willfully falsifying, concealing

or covering up by any trick or device a material fact or making any materially false statements in connection with the delivery

of, or payment for, healthcare benefits, items or services relating to healthcare matters;

HIPAA, as amended by the Health Information

Technology for Economic and Clinical Health Act of 2009, or HITECH, and its implementing regulations, which impose obligations

on covered entities, including healthcare providers, health plans, and healthcare clearinghouses, as well as their respective business

associates that create, receive, maintain or transmit individually identifiable health information for or on behalf of a covered

entity, with respect to safeguarding the privacy, security and transmission of individually identifiable health information;

the federal Open Payments program, created

under Section 6002 of the Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation

Act, or the Affordable Care Act, and its implementing regulations, which imposed annual reporting requirements for manufacturers

of drugs, devices, biologicals and medical supplies for certain payments and “transfers of value”

provided to physicians

and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate family members, where

failure to submit timely, accurately and completely the required information for all covered payments, transfers of value and ownership

or investment interests may result in civil monetary penalties;

analogous state and foreign laws, such as

state anti-kickback and false claims laws, which may apply to sales or marketing arrangements and claims involving healthcare items

or services reimbursed by non-governmental third-party payors, including private insurers;

state laws that require pharmaceutical

companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance

promulgated by the federal government or otherwise

restrict payments that may be made

to healthcare providers;

state and foreign laws that require drug manufacturers to report information related to payments

and other transfers of value to physicians and other healthcare providers or marketing expenditures;

and state and foreign laws

governing the privacy and security of health information in certain circumstances, many of which differ from each other in significant

ways and often are not preempted by HIPAA, thus complicating compliance efforts.

Further, the Affordable Care Act,

among other things, amended the intent requirement of the federal Anti-Kickback Statute and certain criminal statutes governing

healthcare fraud.

the federal Anti-Kickback Statute, which prohibits, among other things, persons from knowingly

and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce

or reward, or in return for, either the referral of an individual for, or the purchase, lease, order or recommendation of, any

good, facility, item or service, for which payment may be made, in whole or in part, under federal and state healthcare programs

such as Medicare and Medicaid;

federal civil and criminal false claims laws and civil monetary penalty laws, including the federal

False Claims Act, which impose criminal and civil penalties, including civil whistleblower or qui tam actions, against individuals

or entities for, among other things, knowingly presenting, or causing to be presented, to the federal government, including the

Medicare and Medicaid programs, claims for payment that are false or fraudulent or making a false statement to avoid, decrease

or conceal an obligation to pay money to the federal government;

the civil monetary penalties statute, which imposes penalties against any person or entity who,

among other things, is determined to have presented or caused to be presented a claim to a federal health program that the person

knows or should know is for an item or service that was not provided as claimed or is false or fraudulent;

the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which created

new federal criminal statutes that prohibit knowingly and willfully executing, or attempting to execute, a scheme to defraud any

healthcare benefit program or obtain, by means of false or fraudulent pretenses, representations or promises, any of the money

or property owned by, or under the custody or control of, any healthcare benefit program, regardless of the payor (e.g., public

or private), knowingly and willfully embezzling or stealing from a healthcare benefit program, willfully obstructing a criminal

investigation of a healthcare offense and knowingly and willfully falsifying, concealing or covering up by any trick or device

a material fact or making any materially false statements in connection with the delivery of, or payment for, healthcare benefits,

items or services relating to healthcare matters;

HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of

2009, or HITECH, and its implementing regulations, which impose obligations on covered entities, including healthcare providers,

health plans, and healthcare clearinghouses, as well as their respective business associates that create, receive, maintain or

transmit individually identifiable health information for or on behalf of a covered entity, with respect to safeguarding the privacy,

security and transmission of individually identifiable health information;

the federal Open Payments program, created under Section 6002 of the Patient Protection and

Affordable Care Act, as amended by the Health Care and Education Reconciliation Act, or the Affordable Care Act, and its implementing

regulations, which imposed annual reporting requirements for manufacturers of drugs, devices, biologicals and medical supplies

for certain payments and “transfers of value”

provided to physicians and teaching hospitals, as well as ownership and

investment interests held by physicians and their immediate family members, where failure to submit timely, accurately and completely

the required information for all covered payments, transfers of value and ownership or investment interests may result in civil

monetary penalties;

analogous state and foreign laws, such as state anti-kickback and false claims laws, which may

apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party

payors, including private insurers;

state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s

voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government or otherwise restrict

payments that may be made to healthcare providers;

state and foreign laws that require drug manufacturers to report information

related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures;

and foreign laws governing the privacy and security of health information in certain circumstances, many of which differ from each

other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.

Further, the Affordable Care Act, among

other things, amended the intent requirement of the federal Anti-Kickback Statute and certain criminal statutes governing healthcare

A person or entity no longer needs to have actual knowledge of the statute or specific intent to violate it.

In addition, the Affordable Care Act provided that the government may assert that a claim including items or services resulting

from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the False Claims

the Affordable Care Act provided that the government may assert that a claim including items or services resulting from a violation

of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the False Claims Act.

Efforts to ensure that our future business

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programs, which could also affect our business.

The impact of recent healthcare reform

legislation and other changes in the healthcare industry and healthcare spending on us is currently unknown and may adversely affect

our business model.

The impact of recent healthcare reform legislation and other

changes in the healthcare industry and healthcare spending on us is currently unknown and may adversely affect our business model.

In the United States and some foreign jurisdictions,

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for which we obtain marketing approval.

Our revenue prospects could be affected by

changes in healthcare spending and policy in the United States and abroad.

We operate in a highly regulated industry and new laws

and judicial decisions, or new interpretations of existing laws or decisions, related to healthcare availability, the method of

delivery or payment for healthcare products and services could negatively impact our business, financial condition, results of

operations and prospects.

There is significant interest in promoting healthcare reform, as evidenced by the enactment in the United

States of the Affordable Care Act.

Our revenue prospects could be affected

by changes in healthcare spending and policy in the United States and abroad.

We operate in a highly regulated industry and new

laws and judicial decisions, or new interpretations of existing laws or decisions, related to healthcare availability, the method

of delivery or payment for healthcare products and services could negatively impact our business, financial condition, results

of operations and prospects.

There is significant interest in promoting healthcare reform, as evidenced by the enactment in the

United States of the Affordable Care Act.

Among other things, the Affordable Care Act contains provisions that may reduce the profitability

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and subjecting drug manufacturers to payment of an annual fee.

We expect that the Affordable Care Act, as

well as other healthcare reform measures that may be adopted in the future, may result in more rigorous coverage criteria and in

additional downward pressure on the price that we receive for any approved product.

Any reduction in reimbursement from Medicare

or other government programs may result in a similar reduction in payments from private payors.

The implementation of cost containment

measures or other healthcare reforms may prevent us from being able to generate revenue or commercialize our drugs.

We expect that the Affordable Care Act,

as well as other healthcare reform measures that may be adopted in the future, may result in more rigorous coverage criteria and

in additional downward pressure on the price that we receive for our product if it is approved for marketing.

Any reduction in

reimbursement from Medicare or other government programs may result in a similar reduction in payments from private payors.

implementation of cost containment measures or other healthcare reforms may prevent us from being able to generate revenue or commercialize

It is likely that federal and state legislatures

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services to contain or reduce costs of healthcare may adversely affect:

the demand for any drug products for which

we may obtain marketing approval;

our ability to set a price that we believe

is fair for our products;

our ability to obtain coverage and reimbursement

approval for a product;

our ability to generate revenues and achieve

or maintain profitability;

the level of taxes that we are required to

the demand for our product;

our ability to set a price that we believe is fair for our product;

our ability to obtain coverage and reimbursement approval for our product;

our ability to generate revenues and achieve or maintain profitability;

the level of taxes that we are required to pay.

If we fail to comply with environmental,

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and suppliers’

activities involve the controlled storage, use, and disposal of

hazardous materials, including the components of our product candidate and other hazardous compounds.

manufacturers and suppliers are subject to laws and regulations governing the use, manufacture, storage, handling, and

disposal of these hazardous materials.

In some cases, these hazardous materials and various wastes resulting from their use

are stored at our and our manufacturers’

activities involve the controlled storage, use, and disposal of hazardous

materials, including the components of our product candidate and other hazardous compounds.

We and our manufacturers and suppliers

are subject to laws and regulations governing the use, manufacture, storage, handling, and disposal of these hazardous materials.

In some cases, these hazardous materials and various wastes resulting from their use are stored at our and our manufacturers’

facilities pending their use and disposal.

We cannot eliminate the risk of

contamination, which could cause an interruption of our commercialization efforts, research and development efforts and

business operations, environmental damage resulting in costly clean-up and liabilities under applicable laws and regulations

governing the use, storage, handling, and disposal of these materials and specified waste products.

Although we believe that

the safety procedures utilized by us and our third-party manufacturers for handling and disposing of these materials

generally comply with the standards prescribed by these laws and regulations, we cannot guarantee that this is the case or

eliminate the risk of accidental contamination or injury from these materials.

In such an event, we may be held liable for

any resulting damages and such liability could exceed our resources and state or federal or other applicable authorities may

curtail our use of specified materials and/or interrupt our business operations.

Furthermore, environmental laws and

regulations are complex, change frequently, and have tended to become more stringent.

We cannot eliminate the risk of contamination, which could cause an interruption of

our commercialization efforts, research and development efforts and business operations, environmental damage resulting in costly

clean-up and liabilities under applicable laws and regulations governing the use, storage, handling, and disposal of these materials

and specified waste products.

Although we believe that the safety procedures utilized by us and our third-party manufacturers for

handling and disposing of these materials generally comply with the standards prescribed by these laws and regulations, we cannot

guarantee that this is the case or eliminate the risk of accidental contamination or injury from these materials.

In such an event,

we may be held liable for any resulting damages and such liability could exceed our resources and state or federal or other applicable

authorities may curtail our use of specified materials and/or interrupt our business operations.

Furthermore, environmental laws

and regulations are complex, change frequently, and have tended to become more stringent.

We cannot predict the impact of such

changes and cannot be certain of our future compliance.

We do not currently carry biological or hazardous waste insurance

Risks Related to Commercialization of Our

Product Candidate

We do not currently carry biological or hazardous waste insurance coverage.

Risks Related to Commercialization of

Our Product Candidate

Even if we obtain the required regulatory

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and acceptance of our product candidates.

Even if we obtain marketing approval for PEDMARK TM

or any other product candidate that we may develop or acquire in the future, the products may not gain market acceptance among

physicians, key opinion leaders, healthcare payors, patients and the medical community.

Market acceptance of any approved products

depends on a number of factors, including:

Even if we obtain marketing approval for

PEDMARK TM , it may not gain market acceptance among physicians, key opinion leaders, healthcare payors, patients and

the medical community.

Market acceptance of PEDMARK TM depends on a number of factors, including:

the timing of market introduction;

the efficacy and safety of the product, as

demonstrated in clinical trials;

the clinical indications for which the product

is approved, and the label approved by regulatory authorities for use with the product, including any precautions, warnings or

contraindications that may be required on the label;

acceptance by physicians, key opinion leaders

and patients of the product as a safe and effective treatment;

the cost, safety and efficacy of treatment

in relation to alternative treatments;

the availability of coverage and adequate

reimbursement and pricing by third-party payors and government authorities;

the number and clinical profile of competing

the growth of drug markets in our various

its efficacy and safety, as demonstrated in clinical trials;

the clinical indications for which it is approved, and the label approved by regulatory authorities

for use with the product, including any precautions, warnings or contraindications that may be required on the label;

acceptance by physicians, key opinion leaders and patients of PEDMARK TM as a safe and

effective treatment;

the cost, safety and efficacy of treatment in relation to alternative treatments;

the availability of coverage and adequate reimbursement and pricing by third-party payors and government

the number and clinical profile of competing products;

the growth of drug markets in our various indications;

relative convenience and ease of administration;

marketing and distribution support;

the prevalence and severity of adverse side

the effectiveness of our sales and marketing

the prevalence and severity of adverse side effects;

the effectiveness of our sales and marketing efforts.

Market acceptance is critical to our ability

to generate revenue.

Any product candidate, if approved and commercialized, may be accepted in only limited capacities or not at

If any approved products are not accepted by the market to the extent that we expect, we may not be able to generate revenue

and our business would suffer.

If the market opportunities for our product

candidates are smaller than we believe they are, then our revenues may be adversely affected, and our business may suffer.

The market opportunities that our current

and future product candidates are being developed to address are rare.

Our projections of both the number of people who are administered

Cisplatin, as well as the subset of people who have the potential to benefit from treatment with our product candidates, and our

assumptions relating to pricing are based on estimates.

Given the small number of patients that we are targeting, our eligible

patient population and pricing estimates may differ significantly from the actual market addressable by our product candidates.

PEDMARK TM , if approved and commercialized, may be accepted in only limited capacities or not at

If PEDMARKTM is not accepted by the market to the extent that we expect, we may not be able to generate revenue and our business

would suffer.

If the market opportunities for our

product candidate are smaller than we believe they are, then our revenues may be adversely affected, and our business may suffer.

The market opportunities that our product

candidate is being developed to address are rare.

Our projections of both the number of people who are administered Cisplatin,

as well as the subset of people who have the potential to benefit from treatment with our product candidate, and our assumptions

relating to pricing are based on estimates.

Given the small number of patients that we are targeting, our eligible patient population

and pricing estimates may differ significantly from the actual market addressable by our product candidate.

Coverage and reimbursement may be limited

or unavailable in certain market segments for our product candidate, which could make it difficult for us to sell our products

There is significant uncertainty related to

third-party coverage and reimbursement of newly approved pharmaceuticals.

Market acceptance and sales of our product candidate,

should it receive marketing approval, will depend significantly on the availability of coverage and adequate reimbursement from

third-party payors and may be affected by existing and future healthcare reform measures.

Patients who are prescribed treatments

for their conditions and providers performing the prescribed services generally rely on third-party payors to reimburse all or

part of the associated healthcare costs.

Government authorities and third-party payors, such as private health insurers, health

maintenance organizations, and government payors like Medicare and Medicaid, decide which drugs they will pay for and establish

reimbursement levels.

Increasingly, third-party payors are requiring that drug companies provide them with predetermined discounts

from list prices and are challenging the prices charged for drugs and products.

Coverage and reimbursement may not be available

for any product that we commercialize and, even if coverage is provided, the level of reimbursement may not be satisfactory.

reimbursement levels may adversely affect the demand for, or the price of, any drug candidate for which we obtain marketing approval.

Reimbursement by a third-party payor may depend

upon a number of factors, including the third-party payor’s determination that use of a product is, among other things:

There is significant uncertainty

related to third-party coverage and reimbursement of newly approved pharmaceuticals.

Market acceptance and sales of our

product candidate, should it receive marketing approval, will depend significantly on the availability of coverage and

adequate reimbursement from third-party payors and may be affected by existing and future healthcare reform measures.

Patients who are prescribed treatments for their conditions and providers performing the prescribed services generally rely

on third-party payors to reimburse all or part of the associated healthcare costs.

Government authorities and third-party

payors, such as private health insurers, health maintenance organizations, and government payors like Medicare and Medicaid,

decide which drugs they will pay for and establish reimbursement levels.

Increasingly, third-party payors are requiring that

drug companies provide them with predetermined discounts from list prices and are challenging the prices charged for drugs

and products.

Coverage and reimbursement may not be available for any product that we commercialize and, even if coverage is

provided, the level of reimbursement may not be satisfactory.

Inadequate reimbursement levels may adversely affect the demand

for, or the price of, our drug candidate if it obtains marketing approval.

Reimbursement by a third-party payor may

depend upon a number of factors, including the third-party payor’s determination that use of a product is, among other things:

a covered benefit under its health plan;

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to conduct expensive pharmacoeconomic studies and provide supporting scientific, clinical and cost-effectiveness data for the use

of our products to the payor.

of our product to the payor.

We may not be able to provide data sufficient to gain acceptance with respect to coverage and adequate

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We cannot be sure that coverage or adequate

reimbursement will be available for any of our product candidate.

Also, we cannot be sure that reimbursement amounts will not reduce

the demand for, or the price of, our products.

If reimbursement is not available or is available only to limited levels, we may

not be able to commercialize certain of our products.

reimbursement will be available for our product candidate.

Also, we cannot be sure that reimbursement amounts will not reduce the

demand for, or the price of, our product.

If reimbursement is not available or is available only to limited levels, we may not

be able to commercialize certain of our products.

In the United States, third-party payors are increasingly attempting to contain

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the pressure on drug pricing.

If third-party payors do not consider our products to be cost-effective compared to other available

therapies, they may not cover the products for which we receive FDA approval or, if they do, the level of payment may not be sufficient

to allow us to sell our products at a profit.

If third-party payors do not consider our product to be cost-effective compared to other available

therapies, they may not cover our product or, if they do, the level of payment may not be sufficient to allow us to sell our product

Coverage policies, third-party reimbursement

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in the foreseeable future.

Even if favorable coverage and reimbursement status is attained for one or more products for which we

receive marketing approval, less favorable coverage policies and reimbursement rates may be implemented in the future.

We face substantial competition, which

may result in others discovering, developing or commercializing products before, or more successfully, than we do.

Even if favorable coverage and reimbursement status is attained for our product, less favorable coverage

policies and reimbursement rates may be implemented in the future.

We face substantial competition,

which may result in others discovering, developing or commercializing products before, or more successfully, than we do.

The life sciences industry is highly competitive,

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and maintain a competitive advantage with respect to the design, development and commercialization of our product candidate for

the treatment of orphan and ultra-orphan diseases for which there is a small patient population in the United States.

A drug designated

an Orphan Drug may receive up to seven years of exclusive marketing in the United States for that indication.

Many of our potential competitors have significantly

greater financial, manufacturing, marketing, development, technical and human resources than we do.

Large pharmaceutical and biotechnology

companies, in particular, have extensive experience in clinical testing, obtaining regulatory approvals, recruiting patients and

in manufacturing clinical products.

These companies also have significantly greater research and marketing capabilities than we

do and may also have products that have been approved or are in late stages of development, and have collaborative arrangements

the treatment of orphan and ultra-orphan diseases for which there is a small patient population in both the United States and in

all other potential markets.

A drug designated an orphan drug may receive up to seven years of exclusive marketing in the United

States for that indication.

Many of our potential competitors have

significantly greater financial, manufacturing, marketing, development, technical and human resources than we do.

Large pharmaceutical

and biotechnology companies, in particular, have extensive experience in clinical testing, obtaining regulatory approvals, recruiting

patients and in manufacturing clinical products.

These companies also have significantly greater research and marketing capabilities

than we do and may also have products that have been approved or are in late stages of development, and have collaborative arrangements

in our target markets with leading companies and research institutions.

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orphan drug designation for our product candidate is essential to our viability since our competitors may, among other things:

have greater name and brand recognition,

financial and human resources;

develop and commercialize products that are

safer, more effective, less expensive, or more convenient or easier to administer;

have greater name and brand recognition, financial and human resources;

develop and commercialize products that are safer, more effective, less expensive, or more convenient

or easier to administer;

obtain quicker marketing approval;

establish superior proprietary positions;

have access to more manufacturing capacity

as well as to more cost-effective manufacturing capacity;

implement more effective approaches to sales

and marketing;

have access to more manufacturing capacity as well as to more cost-effective manufacturing capacity;

implement more effective approaches to sales and marketing;

form more advantageous strategic alliances.

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We believe that our ability to successfully

compete will depend on our ability to obtain Orphan Drug designation as well as:

our ability to design and successfully execute

appropriate clinical trials;

our ability to recruit and enroll patients

for our clinical trials;

the results of our clinical trials and the

efficacy and safety of our product candidate;

the speed at which we develop our product

achieving and maintaining compliance with

regulatory requirements applicable to our business;

the timing and scope of regulatory approvals,

including labeling;

adequate levels of reimbursement under private

and governmental health insurance plans, including Medicare and Medicaid;

our ability to protect intellectual property

rights related to our product candidate;

our ability to commercialize and market any

of our product candidate if it obtains marketing approval;

our ability to manufacture and sell commercial

quantities of any approved our product candidate;

acceptance of our product candidate by physicians,

other healthcare providers and patients;

the cost of treatment in relation to alternative

If our competitors are able to obtain orphan

drug exclusivity for their products that are for the same indication as our product candidate, we may not be able to have competing

products approved by the applicable regulatory authority for a significant period of time or benefit from that exclusivity.

We have orphan drug designation in the United

States for PEDMARK TM for the prevention of platinum induced ototoxicity in pediatric patients.

Generally, if a product with an orphan drug

designation subsequently receives the first marketing approval for the indication for which it has such designation, that product

is entitled to a period of marketing exclusivity, which precludes the applicable regulatory authority from approving another marketing

application for the same drug for the same indication for that time period.

The applicable period is seven and a half years in

the United States.

Maintaining orphan drug designation for PEDMARK TM may be important to its success.

orphan drug designation, we may not be able to maintain it.

For example, if a competitive product that treats the same disease

as our product candidate is shown to be clinically superior to our product candidate, any orphan drug designation we have obtained

will not block the approval of such competitive product and we may effectively lose what had previously been orphan drug designation.

Orphan drug designation for PEDMARK TM also will not bar the FDA from approving another STS drug product for another

In the United States, reforms to the Orphan Drug Act, if enacted, could also materially affect our ability to maintain

orphan drug designation for PEDMARK TM for cisplatin induced ototoxicity in pediatric cancer.

compete will depend on our ability to maintain orphan drug designation as well as:

our ability to design and successfully execute appropriate clinical trials;

our ability to recruit and enroll patients for our clinical trials;

the results of our clinical trials and the efficacy and safety of our product candidate;

the speed at which we develop our product candidate;

achieving and maintaining compliance with regulatory requirements applicable to our business;

the timing and scope of regulatory approvals, including labeling;

adequate levels of reimbursement under private and governmental health insurance plans, including

Medicare and Medicaid;

our ability to protect intellectual property rights related to our product candidate;

our ability to commercialize and market our product candidate if it obtains marketing approval;

our ability to manufacture and sell commercial quantities of any approved our product candidate;

acceptance of our product candidate by physicians, other healthcare providers and patients;

the cost of treatment in relation to alternative therapies.

If our competitors are able to obtain

orphan drug exclusivity for their products that are for the same indication as our product candidate, we may not be able to have

our product approved by the applicable regulatory authority for a significant period of time or benefit from that exclusivity.

have orphan drug designation in the United States for PEDMARK TM for

the prevention of platinum induced ototoxicity in pediatric patients.

if a product with an orphan drug designation subsequently receives the first marketing approval for the indication for which it

has such designation, that product is entitled to a period of marketing exclusivity, which precludes the applicable regulatory

authority from approving another marketing application for the same drug for the same indication for that time period.

The applicable

period is seven and a half years in the United States.

Maintaining orphan drug designation for PEDMARK TM may

be important to its success.

Even with orphan drug designation, we may not be able to maintain it.

For example, if a competitive

product that treats the same disease as our product candidate is shown to be clinically superior to our product candidate, any

orphan drug designation we have obtained will not block the approval of such competitive product and we may effectively lose what

had previously been orphan drug designation.

Orphan drug designation for PEDMARK TM also

will not bar the FDA from approving another STS drug product for another indication.

In the United States, reforms to the Orphan

Drug Act, if enacted, could also materially affect our ability to maintain orphan drug designation for PEDMARK TM for

cisplatin induced ototoxicity in pediatric cancer.

Price controls may be imposed in foreign

markets, which may adversely affect our future profitability.

In some countries, particularly member states

of the European Union, the pricing of prescription drugs is subject to governmental control.

In these countries, pricing negotiations

with governmental authorities can take considerable time after receipt of marketing approval for a product.

In addition, there

can be considerable pressure by governments and other stakeholders on prices and reimbursement levels, including as part of cost

containment measures.

Political, economic and regulatory developments may further complicate pricing negotiations, and pricing

negotiations may continue after reimbursement has been obtained.

Reference pricing used by various European Union member states

and parallel distribution, or arbitrage between low-priced and high-priced member states, can further reduce prices.

In some countries,

we may be required to conduct a clinical trial or other studies that compare the cost-effectiveness of our product candidate to

other available therapies in order to obtain or maintain reimbursement or pricing approval.

Publication of discounts by third-party

payors or authorities may lead to further pressure on the prices or reimbursement levels within the country of publication and

other countries.

In some countries, particularly member

states of the European Union, the pricing of prescription drugs is subject to governmental control.

In these countries, pricing

negotiations with governmental authorities can take considerable time after receipt of marketing approval for a product.

there can be considerable pressure by governments and other stakeholders on prices and reimbursement levels, including as part

of cost containment measures.

Political, economic and regulatory developments may further complicate pricing negotiations, and

pricing negotiations may continue after reimbursement has been obtained.

Reference pricing used by various European Union member

states and parallel distribution, or arbitrage between low-priced and high-priced member states, can further reduce prices.

some countries, we may be required to conduct a clinical trial or other studies that compare the cost-effectiveness of our product

candidate to other available therapies in order to obtain or maintain reimbursement or pricing approval.

Publication of discounts

by third-party payors or authorities may lead to further pressure on the prices or reimbursement levels within the country of publication

and other countries.

If reimbursement of our products is unavailable or limited in scope or amount, or if pricing is set at unsatisfactory

levels, our business could be adversely affected.

Rapid technological change could make

our products obsolete.

Rapid technological change could

make our products obsolete.

Pharmaceutical technologies have undergone

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If our product candidate is rendered obsolete by advancements in pharmaceutical technologies, our prospects

Government controls and healthcare reform

measures could adversely affect our business.

The business and financial condition of pharmaceutical

and biotechnology companies are affected by the efforts of governmental and third-party payors to contain or reduce the costs of

In the United States and in foreign jurisdictions, there have been, and we expect that there will continue to be, a

number of legislative and regulatory proposals aimed at changing the healthcare system.

For example, in some foreign countries,

particularly in Europe, the pricing of prescription pharmaceuticals is subject to governmental control.

In these countries, pricing

negotiations with governmental authorities can take considerable time after the receipt of marketing approval for a product candidate.

To obtain reimbursement or pricing approval in some countries, we may be required to conduct additional clinical trials that compare

the cost-effectiveness of any product candidate to other available therapies.

If reimbursement of any product candidate is unavailable

or limited in scope or amount in a particular country, or if pricing is set at unsatisfactory levels, we may be unable to achieve

or sustain profitability in such country.

In the United States, the Medicare Prescription Drug, Improvement, and Modernization

Act of 2003, or MMA, changed the way Medicare covers and pays for pharmaceutical products.

The legislation established Medicare

Part D, which expanded Medicare coverage for outpatient prescription drug purchases by the elderly but provided authority for limiting

the number of drugs that will be covered in any therapeutic class.

The MMA also introduced a new reimbursement methodology based

on average sales prices for physician-administered drugs.

Any negotiated prices for our product candidate under a Part D prescription

drug plan will likely be lower than the prices that might otherwise be obtained outside of the Medicare Part D prescription drug

Moreover, while Medicare Part D applies only to drug benefits for Medicare beneficiaries, private payors often follow Medicare

coverage policy and payment limitations in setting their own payment rates.

Any reduction in payment under Medicare Part D may

result in a similar reduction in payments from non-governmental payors.

The United States and several other jurisdictions

are considering, or have already enacted, a number of legislative and regulatory proposals to change the healthcare system in ways

that could affect our ability to sell any product candidate.

Among policy-makers and payors in the United States and elsewhere,

there is significant interest in promoting changes in healthcare systems with the stated goals of containing healthcare costs,

improving quality and/or expanding access to healthcare.

In the United States, the pharmaceutical industry has been a particular

focus of these efforts and has been significantly affected by major legislative initiatives.

There have been, and likely will continue

to be, legislative and regulatory proposals at the federal and state levels directed at broadening the availability of healthcare

and containing or lowering the cost of healthcare.

We cannot predict the initiatives that may be adopted in the future.

The continuing

efforts of the government, insurance companies, managed care organizations and other payors of healthcare services to contain or

reduce costs of healthcare may adversely affect:

the demand for any product candidate;

the ability to set a price that we believe

is fair for any product candidate;

our ability to generate revenues and achieve or maintain profitability;

the level of taxes that

we are required to pay;

and the availability of capital.

limited experience as a company in marketing or distributing pharmaceutical products.

If we are unable to expand our marketing

capabilities and effectively commercialize PEDMARK TM , our business, results of operations and financial condition may

be materially adversely affected.

Our strategy is

to build our sales, marketing and distribution capabilities to successfully commercialize PEDMARK TM in the United States

and evaluate commercial opportunities globally for PEDMARK TM .

While we have begun to establish our commercial team,

we have limited experience commercializing pharmaceutical products as an organization.

In order to successfully market PEDMARK TM ,

we must continue to build our sales, marketing, managerial, compliance, and related capabilities or make arrangements with third

parties to perform these services.

If we are unable to establish adequate sales, marketing, and distribution capabilities, whether

independently or with third parties, we may not be able to appropriately commercialize PEDMARK TM and may not become

Included in our

strategy in the United States is to have a sales force to commercialize PEDMARK TM , subject to it receiving marketing

These efforts will continue to be expensive and time-consuming, and we cannot be certain that we will be able to successfully

develop this capability.

We will need to train our sales force to ensure that a consistent and appropriate message about PEDMARK TM

is being delivered to our potential customers.

If we are unable to effectively train our sales force and equip them with effective

materials, including medical and sales literature to help them inform and educate potential customers about the benefits of PEDMARK TM

and its proper administration, all while maintaining compliance with regulatory requirements, our efforts to successfully commercialize

PEDMARK TM could be harmed, which would negatively impact our ability to generate product revenue.

Additionally, we will

need to maintain and further develop our sales force to achieve commercial success, and we will be competing with other pharmaceutical

and biotechnology companies to recruit, hire, train and retain marketing and sales personnel.

In the event we are unable to continue

to develop and effectively maintain our commercial team, our ability to successfully commercialize PEDMARK TM would be

limited, and we would not be able to generate product revenue successfully.

There are risks

involved both with establishing our own sales and marketing capabilities, and with entering into arrangements with third parties

to perform these services.

For example, any efforts to develop a direct sales and marketing organization are subject to numerous

risks, including:

the expense and time required to recruit,

retain, and motivate members of the sales force;

our inability to recruit, retain or motivate

adequate numbers of effective marketing personnel and partner marketing agencies;

the inability to provide adequate training

to sales and marketing personnel;

the expense and time required to monitor

regulatory compliance;

the inability of sales personnel to obtain

access to physicians or convince adequate numbers of physicians to prescribe any product;

unforeseen costs and expenses associated

with creating an independent sales and marketing organization.

Similarly, if

we enter into arrangements with third parties to perform sales, marketing and distribution services, our product revenue or the

profitability associated with any product revenue may be lower than if we were to market and sell any products that we develop

In addition, we may not be successful in entering into arrangements with third parties to sell and market our products

or may be unable to do so on terms that are favorable to us.

We may have little control over such third parties, and any of them

may fail to devote the necessary resources and attention to sell and market our products effectively.

Moreover, we may be negatively

impacted by other factors outside of our control relating to such third parties, including, but not limited to, their inability

to comply with regulatory requirements.

If we do not establish sales, marketing and distribution capabilities successfully, either

on our own or in collaboration with third parties, we will not be successful in commercializing our products.

Finally, because

we are using a very small group of exclusive specialty pharmacies to distribute our product, if the organizations that we work

with to deliver our drug do not perform in a lawful manner or have issues unrelated to our business, our business could be adversely

a risk of product liability claims and may not be able to obtain adequate insurance.

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We currently carry liability insurance that we believe to be adequate.

Our insurance may not reimburse us for certain claims or the coverage may not be sufficient to cover claims made against us.

cannot predict all of the possible harms or side effects that may result from the use of our current drug candidates, or any potential

future products we may acquire and use in clinical trials or after FDA approval and, therefore, the amount of insurance coverage

we currently hold may not be adequate to cover all liabilities we might incur.

If we are sued for any injury allegedly caused by

our products, our liability could exceed our ability to pay the liability.

Whether or not we are ultimately successful in any adverse

litigation, such litigation could consume substantial amounts of our financial and managerial resources, all of which could have

a material adverse effect on our business, financial condition, results of operations, prospects and stock price.

However, our insurance may not reimburse us for certain claims or the coverage may not be sufficient to cover claims made against

We cannot predict all of the possible harms or side effects that may result from the use of our drug candidate and, therefore,

the amount of insurance coverage we currently hold may not be adequate to cover all liabilities we might incur.

If we are sued

for any injury allegedly caused by our products, our liability could exceed our ability to pay the liability.

Whether or not we

are ultimately successful in any adverse litigation, such litigation could consume substantial amounts of our financial and managerial

resources, all of which could have a material adverse effect on our business, financial condition, results of operations, prospects

and stock price.

Risks Related to Third Parties

We rely on third-party suppliers and

other third parties for production of our product candidate and our dependence on these third parties may impair the advancement

of our research and development programs and the development of our product candidate.

We rely on third-party suppliers and other third parties

for production of our product candidate and our dependence on these third parties may impair the advancement of our research and

development programs and the development of our product candidate.

We do not currently own or operate manufacturing

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We lack the resources and the capability to manufacture

any of our product candidate on a clinical or commercial scale.

our product candidate on a clinical or commercial scale.

Instead, we rely on, and expect to continue to rely on, third parties

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our competitors, which would adversely affect our ability to access the formulations we require.

The facilities used by our current

contract manufacturers and any future manufacturers to manufacture our product candidate must be inspected by the FDA during

the review of our NDA.

We do not control the manufacturing process of, and are completely dependent on, our contract

manufacturers for compliance with the regulatory requirements, known as cGMPs, for manufacture of both active drug substances

and finished drug products.

If our contract manufacturers cannot successfully manufacture material that conforms to our

specifications and the strict regulatory requirements of the FDA or others, the FDA may refuse to approve our NDA.

or a comparable foreign regulatory authority does not approve our NDA because of concerns about the manufacture of our

product candidate or if significant manufacturing issues arise in the future, we may need to find alternative manufacturing

facilities, which would significantly impact our ability to develop our product candidate, to obtain marketing approval of

our NDA or to continue to market our product candidate, if approved.

Although we are ultimately responsible for ensuring

compliance with these regulatory requirements, we do not have day-to-day control over a contract manufacturing organization

(“CMO”) or other third-party manufacturer’s compliance with applicable laws and regulations, including

cGMPs and other laws and regulations, such as those related to environmental health and safety matters.

Any failure to

achieve and maintain compliance with these laws, regulations and standards could subject us to the risk that we may have to

suspend the manufacturing of our product candidate or that obtained approvals could be revoked, which would adversely affect

our business and reputation.

In addition, third-party contractors, such as our CMOs, may elect not to continue to work with

us due to factors beyond our control.

Although we have contracts in place, they may also refuse to work with us because

of their own financial difficulties, business priorities or other reasons, at a time that is costly or otherwise inconvenient

If we were unable to find adequate replacement or another acceptable solution in time, our clinical trials could

be delayed or our commercial activities could be harmed.

Problems with the quality of the work of third

parties may lead us to seek to terminate our working relationships and use alternative service providers.

However, making this

change may be costly and may delay clinical trials.

In addition, it may be very challenging, and in some cases impossible, to find

replacement service providers that can develop and manufacture our drug candidates in an acceptable manner and at an acceptable

facilities used by our current contract manufacturers and any future manufacturers to manufacture our product candidate must be

inspected by the FDA during the review of our NDA.

We do not control the manufacturing process of, and are completely dependent

on, our contract manufacturers for compliance with the regulatory requirements, known as cGMPs, for manufacture of both active

drug substances and finished drug products.

If our contract manufacturers cannot successfully manufacture material that conforms

to our specifications and the strict regulatory requirements of the FDA or others, the FDA may refuse to approve our NDA.

CRL that we received from the FDA in August 2020, as a result of deficiencies in the third-party manufacturing facility that

manufactures PEDMARK TM on our behalf is a specific example of the risks associated with our third-party manufacturers.

If the FDA or a comparable foreign regulatory authority does not approve our NDA because of concerns about the manufacture of our

product candidate or if significant manufacturing issues arise in the future, we may need to find alternative manufacturing facilities,

which would significantly impact our ability to develop our product candidate, to obtain marketing approval of our NDA or to continue

to market our product candidate, if approved.

Although we are ultimately responsible for ensuring compliance with these regulatory

requirements, we do not have day-to-day control over a contract manufacturing organization (“CMO”) or other third-party

manufacturer’s compliance with applicable laws and regulations, including cGMPs and other laws and regulations, such as those

related to environmental health and safety matters.

Any failure to achieve and maintain compliance with these laws, regulations

and standards could subject us to the risk that we may have to suspend the manufacturing of our product candidate or that obtained

approvals could be revoked, which would adversely affect our business and reputation.

In addition, third-party contractors, such

as our CMOs, may elect not to continue to work with us due to factors beyond our control.

Although we have contracts in place,

they may also refuse to work with us because of their own financial difficulties, business priorities or other reasons, at a time

that is costly or otherwise inconvenient for us.

If we were unable to find adequate replacement or another acceptable solution in

time, our clinical trials could be delayed or our commercial activities could be harmed.

On August 10, 2020, we received a CRL.

to the CRL, after recent completion of a pre-approval inspection of the manufacturing facility of our drug product manufacturer, the FDA

identified deficiencies resulting in a Form 483, which is a list of conditions or practices that are required to be resolved prior

to the approval of PEDMARK™.

The Company has developed a detailed plan and has dedicated, and continues to commit, significant

resources to addressing the CRL, while, in parallel, working with our third-party drug product manufacturer to be ready for re-inspection

If the FDA determines that these actions were not sufficient, or based on the re-inspection FDA officials do not recommend

approval relative to the drug product manufacturing facility, or if information deemed necessary by the FDA cannot be provided as part

of our NDA submission or during the review period as deemed appropriate on a timely basis, such events could further delay the progress

of our NDA and could require additional Company actions that cannot be completed during the review period which may adversely impact our

Problems with the quality of the work of

third parties may lead us to seek to terminate our working relationships and use alternative service providers.

However, making

this change may be costly and may delay clinical trials.

In addition, it may be very challenging, and in some cases impossible,

to find replacement service providers that can develop and manufacture our drug candidates in an acceptable manner and at an acceptable

cost and on a timely basis.

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reasonable timeframe and at an acceptable cost or at all.

We plan to rely on third parties to

conduct clinical trials for our product candidate.

We plan to rely on third parties

to conduct clinical trials for our product candidate.

If these third parties do not successfully carry out their contractual duties

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be unable to obtain marketing approval for or commercialize our product candidate.

Clinical trials must meet applicable FDA and

foreign regulatory requirements.

Clinical trials must meet applicable FDA

and foreign regulatory requirements.

We do not have the ability to independently conduct clinical trials for our product candidate.

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negative effect on our ability to obtain marketing approval, our business, and our financial condition.

We or the third parties we rely on may encounter

problems in clinical trials that may cause us or the FDA or foreign regulatory agencies to delay, suspend or terminate our clinical

trials at any phase.

These problems could include the possibility that we may not be able to manufacture sufficient quantities

of materials for use in our clinical trials, conduct clinical trials at our preferred sites, enroll a sufficient number of patients

for our clinical trials at one or more sites, or begin or successfully complete clinical trials in a timely fashion, if at all.

Furthermore, we, the FDA or foreign regulatory agencies may suspend clinical trials of our product candidate at any time if we

or they believe the subjects participating in the trials are being exposed to unacceptable health risks, whether as a result of

adverse events occurring in our trials or otherwise, or if we or they find deficiencies in the clinical trial process or conduct

of the investigation.

The FDA and foreign regulatory agencies could

also require additional clinical trials before or after granting of marketing approval for any products, which would result in

increased costs and significant delays in the development and commercialization of such products and could result in the withdrawal

We or the third parties we rely on may

encounter problems in clinical trials that may cause us or the FDA or foreign regulatory agencies to delay, suspend or terminate

our clinical trials at any phase.

These problems could include the possibility that we may not be able to manufacture sufficient

quantities of materials for use in our clinical trials, conduct clinical trials at our preferred sites, enroll a sufficient number

of patients for our clinical trials at one or more sites, or begin or successfully complete clinical trials in a timely fashion,

Furthermore, we, the FDA or foreign regulatory agencies may suspend clinical trials of our product candidate at any

time if we or they believe the subjects participating in the trials are being exposed to unacceptable health risks, whether as

a result of adverse events occurring in our trials or otherwise, or if we or they find deficiencies in the clinical trial process

or conduct of the investigation.

The FDA and foreign regulatory agencies

could also require additional clinical trials before or after granting of marketing approval for any products, which would result

in increased costs and significant delays in the development and commercialization of such products and could result in the withdrawal

of such products from the market after obtaining marketing approval.

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claims that we infringe its patents, any of the following may occur:

we may be required to pay substantial

financial damages if a court decides that our technologies infringe a competitor’s patent, which can be tripled if the infringement

is deemed willful, or be required to discontinue or significantly delay development, marketing, selling and licensing of the affected

products and intellectual property rights;

a court may prohibit us from selling or

licensing our product without a license from the patent holder, which may not be available on commercially acceptable terms or

at all, or which may require us to pay substantial royalties or grant cross-licenses to our patents;

we may have to redesign our product so

that it does not infringe others’

patent rights, which may not be possible or could require substantial funds or time and

require additional studies.

we may be required to pay substantial financial damages if a court decides that our technologies

infringe a competitor’s patent, which can be tripled if the infringement is deemed willful, or be required to discontinue

or significantly delay development, marketing, selling and licensing of our product and intellectual property rights;

a court may prohibit us from selling or licensing our product without a license from the patent

holder, which may not be available on commercially acceptable terms or at all, or which may require us to pay substantial royalties

or grant cross-licenses to our patents;

we may have to redesign our product so that it does not infringe others’

patent rights, which

may not be possible or could require substantial funds or time and require additional studies.

In addition, employees,

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Some of our competitors have considerable resources

available to them and a strong economic incentive to undertake substantial efforts to stop or delay us from commercializing products.

Moreover, regardless of the outcome, intellectual property litigation against or by us could significantly disrupt our development

and commercialization efforts, divert our management’s attention and quickly consume our financial resources.

available to them and may have a strong economic incentive to undertake substantial efforts to stop or delay us from commercializing

Moreover, regardless of the outcome, intellectual property litigation against or by us could significantly disrupt

our development and commercialization efforts, divert our management’s attention and quickly consume our financial resources.

In addition, if

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development or other more productive matters.

Our proprietary rights may not adequately

protect our technologies and product candidate.

Our proprietary rights may not adequately protect our

technologies and product candidate.

Our commercial success will depend in part

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We will be able to

protect our proprietary rights from unauthorized use by third parties only to the extent that our proprietary technologies, product

candidate and any future products are covered by valid and enforceable patents or are effectively maintained as trade secrets.

We apply for patents covering both our technologies

and product candidate, as we deem appropriate.

However, we may fail to apply for patents on important technologies or product candidate

in a timely fashion, or at all.

Our existing patents and any future patents we obtain may not be sufficiently broad to prevent

others from practicing our technologies or from developing competing products and technologies.

We cannot be certain that our patent

applications will be approved or that any patents issued will adequately protect our intellectual property.

While we are responsible for and have control

over the filing and prosecuting of patent applications and maintaining patents which cover making, using or selling PEDMARK TM ,

we may lose any such rights if we decide to allow any licensed patent to lapse.

If we fail to appropriately prosecute and maintain

patent protection for any of our product candidate, our ability to develop and commercialize those product candidate may be adversely

affected and we may not be able to prevent competitors from making, using and selling competing products.

protect our proprietary rights from unauthorized use by third parties only to the extent that our proprietary technologies and

product candidate are covered by valid and enforceable patents or are effectively maintained as trade secrets.

We apply for patents covering both our

technologies and product candidate, as we deem appropriate.

However, we may fail to apply for patents on important technologies

or product candidate in a timely fashion, or at all.

Our existing patents and any future patents we obtain may not be sufficiently

broad to prevent others from practicing our technologies or from developing competing products and technologies.

We cannot be certain

that our patent applications will be approved or that any patents issued will adequately protect our intellectual property.

we are responsible for and have control over the filing and prosecuting of patent applications and maintaining patents which cover

making, using or selling PEDMARK TM , we may lose any such rights if we decide to allow any licensed patent to

If we fail to appropriately prosecute and maintain patent protection for any of our product candidate, our ability to develop

and commercialize those product candidate may be adversely affected and we may not be able to prevent competitors from making,

using and selling competing products.

Moreover, the patent positions of pharmaceutical

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we do not know whether:

we or our licensors were the first to make

the inventions covered by each of our issued patents and pending patent applications;

we or our licensors were the first to file

patent applications for these inventions;

any of the patents that cover our product

candidate will be eligible to be listed in the FDA’s compendium of “Approved Drug Products with Therapeutic Equivalence

Evaluation,”

sometimes referred to as the FDA’s Orange Book;

others will independently develop similar

or alternative technologies or duplicate any of our technologies;

we or our licensors were the first to make the inventions covered by each of our issued patents

and pending patent applications;

we or our licensors were the first to file patent applications for these inventions;

any of the patents that cover our product candidate will be eligible to be listed in the FDA’s

compendium of “Approved Drug Products with Therapeutic Equivalence Evaluation,”

sometimes referred to as the FDA’s

others will independently develop similar or alternative technologies or duplicate any of our technologies;

any of our or our licensors’

patent applications will result in issued patents;

any patents issued to us or our licensors

and collaborators will provide us with any competitive advantages, or will be challenge by third parties;

we will develop additional proprietary technologies

that are patentable;

the United States government will exercise

any of its statutory rights to our intellectual property that was developed with government funding;

our business may infringe the patents or

other proprietary rights of others.

pending patent applications will result in issued patents;

any patents issued to us or our licensors and collaborators will provide us with any competitive

advantages, or will be challenge by third parties;

we will develop additional proprietary technologies that are patentable;

the United States government will exercise any of its statutory rights to our intellectual property

that was developed with government funding;

our business may infringe the patents or other proprietary rights of others.

The actual protection afforded by a patent

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thereby reducing any advantage of the patent.

We may also rely on trade secrets to

protect some of our technology, especially where we do not believe patent protection is appropriate or obtainable.

trade secrets are difficult to maintain.

While we use reasonable efforts to protect our trade secrets, we or any of our

collaborators’

employees, consultants, contractors or scientific and other advisors may unintentionally or willfully

disclose our proprietary information to competitors and we may not have adequate remedies in respect of that disclosure.

Enforcement of claims that a third party has illegally obtained and is using trade secrets is expensive, time consuming and

In addition, foreign courts are sometimes less willing than United States courts to protect trade secrets.

competitors independently develop equivalent knowledge, methods and know-how, we would not be able to assert our trade

secrets against them and our business could be harmed.

We may not be able to protect our intellectual

property rights throughout the world.

We may also rely on trade secrets to protect

some of our technology, especially where we do not believe patent protection is appropriate or obtainable.

However, trade secrets

are difficult to maintain.

While we use reasonable efforts to protect our trade secrets, we or any of our collaborators’

employees, consultants, contractors or scientific and other advisors may unintentionally or willfully disclose our proprietary

information to competitors and we may not have adequate remedies in respect of that disclosure.

Enforcement of claims that a third

party has illegally obtained and is using trade secrets is expensive, time consuming and uncertain.

In addition, foreign courts

are sometimes less willing than United States courts to protect trade secrets.

If our competitors independently develop equivalent

knowledge, methods and know-how, we would not be able to assert our trade secrets against them and our business could be harmed.

We may not be able to protect our

intellectual property rights throughout the world.

Filing, prosecuting and defending patents

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For example, depending on the timing, duration and specifics of FDA marketing

approval of our product candidate, if any, one of the United States patents covering each of such approved product(s) or the use

thereof may be eligible for up to five years of patent term restoration under the Hatch-Waxman Act.

approval of our product candidate, if any, one of the United States patents covering each of such approved product(s) or the

use thereof may be eligible for up to five years of patent term restoration under the Hatch-Waxman Act.

The Hatch-Waxman Act allows

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countries upon regulatory approval of our product candidate.

Nevertheless, we may not be granted patent

term extension either in the United States or in any foreign country because of, for example, failing to apply within applicable

deadlines, failing to apply prior to expiration of relevant patents or otherwise failing to satisfy applicable requirements.

the term of extension, as well as the scope of patent protection during any such extension, afforded by the governmental authority

could be less than we request.

In addition, even though some regulatory authorities may provide some other exclusivity for

a product under their own laws and regulations, we may not be able to qualify the product or obtain the exclusive time period.

If we are unable to obtain patent term extension/restoration or some other exclusivity, we could be subject to increased competition

and our opportunity to establish or maintain product revenue could be substantially reduced or eliminated.

we may not have sufficient time to recover our development costs prior to the expiration of our United States and

foreign patents.

Nevertheless,

we may not be granted patent term extension either in the United States or in any foreign country because of, for example, failing

to apply within applicable deadlines, failing to apply prior to expiration of relevant patents or otherwise failing to satisfy

applicable requirements.

Moreover, the term of extension, as well as the scope of patent protection during any such extension,

afforded by the governmental authority could be less than we request.

even though some regulatory authorities may provide some other exclusivity for a product under their own laws and regulations,

we may not be able to qualify the product or obtain the exclusive time period.

If we are unable to obtain patent term extension/restoration or

some other exclusivity, we could be subject to increased competition and our opportunity to establish or maintain product

revenue could be substantially reduced or eliminated.

Furthermore, we may not have sufficient time to recover our development

costs prior to the expiration of our United States and foreign patents.

Obtaining and maintaining our patent

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patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.

The USPTO and various foreign

governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other similar

provisions during the patent prosecution process.

Periodic maintenance fees, renewal fees, annuity fees and various other

governmental fees on any issued patent and/or pending patent applications are due to be paid to the USPTO and foreign patent

agencies in several stages over the lifetime of a patent or patent application.

We employ an outside firm and rely on our

outside counsel to pay these fees.

While an inadvertent lapse may sometimes be cured by payment of a late fee or by other

means in accordance with the applicable rules, there are many situations in which noncompliance can result in abandonment or

lapse of the patent or patent application, resulting in partial or complete loss of patent rights in the relevant

jurisdiction.

If we fail to maintain the patents and patent applications directed to our product candidate, our competitors

might be able to enter the market earlier than should otherwise have been the case, which would have a material adverse

effect on our business.

We may become involved in lawsuits to

protect our patents or other intellectual property rights, which could be expensive, time-consuming and ultimately unsuccessful.

Competitors may infringe our patents or other

intellectual property rights.

To counter infringement or unauthorized use, we may be required to file infringement claims, directly

or through our licensors, which can be expensive and time consuming.

In addition, in an infringement proceeding, a court may decide

that a patent of our licensor is not valid or is unenforceable, or may refuse to stop the other party from using the technology

at issue on the grounds that our patents do not cover the technology in question.

An adverse result in any litigation or defense

proceedings could put one or more of the patents we license at risk of being invalidated or interpreted narrowly and could put

our licensors’

The USPTO and various foreign governmental

patent agencies require compliance with a number of procedural, documentary, fee payment and other similar provisions during the

patent prosecution process.

Periodic maintenance fees, renewal fees, annuity fees and various other governmental fees on any issued

patent and/or pending patent applications are due to be paid to the USPTO and foreign patent agencies in several stages over the

lifetime of a patent or patent application.

We employ an outside firm and rely on our outside counsel to pay these fees.

an inadvertent lapse may sometimes be cured by payment of a late fee or by other means in accordance with the applicable rules,

there are many situations in which noncompliance can result in abandonment or lapse of the patent or patent application, resulting

in partial or complete loss of patent rights in the relevant jurisdiction.

If we fail to maintain the patents and patent applications

directed to our product candidate, our competitors might be able to enter the market earlier than should otherwise have been the

case, which would have a material adverse effect on our business.

We may become involved in lawsuits

to protect our patents or other intellectual property rights, which could be expensive, time-consuming and ultimately unsuccessful.

Competitors may infringe our patents or

other intellectual property rights.

To counter infringement or unauthorized use, we may be required to file infringement claims,

directly or through our licensors, which can be expensive and time consuming.

In addition, in an infringement proceeding, a court

may decide that a patent of ours or of our licensors is not valid or is unenforceable, or may refuse to stop the other party from

using the technology at issue on the grounds that our patents do not cover the technology in question.

An adverse result in any

litigation or defense proceedings could put one or more of our patents or the patents we license at risk of being invalidated or

interpreted narrowly and could put our ot our licensors’

patent applications at risk of not issuing.

Interference proceedings brought by the USPTO

may be necessary to determine the priority of inventions with respect to our patents or the patents of our licensors.

An unfavorable

outcome could require us to cease using the technology or to attempt to license rights to it from the prevailing party.

could be harmed if a prevailing party does not offer us a license on terms that are acceptable to us.

Litigation or interference

proceedings may fail and, even if successful, may result in substantial costs and distraction of our management and other employees.

We may not be able to prevent, alone or with our licensors, misappropriation of our proprietary rights, particularly in countries

where the laws may not protect those rights as fully as in the United States.

In addition, potential infringers of our intellectual

property rights may have substantially more resources than we do to defend their position, which could adversely affect the outcome

of any such dispute.

Furthermore, because of the substantial amount

of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential and proprietary

information could be compromised by disclosure during this type of litigation.

In addition, there could be public announcements

of the results of hearings, motions or other interim proceedings or developments.

If securities analysts or investors perceive

these results to be negative, it could have a substantial adverse effect on the price of our common stock.

Third-party claims of intellectual property

infringement or misappropriation may adversely affect our business and could prevent us from developing or commercializing our

product candidate.

Our commercial success depends in part on

us not infringing the patents and proprietary rights of third parties.

Interference proceedings brought by the

USPTO may be necessary to determine the priority of inventions with respect to our patents or the patents of our licensors.

unfavorable outcome could require us to cease using the technology or to attempt to license rights to it from the prevailing party.

Our business could be harmed if a prevailing party does not offer us a license on terms that are acceptable to us.

Litigation or

interference proceedings may fail and, even if successful, may result in substantial costs and distraction of our management and

other employees.

We may not be able to prevent, alone or with our licensors, misappropriation of our proprietary rights, particularly

in countries where the laws may not protect those rights as fully as in the United States.

In addition, potential infringers of

our intellectual property rights may have substantially more resources than we do to defend their position, which could adversely

affect the outcome of any such dispute.

Furthermore, because of the substantial

amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential

and proprietary information could be compromised by disclosure during this type of litigation.

In addition, there could be public

announcements of the results of hearings, motions or other interim proceedings or developments.

If securities analysts or investors

perceive these results to be negative, it could have a substantial adverse effect on the price of our common stock.

Third-party claims of intellectual

property infringement or misappropriation may adversely affect our business and could prevent us from developing or commercializing

our product candidate.

Our commercial success depends in part

on us not infringing the patents and proprietary rights of third parties.

There is a substantial amount of litigation, both within

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a third party claims that we infringe on their products or technology, we could face a number of issues, including:

infringement and other intellectual property

claims which, with or without merit, can be expensive and time-consuming to litigate and can divert management’s attention

from our core business;

substantial damages for past infringement,

which we may have to pay if a court decides that our product infringes on a competitor’s patent;

a court prohibiting us from selling or licensing

our product unless the patent holder licenses the patent to us, which the collaborator would not be required to do;

if a license is available from a patent holder,

we may have to pay substantial royalties or grant cross licenses to our patents;

redesigning our processes so they do not

infringe, which may not be possible or could require substantial funds and time.

infringement and other intellectual property claims which, with or without merit, can be expensive

and time-consuming to litigate and can divert management’s attention from our core business;

substantial damages for past infringement, which we may have to pay if a court decides that our

product infringes on a competitor’s patent;

a court prohibiting us from selling or licensing our product unless the patent holder licenses

the patent to us, which the collaborator would not be required to do;

if a license is available from a patent holder, we may have to pay substantial royalties or grant

cross licenses to our patents;

redesigning our processes so they do not infringe, which may not be possible or could require substantial

funds and time.

Third parties may assert that we are employing

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failed to identify.

For example, applications filed before November 29, 2000 and certain applications filed after that date that

will not be filed outside the United States remain confidential until issued as patents.

Except for the preceding exceptions, patent

applications in the United States and elsewhere are generally published only after a waiting period of approximately 18 months

after the earliest filing.

Therefore, patent applications covering our product candidate could have been filed by others without

the knowledge of us or our licensors.

Additionally, pending patent applications which have been published can, subject to certain

limitations, be later amended in a manner that could cover our product candidate or the use or manufacture of our product candidate.

We may also face a claim of misappropriation if a third party believes that we inappropriately obtained and used trade secrets

of such third party.

If we are found to have misappropriated a third party’s trade secrets, we may be prevented from further

using such trade secrets, limiting our ability to develop our product candidate, and we may be required to pay damages.

If any third-party patents were held by a

court of competent jurisdiction to cover aspects of our materials, formulations, methods of manufacture or methods for treatment,

For example, applications filed before November 29, 2000 and certain applications filed after that date

that will not be filed outside the United States remain confidential until issued as patents.

Except for the preceding exceptions,

patent applications in the United States and elsewhere are generally published only after a waiting period of approximately 18

months after the earliest filing.

Therefore, patent applications covering our product candidate could have been filed by others

without the knowledge of us or our licensors.

Additionally, pending patent applications which have been published can, subject

to certain limitations, be later amended in a manner that could cover our product candidate or the use or manufacture of our product

We may also face a claim of misappropriation if a third party believes that we inappropriately obtained and used trade

secrets of such third party.

If we are found to have misappropriated a third party’s trade secrets, we may be prevented from

further using such trade secrets, limiting our ability to develop our product candidate, and we may be required to pay damages.

If any third-party patents were held by

a court of competent jurisdiction to cover aspects of our materials, formulations, methods of manufacture or methods for treatment,

the holders of any such patents would be able to block our ability to develop and commercialize the applicable product candidate

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same intellectual property.

Ultimately, we could be prevented from commercializing

a product, or be forced to cease some aspect of our business operations, if, as a result of actual or threatened patent infringement

claims, we are unable to enter into licenses on acceptable terms.

Parties making claims against us may

obtain injunctive or other equitable relief, which could effectively block our ability to further develop and commercialize

one or more of our product candidate.

Defending against claims of patent infringement or misappropriation of trade secrets

could be costly and time-consuming, regardless of the outcome.

Thus, even if we were to ultimately prevail, or to settle at

an early stage, such litigation could burden us with substantial unanticipated costs.

In addition, litigation or threatened

litigation could result in significant demands on the time and attention of our management team, distracting them from the

pursuit of other company business.

In the event of a successful claim of infringement against us, we may have to pay

substantial damages, including treble damages and attorneys’

fees for willful infringement, pay royalties, redesign our

infringing products or obtain one or more licenses from third parties, which may be impossible or require substantial time

and monetary expenditure.

In addition, the uncertainties associated with litigation could have a material adverse effect on

our ability to raise the funds necessary to continue our clinical trials, continue our research programs, license necessary

technology from third parties, or enter into development collaborations that would help us bring our product candidate to

Ultimately, we could be prevented from

commercializing a product, or be forced to cease some aspect of our business operations, if, as a result of actual or threatened

patent infringement claims, we are unable to enter into licenses on acceptable terms.

Parties making claims against us may obtain

injunctive or other equitable relief, which could effectively block our ability to further develop and commercialize one or more

of our product candidates.

Defending against claims of patent infringement or misappropriation of trade secrets could be costly

and time-consuming, regardless of the outcome.

Thus, even if we were to ultimately prevail, or to settle at an early stage, such

litigation could burden us with substantial unanticipated costs.

In addition, litigation or threatened litigation could result

in significant demands on the time and attention of our management team, distracting them from the pursuit of other company business.

In the event of a successful claim of infringement against us, we may have to pay substantial damages, including treble damages

and attorneys’

fees for willful infringement, pay royalties, redesign our infringing products or obtain one or more licenses

from third parties, which may be impossible or require substantial time and monetary expenditure.

In addition, the uncertainties

associated with litigation could have a material adverse effect on our ability to raise the funds necessary to continue our clinical

trials, continue our research programs, license necessary technology from third parties, or enter into development collaborations

that would help us bring our product candidate to market.

Changes in United States patent law

could diminish the value of patents in general, thereby impairing our ability to protect our product candidate.

As is the case with other pharmaceutical companies,

our success is heavily dependent on intellectual property, particularly on obtaining and enforcing patents and patent rights.

and enforcing patents and patent rights in the pharmaceutical industry involves both technological and legal complexity, and therefore,

is costly, time-consuming and inherently uncertain.

In addition, the United States has recently enacted and is currently implementing

wide-ranging patent reform legislation.

Further, several recent United States Supreme Court rulings have either narrowed the scope

of patent protection available in certain circumstances or weakened the rights of patent owners in certain situations.

to increasing uncertainty with regard to our ability to obtain patents in the future, this combination of events has created uncertainty

with respect to the value of patents and patent rights, once obtained.

As is the case with other pharmaceutical

companies, our success is heavily dependent on intellectual property, particularly on obtaining and enforcing patents and patent

Obtaining and enforcing patents and patent rights in the pharmaceutical industry involves both technological and legal

complexity, and therefore, is costly, time-consuming and inherently uncertain.

In addition, the United States has recently enacted

and is currently implementing wide-ranging patent reform legislation.

Further, several recent United States Supreme Court rulings

have either narrowed the scope of patent protection available in certain circumstances or weakened the rights of patent owners

in certain situations.

In addition to increasing uncertainty with regard to our ability to obtain patents in the future, this combination

of events has created uncertainty with respect to the value of patents and patent rights, once obtained.

For our United States patent applications

containing a claim not entitled to priority before March 16, 2013, there is a greater level of uncertainty in the patent law.

September 2011, the Leahy-Smith America Invents Act (the “America Invents Act”

or “AIA”) was signed into

The AIA includes a number of significant changes to United States patent law, including provisions that affect the way patent

applications will be prosecuted, reviewed after issuance, and may also affect patent litigation.

The USPTO is currently developing

regulations and procedures to govern administration of the AIA, and many of the substantive changes to patent law associated with

In September 2011, the Leahy-Smith America Invents Act (the “America Invents Act”

or “AIA”) was signed

The AIA includes a number of significant changes to United States patent law, including provisions that affect the way

patent applications will be prosecuted, reviewed after issuance, and may also affect patent litigation.

The USPTO is currently

developing regulations and procedures to govern administration of the AIA, and many of the substantive changes to patent law associated

with the AIA.

It is not clear what other, if any, impact the AIA will have on the operation of our business.

Moreover, the AIA and its

implementation could increase the uncertainties and costs surrounding the prosecution of patent applications and the enforcement

Moreover, the AIA

and its implementation could increase the uncertainties and costs surrounding the prosecution of patent applications and the enforcement

or defense of patent rights, all of which could have a material adverse effect on our business and financial condition.

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is that, as of March 16, 2013, the United States transitioned to a “first-inventor-to-file”

system for deciding which

party should be granted a patent when two or more patent applications are filed by different parties claiming the same invention.

system for deciding

which party should be granted a patent when two or more patent applications are filed by different parties claiming the same invention.

A third party that files a patent application in the USPTO after that date but before a licensor or us could therefore be awarded

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United States and most other countries are confidential for a period of time after filing, we cannot be certain that a licensor

or we were the first to either (a) file any patent application related to our product candidate or (b) invent any of the inventions

claimed in our patents or patent applications.

Among some of the other changes

introduced by the AIA are changes that limit where a patentee may file a patent infringement suit and providing opportunities

for third parties to challenge any issued patent in the USPTO.

This applies to all United States patents, even those issued

before March 16, 2013.

Because of a lower evidentiary standard in USPTO proceedings compared to the evidentiary standard in

United States federal court necessary to invalidate a patent claim, a third party could potentially provide evidence in a

USPTO proceeding sufficient for the USPTO to hold a claim invalid as unpatentable even though the same evidence may be

insufficient to invalidate the claim if first presented in a district court action.

Accordingly, a third party may attempt to

use the USPTO procedures to invalidate patent rights that would not have been invalidated if first challenged by the third

party as a defendant in a district court action.

or we were the first to either (a) file any patent application related to our product candidate or (b) invent any of

the inventions claimed in our patents or patent applications.

Among some of the other changes introduced

by the AIA are changes that limit where a patentee may file a patent infringement suit and providing opportunities for third parties

to challenge any issued patent in the USPTO.

This applies to all United States patents, even those issued before March 16,

Because of a lower evidentiary standard in USPTO proceedings compared to the evidentiary standard in United States federal

court necessary to invalidate a patent claim, a third party could potentially provide evidence in a USPTO proceeding sufficient

for the USPTO to hold a claim invalid as unpatentable even though the same evidence may be insufficient to invalidate the claim

if first presented in a district court action.

Accordingly, a third party may attempt to use the USPTO procedures to invalidate

patent rights that would not have been invalidated if first challenged by the third party as a defendant in a district court action.

Depending on decisions by the United States

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address all potential threats to our competitive advantage.

The degree of future protection afforded by

our intellectual property rights is uncertain because intellectual property rights have limitations, and may not adequately protect

our business, or permit us to maintain our competitive advantage.

The degree of future protection afforded

by our intellectual property rights is uncertain because intellectual property rights have limitations, and may not adequately

protect our business, or permit us to maintain our competitive advantage.

The following examples are illustrative:

Others may be able to make products that

are similar to our product candidate but that are not covered by the claims of the patents that we license from others or may license

or own in the future;

Others may independently develop similar

or alternative technologies or otherwise circumvent any of our technologies without infringing our intellectual property rights;

Any of our collaborators might not have been

the first to conceive and reduce to practice the inventions covered by the patents or patent applications that we license or will,

in the future, own or license;

Issued patents that have been licensed to

us may not provide us with any competitive advantage, or may be held invalid or unenforceable, as a result of legal challenges

by our competitors;

Our competitors might conduct research and

development activities in countries where we do not have license rights, or in countries where research and development safe harbor

laws exist, and then use the information learned from such activities to develop competitive products for sale in our major commercial

Ownership of patents or patent applications

licensed to us may be challenged by third parties;

The patents of third parties or pending or

future applications of third parties, if issued, may have an adverse effect on our business.

Others may be able to make products that are similar to our product candidate but that are not

covered by the claims of the patents that we license from others or may license or own in the future;

Others may independently develop similar or alternative technologies or otherwise circumvent any

of our technologies without infringing our intellectual property rights;

Any of our collaborators might not have been the first to conceive and reduce to practice the inventions

covered by the patents or patent applications that we license or will, in the future, own or license;

Issued patents that have been licensed to us may not provide us with any competitive advantage,

or may be held invalid or unenforceable, as a result of legal challenges by our competitors;

Our competitors might conduct research and development activities in countries where we do not

have license rights, or in countries where research and development safe harbor laws exist, and then use the information learned

from such activities to develop competitive products for sale in our major commercial markets;

Ownership of patents or patent applications licensed to us may be challenged by third parties;

The patents of third parties or pending or future applications of third parties, if issued, may

have an adverse effect on our business.

Confidentiality agreements with employees,

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terms or at all, which could materially harm our business.

We may be subject to claims that our

employees, consultants or independent contractors have wrongfully used or disclosed confidential information of third parties.

We have received confidential and

proprietary information from third parties.

In addition, we employ individuals who were previously employed at other

biotechnology or pharmaceutical companies.

We may be subject to claims that we or our employees, consultants or independent

contractors have inadvertently or otherwise improperly used or disclosed confidential information of these third parties or

our employees’

We may be subject to claims that

our employees, consultants or independent contractors have wrongfully used or disclosed confidential information of third parties.

We have received confidential and proprietary

information from third parties.

In addition, we employ individuals who were previously employed at other biotechnology or pharmaceutical

We may be subject to claims that we or our employees, consultants or independent contractors have inadvertently or otherwise

improperly used or disclosed confidential information of these third parties or our employees’

former employers.

Further, we may be subject to ownership disputes

in the future arising, for example, from conflicting obligations of consultants or others who are involved in developing our product

We may also be subject to claims that former employees, consultants, independent contractors, collaborators or other

third parties have an ownership interest in our patents or other intellectual property.

Litigation may be necessary to defend against

these and other claims challenging our right to and use of confidential and proprietary information.

If we fail in defending any

such claims, in addition to paying monetary damages, we may lose our rights therein.

Such an outcome could have a material adverse

effect on our business.

Even if we are successful in defending against

these claims, litigation could result in substantial cost and be a distraction to our management and employees.

Further, we may be subject to ownership

disputes in the future arising, for example, from conflicting obligations of consultants or others who are involved in developing

our product candidate.

We may also be subject to claims that former employees, consultants, independent contractors, collaborators

or other third parties have an ownership interest in our patents or other intellectual property.

Litigation may be necessary to

defend against these and other claims challenging our right to and use of confidential and proprietary information.

in defending any such claims, in addition to paying monetary damages, we may lose our rights therein.

Such an outcome could have

a material adverse effect on our business.

Even if we are successful in defending

against these claims, litigation could result in substantial cost and be a distraction to our management and employees.

Our reliance on third parties requires

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adverse effect on our business.

In addition, these agreements typically restrict

the ability of our advisors, employees, third-party contractors and consultants to publish data potentially relating to our trade

secrets, although our agreements may contain certain limited publication rights.

For example, any academic institution that we

may collaborate with in the future will usually expect to be granted rights to publish data arising out of such collaboration,

In addition, these agreements typically

restrict the ability of our advisors, employees, third-party contractors and consultants to publish data potentially relating to

our trade secrets, although our agreements may contain certain limited publication rights.

For example, any academic institution

that we may collaborate with in the future will usually expect to be granted rights to publish data arising out of such collaboration,

provided that we are notified in advance and given the opportunity to delay publication for a limited time period in order for

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Risks Related to Our Industry

If we are unable to obtain applicable U.S.

and/or foreign regulatory approvals, we will be unable to develop and commercialize our drug candidate.

The preclinical studies and clinical trials

of our product candidate, as well as the manufacturing, labeling, sale and distribution, export or import, marketing, advertising

and promotion of our product candidate, are subject to various regulatory frameworks in the United States, Canada and other countries.

Any products that we develop must receive all relevant regulatory approvals and clearances before any marketing, sale or distribution.

The regulatory process, which includes extensive preclinical studies and clinical testing to establish product safety and efficacy,

can take many years and cost substantial amounts of money.

As a result of the length of time, many challenges and costs are associated

with the drug development process, and the historical rate of failures for drug candidates is extremely high.

Changes in regulatory

policy could also cause delays or affect regulatory approval.

Any regulatory delays may increase our development costs and negatively

impact our competitiveness and prospects.

It is possible that we may not be able to obtain regulatory approval of our drug candidate

or approvals may take longer and cost more to obtain than expected.

Regulatory approvals, if granted, may entail

limitations on the uses for which any product we develop may be marketed, limiting the potential sales for any such products.

granting of product approvals can be withdrawn at any time, and manufacturers of approved products are subject to regular reviews,

including for compliance with FDA Good Manufacturing Practices regulations.

Failure to comply with any applicable regulatory requirement,

which may change from time to time, can result in warning letters, fines, sanctions, penalties, recalling or seizing products,

suspension of production, or even criminal prosecution.

Future sales of our product candidate may

suffer if it fails to achieve market acceptance.

Even if our product candidate is

successfully developed and achieves appropriate regulatory approval, it may not enjoy commercial acceptance or success.

product candidate may compete with a number of new and traditional drugs and therapies developed by major pharmaceutical and

biotechnology companies.

Market acceptance is dependent on the product candidate demonstrating clinical efficacy and safety,

as well as demonstrating advantages over alternative treatment methods.

In addition, market acceptance is influenced by

government reimbursement policies and the ability of third parties to pay for such products.

Physicians, patients, or the

medical community may not accept or utilize any products we may develop.

We face a strong competitive environment.

Other companies may develop or commercialize more effective or cheaper products, which may reduce or eliminate the demand for our

product candidate.

The biotechnology and pharmaceutical industry,

and in particular the field of cancer therapeutics where we are focused, is very competitive.

Many companies and research organizations

are engaged in the research, development and testing of new cancer therapies or means of increasing the effectiveness of existing

therapies, including, among many others, Amgen, AstraZeneca, Bayer, Bristol-Myers Squibb, Eli Lilly, Eisai, Merck KGaA, Novartis,

Johnson & Johnson, Pfizer, Roche, Taiho and Sanofi-Aventis.

Many of these companies have marketed drugs or are developing targeted

cancer therapeutics, which depending upon the mechanism of action of such agents could be competitors.

Many of our existing or potential competitors

have substantially greater financial, technical and human resources than we do and may be better equipped to develop, manufacture

and market products.

In addition, many of these competitors have extensive experience with preclinical testing and human clinical

trials and in obtaining regulatory approvals.

Also, some of the smaller companies that compete with us have formed collaborative

relationships with large, established companies to support the research, development, clinical trials and commercialization of

any products that they may develop.

Academic institutions, government agencies and other public and private research organizations

may also conduct research, seek patent protection and establish collaborative arrangements for research, clinical development and

marketing of products similar to those we seek to develop.

These companies and institutions compete with us in recruiting and retaining

qualified scientific and management personnel as well as in acquiring technologies complementary to our projects.

We are likely to face competition in the areas

of product efficacy and safety, ease of use and adaptability, as well as pricing, product acceptance, regulatory approvals and

intellectual property.

Competitors could develop more effective, safer and more affordable products than we do, and they may obtain

patent protection or product commercialization before we do or even render our product candidate obsolete.

The existence of competitive

products, including products or treatments of which we are not aware, or products or treatments that may be developed in the future,

may adversely affect the marketability of any product that we develop.

We may face product liability claims that

could require us to defend costly lawsuits or incur substantial liabilities that could adversely impact our financial condition,

receipt of regulatory approvals for our product candidate and our results of operation.

The use of our product candidate in clinical

trials and for commercial applications, if any, may expose us to liability claims in the event that such product candidate causes

injury or death or results in other adverse effects.

These claims could be made by health care institutions, contract laboratories,

and subjects participating in our clinical studies, patients or others using our product candidate.

In addition to liability claims,

certain serious adverse events could require interruption, delay and/or discontinuation of a clinical trial and potentially prevent

further development of our product candidate.

Litigation is very expensive, even if we defend successfully against possible litigation.

In addition, our existing insurance coverage may not be adequate to cover certain types or amounts of liability, and future coverage

may not be available in sufficient amounts or at reasonable cost.

Further, it is possible that we may later reduce or terminate

this coverage based on future availability of financial resources.

Adverse liability claims may also harm our ability to obtain

or maintain regulatory approvals.

Drug development

involves a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive

of future trial results.

Conducting clinical

trials is a lengthy, time-consuming and expensive process.

Before obtaining regulatory approvals for the commercial sale of any

products, we, or our potential partners, must demonstrate through preclinical testing and clinical trials that our product candidates

are safe and effective for their intended uses in humans.

We have incurred and may continue to incur substantial expense and devote

a significant amount of time to preclinical testing and clinical trials.

The outcome of

clinical testing is inherently uncertain.

Failure can occur at any time during the clinical trial process.

The results of preclinical

studies and early clinical trials of product candidates may not be predictive of the results of later-stage clinical trials.

addition, regulations are not static, and regulatory agencies, including the FDA, alter their staff, interpretations and practices

and may in the future impose more stringent requirements than are currently in effect, which may adversely affect our planned drug

development and/or our commercialization efforts.

Satisfying regulatory requirements typically takes a significant number of years

and can vary substantially based on the type, complexity and novelty of the product candidate.

Our business, results of operations

and financial condition may be materially adversely affected by any delays in, or termination of, our clinical trials.

that could impede our ability to generate commercially viable products through the conduct of clinical trials include:

insufficient funds to conduct clinical trials;

the inability to find partners, if necessary, for support, including research, development, manufacturing

or clinical needs;

the failure of tests or studies necessary to submit an NDA, such as clinical studies, bioequivalence

studies in support of a 505(b)(2) regulatory filing, or stability studies;

the failure of clinical trials to demonstrate the safety and efficacy of our product candidate

to the extent necessary to obtain regulatory approvals;

the failure by us or third-party investigators, CROs, or other third parties involved in the research

to adhere to regulatory requirements applicable to the conduct of clinical trials;

the failure of preclinical testing and early clinical trials to predict results of later clinical

any delay in completion of clinical trials caused by a regional disturbance where we or our collaborative

partners are enrolling patients in clinical studies, such as pandemic, terrorist activities, or war, or political unrest, a natural

disaster or any other reason or event, resulting in increased costs;

any delay in obtaining advice from the FDA or similar regulatory authorities;

the inability to obtain regulatory approval of our product candidate following completion of clinical

trials, or delays in obtaining such approvals.

can be no assurance that if our clinical trials are successfully initiated and completed, we will be able to obtain approval by

the FDA in the U.S.

or similar regulatory authorities elsewhere in the world in a timely manner, if at all.

For example, as described

elsewhere, we received a CRL from the FDA in August, 2020, regarding our NDA for PEDMARK TM , stating that it was

unable to approve the application in its current form based on deficiencies identified by the FDA after completion of a pre-approval

inspection of the manufacturing facility of our third-party drug product manufacturer.

Even if we are successful in resolving some

or all of the matters raised by the FDA in the CRL, there is significant risk that we will be unable to obtain FDA approval for

PEDMARK TM on a timely basis or at all .

If we fail to successfully develop and commercialize

PEDMARK TM we may be unable to generate sufficient revenues to attain profitability, and our reputation in the industry

and in the investment community would likely be damaged, each of which would cause our stock price to decrease.

We use hazardous materials and chemicals

in our research and development, and our failure to comply with laws related to hazardous materials could materially harm us.

Our research and development processes while

outsources, dies involve the controlled use of hazardous materials, such as flammable organic solvents, corrosive acids and corrosive

Accordingly, we are subject to federal, state, local and foreign laws and regulations governing the use, manufacture, storage,

handling and disposal of such materials and certain waste products.

The risk of accidental contamination or injury from these materials

cannot be completely eliminated.

We could be held liable for any damages that result and any such liability could exceed our resources

and may not be covered by our general liability insurance.

We currently do not carry insurance specifically for hazardous materials

We may be required to incur significant costs to comply with environmental laws and regulations, which may change from

time to time.

Efforts to reduce product pricing and health

care reimbursement and changes to government policies could negatively affect the commercialization of our product candidate.

If our product candidate achieves

regulatory approval, we may be materially adversely affected by the continuing efforts of governmental and third-party payers

to contain or reduce health care costs.

For example, if we succeed in bringing one or more products to market, such products

may not be considered cost-effective and the availability of consumer reimbursement may not exist or be sufficient to allow

the sale of such products on a competitive basis.

The constraints on pricing and availability of competitive products may

further limit our pricing and reimbursement policies as well as adversely impact market acceptance and commercialization of

our product candidate.

Our research and development processes,

while outsourced, does involve the controlled use of hazardous materials, such as flammable organic solvents, corrosive acids and

corrosive bases.

Accordingly, we are subject to federal, state, local and foreign laws and regulations governing the use, manufacture,

storage, handling and disposal of such materials and certain waste products.

The risk of accidental contamination or injury from

these materials cannot be completely eliminated.

We could be held liable for any damages that result and any such liability could

exceed our resources and may not be covered by our general liability insurance.

We currently do not carry insurance specifically

for hazardous materials claims.

We may be required to incur significant costs to comply with environmental laws and regulations,

which may change from time to time.

Efforts to reduce product pricing and

health care reimbursement and changes to government policies could negatively affect the commercialization of our product candidate.

If our product candidate achieves regulatory

approval, we may be materially adversely affected by the continuing efforts of governmental and third-party payers to contain or

reduce health care costs.

For example, if we succeed in bringing one or more products to market, such products may not be considered

cost-effective and the availability of consumer reimbursement may not exist or be sufficient to allow the sale of such products

on a competitive basis.

The constraints on pricing and availability of competitive products may further limit our pricing and reimbursement

policies as well as adversely impact market acceptance and commercialization of our product candidate.

In many markets, the pricing or profitability

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Since its enactment, there have been judicial

and Congressional challenges to numerous aspects of the Affordable Care Act, and Congress and the executive branch are seeking

to replace the Affordable Care Act with new federal legislation.

There may also be federal and state regulatory changes that impact

the Affordable Care Act or healthcare programs, insurance coverage or reimbursement generally.

These efforts have increased uncertainty

regarding the availability of healthcare programs, insurance coverage and reimbursement as a general matter as well as for our

product candidate, and we cannot predict how these events will impact our business.

In addition, there has been heightened governmental

scrutiny over the manner in which manufacturers set prices for their marketed products, which have resulted in several recent Congressional

inquiries and proposed bills designed to, among other things, bring more transparency to product pricing, review the relationship

between pricing and manufacturer patient programs, reduce the price of drugs under Medicare and reform government program reimbursement

methodologies for products.

We expect that additional state and federal healthcare reform measures will be adopted in the future,

any of which could limit the amounts that federal and state governments will pay for healthcare products and services, which could

result in reduced demand for our product candidate or additional pricing pressures.

and Congressional challenges to numerous aspects of the Affordable Care Act.

There may also be federal and state regulatory changes

that impact the Affordable Care Act or healthcare programs, insurance coverage or reimbursement generally.

These efforts have increased

uncertainty regarding the availability of healthcare programs, insurance coverage and reimbursement as a general matter as well

as for our product candidate, and we cannot predict how these events will impact our business.

In addition, there has been heightened

governmental scrutiny over the manner in which manufacturers set prices for their marketed products, which have resulted in several

recent Congressional inquiries and proposed bills designed to, among other things, bring more transparency to product pricing,

review the relationship between pricing and manufacturer patient programs, reduce the price of drugs under Medicare and reform

government program reimbursement methodologies for products.

We expect that additional state and federal healthcare reform measures

will be adopted in the future, any of which could limit the amounts that federal and state governments will pay for healthcare

products and services, which could result in reduced demand for our product candidate or additional pricing pressures.

Any significant changes in the healthcare

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of our common shares.

Our common shares are currently listed on

the Nasdaq Capital Market and the Toronto Stock Exchange (the “TSX”).

Both the Nasdaq Capital Market and the TSX have

rules for continued listing, including minimum market capitalization and other requirements that we might not meet in the future.

While we are exercising diligent efforts to maintain the listing of our common stock on the NASDAQ Capital Market and TSX, there

can be no assurance that we will be able to do so, and our securities could be delisted.

Our common shares are currently listed

on the Nasdaq Capital Market and the Toronto Stock Exchange (the “TSX”).

Both the Nasdaq Capital Market and the TSX

have rules for continued listing, including minimum market capitalization and other requirements that we might not meet in

While we are exercising diligent efforts to maintain the listing of our common shares on the NASDAQ Capital Market

and TSX, there can be no assurance that we will be able to do so, and our securities could be delisted.

from the Nasdaq Capital Market or the TSX would make it more difficult for shareholders to dispose of our common shares and more

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This could have an adverse effect on the price of our common shares.

There can be no assurances that a market maker will make a market in our common shares on the OTCQB o r

any other stock quotation system after delisting.

Furthermore, securities quoted over-the-counter generally have significantly

less liquidity than securities traded on a national securities exchange, not only in the number of shares that can be bought and

sold, but also through delays in the timing of transactions and lower market prices than might otherwise be obtained.

shareholders might find it difficult to resell shares at prices quoted in the market or at all.

Furthermore, because of the limited

market and generally low volume of trading in our common shares, our common shares are more likely to be affected by broad market

fluctuations, general market conditions, fluctuations in our operating results, changes in the market’s perception of our

business, and announcements made by us, our competitors or parties with whom we have business relationships.

Our ability to issue

additional securities for financing or other purposes, or to otherwise arrange for any financing we may need in the future, may

also be materially and adversely affected by the limited market and low trading volume of our common shares.

The market price of our common shares is

highly volatile and could cause the value of your investment to significantly decline.

There can be no assurances that a market maker will make a market in our common shares on the OTCQB o r any other stock quotation

system after delisting.

Furthermore, securities quoted over-the-counter generally have significantly less liquidity than securities

traded on a national securities exchange, not only in the number of shares that can be bought and sold, but also through delays

in the timing of transactions and lower market prices than might otherwise be obtained.

As a result, shareholders might find it

difficult to resell shares at prices quoted in the market or at all.

Furthermore, because of the limited market and generally low

volume of trading in our common shares, our common shares are more likely to be affected by broad market fluctuations, general

market conditions, fluctuations in our operating results, changes in the market’s perception of our business, and announcements

made by us, our competitors or parties with whom we have business relationships.

Our ability to issue additional securities for

financing or other purposes, or to otherwise arrange for any financing we may need in the future, may also be materially and adversely

affected by the limited market and low trading volume of our common shares.

The market price of our common shares

is highly volatile and could cause the value of your investment to significantly decline.

Historically, the market price of our common

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fluctuations, some of which are unrelated to our operating performance.

From March 11, 2013 to February 11, 2020, the closing trading

price of our stock fluctuated from a high of $18.45 Canadian dollars (“CAD”) per share to a low of CAD$0.72 per share

From September 13, 2017 to February 11, 2020, the closing trading price of our stock fluctuated from a high of $14.33

per share to a low of $3.30 on the Nasdaq Capital Market.

Historically, our common shares have had a low trading volume, and may

continue to have a low trading volume in the future.

This low volume may contribute to the volatility of the market price of our

common shares.

It is likely that the market price of our common shares will continue to fluctuate significantly in the future.

From January 1, 2018 to March 29, 2021, the closing

trading price of our stock fluctuated from a high of $18.45 Canadian dollars (“CAD”) per share to a low of CAD$4.38

per share on the TSX.

From September 13, 2017 (the date our common shares were first listed on the Nasdaq Capital Market)

to March 29, 2021, the closing trading price of our stock fluctuated from a high of $14.33 per share to a low of $3.30 on

the Nasdaq Capital Market.

Historically, our common shares have had a low trading volume, and may continue to have a low trading

volume in the future.

This low volume may contribute to the volatility of the market price of our common shares.

It is likely that

the market price of our common shares will continue to fluctuate significantly in the future.

The market price of our common shares may

be significantly affected by many factors, including without limitation:

the development of our sole product candidate,

the need to raise additional capital and

the terms of any transaction we are able to enter into;

other external factors generally or stock

market trends in the pharmaceutical or biotechnology industries specifically;

announcements of licensing agreements, joint

ventures, collaborations or other strategic alliances that involve our product or those of our competitors;

the development of our sole product candidate, PEDMARK TM ;

the need to raise additional capital and the terms of any transaction we are able to enter into;

other external factors generally or stock market trends in the pharmaceutical or biotechnology

industries specifically;

announcements of licensing agreements, joint ventures, collaborations or other strategic alliances

that involve our product or those of our competitors;

innovations related to our or our competitors’

actual or potential clinical trial results

related to our or our competitors’

the status, timing and outcome of regulatory

actual or potential clinical trial results related to our or our competitors’

the status, timing and outcome of regulatory approvals;

our financial results or those of our competitors;

reports of securities analysts regarding

us or our competitors;

developments or disputes concerning our licensed

or owned patents or those of our competitors;

developments with respect to the efficacy

or safety of our product or those of our competitors;

health care reforms and reimbursement policy

changes nationally and internationally.

Our existing principal shareholders hold

a substantial number of our common shares and may be able to exercise influence in matters requiring approval of our shareholders.

At February 11, 2020, our current shareholders

separately representing more than 5% ownership in our Company collectively represented beneficial ownership of approximately 41.86%

reports of securities analysts regarding us or our competitors;

developments or disputes concerning our licensed or owned patents or those of our competitors;

developments with respect to the efficacy or safety of our product or those of our competitors;

health care reforms and reimbursement policy changes nationally and internationally.

Our existing principal shareholders

hold a substantial number of our common shares and may be able to exercise influence in matters requiring approval of our shareholders.

At March 29, 2021, our current shareholders

separately representing more than 5% ownership of our common shares collectively represented beneficial ownership of approximately 47.06%

of our common shares.

In particular, Southpoint Capital Advisors LP (“Southpoint Capital”) owns or exercises control

over approximately 4.0 million common shares, representing approximately 20.1% of our issued and outstanding common shares;

SpA, owns approximately 3.2 million shares, or approximately 16.2% of our issued and outstanding common shares;

and venBio owns

approximately 1.1 million shares, or approximately 5.6% of our issued and outstanding common shares.

Southpoint Capital, Essetifin

SpA, venBio, our other significant shareholders, and other insiders, acting alone or together, might be able to influence the outcomes

of matters that require the approval of our shareholders, including but not limited to certain equity transactions (such as a financing),

an acquisition or merger with another company, a sale of substantially all of our assets, the election and removal of directors,

In particular, Southpoint Capital Advisors LP (“Southpoint Capital”) owns or exercises control over

approximately 4.0 million shares, representing approximately 15.68% of our issued and outstanding common shares;

Essetifin SpA, owns approximately

4.0 million shares, or approximately 15.36% of our issued and outstanding common shares;

Sonic Fund II, LP, owns approximately 2.5 million

shares, or approximately 9.60% of our issued and outstanding common shares;

and Avoro Capital Advisors owns approximately 1.7 million

shares, or approximately 6.42% of our issued and outstanding common shares.

Southpoint Capital, Essetifin SpA, Sonic Fund II, LP, Avoro

Capital Advisors, and our other significant shareholders, and other insiders, acting alone or together, might be able to influence the

outcomes of matters that require the approval of our shareholders, including but not limited to certain equity transactions (such as a

financing), an acquisition or merger with another company, a sale of substantially all of our assets, the election and removal of directors,

or amendments to our incorporating documents.

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The concentration

of ownership could have the effect of delaying, preventing or deterring a change of control of our Company, which could adversely

affect the market price of our common shares or deprive our other shareholders of an opportunity to receive a premium for our common

shares as part of a sale of our Company.

of ownership could have the effect of delaying, preventing or deterring a change of control of our Company, which could adversely affect

the market price of our common shares or deprive our other shareholders of an opportunity to receive a premium for our common shares as

part of a sale of our Company.

There are a large number of our common

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to obtain additional financing.

At February 11, 2020, we had outstanding warrants to purchase approximately 0.04 million shares

At March 29, 2021, we had outstanding warrants to purchase approximately 0.04 million shares

of our common shares at an exercise price of $6.80 per common share.

In addition, at February 11, 2020, there were approximately

3.1 million common shares issuable upon the exercise of outstanding stock options, of which options to purchase approximately $1.2

million were denominated in Canadian dollars and had a weighted average exercise price of CAD $2.43 per common share and options

to purchase approximately $9.9 million were denominated in U.S.

dollars and had a weighted average exercise price of $4.05 per

common share.

We may also issue further warrants as part of any future financings in addition to the additional 1.9 million options

to acquire our common shares currently remaining and available for future awards under our stock option plan.

We may need to raise additional funds in

the future to continue our operations.

Any equity offering could result in significant dilution to the ownership interests of shareholders

and may result in dilution of the value of such interests and any debt offering will increase financial risk.

In addition, at March 29, 2021, there were approximately

3.0 million common shares issuable upon the exercise of outstanding stock options with a weighted average exercise price of $4.82

per common share.

We may also issue further warrants as part of any future financings in addition to the additional 3.5 million

options to acquire our common shares currently remaining and available for future awards under our stock option plan.

We may need to raise additional funds

in the future to continue our operations.

Any equity offering could result in significant dilution to the ownership interests of

shareholders and may result in dilution of the value of such interests and any debt offering will increase financial risk.

In order to satisfy our anticipated capital

requirements to develop our product, we may need to raise additional funds through either the sale of additional equity, the issue

requirements to develop our product, we may need to raise additional funds through either the sale of additional equity, the issuance

of securities convertible into equity, the issuance of debt, the establishment of collaborations that provide us with funding,

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would have an adverse effect on the value of our common stock and could make it more difficult and costlier to raise funds in the

We have not paid any dividends since incorporation

and do not anticipate declaring any dividends in the foreseeable future.

As a result, you may not be able to recoup your investment

through the payment of dividends on your common shares and the lack of a dividend payable on our common shares might depress the

value of your investment.

For the foreseeable future, we plan to use

all available funds to finance the development of our product candidate and operate our business.

have not paid any dividends since incorporation and do not anticipate declaring any dividends in the foreseeable future.

you may not be able to recoup your investment through the payment of dividends on your common shares and the lack of a dividend

payable on our common shares might depress the value of your investment.

For the foreseeable future, we plan to

use all available funds to finance the development of our product candidate and operate our business.

Our directors will determine

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with respect to our status as a PFIC for our current taxable year or any subsequent taxable year.

Moreover, if we are a PFIC for

any taxable year, we intend to provide to a U.S.

Holder such information as the Internal Revenue Service (“IRS”) may

require, including a PFIC annual information statement, in order to enable the U.S.

Holder to make and maintain a “qualified

electing fund”

investors to consult their own tax advisors regarding the possible application of the

If we are a PFIC for any taxable

year, we intend to provide to a U.S.

Holder such information as the Internal Revenue Service (“IRS”) may require, including

a PFIC annual information statement, in order to enable the U.S.

Holder to make and maintain a “qualified electing fund”

For a more detailed explanation of the tax consequences of PFIC classification to U.S.

Holders, see the section of

this Annual Report entitled “Material U.S.

Federal Income Tax Considerations—General Rules Applicable to the Ownership

and Disposition of Common Shares.”

This paragraph is qualified in its entirety by the discussion below under the heading

“Material U.S.

Holders, see the section of this

Annual Report entitled “Material U.S.

Federal Income Tax Considerations.”

shareholder should consult its own tax advisors regarding

the PFIC rules and the U.S.

This paragraph is qualified in its entirety

by the discussion under that heading.

shareholder should consult its own tax advisors regarding the PFIC rules and

federal income tax consequences of the acquisition, ownership, and disposition of our common shares.

Failure to maintain effective internal

controls in accordance with Section 404 of the Sarbanes-Oxley Act of 2002 could have an adverse effect on our business, and our

per share price may be adversely affected.

controls in accordance with Section 404 of the Sarbanes-Oxley Act of 2002 could have an adverse effect on our business.

Pursuant to Section 404 of the Sarbanes-Oxley

Act of 2002 (“Section 404”) and the rules and regulations promulgated by the SEC to implement Section 404, we are required

to include in our Form 10-K a report by our management regarding the effectiveness of our internal control over financial reporting.

The report includes, among other things, an assessment of the effectiveness of our internal control over financial reporting.

assessment must include disclosure of any material weakness in our internal control over financial reporting identified by management.

As part of the evaluation undertaken by management

pursuant to Section 404, our internal control over financial reporting was effective as of December 31, 2019.

we fail to maintain an effective system of disclosure controls or internal controls over financial reporting, we may discover material

weaknesses that we would then be required to disclose.

Any material weaknesses identified in our internal controls could have an

adverse effect on our business.

We may not be able to accurately or timely report on our financial results, and we might be subject

to investigation by regulatory authorities.

This could result in a loss of investor confidence in the accuracy and completeness

of our financial reports, which may have an adverse effect on our stock price.

Act of 2002 (“Section 404”) and the rules and regulations promulgated by the SEC to implement Section 404,

we are required to include in our Form 10-K a report by our management regarding the effectiveness of our internal control

over financial reporting.

The report includes, among other things, an assessment of the effectiveness of our internal control over

financial reporting.

The assessment must include disclosure of any material weakness in our internal control over financial reporting

identified by management.

As part of the evaluation undertaken by

management pursuant to Section 404, our management concluded that our internal control over financial reporting was effective

as of December 31, 2020.

However, if we fail to maintain an effective system of disclosure controls or internal controls over

financial reporting, we may discover material weaknesses that we would then be required to disclose.

Any material weaknesses identified

in our internal controls could have an adverse effect on our business.

We may not be able to accurately or timely report on our

financial results, and we might be subject to investigation by regulatory authorities.

This could result in a loss of investor

confidence in the accuracy and completeness of our financial reports, which may have an adverse effect on our stock price.

No evaluation process can provide complete

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in implementing appropriate controls will increase and may require that we evolve some or all of our internal control processes.

Under applicable SEC rules, our management’s assessment of the effectiveness of our internal control over financial reporting

are not attested to by our registered public accounting firm.

It is also possible that the overall scope

of Section 404 may be revised in the future, thereby causing ourselves to review, revise or reevaluate our internal control processes,

which may result in the expenditure of additional human and financial resources.

of Section 404 may be revised in the future, thereby causing ourselves to review, revise or reevaluate our internal control

processes, which may result in the expenditure of additional human and financial resources.

Unresolved Staff Comments

We have an operating lease in Research Triangle

Park, North Carolina utilizing small space within a commercial building.

The operating lease has payments of $200 per month with

no scheduled increases.

This operating lease is terminable with 30 days’

notice and has no penalties or contingent payments

Legal Proceedings

Mine Safety Disclosures

Not applicable.

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 1A. Risk Factors