Syndax Pharmaceuticals (SNDX) 10-Q 5 Nov 24 2024 Q3 Quarterly report Financial data

Document and Entity Information

Document and Entity Information	9 Months Ended
	Sep. 30, 2024
Cover [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Period End Date	Sep. 30, 2024
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	Q3
Trading Symbol	SNDX
Entity Registrant Name	Syndax Pharmaceuticals, Inc.
Entity Central Index Key	0001395937
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Current Fiscal Year End Date	--12-31
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Title of 12(b) Security	Common Stock
Security Exchange Name	NASDAQ
Entity File Number	001-37708
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	32-0162505
Entity Address, Address Line One	35 Gatehouse Drive
Entity Address, Address Line Two	Building D
Entity Address, Address Line Three	Floor 3
Entity Address, City or Town	Waltham
Entity Address, State or Province	MA
Entity Address, Postal Zip Code	02451
City Area Code	781
Local Phone Number	419-1400
Document Quarterly Report	true
Document Transition Report	false

Condensed Consolidated Balance

Condensed Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Current assets:
Cash and cash equivalents	$ 133,019	$ 295,394
Short-term investments	256,588	275,304
Short-term deposits	16,788	6,885
Other receivable	3,507	0
Prepaid expenses and other current assets	4,907	3,293
Total current assets	414,809	580,876
Long-term investments	10,029	29,829
Property and equipment, net	0	8
Right-of-use asset, net	756	1,487
Restricted cash	217	217
Other assets	0	463
Total assets	425,811	612,880
Current liabilities:
Accounts payable	4,898	9,961
Collaboration payable, net	5,506	7,232
Accrued expenses and other current liabilities	48,121	39,856
Current portion of right-of-use liability	842	1,035
Current portion of capital lease	10	12
Total current liabilities	59,377	58,096
Long-term liabilities:
Right-of-use liability, less current portion	0	578
Capital lease, less current portion	2	10
Total long-term liabilities	2	588
Total liabilities	59,379	58,684
Commitments and contingencies (Note 12)
Stockholders’ equity:
Preferred stock, $0.001 par value, 10,000,000 shares authorized; 0 shares outstanding at September 30, 2024 and December 31, 2023	0	0
Common stock, $0.0001 par value, 200,000,000 shares authorized; 85,571,205 and 84,826,632 shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively	9	8
Additional paid-in capital	1,493,041	1,456,370
Accumulated other comprehensive gain	371	218
Accumulated deficit	(1,126,989)	(902,400)
Total stockholders’ equity	366,432	554,196
Total liabilities and stockholders’ equity	$ 425,811	$ 612,880

Condensed Consolidated Balanc_2

Condensed Consolidated Balance Sheets (Unaudited) (Parenthetical) - $ / shares	Sep. 30, 2024	Dec. 31, 2023
Statement of Financial Position [Abstract]
Preferred stock, par value	$ 0.001	$ 0.001
Preferred stock, shares authorized	10,000,000	10,000,000
Preferred stock, shares outstanding	0	0
Common stock, par value	$ 0.0001	$ 0.0001
Common stock, shares authorized	200,000,000	200,000,000
Common stock, shares issued	85,571,205	84,826,632
Common stock, shares outstanding	85,571,205	84,826,632

CONDENSED CONSOLIDATED STATEMEN

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (Unaudited) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Revenue:
Milestone and license revenue	$ 12,500	$ 0	$ 16,000	$ 0
Total revenues	12,500	0	16,000	0
Operating expenses:
Research and development	70,971	39,087	176,118	107,906
Selling, general and administrative	31,106	17,268	83,189	44,143
Total operating expenses	102,077	56,355	259,307	152,049
Loss from operations	(89,577)	(56,355)	(243,307)	(152,049)
Other income (expense), net:
Interest expense	(23)	(70)	(123)	(145)
Interest income	5,442	5,345	18,982	15,613
Other income (expense)	32	(66)	(141)	(306)
Total other income, net	5,451	5,209	18,718	15,162
Net loss	(84,126)	(51,146)	(224,589)	(136,887)
Other comprehensive loss:
Unrealized gain on marketable securities	1,188	119	371	351
Comprehensive loss	(82,938)	(51,027)	(224,218)	(136,536)
Net loss attributable to common stockholders	$ (84,126)	$ (51,146)	$ (224,589)	$ (136,887)
Net loss per share attributable to common stockholders—basic	$ (0.98)	$ (0.73)	$ (2.63)	$ (1.97)
Net loss per share attributable to common stockholders—diluted	$ (0.98)	$ (0.73)	$ (2.63)	$ (1.97)
Weighted-average number of common shares used to compute net loss per share attributable to common stockholders —basic	85,433,569	69,855,766	85,307,660	69,645,888
Weighted-average number of common shares used to compute net loss per share attributable to common stockholders—diluted	85,433,569	69,855,766	85,307,660	69,645,888

CONDENSED CONSOLIDATED STATEM_2

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023
CASH FLOWS FROM OPERATING ACTIVITIES:
Net loss	$ (224,589)	$ (136,887)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation	8	9
Accretion of investments	(10,528)	(11,344)
Non-cash operating lease expense	781	496
Stock-based compensation	30,729	22,624
Changes in operating assets and liabilities:
Prepaid expenses and other assets	(11,567)	(3,281)
Collaboration (payable) receivable, net	(1,726)	68
Other receivable	(3,507)	0
Other assets	463	0
Accounts payable	(5,063)	1,699
Accrued expenses and other liabilities	7,484	7,364
Net cash used in operating activities	(217,515)	(119,252)
CASH FLOWS FROM INVESTING ACTIVITIES:
Purchases of short and long-term investments	(180,738)	(225,316)
Proceeds from sales and maturities of short-term investments	229,936	359,215
Net cash provided by investing activities	49,198	133,899
CASH FLOWS FROM FINANCING ACTIVITIES:
Proceeds from Employee Stock Purchase Plan	1,081	633
Proceeds from stock option exercises	4,861	5,016
Net cash provided by financing activities	5,942	5,649
NET (DECREASE) INCREASE CASH, CASH EQUIVALENTS AND RESTRICTED CASH	(162,375)	20,296
CASH, CASH EQUIVALENTS AND RESTRICTED CASH—beginning of period	295,611	74,471
CASH, CASH EQUIVALENTS AND RESTRICTED CASH —end of period	$ 133,236	$ 94,767

Pay vs Performance Disclosure

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2024	Sep. 30, 2023
Pay vs Performance Disclosure
Net Income (Loss)	$ (84,126)	$ (68,063)	$ (72,400)	$ (51,146)	$ (44,615)	$ (41,126)	$ (224,589)	$ (136,887)

Insider Trading Arrangements

Insider Trading Arrangements	3 Months Ended
	Sep. 30, 2024 shares
Trading Arrangements, by Individual
Material Terms of Trading Arrangement	Trading Arrangements During the quarter ended September 30, 2024 , our directors and officers (as defined in Rule 16a-1(f) under the Exchange Act) adopted or terminated contracts, instructions or written plans for the purchase or sale of our securities as set forth in the table below. Type of Trading Arrangement Name and Position Action Adoption/Termination Date Rule 10b5-1* Non- Rule 10b5-1** Total Shares of Common Stock to be Sold Total Shares of Common Stock to be Purchased Expiration Date Michael A. Metzger Chief Executive Officer, Director Adoption *** 8/2/2024 X 171,195 - 9/10/2025 * Contract, instructions, or written plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) under the Exchange Act. ** “Non-Rule 10b5-1 trading arrangement” as defined in Item 408(c) of Regulation S-K under the Exchange Act. *** The selling start date of Mr. Metzger’s plan is January 2025, after the Company’s current NDA PDUFA date of December 26, 2024, and the plan solely includes equity grants with expiration dates prior to September 10, 2025.
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false
Michael A Metzger [Member]
Trading Arrangements, by Individual
Name	Michael A. Metzger
Title	Chief Executive Officer, Director
Rule 10b5-1 Arrangement Adopted	true
Non-Rule 10b5-1 Arrangement Adopted	true
Adoption Date	8/2/2024
Expiration Date	9/10/2025
Aggregate Available	171,195

Nature of Business

Nature of Business	9 Months Ended
	Sep. 30, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Nature of Business	1. Nature of Business Syndax Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company with one commercially approved product and an innovative pipeline of cancer therapies under development. We were incorporated in Delaware in 2005. We have operations in New York, NY and Waltham, MA and we operate in one segment. References in these notes to condensed consolidated financial statements to “Syndax,” “the Company,” “we,” “us” or “our” refer to Syndax Pharmaceuticals, Inc. and its wholly owned subsidiaries.

Basis of Presentation

Basis of Presentation	9 Months Ended
	Sep. 30, 2024
Accounting Policies [Abstract]
Basis of Presentation	2. Basis of Presentation The Company has prepared the accompanying condensed consolidated financial statements in conformity with accounting principles generally accepted in the United States of America, or U.S. GAAP. Certain information and footnote disclosures normally included in the Company’s annual financial statements have been condensed or omitted. The interim unaudited condensed financial statements have been prepared on the same basis as the annual audited financial statements and in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for the fair statement of the Company’s financial position as of September 30, 2024, and the results of operations and comprehensive loss for the three and nine months ended September 30, 2024 and 2023, and cash flows for the nine months ended September 30, 2024 and 2023. The results for the three and nine months ended September 30, 2024 are not necessarily indicative of the results to be expected for the year ending December 31, 2024, any other interim periods, or any future year or period. These interim financial statements should be read in conjunction with the audited financial statements as of and for the year ended December 31, 2023, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K as filed with the Securities and Exchange Commission, or the SEC, on February 27, 2024. In 2011, the Company established a wholly owned subsidiary in the United Kingdom, which the Company dissolved in June 2024. In 2014, the Company established a wholly owned U.S. subsidiary, and in 2021, the Company established a wholly owned subsidiary in the Netherlands. To date, there have been no material activities for these entities. All intercompany balances and transactions have been eliminated in consolidation.

Summary of Significant Accounti

Summary of Significant Accounting Policies	9 Months Ended
	Sep. 30, 2024
Accounting Policies [Abstract]
Summary of Significant Accounting Policies	3. Summary of Significant Accounting Policies The Company’s significant accounting policies, which are disclosed in the audited consolidated financial statements for the year ended December 31, 2023, and the notes thereto are included in the Company’s Annual Report on Form 10-K that was filed with the SEC on February 27, 2024 . Since the date of filing, there have been no material changes to the Company’s significant accounting policies except as noted below. Use of Estimates The preparation of condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the condensed consolidated financial statements and the reported amounts of costs and expenses during the reporting period. The Company bases estimates and assumptions on historical experience when available and on various factors that it believes to be reasonable under the circumstances. The Company evaluates its estimates and assumptions on an ongoing basis. Estimates and assumptions about future events and their effects cannot be determined with certainty and therefore require the exercise of judgment. As of the date of issuance of these financial statements, the Company is not aware of any specific event or circumstance that would require the Company to update its estimates, assumptions and judgments or revise the carrying value of its assets or liabilities. These estimates may change as new events occur and additional information is obtained and are recognized in the consolidated financial statements as soon as they become known. Actual results could differ from those estimates and any such differences may be material to the Company’s condensed consolidated financial statements. Significant Risks and Uncertainties We are subject to challenges and risks specific to our business and our ability to execute on our strategy, as well as risks and uncertainties common to companies in the pharmaceutical industry with development and commercial operations, including, without limitation, risks and uncertainties associated with: obtaining regulatory approval of our late-stage product candidate; delays or problems in the supply of our products, loss of single source suppliers or failure to comply with manufacturing regulations; identifying, acquiring or in-licensing additional products or product candidates; pharmaceutical product development and the inherent uncertainty of clinical success; the challenges of protecting and enhancing our intellectual property rights; and complying with applicable regulatory requirements. Income taxes In accordance with ASC 270, Interim Reporting, and ASC 740, Income Taxes, the Company is required at the end of each interim period to determine the best estimate of its annual effective tax rate and then apply that rate in providing for income taxes on a current year-to-date (interim period) basis. For the nine months ended September 30, 2024 and 2023, the Company recorded no tax expense or benefit due to the expected current year loss and its historical losses. As of September 30, 2024 and December 31, 2023, the Company concluded that a full valuation allowance would be necessary for all of its net deferred tax assets. The Company had no amounts recorded for uncertain tax positions, interest or penalties in the accompanying consolidated financial statements. Recently Issued and Adopted Accounting Pronouncements From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board, or FASB, or other accounting standard setting bodies that we adopt as of the specified effective date. Unless otherwise discussed below, we do not believe that the adoption of recently issued standards have or may have a material impact on our consolidated statements or disclosures. Segment Reporting In December 2023, the FASB issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures , which requires all public entities, including public entities with a single reportable segment, to provide in interim and annual periods one or more measures of segment profit or loss used by the chief operating decision maker to allocate resources and assess performance. Additionally, the standard requires disclosures of significant segment expenses and other segment items as well as incremental qualitative disclosures. The guidance in this update is effective for fiscal years beginning after December 15, 2023, and interim periods after December 15, 2024. The Company is currently in the process of evaluating the effects of this pronouncement on our related disclosures.

Significant Collaborative Resea

Significant Collaborative Research and License Agreements	9 Months Ended
	Sep. 30, 2024
Research and Development [Abstract]
Significant Collaborative Research and License Agreements	4. Significant Collaborative Research and License Agreements Incyte Collaboration In September 2021, the Company entered into the Incyte License and Collaboration Agreement, or the Incyte License, with Incyte covering the worldwide development and commercialization of axatilimab. Also in September 2021, the Company entered into a share purchase agreement with Incyte, or the Incyte Share Purchase Agreement. These agreements are collectively referred to as the Incyte Agreements. Under the terms of the Incyte Agreements, Incyte received exclusive commercialization rights outside of the United States, subject to certain royalty payment obligations set forth below. In the United States, Incyte and the Company are co-commercializing and co-promoting axatilimab as Niktimvo™ (axatilimab-csfr). The Company and Incyte share equally the profits and losses from co-commercialization efforts in the United States. The Company and Incyte have agreed to continue to co-develop axatilimab and to share development costs associated with global and additional U.S.-specific clinical trials, with Incyte responsible for 55 % of such costs and the Company responsible for 45 % of such costs. Each company will be responsible for funding any of its own independent development activities. Incyte is responsible for 100 % of future development costs for trials that are specific to ex-U.S. countries. All development costs related to the collaboration will be subject to a joint development plan. Under the terms of the Incyte Agreements, in December 2021, Incyte paid the Company a non-refundable cash payment of $ 117.0 million and the Company issued 1,421,523 shares of common stock with an aggregate purchase price of $ 35.0 million, or $ 24.62 per share. Additionally, under the terms of the Incyte Agreements, the Company was eligible, upon execution, to receive up to $ 220.0 million in future contingent development and regulatory milestones and up to $ 230.0 million in commercialization milestones as well as tiered royalties ranging in the mid-teens percentage on net sales of the licensed product comprising axatilimab in Europe and Japan and low double digit percentage in the rest of the world outside of the United States. The Company’s right to receive royalties in any particular country will expire upon the last to occur of (a) the expiration of licensed patent rights covering the licensed product in that particular country, (b) a specified period of time after the first post - marketing authorization sale of a licensed product in that country, and (c) the expiration of any regulatory exclusivity for that licensed product in that country. In August 2024, the U.S. Food and Drug Administration, or FDA, approved Niktimvo for the treatment of cGVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs). As a result of the approval of Niktimvo, the Company earned a revenue milestone of $ 12.5 million, which was received in September 2024. As of September 30, 2024, the Company has recorded approximately $ 1.7 million as a collaboration receivable due from Incyte related to the Company’s development and pre-commercialization costs under the Incyte Agreements and has recorded approximately $ 7.2 million as a collaboration payable due to Incyte for development and pre-commercialization costs incurred by Incyte as of September 30, 2024. Both expense and cost offset are recorded as part of operating expenses. Vitae Pharmaceuticals, Inc. In October 2017, the Company entered into a license agreement, or the Vitae License Agreement, with Vitae Pharmaceuticals, LLC, or Vitae, a subsidiary of AbbVie, Inc., under which the Company was granted an exclusive, sublicensable, worldwide license to a portfolio of preclinical, orally available, small molecule inhibitors of the Menin–KMT2A binding interaction, or the Menin Assets. Upon execution of the agreement, the Company would potentially be required to pay Allergan up to $ 99.0 million in one-time development a nd regulatory milestone payments over the term of the Vitae License Agreement, subject to the achievement of certain milestone events. In the event that the Company or any of its affiliates or sublicensees commercializes the Menin Assets, the Company will also be obligated to pay Vitae low single to low double-digit royalties on sales, subject to reduction in certain circumstances, as well as up to an aggregate of $ 70.0 million in potential one-time, sales-based milestone payments based on achievement of certain annual sales thresholds. The Company is solely responsible for the development and commercialization of the Menin Assets. Each party may terminate the Vitae License Agreement for the other party’s uncured material breach or insolvency, and the Company may terminate the Vitae License Agreement at any time upon advance written notice to Vitae. Vitae may terminate the Vitae License Agreement if the Company or any of its affiliates or sublicensees institutes a legal challenge to the validity, enforceability, or patentability of the licensed patent rights. Unless terminated earlier in accordance with its terms, the Vitae License Agreement will continue on a country-by-country and product-by-product basis until the later of: (i) the expiration of all of the licensed patent rights in such country; (ii) the expiration of all regulatory exclusivity applicable to the product in such country; and (iii) 10 years from the date of the first commercial sale of the product in such country. As of the date of the Vitae License Agreement, the asset acquired had no alternative future use nor had it reached a stage of technological feasibility. As the processes or activities that were acquired along with the license do not constitute a “business,” the transaction has been accounted for as an asset acquisition. Since the effective date of the Vitae License Agreement, the Company achieved certain development and regulatory milestones, resulting in $ 18.0 m illion in research and development expense, which includes an $ 8.0 million milestone paid during the first quarter of 2024 for the successful completion of the first pivotal trial in the first indication. UCB Biopharma Sprl In 2016, the Company entered into a license agreement, or the UCB License Agreement, as amended from time to time, with UCB Biopharma Sprl, or UCB, under which UCB granted to the Company a worldwide, sublicensable, exclusive license to UCB6352, which the Company refers to as axatilimab, an anti-CSF-1R monoclonal antibody. Upon execution of the agreement, the Company would potentially be required to pay UCB up to $ 119.5 million in one-time development and regulatory milestone payments over the term of the UCB License Agreement, subject to the achievement of certain milestone events. In the event that the Company or any of its affiliates or sublicensees commercializes axatilimab, the Company will also be obligated to pay UCB low double-digit royalties on sales, subject to reduction in certain circumstances, as well as up to an aggregate of $ 250.0 million in potential one-time, sales-based milestone payments based on achievement of certain annual sales thresholds. Under certain circumstances, the Company may be required to share a percentage of non-royalty income from sublicensees, subject to certain deductions, with UCB. The Company is solely responsible for the development and commercialization of axatilimab, except that UCB was responsible for performing a limited set of transitional chemistry, manufacturing and control tasks related to axatilimab. Each party may terminate the UCB License Agreement for the other party’s uncured material breach or insolvency, and the Company may terminate the UCB License Agreement at any time upon advance written notice to UCB. UCB may terminate the UCB License Agreement if the Company or any of its affiliates or sublicensees institutes a legal challenge to the validity, enforceability, or patentability of the licensed patent rights. Unless terminated earlier in accordance with its terms, the UCB License Agreement will continue on a country-by-country and product-by-product basis until the later of: (i) the expiration of all of the licensed patent rights in such country; (ii) the expiration of all regulatory exclusivity applicable to the product in such country; and (iii) 10 years from the date of the first commercial sale of the product in such country. As of the date of the UCB License Agreement, the asset acquired had no alternative future use nor had it reached a stage of technological feasibility. As the processes or activities that were acquired along with the license do not constitute a “business,” the transaction has been accounted for as an asset acquisition. As a result, in 2016, the upfront payment of $ 5.0 million was recorded as research and development expense in the consolidated statements of operations. Additionally, in connection with its most recent amendment of the UCB License Agreement, in the second quarter of 2022 the Company paid UCB $ 5.8 million, which was recognized as a milestone expense. Since the effective date of the license agreement, the Company achieved certain development and regulatory milestones and has recorded $ 31.0 million as research and development expense, which includes a $ 15.0 million milestone paid during the three months ended September 30, 2024, upon the approval of Niktimvo. Bayer Pharma AG (formerly known as Bayer Schering Pharma AG) In March 2007, the Company entered into a license agreement with Bayer Schering Pharma AG, or Bayer, for a worldwide, exclusive license to develop and commercialize entinostat and any other products containing the same active ingredient. The Company will pay Bayer royalties on a sliding scale based on net sales, if any, and make future milestone payments to Bayer of up to $ 150.0 million in the event that certain specified development and regulatory goals and sales levels are achieved. Eddingpharm Investment Company Limited In August 2016, the Company entered into a license agreement with Eddingpharm Investment Company, or Eddingpharm, to develop and commercialize entinostat in China and certain other Asian countries. Eddingpharm will pay the Company royalties on a sliding scale based on net sales, if any, and make future milestone payments up to $ 10.0 million in the event that certain specified development and regulatory goals are achieved. In April 2024, a milestone was achieved under the Eddingpharm license agreement for the marketing approval of entinostat in China. As a result, the Company recognized $ 3.5 million of milestone and license revenue in the second quarter of 2024.

Net Loss per Share Attributable

Net Loss per Share Attributable to Common Stockholders	9 Months Ended
	Sep. 30, 2024
Earnings Per Share [Abstract]
Net Loss per Share Attributable to Common Stockholders	5. Net Loss per Share Attributable to Common Stockholders Basic net loss per share attributable to common stockholders is computed by dividing the net loss attributable to common stockholders by the weighted-average number of common shares outstanding for the period. Because the Company has reported a net loss for all periods presented, diluted net loss per common share is the same as basic net loss per common share for those periods. The following table summarizes the computation of basic and diluted net loss per share attributable to common stockholders of the Company: Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 (In thousands, except share and per (In thousands, except share and per Numerator—basic and diluted: Net loss $ ( 84,126 ) $ ( 51,146 ) $ ( 224,589 ) $ ( 136,887 ) Net loss attributable to common $ ( 84,126 ) $ ( 51,146 ) $ ( 224,589 ) $ ( 136,887 ) Net loss per share attributable to common $ ( 0.98 ) $ ( 0.73 ) $ ( 2.63 ) $ ( 1.97 ) Denominator—basic and diluted: Weighted-average number of common shares 85,433,569 69,855,766 85,307,660 69,645,888 The following potentially dilutive securities have been excluded from the computation of diluted weighted-average shares outstanding because such securities have an antidilutive impact due to losses reported (in common stock equivalent shares): September 30, 2024 2023 Options to purchase common stock 12,205,960 10,530,750 Employee Stock Purchase Plan 73,200 35,479 Non-vested restricted stock units (RSUs) 1,461,005 524,236 For additional information related to the Company’ s common stock see Note 10.

Other Receivables

Other Receivables	9 Months Ended
	Sep. 30, 2024
Other Receivables, Net, Current [Abstract]
Other Receivables	6. Other Receivables Contemporaneous with the Company’s New Drug Application, or NDA, submission to the FDA for revumenib, it was required to pay a $ 6.1 million refundable fee under the Prescription Drug User Fee Act, or PDUFA. The $ 6.1 million fee was paid in January 2024 and was fully refunded in September 2024. In April 2024, entinostat received marketing approval in China. As of September 30, 2024, the Company recorded a $ 3.5 million milestone receivable related to achieved milestones under the license agreement with Eddingpharm.

Fair Value Measurements

Fair Value Measurements	9 Months Ended
	Sep. 30, 2024
Fair Value Disclosures [Abstract]
Fair Value Measurements	7. Fair Value Measurements The carrying amounts of cash and cash equivalents, restricted cash, accounts payable, and accrued expenses approximated their estimated fair values due to the short-term nature of these financial instruments. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value are performed in a manner to maximize the use of observable inputs and minimize the use of unobservable inputs. The accounting standard describes a fair value hierarchy based on three levels of inputs, of which the first two are considered observable and the last unobservable, that may be used to measure fair value, which are the following: Level 1— Quoted prices (unadjusted) in active markets that are accessible at the market date for identical unrestricted assets or liabilities. Level 2— Inputs other than Level 1 that are observable, either directly or indirectly, such as quoted prices for similar assets or liabilities; quoted prices in markets that are not active; or other inputs for which all significant inputs are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities. Level 3— Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities. The table below presents information about the Company’s assets and liabilities that are regularly measured and carried at fair value and indicate the level within the fair value hierarchy of valuation techniques the Company utilized to determine such fair values (in thousands): Fair Value Measurements Using Quoted Prices Significant (unadjusted) Other Significant Total in Active Observable Unobservable Carrying Markets Inputs Inputs Value (Level 1) (Level 2) (Level 3) (In thousands) September 30, 2024 Assets: Cash and cash equivalents $ 133,019 $ 133,019 $ — $ — Short-term investments 256,588 — 256,588 — Long-term investments 10,029 — 10,029 — Total assets $ 399,636 $ 133,019 $ 266,617 $ — December 31, 2023 Assets: Cash and cash equivalents $ 295,394 $ 295,394 $ — $ — Short-term investments 275,304 — 275,304 — Long-term investments 29,829 — 29,829 — Total assets $ 600,527 $ 295,394 $ 305,133 $ — There have been no material impairments of our assets measured and carried at fair value during the period ended September 30, 2024 and 2023. In addition, there have been no changes in valuation techniques during the periods ended September 30, 2024 and 2023. The fair value of Level 1 instruments classified as cash equivalents are valued using quoted market prices in active markets. The fair value of Level 2 instruments classified as short and long-term investments are determined based on quoted prices in active markets, which are either directly or indirectly observable as of the reporting date with fair value being determined using models or other valuation methodologies. The Company’s short and long-term investments are classified as available-for-sale securities. As of September 30, 2024 , the remaining contractual maturities of the available-for-sale securities were 1 to 13 months, and the balance in the Company’s accumulated other comprehensive gain was comprised solely of activity related to the Company’s available-for-sale securities. There were no realized gains or losses recognized on the sale or maturity of available-for-sale securities, during the three and nine months ended September 30, 2024 and 2023. As a result, the Company did not reclassify any amounts out of accumulated other comprehensive gain for the same periods. The following table summarizes the available-for-sale securities: Amortized Unrealized Unrealized Cost Gains Losses Fair Value (In thousands) September 30, 2024 Commercial paper $ 81,096 $ 44 $ — $ 81,140 Corporate bonds 40,456 17 — 40,473 US Treasury 134,716 259 — 134,975 Federal bonds 9,978 51 — 10,029 $ 266,246 $ 371 $ — $ 266,617 December 31, 2023 Commercial paper $ 160,657 $ 149 $ — $ 160,806 Corporate bonds 47,150 62 — 47,212 US Treasury 68,111 46 — 68,157 Federal bonds 28,998 — ( 40 ) 28,958 $ 304,916 $ 257 $ ( 40 ) $ 305,133

Prepaid Expenses and Other Curr

Prepaid Expenses and Other Current Assets	9 Months Ended
	Sep. 30, 2024
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract]
Prepaid Expenses and Other Current Assets	8. Prepaid Expenses and Other Current Assets P repaid expenses and other current assets consisted of the following (in thousands): September 30, 2024 December 31, 2023 Prepaid insurance 1,488 807 Interest receivable on investments 1,183 1,227 Prepaid subscription 1,295 769 Prepaid state and local taxes 601 264 Prepaid rent 122 163 Other 218 63 Total prepaid expenses and other current assets $ 4,907 $ 3,293

Accrued Expenses and Other Curr

Accrued Expenses and Other Current Liabilities	9 Months Ended
	Sep. 30, 2024
Payables and Accruals [Abstract]
Accrued Expenses and Other Current Liabilities	9. Accrued Expenses and Other Current Liabilities Accrued expenses and other current liabilities consisted of the following (in thousands): September 30, 2024 December 31, 2023 Accrued clinical study and trial costs $ 35,692 $ 16,346 Accrued compensation and related costs 11,602 11,172 Accrued professional fees 619 1,450 Accrued milestone costs — 10,000 Other 208 888 Total accrued expenses and other current liabilities $ 48,121 $ 39,856

Stock-Based Compensation

Stock-Based Compensation	9 Months Ended
	Sep. 30, 2024
Share-Based Payment Arrangement [Abstract]
Stock-Based Compensation	10. Stock-Based Compensation In January 2024, the number of shares of common stock available for issuance under the Company’s 2015 Omnibus Incentive Plan, or the 2015 Plan, was increased by 3,393,065 shares of common stock due to the automatic annual provision to increase shares of common stock available under the 2015 Plan. Additionally in December 2023, the Company’s board of directors approved an increase of 1,100,000 shares of common stock available for issuance under the Company’s 2023 Inducement Plan, or Inducement Plan. As of September 30, 2024, there were 3,862,670 shares of common stock available for issuance under the 2015 Plan and 76,763 shares of common stock available for issuance under the Inducement Plan. The Company recognized stock-based compensation expense related to the issuance of stock option awards and restricted stock units to employees and non-employees and related to the Company’s 2015 Employee Stock Purchase Plan, or ESPP, in the condensed consolidated statements of comprehensive loss as follows: Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Research and development $ 5,886 $ 3,853 $ 14,347 $ 10,329 Selling, general and administrative 6,048 4,468 16,382 12,295 Total $ 11,934 $ 8,321 $ 30,729 $ 22,624 Compensation expense by type of award in the three and nine months ended September 30, 2024 and 2023 was as follows: Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Stock options $ 9,300 $ 7,242 $ 24,285 $ 19,654 RSUs 2,455 990 6,071 2,749 ESPP 179 89 373 221 Total $ 11,934 $ 8,321 $ 30,729 $ 22,624 As of September 30, 2024, there were $ 93.3 million of unrecognized compensation costs related to employee and non-employee unvested stock options and RSUs granted under the 2023 Inducement Plan, 2015 Plan and the Company’s 2007 Stock Plan, which are expected to be recognized over a weighted-average remaining service period of 2.61 years.

Stockholders' Equity

Stockholders' Equity	9 Months Ended
	Sep. 30, 2024
Equity [Abstract]
Stockholders' Equity	11. Stockholders’ Equity The following table presents the changes in stockholders’ equity for the three and nine months ended September 30, 2024: (In thousands, except share data) Common Stock Additional Accumulated Accumulated Total Shares Amount Balance as of December 31, 2023 84,826,632 $ 8 $ 1,456,370 $ 218 $ ( 902,400 ) $ 554,196 Stock purchase under ESPP 35,463 — — — — — Stock-based compensation expense — — 8,899 — — 8,899 Unrealized loss on investments — — — ( 974 ) — ( 974 ) Vesting of RSUs 3,750 — — — — — Employee withholdings ESPP — — 309 — — 309 Proceeds from exercise of stock options 113,841 — 1,859 — — 1,859 Net loss — — — — ( 72,400 ) ( 72,400 ) Balance as of March 31, 2024 84,979,686 $ 8 $ 1,467,437 $ ( 756 ) $ ( 974,800 ) $ 491,889 Stock-based compensation expense — — 9,896 — — 9,896 Unrealized loss on investments — — — ( 61 ) — ( 61 ) Vesting of RSUs 1,603 — — — — — Employee withholdings ESPP — — 237 — — 237 Proceeds from exercise of stock options 47,340 — 439 — — 439 Par value adjustment — 1 — — — 1 Net loss — — — — ( 68,063 ) ( 68,063 ) Balance as of June 30, 2024 85,028,629 $ 9 $ 1,478,009 $ ( 817 ) $ ( 1,042,863 ) $ 434,338 Stock-based compensation expense — — 11,934 — — 11,934 Unrealized gains on investments — — — 1,188 — 1,188 Stock purchase under ESPP 29,440 — — — — — Employee withholdings ESPP — — 535 — — 535 Vesting of RSUs 19,057 — — — — — Proceeds from exercise of stock options 208,362 — 2,563 — — 2,563 Net loss — — — — ( 84,126 ) ( 84,126 ) Balance as of September 30, 2024 85,285,488 $ 9 $ 1,493,041 $ 371 $ ( 1,126,989 ) $ 366,432 The following table presents the changes in stockholders’ equity for the three and nine months ended September 30, 2023: (In thousands, except share data) Common Stock Additional Accumulated Accumulated Total Shares Amount Balance as of December 31, 2022 68,111,385 $ 7 $ 1,161,288 $ ( 806 ) $ ( 693,040 ) $ 467,449 Stock purchase under ESPP 16,537 — — — — — Stock-based compensation expense — — 6,238 — — 6,238 Unrealized gain on short-term investments — — — 470 — 470 Vesting of RSUs 5,000 — — — — — Employee withholdings ESPP — — 196 — — 196 Prefunded warrants, exercise 85,998 — — — — — Proceeds from exercise of stock options 276,506 — 2,278 — — 2,278 Net loss — — — — ( 41,126 ) ( 41,126 ) Balance as of March 31, 2023 68,495,426 $ 7 $ 1,170,000 $ ( 336 ) $ ( 734,166 ) $ 435,505 Stock-based compensation expense — — 8,065 — — 8,065 Unrealized loss on short-term investments — — — ( 238 ) — ( 238 ) Vesting of RSUs 1,602 — — — — — Employee withholdings ESPP — — 179 — — 179 Prefunded warrants, exercise 771,133 — ( 1 ) — — ( 1 ) Proceeds from exercise of stock options 163,037 — 1,146 — — 1,146 Net loss — — — — ( 44,615 ) ( 44,615 ) Balance as of June 30, 2023 69,431,198 $ 7 $ 1,179,389 $ ( 574 ) $ ( 778,781 ) $ 400,041 Stock-based compensation expense — — 8,321 — — 8,321 Unrealized gains on short-term investments — — — 119 — 119 Stock purchase under ESPP 18,260 — — — — — Employee withholdings ESPP — — 259 — — 259 Proceeds from exercise of stock options 188,710 — 1,592 — — 1,592 Net loss ( 51,146 ) ( 51,146 ) Balance as of September 30, 2023 69,638,168 $ 7 $ 1,189,561 $ ( 455 ) $ ( 829,927 ) $ 359,186 At-the Market Program In May 2023, the Company entered into a sales agreement with Cowen and Company, or TD Cowen, under which the Company could, from time to time, issue and sell shares of its common stock having aggregate sales proceeds of up to $ 200.0 million, in a series of one or more ATM equity offerings, or the 2023 ATM Program. TD Cowen is not required to sell any specific share amounts but acts as the Company’s sales agent, using commercially reasonable efforts consistent with its normal trading and sales practices. Pursuant to the sales agreement, shares will be sold pursuant to the previous shelf registration statement on Form S-3ASR (Registration No. 333-277424), which became automatically effective upon the filing on February 27, 2024. The Company’s common stock will be sold at prevailing market prices at the time of the sale, and as a result, prices may vary. For the three and nine months ended September 30, 2024, the Company sold no shares of common stock under the 2023 ATM Program. Pre-Funded Warrants In December 2021, the Company sold pre-funded warrants to purchase 1,142,856 shares of common stock. As of September 30, 2024 , 285,714 pre-funded warrants were issued and outstanding.

Commitments and Contingencies

Commitments and Contingencies	9 Months Ended
	Sep. 30, 2024
Commitments and Contingencies Disclosure [Abstract]
Commitments and Contingencies	12. Commitments and Contingencies From time to time, the Company may be subject to various claims and proceedings in the ordinary course of business. If the potential loss from any claim, asserted or unasserted, or proceeding is considered probable and the amount is reasonably estimable, the Company will accrue a liability for the estimated loss. There were no contingent liabilities recorded as of September 30, 2024

Subsequent Events

Subsequent Events	9 Months Ended
	Sep. 30, 2024
Subsequent Events [Abstract]
Subsequent Events	13. Subsequent Events Royalty Pharma Development Funding, LLC Royalty Monetization Agreement In October 2024, the Company entered into a purchase and sale agreement, or Royalty Agreement, with Royalty Pharma Development Funding, LLC, or Royalty Pharma, pursuant to which Royalty Pharma purchased the right to receive 13.8 % on quarterly net sales of Niktimvo in the United States of America and its respective territories, districts, commonwealths and possessions (including Guam and Puerto Rico) in exchange for an upfront payment of $ 350.0 million (gross) at closing, received in November 2024. Aggregate payments to Royalty Pharma pursuant to the Royalty Agreement will be capped at $ 822.5 million or 2.35 times the funded amount. NYC Office Lease Extension In October 2024, the Company entered into a three and a half year lease extension for its office at 730 Third Avenue, New York, NY, commencing on January 1, 2025. The term of the new lease expires on August 31, 2028 .

Summary of Significant Accoun_2

Summary of Significant Accounting Policies (Policies)	9 Months Ended
	Sep. 30, 2024
Accounting Policies [Abstract]
Significant Accounting Policies	The Company’s significant accounting policies, which are disclosed in the audited consolidated financial statements for the year ended December 31, 2023, and the notes thereto are included in the Company’s Annual Report on Form 10-K that was filed with the SEC on February 27, 2024 . Since the date of filing, there have been no material changes to the Company’s significant accounting policies except as noted below.
Use of Estimates	Use of Estimates The preparation of condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the condensed consolidated financial statements and the reported amounts of costs and expenses during the reporting period. The Company bases estimates and assumptions on historical experience when available and on various factors that it believes to be reasonable under the circumstances. The Company evaluates its estimates and assumptions on an ongoing basis. Estimates and assumptions about future events and their effects cannot be determined with certainty and therefore require the exercise of judgment. As of the date of issuance of these financial statements, the Company is not aware of any specific event or circumstance that would require the Company to update its estimates, assumptions and judgments or revise the carrying value of its assets or liabilities. These estimates may change as new events occur and additional information is obtained and are recognized in the consolidated financial statements as soon as they become known. Actual results could differ from those estimates and any such differences may be material to the Company’s condensed consolidated financial statements.
Significant Risks and Uncertainties	Significant Risks and Uncertainties We are subject to challenges and risks specific to our business and our ability to execute on our strategy, as well as risks and uncertainties common to companies in the pharmaceutical industry with development and commercial operations, including, without limitation, risks and uncertainties associated with: obtaining regulatory approval of our late-stage product candidate; delays or problems in the supply of our products, loss of single source suppliers or failure to comply with manufacturing regulations; identifying, acquiring or in-licensing additional products or product candidates; pharmaceutical product development and the inherent uncertainty of clinical success; the challenges of protecting and enhancing our intellectual property rights; and complying with applicable regulatory requirements.
Income taxes	Income taxes In accordance with ASC 270, Interim Reporting, and ASC 740, Income Taxes, the Company is required at the end of each interim period to determine the best estimate of its annual effective tax rate and then apply that rate in providing for income taxes on a current year-to-date (interim period) basis. For the nine months ended September 30, 2024 and 2023, the Company recorded no tax expense or benefit due to the expected current year loss and its historical losses. As of September 30, 2024 and December 31, 2023, the Company concluded that a full valuation allowance would be necessary for all of its net deferred tax assets. The Company had no amounts recorded for uncertain tax positions, interest or penalties in the accompanying consolidated financial statements.
Recently Issued and Adopted Accounting Pronouncements	Recently Issued and Adopted Accounting Pronouncements From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board, or FASB, or other accounting standard setting bodies that we adopt as of the specified effective date. Unless otherwise discussed below, we do not believe that the adoption of recently issued standards have or may have a material impact on our consolidated statements or disclosures. Segment Reporting In December 2023, the FASB issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures , which requires all public entities, including public entities with a single reportable segment, to provide in interim and annual periods one or more measures of segment profit or loss used by the chief operating decision maker to allocate resources and assess performance. Additionally, the standard requires disclosures of significant segment expenses and other segment items as well as incremental qualitative disclosures. The guidance in this update is effective for fiscal years beginning after December 15, 2023, and interim periods after December 15, 2024. The Company is currently in the process of evaluating the effects of this pronouncement on our related disclosures.

Net Loss per Share Attributab_2

Net Loss per Share Attributable to Common Stockholders (Tables)	9 Months Ended
	Sep. 30, 2024
Earnings Per Share [Abstract]
Computation of Basic and Diluted Net Loss per Share Attributable to Common Stockholders	The following table summarizes the computation of basic and diluted net loss per share attributable to common stockholders of the Company: Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 (In thousands, except share and per (In thousands, except share and per Numerator—basic and diluted: Net loss $ ( 84,126 ) $ ( 51,146 ) $ ( 224,589 ) $ ( 136,887 ) Net loss attributable to common $ ( 84,126 ) $ ( 51,146 ) $ ( 224,589 ) $ ( 136,887 ) Net loss per share attributable to common $ ( 0.98 ) $ ( 0.73 ) $ ( 2.63 ) $ ( 1.97 ) Denominator—basic and diluted: Weighted-average number of common shares 85,433,569 69,855,766 85,307,660 69,645,888
Potential Dilutive Securities Excluded from Computation of Diluted Net Loss per Common Share	The following potentially dilutive securities have been excluded from the computation of diluted weighted-average shares outstanding because such securities have an antidilutive impact due to losses reported (in common stock equivalent shares): September 30, 2024 2023 Options to purchase common stock 12,205,960 10,530,750 Employee Stock Purchase Plan 73,200 35,479 Non-vested restricted stock units (RSUs) 1,461,005 524,236

Fair Value Measurements (Tables

Fair Value Measurements (Tables)	9 Months Ended
	Sep. 30, 2024
Fair Value Disclosures [Abstract]
Schedule of Fair Values of Financial Assets and Liabilities Measured at Fair Value	The table below presents information about the Company’s assets and liabilities that are regularly measured and carried at fair value and indicate the level within the fair value hierarchy of valuation techniques the Company utilized to determine such fair values (in thousands): Fair Value Measurements Using Quoted Prices Significant (unadjusted) Other Significant Total in Active Observable Unobservable Carrying Markets Inputs Inputs Value (Level 1) (Level 2) (Level 3) (In thousands) September 30, 2024 Assets: Cash and cash equivalents $ 133,019 $ 133,019 $ — $ — Short-term investments 256,588 — 256,588 — Long-term investments 10,029 — 10,029 — Total assets $ 399,636 $ 133,019 $ 266,617 $ — December 31, 2023 Assets: Cash and cash equivalents $ 295,394 $ 295,394 $ — $ — Short-term investments 275,304 — 275,304 — Long-term investments 29,829 — 29,829 — Total assets $ 600,527 $ 295,394 $ 305,133 $ —
Summary of Available-for-Sale Securities	The following table summarizes the available-for-sale securities: Amortized Unrealized Unrealized Cost Gains Losses Fair Value (In thousands) September 30, 2024 Commercial paper $ 81,096 $ 44 $ — $ 81,140 Corporate bonds 40,456 17 — 40,473 US Treasury 134,716 259 — 134,975 Federal bonds 9,978 51 — 10,029 $ 266,246 $ 371 $ — $ 266,617 December 31, 2023 Commercial paper $ 160,657 $ 149 $ — $ 160,806 Corporate bonds 47,150 62 — 47,212 US Treasury 68,111 46 — 68,157 Federal bonds 28,998 — ( 40 ) 28,958 $ 304,916 $ 257 $ ( 40 ) $ 305,133

Prepaid Expenses and Other Cu_2

Prepaid Expenses and Other Current Assets (Tables)	9 Months Ended
	Sep. 30, 2024
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract]
Schedule of Prepaid Expenses and Other Current Assets	repaid expenses and other current assets consisted of the following (in thousands): September 30, 2024 December 31, 2023 Prepaid insurance 1,488 807 Interest receivable on investments 1,183 1,227 Prepaid subscription 1,295 769 Prepaid state and local taxes 601 264 Prepaid rent 122 163 Other 218 63 Total prepaid expenses and other current assets $ 4,907 $ 3,293

Accrued Expenses and Other Cu_2

Accrued Expenses and Other Current Liabilities (Tables)	9 Months Ended
	Sep. 30, 2024
Payables and Accruals [Abstract]
Schedule of Accrued Expenses and Other Current Liabilities	Accrued expenses and other current liabilities consisted of the following (in thousands): September 30, 2024 December 31, 2023 Accrued clinical study and trial costs $ 35,692 $ 16,346 Accrued compensation and related costs 11,602 11,172 Accrued professional fees 619 1,450 Accrued milestone costs — 10,000 Other 208 888 Total accrued expenses and other current liabilities $ 48,121 $ 39,856

Stock-Based Compensation (Table

Stock-Based Compensation (Tables)	9 Months Ended
	Sep. 30, 2024
Share-Based Payment Arrangement [Abstract]
Share Based Compensation Expense Related to Issuance of Stock Option Awards to Employees and Non Employees Related to Employee Stock Purchase Plan	The Company recognized stock-based compensation expense related to the issuance of stock option awards and restricted stock units to employees and non-employees and related to the Company’s 2015 Employee Stock Purchase Plan, or ESPP, in the condensed consolidated statements of comprehensive loss as follows: Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Research and development $ 5,886 $ 3,853 $ 14,347 $ 10,329 Selling, general and administrative 6,048 4,468 16,382 12,295 Total $ 11,934 $ 8,321 $ 30,729 $ 22,624
Summary in Compensation Expense by Type of Award	Compensation expense by type of award in the three and nine months ended September 30, 2024 and 2023 was as follows: Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Stock options $ 9,300 $ 7,242 $ 24,285 $ 19,654 RSUs 2,455 990 6,071 2,749 ESPP 179 89 373 221 Total $ 11,934 $ 8,321 $ 30,729 $ 22,624

Stockholders' Equity (Tables)

Stockholders' Equity (Tables)	9 Months Ended
	Sep. 30, 2024
Equity [Abstract]
Summary of Changes in Stockholders' Equity	The following table presents the changes in stockholders’ equity for the three and nine months ended September 30, 2024: (In thousands, except share data) Common Stock Additional Accumulated Accumulated Total Shares Amount Balance as of December 31, 2023 84,826,632 $ 8 $ 1,456,370 $ 218 $ ( 902,400 ) $ 554,196 Stock purchase under ESPP 35,463 — — — — — Stock-based compensation expense — — 8,899 — — 8,899 Unrealized loss on investments — — — ( 974 ) — ( 974 ) Vesting of RSUs 3,750 — — — — — Employee withholdings ESPP — — 309 — — 309 Proceeds from exercise of stock options 113,841 — 1,859 — — 1,859 Net loss — — — — ( 72,400 ) ( 72,400 ) Balance as of March 31, 2024 84,979,686 $ 8 $ 1,467,437 $ ( 756 ) $ ( 974,800 ) $ 491,889 Stock-based compensation expense — — 9,896 — — 9,896 Unrealized loss on investments — — — ( 61 ) — ( 61 ) Vesting of RSUs 1,603 — — — — — Employee withholdings ESPP — — 237 — — 237 Proceeds from exercise of stock options 47,340 — 439 — — 439 Par value adjustment — 1 — — — 1 Net loss — — — — ( 68,063 ) ( 68,063 ) Balance as of June 30, 2024 85,028,629 $ 9 $ 1,478,009 $ ( 817 ) $ ( 1,042,863 ) $ 434,338 Stock-based compensation expense — — 11,934 — — 11,934 Unrealized gains on investments — — — 1,188 — 1,188 Stock purchase under ESPP 29,440 — — — — — Employee withholdings ESPP — — 535 — — 535 Vesting of RSUs 19,057 — — — — — Proceeds from exercise of stock options 208,362 — 2,563 — — 2,563 Net loss — — — — ( 84,126 ) ( 84,126 ) Balance as of September 30, 2024 85,285,488 $ 9 $ 1,493,041 $ 371 $ ( 1,126,989 ) $ 366,432 The following table presents the changes in stockholders’ equity for the three and nine months ended September 30, 2023: (In thousands, except share data) Common Stock Additional Accumulated Accumulated Total Shares Amount Balance as of December 31, 2022 68,111,385 $ 7 $ 1,161,288 $ ( 806 ) $ ( 693,040 ) $ 467,449 Stock purchase under ESPP 16,537 — — — — — Stock-based compensation expense — — 6,238 — — 6,238 Unrealized gain on short-term investments — — — 470 — 470 Vesting of RSUs 5,000 — — — — — Employee withholdings ESPP — — 196 — — 196 Prefunded warrants, exercise 85,998 — — — — — Proceeds from exercise of stock options 276,506 — 2,278 — — 2,278 Net loss — — — — ( 41,126 ) ( 41,126 ) Balance as of March 31, 2023 68,495,426 $ 7 $ 1,170,000 $ ( 336 ) $ ( 734,166 ) $ 435,505 Stock-based compensation expense — — 8,065 — — 8,065 Unrealized loss on short-term investments — — — ( 238 ) — ( 238 ) Vesting of RSUs 1,602 — — — — — Employee withholdings ESPP — — 179 — — 179 Prefunded warrants, exercise 771,133 — ( 1 ) — — ( 1 ) Proceeds from exercise of stock options 163,037 — 1,146 — — 1,146 Net loss — — — — ( 44,615 ) ( 44,615 ) Balance as of June 30, 2023 69,431,198 $ 7 $ 1,179,389 $ ( 574 ) $ ( 778,781 ) $ 400,041 Stock-based compensation expense — — 8,321 — — 8,321 Unrealized gains on short-term investments — — — 119 — 119 Stock purchase under ESPP 18,260 — — — — — Employee withholdings ESPP — — 259 — — 259 Proceeds from exercise of stock options 188,710 — 1,592 — — 1,592 Net loss ( 51,146 ) ( 51,146 ) Balance as of September 30, 2023 69,638,168 $ 7 $ 1,189,561 $ ( 455 ) $ ( 829,927 ) $ 359,186

Nature of Business - Additional

Nature of Business - Additional Information (Detail)	9 Months Ended
	Sep. 30, 2024 Segment
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Number of operating segments	1
State of incorporation	DE

Summary of Significant Accoun_3

Summary of Significant Accounting Policies (Additional Information) (Details) - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023
Accounting Policies [Abstract]
Income Tax Expense Benefit	$ 0	$ 0

Significant Collaborative Res_2

Significant Collaborative Research and License Agreements - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands

1 Months Ended

3 Months Ended

9 Months Ended

12 Months Ended

Dec. 31, 2021

Sep. 30, 2021

Oct. 31, 2017

Aug. 31, 2016

Jul. 31, 2016

Sep. 30, 2024

Jun. 30, 2024

Sep. 30, 2023

Jun. 30, 2022

Sep. 30, 2024

Sep. 30, 2023

Dec. 31, 2016

Mar. 31, 2007

Research And Development Arrangement Contract To Perform For Others [Line Items]

Research and development

$ 70,971

$ 39,087

$ 176,118

$ 107,906

Revenue milestone

12,500

Milestone and license revenue

12,500

$ 0

16,000

$ 0

Incyte Agreement [Member]

Research And Development Arrangement Contract To Perform For Others [Line Items]

Common stock shares purchased

1,421,523

Purchase price per share

$ 24.62

Proceeds from issuance of common stock

$ 35,000

Collaboration receivable

1,700

1,700

Collaboration expense due to counterparty

7,200

7,200

Incyte Agreement [Member] | Maximum [Member]

Research And Development Arrangement Contract To Perform For Others [Line Items]

Potential milestone payments to be made

$ 220,000

Commercialization milestones and tiered royalties

$ 230,000

Incyte Agreement [Member] | Non-Refundable Cash Payments [Member]

Research And Development Arrangement Contract To Perform For Others [Line Items]

Upfront milestone payable

$ 117,000

Allergan License Agreement [Member] | Vitae Pharmaceuticals Inc [Member]

Research And Development Arrangement Contract To Perform For Others [Line Items]

Potential milestone payments to be made

$ 99,000

Aggregate potential milestone payable

$ 70,000

Milestone Expenses Paid

8,000

8,000

Development and regulaotry milestones Expenses

18,000

$ 18,000

License expiration year

10 years

Ucb License Agreement [Member] | U C B Biopharma [Member]

Research And Development Arrangement Contract To Perform For Others [Line Items]

Research and development

31,000

$ 5,000

Milestone paid

$ 15,000

Potential milestone payments to be made

$ 119,500

Aggregate potential milestone payable

$ 250,000

License expiration year

10 years

Milestone payment payable

$ 5,800

License Agreement [Member] | Bayer Pharma Ag [Member] | Maximum [Member]

Research And Development Arrangement Contract To Perform For Others [Line Items]

Aggregate payment obligation

$ 150,000

License Agreement [Member] | Eddingpharm Investment [Member]

Research And Development Arrangement Contract To Perform For Others [Line Items]

Milestone and license revenue

$ 3,500

License Agreement [Member] | Eddingpharm Investment [Member] | Maximum [Member]

Research And Development Arrangement Contract To Perform For Others [Line Items]

Milestone payment payable

$ 10,000

Global and U.S. [Member] | Incyte Agreement [Member]

Research And Development Arrangement Contract To Perform For Others [Line Items]

Development costs associated with clinical trials

45%

Global and U.S. [Member] | Incyte Agreement [Member] | Incyte Corporation [Member]

Research And Development Arrangement Contract To Perform For Others [Line Items]

Development costs associated with clinical trials

55%

Ex-U.S. Countries [Member] | Incyte Agreement [Member]

Research And Development Arrangement Contract To Perform For Others [Line Items]

Development costs associated with clinical trials

100%

Net Loss per Share Attributab_3

Net Loss per Share Attributable to Common Stockholders - Computation of Basic and Diluted Net Loss per Share Attributable to Common Stockholders (Detail) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2024	Sep. 30, 2023
Numerator—basic and diluted:
Net Income (Loss)	$ (84,126)	$ (68,063)	$ (72,400)	$ (51,146)	$ (44,615)	$ (41,126)	$ (224,589)	$ (136,887)
Net loss attributable to common stockholders - basic	(84,126)			(51,146)			(224,589)	(136,887)
Net loss attributable to common stockholders - diluted	$ (84,126)			$ (51,146)			$ (224,589)	$ (136,887)
Net loss per share attributable to common stockholders—basic	$ (0.98)			$ (0.73)			$ (2.63)	$ (1.97)
Net loss per share attributable to common stockholders— diluted	$ (0.98)			$ (0.73)			$ (2.63)	$ (1.97)
Denominator—basic and diluted:
Weighted-average number of common shares used to compute net loss per share attributable to common stockholders—basic	85,433,569			69,855,766			85,307,660	69,645,888
Weighted-average number of common shares used to compute net loss per share attributable to common stockholders—diluted	85,433,569			69,855,766			85,307,660	69,645,888

Net Loss per Share Attributab_4

Net Loss per Share Attributable to Common Stockholders - Potential Dilutive Securities Excluded from Computation of Diluted Net Loss per Common Share (Detail) - shares	9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023
Employee Stock Option
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Anti-dilutive securities excluded from computation of diluted net loss per common share	12,205,960	10,530,750
Employee Stock Purchase Plan [Member]
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Anti-dilutive securities excluded from computation of diluted net loss per common share	73,200	35,479
Non-Vested Restricted Stock Units (RSUs) [Member]
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Anti-dilutive securities excluded from computation of diluted net loss per common share	1,461,005	524,236

Significant Agreements - Additi

Significant Agreements - Additional Information (Detail) - USD ($) $ in Thousands	1 Months Ended		3 Months Ended			9 Months Ended		12 Months Ended
	Oct. 31, 2017	Jul. 31, 2016	Sep. 30, 2024	Sep. 30, 2023	Jun. 30, 2022	Sep. 30, 2024	Sep. 30, 2023	Dec. 31, 2016	Mar. 31, 2007
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Research and development expense			$ 70,971	$ 39,087		$ 176,118	$ 107,906
Allergan License Agreement Member | Vitae Pharmaceuticals Inc [Member]
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Potential milestone payments to be made	$ 99,000
Aggregate potential milestone payable	$ 70,000
License expiration year	10 years
Milestone Expenses Paid			8,000			8,000
Development and regulaotry milestones Expenses			18,000			$ 18,000
UCB License Agreement [Member] | UCB Biopharma [Member]
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Potential milestone payments to be made		$ 119,500
Aggregate potential milestone payable		$ 250,000
License expiration year		10 years
Research and development expense			$ 31,000					$ 5,000
Milestone payment payable					$ 5,800
License Agreement [Member] | Bayer Pharma AG [Member] | Maximum [Member]
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Aggregate payment obligation									$ 150,000

Other Receivables (Additional I

Other Receivables (Additional Information) (Details) $ in Millions	9 Months Ended
	Sep. 30, 2024 USD ($)
Other Receivables, Net, Current [Abstract]
Refundable fee payble	$ 6.1
Payment for fully-refundable fee	6.1
Receivables Related to Achieved Milestones	$ 3.5

Fair Value Measurements - Sched

Fair Value Measurements - Schedule of Fair Values of Financial Assets and Liabilities Measured at Fair Value (Detail) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Short-term investments	$ 256,588	$ 275,304
Long-term investments	10,029	29,829
Carrying Value [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	133,019	295,394
Short-term investments	256,588	275,304
Long-term investments	10,029	29,829
Total assets	399,636	600,527
Quoted Prices (Unadjusted) in Active Markets Level 1 [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	133,019	295,394
Total assets	133,019	295,394
Significant Other Observable Inputs Level 2 [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Short-term investments	256,588	275,304
Long-term investments	10,029	29,829
Total assets	$ 266,617	$ 305,133

Fair Value Measurements - Addit

Fair Value Measurements - Additional Information (Detail) - USD ($)	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Fair Value Measurements [Line Items]
Realized gains or losses recognized on the sale or maturity of available-for-sale securities	$ 0	$ 0	$ 0	$ 0

Fair Value Measurements - Summa

Fair Value Measurements - Summary of Available-for-Sale Securities (Detail) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	$ 266,246	$ 304,916
Unrealized Gains	371	257
Unrealized Losses	0	(40)
Fair Value	266,617	305,133
Commercial Paper [Member]
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	81,096	160,657
Unrealized Gains	44	149
Fair Value	81,140	160,806
Corporate Bonds [Member]
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	40,456	47,150
Unrealized Gains	17	62
Fair Value	40,473	47,212
U.S. Treasury [Member]
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	134,716	68,111
Unrealized Gains	259	46
Fair Value	134,975	68,157
Federal Bonds [Member]
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	9,978	28,998
Unrealized Gains	51
Unrealized Losses		(40)
Fair Value	$ 10,029	$ 28,958

Prepaid Expenses and Other Cu_3

Prepaid Expenses and Other Current Assets - Schedule of Prepaid Expenses and Other Current Assets (Detail) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract]
Prepaid insurance	$ 1,488	$ 807
Interest receivable on investments	1,183	1,227
Prepaid subscriptions	1,295	769
Prepaid state and local taxes	601	264
Prepaid rent	122	163
Other	218	63
Total prepaid expenses and other current assets	$ 4,907	$ 3,293

Accrued Expenses and Other Cu_3

Accrued Expenses and Other Current Liabilities - Schedule of Accrued Expenses and Other Current Liabilities (Detail) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Payables and Accruals [Abstract]
Accrued clinical study and trial costs	$ 35,692	$ 16,346
Accrued compensation and related costs	11,602	11,172
Accrued professional fees	619	1,450
Accrued milestone costs	0	10,000
Other	208	888
Total accrued expenses and other current liabilities	$ 48,121	$ 39,856

Stock-Based Compensation - Addi

Stock-Based Compensation - Additional Information (Detail) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023	Jan. 01, 2024	Dec. 31, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based compensation expense, total	$ 11,934	$ 8,321	$ 30,729	$ 22,624
Proceeds from stock option exercises			4,861	5,016
Restricted Stock Units [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based compensation expense, total	$ 2,455	$ 990	$ 6,071	$ 2,749
2015 Plan [Member] | Maximum [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares available for issuance	3,862,670		3,862,670		3,393,065
2015 and 2007 [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized compensation expense related to stock option and RSUs	$ 93,300		$ 93,300
Weighted average period to recognize compensation expense			2 years 7 months 9 days
2023 Inducement Plan [Member] | Maximum [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares available for issuance	76,763		76,763
Common stock reserved for issuance						1,100,000

Stock-Based Compensation - Shar

Stock-Based Compensation - Share Based Compensation Expense Related to Issuance of Stock Option Awards to Employees and Non Employees Related to Employee Stock Purchase Plan (Detail) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based compensation expense, total	$ 11,934	$ 8,321	$ 30,729	$ 22,624
Research and Development [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based compensation expense, total	5,886	3,853	14,347	10,329
Selling, General and Administrative [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based compensation expense, total	$ 6,048	$ 4,468	$ 16,382	$ 12,295

Stock-Based Compensation - Comp

Stock-Based Compensation - Compensation Expense by Type of Award (Detail) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based compensation expense, total	$ 11,934	$ 8,321	$ 30,729	$ 22,624
Stock options [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based compensation expense, total	9,300	7,242	24,285	19,654
Restricted Stock Units [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based compensation expense, total	2,455	990	6,071	2,749
Employee Stock Purchase Plan [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share based compensation expense, total	$ 179	$ 89	$ 373	$ 221

Stockholders' Equity - Summary

Stockholders' Equity - Summary of Changes in Stockholders' Equity (Detail) - USD ($) $ in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2024	Sep. 30, 2023
Class of Stock [Line Items]
Beginning balance	$ 434,338	$ 491,889	$ 554,196	$ 400,041	$ 435,505	$ 467,449	$ 554,196	$ 467,449
Stock-based compensation expense	11,934	9,896	8,899	8,321	8,065	6,238
Unrealized loss on investments	1,188	(61)	(974)	119	(238)	470	371	351
Employee withholdings ESPP	535	237	309	259	$ 179	196
Prefunded warrants, exercise, shares					(1)
Proceeds from exercise of stock options	2,563	439	1,859	1,592	$ 1,146	2,278
Par value adjustment		1
Net Income (Loss)	(84,126)	(68,063)	(72,400)	(51,146)	(44,615)	(41,126)	(224,589)	(136,887)
Ending balance	366,432	434,338	491,889	359,186	400,041	435,505	366,432	359,186
Common Stock [Member]
Class of Stock [Line Items]
Beginning balance	$ 9	$ 8	$ 8	$ 7	$ 7	$ 7	$ 8	$ 7
Beginning balance, shares	85,028,629	84,979,686	84,826,632	69,431,198	68,495,426	68,111,385	84,826,632	68,111,385
Stock purchase under ESPP, shares	29,440		35,463	18,260		16,537
Vesting of RSUs, shares	19,057	1,603	3,750		1,602	5,000
Prefunded warrants, exercise, shares					771,133	85,998
Proceeds from exercise of stock options, shares	208,362	47,340	113,841	188,710	163,037	276,506
Par value adjustment		$ 1
Ending balance	$ 9	$ 9	$ 8	$ 7	$ 7	$ 7	$ 9	$ 7
Ending balance, shares	85,285,488	85,028,629	84,979,686	69,638,168	69,431,198	68,495,426	85,285,488	69,638,168
Additional Paid-In Capital [Member]
Class of Stock [Line Items]
Beginning balance	$ 1,478,009	$ 1,467,437	$ 1,456,370	$ 1,179,389	$ 1,170,000	$ 1,161,288	$ 1,456,370	$ 1,161,288
Stock-based compensation expense	11,934	9,896	8,899	8,321	8,065	6,238
Employee withholdings ESPP	535	237	309	259	$ 179	196
Prefunded warrants, exercise, shares					(1)
Proceeds from exercise of stock options	2,563	439	1,859	1,592	$ 1,146	2,278
Ending balance	1,493,041	1,478,009	1,467,437	1,189,561	1,179,389	1,170,000	1,493,041	1,189,561
Accumulated Other Comprehensive(Loss) / Income [Member]
Class of Stock [Line Items]
Beginning balance	(817)	(756)	218	(574)	(336)	(806)	218	(806)
Unrealized loss on investments	1,188	(61)	(974)	119	(238)	470
Ending balance	371	(817)	(756)	(455)	(574)	(336)	371	(455)
Accumulated Deficit [Member]
Class of Stock [Line Items]
Beginning balance	(1,042,863)	(974,800)	(902,400)	(778,781)	(734,166)	(693,040)	(902,400)	(693,040)
Net Income (Loss)	(84,126)	(68,063)	(72,400)	(51,146)	(44,615)	(41,126)
Ending balance	$ (1,126,989)	$ (1,042,863)	$ (974,800)	$ (829,927)	$ (778,781)	$ (734,166)	$ (1,126,989)	$ (829,927)

Stockholders' Equity - Addition

Stockholders' Equity - Additional Information (Detail) - USD ($) $ in Millions	1 Months Ended
	May 31, 2023	Sep. 30, 2024	Dec. 31, 2021
Pre Funded Warrants [Member]
Class Of Stock [Line Items]
Warrants issued (in shares)			1,142,856
Number of pre funded warrants exercised for common stock		285,714
Cowen and Company LLC [Member] | At The Market Equity Offering Sales Agreement [Member]
Class Of Stock [Line Items]
Proceeds from issuance of common stock	$ 200

Commitments and Contingencies (

Commitments and Contingencies (Additional Information) (Details) $ in Thousands	Sep. 30, 2024 USD ($)
Commitments and Contingencies Disclosure [Abstract]
Contingent liabilities	$ 0

Subsequent Events (Additional I

Subsequent Events (Additional Information) (Details) - Subsequent Event [Member] $ in Millions	1 Months Ended
	Oct. 31, 2024 USD ($)
Subsequent Event [Line Items]
Percentage of net sales	13.80%
Upfront Payment	$ 350
Capped funded amount	$ 822.5
Percentage of Funded Amount	2.35%
lease expiration date	Aug. 31, 2028