QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
(State or other jurisdiction of incorporation or organization) |
(I.R.S. Employer Identification No.) | |
(Address of principal executive offices) |
(Zip Code) |
Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered | ||
Large accelerated filer |
☐ |
Accelerated filer |
☐ | |||
Non-accelerated filer |
☒ |
Smaller reporting company |
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Emerging growth company |
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• |
The novel coronavirus, or COVID-19, pandemic has caused, and could continue to cause, severe disruption 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 employees, business, financial condition and results of operations. |
• |
We are a clinical stage company with a limited operating history, have incurred significant losses since our inception, and anticipate that we will continue to incur significant losses for the foreseeable future. |
• |
We have no products approved for commercial sale and have not generated any revenue from product sales. If we are unable to raise additional capital when needed or on terms acceptable to us, we could be forced to significantly delay, scale back or discontinue our development or commercialization efforts. |
• |
Although we have initiated and conducted clinical trials for some of our product candidates, including MGTA-145, we have not yet demonstrated the ability to successfully advance our clinical trials for our product candidates through the final regulatory processes and obtain marketing approvals for such products or conduct sales and marketing activities necessary for successful commercialization. |
• |
If we are unable to obtain regulatory approval for MGTA-145, MGTA-117 or any other product candidates that we may identify or develop, our business will be substantially harmed. |
• |
We have not yet demonstrated an ability to manufacture or process drug product on a commercial-scale and may not be able to do so for any of our product candidates. |
• |
The results of earlier studies and interim data from our ongoing studies may not be predictive of future clinical trial results, and we may fail to establish an adequate safety or efficacy profile to conduct advanced clinical trials or obtain regulatory approval for MGTA-145, MGTA-117 or any other product candidates that we may pursue. |
• |
Stem cell transplant is a high-risk procedure that may result in complications or adverse events for patients in our clinical trials or for patients that use any of our product candidates, if approved. If serious adverse events, undesirable side effects, or unexpected characteristics are identified during the development of any of our product candidates, we may need to limit, delay or abandon our further clinical development of those product candidates, even if such events, effects or characteristics were the result of stem cell transplant or related procedures generally, and not directly or specifically caused or exacerbated by our product candidates. |
• |
If we are not able to identify a safe and effective dose for any of our antibody-drug conjugates, or ADCs, we may need to delay, abandon or limit our development of any potential product candidates. |
• |
We rely, and expect to continue to rely, on third parties to manufacture our clinical product supplies, and we intend to rely on third parties to produce and process our product candidates, if approved. |
• |
If we are unable to successfully develop our current programs into a comprehensive portfolio of product candidates, or experience significant delays in doing so, we may not realize the full commercial potential of our current and future product candidates. |
• |
We are developing E478 specifically to partner with gene therapy and genome editing companies, and if we are unable to find willing collaborators, this may adversely affect the development of E478 and our business. |
• |
The commercial success of any of our product candidates will depend upon the degree of market acceptance by physicians, patients, third-party payors and others in the medical community. |
• |
We face substantial competition, including from companies with greater financial, technical, research, manufacturing, marketing, distribution and other resources than us, which may result in others discovering, developing or commercializing products before or more successfully than we do. |
• |
We have entered into collaborations and may enter into additional collaborations, strategic alliances or additional licensing arrangements in the future, and we may not realize the benefits of such collaborations, alliances or licensing arrangements. |
• |
We are highly dependent on intellectual property licensed from third parties and termination of any of these licenses could result in the loss of significant rights, which would harm our business. |
• |
It is difficult and costly to protect our intellectual property and our proprietary technologies, and we may not be able to ensure their protection. If we are unable to obtain and maintain sufficient intellectual property protection for MGTA-145, MGTA-117 or any of our other current or any future product candidates, or our technologies, we may not be able to compete effectively in our markets. |
• |
Our future success depends in part upon our ability to attract and retain highly skilled personnel, including the members of our executive team and key scientific and medical personnel employees. |
• |
Changes in tax law could adversely affect our business and financial condition. |
Item 1. |
Financial Statements. |
September 30, 2021 |
December 31, 2020 |
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Assets |
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Current assets: |
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Cash and cash equivalents |
$ | $ | ||||||
Marketable securities |
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Prepaid expenses and other current assets |
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Total current assets |
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Restricted cash |
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Property and equipment, net |
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Total assets |
$ | $ | ||||||
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Liabilities and Stockholders’ Equity |
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Current liabilities: |
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Accounts payable |
$ | $ | ||||||
Accrued expenses and other current liabilities |
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Total current liabilities |
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Deferred rent |
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Total liabilities |
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Commitments and contingencies (Note 6) |
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Stockholders’ Equity: |
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Preferred stock, $ |
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Common stock, $ |
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Additional paid-in capital |
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Accumulated other comprehensive income (loss) |
( |
) | ||||||
Accumulated deficit |
( |
) | ( |
) | ||||
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Total stockholders’ equity |
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Total liabilities and stockholders’ equity |
$ | $ | ||||||
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Three Months Ended September 30, |
Nine Months Ended September 30, |
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2021 |
2020 |
2021 |
2020 |
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Operating expenses: |
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Research and development |
$ | $ | $ | |||||||||||||
General and administrative |
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Total operating expenses |
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Loss from operations |
( |
) | ( |
) | ( |
) | ( |
) | ||||||||
Interest and other income, net |
||||||||||||||||
Net loss |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
Net loss per share, basic and diluted |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
Weighted average common shares outstanding, basic and diluted |
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Comprehensive loss: |
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Net loss |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
Other comprehensive income (loss): |
||||||||||||||||
Unrealized gains (losses) on marketable securities |
( |
) | ( |
) | ||||||||||||
Total other comprehensive income (loss) |
( |
) | ( |
) | ||||||||||||
Total comprehensive loss |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
Accumulated |
||||||||||||||||||||||||
Additional |
Other |
Total |
||||||||||||||||||||||
Common Stock |
Paid-in |
Comprehensive |
Accumulated |
Stockholders’ |
||||||||||||||||||||
Shares |
Amount |
Capital |
Income (Loss) |
Deficit |
Equity |
|||||||||||||||||||
Three Months Ended September 30, 2021 |
||||||||||||||||||||||||
Balances at June 30, 2021 |
$ |
$ |
$ |
$ |
( |
) |
$ |
|||||||||||||||||
Vesting of restricted stock |
— |
— |
— |
— |
— |
|||||||||||||||||||
Issuance of common stock upon exercise of stock options |
— |
— |
— |
|||||||||||||||||||||
Stock-based compensation expense |
— |
— |
— |
— |
||||||||||||||||||||
Unrealized losses on marketable securities |
— |
— |
— |
( |
) |
— |
( |
) | ||||||||||||||||
Net loss |
— |
— |
— |
— |
( |
) |
( |
) | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
Balances at September 30, 2021 |
$ |
$ |
$ |
$ |
( |
) |
$ |
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|
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|
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, 2020 |
||||||||||||||||||||||||
Balances at June 30, 2020 |
$ |
$ |
$ |
$ |
( |
) |
$ |
|||||||||||||||||
Vesting of restricted stock |
— |
— |
— |
— |
— |
|||||||||||||||||||
Issuance of common stock upon exercise of stock options |
— |
— |
— |
|||||||||||||||||||||
Stock-based compensation expense |
— |
— |
— |
— |
||||||||||||||||||||
Unrealized losses on marketable securities |
— |
— |
— |
( |
) |
— |
( |
) | ||||||||||||||||
Net loss |
— |
— |
— |
— |
( |
) |
( |
) | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
Balances at September 30, 2020 |
$ |
$ |
$ |
$ |
( |
) |
$ |
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|
|
|
|
|
|
|
|
|
|
|
|
Accumulated |
||||||||||||||||||||||||
Additional |
Other |
Total |
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Common Stock |
Paid-in |
Comprehensive |
Accumulated |
Stockholders’ |
||||||||||||||||||||
Shares |
Amount |
Capital |
Income (Loss) |
Deficit |
Equity |
|||||||||||||||||||
Nine Months Ended September 30, 2021 |
||||||||||||||||||||||||
Balances at December 31, 2020 |
$ |
$ |
$ |
( |
) |
$ |
( |
) |
$ |
|||||||||||||||
Issuance of common stock upon private investment, net of offering costs |
— |
— |
||||||||||||||||||||||
Vesting of restricted stock |
— |
— |
— |
— |
— |
|||||||||||||||||||
Issuance of common stock upon exercise of stock options |
— |
— |
— |
|||||||||||||||||||||
Issuance of common stock under Employee Stock Purchase Plan |
— |
— |
— |
|||||||||||||||||||||
Stock-based compensation expense |
— |
— |
— |
— |
||||||||||||||||||||
Unrealized gains on marketable securities |
— |
— |
— |
— |
||||||||||||||||||||
Net loss |
— |
— |
— |
— |
( |
) |
( |
) | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
Balances at September 30, 2021 |
$ |
$ |
$ |
$ |
( |
) |
$ |
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|
|
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|
|
|
|
|
|
|
Nine Months Ended September 30, 2020 |
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Balances at December 31, 2019 |
$ |
$ |
$ |
$ |
( |
) |
$ |
|||||||||||||||||
Issuance of common stock upon public offering net of underwriting discounts, commissions and offering costs |
— |
— |
||||||||||||||||||||||
Vesting of restricted stock |
— |
— |
— |
— |
— |
|||||||||||||||||||
Issuance of common stock upon exercise of stock options |
— |
— |
— |
|||||||||||||||||||||
Issuance of common stock under Employee Stock Purchase Plan |
— |
— |
— |
|||||||||||||||||||||
Stock-based compensation expense |
— |
— |
— |
— |
||||||||||||||||||||
Unrealized gains on marketable securities |
— |
— |
— |
— |
||||||||||||||||||||
Net loss |
— |
— |
— |
— |
( |
) |
( |
) | ||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||
Balances at September 30, 2020 |
$ |
$ |
$ |
$ |
( |
) |
$ |
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|
|
|
|
|
|
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|
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|
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|
Nine months ended September 30, |
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2021 |
2020 |
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Cash flows from operating activities: |
||||||||
Net loss |
$ | ( |
) | $ | ( |
) | ||
Adjustments to reconcile net loss to net cash used in operating activities: |
||||||||
Stock-based compensation expense |
||||||||
Depreciation and amortization expense |
||||||||
Loss on disposal of property and equipment |
||||||||
Net amortization of premiums on marketable securities |
||||||||
Changes in operating assets and liabilities: |
||||||||
Prepaid expenses and other current assets |
( |
) | ||||||
Accounts payable |
( |
) | ( |
) | ||||
Accrued expenses and other current liabilities |
( |
) | ||||||
Deferred rent |
||||||||
|
|
|
|
|||||
Net cash used in operating activities |
( |
) | ( |
) | ||||
|
|
|
|
|||||
Cash flows from investing activities: |
||||||||
Purchases of property and equipment |
( |
) | ( |
) | ||||
Purchases of marketable securities |
— | ( |
) | |||||
Maturities of marketable securities |
||||||||
|
|
|
|
|||||
Net cash provided by investing activities |
||||||||
|
|
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|
|||||
Cash flows from financing activities: |
||||||||
Proceeds from private investment |
— | |||||||
Proceeds from public offerings, net of underwriting discounts and commissions |
— | |||||||
Payments of offering costs |
( |
) | ( |
) | ||||
Proceeds from exercise of common stock options |
||||||||
Proceeds from issuance of common stock under Employee Stock Purchase Plan |
||||||||
|
|
|
|
|||||
Net cash provided by financing activities |
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|
|
|
|
|||||
Net increase in cash, cash equivalents and restricted cash |
||||||||
Cash, cash equivalents and restricted cash at beginning of period |
||||||||
|
|
|
|
|||||
Cash, cash equivalents and restricted cash at end of period |
$ | $ | ||||||
|
|
|
|
|||||
Supplemental disclosure of non-cash investing and financing activities: |
||||||||
Offering costs included in accounts payable and accrued expenses |
$ | — | $ |
• |
Level 1—Quoted prices in active markets for identical assets or liabilities. |
• |
Level 2—Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data. |
• |
Level 3—Unobservable inputs that are supported by little or no market activity and that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques. |
As of September 30, |
||||||||
2021 |
2020 |
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Stock options to purchase common stock |
||||||||
Unvested restricted common stock and units |
||||||||
Shares of common stock issuable under Employee Stock Purchase Plan |
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|
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|
Gross |
Gross |
Estimated |
||||||||||||||
Amortized |
Unrealized |
Unrealized |
Fair |
|||||||||||||
Cost |
Gains |
Losses |
Value |
|||||||||||||
U.S. treasury notes (due within one year) |
$ | $ | $ | — | $ | |||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total |
$ | $ | $ | — | $ | |||||||||||
|
|
|
|
|
|
|
|
Gross |
Gross |
Estimated |
||||||||||||||
Amortized |
Unrealized |
Unrealized |
Fair |
|||||||||||||
Cost |
Gains |
Losses |
Value |
|||||||||||||
U.S. treasury notes (due within one year) |
$ |
$ |
— |
$ |
( |
) |
$ |
|||||||||
|
|
|
|
|
|
|
|
|||||||||
Total |
$ |
$ |
— |
$ |
( |
) |
$ |
|||||||||
|
|
|
|
|
|
|
|
Fair Value Measurements at September 30, 2021 Using: |
||||||||||||||||
Level 1 |
Level 2 |
Level 3 |
Total |
|||||||||||||
Cash equivalents: |
||||||||||||||||
Money market funds |
$ |
$ |
— |
$ |
— |
$ |
||||||||||
Marketable securities: |
||||||||||||||||
U.S. treasury notes |
— |
— |
||||||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total |
$ |
$ |
$ |
— |
$ |
|||||||||||
|
|
|
|
|
|
|
|
Fair Value Measurements at December 31, 2020 Using: |
||||||||||||||||
Level 1 |
Level 2 |
Level 3 |
Total |
|||||||||||||
Cash equivalents: |
||||||||||||||||
Money market funds |
$ | $ | — | $ | — | $ | ||||||||||
U.S. treasury notes |
— | — | ||||||||||||||
Marketable securities: |
||||||||||||||||
U.S. treasury notes |
— | — | ||||||||||||||
Total |
$ | $ | $ | — | $ | |||||||||||
September 30, 2021 |
December 31, 2020 |
|||||||
Accrued external research and development expenses |
$ | $ | ||||||
Accrued payroll and related expenses |
||||||||
Accrued professional fees |
||||||||
Deferred rent, current portion |
||||||||
Accrued other |
||||||||
$ | $ | |||||||
Three Months Ended September 30, |
Nine Months Ended September 30, |
|||||||||||||||
2021 |
2020 |
2021 |
2020 |
|||||||||||||
Research and development expenses |
$ | $ | $ | $ | ||||||||||||
General and administrative expenses |
||||||||||||||||
$ | $ | $ | $ | |||||||||||||
Remainder of 2021 (three months) |
$ | |||
2022 |
||||
2023 |
||||
2024 |
||||
2025 |
||||
Thereafter |
||||
$ | ||||
Item 2. |
Management’s Discussion and Analysis of Financial Condition and Results of Operations. |
• | the initiation, timing and success of clinical trials of MGTA-145, MGTA-117 and any other product candidates; |
• | the outcomes of our preclinical studies; |
• | our ability to commence and enroll patients in our clinical trials at the pace that we project; |
• | whether the results of our trials will be sufficient to support domestic or foreign regulatory approvals for MGTA-145, MGTA-117 or any other product candidates we may develop; |
• | our ability to establish clinical programs moving forward in multiple indications, with a rapidly advancing portfolio and sustainable platform; |
• | regulatory actions with respect to our product candidates or our competitors’ products and product candidates |
• | our ability to obtain, including on an expedited basis, and maintain regulatory approval of MGTA-145, MGTA-117 or any other product candidates we may develop; |
• | the level of expenses related to any of our product candidates or clinical development programs; |
• | our expectation that our existing capital resources will be sufficient to enable us to fund our planned development of MGTA-145, MGTA-117 and any other product candidates we may identify and pursue; |
• | the benefits of the use of MGTA-145, MGTA-117 or any other product candidate, if approved; |
• | our ability to successfully commercialize MGTA-145, MGTA-117 or any other product candidates we may identify and pursue, if approved; |
• | our ability to successfully find collaborators for E478 or any of our current and future programs and product candidates; |
• | the rate and degree of market acceptance of MGTA-145, MGTA-117 or any other product candidates we may identify and pursue; |
• | our ability to obtain orphan drug designation for any of our product candidates we may identify and pursue; |
• | our expectations regarding government and third-party payor coverage and reimbursement; |
• | our ability to manufacture MGTA-145, MGTA-117 or any other product candidate in conformity with the U.S. Food and Drug Administration’s requirements and to scale up manufacturing of our product candidates to commercial scale, if approved; |
• | our ability to successfully build a specialty sales force and commercial infrastructure; |
• | our ability to compete with companies currently producing or engaged in the clinical development of treatments for the disease indications that we pursue and treatment modalities that we develop; |
• | our reliance on third parties to conduct our clinical trials; |
• | our reliance on third-party contract manufacturers to manufacture and supply our product candidates for us; |
• | our ability to retain and recruit key personnel; |
• | our ability to obtain and maintain intellectual property protection for MGTA-145, MGTA-117 or any other product candidates we may identify and pursue; |
• | our estimates of our expenses, ongoing losses, future revenue, capital requirements and our needs for or ability to obtain additional financing; |
• | our expectations regarding the time during which we will continue to be an emerging growth company or smaller reporting company as defined in federal securities regulations; |
• | our financial performance; and |
• | developments and projections relating to our competitors or our industry. |
• | initiate, enroll and conduct Phase 2 clinical trials for MGTA-145 and Phase 1/2 clinical trial for MGTA-117; |
• | initiate and conduct preclinical studies and clinical trials of our other product candidates; |
• | develop any other future product candidates we may choose to pursue; |
• | seek marketing approval for any of our product candidates that successfully complete clinical development, if any; |
• | maintain compliance with applicable regulatory requirements; |
• | develop and scale up our capabilities to support our ongoing preclinical activities and clinical trials for our product candidates and commercialization of any of our product candidates for which we obtain marketing approval, if any; |
• | maintain, expand, protect and enforce our intellectual property portfolio; |
• | develop and expand our sales, marketing and distribution capabilities for our product candidates for which we obtain marketing approval, if any; and |
• | expand our operational, financial and management systems and increase personnel, including to support our clinical development and commercialization efforts and our operations as a public company. |
• | employee-related expenses, including salaries and related costs, and stock-based compensation expense, for employees engaged in research and development functions; |
• | expenses incurred in connection with the preclinical and clinical development of our product candidates, including under agreements with contract research organizations, or CROs; |
• | the cost of consultants and contract manufacturing organizations, or CMOs, that manufacture drug products for use in our preclinical studies and clinical trials; |
• | facilities, depreciation and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance and supplies; and |
• | payments made under third-party licensing agreements. |
• | the continuing impact of the COVID-19 pandemic on our industry, the healthcare system, and our current and future operations; |
• | successful completion of preclinical studies and clinical trials; |
• | receipt and related terms of marketing approvals from applicable regulatory authorities; |
• | raising additional funds necessary to complete clinical development of and commercialize our product candidates; |
• | obtaining and maintaining patent, trade secret and other intellectual property protection and regulatory exclusivity for our product candidates; |
• | making arrangements with third-party manufacturers, or establishing manufacturing capabilities, for both clinical and commercial supplies of our product candidates; |
• | developing and implementing marketing and reimbursement strategies; |
• | establishing sales, marketing and distribution capabilities and launching commercial sales of our products, if and when approved, whether alone or in collaboration with others; |
• | acceptance of our products, if and when approved, by patients, the medical community and third-party payors; |
• | effectively competing with other therapies; |
• | obtaining and maintaining third-party coverage and adequate reimbursement; |
• | protecting and enforcing our rights in our intellectual property portfolio; and |
• | maintaining a continued acceptable safety profile of the products following approval. |
• | accrued research and development expenses; and |
• | stock-based compensation. |
Three Months Ended September 30, |
||||||||||||
2021 |
2020 |
Change |
||||||||||
(in thousands) |
||||||||||||
Operating expenses: |
||||||||||||
Research and development |
$ | 10,795 | $ | 11,786 | $ | (991 | ) | |||||
General and administrative |
7,450 | 6,595 | 855 | |||||||||
|
|
|
|
|
|
|||||||
Total operating expenses |
18,245 | 18,381 | (136 | ) | ||||||||
|
|
|
|
|
|
|||||||
Loss from operations |
(18,245 | ) | (18,381 | ) | 136 | |||||||
Interest and other income, net |
818 | 703 | 115 | |||||||||
|
|
|
|
|
|
|||||||
Net loss |
$ | (17,427 | ) | $ | (17,678 | ) | $ | 251 | ||||
|
|
|
|
|
|
Three Months Ended September 30, |
||||||||||||
2021 |
2020 |
Change |
||||||||||
(in thousands) |
||||||||||||
Direct research and development expenses by program: |
||||||||||||
Conditioning |
$ | 1,510 | $ | 3,926 | $ | (2,416 | ) | |||||
Mobilization |
973 | 1,029 | (56 | ) | ||||||||
Cell Therapy |
42 | 417 | (375 | ) | ||||||||
Unallocated expenses: |
||||||||||||
Personnel related (including stock-based compensation) |
4,886 | 3,704 | 1,182 | |||||||||
Consultant (including stock-based compensation) |
544 | 320 | 224 | |||||||||
Facility related and other |
2,840 | 2,390 | 450 | |||||||||
|
|
|
|
|
|
|||||||
Total research and development expenses |
$ | 10,795 | $ | 11,786 | $ | (991 | ) | |||||
|
|
|
|
|
|
Three Months Ended September 30, |
||||||||||||
2021 |
2020 |
Change |
||||||||||
(in thousands) |
||||||||||||
Personnel related (including stock-based compensation) |
$ | 4,213 | $ | 3,539 | $ | 674 | ||||||
Professional and consultant |
1,317 | 1,524 | (207 | ) | ||||||||
Facility related and other |
1,920 | 1,532 | 388 | |||||||||
|
|
|
|
|
|
|||||||
Total general and administrative expenses |
$ | 7,450 | $ | 6,595 | $ | 855 | ||||||
|
|
|
|
|
|
Nine Months Ended September 30, |
||||||||||||
2021 |
2020 |
Change |
||||||||||
(in thousands) |
||||||||||||
Operating expenses: |
||||||||||||
Research and development |
$ | 33,652 | $ | 38,359 | $ | (4,707 | ) | |||||
General and administrative |
20,900 | 21,278 | (378 | ) | ||||||||
|
|
|
|
|
|
|||||||
Total operating expenses |
54,552 | 59,637 | (5,085 | ) | ||||||||
|
|
|
|
|
|
|||||||
Loss from operations |
(54,552 | ) | (59,637 | ) | 5,085 | |||||||
Interest and other income, net |
2,708 | 2,869 | (161 | ) | ||||||||
|
|
|
|
|
|
|||||||
Net loss |
$ | (51,844 | ) | $ | (56,768 | ) | $ | 4,924 | ||||
|
|
|
|
|
|
Nine Months Ended September 30, |
||||||||||||
2021 |
2020 |
Change |
||||||||||
(in thousands) |
||||||||||||
Direct research and development expenses by program: |
||||||||||||
Conditioning |
$ | 6,028 | $ | 11,876 | $ | (5,848 | ) | |||||
Mobilization |
3,265 | 3,049 | 216 | |||||||||
Cell Therapy |
676 | 4,016 | (3,340 | ) | ||||||||
Unallocated expenses: |
||||||||||||
Personnel related (including stock-based compensation) |
14,193 | 11,120 | 3,073 | |||||||||
Consultant (including stock-based compensation) |
1,001 | 960 | 41 | |||||||||
Facility related and other |
8,489 | 7,338 | 1,151 | |||||||||
|
|
|
|
|
|
|||||||
Total research and development expenses |
$ | 33,652 | $ | 38,359 | $ | (4,707 | ) | |||||
|
|
|
|
|
|
Nine Months Ended September 30, |
||||||||||||
2021 |
2020 |
Change |
||||||||||
(in thousands) |
||||||||||||
Personnel related (including stock-based compensation) |
$ | 10,508 | $ | 11,097 | $ | (589 | ) | |||||
Professional and consultant |
4,785 | 5,412 | (627 | ) | ||||||||
Facility related and other |
5,607 | 4,769 | 838 | |||||||||
|
|
|
|
|
|
|||||||
Total general and administrative expenses |
$ | 20,900 | $ | 21,278 | $ | (378 | ) | |||||
|
|
|
|
|
|
Nine Months Ended September 30, |
||||||||
2021 |
2020 |
|||||||
(in thousands) |
||||||||
Cash used in operating activities |
$ | (43,841 | ) | $ | (50,290 | ) | ||
Cash provided by investing activities |
53,751 | 60,339 | ||||||
Cash provided by financing activities |
89,477 | 66,560 | ||||||
|
|
|
|
|||||
Net increase in cash, cash equivalents and restricted cash |
$ | 99,387 | $ | 76,609 | ||||
|
|
|
|
Item 3. |
Quantitative and Qualitative Disclosures about Market Risks. |
Item 4. |
Controls and Procedures. |
Item 1. |
Legal Proceedings. |
Item 1A. |
Risk Factors. |
• | The COVID-19 pandemic has had, and will likely continue to have, an adverse impact on various aspects of our ongoing clinical trials, including our investigator-initiated trial, and on preclinical studies and clinical trials, including investigator-initiated trials. For example, we are staggering the initiation of our Phase 2 trials for MGTA-145 over the course of 2021 due to the clinical trial impacts from COVID-19. |
• | Other potential impacts of the COVID-19 pandemic on our various clinical trials include impacts on patient dosing and study monitoring, which may be paused or delayed due to changes in policies at various clinical sites; federal, state, local or foreign laws, rules and regulations, including quarantines or other travel restrictions; the prioritization of healthcare resources toward pandemic efforts, including diminished attention from physicians serving as our clinical trial investigators and reduced availability of site staff supporting the conduct of our clinical trials; and interruption or delays in the operations of the U.S. Food and Drug Administration, or FDA, among other reasons related to the COVID-19 pandemic. If the COVID-19 pandemic continues, other aspects of our clinical trials will likely be adversely affected, delayed or interrupted, including, for example, site initiation, patient recruitment and enrollment, availability of clinical trial materials and data analysis. Some patients and clinical investigators may not be able to comply with clinical trial protocols and patients may choose to withdraw from our studies or we may choose to, or be required to, pause enrollment and or patient dosing in our ongoing clinical trials in order to preserve health resources and protect trial participants. It is unknown how long these pauses or disruptions could continue. |
• | We currently rely on third parties, including our contract research organizations, or CROs, and our contract manufacturing organizations, or CMOs, and other contractors and consultants to, among other things, conduct our preclinical and clinical trials, manufacture raw materials, manufacture and supply our product candidates, ship investigational drugs and clinical trial samples, perform quality testing and supply other goods and services to run our business. If any such third party is adversely impacted by restrictions resulting from the COVID-19 pandemic, including staffing shortages, production slowdowns and disruptions in delivery systems, our supply chain may be disrupted, which could limit our ability to manufacture our product candidates for our clinical trials and conduct our research and development operations. |
• | We have established a work-from-home policy for all employees, as well as safety measures for those using our offices and laboratory facilities that are designed to comply with applicable federal, state and local guidelines instituted in response to the COVID-19 pandemic. Our increased reliance on personnel working from home may negatively impact productivity, or disrupt, delay, or otherwise adversely impact our business. In addition, this could increase our cyber security risk, create data accessibility concerns and make us more susceptible to communication disruptions, any of which could adversely impact our business operations or delay necessary interactions with local and federal regulators, ethics committees, manufacturing sites, research or clinical trial sites and other important agencies and contractors. |
• | Our employees and contractors conducting non-business critical research and development activities have not been able to, and may not in the future be able to, access our laboratory for an extended period of time as a result of the current work-from-home policy and the possibility that governmental authorities further modify current restrictions. This could delay timely completion of preclinical activities, including completing Investigational New Drug, or IND, enabling studies or our ability to select future development candidates, and initiation of additional clinical trials for our other product candidates. |
• | Certain government agencies, such as health regulatory agencies and patent offices, within the U.S. or internationally have experienced, and may continue to experience, disruptions in their operations as a result of the COVID-19 pandemic. The FDA and comparable foreign regulatory agencies may have slower response times or be under-resourced to continue to monitor our clinical trials and, as a result, review, inspection and other timelines may be materially delayed. It is unknown how long these disruptions could continue. Any elongation or de-prioritization of our clinical trials or delay in regulatory review resulting from such disruptions could materially affect the development and study of our product candidates. For example, regulatory authorities may require that we not distribute a product candidate lot until the relevant agency authorizes its release. Such release authorization may be delayed as a result of the COVID-19 pandemic, which would likely result in delays to our ongoing clinical trials. |
• | The trading prices for our common stock and those of other biopharmaceutical companies have been highly volatile, partly due to the COVID-19 pandemic. As a result, we may face difficulties raising capital through sales of our common stock or such sales may be on unfavorable terms. In addition, a recession, depression or other sustained adverse market event resulting from the COVID-19 pandemic could materially and adversely affect our business and the value of our common stock. |
• | the initiation, progress, timing, costs and results of preclinical studies and clinical trials for our product candidates; |
• | the clinical development plans we establish for these product candidates; |
• | the number and characteristics of product candidates that we develop or may in-license; |
• | the cost of milestone or other payments under any current or future license, acquisition, collaboration or other strategic transaction agreements; |
• | the outcome, timing and cost of meeting regulatory requirements established by the FDA, the European Medical Agency, or EMA, and other comparable foreign regulatory authorities; |
• | the cost of filing, prosecuting, defending and enforcing our patent claims and other intellectual property rights; |
• | the cost of defending intellectual property disputes, including patent infringement actions brought by third parties against us or our product candidates; |
• | the effect of competing technological and market developments; |
• | the cost and timing of completion of commercial-scale outsourced manufacturing activities; |
• | the cost of seeking to attract, hire and retain skilled personnel; and |
• | the cost of establishing sales, marketing and distribution capabilities for any product candidates for which we may receive regulatory approval in regions where we choose to commercialize our products on our own. |
• | identify product candidates and complete research and preclinical and clinical development of any product candidates we may identify; |
• | seek and obtain regulatory and marketing approvals for any of our product candidates for which we complete clinical trials; |
• | launch and commercialize any of our product candidates for which we obtain regulatory and marketing approval by establishing a sales force, marketing, and distribution infrastructure or, alternatively, collaborating with a commercialization partner; |
• | qualify for adequate coverage and reimbursement by government and third-party payors for any of our product candidates for which we obtain regulatory and marketing approval; |
• | develop, maintain, and enhance a sustainable, scalable, reproducible, and transferable manufacturing process for the product candidates we may develop; |
• | establish and maintain supply and manufacturing relationships with third parties that can provide adequate, in both amount and quality, products and services to support clinical development and the market demand for any of our product candidates for which we obtain regulatory and marketing approval; |
• | obtain market acceptance of any product candidates we may develop as viable treatment options; |
• | address competing technological and market developments; |
• | implement internal systems and infrastructure, as needed; |
• | negotiate favorable terms in any collaboration, licensing, or other arrangements into which we may enter and perform our obligations in such collaborations; |
• | maintain, protect, and expand our portfolio of intellectual property rights, including patents, trade secrets, and know-how; |
• | avoid and defend against third-party interference or infringement claims; and |
• | attract, hire, and retain qualified personnel. |
• | successful completion of preclinical studies and successful enrollment and completion of clinical trials, including toxicology studies, biodistribution studies and minimally efficacious dose studies in animals, where applicable, under the FDA’s current Good Clinical Practices, or cGCPs, and the FDA’s current Good Laboratory Practices; |
• | effective IND applications or Clinical Trial Authorizations that allow commencement of our planned clinical trials or future clinical trials for our product candidates; |
• | positive results from our future clinical programs that support a finding of safety and effectiveness and an acceptable risk-benefit profile of our product candidates in the intended populations; |
• | receipt of regulatory approvals from applicable regulatory authorities; |
• | establishment of arrangements with third-party manufacturers for clinical supply and, where applicable, commercial manufacturing capabilities; |
• | successful development of our internal manufacturing processes or transfer to larger-scale facilities operated by either a CMO or by us; |
• | establishment and maintenance of patent and trade secret protection or regulatory exclusivity for our product candidates; |
• | commercial launch of our product candidates, if and when approved, whether alone or in collaboration with others; |
• | acceptance of our product candidates, if and when approved, by patients, the medical community and third-party payors; |
• | effective competition against other therapies; |
• | establishment and maintenance of healthcare coverage and adequate reimbursement; |
• | enforcement and defense of intellectual property rights and claims; and |
• | maintenance of a continued acceptable safety profile of our product candidates following approval. |
• | preclinical study results may show the therapies to be less effective than desired or to have harmful or problematic side effects; |
• | clinical trial results may show the therapies to be less effective than expected (e.g., the trial failed to meet its primary endpoint or the results are not competitive compared to other therapeutic alternatives) or to have unacceptable side effects or toxicities; |
• | failure to receive the necessary regulatory approvals or a delay in receiving such approvals, which delays may be caused by, among other things, slow enrollment in clinical trials, length of time to achieve study endpoints, additional time requirements for data analysis, or biologics license application, or BLA, preparation, discussions with the FDA, an FDA request for additional preclinical or clinical data, or unexpected safety or manufacturing issues; |
• | manufacturing costs, formulation issues, pricing or reimbursement issues, or other factors that make the therapy uneconomical; and |
• | the proprietary rights of others and their competing products and technologies that may prevent the therapy from being commercialized. |
• | regulators, IRBs, or ethics committees may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial at a prospective trial site; |
• | we may experience delays in reaching, or fail to reach, agreement on acceptable terms with prospective trial sites and prospective CROs, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites; |
• | clinical trials of any product candidates may fail to show safety or efficacy, produce negative or inconclusive results and we may decide, or regulators may require us, to conduct additional preclinical studies or clinical trials or we may decide to abandon product development programs; |
• | the number of patients required for clinical trials of any product candidates may be larger than we anticipate, enrollment in these clinical trials may be slower than we anticipate, or participants may drop out of these clinical trials or fail to return for post-treatment follow-up at a higher rate than we anticipate; |
• | our third-party contractors may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all, or may deviate from the clinical trial protocol or drop out of the trial, which may require that we add new clinical trial sites or investigators; |
• | we may elect to, or regulators, IRBs or ethics committees may require, that we or our investigators, suspend or terminate clinical research or trials for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks; |
• | the cost of preclinical studies and clinical trials of any product candidates may be greater than we anticipate; |
• | the supply or quality of our product candidates or other materials necessary to conduct clinical trials of our product candidates may be insufficient or inadequate; and |
• | our product candidates may have undesirable side effects or other unexpected characteristics, causing us or our investigators, regulators, IRBs or ethics committees to suspend or terminate the trials, or reports may arise from preclinical or clinical testing of other blood and immune reset and cell-based therapies that raise safety or efficacy concerns about our product candidates. |
• | the patient eligibility criteria defined in the protocol; |
• | the size of the patient population required for analysis of the trial’s primary endpoints; |
• | the proximity of patients to trial sites; |
• | the design of the trial; |
• | our ability to recruit clinical trial investigators with the appropriate competencies and experience; |
• | our ability to obtain and maintain patient consents; and |
• | the risk that patients enrolled in clinical trials will drop out of the trials before completion. |
• | the research methodology used may not be successful in identifying potential indications and/or product candidates; |
• | potential product candidates may, after further study, be shown to have harmful adverse effects or other characteristics that indicate they are unlikely to be effective drugs; or |
• | it may take greater human and financial resources than we will possess to identify additional therapeutic opportunities for our product candidates or to develop suitable potential product candidates through internal research programs, thereby limiting our ability to develop, diversify and expand our product portfolio. |
• | reliance on the third party for regulatory compliance and quality assurance; |
• | the possible breach of the manufacturing agreement by the third party; |
• | the possible misappropriation of our proprietary information, including our trade secrets and know-how; and |
• | the possible termination or nonrenewal of the agreement by the third party at a time that is costly or inconvenient for us. |
• | the efficacy, durability and safety of such product candidates as demonstrated in clinical trials; |
• | the potential and perceived advantages of product candidates over alternative treatments; |
• | the cost of treatment relative to alternative treatments; |
• | our ability to offer the product for sale at competitive prices; |
• | the clinical indications for which the product candidate is approved by the FDA or the EMA; |
• | the product’s convenience and ease of administration compared to alternative treatments; |
• | the willingness of physicians to prescribe new therapies; |
• | the willingness of the target patient population to try new therapies; |
• | the prevalence and severity of any side effects; |
• | product labeling or product insert requirements of the FDA, EMA or other regulatory authorities, including any limitations or warnings contained in a product’s approved labeling; |
• | relative convenience and ease of administration; |
• | the strength of marketing and distribution support; |
• | the timing of market introduction of competitive products; |
• | publicity concerning our products or competing products and treatments; |
• | changes in the standard of care for the targeted indications for the product; and |
• | sufficient third-party payor coverage and adequate reimbursement. |
• | a covered benefit under its health plan; |
• | safe, effective and medically necessary; |
• | appropriate for the specific patient; |
• | cost-effective; and |
• | neither experimental nor investigational. |
• | decreased demand for our products; |
• | injury to our reputation; |
• | withdrawal of clinical trial participants and inability to continue clinical trials; |
• | initiation of investigations by regulators; |
• | costs to defend the related litigation; |
• | a diversion of management’s time and our resources; |
• | substantial monetary awards to trial participants or patients; |
• | product recalls, withdrawals or labeling, marketing or promotional restrictions; |
• | loss of revenue; |
• | exhaustion of any available insurance and our capital resources; |
• | the inability to commercialize any product candidate; and |
• | a decline in our share price. |
• | the scope of rights, if any, granted under the license agreement and other interpretation-related issues; |
• | whether and the extent to which our technology and processes infringe on intellectual property of the licensor that is not subject to the license agreement; |
• | whether our licensor or its licensor had the right to grant the license agreement; |
• | whether third parties are entitled to compensation or equitable relief, such as an injunction, for our use of the intellectual property without their authorization; |
• | our right to sublicense patent and other rights to third parties under collaborative development relationships; |
• | whether we are complying with our obligations with respect to the use of the licensed technology in relation to our development and commercialization of product candidates; |
• | our involvement in the prosecution of the licensed patents and our licensors’ overall patent enforcement strategy; |
• | the allocation of ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and by us and our partners; and |
• | the amounts of royalties, milestones or other payments due under the license agreement. |
• | the USPTO and various foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other provisions during the patent process. There are situations in which noncompliance can result in abandonment or lapse of a patent or patent application, resulting in partial or complete loss of patent rights in the relevant jurisdiction. In such an event, competitors might be able to enter the market earlier than would otherwise have been the case; |
• | patent applications may not result in any patents being issued; |
• | patents that may be issued or in-licensed may be challenged, invalidated, modified, revoked, circumvented, found to be unenforceable or otherwise may not provide any competitive advantage; |
• | our competitors, many of whom have substantially greater resources and many of whom have made significant investments in competing technologies, may seek or may have already obtained patents that will limit, interfere with or eliminate our ability to make, use, and sell our potential product candidates; |
• | there may be significant pressure on the U.S. government and international governmental bodies to limit the scope of patent protection both inside and outside the U.S. for disease treatments that prove successful, as a matter of public policy regarding worldwide health concerns; and |
• | countries other than the U.S. may have patent laws less favorable to patentees than those upheld by U.S. courts, allowing foreign competitors a better opportunity to create, develop and market competing product candidates. |
• | others may be able to make products that are similar to any product candidates we may develop or utilize similar technology but that are not covered by the claims of the patents that we license or own; |
• | we, or our current or future licensors might not have been the first to make the inventions covered by the issued patent or pending patent application that we license or own; |
• | we, or our current or future licensors might not have been the first to file patent applications covering certain of our or their inventions; |
• | others may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our owned or licensed intellectual property rights; |
• | it is possible that our pending owned or licensed patent applications or those that we may own or license in the future will not lead to issued patents; |
• | issued patents that we hold rights to may be held invalid or unenforceable, including as a result of legal challenges by our competitors; |
• | our competitors might conduct research and development activities in countries where we do not have patent rights and then use the information learned from such activities to develop competitive products for sale in our major commercial markets; |
• | we may not develop additional proprietary technologies that are patentable; |
• | the patents of others may harm our business; and |
• | we may choose not to file a patent in order to maintain certain trade secrets or know-how, and a third party may subsequently file a patent covering such intellectual property. |
• | Collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations. |
• | Collaborators may not pursue development and commercialization of any product candidates we may develop or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborator’s strategic focus or available funding or external factors such as an acquisition that diverts resources or creates competing priorities. |
• | Collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials, or require a new formulation of a product candidate for clinical testing. |
• | A collaborator’s product candidate may have a safety or efficacy profile that would impact the collaborator’s ability to continue to pursue the development and commercialization of its product candidate, which in turn, would negatively impact our ability to continue to pursue the development and commercialization of our product candidate. |
• | Collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our medicines or product candidates if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours. |
• | Collaborators with marketing and distribution rights to one or more medicines may not commit sufficient resources to the marketing and distribution of such medicine or medicines. |
• | Collaborators may not properly obtain, maintain, enforce, or defend our intellectual property or proprietary rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our proprietary information or expose us to potential litigation. |
• | Disputes may arise between the collaborators and us that result in the delay or termination of the research, development, or commercialization of our medicines or product candidates or that result in costly litigation or arbitration that diverts management attention and resources. |
• | We may lose certain valuable rights under circumstances identified in our collaborations, including if we undergo a change of control. |
• | Collaborations may be terminated and, if terminated, may result in a need for additional capital to pursue further development or commercialization of the applicable product candidates. |
• | Collaboration agreements may not lead to development or commercialization of product candidates in the most efficient manner or at all. If a present or future collaborator of ours were to be involved in a business combination, the continued pursuit and emphasis on our product development or commercialization program under such collaboration could be delayed, diminished, or terminated. |
• | identifying, recruiting, integrating, maintaining and motivating additional employees; |
• | managing our internal development efforts effectively, including the clinical, FDA and international regulatory review process for our product candidates, while complying with our contractual obligations to contractors and other third parties; and |
• | improving our operational, financial and management controls, reporting systems and procedures. |
• | the success of existing or new competitive products or technologies; |
• | regulatory actions with respect to our product candidates or our competitors’ products and product candidates; |
• | announcements by us or our competitors of significant acquisitions, strategic partnerships, joint ventures, collaborations or capital commitments; |
• | the timing and results of preclinical studies for any of our product candidates; |
• | the timing and results of clinical trials of MGTA-145 and any other product candidates; |
• | commencement or termination of collaborations for E478 or any of our current and future programs and product candidates; |
• | failure or discontinuation of any of our development programs; |
• | results of clinical trials of product candidates of our competitors; |
• | regulatory or legal developments in the U.S. and other countries; |
• | developments or disputes concerning patent applications, issued patents or other proprietary rights; |
• | the recruitment or departure of key personnel; |
• | the level of expenses related to any of our product candidates or clinical development programs; |
• | the results of our efforts to develop additional product candidates or products; |
• | actual or anticipated changes in estimates as to financial results or development timelines; |
• | announcement or expectation of additional financing efforts; |
• | sales of our common stock by us, our insiders or other stockholders; |
• | variations in our financial results or those of companies that are perceived to be similar to us; |
• | changes in estimates or recommendations by securities analysts, if any, that cover us; |
• | changes in the structure of healthcare payment systems; |
• | market conditions in the pharmaceutical and biotechnology sectors; |
• | disruptions to political, governmental or regulatory systems, including shutdowns of the government and its agencies; |
• | general economic, industry and market conditions; and |
• | the other factors described in this “Risk Factors” section. |
• | establish a classified board of directors such that all members of the board are not elected at one time; |
• | allow the authorized number of our directors to be changed only by resolution of our board of directors; |
• | limit the manner in which stockholders can remove directors from the board; |
• | establish advance notice requirements for nominations for election to the board of directors or for proposing matters that can be acted on at stockholder meetings; |
• | require that stockholder actions must be effected at a duly called stockholder meeting and prohibit actions by our stockholders by written consent; |
• | limit who may call a special meeting of stockholders; |
• | authorize our board of directors to issue preferred stock without stockholder approval, which could be used to institute a “poison pill” that would work to dilute the stock ownership of a potential hostile acquirer, effectively preventing acquisitions that have not been approved by our board of directors; and |
• | require the approval of the holders of at least 66.67% of the votes that all our stockholders would be entitled to cast to amend or repeal certain provisions of our charter or bylaws. |
• | The federal Anti-Kickback Statute, which prohibits, among other things, knowingly and willfully soliciting, receiving, offering, paying or providing any remuneration (including any kickback, bribe, or rebate), directly or indirectly, in cash or in kind, to induce, or in return for, either the referral of an individual, 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 a federal healthcare program, such as the Medicare and Medicaid programs. A person or entity can be found guilty of violating the statute without actual knowledge of the statute or specific intent to violate it. In addition, 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. The Anti-Kickback Statute has been interpreted to apply to arrangements between pharmaceutical manufacturers on the one hand and prescribers, purchasers, and formulary managers on the other. There are a number of statutory exceptions and regulatory safe harbors protecting some common activities from prosecution. |
• | Federal civil and criminal false claims laws and civil monetary penalty laws, including the False Claims Act, which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, false or fraudulent claims for payment to, or approval by Medicare, Medicaid, or other federal healthcare programs, knowingly making, using or causing to be made or used a false record or statement material to a false or fraudulent claim or an obligation to pay or transmit money to the federal government, or knowingly concealing or knowingly and improperly avoiding or decreasing an obligation to pay money to the federal government. Manufacturers can be held liable under the False Claims Act even when they do not submit claims directly to government payors if they are deemed to “cause” the submission of false or fraudulent claims. The False Claims Act also permits a private individual acting as a “whistleblower” to bring actions on behalf of the federal government alleging violations of the False Claims Act and to share in any monetary recovery. |
• | 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) 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. Similar to the federal Anti-Kickback Statute, a person or entity can be found guilty of violating HIPAA without actual knowledge of the statute or specific intent to violate it. |
• | HIPAA, as amended by HITECH, and their respective implementing regulations, which impose, among other things, requirements on certain covered healthcare providers, health plans, and healthcare clearinghouses as well as their respective business associates that perform services for them that involve the use, or disclosure of, individually identifiable health information, relating to the privacy, security and transmission of individually identifiable health information without appropriate authorization. HITECH also created new tiers of civil monetary penalties, amended HIPAA to make civil and criminal penalties directly applicable to business associates, 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 attorneys’ fees and costs associated with pursuing federal civil actions. |
• | The federal Physician Payment Sunshine Act of 2010, as amended by the Health Care and Education Reconciliation Act, which require manufacturers of drugs, devices, biologicals and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program (with certain exceptions) to report annually to CMS information related to any payments and other transfers of value made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors) and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate family members. Effective January 1, 2022, these reporting obligations will extend to include transfers of value made to certain non-physician providers (physician assistants, nurse practitioners, clinical nurse specialists, certified registered nurse anesthetists & anesthesiologist assistants, and certified nurse-midwives). |
• | Additional federal consumer protection and unfair competition laws, which broadly regulate marketplace activities and activities that potentially harm consumers. |
Item 2. |
Unregistered Sales of Equity Securities and Use of Proceeds |
(a) |
Recent Sales of Unregistered Securities |
(b) |
Use of Proceeds from Initial Public Offering |
(c) |
Issuer Purchases of Equity Securities |
Item 6. |
Exhibits. |
* | Filed herewith. |
** | Furnished herewith. |
MAGENTA THERAPEUTICS, INC. | ||||
Date: November 4, 2021 | By: | /s/ Stephen Mahoney | ||
Stephen Mahoney | ||||
Chief Financial and Operating Officer (Principal Financial and Accounting Officer) |
Exhibit 31.1
CERTIFICATION OF PRINCIPAL EXECUTIVE OFFICER PURSUANT TO
RULE 13A-14(A) / RULE 15D-14(A) OF THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
I, Jason Gardner, D.Phil., certify that:
1. I have reviewed this Quarterly Report on Form 10-Q of Magenta Therapeutics, Inc.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrants other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
(a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
(b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
(c) Evaluated the effectiveness of the registrants disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
(d) Disclosed in this report any change in the registrants internal control over financial reporting that occurred during the registrants most recent fiscal quarter (the registrants fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrants internal control over financial reporting; and
5. The registrants other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrants auditors and the audit committee of the registrants board of directors (or persons performing the equivalent functions):
(a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrants ability to record, process, summarize and report financial information; and
(b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrants internal control over financial reporting.
Date: November 4, 2021
/s/ Jason Gardner |
Jason Gardner, D.Phil. |
President, Chief Executive Officer and Director |
(Principal Executive Officer) |
Exhibit 31.2
CERTIFICATION OF PRINCIPAL FINANCIAL OFFICER PURSUANT TO
RULE 13A-14(A) / RULE 15D-14(A) OF THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
I, Stephen Mahoney, certify that:
1. I have reviewed this Quarterly Report on Form 10-Q of Magenta Therapeutics, Inc.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrants other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
(a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
(b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
(c) Evaluated the effectiveness of the registrants disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
(d) Disclosed in this report any change in the registrants internal control over financial reporting that occurred during the registrants most recent fiscal quarter (the registrants fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrants internal control over financial reporting; and
5. The registrants other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrants auditors and the audit committee of the registrants board of directors (or persons performing the equivalent functions):
(a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrants ability to record, process, summarize and report financial information; and
(b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrants internal control over financial reporting.
Date: November 4, 2021
/s/ Stephen Mahoney |
Stephen Mahoney |
Chief Financial and Operating Officer |
(Principal Financial and Accounting Officer) |
Exhibit 32.1
CERTIFICATIONS OF PRINCIPAL EXECUTIVE OFFICER AND PRINCIPAL
FINANCIAL OFFICER PURSUANT TO 18 U.S.C. SECTION 1350,
AS ADOPTED PURSUANT TO
SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002
In connection with this Quarterly Report on Form 10-Q of Magenta Therapeutics, Inc. (the Company) for the quarter ended September 30, 2021 as filed with the Securities and Exchange Commission on the date hereof (the Report), each of the undersigned officers hereby certifies, pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002, that to the best of his or her knowledge:
1. | the Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and |
2. | the information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of the Company. |
Dated: November 4, 2021
/s/ Jason Gardner |
Jason Gardner, D.Phil. |
President, Chief Executive Officer and Director |
(Principal Executive Officer) |
/s/ Stephen Mahoney |
Stephen Mahoney |
Chief Financial and Operating Officer |
(Principal Financial and Accounting Officer) |