Cover
Cover - USD ($) | 12 Months Ended | ||
Dec. 31, 2023 | Feb. 21, 2024 | Jun. 30, 2023 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Document Annual Report | true | ||
Current Fiscal Year End Date | --12-31 | ||
Document Period End Date | Dec. 31, 2023 | ||
Document Transition Report | false | ||
Entity File Number | 001-39044 | ||
Entity Registrant Name | SPRINGWORKS THERAPEUTICS, INC. | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 83-4066827 | ||
Entity Address, Address Line One | 100 Washington Blvd | ||
Entity Address, City or Town | Stamford | ||
Entity Address, State or Province | CT | ||
Entity Address, Postal Zip Code | 06902 | ||
City Area Code | (203) | ||
Local Phone Number | 883-9490 | ||
Title of 12(b) Security | Common Stock, $0.0001 Par Value per Share | ||
Trading Symbol | SWTX | ||
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 | ||
Document Financial Statement Error Correction Flag | false | ||
Entity Shell Company | false | ||
Entity Public Float | $ 1,600,965,538 | ||
Entity Common Stock, Shares Outstanding | 73,786,845 | ||
Documents Incorporated by Reference | Documents Incorporated by Reference The registrant's definitive proxy statement relating to the annual meeting of shareholders will be filed with the Securities and Exchange Commission within 120 days after the close of the registrant's fiscal year ended December 31, 2023 and is incorporated by reference in Part III to the extent described herein . | ||
Entity Central Index Key | 0001773427 | ||
Amendment Flag | false | ||
Document Fiscal Year Focus | 2023 | ||
Document Fiscal Period Focus | FY |
Audit Information
Audit Information | 12 Months Ended |
Dec. 31, 2023 | |
Audit Information [Abstract] | |
Auditor Firm ID | 42 |
Auditor Name | Ernst & Young LLP |
Auditor Location | Hartford, Connecticut |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents | $ 176,053 | $ 67,490 |
Marketable securities | 303,149 | 524,722 |
Accounts receivable, net | 5,930 | 0 |
Inventory | 3,103 | 0 |
Prepaid expenses and other current assets | 12,677 | 7,548 |
Total current assets | 500,912 | 599,760 |
Long-term marketable securities | 183,386 | 4,794 |
Property and equipment, net | 17,943 | 13,571 |
Operating lease right-of-use assets | 6,144 | 4,698 |
Equity method investment | 1,955 | 4,193 |
Restricted cash | 613 | 578 |
Other assets | 14,835 | 2,648 |
Total assets | 725,788 | 630,242 |
Current liabilities: | ||
Accounts payable | 7,396 | 8,010 |
Accrued expenses | 65,569 | 39,242 |
Operating lease liabilities, current | 1,061 | 483 |
Deferred revenue, current | 4,144 | 3,314 |
Total current liabilities | 78,170 | 51,049 |
Operating lease liabilities, long-term | 5,996 | 4,768 |
Deferred revenue, long-term | 15,403 | 16,233 |
Total liabilities | 99,569 | 72,050 |
Commitments and contingencies | ||
Stockholders’ equity: | ||
Preferred stock, $0.0001 par value, 10,000,000 shares authorized, no shares issued or outstanding at December 31, 2023 and December 31, 2022. | 0 | 0 |
Common stock, $0.0001 par value, 150,000,000 shares authorized, 73,620,361 and 62,453,328 shares issued and 73,486,699 and 62,423,129 shares outstanding at December 31, 2023 and December 31, 2022, respectively. | 7 | 6 |
Additional paid-in capital | 1,524,196 | 1,130,224 |
Accumulated deficit | (895,034) | (569,930) |
Treasury stock, at cost (133,662 and 30,199 shares of common stock at December 31, 2023 and December 31, 2022, respectively). | (4,141) | (1,341) |
Accumulated other comprehensive income (loss) | 1,191 | (767) |
Total stockholders’ equity | 626,219 | 558,192 |
Total liabilities and stockholders’ equity | $ 725,788 | $ 630,242 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2023 | Dec. 31, 2022 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value (usd per share) | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized (in shares) | 10,000,000 | 10,000,000 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Common stock, par value (usd per share) | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized (in shares) | 150,000,000 | 150,000,000 |
Common stock, shares issued (in shares) | 73,620,361 | 62,453,328 |
Common stock, shares outstanding (in shares) | 73,486,699 | 62,423,129 |
Treasury stock (in shares) | 133,662 | 30,199 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Revenue: | |||
Total revenue | $ 5,447 | $ 0 | $ 0 |
Operating expenses: | |||
Cost of product revenue | 422 | 0 | 0 |
Research and development | 150,487 | 146,122 | 101,676 |
Selling, general and administrative | 197,551 | 134,552 | 71,792 |
Total operating expenses | 348,460 | 280,674 | 173,468 |
Loss from operations | (343,013) | (280,674) | (173,468) |
Interest and other income: | |||
Interest and other income, net | 22,947 | 6,147 | 546 |
Total interest and other income | 22,947 | 6,147 | 546 |
Equity method investment loss | (5,038) | (2,890) | (988) |
Net loss | $ (325,104) | $ (277,417) | $ (173,910) |
Net loss per share, basic (in usd per share) | $ (5.15) | $ (5.21) | $ (3.59) |
Net loss per share, diluted (in usd per share) | $ (5.15) | $ (5.21) | $ (3.59) |
Weighted average common shares outstanding, basic (in shares) | 63,123,936 | 53,290,528 | 48,497,790 |
Weighted average common shares outstanding, diluted (in shares) | 63,123,936 | 53,290,528 | 48,497,790 |
Revenue from Contract with Customer, Product and Service [Extensible Enumeration] | Product [Member] |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Statement of Comprehensive Income [Abstract] | |||
Net loss | $ (325,104) | $ (277,417) | $ (173,910) |
Changes in other comprehensive income (loss): | |||
Unrealized gain (loss) on marketable securities, net | 1,958 | (455) | (353) |
Total changes in other comprehensive income (loss) | 1,958 | (455) | (353) |
Comprehensive loss | $ (323,146) | $ (277,872) | $ (174,263) |
Consolidated Statement of Stock
Consolidated Statement of Stockholders’ Equity - USD ($) $ in Thousands | Total | Private Placement | Market offering | Underwritten Public Offering | GSK | Common | Common Private Placement | Common Market offering | Common Underwritten Public Offering | Common GSK | Treasury | Additional Paid-In Capital | Additional Paid-In Capital Private Placement | Additional Paid-In Capital Market offering | Additional Paid-In Capital Underwritten Public Offering | Additional Paid-In Capital GSK | Accumulated Other Comprehensive Income (Loss) | Accumulated Deficit |
Beginning balance at Dec. 31, 2020 | $ 557,058 | $ 5 | $ 0 | $ 675,615 | $ 41 | $ (118,603) | ||||||||||||
Balance at the beginning (in shares) at Dec. 31, 2020 | 48,819,591 | |||||||||||||||||
Balance at the beginning (in shares) at Dec. 31, 2020 | 0 | |||||||||||||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||||||||
Equity-based compensation expense | 38,444 | 38,444 | ||||||||||||||||
Issuance of restricted stock award (in shares) | 332,226 | |||||||||||||||||
Forfeitures of restricted stock awards (in shares) | (55,475) | |||||||||||||||||
Exercise of stock options | $ 1,157 | 1,157 | ||||||||||||||||
Exercise of stock options (in shares) | 151,643 | 151,643 | ||||||||||||||||
Other comprehensive income (loss), net of tax | $ (353) | (353) | ||||||||||||||||
Net loss | (173,910) | (173,910) | ||||||||||||||||
Ending balance at Dec. 31, 2021 | 422,396 | $ 5 | $ 0 | 715,216 | (312) | (292,513) | ||||||||||||
Balance at the end (in shares) at Dec. 31, 2021 | 49,247,985 | |||||||||||||||||
Balance at the end (in shares) at Dec. 31, 2021 | 0 | |||||||||||||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||||||||
Equity-based compensation expense | 72,965 | 72,965 | ||||||||||||||||
Issuance of common stock | $ 216,831 | $ 67,782 | $ 55,454 | $ 1 | $ 216,830 | $ 67,782 | $ 55,454 | |||||||||||
Issuance of common stock (in shares) | 8,650,520 | 2,247,500 | 2,050,819 | |||||||||||||||
Issuance of restricted stock award (in shares) | 36,625 | |||||||||||||||||
Forfeitures of restricted stock awards (in shares) | (27,957) | |||||||||||||||||
Restricted stock units vested (in shares) | 24,369 | |||||||||||||||||
Exercise of stock options | $ 1,977 | 1,977 | ||||||||||||||||
Exercise of stock options (in shares) | 223,467 | 223,467 | ||||||||||||||||
Shares of common stock used to satisfy tax withholding obligations | $ (1,341) | $ (1,341) | ||||||||||||||||
Shares of common stock used to satisfy tax withholding obligations (in shares ) | 30,199 | |||||||||||||||||
Other comprehensive income (loss), net of tax | (455) | (455) | ||||||||||||||||
Net loss | (277,417) | (277,417) | ||||||||||||||||
Ending balance at Dec. 31, 2022 | $ 558,192 | $ 6 | $ (1,341) | 1,130,224 | (767) | (569,930) | ||||||||||||
Balance at the end (in shares) at Dec. 31, 2022 | 62,423,129 | 62,453,328 | ||||||||||||||||
Balance at the end (in shares) at Dec. 31, 2022 | 30,199 | 30,199 | ||||||||||||||||
Increase (Decrease) in Stockholders' Equity | ||||||||||||||||||
Equity-based compensation expense | $ 94,534 | 94,534 | ||||||||||||||||
Issuance of common stock | $ 299,300 | $ 1 | $ 299,299 | |||||||||||||||
Issuance of common stock (in shares) | 10,905,171 | |||||||||||||||||
Forfeitures of restricted stock awards (in shares) | (21,113) | |||||||||||||||||
Restricted stock units vested (in shares) | 238,725 | |||||||||||||||||
Exercise of stock options | $ 139 | 139 | ||||||||||||||||
Exercise of stock options (in shares) | 44,250 | 44,250 | ||||||||||||||||
Shares of common stock used to satisfy tax withholding obligations | $ (2,800) | $ (2,800) | ||||||||||||||||
Shares of common stock used to satisfy tax withholding obligations (in shares ) | 103,463 | |||||||||||||||||
Other comprehensive income (loss), net of tax | 1,958 | 1,958 | ||||||||||||||||
Net loss | (325,104) | (325,104) | ||||||||||||||||
Ending balance at Dec. 31, 2023 | $ 626,219 | $ 7 | $ (4,141) | $ 1,524,196 | $ 1,191 | $ (895,034) | ||||||||||||
Balance at the end (in shares) at Dec. 31, 2023 | 73,486,699 | 73,620,361 | ||||||||||||||||
Balance at the end (in shares) at Dec. 31, 2023 | 133,662 | 133,662 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Operating activities | |||
Net loss | $ (325,104) | $ (277,417) | $ (173,910) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Depreciation and amortization expense | 1,667 | 765 | 490 |
Non-cash operating lease expense | 1,653 | 1,131 | 993 |
Equity-based compensation expense | 94,534 | 72,965 | 38,444 |
Equity method investment loss | 5,038 | 2,890 | 988 |
Changes in operating assets and liabilities | |||
Accounts receivable, net | (5,930) | 0 | 0 |
Inventory | (3,103) | 0 | 0 |
Prepaid expenses and other current assets | (5,129) | 1,861 | (4,609) |
Other assets | (838) | 61 | (707) |
Accounts payable | (196) | 4,168 | 2,074 |
Accrued expenses | 15,906 | 13,325 | 9,912 |
Lease liability | (1,293) | (859) | (1,388) |
Deferred revenue | 0 | 19,547 | 0 |
Other liabilities | 0 | 0 | (164) |
Net cash used in operating activities | (222,795) | (161,563) | (127,877) |
Investing activities | |||
Capital expenditures | (7,385) | (10,196) | (2,016) |
Equity method investment | (2,800) | (4,200) | 0 |
Purchases of marketable securities | (575,724) | (481,050) | (305,423) |
Proceeds from sale and maturity of marketable securities | 620,663 | 279,849 | 391,031 |
Net cash provided by (used in) investing activities | 34,754 | (215,597) | 83,592 |
Financing activities | |||
Proceeds from issuance of common stock in underwritten public offering, net of issuance costs | 299,300 | 0 | 0 |
Treasury stock | (2,800) | (1,341) | 0 |
Proceeds from stock option exercises | 139 | 1,977 | 1,157 |
Net cash provided by financing activities | 296,639 | 340,702 | 1,157 |
Net increase (decrease) in cash and cash equivalents | 108,598 | (36,458) | (43,128) |
Cash and cash equivalents including restricted cash, beginning of period | 68,068 | 104,526 | 147,654 |
Cash and cash equivalents including restricted cash, end of period | 176,666 | 68,068 | 104,526 |
Supplemental non-cash disclosure | |||
Right-of-use assets obtained in exchange for operating lease obligations | 2,637 | 5,580 | 0 |
Milestone payment for first commercial sale of OGSIVEO included in accrued expenses as of December 31, 2023 | 11,250 | 0 | 0 |
GSK | |||
Financing activities | |||
Proceeds from issuance of common stock | 0 | 55,454 | 0 |
Private Placement | |||
Financing activities | |||
Proceeds from issuance of common stock | 0 | 216,830 | 0 |
Market offering | |||
Financing activities | |||
Proceeds from issuance of common stock | $ 0 | $ 67,782 | $ 0 |
Nature of Operations
Nature of Operations | 12 Months Ended |
Dec. 31, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Nature of Operations | Nature of Operations SpringWorks Therapeutics, Inc., or the Company, was formed in Delaware on August 18, 2017. The Company is a commercial-stage biopharmaceutical company applying a precision medicine approach to developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. The Company has a differentiated portfolio of small molecule targeted oncology assets, including one approved product and several clinical candidates, and is advancing programs in both rare tumor types as well as highly prevalent, genetically defined cancers. OGSIVEO™ (nirogacestat) is the Company's first commercial product. OGSIVEO was approved by the United States Food and Drug Administration, or FDA, on November 27, 2023 for the treatment of adult patients with progressing desmoid tumors who require systemic treatment. Follow-On Offerings On December 8, 2023, the Company completed the sale of 10,905,171 shares of common stock in an underwritten public offering, including 1,422,413 shares of common stock sold pursuant to the underwriter's full exercise of their option to purchase additional shares, at an offering price of $29.00 per share, resulting in net proceeds to the Company of $299.3 million. On October 13, 2020, the Company completed the sale of 5,637,254 shares of common stock in an underwritten public offering, including 735,294 shares of common stock sold pursuant to the underwriter's full exercise of their option to purchase additional shares, at an offering price of $51.00 per share, resulting in net proceeds to the Company of $269.5 million. Private Placements On September 7, 2022, the Company and certain accredited investors, or the Investors, entered into a securities purchase agreement pursuant to which the Company agreed to sell and issue to the Investors in a private placement transaction, or the Private Placement, an aggregate of 8,650,520 shares of Common Stock at a purchase price of $26.01 per share. In connection with the Private Placement, the Company received gross proceeds of approximately $225 million, and after deducting commissions and offering costs, net proceeds were approximately $216.8 million. In connection with the Private Placement, the Company and the Investors also entered into a registration rights agreement, dated September 7, 2022, providing for the registration for resale of the shares. The shares were registered for resale pursuant to the Registration Statement and the prospectus supplement relating to the shares filed with the SEC on September 26, 2022. On September 6, 2022, the Company entered into an expanded global, non-exclusive license and collaboration agreement with GSK, plc, formerly GlaxoSmithKline plc, or GSK, for nirogacestat in combination with belantamab mafodotin (belamaf) and, concurrent with the execution of such agreement, we entered into a stock purchase agreement, or the Stock Purchase Agreement, with an affiliate of GSK, Glaxo Group Limited, or GGL, under which GGL agreed to purchase from the Company in a private placement transaction 2,050,819 shares of Common Stock for an aggregate purchase price of approximately $75.0 million, or $36.57 per share. The shares were sold at a 25% premium to the volume-weighted average share price of the Company’s Common Stock for a specified 30-day period prior to entering into the Stock Purchase Agreement. At-the-Market Offering In August 2022, the Company sold 2,247,500 shares of Common Stock under an ATM Program, resulting in gross proceeds of $69.7 million, less commissions and other fees of $1.9 million for net proceeds of $67.8 million. |
Risks and Liquidity
Risks and Liquidity | 12 Months Ended |
Dec. 31, 2023 | |
Risks and Uncertainties [Abstract] | |
Risks and Liquidity | Risks and Liquidity The Company has incurred losses and negative operating cash flows since inception and had an accumulated deficit of $895.0 million and $569.9 million, and working capital of $422.7 million and $548.7 million at December 31, 2023 and December 31, 2022, respectively. In November 2023, the FDA approved OGSIVEO (nirogacestat) for the treatment of adult patients with desmoid tumors. In December 2023, the Company began to generate revenue from sales of OGSIVEO in the United States, for which the Company recorded net revenue of $5.4 million in 2023. The Company is subject to those risks associated with any biopharmaceutical company that has substantial expenditures for development. There can be no assurance that the Company’s development projects will be successful, that products developed will obtain necessary regulatory approval, or that any approved product will be commercially viable. In addition, the Company operates in an environment of rapid technological change and is largely dependent on the services of its employees, advisors, consultants and vendors. The Company had cash, cash equivalents and marketable securities of $662.6 million and $597.0 million as of December 31, 2023 and December 31, 2022, respectively. Based on the Company’s cash, cash equivalents and marketable securities at December 31, 2023, management estimates that its current liquidity will enable it to meet operating expenses through at least twelve months after the date that these financial statements were issued. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2023 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies Basis of Presentation These consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States, or U.S. GAAP. Principles of Consolidation The consolidated financial statements include the accounts of SpringWorks Therapeutics, Inc. and its subsidiaries, collectively, the Company. All intercompany transactions and balances have been eliminated in consolidation. Investments in business entities in which the Company lacks control but does have the ability to exercise significant influence over operating and financial policies are accounted for using the equity method of accounting. Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts in the financial statements and accompanying notes. Significant estimates and assumptions reflected in these consolidated financial statements include, but are not limited to, accrued research and development expenses and the valuation of equity-based compensation awards. The Company bases its estimates on historical experience, known trends and other market-specific or relevant factors that it believes to be reasonable under the circumstances. Actual results may differ from those estimates. On an ongoing basis, management evaluates its estimates, and adjusts those estimates and assumptions when facts or circumstances change. Changes in estimates are recorded in the period in which they become known. Segment Information Operating segments are defined as components of an entity about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating segment. Cash and Cash Equivalents The Company considers all highly liquid instruments that have maturities of three months or less when acquired to be cash equivalents. The Company had cash and cash equivalents as of December 31, 2023 and December 31, 2022 of $176.1 million and $67.5 million, respectively. Marketable Securities Marketable debt securities are reported at fair value with unrealized gains and losses included in accumulated other comprehensive income. Each reporting period, the Company evaluates whether there are declines in fair value below amortized cost and if these declines are due to credit losses, as well as the Company’s ability and intent to hold the investment until a forecasted recovery occurs. If both criteria regarding the intent or ability to hold are met, any decline in fair value due to credit losses is recorded as an allowance through other income (expense); limited by the amount that the fair value is less than the amortized costs basis. If either criterion is not met, any previously recorded allowance for credit losses and any excess amortized cost basis over fair value is recorded in other income (expense). As of, and for the years ended December 31, 2023 and December 31, 2022, the Company did not have any allowance for credit losses or impairments of its marketable securities. Concentration of Credit Risk Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of cash, cash equivalents and available-for-sale marketable securities. The Company maintains each of its cash, cash equivalent balances and marketable securities balances with high quality, financial institutions and the Company’s marketable securities are invested in high-quality, highly liquid debt securities including corporate debt securities, U.S. government securities and commercial paper. Accounts Receivable, net Accounts receivable, net consists of trade receivables which are amounts due from customers related to product sales. The Company records trade receivables net of chargebacks, invoice discounts, distribution service fees and any allowances for potential credit losses. An allowance for credit losses is determined based on the financial condition and creditworthiness of customers and the Company considers economic factors and events or trends expected to affect future collections experience. Any allowance would reduce the net receivables to the amount that is expected to be collected. The payment history of the Company’s customers will be considered in future assessments of collectibility as these patterns are established over a longer period of time. As of December 31, 2023, the Company determined an allowance for credit losses was insignificant. Inventory and Pre-Approval Inventory The Company began capitalizing inventory for OGSIVEO upon approval by the FDA in November 2023. OGSIVEO is approved for the treatment of adult patients with desmoid tumors. Prior to regulatory approval, all direct and indirect manufacturing costs were charged to research and development expense in the period incurred. Inventory is comprised of raw materials, work-in-process and finished goods, and includes costs related to third-party contract manufacturing, packaging, freight-in and overhead. Inventory is stated at the lower of cost or net realizable value with cost based on the first-in-first-out method. Property and Equipment Property and equipment consist of computer equipment, software, furniture and leasehold improvements and are recorded at cost. Property and equipment are depreciated on a straight-line basis over their estimated useful lives. Intangible Asset, net The Company’s finite-lived intangible asset resulted from the capitalization of a milestone payment due under a license and collaboration agreement in connection with the first commercial sale of OGSIVEO in the United States in December 2023. The intangible asset will be amortized on a straight-line basis over its remaining useful life, which is estimated to be the remaining patent life of OGSIVEO. Amortization expense is recorded as cost of product revenue in the consolidated statement of operations. Impairment of Long-Lived Assets The Company reviews its long-lived assets, including property and equipment and finite-lived intangible assets, for impairment whenever events or changes in circumstances indicate that the carrying value of an asset may not be recoverable. If such circumstances are determined to exist, an estimate of undiscounted future cash flows produced by the long-lived asset, including its eventual residual value, is compared to the carrying value to determine whether impairment exists. In the event that such cash flows are not expected to be sufficient to recover the carrying amount of the asset, the asset is written-down to its estimated fair value. Revenue Recognition Arrangements Within the Scope of ASC 606, Revenue from Contracts with Customers The Company recognizes revenue in accordance with ASC 606, which applies to all contracts with customers, except for contracts that are within the scope of other standards, such as collaboration arrangements and leases. Pursuant to ASC 606, the Company recognizes revenue when its customers obtain control of promised goods or services, in an amount that reflects the consideration which the Company determines it expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that the Company determines are within the scope of ASC 606, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligation(s) in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligation(s) in the contract; and (v) recognize revenue when (or as) the Company satisfies its performance obligation(s). As part of the accounting for these arrangements, the Company may be required to make significant judgments, including identifying performance obligations in the contract, estimating the amount of variable consideration to include in the transaction price and allocating the transaction price to each performance obligation. Once a contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within the contract and determines those that are performance obligations. The Company assesses whether each promised good or service is distinct for the purpose of identifying the performance obligations in the contract. This assessment involves subjective determinations and may require management to make judgments about the individual promised goods or services and whether such are separable from the other aspects of the contractual relationship. Promised goods and services are considered distinct provided that: (i) the customer can benefit from the good or service either on its own or together with other resources that are readily available to the customer (that is, the good or service is capable of being distinct) and (ii) the entity’s promise to transfer the good or service to the customer is separately identifiable from other promises in the contract (that is, the promise to transfer the good or service is distinct within the context of the contract). In assessing whether a promised good or service is distinct, the Company considers factors such as the research, manufacturing and commercialization capabilities of the customer and the availability of the associated expertise in the general marketplace. The Company also considers the intended benefit of the contract in assessing whether a promised good or service is separately identifiable from other promises in the contract. If a promised good or service is not distinct, an entity is required to combine that good or service with other promised goods or services until it identifies a bundle of goods or services that is distinct. If the consideration promised in a contract includes a variable amount, the Company estimates the amount of consideration to which it will be entitled in exchange for transferring the promised goods or services to a customer. The Company determines the amount of variable consideration by using the expected value method or the most likely amount method. The Company includes the unconstrained amount of estimated variable consideration in the transaction price. The amount included in the transaction price is constrained to the amount for which it is probable that a significant reversal of cumulative revenue recognized will not occur. At the end of each subsequent reporting period, the Company re-evaluates the estimated variable consideration included in the transaction price and any related constraint, and if necessary, adjusts its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis in the period of adjustment. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) each performance obligation is satisfied at a point in time or over time, and if over time based on the use of an output or input method that most faithfully depicts the transfer of goods and services to the customer. Product revenue, net: Revenues from product sales are recorded at the net sales price, or “transaction price,” which includes estimates of variable consideration that result from (i) invoice discounts for prompt payment and specialty distributor and specialty pharmacy service fees, (ii) government and other rebates, chargebacks, discounts and fees, (iii) group purchasing organization (“GPO”) discounts, performance rebates and administrative fees, (iv) product returns and (v) costs of co-pay assistance programs for patients. Reserves are established for the estimates of variable consideration based on the amounts the Company expects to be earned or to be claimed on the related sales. The reserves are classified as reductions to accounts receivable, net or accrued expenses and other current liabilities. Where appropriate, the Company utilizes the expected value method to determine the appropriate amount for estimates of variable consideration based on factors such as current contractual and statutory requirements, specific known market events and trends, industry data and forecasted customer buying, payment patterns and the Company’s historical experience. The amount of variable consideration that is included in the transaction price may be constrained and is included in net product revenues only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results vary from the Company's estimates, these estimates are adjusted, which would affect net product revenue and earnings in the period such variances become known. Licenses of intellectual property: The terms of the Company’s license agreements include the license of functional intellectual property, given the functionality of the intellectual property is not expected to change substantially as a result of the Company’s ongoing activities. For licenses that are bundled with other promises (that is, for licenses that are not distinct from other promised goods and services in an arrangement), the Company utilizes 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, the appropriate method of measuring progress for purposes of recognizing revenue. Up-front Fees: If a license agreement is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenues from the transaction price 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 utilizes judgment to assess the nature of the combined performance obligation to determine whether the license is deemed to be the predominant item and if the combined performance obligation is satisfied over time or at a point in time. Milestone Payments: At the inception of each arrangement that includes milestone payments (variable consideration), 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 it is probable that a significant revenue reversal would not occur, the associated milestone value is included in the transaction price. Milestone payments such as developmental and regulatory approval milestones, are generally not considered probable of being achieved until the related activity has been achieved, due to the uncertain nature of the success of clinical trials and obtaining regulatory approvals, which make it unlikely that a significant revenue reversal could be deemed not probable, until such time that the related event has occurred. Royalties: For arrangements that include sales-based royalties, including commercial milestone payments based on the level of sales, and 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 the royalty has been allocated has been satisfied (or partially satisfied). Reimbursement, cost-sharing and profit-sharing payments: Under certain arrangements, the Company has been reimbursed for a portion of its research and development expenses or participates in the cost-sharing of such research and development expenses. Such reimbursements and cost-sharing arrangements have been reflected in research and development expense in the Company’s consolidated statements of operations, as the Company does not consider performing research and development services for reimbursement to be a part of its ongoing major or central operations. Cost of Product Revenue Our cost of product revenue includes the cost of goods sold, amortization expense for commercial milestones and royalty expense. Our cost of goods sold consists of raw materials, third-party manufacturing costs to manufacture the raw materials into finished product, freight and other costs associated with sales of commercial products. Research and Development Expenditures for clinical development, including upfront licensing fees and milestone payments associated with products that have not yet been approved by the U.S. Food and Drug Administration, are charged to research and development expense as incurred. These expenses consist of expenses incurred in performing development activities, including salaries and benefits, equity-based compensation expense, preclinical expenses, clinical trial and related clinical manufacturing expenses, contract services and other outside expenses. Expenses incurred for certain research and development activities, including expenses associated with particular activities performed by contract research organizations, investigative sites in connection with clinical trials and contract manufacturing organizations, are recognized based on an evaluation of the progress or completion of specific tasks using either time-based measures or data such as information provided to the Company by its vendors on actual activities completed or costs incurred. Payments for these activities are based on the terms of the individual arrangements, which may differ from the pattern of expense recognition. Expenses for research and development activities incurred that have yet to be invoiced by the vendors that perform the related activities are reflected in the consolidated financial statements as accrued expenses. Advance payments for goods or services to be received in the future for research and development activities are deferred and recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed. Selling, General and Administrative Selling, general and administrative expenses consist primarily of salaries and related costs, including equity-based compensation for personnel in executive, finance, corporate, commercial, business development and administrative functions. Selling, general and administrative expenses also include consulting services, legal fees relating to patent and corporate matters; professional fees for accounting, auditing and tax services; insurance costs; administrative travel expenses; and facility-related expenses, which include direct depreciation costs and expenses for rent and maintenance of facilities and other operating expenses. Equity-based compensation expense Equity-based compensation expense is recognized using the straight-line method, based on the grant date fair value, over the requisite service period of the award, which is generally the vesting term. The Company recognizes forfeitures at the time of the actual forfeiture event. For awards subject to performance conditions, as well as awards containing both market and performance conditions, the Company recognizes equity award compensation expense using an accelerated recognition method over the remaining service period when management determines that achievement of the milestone is probable. Management evaluates when the achievement of a performance-based milestone is probable based on the expected satisfaction of the performance conditions as of the reporting date. The grant-date fair value of performance-based awards with market conditions is estimated using a Monte Carlo simulation method that incorporates the probability of the performance conditions being met as of the grant date. For stock options issued, the Company estimates the grant date fair value and the resulting equity-based compensation expense using the Black-Scholes option-pricing model. The Black-Scholes option-pricing model requires the use of certain subjective assumptions which determine the fair value of equity-based awards. Inputs used in the Black-Scholes option-pricing model are: • Fair value of common stock, which is the trading price of the Company’s common stock on the grant date of the award. • Expected term — The expected term represents the period that the equity-based awards are expected to be outstanding. The Company uses the simplified method to calculate the expected term due to the limited Company-specific historical information available for the Company. • Expected volatility — The Company lacks sufficient Company-specific historical and implied volatility information. Therefore, the Company includes the historical volatility of a publicly traded set of peer companies to determine its expected stock volatility and expects to continue to do so until it has adequate historical data regarding the volatility of its own traded stock. • Risk-free interest rate — The risk-free interest rate is based on the U.S. Treasury yield in effect at the time of grant for zero-coupon U.S. Treasury notes with maturities approximately equal to the expected term of the awards. • Expected dividend — The Company has never paid dividends on its common units or stock and has no plans to pay dividends on its common stock. Therefore, the expected dividend yield is zero. Net Loss per Share Basic net loss per share is computed by dividing net loss by the weighted average number of shares outstanding for the period. Diluted net loss per share excludes the potential impact of unvested restricted stock and stock options because their effect would be anti-dilutive due to the Company’s net loss. Since the Company had a net loss in each of the periods presented, basic and diluted net loss per share are the same. Income Taxes Income taxes are accounted for using the asset-and-liability method. Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax basis and operating loss and tax credit carryforwards. Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized as income in the period that includes the enactment date. Changes in deferred tax assets and liabilities are recorded in the provision for income taxes. The Company recognizes deferred tax assets to the extent that it believes that these assets are more likely than not to be realized. In making such a determination, management considers all available positive and negative evidence, including future reversals of existing taxable temporary differences, projected future taxable income, tax-planning strategies and results of recent operations. Valuation allowances are provided, if based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. If management determines that the Company would be able to realize its deferred tax assets in the future in excess of their net recorded amount, management would make an adjustment to the deferred tax asset valuation allowance, which would reduce the provision for income taxes. The Company records uncertain tax positions in accordance with ASC 740 on the basis of a two-step process in which (1) management determines whether it is more likely than not that the tax positions will be sustained on the basis of the technical merits of the position and (2) for those tax positions that meet the more-likely-than-not recognition threshold, management recognizes the largest amount of tax benefit that is more than 50% likely to be realized upon ultimate settlement with the related tax authority. The Company provides reserves for potential payments of tax to various tax authorities related to uncertain tax positions. These reserves are based on a determination of whether and how much of a tax benefit taken by the Company in its filings or positions is more likely than not to be realized following resolution of any potential contingencies related to the tax benefit. Potential interest related to the underpayment of income taxes will be classified as a component of income tax expense and any related penalties will be classified as income tax expense. Recently Adopted and Recently Issued Accounting Pronouncements There were no recently adopted accounting pronouncements that had a material impact on the Company's financial statements. In December 2023, the FASB issued ASU 2023-09, Improvements to Income Tax Disclosures, to provide more detailed income tax disclosure requirements. The guidance requires entities to disclose disaggregated information about their effective tax rate reconciliation as well as information on income taxes paid. The disclosure requirements should be applied on a prospective basis, with the option to apply it retrospectively. The effective date for the standard is for fiscal years beginning after December 15, 2024. Early adoption is permitted. The Company does not expect ASU 2023-09 to have a material impact on the Company's financial statements. |
Marketable Securities
Marketable Securities | 12 Months Ended |
Dec. 31, 2023 | |
Investments, Debt and Equity Securities [Abstract] | |
Marketable Securities | Marketable Securities The following table summarizes the Company’s available-for-sale marketable securities as of December 31, 2023 and December 31, 2022: As of December 31, 2023 (in thousands) Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value Marketable securities: Short-term investments: U.S. government securities $ 228,579 $ 212 $ — $ 228,791 Corporate debt securities 9,629 2 — 9,631 Commercial paper 64,724 3 — 64,727 Long-term investments: U.S. government securities 141,102 755 141,857 Corporate debt securities 41,310 219 — 41,529 Total $ 485,344 $ 1,191 $ — $ 486,535 As of December 31, 2022 (in thousands) Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value Marketable securities: Short-term investments: U.S. government securities $ 232,229 $ — $ (690) $ 231,539 Non-U.S. government securities 9,388 — (31) 9,357 Corporate debt securities 45,710 — (44) 45,666 Commercial paper 238,160 — — 238,160 Long-term investments: Corporate debt securities 4,796 (2) 4,794 Total $ 530,283 $ — $ (767) $ 529,516 The Company’s marketable securities are available-for-sale securities and consist of high-quality, highly liquid debt securities including corporate debt securities, U.S. government securities, non-U.S. government securities, and commercial paper. The Company’s securities classified as short-term marketable securities mature within one year or less of the balance sheet date. Marketable securities that mature greater than one year from the balance sheet date are classified as long-term. As of December 31, 2023, the Company did not hold any investments that matured beyond five years. |
Fair Value Measurements
Fair Value Measurements | 12 Months Ended |
Dec. 31, 2023 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | Fair Value Measurements The fair value of the Company’s financial assets measured on a recurring basis are classified based upon a fair value hierarchy consisting of the following three levels: Level 1 — Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets, or liabilities. Level 2 — Quoted prices for similar assets and liabilities in active markets, quoted prices in markets that are not active, or inputs which are observable, either directly or indirectly, for substantially the full term of the instrument. Level 3 — Prices or valuation techniques that require inputs that are both significant to the fair value measurement and unobservable (i.e., supported by little or no market activity). The fair value hierarchy is based on inputs to valuation techniques used to measure fair value that are either observable or unobservable. Observable inputs reflect assumptions market participants would use in pricing an asset or liability based on market data obtained from independent sources while unobservable inputs reflect a reporting entity's pricing based upon their own market assumptions. The following tables sets forth the fair value hierarchy of the Company’s financial assets and liabilities measured on a recurring basis as of December 31, 2023 and December 31, 2022: As of December 31, 2023 Fair Value Hierarchy (in thousands) Total Level 1 Level 2 Level 3 Financial instruments carried at fair value (asset position): Cash equivalents: Money market funds $ 14,434 $ 14,434 $ — $ — Commercial paper 49,631 — 49,631 — Short-term investments: U.S. government securities 228,791 228,791 — — Corporate debt securities 9,631 — 9,631 — Commercial paper 64,727 — 64,727 — Long-term investments: U.S. government securities 141,857 141,857 — — Corporate debt securities 41,529 — 41,529 — Total $ 550,600 $ 385,082 $ 165,518 $ — As of December 31, 2022 Fair Value Hierarchy (in thousands) Total Level 1 Level 2 Level 3 Financial instruments carried at fair value (asset position): Cash equivalents: Money market funds $ 22,494 $ 22,494 $ — $ — Short-term investments: U.S. government securities 231,539 231,539 — — Non-U.S. government securities 9,357 — 9,357 — Corporate debt securities 45,666 — 45,666 — Commercial paper 238,160 — 238,160 — Long-term investments: Corporate debt securities 4,794 — 4,794 — Total $ 552,010 $ 254,033 $ 297,977 $ — The Company’s financial assets measured at fair value on a recurring basis included cash equivalents, which consist of money market funds, commercial paper and marketable securities. The Company’s money market funds are readily convertible into cash and the net asset value of each fund on the last day of the quarter is used to determine fair value. The U.S. government securities are classified as Level 1 and valued utilizing quoted market prices. The Company’s corporate debt securities, non-U.S. government securities, and commercial paper are classified as Level 2 and valued utilizing various observable market and industry inputs. The carrying amounts reflected in the Company’s consolidated balance sheets for cash equivalents, accounts receivable, accounts payable, and accrued expenses approximate fair value due to their short-term nature. |
Property and Equipment, net
Property and Equipment, net | 12 Months Ended |
Dec. 31, 2023 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, net | Property and Equipment, net Property and equipment, net consisted of the following: December 31, (in thousands) 2023 2022 Useful Life Leasehold improvements $ 826 $ 826 Length of lease or 5 years, whichever is shorter Computer equipment 380 368 3-5 years Lab equipment 2,439 — 5 - 15 years Furniture 437 437 5 years Software 12,954 6,048 3-10 years Construction in process 3,565 7,137 Property and equipment, gross 20,601 14,816 Less accumulated depreciation (2,658) (1,245) Property and equipment, net $ 17,943 $ 13,571 Depreciation expense was $1.6 million, $0.8 million, and $0.5 million for the years ended December 31, 2023, December 31, 2022 and December 31, 2021, respectively. |
Intangible Assets, net
Intangible Assets, net | 12 Months Ended |
Dec. 31, 2023 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Intangible Assets, net | Intangible Assets, net Intangible assets, net, which is included in other assets, consisted of the following: December 31, (in thousands) 2023 2022 Definite-lived intangible assets License agreements $ 11,250 $ — Intangible assets, gross 11,250 — Less accumulated amortization (50) — Intangible assets, net $ 11,200 $ — The Company's finite-lived intangible asset results from the commercial milestone payment due under its license agreement with Pfizer, Inc., or Pfizer. For intangible assets related to products with patent exclusivity, the useful life is the remaining patent exclusivity period of approximately 19.4 years. The Company incurred amortization expense of $0.1 million for the year ended December 31, 2023. The expected future amortization expense for amortizable finite-lived intangible assets as of December 31, 2023 is as follows: (in thousands) 2024 $ 600 2025 600 2026 600 2027 600 2028 600 Thereafter 8,200 Total future expected amortization expense $ 11,200 |
Leases
Leases | 12 Months Ended |
Dec. 31, 2023 | |
Leases [Abstract] | |
Leases | Leases Operating Leases The company’s operating leases relate to real estate. In August 2018, the Company entered into a five-year operating lease in Durham, NC, for additional office space which houses various corporate functions including clinical development operations. In May 2023, the Company amended this lease agreement to extend the lease term through September 30, 2026, with two consecutive five-year renewal options. Pursuant to the amendment, the lease payments increase by 3.0% each year, commencing October 1, 2023. In October 2018, the Company entered into a lease for its corporate headquarters in Stamford, CT. In January 2022, the Company amended this lease agreement to extend the lease term through April 2028, with two five-year renewal options or one ten-year renewal option. Pursuant to the amended agreement, lease payments increase by 2.5% each year. In March 2023, the Company entered into a five-year operating lease in Research Triangle Park in Durham, NC (the location of the Company’s discovery lab and translational operations), with two consecutive five-year renewal options. Pursuant to the amended agreement, lease payments increase by 3.0% in each of the subsequent four years of the five-year operating lease term. Rental payments under the renewal period will be at current market rates for the premises. The components of lease cost recorded in the Company’s consolidated statement of operations were as follows: Twelve Months Ended December 31, (in thousands) 2023 2022 2021 Operating lease cost Fixed $ 1,653 $ 1,131 $ 993 Variable (1) 866 336 548 Total lease cost $ 2,519 $ 1,467 $ 1,541 (1) Variable lease costs consist primarily of taxes, utilities and common area maintenance costs. The Company’s leases are included on its consolidated balance sheets as follows: (in thousands) As of December 31, 2023 As of December 31, 2022 Operating leases Operating lease right-of-use-assets $ 6,144 $ 4,698 Total operating lease assets $ 6,144 $ 4,698 Operating lease liabilities, current $ 1,061 $ 483 Operating lease liabilities, long-term 5,996 4,768 Total operating lease liabilities $ 7,057 $ 5,251 Maturities of the Company’s operating lease liabilities as of December 31, 2023 were as follows: (in thousands) Operating Leases 2024 $ 1,476 2025 1,999 2026 2,002 2027 1,904 2028 646 Thereafter — Total lease payments 8,027 Less: imputed interest (970) Present value of lease liabilities $ 7,057 The weighted-average remaining lease term and discount rate related to the Company’s leases were as follows: As of December 31, 2023 As of December 31, 2022 Weighted-average remaining lease term (in years) Operating leases 4.2 5.2 Weighted-average discount rate Operating leases 5.8 % 4.2 % Supplemental cash flow information related to the Company’s leases was as follows: (in thousands) December 31, 2023 December 31, 2022 December 31, 2021 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 1,293 $ 859 $ 1,388 Right-of-use assets obtained in exchange for new operating lease liabilities 2,637 5,580 — |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Dec. 31, 2023 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | Accrued Expenses Accrued expenses consisted of the following: December 31, December 31, (in thousands) 2023 2022 Accrued compensation and benefits $ 26,047 $ 19,142 Accrued research and development 15,129 12,321 Accrued milestone 11,250 — Accrued other 13,143 7,779 Total accrued expenses $ 65,569 $ 39,242 |
Equity- Based Compensation
Equity- Based Compensation | 12 Months Ended |
Dec. 31, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Equity- Based Compensation | Equity-Based Compensation The Company recorded total equity-based compensation expense for the periods presented as follows: Year Ended December 31, (in thousands) 2023 2022 2021 Research and development $ 34,398 $ 29,373 $ 14,664 Selling, general and administrative 60,136 43,592 23,780 Total equity-based compensation expense $ 94,534 $ 72,965 $ 38,444 2019 Equity Incentive Plan The 2019 Equity Incentive Plan provides for the grant of incentive stock options, nonqualified stock options, stock appreciation rights, restricted stock units, restricted stock awards, unrestricted stock awards and dividend equivalent rights to the Company’s officers, employees, directors and other key persons (including consultants). The number of shares available for issuance under the 2019 Equity Incentive Plan is cumulatively increased each January 1, through and including January 1, 2030, by 5% of the number of shares of the Company’s common stock outstanding on the immediately preceding December 31 or such lesser number of shares determined by the Company’s compensation committee. As of December 31, 2023, there were 2,543,163 shares available for future issuance under the 2019 Equity Incentive Plan. The terms of stock options and restricted stock awards, including vesting requirements, are determined by the Board of Directors or its delegates, subject to the provisions of the 2019 Equity Incentive Plan. Stock options and restricted stock awards granted by the Company to employees generally vest over 3 or 4 years, and stock options and restricted stock awards granted by the Company to directors generally vest over 1 or 3 years. 2019 Employee Stock Purchase Plan On August 30, 2019, the Company’s stockholders approved the 2019 Employee Stock Purchase Plan, or the ESPP, which became effective immediately preceding the effectiveness of the Company’s registration statement on September 12, 2019 in connection with the IPO. A total of 442,153 shares of common stock were reserved for issuance under the ESPP. In addition, the number of shares of common stock that may be issued under the ESPP will automatically increase each January 1, through and including January 1, 2028, by the lesser of (i) 663,229 shares of common stock, (ii) 1% of the number of shares of the Company’s common stock outstanding on the immediately preceding December 31 or (iii) such lesser number of shares determined by the administrator of the ESPP. As of December 31, 2023, there were 2,477,122 shares reserved for issuance under the ESPP. No offering periods under the ESPP had been initiated as of December 31, 2023. Stock Options A summary of the changes in the Company’s stock options during the periods presented is as follows: Shares Weighted Average Exercise Weighted Intrinsic Aggregate Outstanding at December 31, 2020 4,505,546 $ 15.51 8.7 $ 256,860,405 Granted 2,547,813 73.37 — — Exercised (151,643) 7.62 — — Forfeited/cancelled (188,303) 37.34 — — Outstanding at December 31, 2021 6,713,413 37.03 8.2 196,012,147 Granted 3,207,347 43.28 — — Exercised (223,467) 8.84 — — Forfeited/cancelled (520,478) 68.33 — — Outstanding at December 31, 2022 9,176,815 38.13 8.0 53,719,297 Granted 2,994,704 27.65 — — Exercised (44,250) 3.15 — — Forfeited/cancelled (538,693) 44.01 — — Outstanding at December 31, 2023 11,588,576 35.28 7.4 117,631,827 Exercisable at December 31, 2023 6,711,390 32.83 6.6 88,814,151 Aggregate intrinsic value is calculated by subtracting the exercise price of the option from the closing price of the Company’s common stock on closing date, multiplied by the number of shares per each option. Assumptions used in determining the fair value of the stock options granted in 2023 include risk-free interest rates of 3.46% – 4.73%, expected dividend yield of 0.00%, expected term in years of 5.5 years - 6.1 years and expected volatility of 76.7% - 78.8%. At December 31, 2023, the total unrecognized compensation expense related to unvested stock options was $117.7 million, which the Company expects to recognize over a weighted-average remaining period of approximately 2.4 years. For the year ended December 31, 2023, total equity-based compensation expense for stock options was $66.0 million. Restricted Stock Awards A summary of the changes in the Company’s restricted stock awards for the periods presented is as follows: Number Weighted Unvested and outstanding at December 31, 2020 686,868 $ 1.26 Granted 332,226 73.31 Vested (493,309) 1.19 Forfeited (55,475) 13.75 Unvested and outstanding at December 31, 2021 470,310 50.76 Granted 36,625 61.74 Vested (257,219) 32.03 Forfeited (27,957) 79.25 Unvested and outstanding at December 31, 2022 221,759 70.71 Granted — — Vested (93,812) 71.50 Forfeited (21,113) 63.38 Unvested and outstanding at December 31, 2023 106,834 70.48 At December 31, 2023, the total unrecognized compensation expense related to unvested restricted stock awards was $2.6 million, which the Company expects to recognize over a weighted-average remaining period of approximately less than one year. For the year ended December 31, 2023, total restricted stock awards compensation expense was $6.6 million. Restricted Stock Units A summary of the changes in the Company’s restricted stock units for the periods presented is as follows: Number Weighted Unvested and outstanding at December 31, 2021 — $ — Granted 738,508 49.77 Vested (24,369) 24.62 Forfeited (42,185) 58.76 Unvested and outstanding at December 31, 2022 671,954 50.11 Granted 1,163,390 27.68 Vested (238,725) 49.14 Forfeited (110,079) 31.27 Unvested and outstanding at December 31, 2023 1,486,540 34.11 At December 31, 2023, the total unrecognized compensation expense related to unvested restricted stock units was $32.9 million, which the Company expects to recognize over a weighted-average remaining period of approximately 1.9 years. For the year ended December 31, 2023, total restricted stock unit compensation expense was $19.4 million. Performance Stock Units On January 5, 2023, the CEO was granted a supplemental equity award consisting of performance based restricted stock units, or the 2023 CEO PSU award. The 2023 CEO PSU award covers a target of 284,362 shares of the Company's common stock, which are subject to vesting based upon the passage of time, once the Company achieves certain regulatory milestones and the CEO’s continued service with the Company. In addition, the target number of shares of the Company's common stock covered is subject to modification (upwards or downwards) by up to 25% based upon the relative total shareholder return of the Company’s shares compared to the NASDAQ Biotech Index as of the end of the performance period. At December 31, 2023, the total unrecognized compensation expense related to unvested performance stock units was $4.3 million, which the Company expects to recognize over a weighted-average remaining period of approximately 2.0 years. For the year ended December 31, 2023, total performance stock unit compensation expense was $2.4 million. |
License and Collaboration Agree
License and Collaboration Agreements | 12 Months Ended |
Dec. 31, 2023 | |
License and Collaboration Agreements | |
License and Collaboration Agreements | License and Collaboration Agreements Pfizer Inc. Pursuant to the terms of its licenses with Pfizer, the Company is required to pay Pfizer milestones payments of up to an aggregate of $232.5 million for nirogacestat and up to an aggregate of $229.8 million for mirdametinib, each upon achievement of certain commercial milestone events. Royalties are also payable under each License Agreement based on a specified percentage of net sales ranging from mid-single digit percentages to low 20s. Royalty payments under each License Agreement continue until the expiration of the last to expire licensed patent applicable to such product, but not less than ten years after the first commercial sale on a country-by-country basis. A milestone was met upon the first commercial sale of OGSIVEO in December 2023, and a milestone payment of $11.3 million is due to Pfizer, which has been accrued and included in accrued expenses as of December 31, 2023, and is expected to be paid in the second quarter of 2024. In August 2018, the Company entered into a clinical collaboration agreement with BeiGene Ltd., or BeiGene to conduct a clinical study of the combination of mirdametinib and a BeiGene compound designated as lifirafenib. In accordance with the terms of the agreement, the Company and BeiGene share equally the costs associated with the clinical study. BeiGene is required to supply the BeiGene compound and the Company is required to supply mirdametinib to conduct the clinical study. The collaboration is guided by a joint steering committee. Specified areas of development require unanimous agreement among all members of the joint steering committee. The Company recorded expense of $1.9 million for the year ended December 31, 2023, and $1.1 million for each of the years ended December 31, 2022 and December 31, 2021, in connection with this collaboration agreement. TEAD inhibitor portfolio license agreement In May 2021, the Company announced an exclusive worldwide license agreement with Katholieke Universiteit Leuven, or KU Leuven, and the Flanders Institute for Biotechnology, or VIB, pursuant to which the Company in-licensed a portfolio of novel small molecule inhibitors of the TEA Domain, or TEAD, family of transcription factors, designed for the potential treatment of biomarker-defined solid tumors driven by aberrant Hippo pathway signaling. Under the terms of the agreement, the Company made an upfront payment of $11 million to KU Leuven and VIB, which was recorded as research and development expense in the consolidated statement of operations. Pursuant to the terms of the agreement, KU Leuven and VIB are also eligible to receive, in the aggregate, up to $120 million in development milestones, up to $165 million in commercial milestones and tiered single-digit percentage royalties based on any future net sales of products developed based on the in-licensed technology. EGFR inhibitor portfolio license agreement and sponsored research agreement In October 2021, the Company announced an exclusive worldwide license agreement with Dana-Farber Cancer Institute, or Dana-Farber, pursuant to which it in-licensed a portfolio of novel small molecule inhibitors of Epidermal Growth Factor Receptor, or EGFR, designed for the treatment of EGFR-mutant lung cancers. Under the terms of the agreement, the Company made an upfront payment of $0.3 million to Dana-Farber, which was recorded as research and development expense in the consolidated statement of operations. Pursuant to the terms of the agreement, Dana-Farber is also eligible to receive up to $2.3 million in development milestones, up to $39 million in commercial milestones and single-digit percentage royalties based on any future net sales of products developed based on the in-licensed technology. Concurrent with this license agreement, the Company entered a multi-year sponsored research agreement with Stanford Medicine to fund continued research and development in a laboratory at Stanford Medicine as well as collaborating laboratories at Dana-Farber. This sponsored research agreement is intended to support lead optimization and translational biology efforts as the EGFR inhibitor portfolio advances towards development candidate nomination. GSK expanded non-exclusive license and collaboration agreement In September 2022, the Company announced an expansion of its ongoing, non-exclusive clinical collaboration with GSK, which originally commenced in June 2019. The announcement coincided with the entry by the Company and GSK into an amended and restated collaboration and license agreement, or the GSK License Agreement, for the potential continued development and commercialization of nirogacestat in combination with either belantamab mafodotin (belamaf), GSK’s antibody-drug conjugate, or ADC, targeting B-cell maturation antigen, or BCMA, or any other cytotoxic ADC targeting BCMA derived from belantamab that is controlled by GSK, either alone as a combination therapy, or together with other pharmaceutical agents. Pursuant to the terms of the GSK License Agreement and concurrent with the execution of such agreement, the Company entered into a Stock Purchase Agreement with GGL, under which GGL purchased 2,050,819 shares of the Company’s Common Stock in a private placement transaction for an aggregate purchase price of approximately $75.0 million, or $36.57 per share. The shares were sold at a 25% premium to the volume-weighted average share price of the Company’s Common Stock for a specified 30-day period prior to entering into the Stock Purchase Agreement. The fair value of the Common Stock based on the closing price of Common Stock on the day prior to the effective date of the Stock Purchase Agreement was $55.5 million and was recorded to equity. The $19.5 million received in excess of the fair value of the Common Stock represents consideration for the license for the potential continued development and commercialization of nirogacestat in combination with GSK compounds, together with the clinical supply of nirogacestat for future belamaf clinical trials and certain research and development costs associated with nirogacestat. The Company recorded the $19.5 million as deferred revenue in September of 2022 and will recognize revenue as the corresponding performance obligation is satisfied in proportion to expenses incurred, including clinical supply and research and development expenses, associated with the GSK License Agreement. For the year ended December 31, 2023, the Company did not recognize any revenue related to the upfront consideration received from the GSK License Agreement. Under the terms of the GSK License Agreement, the Company is also eligible to receive up to $550.0 million in additional payments, if certain development and commercial milestones are met. The Company continues to retain full commercial rights to nirogacestat. Additionally, SpringWorks will seek to make nirogacestat commercially available in markets where approval has been sought by GSK for a combination with belamaf. Jazz Pharmaceuticals asset purchase and exclusive license agreement In October 2020, the Company and Jazz announced an asset purchase and exclusive license agreement, pursuant to which Jazz acquired the Company’s fatty acid amide hydrolase, or FAAH, inhibitor program including PF-4457845. The FAAH inhibitor program was obtained by the Company as part of the License Agreements in 2018. Jazz made an upfront payment of $35 million to the Company with potential future payments of up to $375 million based upon the achievement of certain clinical development, regulatory, and commercial milestones. In addition, Jazz is obligated to pay the Company tiered sales-based royalties on future net sales of PF-4457845 in the single-digit range. Pursuant to the Jazz Agreement, Jazz is obligated to use commercially reasonable efforts to develop and seek regulatory approval for at least one product in the United States and if regulatory approval is obtained, to commercialize such product in the United States. |
Equity Method Investment
Equity Method Investment | 12 Months Ended |
Dec. 31, 2023 | |
Equity Method Investments and Joint Ventures [Abstract] | |
Equity Method Investment | Equity Method Investment MapKure In June 2019, the Company announced the formation of MapKure LLC, or MapKure, an entity jointly owned by the Company and BeiGene Ltd., or BeiGene. BeiGene licensed to MapKure exclusive rights to brimarafenib (BGB-3245), an investigational oral, small molecule selective inhibitor of specific BRAF driver mutations and genetic fusions. MapKure is advancing brimarafenib through clinical development for solid tumor patients harboring BRAF driver mutations and genetic fusions that were observed to be sensitive to the compound in preclinical studies. In addition to the Company’s equity ownership in MapKure, the Company maintains a member on each of MapKure’s joint steering committee and board of directors. The Company also contributes to clinical development and other operational activities for brimarafenib through a service agreement with MapKure. In conjunction with the formation of MapKure in June 2019, the Company purchased 3,500,000 Series A preferred units of MapKure, or a 25.0% ownership interest, for $3.5 million and in June 2020, the Company purchased an additional 3,500,000 Series A preferred units of MapKure for $3.5 million, increasing its ownership interest to 38.9%, as required by the terms of the Series A unit purchase agreement. In June 2022, the Company made an additional investment in MapKure and purchased 4,200,000 Series B preferred units of MapKure for $4.2 million, pursuant to the terms of a Series B preferred unit purchase agreement. In January 2023, pursuant to terms of the Series B preferred unit purchase agreement, the Company purchased an additional 2,800,000 Series B preferred units of MapKure for $2.8 million. As of December 31, 2023, the Company’s ownership interest in MapKure was 38.9%. The Company determined that MapKure is a variable interest entity. The Company is not the primary beneficiary, as the Company does not have the power to direct the activities that most significantly impact the economic performance of MapKure. Accordingly, the Company does not consolidate the financial statements of this entity and accounts for this investment using the equity method of accounting. The Company reaffirmed its assessment as of December 31, 2023. The Company records its portion of MapKure’s earnings or losses based on a one quarter lag. For the year ended December 31, 2023, the Company recognized a $5.0 million loss for its portion of MapKure’s losses. The Company’s investment in MapKure is included in equity method investment. The balance of the Company’s investment was $2.0 million at December 31, 2023, representing the maximum exposure to loss as a result of the Company’s involvement with MapKure. In January 2024, as part of a Series C financing round, the Company made an additional investment in MapKure and purchased 8,235,200 Series C preferred units of MapKure for $8.2 million, pursuant to the terms of a Series C preferred unit purchase agreement. The Company is required to make subsequent purchases at each of the second and third closings established by such agreement, in each case for an additional 6,176,400 Series C preferred units of MapKure for $6.2 million. This additional investment does not significantly modify the Company’s ownership interest or the Company’s ability to direct the activities that most significantly impact the economic performance of MapKure. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2023 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | Commitments and Contingencies As of December 31, 2023, and December 31, 2022, the Company had obligations consisting of operating leases for facilities. Refer to Footnote 8: Leases for more information. The Company enters into contracts in the normal course of business for clinical trials, preclinical studies, manufacturing and other services and products for operating purposes. These contracts generally provide for termination following a certain period after notice and the Company believes that non-cancelable obligations under these agreements are insignificant. Contingencies From time to time, the Company may be involved in disputes or regulatory inquiries that arise in the ordinary course of business. When the Company determines that a loss is both probable and reasonably estimable, a liability is recorded and disclosed if the amount is material to the financial statements taken as a whole. When a material loss contingency is only reasonably possible, the Company does not record a liability, but instead discloses the nature and the amount of the claim, and an estimate of the loss or range of loss, if such an estimate can reasonably be made. As of December 31, 2023, and December 31, 2022, there was no litigation or contingency that created at least a reasonable possibility of a material loss. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2023 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | Income Taxes As of December 31, 2023 and December 31, 2022, the Company did not have a current or deferred income tax expense or benefit as the Company has incurred losses since inception. As of December 31, 2023, the Company has federal, state and city net operating loss carryforwards of $472.1 million, $365.5 million and $3.7 million, respectively, which are available to reduce future taxable income. Federal net operating loss carryforwards generated 2018 through 2023 of $467.8 million, will be available to offset 80% of taxable income for an indefinite period of time, until fully utilized. Federal net operating loss carryforwards of $4.3 million reported in 2017, and the state and city net operating loss carryforwards expire at various dates through 2037. The Company also has federal tax credits of $33.5 million, which may be used to offset future tax liabilities. These tax credit carryforwards will expire at various dates beginning in 2038. The net operating loss and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities. Net operating loss and tax credit carryforwards may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant shareholders over a three-year period in excess of 50%, as defined under Sections 382 and 383 of the Internal Revenue Code, respectively, as well as similar state provisions and other provisions within the Internal Revenue Code. This could limit the amount of tax attributes that can be utilized annually to offset future taxable income or tax liabilities. The amount of the annual limitation is determined based on the value of the Company immediately prior to an ownership change. Subsequent ownership changes may further affect the limitation in future years. The Company has not recorded any reserves for uncertain tax positions as of December 31, 2023 or December 31, 2022. Interest and penalty charges, if any, related to unrecognized tax benefits will be recorded as income tax expense. As of December 31, 2023, the Company had no accrued interest or penalties related to uncertain tax positions. Since the Company is in a loss carryforward position, it is generally subject to examination by the U.S. federal, state and local income tax authorities for all tax years in which a loss carryforward is available. The Company is not currently under examination by the Internal Revenue Service or any other jurisdictions for any tax years. The principal components of deferred tax assets and liabilities are as follows: As of December 31, (in thousands) 2023 2022 Deferred tax assets: Net operating loss carryforwards $ 121,029 $ 93,032 Research and development credits 6,069 3,422 Orphan drug credit 27,515 19,401 Capitalized research and development 41,588 21,262 Equity-based compensation 26,429 20,286 Deferred revenue 4,105 — Other 3,031 1,525 Total deferred tax assets 229,766 158,928 Deferred tax liability: Operating lease right-of-use assets (1,290) (987) Depreciation (1,255) (113) Other (2) (2) Valuation allowance (227,219) (157,826) Net deferred tax assets $ — $ — A valuation allowance is recorded to reduce the deferred tax assets reported if, based on the weight of available evidence, it is more likely than not that some portion or all of the deferred tax assets will not be realized. After consideration of all evidence, both positive and negative, the Company has recorded a full valuation allowance against its deferred tax assets at December 31, 2023 and December 31, 2022 because the Company has determined that it is more likely than not that these assets will not be realized. The increase in the valuation allowance of $69.4 million in 2023 primarily relates to the net loss incurred by the Company as well as federal research and orphan drug credits generated. The effective tax rate for the Company for the years ended December 31, 2023, December 31, 2022 and December 31, 2021 was zero percent. A reconciliation of the income tax expense at the federal statutory tax rate to the Company’s effective income tax rate follows: Year Ended December 31, 2023 2022 2021 Statutory tax rate 21.00 % 21.00 % 21.00 % U.S. state and local income taxes, net of U.S. federal income tax benefit 2.17 2.33 5.15 Equity-based compensation (1.21) (0.77) (0.03) Research and development credit 0.81 0.27 0.50 Orphan drug credit 2.50 1.87 4.99 Section 162(m) limitation (3.39) — — Other (0.54) — — Change in valuation allowance (21.34) (24.70) (31.61) Effective tax rate — % — % — % |
401(k) Plan
401(k) Plan | 12 Months Ended |
Dec. 31, 2023 | |
Retirement Benefits [Abstract] | |
401(k) Plan | 401(k) Plan |
Net Loss per Share
Net Loss per Share | 12 Months Ended |
Dec. 31, 2023 | |
Earnings Per Share [Abstract] | |
Net Loss per Share | Net Loss per Share Basic and diluted net loss per unit and share is calculated as follows: Year Ended December 31, (in thousands, except share and per-share data) 2023 2022 2021 Numerator: Net loss $ (325,104) $ (277,417) $ (173,910) Net loss attributable to common stockholders (325,104) (277,417) (173,910) Denominator: Weighted average shares outstanding, basic and diluted 63,123,936 53,290,528 48,497,790 Net loss per share, basic and diluted $ (5.15) $ (5.21) $ (3.59) Potentially dilutive securities that were not included in the diluted per share calculations because they would be anti-dilutive were as follows: As of December 31, 2023 2022 Common stock options issued and outstanding 11,588,576 9,176,815 Restricted stock units subject to future vesting 1,486,540 671,954 Performance stock units subject to future vesting 284,362 — Restricted stock awards subject to future vesting 106,834 221,759 Total potentially dilutive securities 13,466,312 10,070,528 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Pay vs Performance Disclosure | |||
Net loss | $ (325,104) | $ (277,417) | $ (173,910) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended |
Dec. 31, 2023 | |
Trading Arrangements, by Individual | |
Rule 10b5-1 Arrangement Adopted | false |
Non-Rule 10b5-1 Arrangement Adopted | false |
Rule 10b5-1 Arrangement Terminated | false |
Non-Rule 10b5-1 Arrangement Terminated | false |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2023 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation These consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States, or U.S. GAAP. |
Principles of Consolidation | Principles of Consolidation The consolidated financial statements include the accounts of SpringWorks Therapeutics, Inc. and its subsidiaries, collectively, the Company. All intercompany transactions and balances have been eliminated in consolidation. Investments in business entities in which the Company lacks control but does have the ability to exercise significant influence over operating and financial policies are accounted for using the equity method of accounting. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts in the financial statements and accompanying notes. Significant estimates and assumptions reflected in these consolidated financial statements include, but are not limited to, accrued research and development expenses and the valuation of equity-based compensation awards. The Company bases its estimates on historical experience, known trends and other market-specific or relevant factors that it believes to be reasonable under the circumstances. Actual results may differ from those estimates. On an ongoing basis, management evaluates its estimates, and adjusts those estimates and assumptions when facts or circumstances change. Changes in estimates are recorded in the period in which they become known. |
Segment Information | Segment Information Operating segments are defined as components of an entity about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating segment. |
Cash and Cash Equivalents | Cash and Cash Equivalents |
Marketable Securities | Marketable Securities Marketable debt securities are reported at fair value with unrealized gains and losses included in accumulated other comprehensive income. Each reporting period, the Company evaluates whether there are declines in fair value below amortized cost and if these declines are due to credit losses, as well as the Company’s ability and intent to hold the investment until a forecasted recovery occurs. If both criteria regarding the intent or ability to hold are met, any decline in fair value due to credit losses is recorded as an allowance through other income (expense); limited by the amount that the fair value is less than the amortized costs basis. If either criterion is not met, any previously recorded allowance for credit losses and any excess amortized cost basis over fair value is recorded in other income (expense). As of, and for the years ended December 31, 2023 and December 31, 2022, the Company did not have any allowance for credit losses or impairments of its marketable securities. |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of cash, cash equivalents and available-for-sale marketable securities. The Company maintains each of its cash, cash equivalent balances and marketable securities balances with high quality, financial institutions and the Company’s marketable securities are invested in high-quality, highly liquid debt securities including corporate debt securities, U.S. government securities and commercial paper. |
Accounts Receivable, net | Accounts Receivable, net Accounts receivable, net consists of trade receivables which are amounts due from customers related to product sales. The Company records trade receivables net of chargebacks, invoice discounts, distribution service fees and any allowances for potential credit losses. An allowance for credit losses is determined based on the financial condition and creditworthiness of customers and the Company considers economic factors and events or trends expected to affect future collections experience. Any allowance would reduce the net receivables to the amount that is expected to be collected. The payment history of the Company’s customers will be considered in future assessments of collectibility as these patterns are established over a longer period of time. As of December 31, 2023, the Company determined an allowance for credit losses was insignificant. |
Inventory and Pre-Approval Inventory | Inventory and Pre-Approval Inventory The Company began capitalizing inventory for OGSIVEO upon approval by the FDA in November 2023. OGSIVEO is approved for the treatment of adult patients with desmoid tumors. Prior to regulatory approval, all direct and indirect manufacturing costs were charged to research and development expense in the period incurred. |
Property and Equipment | Property and Equipment Property and equipment consist of computer equipment, software, furniture and leasehold improvements and are recorded at cost. Property and equipment are depreciated on a straight-line basis over their estimated useful lives. |
Intangible Asset, net | Intangible Asset, net The Company’s finite-lived intangible asset resulted from the capitalization of a milestone payment due under a license and collaboration agreement in connection with the first commercial sale of OGSIVEO in the United States in December 2023. The intangible asset will be amortized on a straight-line basis over its remaining useful life, which is estimated to be the remaining patent life of OGSIVEO. Amortization expense is recorded as cost of product revenue in the consolidated statement of operations. |
Impairment of Long-Lived Assets | Impairment of Long-Lived Assets The Company reviews its long-lived assets, including property and equipment and finite-lived intangible assets, for impairment whenever events or changes in circumstances indicate that the carrying value of an asset may not be recoverable. If such circumstances are determined to exist, an estimate of undiscounted future cash flows produced by the long-lived asset, including its eventual residual value, is compared to the carrying value to determine whether impairment exists. In the event that such cash flows are not expected to be sufficient to recover the carrying amount of the asset, the asset is written-down to its estimated fair value. |
Revenue Recognition & Cost of Product Revenue | Revenue Recognition Arrangements Within the Scope of ASC 606, Revenue from Contracts with Customers The Company recognizes revenue in accordance with ASC 606, which applies to all contracts with customers, except for contracts that are within the scope of other standards, such as collaboration arrangements and leases. Pursuant to ASC 606, the Company recognizes revenue when its customers obtain control of promised goods or services, in an amount that reflects the consideration which the Company determines it expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that the Company determines are within the scope of ASC 606, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligation(s) in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligation(s) in the contract; and (v) recognize revenue when (or as) the Company satisfies its performance obligation(s). As part of the accounting for these arrangements, the Company may be required to make significant judgments, including identifying performance obligations in the contract, estimating the amount of variable consideration to include in the transaction price and allocating the transaction price to each performance obligation. Once a contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within the contract and determines those that are performance obligations. The Company assesses whether each promised good or service is distinct for the purpose of identifying the performance obligations in the contract. This assessment involves subjective determinations and may require management to make judgments about the individual promised goods or services and whether such are separable from the other aspects of the contractual relationship. Promised goods and services are considered distinct provided that: (i) the customer can benefit from the good or service either on its own or together with other resources that are readily available to the customer (that is, the good or service is capable of being distinct) and (ii) the entity’s promise to transfer the good or service to the customer is separately identifiable from other promises in the contract (that is, the promise to transfer the good or service is distinct within the context of the contract). In assessing whether a promised good or service is distinct, the Company considers factors such as the research, manufacturing and commercialization capabilities of the customer and the availability of the associated expertise in the general marketplace. The Company also considers the intended benefit of the contract in assessing whether a promised good or service is separately identifiable from other promises in the contract. If a promised good or service is not distinct, an entity is required to combine that good or service with other promised goods or services until it identifies a bundle of goods or services that is distinct. If the consideration promised in a contract includes a variable amount, the Company estimates the amount of consideration to which it will be entitled in exchange for transferring the promised goods or services to a customer. The Company determines the amount of variable consideration by using the expected value method or the most likely amount method. The Company includes the unconstrained amount of estimated variable consideration in the transaction price. The amount included in the transaction price is constrained to the amount for which it is probable that a significant reversal of cumulative revenue recognized will not occur. At the end of each subsequent reporting period, the Company re-evaluates the estimated variable consideration included in the transaction price and any related constraint, and if necessary, adjusts its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis in the period of adjustment. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) each performance obligation is satisfied at a point in time or over time, and if over time based on the use of an output or input method that most faithfully depicts the transfer of goods and services to the customer. Product revenue, net: Revenues from product sales are recorded at the net sales price, or “transaction price,” which includes estimates of variable consideration that result from (i) invoice discounts for prompt payment and specialty distributor and specialty pharmacy service fees, (ii) government and other rebates, chargebacks, discounts and fees, (iii) group purchasing organization (“GPO”) discounts, performance rebates and administrative fees, (iv) product returns and (v) costs of co-pay assistance programs for patients. Reserves are established for the estimates of variable consideration based on the amounts the Company expects to be earned or to be claimed on the related sales. The reserves are classified as reductions to accounts receivable, net or accrued expenses and other current liabilities. Where appropriate, the Company utilizes the expected value method to determine the appropriate amount for estimates of variable consideration based on factors such as current contractual and statutory requirements, specific known market events and trends, industry data and forecasted customer buying, payment patterns and the Company’s historical experience. The amount of variable consideration that is included in the transaction price may be constrained and is included in net product revenues only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results vary from the Company's estimates, these estimates are adjusted, which would affect net product revenue and earnings in the period such variances become known. Licenses of intellectual property: The terms of the Company’s license agreements include the license of functional intellectual property, given the functionality of the intellectual property is not expected to change substantially as a result of the Company’s ongoing activities. For licenses that are bundled with other promises (that is, for licenses that are not distinct from other promised goods and services in an arrangement), the Company utilizes 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, the appropriate method of measuring progress for purposes of recognizing revenue. Up-front Fees: If a license agreement is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenues from the transaction price 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 utilizes judgment to assess the nature of the combined performance obligation to determine whether the license is deemed to be the predominant item and if the combined performance obligation is satisfied over time or at a point in time. Milestone Payments: At the inception of each arrangement that includes milestone payments (variable consideration), 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 it is probable that a significant revenue reversal would not occur, the associated milestone value is included in the transaction price. Milestone payments such as developmental and regulatory approval milestones, are generally not considered probable of being achieved until the related activity has been achieved, due to the uncertain nature of the success of clinical trials and obtaining regulatory approvals, which make it unlikely that a significant revenue reversal could be deemed not probable, until such time that the related event has occurred. Royalties: For arrangements that include sales-based royalties, including commercial milestone payments based on the level of sales, and 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 the royalty has been allocated has been satisfied (or partially satisfied). Reimbursement, cost-sharing and profit-sharing payments: Under certain arrangements, the Company has been reimbursed for a portion of its research and development expenses or participates in the cost-sharing of such research and development expenses. Such reimbursements and cost-sharing arrangements have been reflected in research and development expense in the Company’s consolidated statements of operations, as the Company does not consider performing research and development services for reimbursement to be a part of its ongoing major or central operations. Cost of Product Revenue Our cost of product revenue includes the cost of goods sold, amortization expense for commercial milestones and royalty expense. Our cost of goods sold consists of raw materials, third-party manufacturing costs to manufacture the raw materials into finished product, freight and other costs associated with sales of commercial products. |
Research and Development | Research and Development Expenditures for clinical development, including upfront licensing fees and milestone payments associated with products that have not yet been approved by the U.S. Food and Drug Administration, are charged to research and development expense as incurred. These expenses consist of expenses incurred in performing development activities, including salaries and benefits, equity-based compensation expense, preclinical expenses, clinical trial and related clinical manufacturing expenses, contract services and other outside expenses. Expenses incurred for certain research and development activities, including expenses associated with particular activities performed by contract research organizations, investigative sites in connection with clinical trials and contract manufacturing organizations, are recognized based on an evaluation of the progress or completion of specific tasks using either time-based measures or data such as information provided to the Company by its vendors on actual activities completed or costs incurred. Payments for these activities are based on the terms of the individual arrangements, which may differ from the pattern of expense recognition. Expenses for research and development activities incurred that have yet to be invoiced by the vendors that perform the related activities are reflected in the consolidated financial statements as accrued expenses. Advance payments for goods or services to be received in the future for research and development activities are deferred and recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed. |
Selling, General and Administrative | Selling, General and Administrative Selling, general and administrative expenses consist primarily of salaries and related costs, including equity-based compensation for personnel in executive, finance, corporate, commercial, business development and administrative functions. Selling, general and administrative expenses also include consulting services, legal fees relating to patent and corporate matters; professional fees for accounting, auditing and tax services; insurance costs; administrative travel expenses; and facility-related expenses, which include direct depreciation costs and expenses for rent and maintenance of facilities and other operating expenses. |
Equity-based compensation expense | Equity-based compensation expense Equity-based compensation expense is recognized using the straight-line method, based on the grant date fair value, over the requisite service period of the award, which is generally the vesting term. The Company recognizes forfeitures at the time of the actual forfeiture event. For awards subject to performance conditions, as well as awards containing both market and performance conditions, the Company recognizes equity award compensation expense using an accelerated recognition method over the remaining service period when management determines that achievement of the milestone is probable. Management evaluates when the achievement of a performance-based milestone is probable based on the expected satisfaction of the performance conditions as of the reporting date. The grant-date fair value of performance-based awards with market conditions is estimated using a Monte Carlo simulation method that incorporates the probability of the performance conditions being met as of the grant date. For stock options issued, the Company estimates the grant date fair value and the resulting equity-based compensation expense using the Black-Scholes option-pricing model. The Black-Scholes option-pricing model requires the use of certain subjective assumptions which determine the fair value of equity-based awards. Inputs used in the Black-Scholes option-pricing model are: • Fair value of common stock, which is the trading price of the Company’s common stock on the grant date of the award. • Expected term — The expected term represents the period that the equity-based awards are expected to be outstanding. The Company uses the simplified method to calculate the expected term due to the limited Company-specific historical information available for the Company. • Expected volatility — The Company lacks sufficient Company-specific historical and implied volatility information. Therefore, the Company includes the historical volatility of a publicly traded set of peer companies to determine its expected stock volatility and expects to continue to do so until it has adequate historical data regarding the volatility of its own traded stock. • Risk-free interest rate — The risk-free interest rate is based on the U.S. Treasury yield in effect at the time of grant for zero-coupon U.S. Treasury notes with maturities approximately equal to the expected term of the awards. • Expected dividend — The Company has never paid dividends on its common units or stock and has no plans to pay dividends on its common stock. Therefore, the expected dividend yield is zero. |
Net Loss per Share | Net Loss per Share Basic net loss per share is computed by dividing net loss by the weighted average number of shares outstanding for the period. Diluted net loss per share excludes the potential impact of unvested restricted stock and stock options because their effect would be anti-dilutive due to the Company’s net loss. Since the Company had a net loss in each of the periods presented, basic and diluted net loss per share are the same. |
Income Taxes | Income Taxes Income taxes are accounted for using the asset-and-liability method. Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax basis and operating loss and tax credit carryforwards. Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized as income in the period that includes the enactment date. Changes in deferred tax assets and liabilities are recorded in the provision for income taxes. The Company recognizes deferred tax assets to the extent that it believes that these assets are more likely than not to be realized. In making such a determination, management considers all available positive and negative evidence, including future reversals of existing taxable temporary differences, projected future taxable income, tax-planning strategies and results of recent operations. Valuation allowances are provided, if based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. If management determines that the Company would be able to realize its deferred tax assets in the future in excess of their net recorded amount, management would make an adjustment to the deferred tax asset valuation allowance, which would reduce the provision for income taxes. The Company records uncertain tax positions in accordance with ASC 740 on the basis of a two-step process in which (1) management determines whether it is more likely than not that the tax positions will be sustained on the basis of the technical merits of the position and (2) for those tax positions that meet the more-likely-than-not recognition threshold, management recognizes the largest amount of tax benefit that is more than 50% likely to be realized upon ultimate settlement with the related tax authority. The Company provides reserves for potential payments of tax to various tax authorities related to uncertain tax positions. These reserves are based on a determination of whether and how much of a tax benefit taken by the Company in its filings or positions is more likely than not to be realized following resolution of any potential contingencies related to the tax benefit. Potential interest related to the underpayment of income taxes will be classified as a component of income tax expense and any related penalties will be classified as income tax expense. |
Recently Adopted and Recently Issued Accounting Pronouncements | Recently Adopted and Recently Issued Accounting Pronouncements There were no recently adopted accounting pronouncements that had a material impact on the Company's financial statements. In December 2023, the FASB issued ASU 2023-09, Improvements to Income Tax Disclosures, to provide more detailed income tax disclosure requirements. The guidance requires entities to disclose disaggregated information about their effective tax rate reconciliation as well as information on income taxes paid. The disclosure requirements should be applied on a prospective basis, with the option to apply it retrospectively. The effective date for the standard is for fiscal years beginning after December 15, 2024. Early adoption is permitted. The Company does not expect ASU 2023-09 to have a material impact on the Company's financial statements. |
Marketable Securities (Tables)
Marketable Securities (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Investments, Debt and Equity Securities [Abstract] | |
Schedule of Available-for-sale Marketable Securities | The following table summarizes the Company’s available-for-sale marketable securities as of December 31, 2023 and December 31, 2022: As of December 31, 2023 (in thousands) Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value Marketable securities: Short-term investments: U.S. government securities $ 228,579 $ 212 $ — $ 228,791 Corporate debt securities 9,629 2 — 9,631 Commercial paper 64,724 3 — 64,727 Long-term investments: U.S. government securities 141,102 755 141,857 Corporate debt securities 41,310 219 — 41,529 Total $ 485,344 $ 1,191 $ — $ 486,535 As of December 31, 2022 (in thousands) Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value Marketable securities: Short-term investments: U.S. government securities $ 232,229 $ — $ (690) $ 231,539 Non-U.S. government securities 9,388 — (31) 9,357 Corporate debt securities 45,710 — (44) 45,666 Commercial paper 238,160 — — 238,160 Long-term investments: Corporate debt securities 4,796 (2) 4,794 Total $ 530,283 $ — $ (767) $ 529,516 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Fair Value Disclosures [Abstract] | |
Schedule of Financial Assets Subject to Fair Value Measurements | The following tables sets forth the fair value hierarchy of the Company’s financial assets and liabilities measured on a recurring basis as of December 31, 2023 and December 31, 2022: As of December 31, 2023 Fair Value Hierarchy (in thousands) Total Level 1 Level 2 Level 3 Financial instruments carried at fair value (asset position): Cash equivalents: Money market funds $ 14,434 $ 14,434 $ — $ — Commercial paper 49,631 — 49,631 — Short-term investments: U.S. government securities 228,791 228,791 — — Corporate debt securities 9,631 — 9,631 — Commercial paper 64,727 — 64,727 — Long-term investments: U.S. government securities 141,857 141,857 — — Corporate debt securities 41,529 — 41,529 — Total $ 550,600 $ 385,082 $ 165,518 $ — As of December 31, 2022 Fair Value Hierarchy (in thousands) Total Level 1 Level 2 Level 3 Financial instruments carried at fair value (asset position): Cash equivalents: Money market funds $ 22,494 $ 22,494 $ — $ — Short-term investments: U.S. government securities 231,539 231,539 — — Non-U.S. government securities 9,357 — 9,357 — Corporate debt securities 45,666 — 45,666 — Commercial paper 238,160 — 238,160 — Long-term investments: Corporate debt securities 4,794 — 4,794 — Total $ 552,010 $ 254,033 $ 297,977 $ — |
Property and Equipment, net (Ta
Property and Equipment, net (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Property, Plant and Equipment [Abstract] | |
Schedule of Property And Equipment | Property and equipment, net consisted of the following: December 31, (in thousands) 2023 2022 Useful Life Leasehold improvements $ 826 $ 826 Length of lease or 5 years, whichever is shorter Computer equipment 380 368 3-5 years Lab equipment 2,439 — 5 - 15 years Furniture 437 437 5 years Software 12,954 6,048 3-10 years Construction in process 3,565 7,137 Property and equipment, gross 20,601 14,816 Less accumulated depreciation (2,658) (1,245) Property and equipment, net $ 17,943 $ 13,571 |
Intangible Assets, net (Tables)
Intangible Assets, net (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Schedule of Finite-Lived Intangible Assets | Intangible assets, net, which is included in other assets, consisted of the following: December 31, (in thousands) 2023 2022 Definite-lived intangible assets License agreements $ 11,250 $ — Intangible assets, gross 11,250 — Less accumulated amortization (50) — Intangible assets, net $ 11,200 $ — |
Schedule of Finite-Lived Intangible Assets, Future Amortization Expense | The expected future amortization expense for amortizable finite-lived intangible assets as of December 31, 2023 is as follows: (in thousands) 2024 $ 600 2025 600 2026 600 2027 600 2028 600 Thereafter 8,200 Total future expected amortization expense $ 11,200 |
Leases (Tables)
Leases (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Leases [Abstract] | |
Schedule of Operating Leases | The components of lease cost recorded in the Company’s consolidated statement of operations were as follows: Twelve Months Ended December 31, (in thousands) 2023 2022 2021 Operating lease cost Fixed $ 1,653 $ 1,131 $ 993 Variable (1) 866 336 548 Total lease cost $ 2,519 $ 1,467 $ 1,541 (1) Variable lease costs consist primarily of taxes, utilities and common area maintenance costs. The weighted-average remaining lease term and discount rate related to the Company’s leases were as follows: As of December 31, 2023 As of December 31, 2022 Weighted-average remaining lease term (in years) Operating leases 4.2 5.2 Weighted-average discount rate Operating leases 5.8 % 4.2 % Supplemental cash flow information related to the Company’s leases was as follows: (in thousands) December 31, 2023 December 31, 2022 December 31, 2021 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 1,293 $ 859 $ 1,388 Right-of-use assets obtained in exchange for new operating lease liabilities 2,637 5,580 — |
Schedule of Operating Lease Assets And Liabilities | The Company’s leases are included on its consolidated balance sheets as follows: (in thousands) As of December 31, 2023 As of December 31, 2022 Operating leases Operating lease right-of-use-assets $ 6,144 $ 4,698 Total operating lease assets $ 6,144 $ 4,698 Operating lease liabilities, current $ 1,061 $ 483 Operating lease liabilities, long-term 5,996 4,768 Total operating lease liabilities $ 7,057 $ 5,251 |
Schedule of Future Minimum Lease Obligations | Maturities of the Company’s operating lease liabilities as of December 31, 2023 were as follows: (in thousands) Operating Leases 2024 $ 1,476 2025 1,999 2026 2,002 2027 1,904 2028 646 Thereafter — Total lease payments 8,027 Less: imputed interest (970) Present value of lease liabilities $ 7,057 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following: December 31, December 31, (in thousands) 2023 2022 Accrued compensation and benefits $ 26,047 $ 19,142 Accrued research and development 15,129 12,321 Accrued milestone 11,250 — Accrued other 13,143 7,779 Total accrued expenses $ 65,569 $ 39,242 |
Equity- Based Compensation (Tab
Equity- Based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Summary of Equity-based Compensation Expense Related to Restricted Stock and Stock Options | The Company recorded total equity-based compensation expense for the periods presented as follows: Year Ended December 31, (in thousands) 2023 2022 2021 Research and development $ 34,398 $ 29,373 $ 14,664 Selling, general and administrative 60,136 43,592 23,780 Total equity-based compensation expense $ 94,534 $ 72,965 $ 38,444 |
Summary of the Changes in the Company's Options During the Period | A summary of the changes in the Company’s stock options during the periods presented is as follows: Shares Weighted Average Exercise Weighted Intrinsic Aggregate Outstanding at December 31, 2020 4,505,546 $ 15.51 8.7 $ 256,860,405 Granted 2,547,813 73.37 — — Exercised (151,643) 7.62 — — Forfeited/cancelled (188,303) 37.34 — — Outstanding at December 31, 2021 6,713,413 37.03 8.2 196,012,147 Granted 3,207,347 43.28 — — Exercised (223,467) 8.84 — — Forfeited/cancelled (520,478) 68.33 — — Outstanding at December 31, 2022 9,176,815 38.13 8.0 53,719,297 Granted 2,994,704 27.65 — — Exercised (44,250) 3.15 — — Forfeited/cancelled (538,693) 44.01 — — Outstanding at December 31, 2023 11,588,576 35.28 7.4 117,631,827 Exercisable at December 31, 2023 6,711,390 32.83 6.6 88,814,151 |
Summary of Share-based Payment Arrangement, Restricted Stock and Restricted Stock Unit, Activity | A summary of the changes in the Company’s restricted stock awards for the periods presented is as follows: Number Weighted Unvested and outstanding at December 31, 2020 686,868 $ 1.26 Granted 332,226 73.31 Vested (493,309) 1.19 Forfeited (55,475) 13.75 Unvested and outstanding at December 31, 2021 470,310 50.76 Granted 36,625 61.74 Vested (257,219) 32.03 Forfeited (27,957) 79.25 Unvested and outstanding at December 31, 2022 221,759 70.71 Granted — — Vested (93,812) 71.50 Forfeited (21,113) 63.38 Unvested and outstanding at December 31, 2023 106,834 70.48 A summary of the changes in the Company’s restricted stock units for the periods presented is as follows: Number Weighted Unvested and outstanding at December 31, 2021 — $ — Granted 738,508 49.77 Vested (24,369) 24.62 Forfeited (42,185) 58.76 Unvested and outstanding at December 31, 2022 671,954 50.11 Granted 1,163,390 27.68 Vested (238,725) 49.14 Forfeited (110,079) 31.27 Unvested and outstanding at December 31, 2023 1,486,540 34.11 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Income Tax Disclosure [Abstract] | |
Schedule of Principal Components of the Subsidiaries Deferred Tax Assets | The principal components of deferred tax assets and liabilities are as follows: As of December 31, (in thousands) 2023 2022 Deferred tax assets: Net operating loss carryforwards $ 121,029 $ 93,032 Research and development credits 6,069 3,422 Orphan drug credit 27,515 19,401 Capitalized research and development 41,588 21,262 Equity-based compensation 26,429 20,286 Deferred revenue 4,105 — Other 3,031 1,525 Total deferred tax assets 229,766 158,928 Deferred tax liability: Operating lease right-of-use assets (1,290) (987) Depreciation (1,255) (113) Other (2) (2) Valuation allowance (227,219) (157,826) Net deferred tax assets $ — $ — |
Schedule of Reconciliation of U.S. Statutory Income Tax Rate to Effective Tax Rate | A reconciliation of the income tax expense at the federal statutory tax rate to the Company’s effective income tax rate follows: Year Ended December 31, 2023 2022 2021 Statutory tax rate 21.00 % 21.00 % 21.00 % U.S. state and local income taxes, net of U.S. federal income tax benefit 2.17 2.33 5.15 Equity-based compensation (1.21) (0.77) (0.03) Research and development credit 0.81 0.27 0.50 Orphan drug credit 2.50 1.87 4.99 Section 162(m) limitation (3.39) — — Other (0.54) — — Change in valuation allowance (21.34) (24.70) (31.61) Effective tax rate — % — % — % |
Net Loss per Share (Tables)
Net Loss per Share (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Earnings Per Share [Abstract] | |
Schedule of Basic and Diluted Net Loss Per Share | Basic and diluted net loss per unit and share is calculated as follows: Year Ended December 31, (in thousands, except share and per-share data) 2023 2022 2021 Numerator: Net loss $ (325,104) $ (277,417) $ (173,910) Net loss attributable to common stockholders (325,104) (277,417) (173,910) Denominator: Weighted average shares outstanding, basic and diluted 63,123,936 53,290,528 48,497,790 Net loss per share, basic and diluted $ (5.15) $ (5.21) $ (3.59) |
Schedule of Potential Common Shares not Included in the Calculation of the Diluted Net Loss Per Share | Potentially dilutive securities that were not included in the diluted per share calculations because they would be anti-dilutive were as follows: As of December 31, 2023 2022 Common stock options issued and outstanding 11,588,576 9,176,815 Restricted stock units subject to future vesting 1,486,540 671,954 Performance stock units subject to future vesting 284,362 — Restricted stock awards subject to future vesting 106,834 221,759 Total potentially dilutive securities 13,466,312 10,070,528 |
Nature of Operations (Details)
Nature of Operations (Details) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | |||||
Dec. 08, 2023 | Sep. 07, 2022 | Sep. 06, 2022 | Oct. 13, 2020 | Sep. 30, 2022 | Aug. 31, 2022 | |
Follow-on Offering | ||||||
Collaborative Arrangement and Arrangement Other than Collaborative | ||||||
Shares issued (in shares) | 10,905,171 | 5,637,254 | ||||
Sales price (in usd per share) | $ 29 | $ 51 | ||||
Sale of stock, consideration received on transaction, net proceeds | $ 299.3 | $ 269.5 | ||||
Underwriter's Option | ||||||
Collaborative Arrangement and Arrangement Other than Collaborative | ||||||
Shares issued (in shares) | 1,422,413 | 735,294 | ||||
Private Placement | ||||||
Collaborative Arrangement and Arrangement Other than Collaborative | ||||||
Shares issued (in shares) | 8,650,520 | |||||
Sales price (in usd per share) | $ 26.01 | |||||
Sale of stock, consideration received on transaction, net proceeds | $ 216.8 | |||||
Sale of stock, consideration received on transaction, gross proceeds | $ 225 | |||||
Private Placement | GSK | ||||||
Collaborative Arrangement and Arrangement Other than Collaborative | ||||||
Shares issued (in shares) | 2,050,819 | |||||
Sales price (in usd per share) | $ 36.57 | |||||
Sale of stock, consideration received on transaction, gross proceeds | $ 75 | |||||
Premium rate paid on weighted average share price (percent) | 25% | |||||
Premium term (days) | 30 days | 30 days | ||||
At-The-Market Offering | Cowen And Company, LLC | ||||||
Collaborative Arrangement and Arrangement Other than Collaborative | ||||||
Shares issued (in shares) | 2,247,500 | |||||
Sale of stock, consideration received on transaction, net proceeds | $ 67.8 | |||||
Sale of stock, consideration received on transaction, gross proceeds | 69.7 | |||||
Commission fee | $ 1.9 |
Risks and Liquidity (Details)
Risks and Liquidity (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Concentration Risk [Line Items] | |||
Accumulated deficit | $ 895,034 | $ 569,930 | |
Working capital | 422,700 | 548,700 | |
Net revenue | 5,447 | 0 | $ 0 |
Cash, cash equivalents and marketable securities | 662,600 | $ 597,000 | |
OGSIVEO | |||
Concentration Risk [Line Items] | |||
Net revenue | $ 5,400 |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Details) | 12 Months Ended | |
Dec. 31, 2023 USD ($) segment | Dec. 31, 2022 USD ($) | |
Share-based Compensation Arrangement by Share-based Payment Award | ||
Number of operating segments | segment | 1 | |
Cash and cash equivalents | $ 176,053,000 | $ 67,490,000 |
Expected dividend yield | $ 0 |
Marketable Securities (Details)
Marketable Securities (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Marketable securities: | ||
Amortized Cost | $ 485,344 | $ 530,283 |
Gross Unrealized Gains | 1,191 | 0 |
Gross Unrealized Losses | 0 | (767) |
Estimated Fair Value | 486,535 | 529,516 |
U.S. government securities | ||
Marketable securities: | ||
Amortized Cost | 232,229 | |
Gross Unrealized Gains | 0 | |
Gross Unrealized Losses | (690) | |
Estimated Fair Value | 231,539 | |
Non-U.S. government securities | ||
Marketable securities: | ||
Amortized Cost | 9,388 | |
Gross Unrealized Gains | 0 | |
Gross Unrealized Losses | (31) | |
Estimated Fair Value | 9,357 | |
Commercial paper | ||
Marketable securities: | ||
Amortized Cost | 64,724 | 238,160 |
Gross Unrealized Gains | 3 | 0 |
Gross Unrealized Losses | 0 | 0 |
Estimated Fair Value | 64,727 | 238,160 |
Short-term investments | U.S. government securities | ||
Marketable securities: | ||
Amortized Cost | 228,579 | |
Gross Unrealized Gains | 212 | |
Gross Unrealized Losses | 0 | |
Estimated Fair Value | 228,791 | |
Short-term investments | Corporate debt securities | ||
Marketable securities: | ||
Amortized Cost | 9,629 | 45,710 |
Gross Unrealized Gains | 2 | 0 |
Gross Unrealized Losses | 0 | (44) |
Estimated Fair Value | 9,631 | 45,666 |
Long-term investments | U.S. government securities | ||
Marketable securities: | ||
Amortized Cost | 141,102 | 4,796 |
Gross Unrealized Gains | 755 | |
Gross Unrealized Losses | (2) | |
Estimated Fair Value | 141,857 | $ 4,794 |
Long-term investments | Corporate debt securities | ||
Marketable securities: | ||
Amortized Cost | 41,310 | |
Gross Unrealized Gains | 219 | |
Gross Unrealized Losses | 0 | |
Estimated Fair Value | $ 41,529 |
Fair Value Measurements (Detail
Fair Value Measurements (Details) - Recurring - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Fair Value Measurements | ||
Total financial assets | $ 550,600 | $ 552,010 |
U.S. government securities | ||
Fair Value Measurements | ||
Short-term investments | 228,791 | 231,539 |
Long-term investment | 141,857 | |
Non-U.S. government securities | ||
Fair Value Measurements | ||
Short-term investments | 9,357 | |
Corporate debt securities | ||
Fair Value Measurements | ||
Short-term investments | 9,631 | 45,666 |
Long-term investment | 41,529 | 4,794 |
Commercial paper | ||
Fair Value Measurements | ||
Short-term investments | 64,727 | 238,160 |
Money market funds | ||
Fair Value Measurements | ||
Cash equivalents | 14,434 | 22,494 |
Commercial paper | ||
Fair Value Measurements | ||
Cash equivalents | 49,631 | |
Level 1 | ||
Fair Value Measurements | ||
Total financial assets | 385,082 | 254,033 |
Level 1 | U.S. government securities | ||
Fair Value Measurements | ||
Short-term investments | 228,791 | 231,539 |
Long-term investment | 141,857 | |
Level 1 | Non-U.S. government securities | ||
Fair Value Measurements | ||
Short-term investments | 0 | |
Level 1 | Corporate debt securities | ||
Fair Value Measurements | ||
Short-term investments | 0 | 0 |
Long-term investment | 0 | 0 |
Level 1 | Commercial paper | ||
Fair Value Measurements | ||
Short-term investments | 0 | 0 |
Level 1 | Money market funds | ||
Fair Value Measurements | ||
Cash equivalents | 14,434 | 22,494 |
Level 1 | Commercial paper | ||
Fair Value Measurements | ||
Cash equivalents | 0 | |
Level 2 | ||
Fair Value Measurements | ||
Total financial assets | 165,518 | 297,977 |
Level 2 | U.S. government securities | ||
Fair Value Measurements | ||
Short-term investments | 0 | 0 |
Long-term investment | 0 | |
Level 2 | Non-U.S. government securities | ||
Fair Value Measurements | ||
Short-term investments | 9,357 | |
Level 2 | Corporate debt securities | ||
Fair Value Measurements | ||
Short-term investments | 9,631 | 45,666 |
Long-term investment | 41,529 | 4,794 |
Level 2 | Commercial paper | ||
Fair Value Measurements | ||
Short-term investments | 64,727 | 238,160 |
Level 2 | Money market funds | ||
Fair Value Measurements | ||
Cash equivalents | 0 | 0 |
Level 2 | Commercial paper | ||
Fair Value Measurements | ||
Cash equivalents | 49,631 | |
Level 3 | ||
Fair Value Measurements | ||
Total financial assets | 0 | 0 |
Level 3 | U.S. government securities | ||
Fair Value Measurements | ||
Short-term investments | 0 | 0 |
Long-term investment | 0 | |
Level 3 | Non-U.S. government securities | ||
Fair Value Measurements | ||
Short-term investments | 0 | |
Level 3 | Corporate debt securities | ||
Fair Value Measurements | ||
Short-term investments | 0 | 0 |
Long-term investment | 0 | 0 |
Level 3 | Commercial paper | ||
Fair Value Measurements | ||
Short-term investments | 0 | 0 |
Level 3 | Money market funds | ||
Fair Value Measurements | ||
Cash equivalents | 0 | $ 0 |
Level 3 | Commercial paper | ||
Fair Value Measurements | ||
Cash equivalents | $ 0 |
Property and Equipment, net - S
Property and Equipment, net - Schedule of Property and Equipment (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Property and Equipment | ||
Property and equipment, gross | $ 20,601 | $ 14,816 |
Less accumulated depreciation | (2,658) | (1,245) |
Property and equipment, net | 17,943 | 13,571 |
Leasehold improvements | ||
Property and Equipment | ||
Property and equipment, gross | $ 826 | 826 |
Leasehold improvements | Maximum | ||
Property and Equipment | ||
Property, plant and equipment, useful life (in years) | 5 years | |
Computer equipment | ||
Property and Equipment | ||
Property and equipment, gross | $ 380 | 368 |
Computer equipment | Minimum | ||
Property and Equipment | ||
Property, plant and equipment, useful life (in years) | 3 years | |
Computer equipment | Maximum | ||
Property and Equipment | ||
Property, plant and equipment, useful life (in years) | 5 years | |
Lab equipment | ||
Property and Equipment | ||
Property and equipment, gross | $ 2,439 | 0 |
Lab equipment | Minimum | ||
Property and Equipment | ||
Property, plant and equipment, useful life (in years) | 5 years | |
Lab equipment | Maximum | ||
Property and Equipment | ||
Property, plant and equipment, useful life (in years) | 15 years | |
Furniture | ||
Property and Equipment | ||
Property and equipment, gross | $ 437 | 437 |
Property, plant and equipment, useful life (in years) | 5 years | |
Software | ||
Property and Equipment | ||
Property and equipment, gross | $ 12,954 | 6,048 |
Software | Minimum | ||
Property and Equipment | ||
Property, plant and equipment, useful life (in years) | 3 years | |
Software | Maximum | ||
Property and Equipment | ||
Property, plant and equipment, useful life (in years) | 10 years | |
Construction in process | ||
Property and Equipment | ||
Property and equipment, gross | $ 3,565 | $ 7,137 |
Property and Equipment, net - N
Property and Equipment, net - Narrative (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Property, Plant and Equipment [Abstract] | |||
Depreciation | $ 1.6 | $ 0.8 | $ 0.5 |
Intangible Assets, net - Schedu
Intangible Assets, net - Schedule of Finite-Lived Intangible Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Definite-lived intangible assets | ||
Intangible assets, gross | $ 11,250 | $ 0 |
Less accumulated amortization | (50) | 0 |
Intangible assets, net | 11,200 | 0 |
License agreements | ||
Definite-lived intangible assets | ||
Intangible assets, gross | $ 11,250 | $ 0 |
Intangible Assets, net - Narrat
Intangible Assets, net - Narrative (Details) $ in Millions | 12 Months Ended |
Dec. 31, 2023 USD ($) | |
Finite-Lived Intangible Assets | |
Amortization expense | $ 0.1 |
Patents | |
Finite-Lived Intangible Assets | |
Intangible asset, useful life | 19 years 4 months 24 days |
Intangible Assets, net - Future
Intangible Assets, net - Future Amortization (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Finite-Lived Intangible Assets, Net, Amortization Expense, Fiscal Year Maturity [Abstract] | ||
2024 | $ 600 | |
2025 | 600 | |
2026 | 600 | |
2027 | 600 | |
2028 | 600 | |
Thereafter | 8,200 | |
Intangible assets, net | $ 11,200 | $ 0 |
Leases - Narrative (Details)
Leases - Narrative (Details) - option | 1 Months Ended | |||
Mar. 31, 2023 | May 31, 2023 | Jan. 31, 2022 | Aug. 31, 2018 | |
Operating Lease in Durham, NC | ||||
Leases | ||||
Lease term (in years) | 5 years | |||
Number of renewal options | 2 | |||
Renewal term (in years) | 5 years | |||
Annual increase in lease payments (as a percent) | 3% | |||
Corporate Headquarters Stamford Connecticut | ||||
Leases | ||||
Annual increase in lease payments (as a percent) | 2.50% | |||
Corporate Headquarters Stamford Connecticut | Option One | ||||
Leases | ||||
Number of renewal options | 2 | |||
Renewal term (in years) | 5 years | |||
Corporate Headquarters Stamford Connecticut | Option Two | ||||
Leases | ||||
Number of renewal options | 1 | |||
Renewal term (in years) | 10 years | |||
Research Triangle Park | ||||
Leases | ||||
Lease term (in years) | 5 years | |||
Number of renewal options | 2 | |||
Renewal term (in years) | 5 years | |||
Annual increase in lease payments (as a percent) | 3% | |||
Research Triangle Park | Minimum | ||||
Leases | ||||
Number of years the lease payment will increase | 4 years | |||
Research Triangle Park | Maximum | ||||
Leases | ||||
Number of years the lease payment will increase | 5 years |
Leases - Statement of Operation
Leases - Statement of Operations (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Operating lease cost | |||
Fixed | $ 1,653 | $ 1,131 | $ 993 |
Variable | 866 | 336 | 548 |
Total lease cost | $ 2,519 | $ 1,467 | $ 1,541 |
Leases - Balance Sheet (Details
Leases - Balance Sheet (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Leases [Abstract] | ||
Operating lease right-of-use assets | $ 6,144 | $ 4,698 |
Operating lease liabilities, current | 1,061 | 483 |
Operating lease liabilities, long-term | 5,996 | 4,768 |
Total operating lease liabilities | $ 7,057 | $ 5,251 |
Leases - Future Lease Obligatio
Leases - Future Lease Obligations (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Operating Leases | ||
2024 | $ 1,476 | |
2025 | 1,999 | |
2026 | 2,002 | |
2027 | 1,904 | |
2028 | 646 | |
Thereafter | 0 | |
Total lease payments | 8,027 | |
Less: imputed interest | (970) | |
Present value of lease liabilities | $ 7,057 | $ 5,251 |
Leases - Supplemental Informati
Leases - Supplemental Information (Details) | Dec. 31, 2023 | Dec. 31, 2022 |
Weighted-average remaining lease term (in years) | ||
Operating leases -Weighted-average remaining lease term (in years) | 4 years 2 months 12 days | 5 years 2 months 12 days |
Weighted-average discount rate | ||
Operating leases - Weighted-average discount rate | 5.80% | 4.20% |
Leases - Cash Flow Statement (D
Leases - Cash Flow Statement (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Cash paid for amounts included in the measurement of lease liabilities: | |||
Operating cash flows from operating leases | $ 1,293 | $ 859 | $ 1,388 |
Right-of-use assets obtained in exchange for new operating lease liabilities | $ 2,637 | $ 5,580 | $ 0 |
Accrued Expenses (Details)
Accrued Expenses (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Payables and Accruals [Abstract] | ||
Accrued compensation and benefits | $ 26,047 | $ 19,142 |
Accrued research and development | 15,129 | 12,321 |
Accrued milestone | 11,250 | 0 |
Accrued other | 13,143 | 7,779 |
Total accrued expenses | $ 65,569 | $ 39,242 |
Equity- Based Compensation - Eq
Equity- Based Compensation - Equity-based compensation expense related to restricted stock and stock options (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Share-based Payment Arrangement, Expensed and Capitalized, Amount | |||
Total equity-based compensation expense | $ 94,534 | $ 72,965 | $ 38,444 |
Research and development | |||
Share-based Payment Arrangement, Expensed and Capitalized, Amount | |||
Total equity-based compensation expense | 34,398 | 29,373 | 14,664 |
Selling, general and administrative | |||
Share-based Payment Arrangement, Expensed and Capitalized, Amount | |||
Total equity-based compensation expense | $ 60,136 | $ 43,592 | $ 23,780 |
Equity- Based Compensation - 20
Equity- Based Compensation - 2019 Equity Incentive Plan (Details) - 2019 Equity Incentive Plan - shares | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2019 | |
Share-based Compensation Arrangement by Share-based Payment Award | ||
Percentage of annual increase in the shares | 5% | |
Number of shares authorized for issuance (in shares) | 2,543,163 | |
Employee | Minimum | ||
Share-based Compensation Arrangement by Share-based Payment Award | ||
Vesting period (in years) | 3 years | |
Employee | Maximum | ||
Share-based Compensation Arrangement by Share-based Payment Award | ||
Vesting period (in years) | 4 years | |
Nonemployee | Minimum | ||
Share-based Compensation Arrangement by Share-based Payment Award | ||
Vesting period (in years) | 1 year | |
Nonemployee | Maximum | ||
Share-based Compensation Arrangement by Share-based Payment Award | ||
Vesting period (in years) | 3 years |
Equity- Based Compensation - _2
Equity- Based Compensation - 2019 Employee Stock Purchase Plan (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | Aug. 30, 2019 | |
Share-based Compensation Arrangement by Share-based Payment Award | ||||
Total equity-based compensation expense | $ 94,534 | $ 72,965 | $ 38,444 | |
Stock Options | ||||
Share-based Compensation Arrangement by Share-based Payment Award | ||||
Minimum risk-free interest rate | 3.46% | |||
Maximum risk-free interest rate | 4.73% | |||
Expected dividend yield | 0% | |||
Stock Options | Minimum | ||||
Share-based Compensation Arrangement by Share-based Payment Award | ||||
Expected term | 5 years 6 months | |||
Expected volatility | 76.70% | |||
Stock Options | Maximum | ||||
Share-based Compensation Arrangement by Share-based Payment Award | ||||
Expected term | 6 years 1 month 6 days | |||
Expected volatility | 78.80% | |||
2019 Employee Stock Purchase Plan | ||||
Share-based Compensation Arrangement by Share-based Payment Award | ||||
Shares available for future issuance (in shares) | 442,153 | |||
Automatic increase in to shares authorized by the number of shares (in shares) | 663,229 | |||
Automatic increase in to shares authorized as percentage of outstanding stock | 1% | |||
Number of shares authorized for issuance (in shares) | 2,477,122 | |||
CEO Performance Award | Stock Options | ||||
Share-based Compensation Arrangement by Share-based Payment Award | ||||
Unrecognized compensation expense, options | $ 117,700 | |||
Share compensation recognition period | 2 years 4 months 24 days | |||
Total equity-based compensation expense | $ 66,000 |
Equity- Based Compensation - St
Equity- Based Compensation - Stock options (Details) - USD ($) | 12 Months Ended | |||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Shares | ||||
Outstanding at the beginning (in shares) | 9,176,815 | 6,713,413 | 4,505,546 | |
Granted (in shares) | 2,994,704 | 3,207,347 | 2,547,813 | |
Exercised (in shares) | (44,250) | (223,467) | (151,643) | |
Forfeited/cancelled (in shares) | (538,693) | (520,478) | (188,303) | |
Outstanding at the end (in shares) | 11,588,576 | 9,176,815 | 6,713,413 | 4,505,546 |
Exercisable (in shares) | 6,711,390 | |||
Weighted Average Exercise Price | ||||
Outstanding at the beginning (usd per share) | $ 38.13 | $ 37.03 | $ 15.51 | |
Granted (usd per share) | 27.65 | 43.28 | 73.37 | |
Exercised (usd per share) | 3.15 | 8.84 | 7.62 | |
Forfeited/cancelled (usd per share) | 44.01 | 68.33 | 37.34 | |
Outstanding at the end (usd per share) | 35.28 | $ 38.13 | $ 37.03 | $ 15.51 |
Exercisable (usd per share) | $ 32.83 | |||
Weighted Average Remaining Contractual Life and Aggregate Intrinsic Value | ||||
Outstanding at the end (in years) | 7 years 4 months 24 days | 8 years | 8 years 2 months 12 days | 8 years 8 months 12 days |
Exercisable (in years) | 6 years 7 months 6 days | |||
Outstanding at the end | $ 117,631,827 | $ 53,719,297 | $ 196,012,147 | $ 256,860,405 |
Exercisable | $ 88,814,151 |
Equity- Based Compensation - Re
Equity- Based Compensation - Restricted stock (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Weighted Average Grant Date Fair Value | |||
Stock compensation expense | $ 94,534 | $ 72,965 | $ 38,444 |
Restricted stock | |||
Number of shares/units | |||
Unvested and outstanding at the beginning (in shares) | 221,759 | 470,310 | 686,868 |
Granted (in shares) | 0 | 36,625 | 332,226 |
Vested (in shares) | (93,812) | (257,219) | (493,309) |
Forfeited (in shares) | (21,113) | (27,957) | (55,475) |
Unvested and outstanding at the end (in shares) | 106,834 | 221,759 | 470,310 |
Weighted Average Grant Date Fair Value | |||
Outstanding at the beginning (in dollars per share) | $ 70.71 | $ 50.76 | $ 1.26 |
Granted (in dollars per share) | 0 | 61.74 | 73.31 |
Vested (in dollars per share) | 71.50 | 32.03 | 1.19 |
Forfeited (in dollars per share) | 63.38 | 79.25 | 13.75 |
Unvested and outstanding at the end (in dollars per share) | $ 70.48 | $ 70.71 | $ 50.76 |
Unrecognized compensation expense, options | $ 2,600 | ||
Share compensation recognition period | 1 year | ||
Stock compensation expense | $ 6,600 | ||
Restricted stock units | |||
Number of shares/units | |||
Unvested and outstanding at the beginning (in shares) | 671,954 | 0 | |
Granted (in shares) | 1,163,390 | 738,508 | |
Vested (in shares) | (238,725) | (24,369) | |
Forfeited (in shares) | (110,079) | (42,185) | |
Unvested and outstanding at the end (in shares) | 1,486,540 | 671,954 | 0 |
Weighted Average Grant Date Fair Value | |||
Outstanding at the beginning (in dollars per share) | $ 50.11 | $ 0 | |
Granted (in dollars per share) | 27.68 | 49.77 | |
Vested (in dollars per share) | 49.14 | 24.62 | |
Forfeited (in dollars per share) | 31.27 | 58.76 | |
Unvested and outstanding at the end (in dollars per share) | $ 34.11 | $ 50.11 | $ 0 |
Unrecognized compensation expense, options | $ 32,900 | ||
Share compensation recognition period | 1 year 10 months 24 days | ||
Stock compensation expense | $ 19,400 |
Equity- Based Compensation - Pe
Equity- Based Compensation - Performance Stock Units (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Jan. 05, 2023 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award | ||||
Granted (in shares) | 2,994,704 | 3,207,347 | 2,547,813 | |
Total equity-based compensation expense | $ 94,534 | $ 72,965 | $ 38,444 | |
Performance Stock Units | ||||
Share-based Compensation Arrangement by Share-based Payment Award | ||||
Percentage of shares subject to modification (percent) | 25% | |||
Unrecognized compensation expense, options | $ 4,300 | |||
Share compensation recognition period | 2 years | |||
Total equity-based compensation expense | $ 2,400 | |||
CEO | Performance Stock Units | ||||
Share-based Compensation Arrangement by Share-based Payment Award | ||||
Granted (in shares) | 284,362 |
License and Collaboration Agr_2
License and Collaboration Agreements (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | |||||||
Sep. 07, 2022 | Sep. 06, 2022 | Sep. 30, 2022 | Oct. 31, 2021 | May 31, 2021 | Oct. 31, 2020 | Oct. 31, 2017 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Research and development expenses | $ 150,487 | $ 146,122 | $ 101,676 | |||||||
Common stock, value issued | 7 | 6 | ||||||||
Private Placement | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Sale of stock (in shares) | 8,650,520 | |||||||||
Sale of stock, consideration received on transaction, gross proceeds | $ 225,000 | |||||||||
Sales price (in usd per share) | $ 26.01 | |||||||||
Asset Purchase and Exclusive License Agreement | Katholieke Universiteit Leuven | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Upfront payment | $ 11,000 | |||||||||
Asset Purchase and Exclusive License Agreement | Dana-Farber Cancer Institute | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Upfront payment | $ 300 | |||||||||
Development Milestones | Katholieke Universiteit Leuven | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Potential future payments | 120,000 | |||||||||
Development Milestones | Dana-Farber Cancer Institute | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Potential future payments | 2,300 | |||||||||
Commercial Milestones | Katholieke Universiteit Leuven | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Potential future payments | $ 165,000 | |||||||||
Commercial Milestones | Dana-Farber Cancer Institute | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Potential future payments | $ 39,000 | |||||||||
Pfizer | License agreement | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Minimum period after first commercial sale on country-by-country basis | 10 years | |||||||||
Pfizer | License agreement | Nirogacestat | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Milestone payments outstanding | $ 232,500 | |||||||||
Pfizer | License agreement | Mirdametinib | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Milestone payments outstanding | $ 229,800 | |||||||||
Pfizer | License agreement | OGSIVEO | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Milestone payments outstanding | 11,300 | |||||||||
BeiGene | Clinical collaboration agreement | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Research and development expenses | 1,900 | $ 1,100 | $ 1,100 | |||||||
GSK | Private Placement | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Sale of stock (in shares) | 2,050,819 | |||||||||
Sale of stock, consideration received on transaction, gross proceeds | $ 75,000 | |||||||||
Sales price (in usd per share) | $ 36.57 | |||||||||
Premium rate paid on weighted average share price (percent) | 25% | |||||||||
Premium term (days) | 30 days | 30 days | ||||||||
Common stock, value issued | $ 55,500 | |||||||||
Other liabilities | $ 19,500 | |||||||||
Collaborative arrangement, maximum proceeds from achievement of commercial milestone | $ 550,000 | |||||||||
Jazz Pharmaceuticals Ireland Limited | Asset Purchase and Exclusive License Agreement | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Upfront payment | $ 35,000 | |||||||||
Jazz Pharmaceuticals Ireland Limited | Asset Purchase and Exclusive License Agreement | Maximum | ||||||||||
Research and Development Arrangement, Contract to Perform for Others | ||||||||||
Potential future payments | $ 375,000 |
Equity Method Investment (Detai
Equity Method Investment (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | ||||||
Jan. 31, 2024 | Jan. 31, 2023 | Jun. 30, 2022 | Jun. 30, 2020 | Jun. 30, 2019 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Schedule of Equity Method Investments | ||||||||
Investment | $ 1,955 | $ 4,193 | ||||||
Payment to equity investment | 2,800 | 4,200 | $ 0 | |||||
Equity method investment loss | 5,038 | $ 2,890 | $ 988 | |||||
MapKure | ||||||||
Schedule of Equity Method Investments | ||||||||
Investment | 2,000 | |||||||
Equity method investment loss | $ 5,000 | |||||||
MapKure | Series A Preferred Stock | ||||||||
Schedule of Equity Method Investments | ||||||||
Shares purchased (in shares) | 3,500,000 | 3,500,000 | ||||||
Ownership interest (in percent) | 38.90% | 25% | ||||||
Investment | $ 3,500 | $ 3,500 | ||||||
MapKure | Series B Preferred Stock | ||||||||
Schedule of Equity Method Investments | ||||||||
Shares purchased (in shares) | 2,800,000 | 4,200,000 | ||||||
Ownership interest (in percent) | 38.90% | |||||||
Payment to equity investment | $ 2,800 | $ 4,200 | ||||||
MapKure | Series C Preferred Stock | Subsequent Event | ||||||||
Schedule of Equity Method Investments | ||||||||
Shares purchased (in shares) | 8,235,200 | |||||||
Payment to equity investment | $ 8,200 | |||||||
MapKure | Series C Preferred Stock | Subsequent Event | Scenario, Plan | ||||||||
Schedule of Equity Method Investments | ||||||||
Shares purchased (in shares) | 6,176,400 | |||||||
Payment to equity investment | $ 6,200 |
Commitments and Contingencies (
Commitments and Contingencies (Details) - USD ($) | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Commitments and Contingencies Disclosure [Abstract] | ||
Litigation or contingency expense | $ 0 | $ 0 |
Income Taxes - Narrative (Detai
Income Taxes - Narrative (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Income Taxes | |||
Net operating loss carryforwards, domestic | $ 472,100,000 | ||
Net operating loss carryforwards | $ 467,800,000 | ||
Available to offset | 80% | ||
Operating loss carryforward subject to expiration | $ 4,300,000 | ||
Tax credit carryforwards | 33,500,000 | ||
Accrued interest or penalties | 0 | ||
Increase in valuation allowance | $ 69,400,000 | ||
Effective tax rate | 0% | 0% | 0% |
State | |||
Income Taxes | |||
Net operating loss carryforwards, domestic state and city | $ 365,500,000 | ||
City | |||
Income Taxes | |||
Net operating loss carryforwards, domestic state and city | $ 3,700,000 |
Income Taxes - Deferred Taxes (
Income Taxes - Deferred Taxes (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Deferred tax assets: | ||
Net operating loss carryforwards | $ 121,029 | $ 93,032 |
Research and development credits | 6,069 | 3,422 |
Orphan drug credit | 27,515 | 19,401 |
Capitalized research and development | 41,588 | 21,262 |
Equity-based compensation | 26,429 | 20,286 |
Deferred revenue | 4,105 | 0 |
Other | 3,031 | 1,525 |
Total deferred tax assets | 229,766 | 158,928 |
Deferred tax liability: | ||
Operating lease right-of-use assets | (1,290) | (987) |
Depreciation | (1,255) | (113) |
Other | (2) | (2) |
Valuation allowance | (227,219) | (157,826) |
Net deferred tax assets | $ 0 | $ 0 |
Income Taxes - Reconciliation o
Income Taxes - Reconciliation of U.S. Statutory Income Tax Rate to Effective Tax Rate (Details) | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Reconciliation of the U.S. statutory income tax rate to the Company's effective tax rate | |||
Statutory tax rate | 21% | 21% | 21% |
U.S. state and local income taxes, net of U.S. federal income tax benefit | 2.17% | 2.33% | 5.15% |
Equity-based compensation | (1.21%) | (0.77%) | (0.03%) |
Research and development credit | 0.81% | 0.27% | 0.50% |
Orphan drug credit | 2.50% | 1.87% | 4.99% |
Section 162(m) limitation | (3.39%) | 0% | 0% |
Other | (0.54%) | 0% | 0% |
Change in valuation allowance | (21.34%) | (24.70%) | (31.61%) |
Effective tax rate | 0% | 0% | 0% |
401(k) Plan (Details)
401(k) Plan (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Retirement Benefits [Abstract] | |||
Contribution expense | $ 1.3 | $ 1 | $ 0.5 |
Net Loss per Share - Basic and
Net Loss per Share - Basic and diluted net loss per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Numerator: | |||
Net loss | $ (325,104) | $ (277,417) | $ (173,910) |
Net loss attributable to common stockholders, basic | (325,104) | (277,417) | (173,910) |
Net loss attributable to common stockholders, diluted | $ (325,104) | $ (277,417) | $ (173,910) |
Denominator: | |||
Weighted average number of shares outstanding, basic (in shares) | 63,123,936 | 53,290,528 | 48,497,790 |
Weighted average number of shares outstanding, diluted (in shares) | 63,123,936 | 53,290,528 | 48,497,790 |
Net loss per share, basic (in usd per share) | $ (5.15) | $ (5.21) | $ (3.59) |
Net loss per share, diluted (in usd per share) | $ (5.15) | $ (5.21) | $ (3.59) |
Net Loss per Share - Potential
Net Loss per Share - Potential common units not included in the calculation of the diluted net loss per unit and share (Details) - shares | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share | ||
Total potentially dilutive securities (in shares) | 13,466,312 | 10,070,528 |
Common stock options issued and outstanding | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share | ||
Total potentially dilutive securities (in shares) | 11,588,576 | 9,176,815 |
Restricted stock units subject to future vesting | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share | ||
Total potentially dilutive securities (in shares) | 1,486,540 | 671,954 |
Performance stock units subject to future vesting | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share | ||
Total potentially dilutive securities (in shares) | 284,362 | 0 |
Restricted stock awards subject to future vesting | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share | ||
Total potentially dilutive securities (in shares) | 106,834 | 221,759 |