Cover Page
Cover Page - USD ($) $ in Billions | 12 Months Ended | ||
Dec. 31, 2020 | Feb. 22, 2021 | Jun. 30, 2020 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Document Annual Report | true | ||
Document Period End Date | Dec. 31, 2020 | ||
Current Fiscal Year End Date | --12-31 | ||
Document Transition Report | false | ||
Entity File Number | 001-36281 | ||
Entity Registrant Name | DICERNA PHARMACEUTICALS, INC. | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 20-5993609 | ||
Entity Address, Address Line One | 75 Hayden Avenue | ||
Entity Address, City or Town | Lexington | ||
Entity Address, State or Province | MA | ||
Entity Address, Postal Zip Code | 02421 | ||
City Area Code | 617 | ||
Local Phone Number | 621-8097 | ||
Title of 12(b) Security | Common Stock, $0.0001 Par Value | ||
Trading Symbol | DRNA | ||
Security Exchange Name | NASDAQ | ||
Entity Well-known Seasoned Issuer | Yes | ||
Entity Voluntary Filers | No | ||
Entity Current Reporting Status | Yes | ||
Entity Interactive Data Current | Yes | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Small Business | false | ||
Entity Emerging Growth Company | false | ||
ICFR Auditor Attestation Flag | true | ||
Entity Shell Company | false | ||
Entity Public Float | $ 1.8 | ||
Entity Common Stock, Shares Outstanding | 76,320,893 | ||
Documents Incorporated by Reference | Portions of the registrant’s definitive proxy statement for its 2021 Annual Meeting of Stockholders are incorporated by reference into Part III hereof. Such proxy statement will be filed with the Securities and Exchange Commission not later than 120 days after the end of the fiscal year covered by this Annual Report on Form 10-K. | ||
Entity Central Index Key | 0001399529 | ||
Document Fiscal Year Focus | 2020 | ||
Document Fiscal Period Focus | FY | ||
Amendment Flag | false |
CONSOLIDATED BALANCE SHEETS
CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
CURRENT ASSETS: | ||
Cash and cash equivalents | $ 126,023 | $ 152,816 |
Held-to-maturity investments | 442,820 | 196,065 |
Restricted cash equivalents, current | 744 | 0 |
Contract receivables | 34,713 | 200,354 |
Prepaid expenses and other current assets | 14,403 | 6,934 |
Total current assets | 618,703 | 556,169 |
NONCURRENT ASSETS: | ||
Property and equipment, net | 17,546 | 7,076 |
Right-of-use operating assets, net | 60,843 | 30,102 |
Restricted cash equivalents, noncurrent | 5,618 | 3,894 |
Other noncurrent assets | 5,136 | 168 |
Total noncurrent assets | 89,143 | 41,240 |
TOTAL ASSETS | 707,846 | 597,409 |
CURRENT LIABILITIES: | ||
Accounts payable | 7,901 | 6,077 |
Accrued expenses and other current liabilities | 28,061 | 20,042 |
Lease liability, current | 3,439 | 3,358 |
Deferred revenue, current | 138,537 | 212,258 |
Total current liabilities | 177,938 | 241,735 |
NONCURRENT LIABILITIES: | ||
Lease liability, noncurrent | 48,744 | 20,141 |
Deferred revenue, noncurrent | 336,236 | 182,730 |
Derivative liability | 6,000 | 0 |
Other noncurrent liabilities | 1,174 | 608 |
Total noncurrent liabilities | 392,154 | 203,479 |
TOTAL LIABILITIES | 570,092 | 445,214 |
COMMITMENTS AND CONTINGENCIES (NOTE 14) | ||
STOCKHOLDERS’ EQUITY: | ||
Preferred stock, $0.0001 par value – 5,000,000 shares authorized; no shares issued or outstanding at December 31, 2020 or 2019 | 0 | 0 |
Common stock, $0.0001 par value – 150,000,000 shares authorized; 75,757,213 and 71,573,196 shares issued and outstanding at December 31, 2020 and 2019, respectively | 8 | 7 |
Additional paid-in capital | 775,809 | 677,504 |
Accumulated deficit | (638,063) | (525,316) |
Total stockholders’ equity | 137,754 | 152,195 |
TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY | $ 707,846 | $ 597,409 |
CONSOLIDATED BALANCE SHEETS (Pa
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Dec. 31, 2020 | Dec. 31, 2019 |
STOCKHOLDERS’ EQUITY: | ||
Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized (in shares) | 5,000,000 | 5,000,000 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Common stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized (in shares) | 150,000,000 | 150,000,000 |
Common stock, shares issued (in shares) | 75,757,213 | 71,573,196 |
Common stock, shares outstanding (in shares) | 75,757,213 | 71,573,196 |
CONSOLIDATED STATEMENTS OF OPER
CONSOLIDATED STATEMENTS OF OPERATIONS - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Income Statement [Abstract] | |||
Revenue | $ 164,307 | $ 23,904 | $ 6,176 |
Operating expenses: | |||
Research and development | 205,384 | 109,339 | 45,711 |
General and administrative | 72,131 | 42,751 | 21,685 |
Litigation expense | 0 | 0 | 29,132 |
Total operating expenses | 277,515 | 152,090 | 96,528 |
Loss from operations | (113,208) | (128,186) | (90,352) |
Other income (expense): | |||
Interest income | 6,011 | 7,537 | 2,102 |
Interest expense | (20) | (3) | (603) |
Other (expense) income | (5,530) | 193 | 0 |
Total other income, net | 461 | 7,727 | 1,499 |
Net loss | $ (112,747) | $ (120,459) | $ (88,853) |
Net loss per share — basic and diluted (in dollars per share) | $ (1.52) | $ (1.76) | $ (1.60) |
Weighted average common shares outstanding — basic and diluted (in shares) | 74,187,251 | 68,428,046 | 55,616,092 |
CONSOLIDATED STATEMENTS OF CHAN
CONSOLIDATED STATEMENTS OF CHANGES IN STOCKHOLDERS’ EQUITY - USD ($) $ in Thousands | Total | Cumulative effect adjustment related to the adoption of ASC 842 | Collaborative Partners | Alnylam | COMMON STOCK | COMMON STOCKCollaborative Partners | COMMON STOCKAlnylam | ADDITIONAL PAID-IN CAPITAL | ADDITIONAL PAID-IN CAPITALCollaborative Partners | ADDITIONAL PAID-IN CAPITALAlnylam | ACCUMULATED DEFICIT | ACCUMULATED DEFICITCumulative effect adjustment related to the adoption of ASC 842 |
Balance at beginning of period (in shares) at Dec. 31, 2017 | 51,644,841 | |||||||||||
Balance at beginning of period at Dec. 31, 2017 | $ 101,086 | $ 5 | $ 417,037 | $ (315,956) | ||||||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||||||||
Issuance of common stock (in shares) | 8,832,565 | 6,250,019 | 983,208 | |||||||||
Issuance of common stock | 107,770 | $ 60,412 | $ 10,315 | $ 1 | $ 1 | 107,769 | $ 60,411 | $ 10,315 | ||||
Exercise of warrants of common stock (in shares) | 45,710 | |||||||||||
Exercise of warrants to purchase common stock | 49 | 49 | ||||||||||
Exercises of common stock options and sales of common stock under Employee Stock Purchase Plan (in shares) | 448,173 | |||||||||||
Exercises of common stock options and sales of common stock under Employee Stock Purchase Plan | 2,061 | 2,061 | ||||||||||
Vesting of restricted common stock (in shares) | 10,000 | |||||||||||
Settlement of restricted stock for tax withholding (in shares) | (3,774) | |||||||||||
Settlement of restricted stock for tax withholding | (35) | (35) | ||||||||||
Stock-based compensation expense | 7,888 | 7,888 | ||||||||||
Net loss | (88,853) | (88,853) | ||||||||||
Balance at end of period (in shares) at Dec. 31, 2018 | 68,210,742 | |||||||||||
Balance at end of period at Dec. 31, 2018 | 200,693 | $ (91) | $ 7 | 605,495 | (404,809) | $ (91) | ||||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||||||||
Issuance of common stock (in shares) | 2,279,982 | |||||||||||
Issuance of common stock | $ 45,828 | $ 45,828 | ||||||||||
Exercises of common stock options and sales of common stock under Employee Stock Purchase Plan (in shares) | 1,082,472 | |||||||||||
Exercises of common stock options and sales of common stock under Employee Stock Purchase Plan | 7,402 | 7,402 | ||||||||||
Stock-based compensation expense | 18,822 | 18,779 | 43 | |||||||||
Net loss | (120,459) | (120,459) | ||||||||||
Balance at end of period (in shares) at Dec. 31, 2019 | 71,573,196 | |||||||||||
Balance at end of period at Dec. 31, 2019 | $ 152,195 | $ 7 | 677,504 | (525,316) | ||||||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||||||||
Accounting standards update, extensible list | us-gaap:AccountingStandardsUpdate201602Member | |||||||||||
Issuance of common stock (in shares) | 2,077,500 | |||||||||||
Issuance of common stock | $ 39,088 | 39,088 | ||||||||||
Exercise of warrants of common stock (in shares) | 1,996,861 | |||||||||||
Exercises of common stock options and sales of common stock under Employee Stock Purchase Plan (in shares) | 2,106,517 | |||||||||||
Exercises of common stock options and sales of common stock under Employee Stock Purchase Plan | $ 20,307 | $ 1 | 20,306 | |||||||||
Stock-based compensation expense | 38,911 | 38,911 | ||||||||||
Net loss | (112,747) | (112,747) | ||||||||||
Balance at end of period (in shares) at Dec. 31, 2020 | 75,757,213 | |||||||||||
Balance at end of period at Dec. 31, 2020 | $ 137,754 | $ 8 | $ 775,809 | $ (638,063) |
CONSOLIDATED STATEMENTS OF CH_2
CONSOLIDATED STATEMENTS OF CHANGES IN STOCKHOLDERS’ EQUITY (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2018 | |
Statement of Stockholders' Equity [Abstract] | ||
Underwriting fees and issuance costs | $ 330 | |
Commissions and offering costs | $ 904 |
CONSOLIDATED STATEMENTS OF CASH
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
CASH FLOWS FROM OPERATING ACTIVITIES: | |||
Net loss | $ (112,747) | $ (120,459) | $ (88,853) |
Adjustments to reconcile net loss to net cash provided by (used in) operating activities: | |||
Non-cash litigation expense | 0 | 0 | 10,315 |
Stock-based compensation expense | 38,911 | 18,822 | 7,888 |
Change in fair value of derivative liability | 6,000 | 0 | 0 |
Depreciation and amortization expense | 2,217 | 1,254 | 774 |
Amortization of premium on investments | 373 | (3,559) | (1,126) |
Lease expense | 8,095 | 2,747 | |
Other | (460) | (191) | 12 |
Changes in operating assets and liabilities: | |||
Litigation settlement payable | 0 | (10,500) | 10,500 |
Deferred revenue | 79,784 | 211,803 | 173,916 |
Prepaid expenses and other assets | (12,511) | (3,856) | 532 |
Accounts payable | 2,208 | 2,067 | (1,217) |
Contract receivables | 165,641 | (100,354) | (100,000) |
Accrued expenses and other liabilities | 6,738 | 9,487 | 3,974 |
Lease liability | (10,420) | (8,966) | |
Deferred charges and other assets | 1,283 | 1,013 | 1,583 |
Net cash provided by (used in) operating activities | 175,112 | (692) | 18,298 |
CASH FLOWS FROM INVESTING ACTIVITIES: | |||
Maturities of held-to-maturity investments | 655,000 | 418,000 | 81,000 |
Purchases of held-to-maturity investments | (902,128) | (362,120) | (283,372) |
Purchases of property and equipment | (11,747) | (6,349) | (359) |
Other | 15 | 0 | 0 |
Net cash (used in) provided by investing activities | (258,860) | 49,531 | (202,731) |
CASH FLOWS FROM FINANCING ACTIVITIES: | |||
Proceeds from issuance of common stock, net of underwriters’ commissions | 39,192 | 0 | 108,099 |
Payments of common stock offering costs | (104) | (50) | (703) |
Proceeds from issuance of common stock to collaboration partners | 0 | 45,828 | 60,412 |
Proceeds from exercises of common stock warrants, stock options, and issuances under Employee Stock Purchase Plan | 20,380 | 7,110 | 2,110 |
Other | (45) | 0 | (35) |
Net cash provided by financing activities | 59,423 | 52,888 | 169,883 |
NET (DECREASE) INCREASE IN CASH, CASH EQUIVALENTS, AND RESTRICTED CASH EQUIVALENTS | (24,325) | 101,727 | (14,550) |
CASH, CASH EQUIVALENTS, AND RESTRICTED CASH EQUIVALENTS – Beginning of year | 156,710 | 54,983 | 69,533 |
CASH, CASH EQUIVALENTS AND RESTRICTED CASH EQUIVALENTS – End of year | 132,385 | 156,710 | 54,983 |
NONCASH OPERATING ACTIVITIES: | |||
Right-of-use assets acquired through operating leases | 32,368 | 25,725 | |
NONCASH INVESTING ACTIVITIES: | |||
Property and equipment purchases included in accounts payable and accrued expenses | 1,612 | 727 | 1,648 |
NONCASH FINANCING ACTIVITIES: | |||
Right-of-use assets acquired through finance leases | 48 | 193 | |
Common stock offering costs included in accounts payable or accrued expenses | 0 | 0 | 50 |
Cash, Cash Equivalents, Restricted Cash and Restricted Cash Equivalents [Abstract] | |||
Total cash, cash equivalents, and restricted cash equivalents shown in the consolidated statements of cash flows | $ 156,710 | $ 156,710 | $ 54,983 |
DESCRIPTION OF BUSINESS
DESCRIPTION OF BUSINESS | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
DESCRIPTION OF BUSINESS | DESCRIPTION OF BUSINESS Business Dicerna Pharmaceuticals, Inc. (“the Company” or “Dicerna”), is a biopharmaceutical company focused on discovering, developing, and commercializing medicines that are designed to leverage ribonucleic acid interference (“RNAi”) to silence selectively genes that cause or contribute to disease. Using the Company’s proprietary GalXC™ and GalXC-Plus™ RNAi technologies, Dicerna is committed to developing RNAi-based therapies with the potential to treat both rare and more prevalent diseases. By silencing disease-causing genes, Dicerna’s GalXC platform has the potential to address conditions that are difficult to treat with other modalities. Initially focused on disease-causing genes in the liver, Dicerna has continued to innovate and is exploring new applications of its RNAi technology with GalXC-Plus, which expands on the functionality and application of our flagship liver-based GalXC technology, yet has the potential to treat diseases across multiple therapeutic areas. In addition to the Company’s own pipeline of core discovery and clinical candidates, Dicerna has established collaborative relationships with some of the world’s leading pharmaceutical companies, including Novo Nordisk A/S (“Novo”), Roche, Eli Lilly and Company (“Lilly”), Alexion Pharmaceuticals, Inc. (together with its affiliates, “Alexion”), Boehringer Ingelheim International GmbH (“BI”), and Alnylam Pharmaceuticals, Inc. (“Alnylam”). Between Dicerna and its collaborative partners, the Company currently has more than 20 active discovery, preclinical, or clinical programs focused on rare, cardiometabolic, viral, chronic liver, and complement-mediated diseases, as well as neurodegenerative diseases and pain. COVID-19 On March 11, 2020, the World Health Organization declared the novel coronavirus (“COVID-19”) a pandemic. The global spread of COVID-19 has created significant volatility, uncertainty, and economic disruption worldwide. Governments in affected regions have implemented, and may continue to implement, safety precautions which include quarantines, travel restrictions, business closures, and other public health safety measures. To date, Dicerna has been impacted by mandatory work from home edicts directed by local governments in the jurisdictions in which the Company operates. However, essential work exemptions continue to permit critical research and development and laboratory activities for limited personnel. Those exemptions enable some continued discovery research and activities supporting the Company’s collaborative agreements and its own programs. Externally, the COVID-19 pandemic has resulted in slower enrollment in the Company’s clinical trials, and Dicerna has undertaken efforts to mitigate potential impacts to our business including those related to conducting clinical trials and managing our supply chain. The Company continues to be alert to the potential for disruptions that could arise from COVID-19 and monitors the Food and Drug Administration’s (“FDA”) and other health authorities’ guidance for the conduct of clinical trials during this time. Current supply of Dicerna’s investigational medicines is sufficient to support ongoing and planned clinical trials. Based on current evaluations, Dicerna’s supply chains continue to appear intact to meet at least the next 12 months of clinical, nonclinical, and chemistry, manufacturing, and control (“CMC”) supply demands across all programs. The Company has undertaken efforts to mitigate potential future impacts to the supply chain by increasing its stock of critical starting materials required to meet its needs and its collaborative partners’ needs through 2021 and by identifying and engaging alternative suppliers. The Company continues to be alert to the potential for disruptions that could arise from COVID-19, including on account of United States (“U.S.”) government utilization of its Defense Production Act authorities, and remains in close contact with suppliers. It is difficult to predict what the lasting impact of the pandemic will be, and what the impact might be if the Company or any of the third parties with whom it engages were to experience additional shutdowns or other prolonged business disruptions. The Company’s ability to conduct its business in the manner and on the timelines presently planned could have a material adverse impact on the Company’s business, results of operations, and financial condition. In addition, depending on the duration and impact of the recurrence or resurgence of COVID-19 cases or continued evolution of further strains of COVID-19, and depending on where the infection rates are highest, and including the ability of regulators to continue ensuring the timely review and approval of applications, the Company’s business, results of operations, and financial condition may be negatively impacted. The Company will continue to monitor developments as it deals with the disruptions and uncertainties relating to the COVID-19 pandemic. |
SUMMARY OF SIGNIFICANT ACCOUNTI
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES Basis of presentation The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”) and include the accounts of Dicerna Pharmaceuticals, Inc. and its wholly owned subsidiaries. The Company believes that the financial statements as presented reflect all normal recurring adjustments necessary for a fair statement of the information for the periods presented. All intercompany balances and transactions have been eliminated in consolidation. Significant judgments and estimates The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the Company’s consolidated financial statements, as well as the revenues and expenses incurred during the reporting periods. On an ongoing basis, the Company evaluates judgments and estimates, including those related to revenue recognition, stock-based compensation, the derivative liability, and accrued expenses. The Company bases its estimates on historical experience and on various other factors that the Company believes are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not apparent from other sources. Changes in estimates are reflected in reported results for the period in which they become known. Actual results could differ materially from those estimates. Cash and cash equivalents Cash and cash equivalents includes all highly liquid investments, including money market funds, maturing within 90 days from the date of purchase. Restricted cash equivalents Restricted cash equivalents are money market funds held in collateral accounts that are restricted to secure letters of credit for corporate lease activity. The letters of credit are required to be maintained throughout the terms of the leases. Held-to-maturity investments The Company invests its excess cash balances in short-term and long-term fixed-income investments. The Company determines the appropriate classification of investments at the time of purchase and re-evaluates such designation as of each balance sheet date. Debt securities carried at amortized cost are classified as held-to-maturity when the Company has the positive intent and ability to hold the securities to maturity. The contractual terms of these investments do not permit the issuer to settle the securities at a price less than the amortized cost bases of the investments. The Company does not intend to sell its investments and the Company has the intent and ability to hold its investments until they mature. The Company’s investment policy mandates that, at the time of purchase, the maturity of each investment within its portfolio shall not exceed two years. In addition, the weighted average maturity of the investment portfolio must not exceed one year. The Company’s policy is not to measure an allowance for credit losses for interest receivable and to write off any uncollectible interest receivable as a reversal of interest income in the period in which it determines the interest will not be collected. The Company did not write off any accrued interest receivables during the years ended December 31, 2020 and 2019. Concentrations of credit risk and significant customers Financial instruments that subject the Company to significant concentrations of credit risk consist of cash, cash equivalents, restricted cash equivalents, held-to-maturity investments, contract receivables, and the withholding tax receivable (see Note 8 – Collaborative Research and License Agreements). All of the Company’s cash, cash equivalents, restricted cash equivalents, and held-to-maturity investments are invested in money market funds or U.S. treasury securities that management believes to be of high credit quality. The Company’s revenues for the years ended December 31, 2020, 2019, and 2018 are primarily related to the Company’s collaboration agreements which are concentrated among a few collaboration partners. All revenues recognized by the Company to date were earned in the United States. Refer to Note 8 – Collaborative Research And License Agreements for composition of significant collaboration relationships. The Company does not currently own or operate any manufacturing facilities for the production of preclinical, clinical, or commercial quantities of any of its product candidates. For each product candidate, the Company currently contracts with manufacturers and expects to continue to do so to meet the preclinical and clinical requirements of its product candidates. Fair Value Measurements Fair value is an exit price, representing the amount that would be received from the sale of an asset or paid to transfer a liability in an orderly transaction between market participants. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. Valuation techniques used to measure fair value are performed in a manner to maximize the use of observable inputs and minimize the use of unobservable inputs. As a basis for considering such assumption, the accounting literature establishes a three-tier value hierarchy which prioritizes the inputs used in measuring fair value as follows: • Level 1 – observable inputs, such as quoted prices in active markets; • Level 2 – inputs other than the quoted prices in active markets that are observable either directly or indirectly; and • Level 3 – unobservable inputs for which there is little or no market data, which requires the Company to develop its own assumptions. Property and equipment Property and equipment are stated at cost. Major betterments are capitalized, whereas expenditures for maintenance and repairs which do not improve or extend the life of the respective assets are charged to operations as incurred. Depreciation is calculated and applied using the straight-line method over the estimated useful lives, as shown below: ASSET CATEGORY ESTIMATED Laboratory equipment 5 years Office and computer equipment 3 - 5 years Furniture and fixtures 5 years Leasehold improvements 5 years or the remaining term of lease, if shorter Construction-in-process is stated at cost, which includes the cost of construction and other direct costs attributable to the construction. No provision for depreciation and amortization expense is recorded related to construction-in-process until the relevant assets are completed and put into use. At December 31, 2020, the balance of construction-in-process includes construction costs for lessee-owned improvements for leased premises and costs associated with laboratory equipment under installation. Cloud computing arrangements The Company adopted Accounting Standards Update (“ASU”) 2018-15, Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That is a Service Contract on January 1, 2020. ASU 2018-15 aligns the requirements for capitalizing implementation costs in a cloud computing arrangement service contract with the requirements for capitalizing implementation costs incurred for internal-use software licenses. Eligible costs associated with cloud computing arrangements, such as software business applications used in the normal course of business, are capitalized in accordance with Accounting Standards Codification (“ASC”) 350 – Intangibles – Goodwill and Other . The Company’s cloud computing arrangements relate to various enterprise resource planning systems. These assets are included in property and equipment, net in the consolidated balance sheets and are amortized on a straight-line basis over their assessed useful lives or the term of the underlying cloud computing hosting contract, whichever is shorter. As of December 31, 2020, the estimated useful lives of these assets were three years. As of December 31, 2020, cloud computing arrangement assets consisted of capitalized implementation costs of $2.7 million. No amortization expense associated with the Company’s cloud computing arrangements has been recognized during the year ended December 31, 2020. The Company evaluates the useful lives of these assets on an annual basis and tests for impairment whenever events or changes in circumstances indicate that the carrying amount may not be recoverable. Impairment of long-lived assets Long-lived assets are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amount of the asset may not be recoverable. When such events occur, the Company compares the carrying amounts of the assets to their undiscounted expected future cash flows. If this comparison indicates that there is an impairment, the amount of the impairment is calculated as the difference between the carrying value and fair value of the related asset. During the years ended December 31, 2020, 2019, and 2018, no impairments were recorded. Leases On January 1, 2019, the Company adopted the new lease standard, Accounting Standards Codification (“ASC”) 842, Leases, which is intended to increase transparency and comparability among organizations by requiring lessees to recognize most leases on their balance sheets and making targeted changes to lessor accounting. The most significant change arising from the new standard is the recognition of right-of-use (“ROU”) assets and lease liabilities for leases classified as operating leases. Under the standard, disclosures are required to enable financial statement users to assess the amount, timing, and uncertainty of cash flows arising from the leases. Initial implementation of the standard did not have a material impact on the consolidated financial statements, but required additional disclosures. The Company elected the package of three practical expedients that permitted an entity not to (a) reassess whether expired or existing contracts contain leases, (b) reassess lease classification for existing or expired leases, and (c) consider whether previously capitalized initial direct costs would be appropriate under the new standard. In adopting ASC 842, the Company elected not to bifurcate payments between lease and nonlease components associated with leases for office and laboratory real estate. The Company determines if an arrangement is a lease at inception. Leases with a term greater than one year are presented on the balance sheet as ROU assets, lease liabilities and, if applicable, long-term lease liabilities. The Company has elected not to recognize leases with terms of one year or less on its balance sheet. At the commencement date, operating lease liabilities and their corresponding ROU assets are recorded based on the present value of future lease payments over the expected lease term. Certain adjustments to the ROU asset may be required for items such as initial direct costs paid or incentives received. Operating lease cost is recognized over the expected term on a straight-line basis. The Company determines the expected term for its operating leases considering the noncancelable period of the lease, plus any additional periods covered by either (a) a Company option to extend (or not to terminate) the lease that the Company is reasonably certain to exercise, or (b) an option to extend (or not to terminate) the lease controlled by the lessor. Derivative liability The derivative liability is recorded at fair value, which is estimated using a Monte Carlo simulation for estimated future contingent royalty and milestone payments. The liability is measured quarterly with any change in fair value recorded in other income (expense) in the consolidated statements of operations. Segment and geographic information Operating segments are defined as components (business activity from which it earns revenue and incurs expenses) of an enterprise about which discrete financial information is available and regularly reviewed by the chief operating decision maker in deciding how to allocate resources and in assessing performance. The Company, through its Chief Executive Officer in his role as chief operating decision maker, views Company operations and manages the business as one operating segment. All long-lived assets of the Company are located in the United States. Research and development costs Research and development costs consist of expenses incurred in performing research and development activities, including compensation and benefits for full-time research and development employees, an allocation of facility expenses and overhead expenses, and other external expenses. Research and development costs are expensed as incurred and were $205.4 million, $109.3 million and $45.7 million for the years ending December 31, 2020, 2019, and 2018, respectively. Research and development costs that are paid in advance of performance are deferred as a prepaid expense and amortized over the service period as the services are provided. Grants and credits The Company sometimes receives assistance from third-party entities such as governmental or non-profit agencies. When assistance is received from a governmental entity, the Company first determines whether the payment represents revenue by considering factors such as whether a commercial purpose exists for the payments and whether the required activity to qualify for the assistance relates to the Company’s ongoing activities. If the Company concludes that the assistance is revenue, the Company applies ASC 606, Revenue from Contracts with Customers . If the assistance is in the form of an income tax credit, the Company applies the guidance in ASC 740, Income Taxes . When the Company determines that the assistance is not revenue and does not fall within the scope of ASC 740, it applies International Accounting Standard 20, Accounting for Government Grants and Disclosure of Government Assistance . Typically, government grants may be considered related to assets or related to income. The Company generally records grants from governmental and non-profit agencies related to income as a reduction in research and development expense. Grants are recognized when there is reasonable assurance that the Company will comply with the conditions attached to the grant arrangement and the grant will be received. Grant payments received related to research and development costs incurred prior to the approval of the qualifying program are recognized immediately upon approval of the program by the grantor. Revenue recognition The Company generates revenue from research collaboration and license agreements with customers. Goods and services in the agreements may include the grant of licenses for the use of the Company’s technology, the provision of services associated with the research and development of product candidates, manufacturing services, and participation on joint steering committees. Such agreements may provide for consideration to the Company in the form of upfront payments; funding or reimbursement of research and development services; reimbursement of certain costs; option exercise payments; payments due upon the achievement of research, development, regulatory, and commercial-based milestones; and royalty payments on licensed products. On January 1, 2018, the Company adopted ASC 606, Revenue from Contracts with Customers , which amended revenue recognition principles and provides a single, comprehensive set of criteria for revenue recognition. The new revenue standard applies to all contracts with customers except for contracts that are within the scope of other standards. The new guidance provides a five-step framework through which revenue is recognized when control of promised goods or services is transferred to a customer at an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. To determine revenue recognition for arrangements that the Company concludes are within the scope of the new revenue recognition standard, management performs the following five steps: (i) identifies the contract(s) with a customer; (ii) identifies the performance obligations in the contract; (iii) determines the transaction price, including whether there are any constraints on variable consideration; (iv) allocates the transaction price to the performance obligations; and (v) recognizes revenue when (or as) the Company satisfies a performance obligation. At contract inception, once a contract is determined to be within the scope of the new revenue standard, Dicerna assesses whether individual goods or services promised within each contract are distinct and, therefore, represent a separate performance obligation. Goods and services that are determined not to be distinct are combined with other promised goods or services until a distinct bundle is identified. Dicerna allocates the transaction price (the amount of consideration to which the Company expects to be entitled in exchange for the promised goods or services) to each performance obligation and recognizes the associated revenue when (or as) each performance obligation is satisfied. The Company’s estimate of the transaction price for each contract includes all variable consideration to which Dicerna expects to be entitled at each measuring period. When two or more contracts are entered into with the same customer at or near the same time, the Company evaluates the contracts to determine whether the contracts should be accounted for as a single arrangement. Contracts are combined and accounted for as a single arrangement if one or more of the following criteria are met: (i) the contracts are negotiated as a package with a single commercial objective; (ii) the amount of consideration to be paid in one contract depends on the price or performance of the other contract; or (iii) the goods or services promised in the contracts (or some goods or services promised in each of the contracts) are a single performance obligation. The evaluation of whether promised goods or services represent distinct performance obligations is subjective and requires the Company to make judgments about the promised goods and services and whether such goods and services are separable from the other aspects of the contract(s). The transaction price is allocated among the performance obligations on a relative standalone selling price basis, and the applicable revenue recognition criteria are applied to each of the separate performance obligations. The Company may estimate the standalone selling price using a residual method when the selling price is highly variable because a representative standalone selling price is not discernible from past transactions or other observable evidence, or when the selling price is uncertain. Determining the standalone selling price for performance obligations requires significant judgment. When an observable price of a promised good or service is not readily available, the Company considers relevant assumptions to estimate the standalone selling price, including, as applicable, market conditions, development timelines, probabilities of technical and regulatory success, reimbursement rates for personnel costs, forecasted revenues, potential limitations to the selling price of the product, and discount rates. The Company applies judgment in determining whether a combined performance obligation is satisfied at a point in time or over time, and, if over time, concluding upon the appropriate method of measuring progress to be applied for purposes of recognizing revenue. The Company evaluates the measure of progress each reporting period and, as estimates related to the measure of progress change, related revenue recognition is adjusted accordingly. Changes in the Company’s estimated measure of progress are accounted for on a cumulative catch-up basis as a change in accounting estimate and are recorded through earnings in the period of adjustment. The Company receives payments from its licensees as established in each contract. Upfront payments and fees are recorded as deferred revenue upon receipt or when due and most often require deferral of revenue recognition to a future period until the Company performs its obligations under the underlying arrangements. Where applicable, amounts are recorded as contracts receivable when the Company’s right to consideration is unconditional. Licenses of intellectual property If a license granted to a customer to use the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenue from consideration allocated to the license when the license is transferred to the licensee and the licensee is able to use and benefit from the license. For licenses that are bundled with other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, to conclude upon the appropriate method of measuring progress for purposes of recognizing revenue related to consideration allocated to the performance obligation. Research and development services Arrangements that include a promise for the Company to provide research or development services are assessed to determine whether the services are capable of being distinct, are not highly interdependent or do not significantly modify one another, and if so, the services are accounted for as a separate performance obligation as the services are provided to the customer. Otherwise, when research or development services are determined not to be capable of being distinct, such services are added to the performance obligation that includes the underlying license. For research and development services that are bundled with other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, to conclude upon the appropriate method of measuring progress for purposes of recognizing revenue related to consideration allocated to the performance obligation. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. Options Customer options, such as options granted to allow a licensee to choose to research and develop additional product candidates or reserve product candidates against target genes to be identified in the future, or options that allow a customer to designate a target as a lead product, are evaluated at contract inception in order to determine whether those options provide a material right (i.e., an optional good or service offered for free or at a discount) to the customer. If the customer option represents a material right, the material right is treated as a separate performance obligation at the outset of the arrangement. The Company allocates the transaction price to material rights based on the standalone selling price, and revenue is recognized when or as the future goods or services are transferred or when the option expires. Customer options that are not material rights do not give rise to separate performance obligations, and as such, the additional consideration that would result from a customer exercising an option in the future is not included in the transaction price for the current contract. Instead, the option is deemed a marketing offer, and additional option fee payments are recognized or begin being recognized as revenue when the licensee exercises the option. The exercise of an option that does not represent a material right is treated as a separate contract for accounting purposes. Milestone payments At the inception of each contract with a customer that includes development or regulatory milestone payments, the Company evaluates whether the milestones are considered probable of being reached and estimates the amount to be included in the transaction price using the most likely amount method. If the Company concludes it is probable that a significant revenue reversal would not occur, the associated milestone payment is included in the transaction price. Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are generally not considered probable of being achieved until those approvals are received. The transaction price is then allocated to each performance obligation on a relative stand-alone selling price basis, for which the Company recognizes revenue as or when the performance obligations under the contract are satisfied. At the end of each subsequent reporting period, the Company re-evaluates the probability of achievement of all milestones and any related constraints, and, if necessary, adjusts the estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis and are recorded as revenue and through earnings in the period of adjustment. Royalties For arrangements that include sales-based royalties, including milestone payments based on the level of sales, and when the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). Contract costs The Company recognizes as an asset the incremental costs of obtaining a contract with a customer if the costs are expected to be recovered. The Company has elected a practical expedient wherein it recognizes the incremental costs of obtaining a contract as an expense when incurred if the amortization period of the asset that it otherwise would have recognized is one year or less. To date, the Company has not incurred any incremental costs of obtaining a contract with a customer. Contract modifications Contract modifications, defined as changes in the scope or price (or both) of a contract that are approved by the parties to the contract, such as a contract amendment, exist when the parties to a contract approve a modification that either creates new or changes existing enforceable rights and obligations of the parties to the contract. Depending on facts and circumstances, the Company accounts for a contract modification as one of the following: (i) a separate contract; (ii) a termination of the existing contract and a creation of a new contract; or (iii) a combination of the preceding treatments. A contract modification is accounted for as a separate contract if the scope of the contract increases because of the addition of promised goods or services that are distinct and the price of the contract increases by an amount of consideration that reflects the Company’s standalone selling prices of the additional promised goods or services. When a contract modification is not considered a separate contract and the remaining goods or services are distinct from the goods or services transferred on or before the date of the contract modification, the Company accounts for the contract modification as a termination of the existing contract and a creation of a new contract. When a contract modification is not considered a separate contract and the remaining goods or services are not distinct, the Company accounts for the contract modification as an add-on to the existing contract and as an adjustment to revenue on a cumulative catch-up basis. The Company receives payments from its licensees as established in each contract. Upfront payments and fees are recorded as deferred revenue upon receipt or when due and may require deferral of revenue recognition to a future period until the Company performs its obligations under these arrangements. Where applicable, amounts are recorded as contracts receivable when the Company’s right to consideration is unconditional. The Company does not assess whether a contract with a customer has a significant financing component if the expectation at contract inception is such that the period between payment by the licensees and the transfer of the promised goods or services to the licensees will be one year or less. Stock-based compensation The Company’s stock-based compensation cost is measured at the grant date of the stock-based award based on the fair value of the award and is recognized as expense over the requisite service period, which generally represents the vesting period, and includes an estimate of the awards that will be forfeited. The Company uses the Black-Scholes valuation model for estimating the fair value of stock options. The fair value of stock option awards is affected by the valuation assumptions, including the expected volatility based on comparable market participants, expected term of the stock option, risk-free interest rate, and expected dividends. Income taxes The Company records deferred tax assets and liabilities for the expected future tax consequences of temporary differences between the Company’s financial statement carrying amounts and the tax basis of assets and liabilities using enacted tax rates expected to be in effect in the years in which the differences are expected to reverse. A valuation allowance is provided to reduce the net deferred tax assets to the amount that will more likely than not (more than 50 percent) be realized. The Company also assesses the probability that the positions taken or expected to be taken in its income tax returns will be sustained by taxing authorities. A “more likely than not” recognition threshold must be met before a tax benefit can be recognized. Tax positions that are more likely than not to be sustained are reflected in the Company’s consolidated financial statements. Tax positions are measured as the largest amount of tax benefit that is greater than 50 percent likely of being realized upon settlement with a taxing authority that has full knowledge of all relevant information. The difference between the benefit recognized for a position and the tax benefit claimed on a tax return is referred to as an unrecognized tax benefit. Potential interest and penalties associated with such uncertain tax positions are recorded as a component of income tax expense. Net loss per common share The Company computes basic net loss per common share by dividing net loss by the weighted average number of common shares outstanding. In periods of net income, the Company’s accounting policy includes allocating a proportional share of net income to participating securities, as determined by dividing total weighted average participating securities by the sum of the total weighted average common shares and participating securities (the “two-class method”). Participating securities have the effect of diluting both basic and diluted earnings per share during periods of income. During periods when the Company incurs a net loss, the Company does not allocate a loss to participating securities because they have no contractual obligation to share in the losses of the Company. The Company computes diluted net loss per common share after giving consideration to the dilutive effect of stock options, warrants, and nonvested restricted stock units that are outstanding during the period, except where such non-participating securities would be anti-dilutive. The outstanding securities presented below were excluded from the calculation of net loss per share because such securities would have been anti-dilutive due to the Company’s net loss per share during the periods ending on the dates presented. DECEMBER 31, 2020 2019 2018 Options to purchase common stock 14,323,689 12,467,150 7,787,690 Nonvested restricted common stock 1,055,405 — — Warrants to purchase common stock — 2,198 2,198 Total 15,379,094 12,469,348 7,789,888 Recent accounting pronouncements In December 2019, the Financial Accounting Standards Board (“FASB”) issued ASU 2019-12, Simplifying the Accounting for Income Taxes , amending accounting guidance that simplifies the accounting for income taxes as part of its initiative to reduce complexity in the accounting standards. The amendments eliminate certain exceptions related to the approach for intraperiod tax allocation, the methodology for calculating income taxes in an interim period, and the recognition of deferred tax liabilities for outside basis differences. |
HELD-TO-MATURITY INVESTMENTS
HELD-TO-MATURITY INVESTMENTS | 12 Months Ended |
Dec. 31, 2020 | |
Investments, Debt and Equity Securities [Abstract] | |
HELD-TO-MATURITY INVESTMENTS | HELD-TO-MATURITY INVESTMENTS A summary of the Company’s held-to-maturity investments is presented below: DECEMBER 31, 2020 DESCRIPTION AMORTIZED COST GROSS UNREALIZED GAINS GROSS LOSSES FAIR VALUE U.S. Treasury securities maturing in one year or less $ 442,820 $ 163 $ (12) $ 442,971 DECEMBER 31, 2019 DESCRIPTION AMORTIZED COST GROSS UNREALIZED GAINS GROSS LOSSES FAIR VALUE U.S. Treasury securities maturing in one year or less $ 196,065 $ 160 $ (6) $ 196,219 |
FAIR VALUE MEASUREMENTS
FAIR VALUE MEASUREMENTS | 12 Months Ended |
Dec. 31, 2020 | |
Fair Value Disclosures [Abstract] | |
FAIR VALUE MEASUREMENTS | FAIR VALUE MEASUREMENTS A summary of the Company’s assets and liabilities that are measured or disclosed at fair value on a recurring basis is presented below: DECEMBER 31, 2020 DESCRIPTION TOTAL FAIR VALUE LEVEL 1 LEVEL 2 LEVEL 3 Financial assets Cash equivalents Money market funds $ 126,006 $ 126,006 $ — $ — Held-to-maturity investments U.S. Treasury securities 442,971 — 442,971 — Restricted cash equivalents Money market funds 6,362 6,362 — — Total financial assets $ 575,339 $ 132,368 $ 442,971 $ — Financial liabilities Derivative liability 6,000 — — 6,000 Total financial liabilities $ 6,000 $ — $ — $ 6,000 DECEMBER 31, 2019 DESCRIPTION TOTAL FAIR VALUE LEVEL 1 LEVEL 2 LEVEL 3 Financial assets Cash equivalents Money market funds $ 152,903 $ 152,903 $ — $ — Held-to-maturity investments U.S. Treasury securities 196,219 — 196,219 — Restricted cash equivalents Money market funds 3,894 3,894 — — Total financial assets $ 353,016 $ 156,797 $ 196,219 $ — The Company’s cash equivalents and restricted cash equivalents, which are held in money market funds, are classified within Level 1 of the fair value hierarchy because they are valued using quoted prices in active markets as of December 31, 2020 and 2019. Restricted cash equivalents represent money market investments which secure letters of credit established in connection with the Company’s facility leases. The Company’s held-to-maturity investments bore interest at the prevailing market rates for instruments with similar characteristics and therefore the amortized cost approximated fair value. These financial instruments were classified within Level 2 of the fair value hierarchy because the inputs to the fair value measurements were valued using observable inputs as of December 31, 2020 and 2019. The Company’s derivative liability associated with certain contingent payments under our collaboration agreement with Alnylam is classified within Level 3 of the fair value hierarchy because the fair value utilizes unobservable inputs for which there is no market data and therefore requires the Company to develop its own assumptions. Such assumptions include the probability of success of development, the probability that Alnylam exercises its commercialization option, the timing of regulatory approval and the first commercial sale, and the volume of sales. As of December 31, 2020 and 2019, the Company’s contract receivables, accounts payable, and accrued expenses approximated their estimated fair values because of the short-term nature of these financial instruments. |
PREPAID EXPENSES AND OTHER CURR
PREPAID EXPENSES AND OTHER CURRENT ASSETS | 12 Months Ended |
Dec. 31, 2020 | |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | |
PREPAID EXPENSES AND OTHER CURRENT ASSETS | PREPAID EXPENSES AND OTHER CURRENT ASSETS Prepaid expenses and other current assets consist of the following: DECEMBER 31, 2020 2019 Prepaid clinical, contract research, and manufacturing costs $ 9,651 $ 4,288 Interest receivable 1,345 918 Prepaid insurance 817 583 Other prepaid expenses and other current assets 2,590 1,145 Prepaid expenses and other current assets $ 14,403 $ 6,934 |
PROPERTY AND EQUIPMENT, NET
PROPERTY AND EQUIPMENT, NET | 12 Months Ended |
Dec. 31, 2020 | |
Property, Plant and Equipment [Abstract] | |
PROPERTY AND EQUIPMENT, NET | PROPERTY AND EQUIPMENT, NET Property and equipment, net, consists of the following: DECEMBER 31, 2020 2019 Laboratory equipment $ 8,637 $ 9,147 Office and computer equipment 2,049 2,425 Furniture and fixtures 1,251 1,569 Leasehold improvements 1,003 257 Construction-in-process 10,038 238 Property and equipment, at cost 22,978 13,636 Less: accumulated depreciation and amortization expense (5,432) (6,560) Property and equipment, net $ 17,546 $ 7,076 Depreciation and amortization expense was $2.2 million, $1.3 million, and $0.8 million for the years ended December 31, 2020, 2019, and 2018. |
ACCRUED EXPENSES AND OTHER CURR
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | 12 Months Ended |
Dec. 31, 2020 | |
Payables and Accruals [Abstract] | |
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES Accrued expenses and other current liabilities consist of the following: DECEMBER 31, 2020 2019 Accrued clinical, contract research, and manufacturing costs $ 9,297 $ 10,347 Accrued compensation and other employee-related benefits 14,031 7,298 Accrued professional fees 2,117 1,519 Other accrued expenses and current liabilities 2,616 878 Total 28,061 $ 20,042 |
COLLABORATIVE RESEARCH AND LICE
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS | 12 Months Ended |
Dec. 31, 2020 | |
Revenue from Contract with Customer [Abstract] | |
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS | COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS Alnylam collaboration and patent cross-license agreements Background On April 3, 2020, the Company and Alnylam (collectively, the “parties”) entered into a Collaboration and License Agreement (the “A1AT Agreement”) and a Patent Cross License Agreement (the “PH Agreement”). Pursuant to the A1AT Agreement, Dicerna will lead efforts on investigational RNAi therapeutics for the treatment of alpha-1 antitrypsin (“AAT”) deficiency (“AATD”)-associated liver disease (“AATLD”). Pursuant to the PH Agreement, the parties completed a cross-license of their respective intellectual property for Alnylam’s lumasiran and Dicerna’s nedosiran investigational programs for the treatment of primary hyperoxaluria (“PH”). No upfront cash consideration was exchanged in either transaction. Pursuant to the A1AT Agreement, Alnylam’s AAT product (ALN-AAT02, or the “Alnylam Product”) and Dicerna’s AAT product (belcesiran, or the “Dicerna Product”) would be explored for the treatment of AATLD. Under the A1AT Agreement, the Company obtained an exclusive worldwide license to Alnylam’s intellectual property to exploit the Alnylam Product. Dicerna assumed responsibility for the development of both the Alnylam Product and the Dicerna Product at its cost. Dicerna selected belcesiran to advance in development for the treatment of patients with AATLD. At the completion of Phase 3, Alnylam has the no-cost opportunity to opt-in to commercialize belcesiran in countries outside the U.S. where it already has a commercialization infrastructure in place (the “Commercialization Option”). If Alnylam exercises its Commercialization Option, the parties will share future development costs. Further, each party will pay tiered royalties to the other party based on a percentage of net product sales generated in its territory ranging from low single-digits to high teens. In the event Alnylam waives its Commercialization Option, the Company will retain worldwide rights to commercialize belcesiran in exchange for payments upon the satisfaction of certain milestones, up to an aggregate of $45.0 million, and royalties will be payable to Alnylam based on net product sales in the low to mid-single-digits. As a result of these uncertain contingent payments the Company may owe to Alnylam, the Company recorded a derivative liability on the consolidated balance sheet for the year ended December 31, 2020. The A1AT Agreement is subject to customary termination provisions, and the Company may terminate the A1AT Agreement at any time without cause following the notice period described in the A1AT Agreement. Pursuant to the PH Agreement, the parties granted to each other a perpetual non-exclusive cross-license to their respective intellectual property related to their respective PH treatment investigational programs to ensure that each party has the freedom to develop and commercialize its respective product with Alnylam’s lumasiran targeting glycolate oxidase (“GO”) for the treatment of PH type 1 and Dicerna’s nedosiran targeting lactate dehydrogenase A (“LDHA”) for the treatment of PH types 1, 2, and 3. Each party will have sole discretion concerning the research and development of its products in the field. In exchange for the license, Alnylam is required to pay mid- to high-single-digit royalties to Dicerna based on global net sales of lumasiran and Dicerna is required to pay low-single-digit royalties to Alnylam on global net sales of nedosiran. The PH Agreement cannot be terminated by either party for the other party’s breach. However, either party may terminate the PH Agreement or may reduce the royalty payable to the other party upon a patent-related challenge by the other party unless the challenge is withdrawn and no longer pending within the time periods specified in the PH Agreement. Further, the PH Agreement will terminate upon the expiration of the last-to-expire patent rights licensed thereunder. Accounting Analysis The Company determined that the A1AT Agreement and the PH Agreement represent separate agreements for accounting purposes, as the transactions have different commercial objectives, the consideration under each contract is not dependent upon the price or performance of the other contract, and the goods or services under each contract are separate performance obligations. A1AT Agreement The Company concluded that the A1AT Agreement was within the scope of ASC 606, Contracts with Customers , as the provision of research and development services is considered an output of the entity’s ordinary activities in exchange for consideration. The Company identified a single performance obligation under the A1AT Agreement, which consists of the provision of certain nonclinical and clinical services through the completion of the Phase 1 clinical trial for any product. The Company determined that the transaction price at inception of the A1AT Agreement consists of non-cash consideration in the form of the license received from Alnylam. The Company determined that the fair value of the non-cash consideration (the license received from Alnylam) is insignificant given the early-stage development status of the Alnylam Product and the related risks associated with developing a commercial product. The sales-based royalties that Dicerna may be entitled to receive in the event that Alnylam exercises its Commercialization Option have been excluded from the transaction price and will be recognized only if Alnylam exercises its Commercialization Option and the related sales occur. As described above, Dicerna may be required to pay contingent milestones and royalties to compensate Alnylam for the license provided under the agreement. Given the uncertainty associated with these contingent payments, the Company recognized $6.0 million of other expense in the three months ended December 31, 2020. The Company has recorded development costs incurred under the A1AT Agreement as research and development expenses in the Company’s consolidated statement of operations. PH Agreement The Company concluded that the PH Agreement is within the scope of ASC 610, Other Income – Gains and Losses from the Derecognition of Nonfinancial Assets , as the exchange of non-exclusive licenses is considered an exchange of non-financial assets outside the ordinary scope of business. Pursuant to ASC 610-20, the Company applied the guidance in ASC 606 to determine if a contract exists, identify the distinct non-financial assets, and determine when control transfers and, therefore, when to derecognize the asset. Additionally, the Company applied the measurement principles of ASC 606 to determine the amount of consideration, if any, to include in the calculation of the gain or loss for the non-financial asset. The Company determined that it transferred control of a non-financial asset (the non-exclusive license granted to Alnylam) at contract inception. Applying the non-cash consideration guidance in ASC 606, the Company further determined that the fair value of the non-financial asset received (the non-exclusive license from Alnylam) was insignificant. Therefore, the Company concluded that no gain or loss would be recorded related to the PH Agreement at contract inception. The Company has recorded costs related to its PH program as research and development expenses in the Company’s consolidated statement of operations. Novo collaboration and share purchase agreements Background On November 15, 2019, Dicerna and Novo entered into a Collaboration and License Agreement (the “Novo Collaboration Agreement”). Under the terms of the Novo Collaboration Agreement, the Company and Novo will seek to use GalXC to explore more than 30 gene targets associated with liver disease with the goal of delivering multiple clinical candidates for disorders including chronic liver disease, non-alcoholic steatohepatitis (“NASH”), type 2 diabetes, obesity, and rare diseases. The Company will conduct and fund discovery and preclinical development to clinical candidate selection for each liver cell target. Novo will be responsible for all further development and the commercialization of each candidate selected for development, with the Company manufacturing clinical candidates selected for Phase 1-related clinical development, subject to reimbursement for its manufacturing costs. In addition, the Company will assist Novo with the Investigational New Drug (“IND”) filing for the first development candidate. The Company also retains the ability to opt in to co-development of a total of two programs during clinical development in Phases 1-3, subject to limitations in the event of a change in control. If the Company exercises a co-development option, it also has an option to co-promote the product in the United States, subject to limitations in the event of a change in control of the Company. Additionally, the Company may lead the development and commercialization of two programs targeting orphan liver diseases, with Novo retaining the ability to opt in to both programs in Phases 1-3. The Company and Novo will share in profits and losses for the Company’s orphan liver and Novo products should both parties elect to co-develop. The Company is working exclusively with Novo during the research collaboration period on the discovery, research, development, and commercialization of hepatocyte targets subject to certain exclusions including those targets subject to the Company’s existing partnerships, and Novo is, during a specified discovery period, working exclusively with the Company in any new research and development of compounds and products directed to collaboration targets using small interfering RNA (“siRNA”) conjugated to the sugar N-acetyl-D-galactosamine (“GalNAc”) to reduce the expression of specific target genes in the liver. Under the Novo Collaboration Agreement, the Company is providing Novo with exclusive and non-exclusive licenses and manufacturing support to enable Novo to commercialize products derived from or containing compounds developed pursuant to such agreement. Under the terms of the Novo Collaboration Agreement, Novo paid the Company an upfront payment of $175.0 million, subject to delivery of target information, in January 2020. The Company is also eligible to receive an additional $75.0 million ($25.0 million at the end of each of the first three years of the Novo Collaboration Agreement), contingent upon the Company delivering GalXC molecules for a defined number of targets on an annual basis, and additional payments totaling up to approximately $357.5 million per target upon achievement of specified development, regulatory, and commercial milestones. In addition, Novo will pay the Company mid-single-digits to mid-teens royalties on product sales on a country-by-country and product-by-product basis until the later of 10 years after the date of first commercial sale of each product in such country, expiration of specified patent rights in such country, or the expiration of specified regulatory exclusivity in such country for GalXC products, subject to royalty step-down provisions set forth in the agreement. In connection with the Novo Collaboration Agreement, the Company and Novo entered into the Novo Share Issuance Agreement on November 15, 2019, pursuant to which the Company agreed to issue to Novo 2,279,982 shares (the “Novo Shares”) of the Company’s common stock, par value $0.0001 per share (“Common Stock”), at a purchase price of $21.93 per share, for an aggregate purchase price of approximately $50.0 million. During the fourth quarter of 2020, Novo nominated its first candidate under the Novo Collaboration Agreement. In conjunction with the nomination of the first development candidate, Dicerna earned a $2.5 million milestone, which the Company received in February 2021. Also during the fourth quarter of 2020, Novo confirmed that Dicerna met its annual obligation to deliver GalXC molecules for a defined number of targets for the first year of the Novo Collaboration Agreement, entitling the Company to a $25.0 million payment, which the Company received in February 2021. Accounting Analysis The Novo Collaboration Agreement and the Novo Share Issuance Agreement (collectively, “the Novo Agreements”) were executed on the same date and negotiated as a package. Management therefore concluded that the Novo Agreements are to be combined for accounting purposes and concluded that Novo is a customer in this arrangement pursuant to the revenue recognition guidance. The Company identified contract promises under the agreement for the license of intellectual property and know-how rights for selected gene targets and research and development services to develop a clinical candidate for each selected gene target, including manufacturing activities. The Company may also be required to provide research and development services for an unspecified number of targets, with the goal of the collaboration being to develop clinical candidates for each of the selected gene targets. The Company determined that the license and research and development services were not capable of being distinct or distinct within the context of the contract. The research and development services to be provided by Dicerna are specialized in nature, specifically with respect to the Company’s therapeutic expertise related to RNAi and the Company’s GalXC conjugates. In addition, there is an interdependent relationship between the contract promises. As such, the Company concluded that there is a single identified combined performance obligation consisting of a license and research and development services. The Company may be required to perform certain additional services after Novo’s nomination of a development candidate. These services include Phase 1-related activities, such as manufacturing through the approval of an IND application for a development candidate, research and development activities to support the filing of an IND application for the first development candidate, and other development services to support Novo’s development activities related to any development candidates. The Company will be reimbursed by Novo for these additional services. Because the provision of these additional goods and services are conditional on Novo electing to nominate a development candidate, the Company has concluded that these goods and services represent customer options and are not considered performance obligations. The total transaction price for the Novo Agreements is $256.7 million, consisting of the total $175.0 million upfront compensation, the $75.0 million additional aggregate payments described above (payable in equal annual payments of $25.0 million), and a $4.2 million premium on the sale of shares under the Novo Share Issuance Agreement. The Company applied equity accounting guidance to measure the $45.8 million recorded in equity upon the issuance of the shares. The upfront payment of $175.0 million was payable to the Company upon the delivery of a bioinformatics package and mapping plan for at least one of the initial targets selected by Novo and was paid in January 2020. If the Novo Collaboration Agreement is terminated prior to the third anniversary of its effective date, the Company is entitled to 80% of the outstanding and unearned annual payments. The Company assumed that the mapped targets will be delivered and that the contract will not be canceled. The Company has experience with mapping targets, and therefore, concluded that such amount does not need to be constrained. Accordingly, the Company included the $75.0 million of additional payments in the transaction price. As Novo nominated its first candidate under the Novo Collaboration Agreement, Dicerna earned a $2.5 million milestone in the fourth quarter of 2020, and this milestone is included in the transaction price. The Company used the most likely amount method to estimate variable consideration and estimated that the most likely amount for each potential preclinical, development, and regulatory variable consideration milestone payment under this agreement was zero, as the achievement of those milestones is uncertain and highly susceptible to factors outside of the Company’s control. Accordingly, all such milestones were excluded from the transaction price. Management will reevaluate the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and will adjust the transaction price as necessary. Sales-based royalties, including milestone payments based on the level of sales, were also excluded from the transaction price, as the license is deemed to be the predominant item to which the royalties relate. The Company will recognize such revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). Revenue associated with the performance obligation is being recognized as services are provided using a cost-to-cost measure of progress method. The transfer of control occurs over time, as the Company’s performance does not create an asset with alternative use, and the Company has an enforceable right to payment for performance completed to date. In management’s judgment, this input method is the best measure of progress towards satisfying the performance obligation and reflects a faithful depiction of the transfer of goods and services. Roche collaboration agreement Background On October 30, 2019, the Company and Roche entered into a Collaboration and License Agreement (the “Roche Collaboration Agreement”). Under the terms of the Roche Collaboration Agreement, the Company and Roche will seek to progress RG6346, the companies’ investigational therapy in Phase 1 clinical development, toward worldwide development and commercialization, as well as provide an option for the companies to collaborate in the discovery, development, and commercialization of oligonucleotide therapeutics intended for the treatment of hepatitis B virus (“HBV”). The Roche Collaboration Agreement requires that Dicerna complete the ongoing Phase 1 clinical trial, along with additional Phase 1 cohorts that were requested by Roche for which Roche will reimburse the Company for the cost of the additional cohorts, after which Roche will lead the development and commercialization of the RG6346 program. Roche also had until receipt of interim Phase 1 data from the RG6346 Phase 1 study (but no later than December 31, 2020) to initiate a research and development collaboration with the Company to pursue up to five targets selected by Roche, (each a “Selected Target”), which are intended primarily to treat HBV. Under an amendment to the Roche Collaboration Agreement in June 2020, Roche and Dicerna agreed to extend the date for nomination of targets from December 31, 2020 to January 15, 2021, subject to further potential extension by the parties due to the COVID-19 global pandemic shutdowns. Under the terms of the Roche Collaboration Agreement, the goal of such research and development collaboration will be to select compounds developed by the Company or Roche for Roche’s continued development and commercialization. The Company’s and Roche’s research and early development organizations will work exclusively with each other during the research and development collaboration period on the discovery, research, and development of such targets selected by Roche, which includes the performance of certain services by Dicerna. Under the Roche Collaboration Agreement, the Company is providing Roche with exclusive and non-exclusive licenses to support Roche’s activities and to enable Roche to commercialize products derived from or containing compounds developed pursuant to such agreement. In April 2020, Roche nominated the first of up to five targets under the research and development portion of the Roche Collaboration Agreement. Under the terms of the Roche Collaboration Agreement, Roche paid the Company a non-refundable upfront payment of $200.0 million in January 2020. The Company is also eligible to receive additional payments totaling up to approximately $1.47 billion, which includes payments upon achievement of specified development, regulatory, and commercial milestones. In addition, Roche will pay the Company up to mid-teens percent royalties on worldwide product sales. Royalties are payable until the later of 10 years after first commercial sale of each product in a country, expiration of patent rights in a country, or for products containing RG6346 in a given country, the expiration of data or regulatory exclusivity, subject to certain royalty step-down provisions set forth in the agreement. In addition, the Company has an option to co-fund the development of products under the agreement and, if exercised, receive high-twenties to mid-thirties royalty rates on net sales of products in the United States. If the Company exercises the co-funding option, it also has an option to co-promote products containing RG6346 in the United States. Accounting Analysis The Company concluded that Roche is a customer in this arrangement pursuant to the revenue recognition guidance. The Company identified contract promises under the agreement for (i) the license of intellectual property and know-how rights related to the lead compound, (ii) research and development services to complete the Phase 1 study associated with the lead compound, (iii) lead compound transfer activities, (iv) manufacturing of clinical supply for the lead compound Phase 1 study, and (v) Roche’s option to receive additional goods and services related to the research and development collaboration. The Company determined that the Roche Collaboration Agreement contains two performance obligations consisting of: (i) a combined performance obligation that includes a license, related development and manufacturing services to complete the Phase 1 study, and manufacturing obligations through the completion of the Phase 1 study related to the lead compound (the “RG6346 Performance Obligation”), and (ii) a material right to enter into a research and development collaboration to develop additional targets. While evaluating contract promises to determine whether each was capable of being distinct and distinct within the context of the contract, management considered the specialized nature of the services to be provided by Dicerna, specifically with respect to the Company’s therapeutic expertise related to RNAi and the Company’s GalXC conjugates, and the interdependent relationship between the contract promises. As such, the Company concluded that the promises of the license and research and development services related to the lead compound were not distinct from each other. Accordingly, these promises were combined into one performance obligation, the RG6346 Performance Obligation. Upon Roche’s exercise of its option to enter into the research and development collaboration for which no additional consideration will be received, Roche has the right to nominate up to five additional targets. For each target nominated, Roche will receive a license to the Selected Target, for which the Company will perform research services through clinical candidate selection. Upon Roche’s nomination of a target, the Company has concluded that the license and related research services through clinical candidate selection for each Selected Target represents a combined performance obligation, which is separate from the RG6346 Performance Obligation. The Company is not required to perform services on more than three Selected Targets at any time. Roche also has the right to replace up to three Selected Targets if a clinical candidate cannot be identified during the research term. The total transaction price for the Roche Collaboration Agreement is $206.5 million, consisting of the $200.0 million upfront payment and the estimated reimbursement from Roche related to the additional cohorts. The Company used the most likely amount method to estimate the amount of reimbursement, which was considered variable consideration, related to the additional cohorts. As reimbursement will be made as the Company performs the related services, the Company concluded that such amount does not need to be constrained, and therefore, included the full amount of the estimated reimbursement by Roche in the transaction price. The Company also estimated that the most likely amount for each potential development and regulatory variable consideration milestone payment under this agreement was zero, as the achievement of those milestones is uncertain and highly susceptible to factors outside of the Company’s control. Accordingly, all such milestones were excluded from the transaction price. Management will reevaluate the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and will adjust the transaction price as necessary. Sales-based royalties, including milestone payments based on the level of sales, were also excluded from the transaction price, as the license is deemed to be the predominant item to which the royalties relate. The Company will recognize such revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). The Company allocated the $206.5 million transaction price to the performance obligations on a relative standalone selling price basis. The Company estimated the standalone selling price for the lead compound performance obligation of $161.0 million using the adjusted market assessment approach, whereby the Company adjusted comparable third-party transactions to reflect the stage of development of the Company’s asset. To determine the estimated standalone selling price of the material right of $45.5 million, the Company estimated the standalone selling price of the underlying performance obligations included in the material right and estimated the probability of Roche exercising such underlying performance obligations. The Company concluded that the research and development collaboration material right contained (i) five material rights to receive a selected target license and related research and development services, and (ii) three material rights to receive a replacement selected target license and related research and development services. Upon the nomination of a target, the Selected Target license and related research services through clinical candidate selection will be accounted for as a combined performance obligation. The value of the material right related to the Selected Target is included in the transaction price for the combined performance obligation. The variable consideration related to the reimbursement from Roche for the additional Phase 1 cohorts and any milestones and royalties that are achieved will be allocated specifically to the lead compound performance obligation, as this variable consideration relates specifically to the Company’s satisfaction of the lead compound performance obligation and such allocation has been determined to be consistent with the allocation objective of revenue recognition guidance. Revenue associated with the lead compound performance obligation is recognized as services are provided using a cost-to-cost measure of progress method. The transfer of control occurs over time, as the Company’s performance does not create an asset with alternative use, and the Company has an enforceable right to payment for performance completed to date. In management’s judgment, this input method is the best measure of progress towards satisfying the performance obligation and reflects a faithful depiction of the transfer of goods and services. The transaction price allocated to the research and development collaboration material right will be recognized based on the timing of recognition of the underlying performance obligations that comprise the material right, or upon expiry of the material right if such right is not exercised. The aggregate amount of the transaction price allocated to the Company’s unsatisfied or partially unsatisfied performance obligations under the Roche Collaboration Agreement at December 31, 2020 was $136.3 million. As of December 31, 2020, the Company expects to recognize the balance of deferred revenue during the estimated three-year research term, which may be extended for up to two years. Lilly collaboration and share purchase agreements Background On October 25, 2018, the Company entered into a Collaboration and License Agreement with Lilly (the “Lilly Collaboration Agreement”) for the discovery, development, and commercialization of potential new medicines in the areas of cardiometabolic disease, neurodegenerative diseases, and pain. Under the terms of the Lilly Collaboration Agreement, the Company and Lilly will use the Company’s proprietary GalXC RNAi technology to progress new drug targets toward clinical development and commercialization. In addition, the Company and Lilly will collaborate on non-liver (i.e., extrahepatic) tissues, including neural tissues. The Company will work exclusively with Lilly in the neurodegenerative disease and pain fields, with the exception of mutually agreed upon orphan indications. Additionally, the Company will work exclusively with Lilly on select targets in the cardiometabolic field. Under the Lilly Collaboration Agreement, the Company will provide Lilly with exclusive and non-exclusive licenses to support the companies’ activities and to enable Lilly to commercialize products derived from or containing compounds developed pursuant to such agreement. The Lilly Collaboration Agreement provides for three initially named hepatocyte targets, and the Company and Lilly developed research programs with the goal of researching and developing multiple lead candidates directed to each of these initial targets. The Lilly Collaboration Agreement contemplates in excess of ten targets. Lilly paid the Company a non-refundable upfront payment of $100.0 million. The Company is also eligible to receive up to $350.0 million per target in development and commercialization milestones, in addition to a $5.0 million payment, which will become due for each of the extrahepatic targets when a product candidate achieves proof of principle in an animal model. In addition, the Company is eligible to earn mid-single- to low-double-digit royalties on product sales on a country-by-country and product-by-product basis until the later of expiration of patent rights in a country, the expiration of data or regulatory exclusivity in such country, or 10 years after the first product sale in such country, subject to certain royalty step-down provisions set forth in the agreement. Simultaneously with the entry into the Lilly Collaboration Agreement, the Company and Lilly entered into a Share Purchase Agreement (the “Lilly Share Issuance Agreement”), pursuant to which Lilly purchased 5,414,185 shares of the Company’s common stock at $18.47 per share, for an aggregate purchase price of $100.0 million. During the fourth quarter of 2020, Dicerna achieved a milestone associated with the first filing of an Investigational New Drug (“IND”) application for LY3561774, targeting the ANGPTL3 gene for the treatment of dyslipidemia, with the FDA, entitling the Company to a $10.0 million payment. The Company received this payment in the fourth quarter of 2020. In February 2021, Lilly notified us of their decision to extend for an additional year the initial research collaboration term for the extrahepatic targets subject to the Lilly Collaboration Agreement. Under the agreement between the companies, Lilly has the option to extend the three-year initial research collaboration term for these extrahepatic targets for up to three consecutive one-year periods. This first extension allows the research program for these extrahepatic targets under the collaboration between the two companies to continue through October 2022. Accounting Analysis The Lilly Share Issuance Agreement is to be combined with the Lilly Collaboration Agreement (together, the “Combined Agreements”) for accounting purposes. The Company concluded that Lilly is a customer in this arrangement. As such, the element of the arrangement unrelated to the issuance of the shares falls within the scope of the revenue recognition guidance. The Company identified contract promises under the Combined Agreements for licenses of intellectual property and know-how rights, associated research and d |
STOCKHOLDERS' EQUITY
STOCKHOLDERS' EQUITY | 12 Months Ended |
Dec. 31, 2020 | |
Equity [Abstract] | |
STOCKHOLDERS' EQUITY | STOCKHOLDERS' EQUITY Preferred stock The Company has authorized up to 5,000,000 shares of preferred stock, $0.0001 par value per share, for issuance. The preferred stock will have such rights, preferences, privileges and restrictions, including voting rights, dividend rights, conversion rights, redemption privileges, and liquidation preferences, as shall be determined by the Company’s Board of Directors upon its issuance. At December 31, 2020 and 2019, there were no shares of preferred stock outstanding. Issuances of Common Stock On April 20, 2018, the Company entered into a Share Issuance Agreement with Alnylam (“Alnylam Share Issuance Agreement”), pursuant to which the Company agreed to issue to Alnylam 983,208 shares in satisfaction of the Company’s obligation under the Settlement Agreement to deliver shares to Alnylam (see Note 14). The Alnylam Share Issuance Agreement contains customary representations and warranties of each party. The transaction contemplated by the Alnylam Share Issuance Agreement was closed on April 24, 2018. On September 11, 2018, the Company completed an underwritten follow-on public offering of 7,680,492 shares of common stock (the “2018 Offering”). In connection with the 2018 Offering, the Company entered into an underwriting agreement (the “2018 Underwriting Agreement”) with Citigroup Global Markets Inc. and Leerink Partners LLC as representatives of the underwriters listed in the 2018 Underwriting Agreement (collectively, the “2018 Underwriters”), pursuant to which the Company granted to the 2018 Underwriters a 30-day option to purchase up to an additional 1,152,073 shares of the Company’s common stock. Upon completion of the sale of 8,832,565 shares to the 2018 Underwriters, the Company received gross proceeds of $115.0 million. In connection with the Alexion Collaboration Agreement, the Company and Alexion entered into the Alexion Share Issuance Agreement on October 22, 2018, pursuant to which the Company sold to Alexion 835,834 shares of the Company’s common stock at $17.95 per share for an aggregate purchase price of approximately $15.0 million, of which $9.1 million was allocated to the share issuance for accounting purposes. In connection with the Lilly Collaboration Agreement, the Company and Lilly entered into the Lilly Share Issuance Agreement on October 25, 2018, pursuant to which the Company sold to Lilly 5,414,185 shares of common stock at $18.47 per share for an aggregate purchase price of approximately $100.0 million, of which $51.3 million was allocated to the share issuance for accounting purposes. The closing of the transactions contemplated by the Lilly Collaboration Agreement and the Lilly Share Issuance Agreement occurred on December 19, 2018. In connection with the Novo Collaboration Agreement, the Company and Novo entered into the Novo Share Issuance Agreement on November 15, 2019, pursuant to which the Company sold to Novo 2,279,982 shares of common stock at $21.93 per share for an aggregate purchase price of approximately $50.0 million, of which $45.8 million was allocated to the share issuance for accounting purposes. The closing of the transactions contemplated by the Novo Collaboration Agreement and the Novo Share Issuance Agreement occurred on December 27, 2019. On February 6, 2020, the Company issued and sold an aggregate of approximately $40.0 million of shares of its common stock to a single institutional investor pursuant to a common stock Sales Agreement with Cowen and Company, LLC as the sales agent. In this transaction, the Company sold an aggregate of 2,077,500 shares of common stock at a price of $19.25 per share, resulting in net proceeds of approximately $39.2 million after a deduction of approximately $0.8 million in sales commissions. The shares in the offering were sold pursuant to a shelf registration statement declared effective by the SEC on May 31, 2018 and a prospectus supplement filed with the SEC on June 1, 2018. |
STOCK-BASED COMPENSATION
STOCK-BASED COMPENSATION | 12 Months Ended |
Dec. 31, 2020 | |
Share-based Payment Arrangement [Abstract] | |
STOCK-BASED COMPENSATION | STOCK-BASED COMPENSATION Equity Incentive Plans As of December 31, 2020 the Company’s approved equity incentive plans include: the Third Amended and Restated 2007 Employee, Director and Consultant Stock Plan (“2007 Plan”); the 2010 Employee, Director and Consultant Equity Incentive Plan (“2010 Plan”); the 2014 Employee Stock Purchase Plan (“2014 ESPP”); the Amended and Restated 2014 Performance Incentive Plan (“2014 Plan”); and the 2016 Inducement Plan (“2016 Plan”). These plans are administered by the Board of Directors and permit the granting of stock options, stock appreciation rights, stock bonuses, restricted stock, performance stock, stock units, phantom stock, or similar rights to purchase or acquire shares. Upon adoption of the 2014 Plan, the Company no longer grants new equity awards under its 2007 Plan or 2010 Plan. Amended and Restated 2014 Performance Incentive Plan On January 14, 2014, the Board of Directors adopted the 2014 Plan which authorized the issuance of up to 1,900,000 shares of the Company’s common stock, with an additional increase on the first trading day in January of each calendar year during the term of the plan by an amount equal to 4.0% of the total number of shares of Common Stock issued and outstanding on December 31 of the immediately preceding calendar year. In June 2015, the 2014 Plan was amended to increase the replenishment percentage to 5.0% of outstanding common shares outstanding on December 31 of the immediately preceding calendar year and to allow the reissuance thereunder of awards and grants that expire or are canceled, terminated, forfeited, or fail to vest under the 2007 Plan and 2010 Plan, as amended. Stock options for new hires granted under this plan generally vest 25% after 12 months, followed by ratable vesting over the remaining 36-month term and expire 10 years from the grant date. Annual promotional and incentive-related grants generally vest ratably over a period of 48 months. As of December 31, 2020, there were 9,262,744 stock options outstanding, 630,755 restricted stock awards outstanding, and 2,189,910 shares of common stock reserved for future issuance under the 2014 Plan. Inducement Grants During 2014 and 2015, the Company granted 470,272 and 450,700 stock options, respectively, as inducement material to individuals entering into employment with the Company (“Inducement Grants”). The Inducement Grants were approved by the Compensation Committee of the Company’s Board of Directors and were awarded in accordance with Nasdaq Listing Rule 5635(c)(4) and outside of the 2014 Plan. As such, any shares underlying the Inducement Grants are not, upon forfeiture, cancellation, or expiration, returned to a pool of shares reserved for future issuance. As of December 31, 2020, there were 52,400 Inducement Grants that remained outstanding. 2016 Inducement Plan On March 4, 2016, the Board of Directors adopted the 2016 Plan pursuant to which the Company may grant options to purchase common shares as an inducement to individuals to join the Company. The 2016 Plan, as adopted, allowed the Company to deliver up to 250,000 shares (the “Share Limit”) of its common stock to eligible persons, as defined. The Share Limit is subject to adjustment as contemplated by the provisions of the 2014 Plan. In February and May 2017, the Share Limit was adjusted to increase the pool of issuable options by 125,000 and 200,000 underlying shares, respectively. On December 11, 2018, the Board of Directors approved a resolution to further increase the Share Limit under the 2016 Plan by 2,700,000 to 3,275,000 underlying shares. In December 2019, the Company’s Board of Directors authorized an additional 2,900,000 shares for issuance under the 2016 Plan. As of December 31, 2020, there were 4,383,484 stock options outstanding, 424,650 restricted stock awards outstanding, and 895,114 shares of common stock reserved for future issuance under the 2016 Plan. Stock-based compensation expense The Company has classified stock-based compensation expense in its consolidated statements of operations as follows: YEAR ENDED 2020 2019 2018 Research and development $ 20,157 $ 8,413 $ 3,062 General and administrative 18,754 10,409 4,826 Total $ 38,911 $ 18,822 $ 7,888 Stock options Expected volatility for the Company’s common stock was determined based on an average of the historical volatility of a peer group of similar companies due to limited historical volatility of the Company’s own common stock. The Company also has limited stock option exercise information, and as such, the expected term of stock options granted was calculated in most cases using the simplified method, which represents the average of the contractual term of the stock option and the weighted-average vesting period of the stock option. The assumed dividend yield is based upon the Company’s expectation of not paying dividends in the foreseeable future. The risk-free rate for periods within the expected life of the stock option is based upon the U.S. Treasury yield curve in effect at the time of grant. The assumptions used in the Black-Scholes option-pricing model for all stock options granted during each period presented are as follows: YEAR ENDED 2020 2019 2018 Common stock price $15.61 - $26.62 $10.31 - $26.48 $9.14 - $15.74 Expected option term (in years) 5.50 - 6.08 5.28 - 6.08 5.50 - 6.25 Expected volatility 78.3% - 80.5% 78.3% - 80.8% 75.9% - 78.3% Risk-free interest rate —% - 1.7% 1.4% - 2.6% 2.3% - 3.0% Expected dividend yield 0.0% 0.0% 0.0% The table below summarizes the activity for stock options granted under the Company’s equity incentive plans: NUMBER WEIGHTED- WEIGHTED- AGGREGATE INTRINSIC VALUE OUTSTANDING – January 1, 2020 12,467,150 $11.38 Granted 4,376,125 $21.69 Exercised (1,996,861) $9.44 Forfeited/Canceled (510,169) $15.05 Expired (12,556) $21.14 OUTSTANDING – December 31, 2020 14,323,689 $14.66 7.4 $ 108,532 EXERCISABLE – December 31, 2020 7,317,376 $12.13 6.2 $ 72,968 VESTED AND EXPECTED TO VEST – December 31, 2020 13,723,693 $14.49 7.4 $ 106,213 The weighted-average grant date fair value of stock options granted during the years ended December 31, 2020, 2019, and 2018 was $14.83, $9.11, and $7.64 per share, respectively. As of December 31, 2020, there was $76.2 million of unrecognized compensation cost related to unvested employee stock options which are expected to be recognized over a weighted-average period of 2.75 years. The intrinsic value of stock options exercised was $25.7 million, $15.2 million, and $2.9 million for the years ended December 31, 2020, 2019, and 2018, respectively. The Company does not currently hold any treasury shares. Upon stock option exercise, the Company issues new shares and delivers them to the participant. Restricted common stock The table below summarizes the activity for restricted stock units granted under the Company’s equity incentive plans: UNITS WEIGHTED- NONVESTED RESTRICTED STOCK UNITS – January 1, 2020 — $0.00 Granted 1,096,780 $21.72 Vested — $0.00 Forfeited (41,375) $22.11 NONVESTED RESTRICTED STOCK UNITS – December 31, 2020 1,055,405 $21.70 As of December 31, 2020, there was $17.9 million of total unrecognized compensation cost related to nonvested share-based compensation arrangements granted under the 2014 Plan. That cost is expected to be recognized over a weighted-average period of 3.17 years. Employee stock purchase plan On January 28, 2014, the Company’s stockholders approved the 2014 ESPP, which authorized the issuance of up to 1,000,000 shares of common stock thereunder. The 2014 ESPP provides for an automatic reserve increase equivalent to the lesser of 1% of the total number of shares of common stock issued and outstanding on December 31 of the immediately preceding calendar year and 1,000,000 shares of common stock, unless otherwise determined by the Company’s Board of Directors. As of December 31, 2020, there were 3,505,629 shares of common stock authorized and 3,006,226 shares of common stock available for issuance under the 2014 ESPP. Eligible employees may purchase shares of the Company’s common stock through regular payroll deductions up to 15% of their eligible compensation. Under the terms of the offering under the 2014 ESPP, the number of shares purchased by an individual participant in the plan may not exceed 10,000 shares in any one purchase period. In addition, the fair market value of shares purchased by an individual participant in the plan may not exceed $25,000 if the contribution period is within any one calendar year. Participants are allowed to terminate their participation in the ESPP at any time during the purchase period prior to the purchase of the shares. The offering periods have a 24‑month term, which consists of four purchase periods, each of which is six months in duration. New offering periods commence on the first day of January and July each year and end on the last business day of the immediately following June or December, respectively. The per-share purchase price at the end of each offering period is equal to the lesser of 85% of the fair market value of the common stock on the grant date of the offering period to which the purchase period relates or 85% of the fair market value of the common stock on the purchase date of the applicable purchase period. In the event that the fair value of the common stock on any purchase date during an offering period is lower than the fair market value of the common stock on the grant date of that offering period, that offering period will terminate on such purchase date, and each participant in such terminated offering period will be automatically enrolled in the new offering period that commences on the first business day of the next offering period that immediately follows such purchase date. Shares issued under the 2014 ESPP are considered compensatory. Accordingly, the Company is required to measure the fair value of the stock purchase rights granted and record compensation expense for share purchase rights granted under the 2014 ESPP. The fair values of the stock purchase rights are estimated using the Black-Scholes option-pricing model, which relies on a number of key assumptions in calculating the estimates of fair value. Stock-based compensation expense related to stock purchase rights under the 2014 ESPP was $1.0 million, $0.7 million, and $0.1 million for the years ended December 31, 2020, 2019, and 2018, respectively. During the years ended December 31, 2020, 2019, and 2018, the Company issued 113,792, 122,999, and 118,239 shares of common stock under the 2014 ESPP, respectively. The weighted-average purchase prices of shares issued under the 2014 ESPP were $13.51, $6.97, and $2.61 per share for the years ended December 31, 2020, 2019, and 2018, respectively. |
401(K) PROFIT SHARING PLAN AND
401(K) PROFIT SHARING PLAN AND TRUST | 12 Months Ended |
Dec. 31, 2020 | |
Retirement Benefits [Abstract] | |
401(K) PROFIT SHARING PLAN AND TRUST | 401(K) PROFIT SHARING PLAN AND TRUST The Company has a 401(k) Profit Sharing Plan and Trust (“401(k) Plan”), which is a retirement plan in which substantially all employees are eligible to participate. Eligible employees may elect to contribute up to the maximum limits, as set by the Internal Revenue Service, of their eligible compensation. Under the terms of the 401(k) Plan, employees may elect to make pre-tax and Roth contributions through payroll deductions within statutory and plan limits. The Company makes matching contributions of 300% of eligible employee salary deferrals that do not exceed 2% of the eligible participant’s compensation. All matching contributions vest immediately. Each year, the Company may also make a discretionary profit-sharing contribution to the plan. Such contributions to the Plan are allocated among eligible participants in the proportion of their salaries to the total salaries of all participants. Expense recognized by the Company for matching contributions made to the 401(k) Plan was $2.3 million, $1.2 million, and $0.6 million for the years ended December 31, 2020, 2019, and 2018, respectively. There were no discretionary profit-sharing contributions made by the Company during the years ended December 31, 2020, 2019, or 2018. |
INCOME TAXES
INCOME TAXES | 12 Months Ended |
Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | |
INCOME TAXES | INCOME TAXES The Company has no current and no deferred income tax expense for the years ended December 31, 2020 and 2019. The Company did not record a federal income tax provision or benefit for the years ended December 31, 2020, 2019, or 2018. The reconciliation between income taxes computed at the federal statutory income tax rate and the provision for (benefit from) income taxes is as follows: YEAR ENDED 2020 2019 2018 Federal statutory rate 21.0 % 21.0 % 21.0 % Effect of: Foreign rate differential — % (5.9) % (9.5) % Tax credits 19.6 % — % — % Net operating loss limitation 1.9 % 17.0 % (23.0) % Change in valuation allowance (39.6) % (29.7) % 10.6 % Foreign income/GILTI (a) — % (8.0) % — % Stock-based compensation expense (2.9) % 1.7 % 0.4 % Other — % 3.9 % 0.5 % Total — % — % — % (a) GILTI represents Global Intangible Low-Tax Income The components of the Company’s deferred tax assets and liabilities are as follows: DECEMBER 31, 2020 2019 Deferred tax assets: Net operating loss carryforwards $ 84,653 $ 58,319 Capitalized research and development costs 318 415 Research and development credit carryforwards 30,580 7,386 Lease liability 15,107 6,905 Stock-based compensation expense 14,911 11,751 Depreciation expense and other costs 1,346 408 Derivative liability 1,724 — Deferred tax assets 148,639 85,184 Deferred tax liabilities: Right-of-use assets (14,848) (6,833) Intangible assets (2,679) (1,939) Deferred revenue (1,688) (492) Deferred tax liabilities (19,215) (9,264) Valuation allowance (129,424) (75,920) Net deferred tax assets $ — $ — As a result of certain changes in applicable Cayman Islands law, certain changes in U.S. tax law, and related business considerations, the Company undertook certain restructuring transactions in 2019 that resulted in the liquidation of Dicerna Cayman, which was a wholly owned subsidiary of Dicerna Pharmaceuticals, Inc. that previously held intellectual property rights. That liquidation was accomplished initially via an election to treat Dicerna Cayman as a disregarded entity separate from Dicerna Pharmaceuticals, Inc. for U.S. federal income tax purposes, effective July 1, 2019; thereafter, Dicerna Cayman was dissolved under applicable Cayman Islands law. As a result of these transactions, all the pre-liquidation assets and liabilities of Dicerna Cayman, including certain intellectual property rights, are now assets and liabilities of Dicerna Pharmaceuticals, Inc. Management has evaluated the positive and negative evidence bearing upon the realizability of the Company’s net deferred tax assets and has determined that it is more likely than not that the Company will not recognize the benefits of the net deferred tax assets. As a result, the Company has recorded a full valuation allowance at December 31, 2020 and 2019. Realization of the future tax benefits is dependent on many factors, including the Company’s ability to generate taxable income within the net operating loss carryforward period. Under the provisions of the Internal Revenue Code (“IRC”), certain substantial changes in the Company’s ownership, including a sale of the Company or significant changes in ownership due to sales of equity, may have limited, or may limit in the future, the amount of net operating loss carryforwards which could be used annually to offset future taxable income. As of December 31, 2020, the Company had approximately $279.6 million of federal net operating losses, of which $125.1 million is subject to the IRC 382 limitation. None of these IRC 382 limited net operating losses are expected to expire before utilization. As of December 31, 2020, the Company had $324.0 million of state net operating loss carryforwards. If not utilized, the federal and state net operating loss carryforwards expire starting in 2029 and 2030, respectively. Additionally, as of December 31, 2020, the Company had $29.9 million of federal and $6.1 million of Massachusetts tax credits that expire starting in 2028 and 2023, respectively. As of December 31, 2020 and 2019, the Company had $5.5 million and $3.0 million of unrecognized tax benefits, respectively, all of which would affect income tax expense if recognized, before consideration of the Company’s valuation allowance. The Company does not expect the unrecognized tax benefits to change significantly over the next 12 months. The Company recognizes both interest and penalties associated with uncertain tax positions as a component of income tax expense. As of December 31, 2020 and 2019, the Company had no accrued penalties or provisions for interest. A reconciliation of the gross unrecognized tax benefits are as follows: YEAR ENDED DECEMBER 31, 2020 2019 Unrecognized tax benefits at the beginning of the period $ 3,040 $ 1,631 Increases for current tax positions 2,847 1,426 Decreases for current tax positions — — Increases for previous tax positions 1,350 — Decreases for previous tax positions (1,769) (17) Unrecognized tax benefits at the end of the period $ 5,468 $ 3,040 The Company files income tax returns in the United States, the Commonwealth of Massachusetts, Colorado, Maryland, North Carolina, New York, and New Jersey. The tax years 2008 through 2019 remain open to examination by these jurisdictions, as carryforward attributes generated in past years may be adjusted in a future period. The Company is not currently under examination by the Internal Revenue Service or any other jurisdiction for these years. The Company has not recorded any interest or penalties for unrecognized tax benefits since its inception. |
LEASES
LEASES | 12 Months Ended |
Dec. 31, 2020 | |
Leases [Abstract] | |
LEASES | LEASES 75 Hayden Avenue On January 14, 2020, the Company entered into a non-cancelable real property lease agreement for 61,282 square feet of laboratory and office space at 75 Hayden Avenue in Lexington, Massachusetts. The original term commenced during the fourth quarter of 2020 and is for 125 months with options to extend for two additional successive periods of five years thereafter. Payments for the extensions are not included in measurement of the ROU asset and lease liability, as it is not reasonably certain that the Company will exercise its options to extend the lease term. The aggregate total fixed rent for the original lease is approximately $41.8 million with the annual fixed rental payments escalating from $3.6 million to $4.8 million during the original term. In conjunction with the agreement, the Company was required to establish a $1.5 million letter of credit that has been secured by money market investments and presented as restricted cash equivalents. The interest rate implicit in the lease agreement is not readily determinable, and as such, the Company utilizes its incremental borrowing rate to calculate the lease liability, which is the rate incurred to borrow, on a collateralized basis for a similar term, an amount equal to the lease payments in a similar economic environment at the time the asset is made available to the Company. On July 1, 2020, the Company entered into an amendment (the “75 Hayden Amendment”) to the 75 Hayden Avenue lease. The 75 Hayden Amendment expands the square footage leased under the 75 Hayden Avenue lease to contain a total of 91,728 rentable square feet. The 75 Hayden Amendment increased monthly base rent by an average of $0.2 million per month. Consistent with the terms of the original 75 Hayden Avenue lease, throughout the term of the amended 75 Hayden Avenue lease, the Company is responsible for paying certain variable costs and expenses, including insurance costs and a proportionate share of applicable taxes and operating expenses for the premises. In conjunction with the 75 Hayden Amendment, the Company was required to establish an additional $0.8 million letter of credit that has been secured by money market investments and presented as restricted cash equivalents. 33 Hayden Avenue On January 2, 2019, Dicerna executed a lease for laboratory and office space in Lexington, Massachusetts (the “Lexington Lease”) that commenced for accounting purposes on November 3, 2019. The term of the Lexington Lease is seven years with approximately $30.1 million in fixed payments and consideration for the first partial calendar month. The Company has the option to extend the lease term at a prevailing market rate as of the extension date, which is seven years after the Lexington Lease commencement date. Payments for the extension is not included in measurement of the ROU asset and lease liability, as it is not reasonably certain that the Company will exercise its option to extend the lease term. As part of the Company’s lease agreement, the Company is required to establish a $2.8 million letter of credit, secured by money market investments, which is presented as restricted cash equivalents at December 31, 2020. Colorado Leases On August 26, 2019, the Company entered into a lease agreement for 15,781 square feet of office space in Boulder, Colorado (the “Original Colorado Lease”). The term of the Original Colorado Lease commenced for accounting purposes on December 4, 2020. The term of the Original Colorado Lease ends September 30, 2027 with approximately $3.0 million in aggregate fixed payments over the term of the lease arrangement. The Boulder Lease also provides the option to extend the term for up to two additional periods of 60 months each. Payments for the extensions are not included in measurement of the ROU asset and lease liability, as it is not reasonably certain that the Company will exercise its options to extend the lease term. As part of the agreement for the Boulder Lease, the Company was required to establish a $0.4 million letter of credit, secured by money market investments, which is presented as restricted cash equivalents at December 31, 2020 and 2019. On February 4, 2020, the Company entered into an amendment to a real property lease agreement for its office location in Boulder, Colorado (“the First Colorado Lease Amendment”). The First Colorado Lease Amendment provided for an additional 6,985 square feet of office space within the same building. The term of the lease for the additional space provided for under the First Colorado Lease Amendment commenced on February 5, 2020. The term of the First Colorado Lease Amendment ends September 30, 2027 with approximately $1.5 million in aggregate fixed payments over the term of the lease arrangement. The Boulder Lease also provides the option to extend the term for up to two additional periods of 60 months each. As part of the agreement for the First Colorado Lease Amendment, the Company was required to establish a $0.2 million letter of credit, secured by money market investments, which is presented as restricted cash equivalents at December 31, 2020. On July 8, 2020, the Company entered into a second amendment (the “Second Colorado Lease Amendment”) to the lease agreement dated August 26, 2019 for office space in Boulder, Colorado. The Second Colorado Lease Amendment provided the Company with permission to operate a designated area of the Original Colorado Premises as biotechnology laboratory space. Payments due under the amended Colorado leases include fixed and variable payments. Variable payments relate to the Company’s share of the lessor’s operating costs associated with the underlying assets and are recognized when the event on which those payments are assessed occurs. The term for each of the lease components under the Amended Colorado Lease will end on September 30, 2027. The Company has the option to extend the term for two additional successive periods of five years thereafter. Cambridge Leases On July 11, 2014, the Company executed a noncancelable operating lease for office and laboratory space in Cambridge, Massachusetts (the “First Cambridge Lease”). The lease agreement, the term of which commenced on December 1, 2014, obligated the Company to make minimum payments totaling $9.6 million over a six-year lease term ending November 30, 2020. The Company had the option to extend the lease term for one additional five-year period, but did not extend and the lease ended. Rent expense was recorded on a straight-line basis. As part of the Company’s lease agreement, the Company established a $0.7 million letter of credit, secured by a money market investment, the balance of which is presented as restricted cash equivalents at December 31, 2020 and 2019. The Company also leases a small office in Cambridge, Massachusetts which has a 30-month term without a renewal option (the “Second Cambridge Lease” and together with the First Cambridge Lease, the “Cambridge Leases”). Payments due under each lease agreement include fixed and variable payments. Variable payments relate to the Company’s share of the lessors’ operating costs associated with the underlying assets and are recognized when the event on which those payments are assessed occurs. None of the Company’s operating leases contain residual value guarantees. The interest rate implicit in lease agreements is typically not readily determinable, and as such, the Company utilizes the incremental borrowing rate to calculate lease liabilities, which is the rate incurred to borrow, on a collateralized basis over a similar term, an amount equal to the lease payments in a similar economic environment. ROU assets from finance leases are recorded within property and equipment on the consolidated balance sheets. Future lease payments for noncancelable leases as of December 31, 2020 are as follows: OPERATING LEASES (1) FINANCE 2021 $ 7,351 $ 64 2022 8,480 60 2023 8,735 60 2024 8,997 56 2025 9,267 2 Thereafter 28,918 — Total undiscounted lease payments 71,748 242 Less: imputed interest expense (19,770) (37) Total lease liabilities $ 51,978 $ 205 __________________________ (1) Excluded from the table above are a portion of our lease payments associated with our newest lease in Lexington, Massachusetts, as the space leased under the amendment has not commenced for accounting purposes as of December 31, 2020. Under generally accepted accounting principles, the commencement date is the date on which the asset is made available to the Company by the lessor. The components of lease cost in the Company’s consolidated statements of operations are as follows: YEAR ENDED DECEMBER 31, 2020 2019 2018 Operating leases Fixed lease cost $ 8,095 $ 2,747 $ 1,634 Variable lease cost 3,064 1,915 — Total operating lease cost $ 11,159 $ 4,662 $ 1,634 Finance lease Amortization expense $ 48 $ 6 $ — Interest expense 20 3 — Total finance lease cost $ 68 $ 9 $ — Amounts reported in the consolidated balance sheet for leases in which the Company is the lessee as of December 31, 2020 were as follows: OPERATING LEASES FINANCE Lease ROU assets $ 60,843 $ 187 Lease liabilities $ 51,978 205 Weighted-average remaining lease term 8.37 3.94 Weighted-average discount rate 8.00 % 9.00 % Other information related to the Company’s leases is as follows: YEAR ENDED 2020 2019 2018 Cash paid for amounts included in the measurement of lease liabilities Operating cash flows from operating leases $ 10,420 $ 8,966 $ 1,634 Financing cash flows from finance leases $ 45 $ — $ — Right-of-use assets obtained in exchange for lease liabilities Operating leases $ 32,368 $ 32,412 $ — Finance leases $ 48 $ 193 $ — |
LEASES | LEASES 75 Hayden Avenue On January 14, 2020, the Company entered into a non-cancelable real property lease agreement for 61,282 square feet of laboratory and office space at 75 Hayden Avenue in Lexington, Massachusetts. The original term commenced during the fourth quarter of 2020 and is for 125 months with options to extend for two additional successive periods of five years thereafter. Payments for the extensions are not included in measurement of the ROU asset and lease liability, as it is not reasonably certain that the Company will exercise its options to extend the lease term. The aggregate total fixed rent for the original lease is approximately $41.8 million with the annual fixed rental payments escalating from $3.6 million to $4.8 million during the original term. In conjunction with the agreement, the Company was required to establish a $1.5 million letter of credit that has been secured by money market investments and presented as restricted cash equivalents. The interest rate implicit in the lease agreement is not readily determinable, and as such, the Company utilizes its incremental borrowing rate to calculate the lease liability, which is the rate incurred to borrow, on a collateralized basis for a similar term, an amount equal to the lease payments in a similar economic environment at the time the asset is made available to the Company. On July 1, 2020, the Company entered into an amendment (the “75 Hayden Amendment”) to the 75 Hayden Avenue lease. The 75 Hayden Amendment expands the square footage leased under the 75 Hayden Avenue lease to contain a total of 91,728 rentable square feet. The 75 Hayden Amendment increased monthly base rent by an average of $0.2 million per month. Consistent with the terms of the original 75 Hayden Avenue lease, throughout the term of the amended 75 Hayden Avenue lease, the Company is responsible for paying certain variable costs and expenses, including insurance costs and a proportionate share of applicable taxes and operating expenses for the premises. In conjunction with the 75 Hayden Amendment, the Company was required to establish an additional $0.8 million letter of credit that has been secured by money market investments and presented as restricted cash equivalents. 33 Hayden Avenue On January 2, 2019, Dicerna executed a lease for laboratory and office space in Lexington, Massachusetts (the “Lexington Lease”) that commenced for accounting purposes on November 3, 2019. The term of the Lexington Lease is seven years with approximately $30.1 million in fixed payments and consideration for the first partial calendar month. The Company has the option to extend the lease term at a prevailing market rate as of the extension date, which is seven years after the Lexington Lease commencement date. Payments for the extension is not included in measurement of the ROU asset and lease liability, as it is not reasonably certain that the Company will exercise its option to extend the lease term. As part of the Company’s lease agreement, the Company is required to establish a $2.8 million letter of credit, secured by money market investments, which is presented as restricted cash equivalents at December 31, 2020. Colorado Leases On August 26, 2019, the Company entered into a lease agreement for 15,781 square feet of office space in Boulder, Colorado (the “Original Colorado Lease”). The term of the Original Colorado Lease commenced for accounting purposes on December 4, 2020. The term of the Original Colorado Lease ends September 30, 2027 with approximately $3.0 million in aggregate fixed payments over the term of the lease arrangement. The Boulder Lease also provides the option to extend the term for up to two additional periods of 60 months each. Payments for the extensions are not included in measurement of the ROU asset and lease liability, as it is not reasonably certain that the Company will exercise its options to extend the lease term. As part of the agreement for the Boulder Lease, the Company was required to establish a $0.4 million letter of credit, secured by money market investments, which is presented as restricted cash equivalents at December 31, 2020 and 2019. On February 4, 2020, the Company entered into an amendment to a real property lease agreement for its office location in Boulder, Colorado (“the First Colorado Lease Amendment”). The First Colorado Lease Amendment provided for an additional 6,985 square feet of office space within the same building. The term of the lease for the additional space provided for under the First Colorado Lease Amendment commenced on February 5, 2020. The term of the First Colorado Lease Amendment ends September 30, 2027 with approximately $1.5 million in aggregate fixed payments over the term of the lease arrangement. The Boulder Lease also provides the option to extend the term for up to two additional periods of 60 months each. As part of the agreement for the First Colorado Lease Amendment, the Company was required to establish a $0.2 million letter of credit, secured by money market investments, which is presented as restricted cash equivalents at December 31, 2020. On July 8, 2020, the Company entered into a second amendment (the “Second Colorado Lease Amendment”) to the lease agreement dated August 26, 2019 for office space in Boulder, Colorado. The Second Colorado Lease Amendment provided the Company with permission to operate a designated area of the Original Colorado Premises as biotechnology laboratory space. Payments due under the amended Colorado leases include fixed and variable payments. Variable payments relate to the Company’s share of the lessor’s operating costs associated with the underlying assets and are recognized when the event on which those payments are assessed occurs. The term for each of the lease components under the Amended Colorado Lease will end on September 30, 2027. The Company has the option to extend the term for two additional successive periods of five years thereafter. Cambridge Leases On July 11, 2014, the Company executed a noncancelable operating lease for office and laboratory space in Cambridge, Massachusetts (the “First Cambridge Lease”). The lease agreement, the term of which commenced on December 1, 2014, obligated the Company to make minimum payments totaling $9.6 million over a six-year lease term ending November 30, 2020. The Company had the option to extend the lease term for one additional five-year period, but did not extend and the lease ended. Rent expense was recorded on a straight-line basis. As part of the Company’s lease agreement, the Company established a $0.7 million letter of credit, secured by a money market investment, the balance of which is presented as restricted cash equivalents at December 31, 2020 and 2019. The Company also leases a small office in Cambridge, Massachusetts which has a 30-month term without a renewal option (the “Second Cambridge Lease” and together with the First Cambridge Lease, the “Cambridge Leases”). Payments due under each lease agreement include fixed and variable payments. Variable payments relate to the Company’s share of the lessors’ operating costs associated with the underlying assets and are recognized when the event on which those payments are assessed occurs. None of the Company’s operating leases contain residual value guarantees. The interest rate implicit in lease agreements is typically not readily determinable, and as such, the Company utilizes the incremental borrowing rate to calculate lease liabilities, which is the rate incurred to borrow, on a collateralized basis over a similar term, an amount equal to the lease payments in a similar economic environment. ROU assets from finance leases are recorded within property and equipment on the consolidated balance sheets. Future lease payments for noncancelable leases as of December 31, 2020 are as follows: OPERATING LEASES (1) FINANCE 2021 $ 7,351 $ 64 2022 8,480 60 2023 8,735 60 2024 8,997 56 2025 9,267 2 Thereafter 28,918 — Total undiscounted lease payments 71,748 242 Less: imputed interest expense (19,770) (37) Total lease liabilities $ 51,978 $ 205 __________________________ (1) Excluded from the table above are a portion of our lease payments associated with our newest lease in Lexington, Massachusetts, as the space leased under the amendment has not commenced for accounting purposes as of December 31, 2020. Under generally accepted accounting principles, the commencement date is the date on which the asset is made available to the Company by the lessor. The components of lease cost in the Company’s consolidated statements of operations are as follows: YEAR ENDED DECEMBER 31, 2020 2019 2018 Operating leases Fixed lease cost $ 8,095 $ 2,747 $ 1,634 Variable lease cost 3,064 1,915 — Total operating lease cost $ 11,159 $ 4,662 $ 1,634 Finance lease Amortization expense $ 48 $ 6 $ — Interest expense 20 3 — Total finance lease cost $ 68 $ 9 $ — Amounts reported in the consolidated balance sheet for leases in which the Company is the lessee as of December 31, 2020 were as follows: OPERATING LEASES FINANCE Lease ROU assets $ 60,843 $ 187 Lease liabilities $ 51,978 205 Weighted-average remaining lease term 8.37 3.94 Weighted-average discount rate 8.00 % 9.00 % Other information related to the Company’s leases is as follows: YEAR ENDED 2020 2019 2018 Cash paid for amounts included in the measurement of lease liabilities Operating cash flows from operating leases $ 10,420 $ 8,966 $ 1,634 Financing cash flows from finance leases $ 45 $ — $ — Right-of-use assets obtained in exchange for lease liabilities Operating leases $ 32,368 $ 32,412 $ — Finance leases $ 48 $ 193 $ — |
COMMITMENTS AND CONTINGENCIES
COMMITMENTS AND CONTINGENCIES | 12 Months Ended |
Dec. 31, 2020 | |
Commitments and Contingencies Disclosure [Abstract] | |
COMMITMENTS AND CONTINGENCIES | COMMITMENTS AND CONTINGENCIES On June 10, 2015, Alnylam filed a complaint against the Company in the Superior Court of Middlesex County, Massachusetts. The complaint alleged misappropriation of confidential, proprietary, and trade secret information, as well as other related claims, in connection with the Company’s hiring of a number of former employees of Merck & Co., Inc. (“Merck”) and its discussions with Merck regarding the acquisition of its subsidiary, Sirna Therapeutics, Inc., which was subsequently acquired by Alnylam. On April 18, 2018, the Company and Alnylam entered into the Settlement Agreement, resolving all ongoing litigation between the Company and Alnylam. Pursuant to the terms of the Settlement Agreement, the Company agreed to make the following payments to Alnylam: (i) a $2.0 million upfront payment in cash, which the Company made in May 2018; (ii) an additional $13.0 million in cash to be paid as 10% of any upfront or first year cash consideration that the Company receives pursuant to future collaborations related to Ga1NAc-conjugated RNAi research and development (excluding any amounts received or to be received by the Company from its existing collaboration with BI), provided that the $13.0 million must be paid by no later than April 28, 2022; and (iii) issuance of shares of the Company’s common stock pursuant to the Alnylam Share Issuance Agreement. Under the Settlement Agreement, for periods ranging from 18 months up to four years, the Company will be restricted in its development and other activities relating to oligonucleotide-based therapeutics directed toward a defined set of eight Alnylam targets (the “Oligo Restrictions”). The Oligo Restrictions pertain to targets where Dicerna does not have, or does not currently intend to have, a therapeutic program, or are expected to be consistent with Dicerna’s execution on programs in the normal course of business. The Settlement Agreement did not include any admission of liability or wrongdoing by either party or any licenses to any intellectual property from either party. On April 20, 2018, the Company and Alnylam entered into the Alnylam Share Issuance Agreement, pursuant to which the Company agreed to issue to Alnylam 983,208 shares in satisfaction of the Company’s obligation under the Settlement Agreement to deliver shares to Alnylam. The 983,208 shares issued pursuant to the Alnylam Share Issuance Agreement was recorded at fair market value of $10.3 million based on the Company’s closing share price on April 18, 2018, the date the Settlement Agreement was executed. The Company did not assign any value to the Oligo Restrictions as the Company did not incur additional losses or give up any value as a result of the restrictions. In May 2018, the Company recorded the cash obligation of $13.0 million as a liability discounted to the estimated present value of $8.7 million at an effective interest rate of 10%. The Company applied the effective interest method, as the present value is accreted through maturity. In October 2018, the Company entered into collaboration agreements with Alexion and Lilly, under which the Company was entitled to upfront cash consideration of $22.0 million and $100.0 million, respectively (see Note 8). Accordingly, the Company revised its estimate of the present value of the litigation settlement payable from $8.7 million to $13.0 million based on the expected timing of the remaining payments. The impact of revising the expected timing of repayment was recorded as a $3.7 million charge to litigation expense in the consolidated statement of operations for the year ended December 31, 2018. In connection with the execution of the Alexion Collaboration Agreement and the related receipt of the non-refundable upfront payment of $22.0 million and proceeds of $15.0 million from the Alexion Share Issuance Agreement in October 2018, the Company determined that $2.5 million became payable to Alnylam under the terms of the Settlement Agreement. The Company issued a payment to Alnylam of $2.5 million in November 2018 for the amount of the litigation settlement payable due in connection with the cash consideration received from Alexion during 2018. At December 31, 2018, the outstanding balance of the litigation settlement payable was $10.5 million. The Company paid the remaining outstanding balance of litigation settlement payable in full on January 22, 2019 upon receipt of the upfront cash payment associated with the Lilly Collaboration Agreement. During the year ended December 31, 2018, the Company recognized interest expense of $0.6 million on the outstanding balance of the litigation settlement payable during the year. Total litigation expense was $29.1 million for the year ended December 31, 2018, all of which related to the litigation and settlement agreement with Alnylam. The litigation expense for the year ended December 31, 2018 includes $24.7 million related to the Settlement Agreement. Legal proceedings From time to time, the Company may be subject to various claims and legal proceedings in the ordinary course of business. If the potential loss from any claim, asserted or unasserted, or legal proceeding is considered probable and the amount is reasonably estimable, the Company will accrue a liability for the estimated loss. There were no contingent liabilities recorded as of December 31, 2020 or 2019. |
SUMMARY OF SIGNIFICANT ACCOUN_2
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies) | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
Basis of presentation | Basis of presentation The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”) and include the accounts of Dicerna Pharmaceuticals, Inc. and its wholly owned subsidiaries. The Company believes that the financial statements as presented reflect all normal recurring adjustments necessary for a fair statement of the information for the periods presented. All intercompany balances and transactions have been eliminated in consolidation. |
Significant judgments and estimates | Significant judgments and estimates The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the Company’s consolidated financial statements, as well as the revenues and expenses incurred during the reporting periods. On an ongoing basis, the Company evaluates judgments and estimates, including those related to revenue recognition, stock-based compensation, the derivative liability, and accrued expenses. The Company bases its estimates on historical experience and on various other factors that the Company believes are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not apparent from other sources. Changes in estimates are reflected in reported results for the period in which they become known. Actual results could differ materially from those estimates. |
Cash and cash equivalents | Cash and cash equivalents Cash and cash equivalents includes all highly liquid investments, including money market funds, maturing within 90 days from the date of purchase. |
Restricted cash equivalents | Restricted cash equivalentsRestricted cash equivalents are money market funds held in collateral accounts that are restricted to secure letters of credit for corporate lease activity. The letters of credit are required to be maintained throughout the terms of the leases. |
Held-to-maturity investments | Held-to-maturity investments The Company invests its excess cash balances in short-term and long-term fixed-income investments. The Company determines the appropriate classification of investments at the time of purchase and re-evaluates such designation as of each balance sheet date. Debt securities carried at amortized cost are classified as held-to-maturity when the Company has the positive intent and ability to hold the securities to maturity. The contractual terms of these investments do not permit the issuer to settle the securities at a price less than the amortized cost bases of the investments. The Company does not intend to sell its investments and the Company has the intent and ability to hold its investments until they mature. The Company’s investment policy mandates that, at the time of purchase, the maturity of each investment within its portfolio shall not exceed two years. In addition, the weighted average maturity of the investment portfolio must not exceed one year. |
Concentrations of credit risk and significant customers | Concentrations of credit risk and significant customers Financial instruments that subject the Company to significant concentrations of credit risk consist of cash, cash equivalents, restricted cash equivalents, held-to-maturity investments, contract receivables, and the withholding tax receivable (see Note 8 – Collaborative Research and License Agreements). All of the Company’s cash, cash equivalents, restricted cash equivalents, and held-to-maturity investments are invested in money market funds or U.S. treasury securities that management believes to be of high credit quality. The Company’s revenues for the years ended December 31, 2020, 2019, and 2018 are primarily related to the Company’s collaboration agreements which are concentrated among a few collaboration partners. All revenues recognized by the Company to date were earned in the United States. Refer to Note 8 – Collaborative Research And License Agreements for composition of significant collaboration relationships. |
Fair value measurements | Fair Value Measurements Fair value is an exit price, representing the amount that would be received from the sale of an asset or paid to transfer a liability in an orderly transaction between market participants. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. Valuation techniques used to measure fair value are performed in a manner to maximize the use of observable inputs and minimize the use of unobservable inputs. As a basis for considering such assumption, the accounting literature establishes a three-tier value hierarchy which prioritizes the inputs used in measuring fair value as follows: • Level 1 – observable inputs, such as quoted prices in active markets; • Level 2 – inputs other than the quoted prices in active markets that are observable either directly or indirectly; and • Level 3 – unobservable inputs for which there is little or no market data, which requires the Company to develop its own assumptions. |
Property and equipment and Cloud computing arrangements | Property and equipment Property and equipment are stated at cost. Major betterments are capitalized, whereas expenditures for maintenance and repairs which do not improve or extend the life of the respective assets are charged to operations as incurred. Depreciation is calculated and applied using the straight-line method over the estimated useful lives, as shown below: ASSET CATEGORY ESTIMATED Laboratory equipment 5 years Office and computer equipment 3 - 5 years Furniture and fixtures 5 years Leasehold improvements 5 years or the remaining term of lease, if shorter Construction-in-process is stated at cost, which includes the cost of construction and other direct costs attributable to the construction. No provision for depreciation and amortization expense is recorded related to construction-in-process until the relevant assets are completed and put into use. At December 31, 2020, the balance of construction-in-process includes construction costs for lessee-owned improvements for leased premises and costs associated with laboratory equipment under installation. Cloud computing arrangements The Company adopted Accounting Standards Update (“ASU”) 2018-15, Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That is a Service Contract on January 1, 2020. ASU 2018-15 aligns the requirements for capitalizing implementation costs in a cloud computing arrangement service contract with the requirements for capitalizing implementation costs incurred for internal-use software licenses. Eligible costs associated with cloud computing arrangements, such as software business applications used in the normal course of business, are capitalized in accordance with Accounting Standards Codification (“ASC”) 350 – Intangibles – Goodwill and Other . |
Impairment of long-lived assets | Impairment of long-lived assetsLong-lived assets are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amount of the asset may not be recoverable. When such events occur, the Company compares the carrying amounts of the assets to their undiscounted expected future cash flows. If this comparison indicates that there is an impairment, the amount of the impairment is calculated as the difference between the carrying value and fair value of the related asset. |
Leases | Leases On January 1, 2019, the Company adopted the new lease standard, Accounting Standards Codification (“ASC”) 842, Leases, which is intended to increase transparency and comparability among organizations by requiring lessees to recognize most leases on their balance sheets and making targeted changes to lessor accounting. The most significant change arising from the new standard is the recognition of right-of-use (“ROU”) assets and lease liabilities for leases classified as operating leases. Under the standard, disclosures are required to enable financial statement users to assess the amount, timing, and uncertainty of cash flows arising from the leases. Initial implementation of the standard did not have a material impact on the consolidated financial statements, but required additional disclosures. The Company elected the package of three practical expedients that permitted an entity not to (a) reassess whether expired or existing contracts contain leases, (b) reassess lease classification for existing or expired leases, and (c) consider whether previously capitalized initial direct costs would be appropriate under the new standard. In adopting ASC 842, the Company elected not to bifurcate payments between lease and nonlease components associated with leases for office and laboratory real estate. The Company determines if an arrangement is a lease at inception. Leases with a term greater than one year are presented on the balance sheet as ROU assets, lease liabilities and, if applicable, long-term lease liabilities. The Company has elected not to recognize leases with terms of one year or less on its balance sheet. At the commencement date, operating lease liabilities and their corresponding ROU assets are recorded based on the present value of future lease payments over the expected lease term. Certain adjustments to the ROU asset may be required for items such as initial direct costs paid or incentives received. Operating lease cost is recognized over the expected term on a straight-line basis. The Company determines the expected term for its operating leases considering the noncancelable period of the lease, plus any additional periods covered by either (a) a Company option to extend (or not to terminate) the lease that the Company is reasonably certain to exercise, or (b) an option to extend (or not to terminate) the lease controlled by the lessor. |
Derivative liability | Derivative liability The derivative liability is recorded at fair value, which is estimated using a Monte Carlo simulation for estimated future contingent royalty and milestone payments. The liability is measured quarterly with any change in fair value recorded in other income (expense) in the consolidated statements of operations. |
Segment and geographic information | Segment and geographic information Operating segments are defined as components (business activity from which it earns revenue and incurs expenses) of an enterprise about which discrete financial information is available and regularly reviewed by the chief operating decision maker in deciding how to allocate resources and in assessing performance. The Company, through its Chief Executive Officer in his role as chief operating decision maker, views Company operations and manages the business as one operating segment. All long-lived assets of the Company are located in the United States. |
Research and development costs and grants and credits | Research and development costs Research and development costs consist of expenses incurred in performing research and development activities, including compensation and benefits for full-time research and development employees, an allocation of facility expenses and overhead expenses, and other external expenses. Research and development costs are expensed as incurred and were $205.4 million, $109.3 million and $45.7 million for the years ending December 31, 2020, 2019, and 2018, respectively. Research and development costs that are paid in advance of performance are deferred as a prepaid expense and amortized over the service period as the services are provided. The Company sometimes receives assistance from third-party entities such as governmental or non-profit agencies. When assistance is received from a governmental entity, the Company first determines whether the payment represents revenue by considering factors such as whether a commercial purpose exists for the payments and whether the required activity to qualify for the assistance relates to the Company’s ongoing activities. If the Company concludes that the assistance is revenue, the Company applies ASC 606, Revenue from Contracts with Customers . If the assistance is in the form of an income tax credit, the Company applies the guidance in ASC 740, Income Taxes . When the Company determines that the assistance is not revenue and does not fall within the scope of ASC 740, it applies International Accounting Standard 20, Accounting for Government Grants and Disclosure of Government Assistance |
Revenue recognition | Revenue recognition The Company generates revenue from research collaboration and license agreements with customers. Goods and services in the agreements may include the grant of licenses for the use of the Company’s technology, the provision of services associated with the research and development of product candidates, manufacturing services, and participation on joint steering committees. Such agreements may provide for consideration to the Company in the form of upfront payments; funding or reimbursement of research and development services; reimbursement of certain costs; option exercise payments; payments due upon the achievement of research, development, regulatory, and commercial-based milestones; and royalty payments on licensed products. On January 1, 2018, the Company adopted ASC 606, Revenue from Contracts with Customers , which amended revenue recognition principles and provides a single, comprehensive set of criteria for revenue recognition. The new revenue standard applies to all contracts with customers except for contracts that are within the scope of other standards. The new guidance provides a five-step framework through which revenue is recognized when control of promised goods or services is transferred to a customer at an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. To determine revenue recognition for arrangements that the Company concludes are within the scope of the new revenue recognition standard, management performs the following five steps: (i) identifies the contract(s) with a customer; (ii) identifies the performance obligations in the contract; (iii) determines the transaction price, including whether there are any constraints on variable consideration; (iv) allocates the transaction price to the performance obligations; and (v) recognizes revenue when (or as) the Company satisfies a performance obligation. At contract inception, once a contract is determined to be within the scope of the new revenue standard, Dicerna assesses whether individual goods or services promised within each contract are distinct and, therefore, represent a separate performance obligation. Goods and services that are determined not to be distinct are combined with other promised goods or services until a distinct bundle is identified. Dicerna allocates the transaction price (the amount of consideration to which the Company expects to be entitled in exchange for the promised goods or services) to each performance obligation and recognizes the associated revenue when (or as) each performance obligation is satisfied. The Company’s estimate of the transaction price for each contract includes all variable consideration to which Dicerna expects to be entitled at each measuring period. When two or more contracts are entered into with the same customer at or near the same time, the Company evaluates the contracts to determine whether the contracts should be accounted for as a single arrangement. Contracts are combined and accounted for as a single arrangement if one or more of the following criteria are met: (i) the contracts are negotiated as a package with a single commercial objective; (ii) the amount of consideration to be paid in one contract depends on the price or performance of the other contract; or (iii) the goods or services promised in the contracts (or some goods or services promised in each of the contracts) are a single performance obligation. The evaluation of whether promised goods or services represent distinct performance obligations is subjective and requires the Company to make judgments about the promised goods and services and whether such goods and services are separable from the other aspects of the contract(s). The transaction price is allocated among the performance obligations on a relative standalone selling price basis, and the applicable revenue recognition criteria are applied to each of the separate performance obligations. The Company may estimate the standalone selling price using a residual method when the selling price is highly variable because a representative standalone selling price is not discernible from past transactions or other observable evidence, or when the selling price is uncertain. Determining the standalone selling price for performance obligations requires significant judgment. When an observable price of a promised good or service is not readily available, the Company considers relevant assumptions to estimate the standalone selling price, including, as applicable, market conditions, development timelines, probabilities of technical and regulatory success, reimbursement rates for personnel costs, forecasted revenues, potential limitations to the selling price of the product, and discount rates. The Company applies judgment in determining whether a combined performance obligation is satisfied at a point in time or over time, and, if over time, concluding upon the appropriate method of measuring progress to be applied for purposes of recognizing revenue. The Company evaluates the measure of progress each reporting period and, as estimates related to the measure of progress change, related revenue recognition is adjusted accordingly. Changes in the Company’s estimated measure of progress are accounted for on a cumulative catch-up basis as a change in accounting estimate and are recorded through earnings in the period of adjustment. The Company receives payments from its licensees as established in each contract. Upfront payments and fees are recorded as deferred revenue upon receipt or when due and most often require deferral of revenue recognition to a future period until the Company performs its obligations under the underlying arrangements. Where applicable, amounts are recorded as contracts receivable when the Company’s right to consideration is unconditional. Licenses of intellectual property If a license granted to a customer to use the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenue from consideration allocated to the license when the license is transferred to the licensee and the licensee is able to use and benefit from the license. For licenses that are bundled with other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, to conclude upon the appropriate method of measuring progress for purposes of recognizing revenue related to consideration allocated to the performance obligation. Research and development services Arrangements that include a promise for the Company to provide research or development services are assessed to determine whether the services are capable of being distinct, are not highly interdependent or do not significantly modify one another, and if so, the services are accounted for as a separate performance obligation as the services are provided to the customer. Otherwise, when research or development services are determined not to be capable of being distinct, such services are added to the performance obligation that includes the underlying license. For research and development services that are bundled with other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, to conclude upon the appropriate method of measuring progress for purposes of recognizing revenue related to consideration allocated to the performance obligation. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. Options Customer options, such as options granted to allow a licensee to choose to research and develop additional product candidates or reserve product candidates against target genes to be identified in the future, or options that allow a customer to designate a target as a lead product, are evaluated at contract inception in order to determine whether those options provide a material right (i.e., an optional good or service offered for free or at a discount) to the customer. If the customer option represents a material right, the material right is treated as a separate performance obligation at the outset of the arrangement. The Company allocates the transaction price to material rights based on the standalone selling price, and revenue is recognized when or as the future goods or services are transferred or when the option expires. Customer options that are not material rights do not give rise to separate performance obligations, and as such, the additional consideration that would result from a customer exercising an option in the future is not included in the transaction price for the current contract. Instead, the option is deemed a marketing offer, and additional option fee payments are recognized or begin being recognized as revenue when the licensee exercises the option. The exercise of an option that does not represent a material right is treated as a separate contract for accounting purposes. Milestone payments At the inception of each contract with a customer that includes development or regulatory milestone payments, the Company evaluates whether the milestones are considered probable of being reached and estimates the amount to be included in the transaction price using the most likely amount method. If the Company concludes it is probable that a significant revenue reversal would not occur, the associated milestone payment is included in the transaction price. Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are generally not considered probable of being achieved until those approvals are received. The transaction price is then allocated to each performance obligation on a relative stand-alone selling price basis, for which the Company recognizes revenue as or when the performance obligations under the contract are satisfied. At the end of each subsequent reporting period, the Company re-evaluates the probability of achievement of all milestones and any related constraints, and, if necessary, adjusts the estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis and are recorded as revenue and through earnings in the period of adjustment. Royalties For arrangements that include sales-based royalties, including milestone payments based on the level of sales, and when the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). Contract costs The Company recognizes as an asset the incremental costs of obtaining a contract with a customer if the costs are expected to be recovered. The Company has elected a practical expedient wherein it recognizes the incremental costs of obtaining a contract as an expense when incurred if the amortization period of the asset that it otherwise would have recognized is one year or less. To date, the Company has not incurred any incremental costs of obtaining a contract with a customer. Contract modifications Contract modifications, defined as changes in the scope or price (or both) of a contract that are approved by the parties to the contract, such as a contract amendment, exist when the parties to a contract approve a modification that either creates new or changes existing enforceable rights and obligations of the parties to the contract. Depending on facts and circumstances, the Company accounts for a contract modification as one of the following: (i) a separate contract; (ii) a termination of the existing contract and a creation of a new contract; or (iii) a combination of the preceding treatments. A contract modification is accounted for as a separate contract if the scope of the contract increases because of the addition of promised goods or services that are distinct and the price of the contract increases by an amount of consideration that reflects the Company’s standalone selling prices of the additional promised goods or services. When a contract modification is not considered a separate contract and the remaining goods or services are distinct from the goods or services transferred on or before the date of the contract modification, the Company accounts for the contract modification as a termination of the existing contract and a creation of a new contract. When a contract modification is not considered a separate contract and the remaining goods or services are not distinct, the Company accounts for the contract modification as an add-on to the existing contract and as an adjustment to revenue on a cumulative catch-up basis. The Company receives payments from its licensees as established in each contract. Upfront payments and fees are recorded as deferred revenue upon receipt or when due and may require deferral of revenue recognition to a future period until the Company performs its obligations under these arrangements. Where applicable, amounts are recorded as contracts receivable when the Company’s right to consideration is unconditional. The Company does not assess whether a contract with a customer has a significant financing component if the expectation at contract inception is such that the period between payment by the licensees and the transfer of the promised goods or services to the licensees will be one year or less. |
Stock-based compensation | Stock-based compensation The Company’s stock-based compensation cost is measured at the grant date of the stock-based award based on the fair value of the award and is recognized as expense over the requisite service period, which generally represents the vesting period, and includes an estimate of the awards that will be forfeited. The Company uses the Black-Scholes valuation model for estimating the fair value of stock options. The fair value of stock option awards is affected by the valuation assumptions, including the expected volatility based on comparable market participants, expected term of the stock option, risk-free interest rate, and expected dividends. |
Income taxes | Income taxes The Company records deferred tax assets and liabilities for the expected future tax consequences of temporary differences between the Company’s financial statement carrying amounts and the tax basis of assets and liabilities using enacted tax rates expected to be in effect in the years in which the differences are expected to reverse. A valuation allowance is provided to reduce the net deferred tax assets to the amount that will more likely than not (more than 50 percent) be realized. The Company also assesses the probability that the positions taken or expected to be taken in its income tax returns will be sustained by taxing authorities. A “more likely than not” recognition threshold must be met before a tax benefit can be recognized. Tax positions that are more likely than not to be sustained are reflected in the Company’s consolidated financial statements. Tax positions are measured as the largest amount of tax benefit that is greater than 50 percent likely of being realized upon settlement with a taxing authority that has full knowledge of all relevant information. The difference between the benefit recognized for a position and the tax benefit claimed on a tax return is referred to as an unrecognized tax benefit. Potential interest and penalties associated with such uncertain tax positions are recorded as a component of income tax expense. |
Net loss per common share | Net loss per common share The Company computes basic net loss per common share by dividing net loss by the weighted average number of common shares outstanding. In periods of net income, the Company’s accounting policy includes allocating a proportional share of net income to participating securities, as determined by dividing total weighted average participating securities by the sum of the total weighted average common shares and participating securities (the “two-class method”). Participating securities have the effect of diluting both basic and diluted earnings per share during periods of income. During periods when the Company incurs a net loss, the Company does not allocate a loss to participating securities because they have no contractual obligation to share in the losses of the Company. The Company computes diluted net loss per common share after giving consideration to the dilutive effect of stock options, warrants, and nonvested restricted stock units that are outstanding during the period, except where such non-participating securities would be anti-dilutive. |
Recent accounting pronouncement | Recent accounting pronouncements In December 2019, the Financial Accounting Standards Board (“FASB”) issued ASU 2019-12, Simplifying the Accounting for Income Taxes , amending accounting guidance that simplifies the accounting for income taxes as part of its initiative to reduce complexity in the accounting standards. The amendments eliminate certain exceptions related to the approach for intraperiod tax allocation, the methodology for calculating income taxes in an interim period, and the recognition of deferred tax liabilities for outside basis differences. The amendments also clarify and simplify other aspects of the accounting for income taxes. For public business entities, ASU 2019-12 is required to be adopted effective for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2020. The Company is currently evaluating the effect this standard will have on its financial statements and related disclosures. In June 2016, the FASB issued ASU 2016-13, Financial Instruments – Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments (“ASU 2016-13”). The FASB subsequently issued amendments to ASU 2016-13 which had the same effective date as ASU 2016-13 of January 1, 2020. These standards require that credit losses be reported using an expected losses model rather than the incurred losses model that was previously used and establish additional disclosures related to credit risks associated with financial assets. The adoption of this standard did not have a significant impact on the Company’s financial statements, but required additional disclosures. |
SUMMARY OF SIGNIFICANT ACCOUN_3
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
Estimated Useful Lives | Depreciation is calculated and applied using the straight-line method over the estimated useful lives, as shown below: ASSET CATEGORY ESTIMATED Laboratory equipment 5 years Office and computer equipment 3 - 5 years Furniture and fixtures 5 years Leasehold improvements 5 years or the remaining term of lease, if shorter |
Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share | The outstanding securities presented below were excluded from the calculation of net loss per share because such securities would have been anti-dilutive due to the Company’s net loss per share during the periods ending on the dates presented. DECEMBER 31, 2020 2019 2018 Options to purchase common stock 14,323,689 12,467,150 7,787,690 Nonvested restricted common stock 1,055,405 — — Warrants to purchase common stock — 2,198 2,198 Total 15,379,094 12,469,348 7,789,888 |
HELD-TO-MATURITY INVESTMENTS (T
HELD-TO-MATURITY INVESTMENTS (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Investments, Debt and Equity Securities [Abstract] | |
Schedule of Held-To-Maturity Investments | A summary of the Company’s held-to-maturity investments is presented below: DECEMBER 31, 2020 DESCRIPTION AMORTIZED COST GROSS UNREALIZED GAINS GROSS LOSSES FAIR VALUE U.S. Treasury securities maturing in one year or less $ 442,820 $ 163 $ (12) $ 442,971 DECEMBER 31, 2019 DESCRIPTION AMORTIZED COST GROSS UNREALIZED GAINS GROSS LOSSES FAIR VALUE U.S. Treasury securities maturing in one year or less $ 196,065 $ 160 $ (6) $ 196,219 |
FAIR VALUE MEASUREMENTS (Tables
FAIR VALUE MEASUREMENTS (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Fair Value Disclosures [Abstract] | |
Schedule of Assets Measured or Disclosed at Fair Value | A summary of the Company’s assets and liabilities that are measured or disclosed at fair value on a recurring basis is presented below: DECEMBER 31, 2020 DESCRIPTION TOTAL FAIR VALUE LEVEL 1 LEVEL 2 LEVEL 3 Financial assets Cash equivalents Money market funds $ 126,006 $ 126,006 $ — $ — Held-to-maturity investments U.S. Treasury securities 442,971 — 442,971 — Restricted cash equivalents Money market funds 6,362 6,362 — — Total financial assets $ 575,339 $ 132,368 $ 442,971 $ — Financial liabilities Derivative liability 6,000 — — 6,000 Total financial liabilities $ 6,000 $ — $ — $ 6,000 DECEMBER 31, 2019 DESCRIPTION TOTAL FAIR VALUE LEVEL 1 LEVEL 2 LEVEL 3 Financial assets Cash equivalents Money market funds $ 152,903 $ 152,903 $ — $ — Held-to-maturity investments U.S. Treasury securities 196,219 — 196,219 — Restricted cash equivalents Money market funds 3,894 3,894 — — Total financial assets $ 353,016 $ 156,797 $ 196,219 $ — |
PREPAID EXPENSES AND OTHER CU_2
PREPAID EXPENSES AND OTHER CURRENT ASSETS (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | |
Summary of Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consist of the following: DECEMBER 31, 2020 2019 Prepaid clinical, contract research, and manufacturing costs $ 9,651 $ 4,288 Interest receivable 1,345 918 Prepaid insurance 817 583 Other prepaid expenses and other current assets 2,590 1,145 Prepaid expenses and other current assets $ 14,403 $ 6,934 |
PROPERTY AND EQUIPMENT, NET (Ta
PROPERTY AND EQUIPMENT, NET (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Property, Plant and Equipment [Abstract] | |
Summary of Property and Equipment, Net | Property and equipment, net, consists of the following: DECEMBER 31, 2020 2019 Laboratory equipment $ 8,637 $ 9,147 Office and computer equipment 2,049 2,425 Furniture and fixtures 1,251 1,569 Leasehold improvements 1,003 257 Construction-in-process 10,038 238 Property and equipment, at cost 22,978 13,636 Less: accumulated depreciation and amortization expense (5,432) (6,560) Property and equipment, net $ 17,546 $ 7,076 |
ACCRUED EXPENSES AND OTHER CU_2
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses and Other Current Liabilities | Accrued expenses and other current liabilities consist of the following: DECEMBER 31, 2020 2019 Accrued clinical, contract research, and manufacturing costs $ 9,297 $ 10,347 Accrued compensation and other employee-related benefits 14,031 7,298 Accrued professional fees 2,117 1,519 Other accrued expenses and current liabilities 2,616 878 Total 28,061 $ 20,042 |
COLLABORATIVE RESEARCH AND LI_2
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Revenue from Contract with Customer [Abstract] | |
Contract with Customer, Asset and Liability | The following tables provide a summary of deferred revenue balances for the Alexion Agreement and the Alexion Amendment: DECEMBER 31, 2020 CURRENT NONCURRENT TOTAL Alexion Agreement $ 9,216 $ 7,528 $ 16,744 Alexion Amendment 9,464 20,323 29,787 Total $ 18,680 $ 27,851 $ 46,531 DECEMBER 31, 2019 CURRENT NONCURRENT TOTAL Alexion Agreement $ 26,648 $ 6,348 $ 32,996 Alexion Amendment 1,197 18,803 20,000 Total $ 27,845 $ 25,151 $ 52,996 The following tables provide a summary of deferred revenue balances: DECEMBER 31, 2020 CURRENT NONCURRENT TOTAL Novo $ 30,169 $ 162,630 $ 192,799 Roche 49,493 81,273 130,766 Lilly 40,195 64,482 104,677 Alexion 18,680 27,851 46,531 Total $ 138,537 $ 336,236 $ 474,773 DECEMBER 31, 2019 CURRENT NONCURRENT TOTAL Novo $ 813 $ 3,359 $ 4,172 Roche 118,094 81,906 200,000 Lilly 63,233 72,314 135,547 Alexion 27,845 25,151 52,996 BI 2,273 — 2,273 Total $ 212,258 $ 182,730 $ 394,988 During the years ended December 31, 2020, 2019, and 2018, the Company recognized the following revenues as a result of changes in contract liability balances: YEAR ENDED Revenue recognized in the period from: 2020 2019 2018 Amounts included in deferred revenue at the beginning of the period (1) $ 149,716 $ 23,829 $ 6,066 Performance obligations satisfied (or partially satisfied) in previous reporting periods (2) $ 2,109 $ — $ — (1) The Company determines the revenue recognized in each period from contract liabilities by first attributing revenue to the individual contract liability balance outstanding at the beginning of the period. If additional consideration is received on those contracts in subsequent periods, we assume all revenue recognized in the reporting period first applies to the beginning contract liability as opposed to the new consideration for the period. (2) Relates to changes in estimated costs for the Company’s future performance obligations and estimated variable consideration. |
Schedule of Payments Received from Collaboration Partners | Payments received from our collaboration partners during the year ended December 31, 2020 were as follows: AMOUNT Novo $ 175,000 Roche 201,981 Lilly 10,000 Alexion 22,594 BI 260 Total $ 409,835 |
Disaggregation of Revenue | The following table provides a summary of revenue recognized for the Alexion Agreement and the Alexion Amendment: YEAR ENDED 2020 2019 2018 Alexion Agreement $ 27,030 $ 4,405 $ 110 Alexion Amendment 5,213 — — Total $ 32,243 $ 4,405 $ 110 The following table provides a summary of revenue recognized: YEAR ENDED 2020 2019 2018 Novo $ 13,874 $ — $ — Roche 73,927 — — Lilly 41,529 13,127 — Alexion 32,243 4,405 110 BI 2,734 6,297 6,066 Other — 75 — Total $ 164,307 $ 23,904 $ 6,176 |
STOCK-BASED COMPENSATION (Table
STOCK-BASED COMPENSATION (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Share-based Payment Arrangement [Abstract] | |
Stock-Based Compensation Expense | The Company has classified stock-based compensation expense in its consolidated statements of operations as follows: YEAR ENDED 2020 2019 2018 Research and development $ 20,157 $ 8,413 $ 3,062 General and administrative 18,754 10,409 4,826 Total $ 38,911 $ 18,822 $ 7,888 |
Schedule of Valuation Assumptions | The assumptions used in the Black-Scholes option-pricing model for all stock options granted during each period presented are as follows: YEAR ENDED 2020 2019 2018 Common stock price $15.61 - $26.62 $10.31 - $26.48 $9.14 - $15.74 Expected option term (in years) 5.50 - 6.08 5.28 - 6.08 5.50 - 6.25 Expected volatility 78.3% - 80.5% 78.3% - 80.8% 75.9% - 78.3% Risk-free interest rate —% - 1.7% 1.4% - 2.6% 2.3% - 3.0% Expected dividend yield 0.0% 0.0% 0.0% |
Stock Option Activity for Employee and Nonemployee | The table below summarizes the activity for stock options granted under the Company’s equity incentive plans: NUMBER WEIGHTED- WEIGHTED- AGGREGATE INTRINSIC VALUE OUTSTANDING – January 1, 2020 12,467,150 $11.38 Granted 4,376,125 $21.69 Exercised (1,996,861) $9.44 Forfeited/Canceled (510,169) $15.05 Expired (12,556) $21.14 OUTSTANDING – December 31, 2020 14,323,689 $14.66 7.4 $ 108,532 EXERCISABLE – December 31, 2020 7,317,376 $12.13 6.2 $ 72,968 VESTED AND EXPECTED TO VEST – December 31, 2020 13,723,693 $14.49 7.4 $ 106,213 |
Summary of Restricted Stock Units | The table below summarizes the activity for restricted stock units granted under the Company’s equity incentive plans: UNITS WEIGHTED- NONVESTED RESTRICTED STOCK UNITS – January 1, 2020 — $0.00 Granted 1,096,780 $21.72 Vested — $0.00 Forfeited (41,375) $22.11 NONVESTED RESTRICTED STOCK UNITS – December 31, 2020 1,055,405 $21.70 |
INCOME TAXES (Tables)
INCOME TAXES (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | |
Reconciliation Between Income Taxes Computed at the Federal Statutory Income Tax Rate and the Provision | The reconciliation between income taxes computed at the federal statutory income tax rate and the provision for (benefit from) income taxes is as follows: YEAR ENDED 2020 2019 2018 Federal statutory rate 21.0 % 21.0 % 21.0 % Effect of: Foreign rate differential — % (5.9) % (9.5) % Tax credits 19.6 % — % — % Net operating loss limitation 1.9 % 17.0 % (23.0) % Change in valuation allowance (39.6) % (29.7) % 10.6 % Foreign income/GILTI (a) — % (8.0) % — % Stock-based compensation expense (2.9) % 1.7 % 0.4 % Other — % 3.9 % 0.5 % Total — % — % — % (a) GILTI represents Global Intangible Low-Tax Income |
Deferred Tax Assets and Liabilities | The components of the Company’s deferred tax assets and liabilities are as follows: DECEMBER 31, 2020 2019 Deferred tax assets: Net operating loss carryforwards $ 84,653 $ 58,319 Capitalized research and development costs 318 415 Research and development credit carryforwards 30,580 7,386 Lease liability 15,107 6,905 Stock-based compensation expense 14,911 11,751 Depreciation expense and other costs 1,346 408 Derivative liability 1,724 — Deferred tax assets 148,639 85,184 Deferred tax liabilities: Right-of-use assets (14,848) (6,833) Intangible assets (2,679) (1,939) Deferred revenue (1,688) (492) Deferred tax liabilities (19,215) (9,264) Valuation allowance (129,424) (75,920) Net deferred tax assets $ — $ — |
Unrecognized Tax Benefit | A reconciliation of the gross unrecognized tax benefits are as follows: YEAR ENDED DECEMBER 31, 2020 2019 Unrecognized tax benefits at the beginning of the period $ 3,040 $ 1,631 Increases for current tax positions 2,847 1,426 Decreases for current tax positions — — Increases for previous tax positions 1,350 — Decreases for previous tax positions (1,769) (17) Unrecognized tax benefits at the end of the period $ 5,468 $ 3,040 |
LEASES (Tables)
LEASES (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Leases [Abstract] | |
Schedule of Future Minimum Lease Payments | Future lease payments for noncancelable leases as of December 31, 2020 are as follows: OPERATING LEASES (1) FINANCE 2021 $ 7,351 $ 64 2022 8,480 60 2023 8,735 60 2024 8,997 56 2025 9,267 2 Thereafter 28,918 — Total undiscounted lease payments 71,748 242 Less: imputed interest expense (19,770) (37) Total lease liabilities $ 51,978 $ 205 __________________________ (1) Excluded from the table above are a portion of our lease payments associated with our newest lease in Lexington, Massachusetts, as the space leased under the amendment has not commenced for accounting purposes as of December 31, 2020. Under generally accepted accounting principles, the commencement date is the date on which the asset is made available to the Company by the lessor. |
Components of Lease Cost | The components of lease cost in the Company’s consolidated statements of operations are as follows: YEAR ENDED DECEMBER 31, 2020 2019 2018 Operating leases Fixed lease cost $ 8,095 $ 2,747 $ 1,634 Variable lease cost 3,064 1,915 — Total operating lease cost $ 11,159 $ 4,662 $ 1,634 Finance lease Amortization expense $ 48 $ 6 $ — Interest expense 20 3 — Total finance lease cost $ 68 $ 9 $ — Other information related to the Company’s leases is as follows: YEAR ENDED 2020 2019 2018 Cash paid for amounts included in the measurement of lease liabilities Operating cash flows from operating leases $ 10,420 $ 8,966 $ 1,634 Financing cash flows from finance leases $ 45 $ — $ — Right-of-use assets obtained in exchange for lease liabilities Operating leases $ 32,368 $ 32,412 $ — Finance leases $ 48 $ 193 $ — |
Lease Amounts Reported in Balance Sheets | Amounts reported in the consolidated balance sheet for leases in which the Company is the lessee as of December 31, 2020 were as follows: OPERATING LEASES FINANCE Lease ROU assets $ 60,843 $ 187 Lease liabilities $ 51,978 205 Weighted-average remaining lease term 8.37 3.94 Weighted-average discount rate 8.00 % 9.00 % |
DESCRIPTION OF BUSINESS - Addit
DESCRIPTION OF BUSINESS - Additional Information (Details) | Dec. 31, 2020program |
Accounting Policies [Abstract] | |
Number of medical programs | 20 |
SUMMARY OF SIGNIFICANT ACCOUN_4
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Narrative (Details) | 12 Months Ended | ||
Dec. 31, 2020USD ($)segment | Dec. 31, 2019USD ($) | Dec. 31, 2018USD ($) | |
Schedule of Held-to-maturity Securities [Line Items] | |||
Held-to-maturity investments portfolio maximum period | 2 years | ||
Accrued interest receivable write-off | $ 0 | $ 0 | |
Contract receivables | 34,713,000 | 200,354,000 | |
Capitalized implementation costs | 2,700,000 | ||
Amortization expense associated with cloud computing arrangements | 0 | ||
Long-lived assets, impairment charge | $ 0 | 0 | |
Number of operating segment | segment | 1 | ||
Research and development | $ 205,384,000 | $ 109,339,000 | $ 45,711,000 |
Cloud computing | |||
Schedule of Held-to-maturity Securities [Line Items] | |||
Useful life | 3 years | ||
Maximum | |||
Schedule of Held-to-maturity Securities [Line Items] | |||
Weighted average maturity investment portfolio period | 1 year |
SUMMARY OF SIGNIFICANT ACCOUN_5
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Estimated Useful Lives (Details) | 12 Months Ended |
Dec. 31, 2020 | |
Laboratory equipment | |
Property, Plant and Equipment [Line Items] | |
ESTIMATED USEFUL LIVES | 5 years |
Office and computer equipment | Minimum | |
Property, Plant and Equipment [Line Items] | |
ESTIMATED USEFUL LIVES | 3 years |
Office and computer equipment | Maximum | |
Property, Plant and Equipment [Line Items] | |
ESTIMATED USEFUL LIVES | 5 years |
Furniture and fixtures | |
Property, Plant and Equipment [Line Items] | |
ESTIMATED USEFUL LIVES | 5 years |
Leasehold improvements | |
Property, Plant and Equipment [Line Items] | |
ESTIMATED USEFUL LIVES | 5 years |
SUMMARY OF SIGNIFICANT ACCOUN_6
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share (Details) - shares | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Anti-dilutive securities (in shares) | 15,379,094 | 12,469,348 | 7,789,888 |
Options to purchase common stock | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Anti-dilutive securities (in shares) | 14,323,689 | 12,467,150 | 7,787,690 |
Nonvested restricted common stock | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Anti-dilutive securities (in shares) | 1,055,405 | 0 | 0 |
Warrants to purchase common stock | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Anti-dilutive securities (in shares) | 0 | 2,198 | 2,198 |
HELD-TO-MATURITY INVESTMENTS -
HELD-TO-MATURITY INVESTMENTS - Schedule of Held-To-Maturity Investments (Details) - U.S. Treasury securities - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Schedule of Held-to-maturity Securities [Line Items] | ||
AMORTIZED COST | $ 442,820 | $ 196,065 |
GROSS UNREALIZED HOLDING GAINS | 163 | 160 |
GROSS UNREALIZED HOLDING LOSSES | (12) | (6) |
FAIR VALUE | $ 442,971 | $ 196,219 |
FAIR VALUE MEASUREMENTS - Sched
FAIR VALUE MEASUREMENTS - Schedule of Assets Measured or Disclosed at Fair Value (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Restricted cash equivalents | ||
Total financial assets | $ 575,339 | $ 353,016 |
Financial liabilities | ||
Derivative liability | 6,000 | |
Total financial liabilities | 6,000 | |
U.S. Treasury securities | ||
Held-to-maturity investments | ||
U.S. Treasury securities | 442,971 | 196,219 |
Money market funds | ||
Cash equivalents | ||
Money market funds | 126,006 | 152,903 |
Restricted cash equivalents | ||
Money market funds | 6,362 | 3,894 |
LEVEL 1 | ||
Restricted cash equivalents | ||
Total financial assets | 132,368 | 156,797 |
Financial liabilities | ||
Derivative liability | 0 | |
Total financial liabilities | 0 | |
LEVEL 1 | U.S. Treasury securities | ||
Held-to-maturity investments | ||
U.S. Treasury securities | 0 | 0 |
LEVEL 1 | Money market funds | ||
Cash equivalents | ||
Money market funds | 126,006 | 152,903 |
Restricted cash equivalents | ||
Money market funds | 6,362 | 3,894 |
LEVEL 2 | ||
Restricted cash equivalents | ||
Total financial assets | 442,971 | 196,219 |
Financial liabilities | ||
Derivative liability | 0 | |
Total financial liabilities | 0 | |
LEVEL 2 | U.S. Treasury securities | ||
Held-to-maturity investments | ||
U.S. Treasury securities | 442,971 | 196,219 |
LEVEL 2 | Money market funds | ||
Cash equivalents | ||
Money market funds | 0 | 0 |
Restricted cash equivalents | ||
Money market funds | 0 | 0 |
LEVEL 3 | ||
Restricted cash equivalents | ||
Total financial assets | 0 | 0 |
Financial liabilities | ||
Derivative liability | 6,000 | |
Total financial liabilities | 6,000 | |
LEVEL 3 | U.S. Treasury securities | ||
Held-to-maturity investments | ||
U.S. Treasury securities | 0 | 0 |
LEVEL 3 | Money market funds | ||
Cash equivalents | ||
Money market funds | 0 | 0 |
Restricted cash equivalents | ||
Money market funds | $ 0 | $ 0 |
PREPAID EXPENSES AND OTHER CU_3
PREPAID EXPENSES AND OTHER CURRENT ASSETS - Summary of Prepaid Expenses and Other Current Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | ||
Prepaid clinical, contract research, and manufacturing costs | $ 9,651 | $ 4,288 |
Interest receivable | 1,345 | 918 |
Prepaid insurance | 817 | 583 |
Other prepaid expenses and other current assets | 2,590 | 1,145 |
Prepaid expenses and other current assets | $ 14,403 | $ 6,934 |
PROPERTY AND EQUIPMENT, NET - S
PROPERTY AND EQUIPMENT, NET - Summary of Property and Equipment, Net (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Property, Plant and Equipment [Line Items] | ||
Property and equipment, at cost | $ 22,978 | $ 13,636 |
Less: accumulated depreciation and amortization expense | (5,432) | (6,560) |
Property and equipment, net | 17,546 | 7,076 |
Laboratory equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, at cost | 8,637 | 9,147 |
Office and computer equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, at cost | 2,049 | 2,425 |
Furniture and fixtures | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, at cost | 1,251 | 1,569 |
Leasehold improvements | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, at cost | 1,003 | 257 |
Construction-in-process | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, at cost | $ 10,038 | $ 238 |
PROPERTY AND EQUIPMENT, NET - A
PROPERTY AND EQUIPMENT, NET - Additional Information (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Property, Plant and Equipment [Abstract] | |||
Depreciation and amortization expense | $ 2,217 | $ 1,254 | $ 774 |
ACCRUED EXPENSES AND OTHER CU_3
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES - Schedule of Accrued Expenses and Other Current Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Payables and Accruals [Abstract] | ||
Accrued clinical, contract research, and manufacturing costs | $ 9,297 | $ 10,347 |
Accrued compensation and other employee-related benefits | 14,031 | 7,298 |
Accrued professional fees | 2,117 | 1,519 |
Other accrued expenses and current liabilities | 2,616 | 878 |
Total | $ 28,061 | $ 20,042 |
COLLABORATIVE RESEARCH AND LI_3
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - Alnylam (Details) - A1AT Agreement - USD ($) $ in Millions | 3 Months Ended | |
Dec. 31, 2020 | Apr. 03, 2020 | |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||
Other expense associated to contingent payments | $ 6 | |
Maximum | ||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||
Aggregate sales milestones receivable | $ 45 |
COLLABORATIVE RESEARCH AND LI_4
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - Novo Collaboration Agreement and Novo Share Issuance Agreement (Details) | Nov. 15, 2019USD ($)targetprogram | Dec. 31, 2020USD ($)$ / shares | Dec. 31, 2020USD ($)$ / shares | Dec. 31, 2019$ / shares |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Common stock, par value (in dollars per share) | $ / shares | $ 0.0001 | $ 0.0001 | $ 0.0001 | |
Novo Agreements | ||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Milestone earned | $ 2,500,000 | |||
Consideration received | $ 256,700,000 | |||
Premium on the sale of shares | $ 4,200,000 | |||
Percent entitles to outstanding and unearned annual payments | 80.00% | |||
Variable consideration for potential development and regulatory milestone | $ 0 | |||
Novo Collaboration Agreement | ||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Number of gene targets | target | 30 | |||
Number of programs | program | 2 | |||
Initial non-refundable upfront payment | $ 175,000,000 | |||
Development and commercialization milestones receivable, year one | 25,000,000 | |||
Development and commercialization milestones receivable, year two | 25,000,000 | |||
Development and commercialization milestones receivable, year three | $ 25,000,000 | |||
Development and commercialization milestones receivable, term | 3 years | |||
Payment due on specified development, regulatory, and commercial milestones | $ 357,500,000 | |||
Collaborative arrangement term | 10 years | 5 years | ||
Milestone earned | 2,500,000 | |||
Payment from targets delivered | $ 25,000,000 | |||
Consideration received | $ 242,800,000 | |||
Collaborative arrangement term, other option | 4 years | |||
Extension term | 2 years | |||
Novo Collaboration Agreement | Maximum | ||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Development and commercialization milestones receivable | $ 75,000,000 | |||
Novo Share Issuance Agreement | ||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||
Expected proceeds from issuance of common stock | 50,000,000 | |||
Compensation recorded in equity upon the issuance of the shares | $ 45,800,000 |
COLLABORATIVE RESEARCH AND LI_5
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - Roche Collaboration Agreement (Details) - Roche Collaboration Agreement | Oct. 30, 2019USD ($)target | Apr. 30, 2020target | Nov. 30, 2019target | Dec. 31, 2020USD ($) | Jan. 31, 2020USD ($) |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||
Number of targets | target | 5 | 5 | |||
Initial non-refundable upfront payment | $ 200,000,000 | $ 200,000,000 | |||
Collaborative arrangement term | 10 years | 3 years | |||
Number of additional targets | target | 5 | ||||
Consideration received | $ 206,500,000 | $ 136,300,000 | |||
Variable consideration for potential development and regulatory milestone | 0 | ||||
Lead compound performance obligation | 161,000,000 | ||||
Material right | 45,500,000 | ||||
Extension term | 2 years | ||||
Maximum | |||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||
Development and commercialization milestones receivable | $ 1,470,000,000 | ||||
Number of replaced targets | target | 3 |
COLLABORATIVE RESEARCH AND LI_6
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - Lilly Collaboration and Share Purchase Agreements (Details) | Oct. 25, 2018USD ($)target | Feb. 28, 2021extensionOption | Dec. 31, 2020USD ($) | Dec. 31, 2019USD ($) |
Disaggregation of Revenue [Line Items] | ||||
TOTAL | $ 474,773,000 | $ 394,988,000 | ||
Lilly Collaboration And License Agreement | ||||
Disaggregation of Revenue [Line Items] | ||||
Number of hepatocyte targets | target | 3 | |||
Number of targets in excess to contemplate agreement | target | 10 | |||
Payment due on first non-hepatocyte target achievement | $ 5,000,000 | |||
Collaborative arrangement term | 10 years | |||
TOTAL | 104,677,000 | $ 135,547,000 | ||
Lilly Collaboration And License Agreement | Subsequent Event | ||||
Disaggregation of Revenue [Line Items] | ||||
Collaborative arrangement term | 3 years | |||
Number of extension options | extensionOption | 3 | |||
Extension term | 1 year | |||
Lilly Collaboration And License Agreement | Maximum | ||||
Disaggregation of Revenue [Line Items] | ||||
Development and commercialization milestones receivable | $ 350,000,000 | |||
Lilly Share Issuance Agreement | ||||
Disaggregation of Revenue [Line Items] | ||||
Initial non-creditable upfront payment | 100,000,000 | |||
Milestone earned | $ 10,000,000 | |||
Consideration received | 158,700,000 | |||
Premium on the sale of shares | 48,700,000 | |||
Variable consideration for potential development and regulatory milestone | $ 0 |
COLLABORATIVE RESEARCH AND LI_7
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - Alexion Collaboration and Equity Agreements (Details) | Oct. 22, 2018USD ($)targetmilestone | Dec. 31, 2019USD ($)target | Dec. 31, 2020USD ($) |
Disaggregation of Revenue [Line Items] | |||
TOTAL | $ 394,988,000 | $ 474,773,000 | |
Alexion Agreements | |||
Disaggregation of Revenue [Line Items] | |||
TOTAL | 46,500,000 | ||
Alexion Agreement | |||
Disaggregation of Revenue [Line Items] | |||
Number of treatment candidates | target | 2 | ||
Initial non-creditable upfront payment | $ 22,000,000 | ||
Collaborative arrangement option exercise fee for each of candidates selected | $ 10,000,000 | ||
Collaborative arrangement term | 10 years | ||
Consideration received | $ 5,900,000 | ||
Transaction price | 48,200,000 | ||
Aggregate contingent milestone payments | 17,000,000 | ||
Variable consideration for potential development and regulatory milestone | 3,300,000 | ||
Variable consideration beyond initial milestones | $ 0 | ||
Number of initial research program milestones | milestone | 3 | ||
TOTAL | $ 32,996,000 | 16,744,000 | |
Alexion Agreement | Maximum | |||
Disaggregation of Revenue [Line Items] | |||
Additional payment receivable | $ 600,000,000 | ||
Option exercise fee | 20,000,000 | ||
Development milestones receivable for each product | 105,000,000 | ||
Aggregate sales milestones receivable | 160,000,000 | ||
Alexion Share Issuance Agreement | |||
Disaggregation of Revenue [Line Items] | |||
Expected proceeds from issuance of common stock | 15,000,000 | ||
Share purchase price allocated to equity | 9,100,000 | ||
Consideration received | 5,900,000 | ||
Alexion Amendment | |||
Disaggregation of Revenue [Line Items] | |||
Option exercise fee | 20,000,000 | ||
Collaborative arrangement option exercise fee for each of candidates selected | 10,000,000 | ||
Transaction price | $ 35,000,000 | ||
Number of additional pathway targets | target | 2 | ||
Number of pathway targets | target | 4 | ||
TOTAL | $ 20,000,000 | $ 29,787,000 |
COLLABORATIVE RESEARCH AND LI_8
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - Alexion Summary of Revenue (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||
Revenue | $ 164,307 | $ 23,904 | $ 6,176 |
Total | |||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||
Revenue | 32,243 | 4,405 | 110 |
Alexion Agreement | |||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||
Revenue | 27,030 | 4,405 | 110 |
Alexion Amendment | |||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||
Revenue | $ 5,213 | $ 0 | $ 0 |
COLLABORATIVE RESEARCH AND LI_9
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - Alexion Deferred Revenue (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||
CURRENT | $ 138,537 | $ 212,258 |
NONCURRENT | 336,236 | 182,730 |
TOTAL | 474,773 | 394,988 |
Total | ||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||
CURRENT | 18,680 | 27,845 |
NONCURRENT | 27,851 | 25,151 |
TOTAL | 46,531 | 52,996 |
Alexion Agreement | ||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||
CURRENT | 9,216 | 26,648 |
NONCURRENT | 7,528 | 6,348 |
TOTAL | 16,744 | 32,996 |
Alexion Amendment | ||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||
CURRENT | 9,464 | 1,197 |
NONCURRENT | 20,323 | 18,803 |
TOTAL | $ 29,787 | $ 20,000 |
COLLABORATIVE RESEARCH AND L_10
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - BI Agreement and Related Amendments (Details) - USD ($) | Oct. 27, 2017 | Jan. 31, 2019 | Oct. 31, 2018 | Dec. 31, 2020 | Dec. 31, 2018 |
Boehringer Ingelheim Agreement | |||||
Disaggregation of Revenue [Line Items] | |||||
Initial non-refundable upfront payment | $ 10,000,000 | ||||
Reimbursable third-party expenses | 300,000 | ||||
Consideration receivable upon potential development and commercial milestones | 191,000,000 | ||||
Contingent milestone payments | 99,000,000 | ||||
Net sales milestones | 95,000,000 | ||||
Boehringer Ingelheim Agreement | Transferred over Time | |||||
Disaggregation of Revenue [Line Items] | |||||
Remaining performance obligation amount | $ 10,300,000 | ||||
Additional Target Agreement | |||||
Disaggregation of Revenue [Line Items] | |||||
Initial non-refundable upfront payment | $ 5,000,000 | ||||
Option exercise fee | $ 5,000,000 | $ 5,000,000 | |||
Reimbursable expenses | 700,000 | ||||
Consideration received | $ 5,700,000 | ||||
Additional Target Agreement | Maximum | |||||
Disaggregation of Revenue [Line Items] | |||||
Consideration receivable upon potential development and commercial milestones | $ 170,000,000 |
COLLABORATIVE RESEARCH AND L_11
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - Summary of Payments Received (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2020USD ($) | |
Disaggregation of Revenue [Line Items] | |
AMOUNT | $ 409,835 |
Novo | |
Disaggregation of Revenue [Line Items] | |
AMOUNT | 175,000 |
Roche | |
Disaggregation of Revenue [Line Items] | |
AMOUNT | 201,981 |
Lilly | |
Disaggregation of Revenue [Line Items] | |
AMOUNT | 10,000 |
Alexion | |
Disaggregation of Revenue [Line Items] | |
AMOUNT | 22,594 |
BI | |
Disaggregation of Revenue [Line Items] | |
AMOUNT | $ 260 |
COLLABORATIVE RESEARCH AND L_12
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - Summary of Revenue Recognized (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Disaggregation of Revenue [Line Items] | |||
Revenue | $ 164,307 | $ 23,904 | $ 6,176 |
Novo | |||
Disaggregation of Revenue [Line Items] | |||
Revenue | 13,874 | 0 | 0 |
Roche | |||
Disaggregation of Revenue [Line Items] | |||
Revenue | 73,927 | 0 | 0 |
Lilly | |||
Disaggregation of Revenue [Line Items] | |||
Revenue | 41,529 | 13,127 | 0 |
Alexion | |||
Disaggregation of Revenue [Line Items] | |||
Revenue | 32,243 | 4,405 | 110 |
BI | |||
Disaggregation of Revenue [Line Items] | |||
Revenue | 2,734 | 6,297 | 6,066 |
Other | |||
Disaggregation of Revenue [Line Items] | |||
Revenue | $ 0 | $ 75 | $ 0 |
COLLABORATIVE RESEARCH AND L_13
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - Summary of Deferred Revenue Balances (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Disaggregation of Revenue [Line Items] | ||
CURRENT | $ 138,537 | $ 212,258 |
NONCURRENT | 336,236 | 182,730 |
TOTAL | 474,773 | 394,988 |
Novo | ||
Disaggregation of Revenue [Line Items] | ||
CURRENT | 30,169 | 813 |
NONCURRENT | 162,630 | 3,359 |
TOTAL | 192,799 | 4,172 |
Roche | ||
Disaggregation of Revenue [Line Items] | ||
CURRENT | 49,493 | 118,094 |
NONCURRENT | 81,273 | 81,906 |
TOTAL | 130,766 | 200,000 |
Lilly | ||
Disaggregation of Revenue [Line Items] | ||
CURRENT | 40,195 | 63,233 |
NONCURRENT | 64,482 | 72,314 |
TOTAL | 104,677 | 135,547 |
Alexion | ||
Disaggregation of Revenue [Line Items] | ||
CURRENT | 18,680 | 27,845 |
NONCURRENT | 27,851 | 25,151 |
TOTAL | $ 46,531 | 52,996 |
BI | ||
Disaggregation of Revenue [Line Items] | ||
CURRENT | 2,273 | |
NONCURRENT | 0 | |
TOTAL | $ 2,273 |
COLLABORATIVE RESEARCH AND L_14
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - Revenues as a Result of Changes in Contract Liability (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Revenue from Contract with Customer [Abstract] | |||
Amounts included in deferred revenue at the beginning of the period | $ 149,716 | $ 23,829 | $ 6,066 |
Performance obligations satisfied (or partially satisfied) in previous reporting periods | $ 2,109 | $ 0 | $ 0 |
STOCKHOLDERS' EQUITY - Preferre
STOCKHOLDERS' EQUITY - Preferred Stock (Details) - $ / shares | Dec. 31, 2020 | Dec. 31, 2019 |
Equity [Abstract] | ||
Preferred stock, shares authorized (in shares) | 5,000,000 | 5,000,000 |
Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
STOCKHOLDERS' EQUITY - Issuance
STOCKHOLDERS' EQUITY - Issuances of Common Stock (Details) - USD ($) | Feb. 06, 2020 | Nov. 15, 2019 | Oct. 25, 2018 | Oct. 22, 2018 | Sep. 11, 2018 | Apr. 20, 2018 | Dec. 18, 2017 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 |
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Issuance of common stock (in shares) | 983,208 | |||||||||
Aggregate proceeds | $ 115,000,000 | $ 39,192,000 | $ 0 | $ 108,099,000 | ||||||
Alexion Share Issuance Agreement | ||||||||||
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Share purchase price allocated to equity | $ 9,100,000 | |||||||||
Expected proceeds from issuance of common stock | $ 15,000,000 | |||||||||
Lilly Share Issuance Agreement | ||||||||||
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Expected proceeds from issuance of common stock | $ 100,000,000 | |||||||||
Initial non-refundable upfront payment | 100,000,000 | |||||||||
Combined Agreements | ||||||||||
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Compensation recorded in equity upon the issuance of the shares | $ 51,300,000 | |||||||||
Novo Share Issuance Agreement | ||||||||||
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Compensation recorded in equity upon the issuance of the shares | $ 45,800,000 | |||||||||
Expected proceeds from issuance of common stock | $ 50,000,000 | |||||||||
Common Stock | ||||||||||
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Issuance of common stock (in shares) | 2,077,500 | 8,832,565 | ||||||||
Common Stock | Underwriting Agreement | ||||||||||
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Issuance of common stock (in shares) | 8,832,565 | |||||||||
2018 Offering | Common Stock | Underwriting Agreement | ||||||||||
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Issuance of common stock (in shares) | 7,680,492 | |||||||||
Overallotment | ||||||||||
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Period to purchase additional shares | 30 days | |||||||||
Overallotment | Common Stock | Underwriting Agreement | ||||||||||
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Issuance of common stock (in shares) | 1,152,073 | |||||||||
Alexion Share Issuance Agreement | ||||||||||
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Number of shares issuable (in shares) | 835,834 | |||||||||
Price per share (in dollars per share) | $ 17.95 | |||||||||
Aggregate gross proceeds | $ 15,000,000 | |||||||||
Lilly Share Issuance Agreement | ||||||||||
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Number of shares issuable (in shares) | 5,414,185 | |||||||||
Price per share (in dollars per share) | $ 18.47 | |||||||||
Novo Share Issuance Agreement | ||||||||||
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Number of shares issuable (in shares) | 2,279,982 | |||||||||
Price per share (in dollars per share) | $ 21.93 | |||||||||
Shelf Registration | ||||||||||
Subsidiary, Sale of Stock [Line Items] | ||||||||||
Aggregate proceeds | $ 39,200,000 | |||||||||
Price per share (in dollars per share) | $ 19.25 | |||||||||
Aggregate gross proceeds | $ 40,000,000 | |||||||||
Common stock sold (in shares) | 2,077,500 | |||||||||
Sales commissions | $ 800,000 |
STOCK-BASED COMPENSATION - Addi
STOCK-BASED COMPENSATION - Additional Information (Details) - USD ($) $ / shares in Units, $ in Millions | Dec. 11, 2018 | Dec. 10, 2018 | May 31, 2015 | Jan. 14, 2014 | Dec. 31, 2019 | May 31, 2017 | Feb. 28, 2017 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2015 | Dec. 31, 2014 | Mar. 04, 2016 |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock options awarded (in shares) | 4,376,125 | ||||||||||||
Number of inducement grants outstanding (in shares) | 12,467,150 | 14,323,689 | 12,467,150 | ||||||||||
Weighted-average grant date fair value of stock options granted (in dollars per share) | $ 14.83 | $ 9.11 | $ 7.64 | ||||||||||
Unrecognized compensation cost | $ 76.2 | ||||||||||||
Aggregate intrinsic value, outstanding | $ 25.7 | $ 15.2 | $ 2.9 | ||||||||||
Restricted Stock Units | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Unvested restricted stock outstanding (in shares) | 0 | 1,055,405 | 0 | ||||||||||
Weighted-average period | 3 years 2 months 1 day | ||||||||||||
Employee Stock Option | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Weighted-average period | 2 years 9 months | ||||||||||||
2014 Plan | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Maximum number of common stock shares granted (in shares) | 1,900,000 | ||||||||||||
Stock option plan, percentage of outstanding common stock | 5.00% | 4.00% | |||||||||||
Stock option plan, vesting period | 36 months | ||||||||||||
Stock option plan, expiration period | 10 years | ||||||||||||
Stock option plan, reserved shares of common stock for future issuance (in shares) | 2,189,910 | ||||||||||||
Stock options awarded (in shares) | 9,262,744 | ||||||||||||
2014 Plan | Tranche One | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock option plan, vested rate | 25.00% | ||||||||||||
Stock option plan, vesting period | 12 months | ||||||||||||
2014 Plan | Annual Promotional and Incentive Related Grants | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock option plan, vesting period | 48 months | ||||||||||||
2014 Plan | Restricted Stock Units | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Unvested restricted stock outstanding (in shares) | 630,755 | ||||||||||||
Inducement Grants | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock options awarded (in shares) | 450,700 | 470,272 | |||||||||||
Number of inducement grants outstanding (in shares) | 52,400 | ||||||||||||
2016 Inducement Plan | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Maximum number of common stock shares granted (in shares) | 250,000 | ||||||||||||
Stock option plan, reserved shares of common stock for future issuance (in shares) | 895,114 | ||||||||||||
Stock options awarded (in shares) | 4,383,484 | ||||||||||||
Issuable common stock shares (in shares) | 3,275,000 | 2,700,000 | 2,900,000 | 200,000 | 125,000 | ||||||||
2016 Inducement Plan | Restricted Stock Units | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Unvested restricted stock outstanding (in shares) | 424,650 |
STOCK-BASED COMPENSATION - Clas
STOCK-BASED COMPENSATION - Classification of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | $ 38,911 | $ 18,822 | $ 7,888 |
Research and development | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | 20,157 | 8,413 | 3,062 |
General and administrative | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | $ 18,754 | $ 10,409 | $ 4,826 |
STOCK-BASED COMPENSATION - Sche
STOCK-BASED COMPENSATION - Schedule of Valuation Assumptions (Details) - Employee Stock Option - $ / shares | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Expected volatility, minimum | 78.30% | 78.30% | 75.90% |
Expected volatility, maximum | 80.50% | 80.80% | 78.30% |
Risk-free interest rate, maximum | 1.70% | 2.60% | 3.00% |
Risk-free interest rate, minimum | 0.00% | 1.40% | 2.30% |
Expected dividend yield | 0.00% | 0.00% | 0.00% |
Minimum | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Common stock price (in dollars per share) | $ 15.61 | $ 10.31 | $ 9.14 |
Expected option term (in years) | 5 years 6 months | 5 years 3 months 10 days | 5 years 6 months |
Maximum | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Common stock price (in dollars per share) | $ 26.62 | $ 26.48 | $ 15.74 |
Expected option term (in years) | 6 years 29 days | 6 years 29 days | 6 years 3 months |
STOCK-BASED COMPENSATION - Summ
STOCK-BASED COMPENSATION - Summary of Activity Under Equity Incentive Plans (Details) $ / shares in Units, $ in Thousands | 12 Months Ended |
Dec. 31, 2020USD ($)$ / sharesshares | |
NUMBER OF OPTIONS | |
Outstanding at beginning of year (in shares) | shares | 12,467,150 |
Granted (in shares) | shares | 4,376,125 |
Exercised (in shares) | shares | (1,996,861) |
Forfeited/cancelled (in shares) | shares | (510,169) |
Expired (in shares) | shares | (12,556) |
Outstanding at end of year (in shares) | shares | 14,323,689 |
Exercisable at end of year (in shares) | shares | 7,317,376 |
Vested and expected to vest at end of year (in shares) | shares | 13,723,693 |
WEIGHTED- AVERAGE EXERCISE PRICE | |
Outstanding at beginning of year (in dollars per share) | $ / shares | $ 11.38 |
Granted (in dollars per share) | $ / shares | 21.69 |
Exercised (in dollars per share) | $ / shares | 9.44 |
Forfeited/cancelled (in dollars per share) | $ / shares | 15.05 |
Expired (in dollars per share) | $ / shares | 21.14 |
Outstanding at end of year (in dollars per share) | $ / shares | 14.66 |
Exercisable at end of year (in dollars per share) | $ / shares | 12.13 |
Vested and expected to vest at end of year (in dollars per share) | $ / shares | $ 14.49 |
WEIGHTED- AVERAGE REMAINING CONTRACTUAL TERM (YEARS) | |
Outstanding | 7 years 4 months 24 days |
Exercisable | 6 years 2 months 12 days |
Vested and expected to vest | 7 years 4 months 24 days |
AGGREGATE INTRINSIC VALUE | |
Outstanding | $ | $ 108,532 |
Exercisable | $ | 72,968 |
Vested and expected to vest | $ | $ 106,213 |
STOCK-BASED COMPENSATION - Rest
STOCK-BASED COMPENSATION - Restricted Common Stock (Details) - Restricted Stock Units $ / shares in Units, $ in Millions | 12 Months Ended |
Dec. 31, 2020USD ($)$ / sharesshares | |
UNITS | |
Nonvested restricted stock, beginning balance (in shares) | shares | 1,055,405 |
Granted (in shares) | shares | 1,096,780 |
Vested (in shares) | shares | 0 |
Forfeited (in shares) | shares | (41,375) |
Nonvested restricted stock, ending balance (in shares) | shares | 0 |
WEIGHTED- AVERAGE GRANT DATE FAIR VALUE | |
Nonvested restricted stock units, beginning balance (in dollars per share) | $ / shares | $ 0 |
Nonvested restricted stock units, ending balance (in dollars per share) | $ / shares | 21.70 |
Vested (in dollars per share) | $ / shares | 0 |
Forfeited (in dollars per share) | $ / shares | 22.11 |
Granted (in dollars per share) | $ / shares | $ 21.72 |
Unrecognized compensation cost | $ | $ 17.9 |
Weighted-average period | 3 years 2 months 1 day |
STOCK-BASED COMPENSATION - Empl
STOCK-BASED COMPENSATION - Employee Stock Purchase Plan (Details) - 2014 Employee Stock Purchase Plan | Jan. 28, 2014USD ($)segmentshares | Dec. 31, 2020USD ($)$ / sharesshares | Dec. 31, 2019USD ($)$ / sharesshares | Dec. 31, 2018USD ($)$ / sharesshares |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Issuable common stock shares (in shares) | 1,000,000 | |||
Automatic reserve increase equivalent to common stock issued and outstanding percentage | 1.00% | |||
Maximum number of common stock shares granted (in shares) | 3,505,629 | |||
Number of shares available for issuance (in shares) | 3,006,226 | |||
Payroll deduction percentage limit of eligible compensation | 15.00% | |||
Maximum shares per employee (in shares) | 10,000 | |||
Maximum employee contribution amount | $ | $ 25,000 | |||
Offering period | 24 months | |||
Number of purchase periods | segment | 4 | |||
Purchase period | 6 months | |||
Stock-based compensation expense related to stock purchase rights | $ | $ 1,000,000 | $ 700,000 | $ 100,000 | |
Share issued under stock option plan (in shares) | 113,792 | 122,999 | 118,239 | |
Average purchase price of share issued (in dollars per share) | $ / shares | $ 13.51 | $ 6.97 | $ 2.61 | |
Maximum | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Percentage of fair market value of common stock | 85.00% |
401(K) PROFIT SHARING PLAN AN_2
401(K) PROFIT SHARING PLAN AND TRUST (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Retirement Benefits [Abstract] | |||
Employer matching contribution, percent of match | 300.00% | ||
Employer matching contribution, percent of employees' gross pay | 2.00% | ||
Contributions | $ 2,300,000 | $ 1,200,000 | $ 600,000 |
Discretionary costs | $ 0 | $ 0 | $ 0 |
INCOME TAXES - Additional Infor
INCOME TAXES - Additional Information (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Income Tax [Line Items] | |||
Current income tax expense | $ 0 | $ 0 | |
Deferred income tax expense | 0 | 0 | |
Net operating loss, estimated limitation | 125,100,000 | ||
Unrecognized tax benefits that would affect income tax expense if recognized | 5,500,000 | 3,000,000 | |
Accrued penalties or provisions for interest | 0 | 0 | |
Federal | |||
Income Tax [Line Items] | |||
Deferred income tax expense | 0 | $ 0 | $ 0 |
Net operating loss carryforwards | 279,600,000 | ||
Tax credits | 29,900,000 | ||
State | |||
Income Tax [Line Items] | |||
Net operating loss carryforwards | 324,000,000 | ||
Massachusetts | |||
Income Tax [Line Items] | |||
Tax credits | $ 6,100,000 |
INCOME TAXES - Reconciliation B
INCOME TAXES - Reconciliation Between Income Taxes Computed at the Federal Statutory Income Tax Rate and the Provision for Benefit from Income Taxes (Details) | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Income Tax Disclosure [Abstract] | |||
Federal statutory rate | 21.00% | 21.00% | 21.00% |
Effect of: | |||
Foreign rate differential | 0.00% | (5.90%) | (9.50%) |
Tax credits | 19.60% | 0.00% | 0.00% |
Net operating loss limitation | 1.90% | 17.00% | (23.00%) |
Change in valuation allowance | (39.60%) | (29.70%) | 10.60% |
Foreign income/GILTI | 0 | (0.080) | 0 |
Stock-based compensation expense | (2.90%) | 1.70% | 0.40% |
Other | 0.00% | 3.90% | 0.50% |
Total | 0.00% | 0.00% | 0.00% |
INCOME TAXES - Deferred Tax Ass
INCOME TAXES - Deferred Tax Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Deferred tax assets: | ||
Net operating loss carryforwards | $ 84,653 | $ 58,319 |
Capitalized research and development costs | 318 | 415 |
Research and development credit carryforwards | 30,580 | 7,386 |
Lease liability | 15,107 | 6,905 |
Stock-based compensation expense | 14,911 | 11,751 |
Depreciation expense and other costs | 1,346 | 408 |
Derivative liability | 1,724 | 0 |
Deferred tax assets | 148,639 | 85,184 |
Deferred tax liabilities: | ||
Right-of-use assets | (14,848) | (6,833) |
Intangible assets | (2,679) | (1,939) |
Deferred revenue | (1,688) | (492) |
Deferred tax liabilities | (19,215) | (9,264) |
Valuation allowance | (129,424) | (75,920) |
Net deferred tax assets | $ 0 | $ 0 |
INCOME TAXES - Reconciliation o
INCOME TAXES - Reconciliation of the Gross Unrecognized Tax Benefit (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Reconciliation of Unrecognized Tax Benefits, Excluding Amounts Pertaining to Examined Tax Returns [Roll Forward] | ||
Unrecognized tax benefits at the beginning of the period | $ 3,040 | $ 1,631 |
Increases for current tax positions | 2,847 | 1,426 |
Decreases for current tax positions | 0 | 0 |
Increases for previous tax positions | 1,350 | 0 |
Decreases for previous tax positions | (1,769) | (17) |
Unrecognized tax benefits at the end of the period | $ 5,468 | $ 3,040 |
LEASES - Narrative (Details)
LEASES - Narrative (Details) $ in Thousands | Jul. 08, 2020renewalOption | Jul. 01, 2020USD ($)ft² | Jan. 14, 2020USD ($)ft²renewalOption | Jan. 02, 2019USD ($) | Dec. 31, 2020USD ($)renewalOption | Dec. 31, 2019USD ($) | Dec. 31, 2018USD ($) | Jul. 18, 2020 | Feb. 05, 2020USD ($)ft² | Aug. 26, 2019USD ($)ft² | Dec. 01, 2014USD ($) |
Lessee, Lease, Description [Line Items] | |||||||||||
Area of real estate (in sqft) | ft² | 91,728 | ||||||||||
Increased monthly base rent | $ 200 | ||||||||||
Term of contract | 7 years | 30 months | 6 years | ||||||||
Lease payments | $ 30,100 | $ 10,420 | $ 8,966 | $ 1,634 | |||||||
Letter of credit | 2,800 | ||||||||||
Lease liability payments | $ 71,748 | $ 9,600 | |||||||||
Number of renewal options | renewalOption | 1 | ||||||||||
Renewal term | 5 years | ||||||||||
Hayden Avenue Letter Of Credit | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Restricted cash equivalents | $ 800 | ||||||||||
Cambridge Leases Letter Of Credit | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Restricted cash equivalents | $ 700 | ||||||||||
Lexington, Massachusetts | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Area of real estate (in sqft) | ft² | 61,282 | ||||||||||
Lease not yet commenced, lease term | 125 months | ||||||||||
Number of additional terms | renewalOption | 2 | ||||||||||
Option to extend lease, term | 5 years | ||||||||||
Aggregate total fixed rent | $ 41,800 | ||||||||||
Lexington, Massachusetts | Hayden Avenue Letter Of Credit | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Restricted cash equivalents | 1,500 | ||||||||||
Lexington, Massachusetts | Maximum | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Annual fixed rental payments | 4,800 | ||||||||||
Lexington, Massachusetts | Minimum | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Annual fixed rental payments | $ 3,600 | ||||||||||
Boulder, Colorado | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Area of real estate (in sqft) | ft² | 6,985 | 15,781 | |||||||||
Letter of credit | $ 200 | $ 400 | |||||||||
Lease liability payments | $ 1,500 | $ 3,000 | |||||||||
Number of renewal options | renewalOption | 2 | 2 | |||||||||
Renewal term | 60 months | 5 years |
LEASES - Future Lease Payments
LEASES - Future Lease Payments (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 01, 2014 |
OPERATING LEASES | ||
2021 | $ 7,351 | |
2022 | 8,480 | |
2023 | 8,735 | |
2024 | 8,997 | |
2025 | 9,267 | |
Thereafter | 28,918 | |
Total undiscounted lease payments | 71,748 | $ 9,600 |
Less: imputed interest expense | (19,770) | |
Lease liabilities | 51,978 | |
FINANCE LEASES | ||
2021 | 64 | |
2022 | 60 | |
2023 | 60 | |
2024 | 56 | |
2025 | 2 | |
Thereafter | 0 | |
Total undiscounted lease payments | 242 | |
Less: imputed interest expense | (37) | |
Lease liabilities | $ 205 |
LEASES - Components of Lease Co
LEASES - Components of Lease Cost (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Operating leases | |||
Fixed lease cost | $ 8,095 | $ 2,747 | $ 1,634 |
Variable lease cost | 3,064 | 1,915 | 0 |
Total operating lease cost | 11,159 | 4,662 | 1,634 |
Finance lease | |||
Amortization expense | 48 | 6 | 0 |
Interest expense | 20 | 3 | 0 |
Total finance lease cost | $ 68 | $ 9 | $ 0 |
Finance lease, right-of-use, statement of financial position, extensible list | us-gaap:PropertyPlantAndEquipmentAndFinanceLeaseRightOfUseAssetAfterAccumulatedDepreciationAndAmortization | ||
Operating lease, liability, current, statement of financial position, extensible list | drna:OperatingAndFinanceLeaseLiabilityCurrent | ||
Operating lease, liability, noncurrent, statement of financial position, extensible list | drna:OperatingAndFinanceLeaseLiabilityNoncurrent | ||
Finance lease, liability, current, statement of financial position, extensible list | drna:OperatingAndFinanceLeaseLiabilityCurrent | ||
Finance lease, liability, noncurrent, statement of financial position, extensible list | drna:OperatingAndFinanceLeaseLiabilityNoncurrent |
LEASES - Amounts Reported in Ba
LEASES - Amounts Reported in Balance Sheets (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
OPERATING LEASES | ||
Lease ROU assets | $ 60,843 | $ 30,102 |
Lease liabilities | $ 51,978 | |
Weighted-average remaining lease term | 8 years 4 months 13 days | |
Weighted-average discount rate | 8.00% | |
FINANCE LEASES | ||
Lease ROU assets | $ 187 | |
Lease liabilities | $ 205 | |
Weighted-average remaining lease term | 3 years 11 months 8 days | |
Weighted-average discount rate | 9.00% |
LEASES - Other Information Rela
LEASES - Other Information Related to Leases (Details) - USD ($) $ in Thousands | Jan. 02, 2019 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 |
Cash paid for amounts included in the measurement of lease liabilities | ||||
Operating cash flows from operating leases | $ 30,100 | $ 10,420 | $ 8,966 | $ 1,634 |
Financing cash flows from finance leases | 45 | 0 | 0 | |
Right-of-use assets obtained in exchange for lease liabilities | ||||
Operating leases | 32,368 | 32,412 | 0 | |
Finance leases | $ 48 | $ 193 | $ 0 |
COMMITMENTS AND CONTINGENCIES -
COMMITMENTS AND CONTINGENCIES - Additional Information (Details) | Oct. 25, 2018USD ($) | Oct. 22, 2018USD ($) | Apr. 20, 2018USD ($)shares | Apr. 18, 2018USD ($)target | Nov. 30, 2018USD ($) | Dec. 31, 2020USD ($) | Dec. 31, 2019USD ($) | Dec. 31, 2018USD ($) | Oct. 31, 2018USD ($) | May 31, 2018USD ($) |
Loss Contingencies [Line Items] | ||||||||||
Issuance of common stock (in shares) | shares | 983,208 | |||||||||
Issuance of common stock | $ 39,088,000 | $ 107,770,000 | ||||||||
Litigation expense | 0 | $ 0 | 29,132,000 | |||||||
Estimated litigation liability, current | 10,500,000 | |||||||||
Interest expense | 20,000 | 3,000 | 603,000 | |||||||
Outstanding litigation liabilities | 0 | $ 0 | ||||||||
Alexion Share Issuance Agreement | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Aggregate gross proceeds | $ 15,000,000 | |||||||||
Additional Paid-in Capital | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Issuance of common stock | $ 39,088,000 | 107,769,000 | ||||||||
Alnylam Share Issuance Agreement | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Issuance of common stock | $ 10,300,000 | |||||||||
Alexion Agreement | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Initial non-refundable upfront payment | 22,000,000 | |||||||||
Litigation settlement payable | $ 2,500,000 | |||||||||
Payments for litigation | $ 2,500,000 | |||||||||
Lilly Share Issuance Agreement | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Initial non-refundable upfront payment | $ 100,000,000 | |||||||||
Expected proceeds from issuance of common stock | $ 100,000,000 | |||||||||
Alexion Share Issuance Agreement | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Expected proceeds from issuance of common stock | $ 15,000,000 | |||||||||
Alexion Pharmaceuticals | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Litigation settlement cash obligation payable | $ 13,000,000 | |||||||||
Litigation settlement discount present value | $ 8,700,000 | |||||||||
Effective interest rate | 10.00% | |||||||||
Alexion Pharmaceuticals | Additional Paid-in Capital | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Litigation expense | 3,700,000 | |||||||||
Alnylam | Settled Litigation | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Litigation settlement upfront payment payable | $ 2,000,000 | |||||||||
Additional litigation settlement payable amount | $ 13,000,000 | $ 13,000,000 | ||||||||
Percentage of additional litigation settlement payable amount | 10.00% | |||||||||
Litigation settlement cash obligation payable | $ 13,000,000 | |||||||||
Number of targets | target | 8 | |||||||||
Litigation expense | $ 24,700,000 | |||||||||
Alnylam | Settled Litigation | Minimum | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Restricted development period | 18 months | |||||||||
Alnylam | Settled Litigation | Maximum | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Restricted development period | 4 years |