What changed in this filing

/stocks — the workspaceLOADING

Item 1A. Risk Factors

2020-11-05 compared with 2020-08-06 · 56 added, 52 removed, 1538 unchanged (7% of the section changed)

11 unchanged sentences

*The current outbreak of the novel coronavirus, or COVID-19,

has caused, and could continue to cause, severe disruptions in the U.S., regional and global economies and could seriously harm our development efforts, increase our costs and expenses and have a material adverse effect on our business, financial condition and results of operations.

pandemic has caused, and could continue to cause, severe disruptions in the U.S., regional and global economies and could seriously harm our development efforts, increase our costs and expenses and have a material adverse effect on our business, financial condition and results of operations.

Widespread outbreak of illness or other communicable diseases, health epidemics, or any other public health crisis could adversely affect our ongoing or planned research and development activities.

7 unchanged sentences

pandemic impacts our business, financial condition and results of operations will depend on future developments, which are highly uncertain and cannot be predicted with confidence, including the scope, severity and duration of such pandemic, the actions taken to contain the pandemic or mitigate its impact, as well as the effect of any relaxation of current restrictions within the Cambridge community or regions in which our partners and clinical sites are located, and the direct and indirect economic effects of the pandemic and containment measures, among others.

The rapid development and fluidity of this situation precludes any prediction as to the full adverse impact of the COVID-19

The rapid development and fluidity of this situation

precludes any prediction as to the full adverse impact of the COVID-19

Nevertheless, the COVID-19

6 unchanged sentences

Additionally, based in part on enrollment challenges common to rare disease populations that were exacerbated during the COVID-19

pandemic, we recently discontinued enrollment in our Phase 2 trial for MGTA-456

in inherited metabolic disorders.

pandemic, we discontinued enrollment in our Phase 2 trial for MGTA-456

in inherited metabolic disorders in June 2020.

Other potential impacts of the COVID-19

37 unchanged sentences

As a result, we are not profitable and have incurred losses in each period since our inception in June 2015.

For the six months ended June 30, 2020 and the years ended December 31, 2019 and 2018, we reported net losses of $39.1 million, $76.8 million and $57.5 million, respectively.

As of June 30, 2020, we had an accumulated deficit of $218.6 million.

For the nine months ended September 30, 2020 and the years ended December 31, 2019 and 2018, we reported net losses of $56.8 million, $76.8 million and $57.5 million, respectively.

As of September 30, 2020, we had an accumulated deficit of $236.3 million.

We expect to continue to incur significant losses for the foreseeable future, and we expect these losses to increase as we continue our research and development of, and seek regulatory approvals for, our product candidates.

7 unchanged sentences

public offerings in May 2019 and June 2020) to conduct further research and development and preclinical testing and clinical trials of our product candidates, to seek regulatory approvals for our product candidates and to launch and commercialize any product candidates for which we receive regulatory approval, including potentially building our own commercial organization to address the U.S., the European Union and certain other markets.

As of June 30, 2020, we had approximately $176.5 million in cash, cash equivalents and marketable securities.

As of September 30, 2020, we had approximately $161.7 million in cash, cash equivalents and marketable securities.

Our monthly spending levels will vary based on new and ongoing development and corporate activities.

118 unchanged sentences

There is typically an extremely high rate of attrition from the failure of product candidates proceeding through clinical trials.

For example, we have observed a limited number of serious adverse events in the Phase 2 clinical trial of MGTA-456

For example, we have observed a limited number of serious

adverse events in the Phase 2 clinical trial of MGTA-456

in blood cancers that were considered to be related to the investigational treatment, and there is no guarantee that we will not see more serious adverse events in the future.

22 unchanged sentences

For example, based in part on enrollment challenges common to rare disease populations that were exacerbated during the COVID-19

pandemic, we recently discontinued enrollment in our Phase 2 trial for MGTA-456

in inherited metabolic disorders and our plans to initiate multiple Phase 2 trials for MGTA-145

pandemic, we discontinued enrollment in our Phase 2 trial for MGTA-456

in inherited metabolic disorders in June 2020 and our plans to initiate multiple Phase 2 trials for MGTA-145

may be staggered over the course of 2020.

42 unchanged sentences

In particular, immune reset involves certain known potential post-procedure complications that may manifest several weeks or months after a transplant and which may be more common in certain patient populations.

For example, up to 20% of patients with inherited metabolic disorders treated with a transplant experience primary engraftment failure, resulting in severe complications,

including death.

For example, up to 20% of patients with inherited metabolic disorders treated with a transplant experience primary engraftment failure, resulting in severe complications, including death.

Another example is autoimmune cytopenia, a known and severe frequent complication of the transplant procedure in patients with non-malignant

59 unchanged sentences

Gene therapies, including genetically modified cells that lead to a durable modification of cells or tissues may meet the definition of a regenerative medicine therapy.

The RMAT program is intended to facilitate efficient development and expedite review of RMATs, which are intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition and for which preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.

for which preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.

A BLA for a regenerative medicine therapy that has received RMAT designation may be eligible for priority review or accelerated approval.

62 unchanged sentences

If the FDA determines that a product candidate offers a treatment for a serious condition and, if approved, the product would provide a significant improvement in safety or effectiveness, the FDA may designate the product candidate for priority review.

A priority review designation means that the goal for the FDA to review an application is six months, rather than the standard review period of ten months.

A priority review designation means that the goal for the FDA to review an application is six months, rather than the standard review

period of ten months.

We may request priority review for our product candidates, however, we cannot assume that MGTA-145,

195 unchanged sentences

evaluations required following the use of product candidates.

Patients are unlikely to use our product candidates unless coverage is provided and reimbursement is adequate to cover a

significant portion of the cost of our product candidates.

Patients are unlikely to use our product candidates unless coverage is provided and reimbursement is adequate to cover a significant portion of the cost of our product candidates.

Because our product candidates may have a higher cost of goods than conventional therapies, and may require long-term follow up evaluations, the risk that coverage and reimbursement rates may be inadequate for us to achieve profitability may be greater.

55 unchanged sentences

In some foreign countries, particularly those in the European Union, the pricing of biologics is subject to governmental control and other market regulations which could put pressure on the pricing and usage of our product candidates.

In these countries, pricing negotiations with governmental authorities can take considerable time after obtaining marketing approval of a product candidate.

In these countries,

pricing negotiations with governmental authorities can take considerable time after obtaining marketing approval of a product candidate.

In addition, market acceptance and sales of our product candidates will depend significantly on the availability of adequate coverage and reimbursement from third-party payors for our product candidates and may be affected by existing and future health care reform measures.

41 unchanged sentences

Additionally, HIPAA, as amended by the Health Information Technology and Clinical Health Act, or HITECH, and its implementing regulations, imposes certain requirements relating to the privacy, security and transmission of individually identifiable health information.

Among other things, HITECH makes HIPAA’s privacy and security standards directly applicable to “business

associates,” those independent contractors or agents of covered entities that create, receive, maintain, transmit or obtain protected health information in connection with providing a service on behalf of a covered entity.

Among other things, HITECH makes HIPAA’s privacy and security standards directly applicable to “business associates,” those independent contractors or agents of covered entities that create, receive, maintain, transmit or obtain protected health information in connection with providing a service on behalf of a covered entity.

HITECH also increased the civil and criminal penalties that may be imposed against covered entities, business associates and possibly other persons, and gave state attorneys general new authority to file civil actions for damages or injunctions in federal courts to enforce the federal HIPAA laws and seek attorney’s fees and costs associated with pursuing federal civil actions.

54 unchanged sentences

While any proposed measures will require authorization through additional legislation to become effective, Congress and the Trump administration have each indicated that it will continue to seek new legislative and/or administrative measures to control drug costs.

For example, on September 25, 2019, the Senate Finance Committee introduced the Prescription Drug Pricing Reduction Action of 2019, a bill intended to reduce Medicare and Medicaid prescription drug prices.

For example, on September 25, 2019, the Senate Finance

Committee introduced the Prescription Drug Pricing Reduction Action of 2019, a bill intended to reduce Medicare and Medicaid prescription drug prices.

The proposed legislation would restructure the Part D benefit, modify payment methodologies for certain drugs, and impose an inflation cap on drug price increases.

22 unchanged sentences

(1) eliminate protection under an Anti-Kickback Statute safe harbor for certain retrospective price reductions provided by drug manufacturers to sponsors of Medicare Part D plans or pharmacy benefit managers that are not applied at the point-of-sale;

(2) allow the importation of certain drugs from other countries through individual waivers, permit the re-importation of insulin products, and prioritize finalization of the FDA’s December 2019 proposed rule to permit the importation of drugs from Canada;

(2) allow the importation of certain drugs from other countries through individual waivers, permit the re-importation

of insulin products, and prioritize finalization of the FDA’s December 2019 proposed rule to permit the importation of drugs from Canada;

(3) ensure that payment by the Medicare program for certain Medicare Part B drugs is not higher than the payment by other comparable countries (depending on whether pharmaceutical manufacturers agree to other measures);

and (4) allow certain low-income individuals receiving insulin and epinephrine purchased by a Federally Qualified Health Center as part of the 340B drug pricing program to purchase those drugs at the discounted price paid by the Federally Qualified Health Center.

and (4) allow certain low-income

individuals receiving insulin and epinephrine purchased by a Federally Qualified Health Center as part of the 340B drug pricing program to purchase those drugs at the discounted price paid by the Federally Qualified Health Center.

It is unclear if, when, and to what extent the Executive Orders and the Draft Guidance may be implemented.

4 unchanged sentences

We face an inherent risk of product liability as a result of the clinical testing of our product candidates and will face an even greater risk if we commercialize any products.

For example, we may be sued if our product candidates cause or are perceived to cause injury or are found to be otherwise unsuitable during clinical testing, manufacturing, marketing or sale.

For example, we may be sued if our product candidates cause or are perceived to cause

injury or are found to be otherwise unsuitable during clinical testing, manufacturing, marketing or sale.

Any such product liability claims may include allegations of defects in manufacturing, defects in design, a failure to warn of dangers inherent in the product, negligence, strict liability or a breach of warranties.

17 unchanged sentences

Although we currently carry clinical trial insurance, the amount of such insurance coverage may not be adequate, we may be unable to maintain such insurance, or we may not be able to obtain additional or replacement insurance at a reasonable cost, if at all.

Our insurance policies

may also have various exclusions, and we may be subject to a product liability claim for which we have no coverage.

Our insurance policies may also have various exclusions, and we may be subject to a product liability claim for which we have no coverage.

We may have to pay any amounts awarded by a court or negotiated in a settlement that exceed our coverage limitations or that are not covered by our insurance, and we may not have, or be able to obtain, sufficient capital to pay such amounts.

82 unchanged sentences

In some instances, agreements through which we license patent rights may not give us control over patent prosecution or maintenance, so that we may not be able to control which claims or arguments are presented, how claims are amended, and may not be able to secure, maintain, or successfully enforce necessary or desirable patent protection from those patent rights.

We have not had and do not have primary control over patent prosecution and maintenance for certain of the patents and patent applications we license, including under our agreement with Novartis, and therefore cannot guarantee that these patents and applications will be prosecuted or maintained in a manner consistent with the best interests of our business.

maintained in a manner consistent with the best interests of our business.

We cannot be certain that patent prosecution and maintenance activities by our licensors have been or will be conducted in compliance with applicable laws and regulations or will result in valid and enforceable patents.

56 unchanged sentences

or in various foreign patent offices, including both national and regional, challenging patents or patent applications in which we have rights, including patents on which we rely to protect our business.

An adverse determination in any such challenges may result in loss of the patent or in patent claims being narrowed, invalidated or held

unenforceable, in whole or in part, or in denial of the patent application or loss or reduction in the scope of one or more claims of the patent application, any of which could limit our ability to stop others from using or commercializing similar or identical technology and products, or limit the duration of the patent protection of our technology and products.

An adverse determination in any such challenges may result in loss of the patent or in patent claims being narrowed, invalidated or held unenforceable, in whole or in part, or in denial of the patent application or loss or reduction in the scope of one or more claims of the patent application, any of which could limit our ability to stop others from using or commercializing similar or identical technology and products, or limit the duration of the patent protection of our technology and products.

In addition, given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized.

34 unchanged sentences

However, we may not obtain these agreements in all circumstances, and individuals with whom we have these agreements may not comply with their terms.

The assignment of intellectual property rights under these agreements may not be self-executing or the assignment agreements may be breached, and we may be forced to bring claims against third parties, or defend claims that they may bring against us, to determine the ownership of what we

regard as our intellectual property.

In addition, we may not be able to prevent the unauthorized disclosure or use of our technical know-how

39 unchanged sentences

Defense of these claims, regardless of their merit, would involve substantial litigation expense and would be a substantial diversion of employee resources from our business.

We may not have

sufficient resources to bring these actions to a successful conclusion.

We may not have sufficient resources to bring these actions to a successful conclusion.

There could also be public announcements of the results of hearings, motions or other interim proceedings or developments.

46 unchanged sentences

To counter infringement or unauthorized use, we may be required to file infringement claims, which can be expensive and time-consuming and divert the time and attention of our management and scientific personnel.

Any claims we assert against perceived infringers could provoke these

parties to assert counterclaims against us alleging that we infringe their patents, in addition to counterclaims asserting that our patents are invalid or unenforceable, or both.

Any claims we assert against perceived infringers could provoke these parties to assert counterclaims against us alleging that we infringe their patents, in addition to counterclaims asserting that our patents are invalid or unenforceable, or both.

In any patent infringement proceeding, there is a risk that a court will decide that a patent of ours is invalid or unenforceable, in whole or in part, and that we do not have the right to stop the other party from using the invention at issue.

15 unchanged sentences

In addition, the U.S.

has recently enacted and is currently implementing wide-ranging patent reform legislation.

has recently enacted and is currently implementing

wide-ranging patent reform legislation.

Supreme Court rulings have narrowed the scope of patent protection available in certain circumstances and weakened the rights of patent owners in certain situations.

7 unchanged sentences

affects the way antibody claims are examined and litigated.

While we do not believe that any of the patents owned or licensed by us will be found invalid based on this decisions, we cannot predict how future decisions by the courts, the Congress or the USPTO may impact the value of our patents.

While we do not believe that any of the patents owned or licensed by us will be found invalid based on these decisions, we cannot predict how future decisions by the courts, the Congress or the USPTO may impact the value of our patents.

We may not be able to protect our intellectual property rights throughout the world.

28 unchanged sentences

Moreover, the length of the extension could be less than we request.

Only one patent per approved product can be extended, the extension cannot extend the total patent term beyond 14 years from approval and only those claims covering the approved drug, a method for using it or a method for manufacturing it may be extended.

Only one patent per approved product can be extended, the extension cannot extend the

total patent term beyond 14 years from approval and only those claims covering the approved drug, a method for using it or a method for manufacturing it may be extended.

If we are unable to obtain patent term extension or the term of any such extension is less than we request, the period during which we can enforce our patent rights for the applicable product candidate will be shortened and our competitors may obtain approval to market competing products sooner.

65 unchanged sentences

Whether we reach a definitive agreement for a collaboration will depend, among other things, upon our assessment of the collaborator’s resources and expertise, the terms and conditions of the proposed collaboration, and the proposed collaborator’s evaluation of a number of factors.

Those factors may include

the design or results of clinical trials, the likelihood of approval by the FDA or similar regulatory authorities outside the U.S., the potential market for the subject product candidate, the costs and complexities of manufacturing and delivering such product candidate to patients, the potential of competing products, the existence of uncertainty with respect to our ownership of technology, which can exist if there is a challenge to such ownership without regard to the merits of the challenge, and industry and market conditions generally.

Those factors may include the design or results of clinical trials, the likelihood of approval by the FDA or similar regulatory authorities outside the U.S., the potential market for the subject product candidate, the costs and complexities of manufacturing and delivering such product candidate to patients, the potential of competing products, the existence of uncertainty with respect to our ownership of technology, which can exist if there is a challenge to such ownership without regard to the merits of the challenge, and industry and market conditions generally.

The collaborator may also consider alternative product candidates or technologies for similar indications that may be available to collaborate on and whether such a collaboration could be more attractive than the one with us.

9 unchanged sentences

The manufacture of clinical supplies for trials and commercial quantities of our current product candidates and any future product candidates are likely to be inherently more difficult and costly than typical chemical pharmaceuticals.

This could delay commercialization of any of our product candidates, if approved, or reduce the profitability of these candidates for us.

could delay commercialization of any of our product candidates, if approved, or reduce the profitability of these candidates for us.

If any of these occur, the development and commercialization of our product candidates could be delayed, curtailed or terminated because we may not have sufficient financial resources or capabilities to continue such development and commercialization on our own.

11 unchanged sentences

We will need to grow the size of our organization, and we may experience difficulties in managing this growth.

As of June 30, 2020, we had 72 full time employees.

As of September 30, 2020, we had 70 full time employees.

As our development, manufacturing and commercialization plans and strategies develop, and as we continue to operate as a public company, we expect to need and are actively recruiting additional managerial, operational, sales, marketing, financial and other personnel.

39 unchanged sentences

We will be required to disclose changes made in our internal controls and procedures on a quarterly basis and our management will be required to assess the effectiveness of these controls annually.

However, for as long as we are an “emerging growth company” under the Jumpstart Our Business Startups Act, or the JOBS Act, enacted in April 2012, our independent registered public accounting firm will not be required to attest to the effectiveness of our internal controls over financial reporting pursuant to Section 404 of the Sarbanes-Oxley Act.

However, for as long as we are an “emerging growth company”

under the Jumpstart Our Business Startups Act, or the JOBS Act, enacted in April 2012, our independent registered public accounting firm will not be required to attest to the effectiveness of our internal controls over financial reporting pursuant to Section 404 of the Sarbanes-Oxley Act.

We could be an “emerging growth company” for up to five years.

8 unchanged sentences

In addition, there is a risk that one or more of our current service providers, manufacturers and other partners may not survive these difficult economic times, which could directly affect our ability to attain our operating goals on schedule and on budget.

At June 30, 2020, we had $176.5 million of cash, cash equivalents and marketable securities.

While we are not aware of any downgrades, material losses, or other significant deterioration in the fair value of our cash equivalents or marketable securities since June 30, 2020, no assurance can be given that further deterioration of the global credit and financial markets would not negatively impact our current portfolio of cash equivalents or our ability to meet our financing objectives.

At September 30, 2020, we had $161.7 million of cash, cash equivalents and marketable securities.

While we are not aware of any downgrades, material losses, or other significant deterioration in the fair value of our cash equivalents or marketable securities since September 30, 2020, no assurance can be given that further deterioration of the global credit and financial markets would not negatively impact our current portfolio of cash equivalents or our ability to meet our financing objectives.

Furthermore, our stock price may decline due in part to the volatility of the stock market and the general economic downturn.

16 unchanged sentences

marketing or other prohibited activities.

For example, patients may use social media channels to comment on their experience in an ongoing blinded clinical trial or to report an alleged adverse event.

For example, patients may use social

media channels to comment on their experience in an ongoing blinded clinical trial or to report an alleged adverse event.

When such disclosures occur, there is a risk that trial enrollment may be adversely impacted, we fail to monitor and comply with applicable adverse event reporting obligations or that we may not be able to defend our business or the public’s legitimate interests in the face of the political and market pressures generated by social media due to restrictions on what we may say about our product candidates.

12 unchanged sentences

In particular, the promotion, sales and marketing of healthcare items and services, as well as certain business arrangements in the healthcare industry, are subject to extensive laws designed to prevent fraud, kickbacks, self-dealing and other abusive practices.

These laws and regulations may restrict or prohibit a wide range of pricing, discounting, marketing and promotion, structuring and commission(s), certain customer incentive programs and other business

arrangements generally.

Activities subject to these laws also involve the improper use of information obtained in the course of patient recruitment for clinical trials.

20 unchanged sentences

State laws may require pharmaceutical or medical device companies to comply with the industry’s voluntary compliance guidelines and the applicable compliance guidance promulgated by the federal government or otherwise restrict payments that may be made to healthcare providers and other potential referral sources.

State and local laws may

also require the licensure of sales representatives, and require drug or device manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures and pricing information.

State and local laws may also require the licensure of sales representatives, and require drug or device manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures and pricing information.

Further data privacy and security laws and regulations in foreign jurisdictions that may be more stringent than those in the U.S.

172 unchanged sentences

However, while we remain an emerging growth company, we will not be required to include an attestation report on internal control over financial reporting issued by our independent registered public accounting firm.

We conduct a process each year to document and evaluate our internal control over financial reporting, which is both costly and challenging.

We conduct a process each year to document and evaluate our internal control over financial reporting, which is both costly and

In this regard, we dedicate internal resources, engage outside consultants and adopt a detailed work plan to assess and document the adequacy of internal control over financial reporting, continue steps to improve control processes as appropriate, validate through testing that controls are functioning as documented and implement a continuous reporting and improvement process for internal control over financial reporting.

8 unchanged sentences

Concentration of ownership of our common stock among our existing executive officers, directors and principal stockholders may prevent new investors from influencing significant corporate decisions.

Our executive officers, directors and principal stockholders, together with their respective affiliates, beneficially owned approximately 62% of our capital stock as of June 30, 2020.

Our executive officers, directors and principal stockholders, together with their respective affiliates, beneficially owned approximately 62% of our capital stock as of September 30, 2020.

This concentration of ownership control could delay, defer or prevent a change in control, entrench our management or the board of directors, or impede a merger, consolidation, takeover or other business combination involving us that other stockholders may desire.

39 unchanged sentences

We do not currently have and may never obtain research coverage by securities and industry analysts.

If no or few securities or industry analysts commence coverage of us, or one or more of the analysts who cover us issues an adverse opinion about our company, our stock price would likely decline.

If no or few securities or industry analysts commence coverage of us, or one or more of the analysts who cover us issues an

adverse opinion about our company, our stock price would likely decline.

If one or more of these analysts ceases research coverage of us or fails to regularly publish reports on us, we could lose visibility in the financial markets, which in turn could cause our stock price or trading volume to decline.

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