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.) | |
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||
(Address of principal executive offices) |
(Zip Code) |
Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered | ||
☒ | Accelerated filer | ☐ | ||||
Non-accelerated filer |
☐ | Smaller reporting company | ||||
Emerging growth company |
• |
the impacts of the COVID-19 pandemic; |
• |
the initiation, timing, progress and results of our current and future preclinical studies and clinical trials and our research and development programs; |
• |
our estimates regarding expenses, future revenue, capital requirements and need for additional financing; |
• |
our expectations regarding our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents, and investments and the period in which we expect that such cash, cash equivalents, and investments will enable us to fund such operating expenses and capital expenditure requirements; |
• |
our plans to develop our product candidates; |
• |
the timing of and our ability to submit applications for, obtain and maintain regulatory approvals for our product candidates; |
• |
the potential advantages of our product candidates; |
• |
the rate and degree of market acceptance and clinical utility of our product candidates; |
• |
our estimates regarding the potential market opportunity for our product candidates; |
• |
our commercialization, marketing and manufacturing capabilities and strategy; |
• |
our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates; |
• |
our ability to identify additional products, product candidates or technologies with significant commercial potential that are consistent with our commercial objectives; |
• |
the impact of government laws and regulations; |
• |
our competitive position; |
• |
developments relating to our competitors and our industry; and |
• |
our ability to establish collaborations or obtain additional funding. |
March 31, 2021 |
December 31, 2020 |
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Assets |
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Current assets: |
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Cash and cash equivalents |
$ | $ | ||||||
Investments |
||||||||
Collaboration receivables |
||||||||
Prepaid expenses and other current assets |
||||||||
Restricted cash |
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|
|||||
Total current assets |
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Property and equipment, net |
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Right-of-use |
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Goodwill |
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Intangible assets, net |
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Other assets |
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|
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Total assets |
$ | $ | ||||||
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|
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|
|||||
Liabilities and Stockholders’ Equity |
||||||||
Current liabilities: |
||||||||
Accounts payable |
$ | $ | ||||||
Accrued expenses |
||||||||
Current portion of deferred revenue |
||||||||
Current portion of operating lease liability |
||||||||
Income tax liability |
|
|
|
|
|
|
— |
|
Total current liabilities |
||||||||
Contingent consideration |
||||||||
Deferred revenue, net of current portion |
||||||||
Operating lease liability, net of current portion |
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|
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Total liabilities |
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|
|||||
Commitments and contingencies (Notes 3 and 12) |
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Stockholders’ equity: |
||||||||
Preferred stock, $ |
— | |||||||
Common stock, $ |
||||||||
Additional paid-in capital |
||||||||
Accumulated deficit |
( |
) | ( |
) | ||||
Accumulated other comprehensive income |
— | |||||||
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|
|||||
Total stockholders’ equity |
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|
|||||
Total liabilities and stockholders’ equity |
$ | $ | ||||||
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|
|
Three Months Ended March 31, |
||||||||
2021 |
2020 |
|||||||
Collaboration revenue |
$ | $ | ||||||
Operating expenses: |
||||||||
Research and development |
||||||||
General and administrative |
||||||||
Change in fair value of contingent consideration |
( |
) | ( |
) | ||||
|
|
|
|
|||||
Total operating expenses |
||||||||
|
|
|
|
|||||
Income (loss) from operations |
( |
) | ||||||
Other income, net |
||||||||
|
|
|
|
|||||
Income (loss) before income tax provision |
( |
) | ||||||
Income tax provision |
( |
) |
— | |||||
|
|
|
|
|||||
Net income (loss) |
$ | $ | ( |
) | ||||
|
|
|
|
|||||
Net income (loss) per share—basic |
$ | $ | ( |
) | ||||
|
|
|
|
|||||
Weighted average common shares outstanding—basic |
||||||||
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|
|||||
Net income (loss) per share—diluted |
$ | $ | ( |
) | ||||
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|
|||||
Weighted average common shares outstanding—diluted |
||||||||
|
|
|
|
Three Months Ended March 31, |
||||||||
2021 |
2020 |
|||||||
Net income (loss) |
$ | $ | ( |
) | ||||
Other comprehensive income (loss): |
||||||||
Unrealized gains on available-for-sale |
||||||||
|
|
|
|
|||||
Comprehensive income (loss) |
$ | $ | ( |
) | ||||
|
|
|
|
Common Stock |
Additional Paid-in Capital |
Accumulated Deficit |
Accumulated Other Comprehensive Income |
Total Stockholders’ Equity |
||||||||||||||||||||
Shares |
Amount |
|||||||||||||||||||||||
Balances at December 31, 2020 |
$ |
$ |
$ |
( |
) | $ |
$ |
|||||||||||||||||
Exercise of stock options |
— | — | — | |||||||||||||||||||||
Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
Unrealized gains on available-for-sale |
— | — | — | — | ||||||||||||||||||||
Net income |
— | — | — | — | ||||||||||||||||||||
|
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|
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|
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|
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|
|||||||||||||
Balances at March 31, 2021 |
$ | $ | $ | ( |
) | $ | $ | |||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
Common Stock |
Additional Paid-in Capital |
Accumulated Deficit |
Accumulated Other Comprehensive Income |
Total Stockholders’ Equity |
||||||||||||||||||||
Shares |
Amount |
|||||||||||||||||||||||
Balances at December 31, 2019 |
$ | $ | $ | ( |
) | $ | $ | |||||||||||||||||
Exercise of stock options |
— | — | — | |||||||||||||||||||||
Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
Unrealized gains on available-for-sale |
— | — | — | — | ||||||||||||||||||||
Net loss |
— | — | — | ( |
) | — | ( |
) | ||||||||||||||||
|
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|
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|||||||||||||
Balances at March 31, 2020 |
$ | $ | $ | ( |
) | $ | $ | |||||||||||||||||
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|
|
|
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|
|
|
|
|
|
Three Months Ended March 31, |
||||||||
2021 |
2020 |
|||||||
Cash flows from operating activities: |
||||||||
Net income (loss) |
$ | $ | ( |
) | ||||
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities: |
||||||||
Depreciation and amortization expense |
||||||||
Stock-based compensation expense |
||||||||
Change in fair value of contingent consideration |
( |
) | ( |
) | ||||
Changes in operating assets and liabilities: |
||||||||
Collaboration receivables |
( |
) | ||||||
Prepaid expenses and other assets |
( |
) | ||||||
Right-of-use |
||||||||
Long-term prepaid rent |
( |
) |
( |
) | ||||
Accounts payable |
( |
) | ||||||
Accrued expenses |
||||||||
Income tax liabilit y |
— |
|||||||
Lease liability |
( |
) | ( |
) | ||||
Deferred revenue |
( |
) | ||||||
Net cash provided by (used in) operating activities |
( |
) | ||||||
Cash flows from investing activities: |
||||||||
Purchases of investments |
( |
) | ( |
) | ||||
Sales and maturities of investments |
— | |||||||
Purchases of property and equipment |
( |
) | ( |
) | ||||
Net cash provided by (used in) investing activities |
( |
) | ||||||
Cash flows from financing activities: |
||||||||
Payments of public offering costs |
— | ( |
) | |||||
Proceeds from option exercises |
||||||||
Net cash provided by financing activities |
||||||||
Net increase (decrease) 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 |
$ | $ | ||||||
Cash, cash equivalents and restricted cash at end of period: |
||||||||
Cash and cash equivalents |
$ | $ | ||||||
Restricted cash |
||||||||
Total cash, cash equivalents and restricted cash at end of period |
$ | $ | ||||||
Supplemental disclosure of non-cash investing and financing activities: |
||||||||
Purchases of property and equipment included in accounts payable and accrued expenses |
$ | $ | ||||||
Deferred offering costs included in accounts payable and accrued expenses |
$ | — | $ |
Three Months Ended March 31, |
||||||||
2021 |
2020 |
|||||||
Collaboration revenue |
$ | $ |
March 31, |
December 31, |
|||||||
2021 |
2020 |
|||||||
Contract liabilities |
||||||||
Deferred revenue |
$ | $ | |
March 31, 2021 |
||||||||||||||||
Estimated Life |
Gross Carrying Amount |
Accumulated Amortization |
Net Carrying Amount |
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(In thousands) |
||||||||||||||||
Definite-lived intangible assets: |
||||||||||||||||
MRT |
|
|
$ | $ | ( |
) | $ | |||||||||
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Indefinite-lived intangible assets: |
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||||||||||||||
IPR&D - CF |
|
Indefinite | |
— | ||||||||||||
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|||||||||
Total intangible assets, net |
|
|
$ | $ | ( |
) | $ | |||||||||
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December 31, 2020 |
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Estimated Life |
|
Gross Carrying Amount |
Accumulated Amortization |
Net Carrying Amount |
||||||||||||
|
(In |
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Definite-lived intangible assets: |
|
|
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MRT |
|
|
$ | $ | ( |
) | $ | |||||||||
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|
|
|
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|
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|
|||||||||
Indefinite-lived intangible assets: |
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||||||||||||||
IPR&D - CF |
|
Indefinite | |
— | ||||||||||||
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|||||||||
Total intangible assets, net |
|
|
$ | $ | ( |
) | $ | |||||||||
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|
|
|
|
|
|
Fair Value Measurements as of March 31, 2021 Using: |
||||||||||||||||
Level 1 |
Level 2 |
Level 3 |
Total |
|||||||||||||
Assets: |
||||||||||||||||
Money market funds |
$ | |
$ | $ | $ | |||||||||||
U.S. treasuries |
||||||||||||||||
U.S. government agency bonds |
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|
|
|
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|
|||||||||
$ | |
$ | $ | $ | ||||||||||||
|
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|||||||||
Liabilities: |
||||||||||||||||
Contingent consideration |
$ | $ | $ | $ | ||||||||||||
|
|
|
|
|
|
|
|
|||||||||
$ | $ | $ | $ | |||||||||||||
|
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|
|||||||||
Fair Value Measurements as of December 31, 2020 Using: |
||||||||||||||||
Level 1 |
Level 2 |
Level 3 |
Total |
|||||||||||||
Assets: |
||||||||||||||||
Money market funds |
$ | — | $ | $ | — | $ | ||||||||||
U.S. treasuries |
— | |||||||||||||||
U.S. government agency bonds |
— | — | ||||||||||||||
|
|
|
|
|
|
|
|
|||||||||
$ | — | $ | $ | — | $ | |||||||||||
|
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|
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|
|||||||||
Liabilities: |
||||||||||||||||
Contingent consideration |
$ | — | $ | — | $ | $ | ||||||||||
|
|
|
|
|
|
|
|
|||||||||
$ | — | $ | — | $ | $ | |||||||||||
|
|
|
|
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|
|
|
March 31, 2021 |
December 31, 2020 |
|||||||||||||||
Amortized Cost |
Fair Value |
Amortized Cost |
Fair Value |
|||||||||||||
Due within one year |
$ |
$ |
$ |
$ |
||||||||||||
Due after one year through two years |
||||||||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total available-for-sale |
$ |
$ |
$ |
$ |
||||||||||||
|
|
|
|
|
|
|
|
Unobservable Inputs Projected Year of Payment |
Fair Value at |
|||||||||||
March 31, |
December 31, |
|||||||||||
2021 |
2020 |
|||||||||||
Earnout payments |
|
|
$ |
$ |
||||||||
Milestone payments |
|
|
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|
|
|||||||
|
|
$ | $ | |||||||||
|
|
|
|
|
|
Fair Value |
||||
Balance as of December 31, 2020 |
$ | |||
Decrease in fair value of contingent consideration |
( |
) | ||
|
|
|||
Balance as of March 31, 2021 |
$ | |||
|
|
March 31, 2021 |
December 2020 |
|||||||
Laboratory equipment |
$ | $ | ||||||
Computer equipment |
||||||||
Office equipment |
||||||||
Leasehold improvements |
||||||||
Construction in progress |
||||||||
|
|
|
|
|||||
Less: Accumulated depreciation and amortization |
( |
) | ( |
) | ||||
|
|
|
|
|||||
$ | $ | |||||||
|
|
|
|
March 31, 2021 |
December 31, 2020 |
|||||||
Accrued external research and development expenses |
$ | $ |
||||||
Accrued employee compensation and benefits |
||||||||
Accrued consultant and professional fees |
||||||||
Other |
||||||||
|
|
|
|
|||||
$ | $ | |||||||
|
|
|
|
Number of Shares |
Weighted Average Exercise Price |
Weighted Average Remaining Contractual Term |
Intrinsic Value |
|||||||||||||
(in years) |
||||||||||||||||
Outstanding as of December 31, 2020 |
$ | $ | ||||||||||||||
Granted |
$ | |||||||||||||||
Exercised |
( |
) | $ | |||||||||||||
Forfeited |
( |
) | $ | |||||||||||||
|
|
|||||||||||||||
Outstanding as of March 31, 2021 |
$ | $ | ||||||||||||||
|
|
|||||||||||||||
Exercisable as of March 31, 2021 |
$ | $ | ||||||||||||||
Vested and expected to vest as of March 31, 2021 |
$ | $ |
Three Months Ended March 31, |
||||||||
2021 |
2020 |
|||||||
Risk-free interest rate |
% | % | ||||||
Expected term (in years) |
||||||||
Expected volatility |
% | % | ||||||
Expected dividend yield |
% | % |
Three Months Ended March 31, |
||||||||
2021 |
2020 |
|||||||
Research and development expenses |
$ | $ | ||||||
General and administrative expenses |
||||||||
|
|
|
|
|||||
$ | $ | |||||||
|
|
|
|
Three Months Ended March 31, |
||||||||
2021 |
2020 |
|||||||
Basic net income (loss) per common share: |
||||||||
Numerator: |
||||||||
Net income (loss) |
$ | $ | ( |
) | ||||
Denominator: |
||||||||
Weighted average common shares outstanding—basic |
||||||||
Net income (loss) per share—basic |
$ | $ | ( |
) | ||||
Diluted net income (loss) per common share: |
||||||||
Numerator: |
||||||||
Net income (loss) |
$ | $ | ( |
) | ||||
Denominator: |
||||||||
Weighted average common shares outstanding—diluted |
||||||||
Net income (loss) per share—diluted |
$ | $ | ( |
) |
Three Months Ended March 31, |
||||||||
2021 |
2020 |
|||||||
Options to purchase common stock |
||||||||
Unvested restricted common stock |
||||||||
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|
|||||
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|
|
Three Months Ended March 31, |
||||||||
2021 |
2020 |
|||||||
Lease cost |
||||||||
Operating lease cost |
$ | $ | ||||||
|
|
|
|
|||||
Total lease cost |
$ | $ | ||||||
|
|
|
|
|||||
Other information |
||||||||
Operating cash flows from operating leases |
$ | $ | ||||||
Operating lease liabilities arising from obtaining right-of-use |
— | |||||||
Weighted-average remaining lease term |
||||||||
Weighted-average discount rate |
% | % |
March 31, 2021 |
December 31, 2020 |
|||||||
2021 |
$ | $ | ||||||
2022 |
||||||||
2023 |
||||||||
2024 |
||||||||
2025 |
||||||||
2026 and thereafter |
||||||||
|
|
|
|
|||||
Total future minimum lease payments |
||||||||
Less: imputed interest |
( |
) | ( |
) | ||||
|
|
|
|
|||||
Present value of lease liabilities |
$ | $ | ||||||
|
|
|
|
• | employee-related expenses, including salaries, related benefits 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 third parties, such as consultants and contract research organizations, or CROs; |
• | the cost of manufacturing drug products for use in our preclinical studies and clinical trials, including under agreements with third parties, such as consultants and contract manufacturing organizations, or CMOs; |
• | laboratory supplies; |
• | facilities, depreciation and other expenses, which include direct or allocated expenses for rent and maintenance of facilities and insurance; |
• | costs to fulfill our obligations under our collaboration with Sanofi; |
• | costs related to compliance with regulatory requirements; and |
• | payments made under third-party licensing agreements. |
Three Months Ended March 31, |
||||||||
2021 |
2020 |
|||||||
(in thousands) |
||||||||
Discovery program |
$ | 8,616 | $ | 3,775 | ||||
Vaccine program |
7,570 | 2,589 | ||||||
MRT5005 program |
6,214 | 6,094 | ||||||
Unallocated research and development expenses |
18,740 | 8,981 | ||||||
|
|
|
|
|||||
Total research and development expenses |
$ | 41,140 | $ | 21,439 | ||||
|
|
|
|
• | the timing and progress of preclinical and clinical development activities, including delays resulting from the COVID-19 pandemic; |
• | the number and scope of preclinical and clinical programs we decide to pursue; |
• | our ability to maintain our current research and development programs and to establish new ones; |
• | establishing an appropriate safety profile with IND enabling studies; |
• | successful patient enrollment in, and the initiation and completion of, clinical trials; |
• | the successful completion of clinical trials with safety, tolerability and efficacy profiles that are satisfactory to the FDA or any comparable foreign regulatory authority; |
• | the receipt of regulatory approvals from applicable regulatory authorities; |
• | the timing, receipt and terms of any marketing approvals from applicable regulatory authorities; |
• | the success of our collaboration with Sanofi; |
• | our ability to establish new licensing or collaboration arrangements; |
• | the performance of our future collaborators, if any; |
• | establishing commercial manufacturing capabilities or making arrangements with third-party manufacturers; |
• | development and timely delivery of commercial-grade drug formulations that can be used in our clinical trials and for commercial launch; |
• | obtaining, maintaining, defending and enforcing patent claims and other intellectual property rights; |
• | launching commercial sales of our product candidates, if approved, whether alone or in collaboration with others; and |
• | maintaining a continued acceptable safety profile of the product candidates following approval. |
Three Months Ended March 31, |
||||||||||||
2021 |
2020 |
Change |
||||||||||
(in thousands) |
||||||||||||
Collaboration revenue |
$ | 34,600 | $ | 4,654 | $ | 29,946 | ||||||
Operating expenses: |
||||||||||||
Research and development |
41,140 | 21,439 | 19,701 | |||||||||
General and administrative |
10,817 | 7,458 | 3,359 | |||||||||
Change in fair value of contingent consideration |
(43,979 | ) | (9,452 | ) | (34,527 | ) | ||||||
|
|
|
|
|
|
|||||||
Total operating expenses |
7,978 | 19,445 | (11,467 | ) | ||||||||
|
|
|
|
|
|
|||||||
Income (loss) from operations |
26,622 | (14,791 | ) | 41,413 | ||||||||
Other income, net |
154 | 509 | (355 | ) | ||||||||
|
|
|
|
|
|
|||||||
Income (loss) before income tax provision |
26,776 | (14,282 | ) | 41,058 | ||||||||
Income tax provision |
(254 | ) | — | (254 | ) | |||||||
|
|
|
|
|
|
|||||||
Net income (loss) |
$ | 26,522 | $ | (14,282 | ) | $ | 40,804 | |||||
|
|
|
|
|
|
Three Months Ended March 31, |
||||||||||||
2021 |
2020 |
Change |
||||||||||
(in thousands) |
||||||||||||
Direct external research and development expenses by program: |
|
|||||||||||
Discovery program |
$ | 8,616 | $ | 3,775 | $ | 4,841 | ||||||
Vaccine program |
7,570 | 2,589 | 4,981 | |||||||||
MRT5005 program |
6,214 | 6,094 | 120 | |||||||||
Unallocated research and development expenses: |
||||||||||||
Personnel related (including stock-based compensation) |
8,757 | 5,985 | 2,772 | |||||||||
Other |
9,983 | 2,996 | 6,987 | |||||||||
|
|
|
|
|
|
|||||||
Total research and development expenses |
$ | 41,140 | $ | 21,439 | $ | 19,701 | ||||||
|
|
|
|
|
|
Three Months Ended March 31, |
||||||||
2021 |
2020 |
|||||||
(in thousands) |
||||||||
Net cash provided by (used in) operating activities |
$ | 689 | $ | (31,447 | ) | |||
Net cash provided by (used in) investing activities |
(188,520 | ) | 44,260 | |||||
Net cash provided by financing activities |
1,550 | 75 | ||||||
|
|
|
|
|||||
Net increase (decrease) in cash, cash equivalents and restricted cash |
$ | (186,281 | ) | $ | 12,888 | |||
|
|
|
|
• | continue the clinical development of MRT5005; |
• | continue the development of mRNA vaccine candidates against infectious diseases, including MRT5500, the lead vaccine candidate against SARS-CoV-2; |
• | leverage our programs to advance our other product candidates into preclinical and clinical development; |
• | seek regulatory approvals for any product candidates that successfully complete clinical trials; |
• | seek to discover and develop additional product candidates; |
• | expand our manufacturing, operational, financial and management systems; |
• | increase personnel, including personnel to support our clinical development, manufacturing and commercialization efforts and our operations as a public company; |
• | maintain, expand and protect our intellectual property portfolio; |
• | acquire or in-license other product candidates and technologies; |
• | incur additional legal, accounting and other expenses in operating as a public company; and |
• | establish a sales, marketing, medical affairs and distribution infrastructure to commercialize any product candidates for which we may not obtain marketing approval and intend to commercialize on our own or jointly. |
• | the impacts of the COVID-19 pandemic and our response to it; |
• | the scope, progress, results and costs of researching and developing our product candidates, and conducting preclinical studies and clinical trials; |
• | the success of our collaboration with Sanofi; |
• | the costs, timing and outcome of regulatory review of our product candidates; |
• | the costs of future activities, including product sales, medical affairs, marketing, manufacturing and distribution, for any of our product candidates for which we receive marketing approval; |
• | the costs of manufacturing commercial-grade products and sufficient inventory to support commercial launch; |
• | the ability to receive additional non-dilutive funding, including grants from organizations and foundations; |
• | the revenue, if any, received from commercial sale of our products, should any of our product candidates receive marketing approval; |
• | the cost and timing of hiring new employees to support our continued growth; |
• | the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending intellectual property-related claims; |
• | the ability to establish and maintain collaborations on favorable terms, if at all; |
• | the extent to which we acquire or in-license other product candidates and technologies; and |
• | the timing, receipt and amount of sales of, or milestone payments related to or royalties on, our current or future product candidates, if any. |
• | diversion of healthcare resources away from the conduct of our clinical trials in order to focus on pandemic concerns, including the availability of necessary materials, the attention of physicians serving as our clinical trial investigators, access to hospitals serving as our clinical trial sites, and availability of hospital staff supporting the conduct of our clinical trials; |
• | potential interruptions in global shipping affecting the transport of clinical trial materials, such as investigational drug product, patient samples, and other supplies used in our clinical trials; |
• | the impact of further limitations on travel that could interrupt key clinical trial activities, such as clinical trial site initiations and monitoring activities, travel by our employees, contractors or patients to clinical trial sites, or the ability of employees at any of our contract manufacturers or contract research organizations to report to work, any of which could delay or adversely impact the conduct or progress of our clinical trials for MRT5005 and other research and manufacturing activities, and limit the amount of clinical data we will be able to report; |
• | any future interruption of, or delays in receiving, supplies of clinical trial material from our contract manufacturing organizations due to staffing shortages, production slowdowns or stoppages, or disruptions in delivery systems; |
• | availability of future capacity at our contract manufacturers to produce sufficient drug substance and drug product to meet forecasted clinical trial demand if any of these manufacturers elect or are required to divert attention or resources to the manufacture of other pharmaceutical products; |
• | delays in ongoing laboratory experiments and operations if we are required to further reduce the number of employees in our laboratories, or if the contract research organizations, or CROs, we have retained to supplement our internal research efforts are unable to perform as anticipated, whether due to capacity constraints, staffing shortages, or otherwise; and |
• | business disruptions caused by potential workplace closures and an increased reliance on employees working from home, challenges in recruiting employees required to execute on our research and development plans, cybersecurity and data accessibility issues, and communication or transit disruptions, any of which could adversely impact our business operations and delay necessary interactions among our employees and between our company and the third parties upon which we rely. |
• | continue the clinical development of MRT5005; |
• | continue the development of mRNA vaccine candidates against infectious diseases, including MRT5500, the lead vaccine candidate against SARS-CoV-2; |
• | leverage our programs to advance our other product candidates into preclinical and clinical development; |
• | seek regulatory approvals for any product candidates that successfully complete clinical trials; |
• | seek to discover and develop additional product candidates; |
• | expand our manufacturing, operational, financial and management systems; |
• | increase personnel, including personnel to support our research, clinical development, manufacturing and commercialization efforts and our operations as a public company; |
• | maintain, expand and protect our intellectual property portfolio; |
• | acquire or in-license other product candidates and technologies; |
• | incur additional legal, accounting and other expenses in operating as a public company; and |
• | establish a sales, marketing, medical affairs and distribution infrastructure to commercialize any product candidates for which we may obtain marketing approval and intend to commercialize on our own or jointly. |
• | completing preclinical and clinical development of our product candidates and identifying and developing new product candidates; |
• | seeking and obtaining marketing approvals for any of our product candidates; |
• | launching and commercializing product candidates for which we obtain marketing approval by establishing a sales force, marketing, medical affairs and distribution infrastructure or, alternatively, collaborating with a commercialization partner; |
• | achieving formulary status in hospitals and adequate coverage and reimbursement by government and third-party payors for our product candidates; |
• | establishing and maintaining 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 our product candidates, if approved; |
• | obtaining market acceptance of our product candidates as viable treatment options; |
• | addressing any competing technological and market developments; |
• | negotiating favorable terms in any collaboration, licensing or other arrangements into which we may enter and performing our obligations in such collaborations; |
• | maintaining, protecting and expanding our portfolio of intellectual property rights, including patents, trade secrets and know-how; |
• | defending against third-party interference or infringement claims, if any; and |
• | attracting, hiring and retaining qualified personnel. |
• | the impacts of the COVID-19 pandemic and our response to it; |
• | the scope, progress, results and costs of researching and developing our product candidates, and conducting preclinical studies and clinical trials; |
• | the success of our collaboration with Sanofi; |
• | the costs, timing and outcome of regulatory review of our product candidates; |
• | the costs of future activities, including product sales, medical affairs, marketing, manufacturing and distribution, for any of our product candidates for which we receive marketing approval; |
• | the costs of manufacturing commercial-grade products and sufficient inventory to support commercial launch; |
• | the ability to receive additional non-dilutive funding, including grants from organizations and foundations; |
• | the revenue, if any, received from commercial sale of our products, should any of our product candidates receive marketing approval; |
• | the cost and timing of hiring new employees to support our continued growth; |
• | the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending intellectual property-related claims; |
• | our ability to establish and maintain collaborations on favorable terms, if at all; |
• | the extent to which we acquire or in-license other product candidates and technologies; and |
• | the timing, receipt and amount of sales of, or milestone payments related to or royalties on, our current or future product candidates, if any. |
• | successful patient enrollment in and completion of clinical trials; |
• | a safety, tolerability and efficacy profile that is satisfactory to the FDA, EMA or other regulatory authorities for marketing approval; |
• | timely receipt of marketing approvals from applicable regulatory authorities; |
• | the extent of any required post-marketing approval commitments to applicable regulatory authorities; |
• | establishment and maintenance of arrangements with third-party manufacturers for both clinical and any future commercial manufacturing; |
• | adequate ongoing availability of raw materials and drug product for clinical development and any commercial sales; |
• | obtaining and maintaining patent, trade secret protection and regulatory exclusivity, both in the United States and internationally; |
• | protection of our rights in our intellectual property portfolio; |
• | successful launch of commercial sales following any marketing approval; |
• | a continued acceptable safety profile following any marketing approval; |
• | commercial acceptance by hospitals, the patient community, the medical community and third-party payors; |
• | the availability of coverage and adequate reimbursement from third-party payors; |
• | the performance of our future collaborators, if any; and |
• | our ability to compete with other therapies. |
• | delays in reaching a consensus with regulatory authorities or collaborators on trial design; |
• | delays in reaching agreement on acceptable terms with CROs and clinical trial sites; |
• | delays in opening clinical trial sites or obtaining required institutional review board or independent ethics committee approval at each clinical trial site; |
• | delays in recruiting suitable subjects or a sufficient number of subjects to participate in our clinical trials; |
• | imposition of a clinical hold by regulatory authorities, including upon submission of an IND, or as a result of a serious adverse event or after an inspection of our clinical trial operations or trial sites; |
• | failure by us, any CROs we engage, clinical investigators or any other third parties to adhere to clinical trial requirements; |
• | failure to perform the clinical trial in accordance with good clinical practices, or GCP, or applicable regulatory requirements in the European Union, the United States, or other countries; |
• | delays in the testing, validation, manufacturing and delivery of our product candidates to the clinical sites, including delays by third parties with whom we have contracted to perform certain of those functions; |
• | delays or failures in demonstrating the comparability of product manufactured at one facility or with one process to product manufactured at another facility or with another process, including clinical trials to demonstrate such comparability; |
• | delays in having patients complete participation in a trial or return for post-treatment follow-up; |
• | clinical trial sites or subjects dropping out of a trial; |
• | selection of clinical endpoints that require prolonged periods of clinical observation or analysis of the resulting data; |
• | occurrence of serious adverse events associated with the product candidate that are viewed to outweigh its potential benefits; |
• | occurrence of serious adverse events in trials of the same class of agents conducted by other sponsors; and |
• | changes in regulatory requirements and guidance that require amending or submitting new clinical protocols. |
• | inability to generate sufficient preclinical or other in vivo in vitro |
• | delays in reaching a consensus with regulatory agencies on study design. |
• | coordination between us, CROs and any future collaborators in our efforts to enroll and administer the clinical trial; |
• | size of the patient population and process for identifying patients; |
• | design of the trial protocol; |
• | eligibility and exclusion criteria; |
• | perceived risks and benefits of the product candidate under study; |
• | availability of competing commercially available therapies and other competing product candidates’ clinical trials; |
• | time of year in which the trial is initiated or conducted; |
• | variations in the seasonal incidence of the target indication; |
• | severity of the disease under investigation; |
• | ability to obtain and maintain subject consent; |
• | ability to enroll and treat patients in a timely manner; |
• | risk that enrolled subjects will drop out before completion of the trial; |
• | proximity and availability of clinical trial sites for prospective patients; |
• | patient referral practices of physicians; and |
• | ability to monitor subjects adequately during and after treatment. |
• | be delayed in obtaining marketing approval for our product candidates, if at all; |
• | obtain approval for indications or patient populations that are not as broad as intended or desired; |
• | obtain approval with labeling that includes significant use or distribution restrictions or safety warnings; |
• | be subject to changes in the way the product is administered; |
• | be required to perform additional clinical trials to support approval or be subject to additional post-marketing testing requirements; |
• | have regulatory authorities withdraw, or suspend, their approval of the product or impose restrictions on its distribution in the form of a modified risk evaluation and mitigation strategy, or REMS; |
• | be subject to the addition of labeling statements, such as contraindications or warnings, including a black box warning; |
• | be sued; or |
• | experience damage to our reputation. |
• | clinical practice patterns and standards of care that vary widely among countries; |
• | non-U.S. regulatory authority requirements that could restrict or limit our ability to conduct our clinical trials; |
• | administrative burdens of conducting clinical trials under multiple non-U.S. regulatory authority schema; |
• | foreign exchange fluctuations; and |
• | diminished protection of intellectual property in some countries. |
• | Sanofi has significant discretion in determining the efforts and resources that it will apply to our collaboration; |
• | Sanofi may not perform its obligations as expected; |
• | The clinical trials conducted as part of our collaboration with Sanofi may not be successful; |
• | Sanofi may not pursue development and/or commercialization of any vaccine candidates that achieve regulatory approval or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in Sanofi’s strategic focus or available funding or external factors, such as an acquisition, that divert resources or create competing priorities; |
• | Sanofi has final decision-making authority for conducting clinical trials, and this may result in Sanofi delaying clinical trials, providing insufficient funding for clinical trials, stopping a clinical trial or abandoning a vaccine candidate, repeating or conducting new clinical trials or requiring a new formulation of a vaccine candidate for clinical testing; |
• | We may not have access to, or may be restricted from disclosing, certain information regarding vaccine candidates being developed or commercialized under our collaboration with Sanofi and, consequently, may have limited ability to inform our stockholders about the status of such vaccine candidates; |
• | Sanofi has an existing collaboration with GlaxoSmithKline plc to develop a SARS-CoV-2 |
• | Sanofi may view vaccine candidates developed in collaboration with us as competitive with their own product candidates or products, which may cause Sanofi to cease to devote resources to the commercialization of our vaccine candidates; |
• | Sanofi may not commit sufficient resources to the marketing and distribution of any such of our vaccine candidates that achieve regulatory approval; |
• | Disagreements with Sanofi, including disagreements over proprietary rights, contract interpretation or the preferred course of development of any of our vaccine candidates, may cause delays or termination of the research, development, manufacture or commercialization of such vaccine candidates, may lead to additional responsibilities for us with respect to such vaccine candidates or may result in litigation or arbitration, any of which would be time-consuming and expensive. Moreover, in certain circumstances, there could be a misalignment between our contractual obligations to Sanofi and any upstream contractual obligations we may owe to our licensors or other third parties; |
• | Sanofi may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential litigation. For example, Sanofi has the first right to enforce or defend certain of our intellectual property rights under our collaboration with respect to products in Licensed Fields, and although we may have the right to assume the enforcement and defense of such intellectual property rights if Sanofi does not, our ability to do so may be compromised by Sanofi’s actions; |
• | Disputes may arise with respect to the ownership of intellectual property developed pursuant to our collaboration with Sanofi; |
• | Sanofi may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability; and |
• | Sanofi may terminate our collaboration for convenience after a specified notice period and, if terminated, we could be required to raise additional capital to pursue further development or commercialization of the applicable vaccine candidates. |
• | 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. |
• | a covered benefit under the applicable health plan; |
• | safe, effective and medically necessary; |
• | appropriate for the specific patient population; |
• | cost-effective; and |
• | neither experimental nor investigational. |
• | the efficacy and safety of such product candidates as demonstrated in clinical trials; |
• | the potential and perceived advantages of our product candidates over other treatments; |
• | the cost-effectiveness of treatment relative to alternative treatments; |
• | the clinical indications for which the product candidate is approved by the FDA, the EMA or other regulatory body; |
• | the willingness of physicians to prescribe new therapies over the existing standard of care and future 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, including any black box warning; |
• | relative convenience and ease of administration; |
• | our ability to educate the medical community and third-party payors about the benefit of our product candidates; |
• | the strength of marketing and distribution support; |
• | the timing of market introduction of competitive products; |
• | any restrictions on the use of our products together with other medications; |
• | publicity concerning our products or competing products and treatments; and |
• | sufficient third-party payor insurance coverage and adequate reimbursement. |
• | different regulatory requirements for approval of drugs and biologics in foreign countries; |
• | reduced protection for intellectual property rights; |
• | unexpected changes in tariffs, trade barriers and regulatory requirements; |
• | economic weakness, including inflation, or political instability in foreign economies and markets; |
• | different pricing and reimbursement regimes; |
• | compliance with tax, employment, immigration and labor laws for employees living or traveling abroad; |
• | foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing business in another country; |
• | workforce uncertainty in countries where labor unrest is more common than in the United States; |
• | production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and |
• | business interruptions resulting from geopolitical actions, including war and terrorism or natural disasters, including earthquakes, typhoons, floods and fires. |
• | decreased demand for any product candidates that we may develop; |
• | loss of revenue; |
• | substantial monetary awards to trial participants or patients; |
• | significant time and costs to defend the related litigation; |
• | withdrawal of clinical trial participants; |
• | the inability to commercialize any product candidates that we may develop; and |
• | injury to our reputation and significant negative media attention. |
• | comply with FDA regulations or the regulations applicable in the European Union and other jurisdictions; |
• | provide accurate information to the FDA, the EMA and other regulatory authorities; |
• | comply with health care fraud and abuse laws and regulations in the United States and abroad; |
• | comply with the U.S. Foreign Corrupt Practices Act, or FCPA, or other anti-corruption laws and regulations; |
• | comply with U.S. federal securities laws relating to trading in our common stock; |
• | report financial information or data accurately; or |
• | disclose unauthorized activities to us. |
• | the scope of rights granted under the license agreement and other interpretation-related issues; |
• | the extent to which our technology and processes infringe the intellectual property of the licensor that is not subject to the licensing agreement; |
• | the sublicensing of patent and other rights under any collaborative development relationships; |
• | our diligence obligations under the license agreement and what activities satisfy those diligence obligations; |
• | the inventorship or ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and us and our partners; and |
• | the priority of invention of patented technology. |
• | others may be able to make products that are similar to our product candidates but that are not covered by the claims of the patents that we own or license or may own in the future; |
• | we, or any partners or collaborators, might not have been the first to make the inventions covered by the issued patent or pending patent application that we license or may own in the future; |
• | we, or any partners or collaborators, 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 licensed patent applications or those that we may own 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 have an adverse effect on our business; and |
• | we may choose not to file a patent for certain trade secrets or know-how, and a third party may subsequently file a patent covering such intellectual property. |
• | the FDA may disagree with the design or implementation of our clinical trials; |
• | we may be unable to demonstrate to the satisfaction of the FDA that a product candidate is safe, pure and potent or effective for its proposed indication; |
• | results of clinical trials may not meet the level of statistical significance required by the FDA for approval; |
• | we may be unable to demonstrate that a product candidate’s clinical and other benefits outweigh its safety risks; |
• | the FDA may disagree with our interpretation of data from preclinical studies or clinical trials; |
• | data collected from clinical trials of our product candidates may not be sufficient to support the submission of a BLA to the FDA or other submission or to obtain regulatory approval in the United States; |
• | the FDA may find deficiencies with or fail to approve the manufacturing processes or facilities of third-party manufacturers with which we contract for clinical and commercial supplies; and |
• | the approval policies or regulations of the FDA may significantly change in a manner rendering our clinical data insufficient for approval. |
• | restrictions on such products, manufacturers or manufacturing processes; |
• | restrictions on the labeling or marketing of a product; |
• | restrictions on product distribution or use; |
• | requirements to conduct post-marketing studies or clinical trials; |
• | warning letters or untitled letters; |
• | withdrawal of the products from the market; |
• | refusal to approve pending applications or supplements to approved applications that we submit; |
• | recall of products; |
• | restrictions on coverage by third-party payors; |
• | fines, restitution or disgorgement of profits or revenue; |
• | suspension or withdrawal of marketing approvals, including license revocation; |
• | refusal to permit the import or export of products; |
• | product seizure; and |
• | injunctions or the imposition of civil or criminal penalties. |
• | Anti-Kickback Statute |
• | False Claims Act per-claim penalties; |
• | HIPAA |
• | HIPAA and HITECH |
• | Transparency Requirements |
• | Analogous State and Foreign Laws |
• | an annual, non-deductible fee on any entity that manufactures or imports specified branded prescription drugs and biologic products; |
• | an increase in the statutory minimum rebates a manufacturer must pay under the Medicaid Drug Rebate Program; |
• | expansion of federal health care fraud and abuse laws, including the civil False Claims Act and the federal Anti-Kickback Statute, new government investigative powers and enhanced penalties for noncompliance; |
• | a new Medicare Part D coverage gap discount program, in which manufacturers must agree to offer 50% point-of-sale |
• | extension of manufacturers’ Medicaid rebate liability; |
• | expansion of eligibility criteria for Medicaid programs; |
• | expansion of the entities eligible for discounts under the Public Health Service pharmaceutical pricing program; |
• | new requirements to report certain financial arrangements with physicians and teaching hospitals; |
• | a new requirement to annually report drug samples that manufacturers and distributors provide to physicians; and |
• | a new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research. |
• | results of preclinical studies and clinical trials of our product candidates or those of our competitors; |
• | public announcements about the scientific community’s evolving understanding of the COVID-19 pandemic and the potential effectiveness of vaccines, treatments, public health measures and other approaches to addressing the disease; |
• | the success of competitive products or technologies; |
• | commencement or termination of collaborations; |
• | regulatory or legal developments in the United States 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 discover, develop, acquire or in-license additional product candidates; |
• | actual or anticipated changes in estimates as to financial results, development timelines or recommendations by securities analysts; |
• | variations in our financial results or those of companies that are perceived to be similar to us; |
• | changes in the structure of health care payment systems; |
• | market conditions in the pharmaceutical and biotechnology sectors; |
• | the entry into significant acquisitions, strategic partnerships or divestitures by us or our competitors; |
• | significant sales of our common stock, including sales by our directors, executive officers or 5% stockholders; |
• | general economic, industry and market conditions, such as the impact of the COVID-19 pandemic on our industry and market conditions; and |
• | the other factors described in this “Risk Factors” section. |
• | establish a classified board of directors such that not all members of the board are 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 stockholder proposals that can be acted on at stockholder meetings and nominations to our board of directors; |
• | 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 stockholder meetings; |
• | authorize our board of directors to issue preferred stock without stockholder approval, which could be used to institute a stockholder rights plan, or so-called “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 75% of the votes that all our stockholders would be entitled to cast to amend or repeal certain provisions of our certificate of incorporation or bylaws. |
* | Filed herewith. |
** | Furnished herewith. |
+ | Indicates a management contract or compensatory arrangement. |
† | Submitted electronically herewith. |
Translate Bio, Inc. | ||||||
Date: May 6, 2021 | By: | /s/ Ronald C. Renaud, Jr. | ||||
Ronald C. Renaud, Jr. | ||||||
Chief Executive Officer | ||||||
Date: May 6, 2021 | By: | /s/ Brendan Smith | ||||
Brendan Smith | ||||||
Chief Financial Officer and Treasurer |