What changed in this filing

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Item 1A. Risk Factors

2020-11-16 compared with 2020-08-05 · 132 added, 18 removed, 44 unchanged (77% of the section changed)

Risk Factors.

Our Annual Report on Form 10-K for

the fiscal year ended December 31, 2019, filed with the SEC on February 14, 2020 (the “Annual Report”), includes

a detailed discussion of our risk factors under the heading “PART I, Item 1A –

the fiscal year ended December 31, 2019, filed with the SEC on February 14, 2020 (the “Annual Report”),

includes a detailed discussion of our risk factors under the heading “PART I, Item 1A –

Risk Factors.”

carefully consider the risk factors discussed in our Annual Report, as well as other information in this quarterly report.

of these risks could cause our business, financial condition, results of operations and future growth prospects to suffer.

not aware of any material changes from the risk factors previously disclosed.

Our business and operations may be materially

and adversely affected by the recent coronavirus outbreak.

In December 2019, a novel strain of

coronavirus was reported to have surfaced in Wuhan, China and has since spread to other parts of the world, including the United

You should carefully consider the risk factors discussed in our Annual Report, as well as other information in this quarterly

Any of these risks could cause our business, financial condition, results of operations and future growth prospects to

We are not aware of any material changes from the risk factors previously disclosed.

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.

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 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 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;

may otherwise take the position that PEDMARK fails to meet the requirements and standards for regulatory approval.

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 ( “

or other data and information, and the development and provision of these data and information may be time consuming and

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 a New Drug Application

to the FDA for PEDMARK 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 Complete Response Letter ( “

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™.

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 by the FDA.

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

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 revenues.

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

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.

Although historically

we have been a research and development company, we currently plan to commercialize our lead product candidate internally rather

than license such asset.

There can be no assurance that we will be successful in developing and expanding commercial operations

or balancing our research and development activities with our commercialization activities.

We have historically

been engaged primarily in research and development activities, but plan to commercialize our lead product candidate, PEDMARK,

There can be no assurance that we will be able to successfully manage the balance of our research and development operations

with our planned commercialization activities.

Potential investors should be aware of the problems, delays, expenses and difficulties

frequently encountered by companies balancing development of product candidates, which can include problems such as unanticipated

issues relating to clinical trials and receipt of approvals from the FDA and foreign regulatory bodies, with commercialization

efforts, which can include problems relating to managing manufacturing and supply, reimbursement, marketing problems and additional

Our product candidate may require significant additional research and clinical trials, and we will need to overcome significant

regulatory burdens prior to commercialization in the United States and other countries.

In addition, we may be required to spend

significant funds on building out our commercial operations.

There can be no assurance that after the expenditure of substantial

funds and efforts, we will successfully develop and commercialize PEDMARK, generate any significant revenues or ever achieve and

maintain a substantial level of sales of our product.

We are 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.

Moreover, 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 Complete Response Letter

from the FDA regarding our NDA for PEDMARK™.

See Item 1 under Legal Proceedings to our unaudited condensed consolidated

financial statements included in this report.

Regardless of the outcome, 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.

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

certificate of incorporation limits, 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 bylaws 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 ’

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

However, in 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 there from.

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.

Our business and operations may be

materially and adversely affected by the recent coronavirus outbreak.

In December 2019, a novel strain

of coronavirus was reported to have surfaced in Wuhan, China and has since spread to other parts of the world, including the United

States and Europe.

22 unchanged sentences

We do not yet know

the full extent of potential delays or impacts on our business, financing or preclinical and clinical trial activities or the global

economy as a whole.

However, these effects could have a material impact on our liquidity, capital resources, operations and business

and those of the third parties on which we rely.

the full extent of potential delays or impacts on our business, financing or preclinical and clinical trial activities or the

global economy as a whole.

However, these effects could have a material impact on our liquidity, capital resources, operations

and business and those of the third parties on which we rely.

Natural disasters, epidemic or pandemic

13 unchanged sentences

timely perform basic business functions, including, without limitation, making SEC filings and preparing financial reports.

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

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.

Recent Sales of Unregistered

Default Upon Senior Securities.

Sales of Unregistered Securities.

Upon Senior Securities.

Mine Safety Disclosure.

1 unchanged sentence

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-Q

Item 1A. Risk Factors