0001689548-25-000040 | 10-K | XBRL Viewer | Praxis Precision Medicines INC

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Stock-Based Compensation - Summary of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]
Stock-based compensation expense	$ 41,360	$ 24,857
Research and development
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]
Stock-based compensation expense	15,620	8,055
General and administrative
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]
Stock-based compensation expense	$ 25,740	$ 16,802

Summary of Significant Accounting Policies - Summary of Cash, Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Accounting Policies [Abstract]
Cash and cash equivalents	$ 215,372	$ 81,300
Restricted cash	416	416
Total cash, cash equivalents and restricted cash as shown on the consolidated statements of cash flows	$ 215,788	$ 81,716	$ 62,031

Summary of Significant Accounting Policies - Narrative (Details) - USD ($)	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Accounting Policies [Abstract]
Total available-for-sale securities, estimated fair value	$ 254,156,000	$ 0
Impairment losses	$ 0	0
Expected dividend yield	0.00%
Foreign currency gain (loss)	$ 0	$ 0

- Definition

Amount of investment in debt security measured at fair value with change in fair value recognized in other comprehensive income (available-for-sale).

+ References

Reference 1: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 103
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-103

Reference 2: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 100
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-100

Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (bbb)(2)(i)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 820
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-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (bbb)(2)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 5: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (bbb)(1)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (aa)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 45
-Paragraph 1
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481830/320-10-45-1

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 942
-SubTopic 210
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.9-03(6))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147478546/942-210-S99-1

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 326
-SubTopic 30
-Name Accounting Standards Codification
-Section 45
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479130/326-30-45-1

+ Details

Restricted Cash	12 Months Ended
Restricted Cash	Dec. 31, 2024
Cash and Cash Equivalents [Abstract]
Restricted Cash	Restricted Cash As of December 31, 2024 and 2023, the Company had restricted cash of $0.4 million and $0.4 million, respectively, held as letters of credit for the benefit of its current landlord. The Company's Boston, Massachusetts sublease expires on January 31, 2026, and the related letter of credit of $0.4 million was classified within other non-current assets on the consolidated balance sheet as of December 31, 2024 and 2023.

Consolidated Balance Sheets - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Current assets:
Cash and cash equivalents	$ 215,372	$ 81,300
Marketable securities	177,195	0
Prepaid expenses and other current assets	11,805	3,580
Total current assets	404,372	84,880
Long-term marketable securities	76,961	0
Property and equipment, net	230	588
Operating lease right-of-use assets	1,131	2,064
Other non-current assets	416	416
Total assets	483,110	87,948
Current liabilities:
Accounts payable	12,528	5,815
Accrued expenses	23,763	7,416
Current operating lease liabilities	1,259	1,126
Current portion of deferred revenue	0	1,392
Total current liabilities	37,550	15,749
Long-term liabilities:
Non-current portion of operating lease liabilities	110	1,369
Non-current portion of deferred revenue	0	1,161
Total liabilities	37,660	18,279
Commitments and contingencies (Note 8)
Stockholders’ equity:
Preferred stock, $0.0001 par value; 10,000,000 shares authorized and no shares issued or outstanding as of December 31, 2024 and December 31, 2023	0	0
Common stock, $0.0001 par value; 150,000,000 shares authorized; 19,422,358 shares issued and outstanding as of December 31, 2024, and 8,791,877 shares issued and outstanding as of December 31, 2023	14	13
Additional paid-in capital	1,281,522	723,577
Accumulated other comprehensive gain	654	0
Accumulated deficit	(836,740)	(653,921)
Total stockholders’ equity	445,450	69,669
Total liabilities and stockholders’ equity	$ 483,110	$ 87,948

Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands	Total	Follow-On Offering	At-The-Market Offering	License And Collaboration Agreement	Common Stock	Common Stock Follow-On Offering	Common Stock At-The-Market Offering	Common Stock License And Collaboration Agreement	Additional Paid-In Capital	Additional Paid-In Capital Follow-On Offering	Additional Paid-In Capital At-The-Market Offering	Additional Paid-In Capital License And Collaboration Agreement	Accumulated Deficit	Accumulated Other Comprehensive Gain (Loss)
Beginning balance (in shares) at Dec. 31, 2022					3,292,163
Beginning balance at Dec. 31, 2022	$ 76,106				$ 5				$ 606,918				$ (530,644)	$ (173)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	7,593								7,593
Issuance of common stock from at-the-market and follow-on public offerings (in shares)					560,253
Issuance of common stock from at-the-market and follow-on public offerings	18,096					$ 1			18,095
Vesting of restricted stock units (in shares)					11,516
Shares withheld for taxes for vesting of restricted stock units (in shares)					(2,875)
Shares withheld for taxes for vesting of restricted stock units	(127)								(127)
Issuance of common stock from exercise of stock options (in shares)					2,970
Issuance of common stock upon exercise of stock options	101								101
Change in unrealized gain (loss) on marketable securities, net of tax	154													154
Net loss	(37,455)												(37,455)
Ending balance (in shares) at Mar. 31, 2023					3,864,027
Ending balance at Mar. 31, 2023	64,468				$ 6				632,580				(568,099)	(19)
Beginning balance (in shares) at Dec. 31, 2022					3,292,163
Beginning balance at Dec. 31, 2022	76,106				$ 5				606,918				(530,644)	(173)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Change in unrealized gain (loss) on marketable securities, net of tax	173
Net loss	$ (123,277)
Ending balance (in shares) at Dec. 31, 2023	8,791,877				8,791,877
Ending balance at Dec. 31, 2023	$ 69,669				$ 13				723,577				(653,921)	0
Beginning balance (in shares) at Mar. 31, 2023					3,864,027
Beginning balance at Mar. 31, 2023	64,468				$ 6				632,580				(568,099)	(19)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	5,775								5,775
Issuance of common stock from at-the-market and follow-on public offerings (in shares)						4,296,646	392,541
Issuance of common stock from at-the-market and follow-on public offerings		$ 63,439	$ 6,032			$ 6	$ 1			$ 63,433	$ 6,031
Issuance of common stock under employee stock purchase plan (in shares)					15,663
Issuance of common stock under employee stock purchase plan	208								208
Vesting of restricted stock units (in shares)					720
Shares withheld for taxes for vesting of restricted stock units (in shares)					(249)
Shares withheld for taxes for vesting of restricted stock units	(4)								(4)
Change in unrealized gain (loss) on marketable securities, net of tax	19													19
Net loss	(34,312)												(34,312)
Ending balance (in shares) at Jun. 30, 2023					8,569,348
Ending balance at Jun. 30, 2023	105,625				$ 13				708,023				(602,411)	0
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	5,763								5,763
Vesting of restricted stock units (in shares)					233
Shares withheld for taxes for vesting of restricted stock units (in shares)					(70)
Shares withheld for taxes for vesting of restricted stock units	(1)								(1)
Issuance of common stock from exercise of stock options (in shares)					311
Issuance of common stock upon exercise of stock options	1								1
Net loss	(24,632)												(24,632)
Ending balance (in shares) at Sep. 30, 2023					8,569,822
Ending balance at Sep. 30, 2023	86,756				$ 13				713,786				(627,043)	0
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	5,726								5,726
Issuance of common stock from at-the-market and follow-on public offerings (in shares)					212,453
Issuance of common stock from at-the-market and follow-on public offerings	3,964								3,964
Issuance of common stock under employee stock purchase plan (in shares)					9,027
Issuance of common stock under employee stock purchase plan	106								106
Vesting of restricted stock units (in shares)					824
Shares withheld for taxes for vesting of restricted stock units (in shares)					(249)
Shares withheld for taxes for vesting of restricted stock units	(5)								(5)
Net loss	$ (26,878)												(26,878)
Ending balance (in shares) at Dec. 31, 2023	8,791,877				8,791,877
Ending balance at Dec. 31, 2023	$ 69,669				$ 13				723,577				(653,921)	0
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	14,475								14,475
Issuance of common stock from at-the-market and follow-on public offerings (in shares)						3,802,025	209,852	443,253
Issuance of common stock from at-the-market and follow-on public offerings		161,649	6,169	$ 17,265						161,649	6,169	$ 17,265
Vesting of restricted stock units (in shares)					11,095
Shares withheld for taxes for vesting of restricted stock units (in shares)					(3,689)
Shares withheld for taxes for vesting of restricted stock units	(137)								(137)
Issuance of common stock from exercise of stock options (in shares)					3,634
Issuance of common stock upon exercise of stock options	143								143
Change in unrealized gain (loss) on marketable securities, net of tax	3													3
Net loss	(39,553)												(39,553)
Ending balance (in shares) at Mar. 31, 2024					13,258,047
Ending balance at Mar. 31, 2024	$ 229,683				$ 13				923,141				(693,474)	3
Beginning balance (in shares) at Dec. 31, 2023	8,791,877				8,791,877
Beginning balance at Dec. 31, 2023	$ 69,669				$ 13				723,577				(653,921)	0
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock from exercise of stock options (in shares)	45,177
Change in unrealized gain (loss) on marketable securities, net of tax	$ 654
Net loss	$ (182,819)
Ending balance (in shares) at Dec. 31, 2024	19,422,358				19,422,358
Ending balance at Dec. 31, 2024	$ 445,450				$ 14				1,281,522				(836,740)	654
Beginning balance (in shares) at Mar. 31, 2024					13,258,047
Beginning balance at Mar. 31, 2024	229,683				$ 13				923,141				(693,474)	3
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	5,878								5,878
Issuance of common stock from at-the-market and follow-on public offerings (in shares)						3,849,558	622,123
Issuance of common stock from at-the-market and follow-on public offerings		215,988	0			$ 1				215,987
Issuance of common stock under employee stock purchase plan (in shares)					25,146
Issuance of common stock under employee stock purchase plan	311								311
Vesting of restricted stock units (in shares)					726
Shares withheld for taxes for vesting of restricted stock units (in shares)					(242)
Shares withheld for taxes for vesting of restricted stock units	(13)								(13)
Issuance of common stock from exercise of stock options (in shares)					148
Issuance of common stock upon exercise of stock options	4								4
Change in unrealized gain (loss) on marketable securities, net of tax	(74)													(74)
Net loss	(32,677)												(32,677)
Ending balance (in shares) at Jun. 30, 2024					17,755,506
Ending balance at Jun. 30, 2024	419,100				$ 14				1,145,308				(726,151)	(71)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	12,432								12,432
Issuance of common stock from at-the-market and follow-on public offerings (in shares)							25,189
Issuance of common stock from at-the-market and follow-on public offerings			$ 1,442								$ 1,442
Vesting of restricted stock units (in shares)					51
Shares withheld for taxes for vesting of restricted stock units (in shares)					(20)
Shares withheld for taxes for vesting of restricted stock units	(1)								(1)
Issuance of common stock from exercise of stock options (in shares)					4,971
Issuance of common stock upon exercise of stock options	201								201
Change in unrealized gain (loss) on marketable securities, net of tax	1,402													1,402
Net loss	(51,910)												(51,910)
Ending balance (in shares) at Sep. 30, 2024					17,785,697
Ending balance at Sep. 30, 2024	382,666				$ 14				1,159,382				(778,061)	1,331
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	8,575								8,575
Issuance of common stock from at-the-market and follow-on public offerings (in shares)					36,424	1,588,611
Issuance of common stock from at-the-market and follow-on public offerings	1,741	$ 111,748							1,741	$ 111,748
Issuance of common stock under employee stock purchase plan (in shares)					6,235
Issuance of common stock under employee stock purchase plan	233								233
Vesting of restricted stock units (in shares)					7,603
Shares withheld for taxes for vesting of restricted stock units (in shares)					(2,212)
Shares withheld for taxes for vesting of restricted stock units	(157)								(157)
Change in unrealized gain (loss) on marketable securities, net of tax	(677)													(677)
Net loss	$ (58,679)												(58,679)
Ending balance (in shares) at Dec. 31, 2024	19,422,358				19,422,358
Ending balance at Dec. 31, 2024	$ 445,450				$ 14				$ 1,281,522				$ (836,740)	$ 654

Insider Trading Policies and Procedures	12 Months Ended
Insider Trading Policies and Procedures	Dec. 31, 2024
Insider Trading Policies and Procedures [Line Items]
Insider Trading Policies and Procedures Adopted	true

Employee Benefit Plan (Details) - USD ($) $ in Millions	12 Months Ended
Employee Benefit Plan (Details) - USD ($) $ in Millions	Dec. 31, 2024	Dec. 31, 2023
Retirement Benefits [Abstract]
Defined contribution plan, employer discretionary contribution amount	$ 1.2	$ 1.0

Common Stock and Preferred Stock

12 Months Ended

Dec. 31, 2024

Stockholders' Equity Note [Abstract]

Common Stock and Preferred Stock

Common Stock

In November 2021, the Company entered into an Open Market Sale Agreement (the "2021 Sales Agreement") with Jefferies LLC ("Jefferies"), to provide for the offering, issuance and sale of up to an aggregate amount of $125.0 million of common stock from time to time in at-the-market offerings for which Jefferies acted as sales agent. The Company terminated the 2021 Sales Agreement in June 2023. During the year ended December 31, 2023, the Company issued and sold an aggregate of 952,794 shares under the 2021 Sales Agreement for aggregate net proceeds of $24.1 million, after deducting commissions and offering expenses payable by the Company.

In December 2023, the Company entered into an Open Market Sale Agreement (the "2023 Sales Agreement"), with Jefferies, to provide for the offering, issuance and sale of up to an aggregate amount of $75.0 million of common stock from time to time in at-the-market offerings. The 2023 Sales Agreement was terminated in January 2024. During the year ended December 31, 2024, the Company issued and sold an aggregate of 192,190 shares under the 2023 Sales Agreement for aggregate net proceeds of $5.3 million, after deducting commissions and offering expenses payable by the Company.

In March 2024, the Company entered into an Open Market Sale Agreement (the "March 2024 Sales Agreement"), with Jefferies, to provide for the offering, issuance, and sale of up to an aggregate amount of $150.0 million of common stock from time to time in at-the-market offerings. During the year ended December 31, 2024, the Company issued and sold an aggregate of 1,614,975 shares under the March 2024 Sales Agreement for aggregate net proceeds of $113.1 million, after deducting commissions and offering expenses payable by the Company.

In December 2024, the Company entered into an amendment to the March Sales Agreement with Jefferies, to provide for the offering, issuance, and sale of up to an aggregate amount of $250.0 million of common stock from time to time in at-the-market offerings. During the year ended December 31, 2024, the Company issued and sold an aggregate of 16,487 shares under the amended March 2024 Sales Agreement for aggregate net proceeds of $1.0 million, after deducting commissions and offering expenses payable by the Company.

As of December 31, 2024 and 2023, the authorized capital stock of the Company included 150,000,000 shares of common stock, $0.0001 par value, pursuant to the Amended and Restated Certificate of Incorporation effective upon the completion of the IPO. Holders of such shares of common stock have the exclusive right to vote for the election of the Company's directors and are entitled to one vote per share. In the event of the voluntary or involuntary liquidation, dissolution or winding up of the Company, the holders of common stock are entitled to a pro rata distribution of the Company's net assets. Dividends may be declared and paid to such holders only when, as, and if declared by the Board or an authorized committee thereof.

As of December 31, 2024, the Company did not hold any treasury shares.

Follow-On Public Offerings

June 2023 Public Offering

On June 21, 2023, the Company completed a public offering of: (i) an aggregate of 4,296,646 shares of its common stock at a public offering price of $14.25 per share, including the underwriters’ full exercise of their option to purchase 619,979 additional shares of common stock, and (ii) pre-funded warrants to purchase 470,000 shares of common stock at a public offering price of $14.2485 per share of common stock underlying the warrants. The purchase price per share for each pre-funded warrant represents the per share offering price for the common stock, less the $0.0015 per share exercise price for each underlying share. Total net proceeds generated from the offering were approximately $63.4 million, after deducting underwriting discounts, commissions and other offering expenses payable by the Company.

During the year ended December 31, 2024, all pre-funded warrants associated with this offering were exercised via a cashless exercise, resulting in 469,981 shares of common stock issued. No cash proceeds associated with the exercise were received by the Company. As of December 31, 2024, there were no pre-funded warrants associated with this offering outstanding.

January 2024 Public Offering

On January 16, 2024, the Company completed a public offering of: (i) an aggregate of 3,802,025 shares of its common stock at a public offering price of $35.50 per share, including the underwriters' full exercise of their option to purchase 633,750 additional shares of common stock, and (ii) pre-funded warrants to purchase 1,056,725 shares of common stock at a public offering price of $35.4999 per share of common stock underlying the warrants. The purchase price per share of each pre-funded warrant represents the per share offering price for the common stock, less the $0.0001 per share exercise price of each underlying share. Total net proceeds generated from the offering were approximately $161.6 million, after deducting underwriting discounts, commissions and other offering expenses payable by the Company.

The pre-funded warrants are exercisable at any time on or after the date of issuance at the option of the holder, subject to a beneficial ownership blocker that may limit exercisability. No holder may exercise any portion of the warrants that would cause the aggregate number of shares of common stock beneficially owned by such holder, together with its affiliates, to exceed 4.99% (or 9.99%) of the issued and outstanding common stock. A holder of a pre-funded warrant may increase or decrease this percentage up to 19.99% by providing at least 61 days’ prior notice to the Company. The pre-funded warrants do not expire. The pre-funded warrants may be settled through either physical or net share settlement. Following the occurrence of certain fundamental transactions, the holders of the pre-funded warrants have the right to receive upon exercise of the warrants the same amount and kind of securities, cash, or property as they would have been entitled to receive if they had been holders of the common shares issuable under the warrants immediately prior to such transaction. During the year ended December 31, 2024, 152,145 pre-funded warrants were exercised via a cashless exercise, resulting in 152,142 shares of common stock issued. No cash proceeds associated with the exercise were received by the Company. As of December 31, 2024, a total of 904,580 pre-funded warrants associated with this offering remained outstanding.

April 2024 Public Offering

On April 2, 2024 the Company completed a public offering of: (i) an aggregate of 3,849,558 shares of its common stock at a public offering price of $56.50 per share, including the underwriters' full exercise of their option to purchase 530,973 additional shares of common stock, and (ii) pre-funded warrants to purchase 221,238 shares of common stock at a public offering price of $56.4999 per share of common stock underlying the warrants. The purchase price per share for each pre-funded warrant represents the per share offering price for the common stock, less the $0.0001 per share exercise price of each underlying share. Total net proceeds generated from the offering were approximately $216.0 million, after deducting underwriting discounts, commissions and other offering expenses payable by the Company.

The pre-funded warrants are exercisable at any time on or after the date of issuance at the option of the holder, subject to a beneficial ownership blocker that may limit exercisability. No holder may exercise any portion of the warrants that would cause the aggregate number of shares of common stock beneficially owned by such holder, together with its affiliates, to exceed 4.99% (or 9.99%) of the issued and outstanding common stock. A holder of a pre-funded warrant may increase or decrease this percentage up to 19.99% by providing at least 61 days’ prior notice to the Company. The pre-funded warrants do not expire. The pre-funded warrants may be settled through either physical or net share settlement. Following the occurrence of certain fundamental transactions, the holders of the pre-funded warrants have the right to receive upon exercise of the warrants the same amount and kind of securities, cash, or property as they would have been entitled to receive if they had been holders of the common shares issuable under the warrants immediately prior to such transaction. As of December 31, 2024, none of the pre-funded warrants had been exercised and all remained outstanding.

The Company determined that the pre-funded warrants related to the June 2023, January 2024, and April 2024 public offerings are freestanding financial instruments because they are both legally detachable and separately exercisable from the common stock sold in the offering. As such, the Company evaluated the pre-funded warrants to determine whether they represent instruments that require liability classification pursuant to the guidance in ASC 480. However, the Company concluded that the pre-funded warrants are not a liability within the scope of ASC 480 due to their characteristics. Further, the Company determined that the pre-funded warrants do not meet the definition of a derivative under ASC 815 because they do not meet the criteria regarding no or little initial net investment. Accordingly, the Company assessed the pre-funded warrants relative to the guidance in ASC No. 815-40, Contracts in Entity’s Own Equity, to determine the appropriate treatment. The Company concluded that

the pre-funded warrants are both indexed to its own stock and meet all other conditions for equity classification. Accordingly, the Company has classified the pre-funded warrants as permanent equity.

Shares Reserved for Future Issuance

The Company had reserved the following shares of common stock for future issuance:


	December 31,
	2024	2023
Shares reserved for exercise of outstanding stock options	1,809,343	666,163
Shares reserved for exercise of pre-funded warrants related to the June 2023 Financing	—	470,000
Shares reserved for exercise of pre-funded warrants related to the January 2024 Financing	904,580	—
Shares reserved for exercise of pre-funded warrants related to the April 2024 Financing	221,238	—
Shares reserved for future awards under the 2020 Stock Option and Incentive Plan	91,343	148,264
Shares reserved for future awards under the 2020 Employee Stock Purchase Plan	109,648	53,111
Shares reserved for future awards under the 2024 Inducement Plan	112,774	—
Shares reserved for vesting of restricted stock units	224,473	47,145
Total shares of authorized common stock reserved for future issuance	3,473,399	1,384,683

Preferred Stock

As of December 31, 2024 and 2023, the Company was authorized to issue 10,000,000 shares of undesignated preferred stock, $0.0001 par value, in one or more series, and is authorized to fix the powers, designations, preferences and relative participating option or other rights thereof, including dividend rights, conversion rights, voting rights, redemption rights, liquidation preferences and the number of shares constituting any series, without any further vote or action by the Company's shareholders. As of December 31, 2024 and 2023, the Company had no shares of undesignated preferred stock issued or outstanding.

- Definition

The entire disclosure for equity.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-13

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-13

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 14
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-14

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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-Paragraph 2
-Subparagraph (a)
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-URI https://asc.fasb.org/1943274/2147477968/946-235-50-2

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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-Paragraph 2
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-URI https://asc.fasb.org/1943274/2147477968/946-235-50-2

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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-URI https://asc.fasb.org/1943274/2147478448/946-505-50-6

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org/1943274/2147480237/815-40-50-6

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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-Paragraph 1
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org/1943274/2147480678/235-10-S99-1

Reference 10: http://fasb.org/us-gaap/role/ref/legacyRef
-Topic 505
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-URI https://asc.fasb.org/505/tableOfContent

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
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Reference 13: http://www.xbrl.org/2003/role/disclosureRef
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Reference 14: http://www.xbrl.org/2003/role/disclosureRef
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Reference 15: http://www.xbrl.org/2003/role/disclosureRef
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Reference 16: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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Reference 17: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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Reference 18: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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Reference 19: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Section 50
-Paragraph 18
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-18

+ Details

Commitments and Contingencies - Operating Lease, Consolidated Balance Sheet (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Commitments and Contingencies Disclosure [Abstract]
Operating lease right-of-use assets	$ 1,131	$ 2,064
Current operating lease liabilities	1,259	1,126
Non-current portion of operating lease liabilities	110	1,369
Total lease liabilities	$ 1,369	$ 2,495

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Dec. 31, 2024
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

Segment Information - Schedule of Segment Reporting Information, by Segment (Details) - USD ($) $ in Thousands	3 Months Ended								12 Months Ended
	Dec. 31, 2024	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Dec. 31, 2023	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Dec. 31, 2024	Dec. 31, 2023
Segment Reporting Information [Line Items]
Collaboration revenue									$ 8,553	$ 2,447
Stock-based compensation expense									41,360	24,857
Depreciation									358	432
Net loss	$ (58,679)	$ (51,910)	$ (32,677)	$ (39,553)	$ (26,878)	$ (24,632)	$ (34,312)	$ (37,455)	(182,819)	(123,277)
Tenacia
Segment Reporting Information [Line Items]
Sale of stock, value transferred in excess of consideration received									2,500
Reportable Segment
Segment Reporting Information [Line Items]
Collaboration revenue									8,553	2,447
Personnel-related expenses									42,853	33,160
Stock-based compensation expense									41,361	24,857
Depreciation									358	432
Other segment expenses									23,572	18,986
Interest income									17,346	3,096
Net loss									(182,819)	(123,277)
Reportable Segment \| Ulixacaltamide
Segment Reporting Information [Line Items]
Program-specific expenses									69,172	19,464
Reportable Segment \| Vormatrigine
Segment Reporting Information [Line Items]
Program-specific expenses									18,360	6,275
Reportable Segment \| Relutrigine
Segment Reporting Information [Line Items]
Program-specific expenses									6,273	3,996
Reportable Segment \| Elsunersen
Segment Reporting Information [Line Items]
Program-specific expenses									2,135	14,595
Reportable Segment \| Other Early State Assets
Segment Reporting Information [Line Items]
Program-specific expenses									$ 4,634	$ 7,055

Summary of Significant Accounting Policies - Property and Equipment Estimated Useful Life (Details)	Dec. 31, 2024
Office furniture and equipment
Property, Plant and Equipment [Line Items]
Estimated Useful Life	5 years
Laboratory equipment
Property, Plant and Equipment [Line Items]
Estimated Useful Life	3 years
Computer equipment
Property, Plant and Equipment [Line Items]
Estimated Useful Life	3 years

Net Loss per Share - Summary of antidilutive securities excluded from computation of earnings per share (Details) - shares	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities excluded from computation of earnings per share (in shares)	2,041,131	738,280
Outstanding stock options
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities excluded from computation of earnings per share (in shares)	1,809,343	666,163
Unvested restricted stock units
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities excluded from computation of earnings per share (in shares)	224,473	47,145
Potential shares issuable under the ESPP
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities excluded from computation of earnings per share (in shares)	7,315	24,972

Common Stock and Preferred Stock - Narrative (Details) $ / shares in Units, $ in Thousands				1 Months Ended				12 Months Ended
	Apr. 02, 2024 USD ($) $ / shares shares	Jan. 16, 2024 USD ($) $ / shares shares	Jun. 21, 2023 USD ($) $ / shares shares	Dec. 31, 2024 USD ($) vote $ / shares shares	Mar. 31, 2024 USD ($)	Dec. 31, 2023 USD ($) $ / shares shares	Nov. 30, 2021 USD ($)	Dec. 31, 2024 USD ($) vote $ / shares shares	Dec. 31, 2023 USD ($) $ / shares shares
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Common stock, authorized (in shares)				150,000,000		150,000,000		150,000,000	150,000,000
Common stock, par value (in USD per share) \| $ / shares				$ 0.0001		$ 0.0001		$ 0.0001	$ 0.0001
Number of votes per share \| vote				1				1
Treasury shares (in shares)				0				0
Preferred stock, authorized (in shares)				10,000,000		10,000,000		10,000,000	10,000,000
Preferred stock, par value (in USD per share) \| $ / shares				$ 0.0001		$ 0.0001		$ 0.0001	$ 0.0001
Preferred stock, issued (in shares)				0		0		0	0
Preferred stock, outstanding (in shares)				0		0		0	0
Pre-Funded Warrant \| Maximum
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Percentage of ownership held by sole owner								4.99%
Percentage of ownership held by sole owner and affiliates								9.99%
Change in percentage of ownership								19.99%
Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Notice of change, term								61 days
Follow-On Offering
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Sale of stock, net of discounts, commissions and offering expenses \| $							$ 125,000
Proceeds from stock offerings, net of issuance costs \| $			$ 63,400					$ 377,636	$ 63,439
2021 Sales Agreement
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Shares sold in follow-on public offering (in shares)									952,794
Proceeds from stock offerings, net of issuance costs \| $									$ 24,100
2023 Sales Agreement
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Shares sold in follow-on public offering (in shares)								192,190
Proceeds from stock offerings, net of issuance costs \| $								$ 5,300
Sale of stock, aggregate authorized amount \| $						$ 75,000
2024 Sales Agreement
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Shares sold in follow-on public offering (in shares)								1,614,975
Proceeds from stock offerings, net of issuance costs \| $								$ 113,100
Sale of stock, aggregate authorized amount \| $					$ 150,000
March Sales Agreement with Jeffries
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Shares sold in follow-on public offering (in shares)								16,487
Proceeds from stock offerings, net of issuance costs \| $								$ 1,000
Sale of stock, aggregate authorized amount \| $				$ 250,000
June 2023 Public Offering
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Common stock, authorized (in shares)			4,296,646
Sale of stock, price (in USD per share) \| $ / shares			$ 14.25
June 2023 Public Offering \| Pre-Funded Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Warrants (in shares)			470,000
Sale of stock, price (in USD per share) \| $ / shares			$ 14.2485
Stock issued during period, shares, exercise of warrants								469,981
June 2023 Public Offering \| Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Class of warrant or right, exercise price of warrants or rights (in dollars per share) \| $ / shares			$ 0.0015
Over-Allotment Option
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Shares sold in follow-on public offering (in shares)	530,973	633,750	619,979
January 2024 Follow-On Offering
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Proceeds from stock offerings, net of issuance costs \| $		$ 161,600
Sale of stock, price (in USD per share) \| $ / shares		$ 35.50
Sale of stock, shares, aggregate authorized amount (in shares)		3,802,025
January 2024 Follow-On Offering \| Pre-Funded Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Warrants (in shares)		1,056,725
Share price (in dollars per share) \| $ / shares		$ 35.4999
January 2024 Follow-On Offering \| Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Class of warrant or right, exercise price of warrants or rights (in dollars per share) \| $ / shares		$ 0.0001
January 2024 Public Offering \| Pre-Funded Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Stock issued during period, shares, exercise of warrants								152,142
Class of warrants or rights, exercised (in shares)								152,145
Class of warrant or right, outstanding (in shares)				904,580				904,580
April 2024 Public Offering
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Proceeds from stock offerings, net of issuance costs \| $	$ 216,000
Sale of stock, price (in USD per share) \| $ / shares	$ 56.50
Sale of stock, shares, aggregate authorized amount (in shares)	3,849,558
April 2024 Public Offering \| Pre-Funded Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Warrants (in shares)	221,238
Sale of stock, price (in USD per share) \| $ / shares	$ 56.4999
April 2024 Public Offering \| Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Class of warrant or right, exercise price of warrants or rights (in dollars per share) \| $ / shares	$ 0.0001

Accrued Expenses - Summary of Accrued Expenses (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Payables and Accruals [Abstract]
Accrued external research and development expenses	$ 13,363	$ 2,957
Accrued personnel-related expenses	9,365	3,716
Accrued other expenses	1,035	743
Total accrued expenses	$ 23,763	$ 7,416

Commitments and Contingencies - Weighted Average Remaining Lease Term and Weighted Average Incremental Borrowing Rate (Details)	Dec. 31, 2024	Dec. 31, 2023
Commitments and Contingencies Disclosure [Abstract]
Weighted average remaining lease term (in years)	1 year 1 month 6 days	2 years 1 month 6 days
Weighted average incremental borrowing rate	9.00%	9.00%

Nature of the Business	12 Months Ended
Nature of the Business	Dec. 31, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Nature of the Business	Nature of the Business Praxis Precision Medicines, Inc. (“Praxis” or the “Company”) is a clinical-stage biopharmaceutical company translating insights from genetic epilepsies into the development of therapies for central nervous system ("CNS"), disorders characterized by neuronal excitation-inhibition imbalance. Normal brain function requires a delicate balance of excitation and inhibition in neuronal circuits, which, when dysregulated, can lead to abnormal function and both rare and more prevalent neurological disorders. The Company is applying genetic insights to the discovery and development of therapies for neurological disorders through two proprietary platforms, using its understanding of shared biological targets and circuits in the brain. Each platform has multiple programs currently, with significant potential for additional program and indication expansion: •Cerebrum™, the Company's small molecule platform, utilizes deep understanding of neuronal excitability and neuronal networks and applies a series of computational and experimental tools to develop orally available precision therapies •Solidus™, the Company's antisense oligonucleotide ("ASO"), platform, is an efficient, targeted precision medicine discovery and development engine anchored on a proprietary, computational methodology The Company's platforms utilize a deliberate, pragmatic and patient-guided approach, leveraging a suite of translational tools, including novel transgenic and predictive translational animal models and electrophysiology markers, to enable an efficient path to proof-of-concept in patients. Through this approach, the Company has established a diversified, multimodal CNS portfolio with four clinical-stage product candidates across movement disorders and epilepsy. Praxis was incorporated in 2015 and commenced operations in 2016. The Company has funded its operations primarily with proceeds from the issuance of redeemable convertible preferred stock, from the sale of common stock through an initial public offering ("IPO"), at-the-market offerings under its shelf registration statement, and follow-on public offerings of common stock and pre-funded warrants to purchase common stock. From inception through December 31, 2024, the Company raised $1.1 billion in aggregate cash proceeds from these transactions, net of issuance costs. The Company is subject to risks and uncertainties common to clinical-stage companies in the biotechnology industry, including but not limited to, risks associated with completing preclinical studies and clinical trials, receiving regulatory approvals for product candidates, development by competitors of new biopharmaceutical products, dependence on key personnel, protection of proprietary technology, compliance with government regulations and the ability to secure additional capital to fund operations. Programs currently under development will require significant additional research and development efforts, including preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will realize revenue from product sales. Liquidity In accordance with the Financial Accounting Standards Board ("FASB") Accounting Standards Update ("ASU") 2014-15, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern (Subtopic 205-40), the Company has evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date that the consolidated financial statements are issued. The Company has incurred recurring losses since its inception, including a net loss of $182.8 million for the year ended December 31, 2024. In addition, as of December 31, 2024, the Company had an accumulated deficit of $836.7 million. The Company expects to continue to generate operating losses for the foreseeable future. The Company expects that its cash, cash equivalents and marketable securities as of December 31, 2024 of $469.5 million will be sufficient to fund its operating expenditures and capital expenditure requirements necessary to advance its research efforts and clinical trials for at least one year from the date of the issuance of these consolidated financial statements. The future viability of the Company is dependent on its ability to raise additional capital to finance its operations. The Company's inability to raise capital as and when needed could have a negative impact on its financial condition and ability to pursue its business strategies. There can be no assurance that the current operating plan will be achieved or that additional funding will be available on terms acceptable to the Company, or at all.

Common Stock and Preferred Stock - Summary of Common Stock Reserved For Future Issuance (Details) - shares	Dec. 31, 2024	Dec. 31, 2023
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	3,473,399	1,384,683
Shares reserved for exercise of outstanding stock options
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	1,809,343	666,163
Pre-Funded Warrant \| June 2023 Follow-On Offering
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	0	470,000
Pre-Funded Warrant \| January 2024 Follow-On Offering
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	904,580	0
Pre-Funded Warrant \| April 2024 Follow-On Offering
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	221,238	0
Future Share-based Awards \| Shares reserved for future awards under the 2020 Stock Option and Incentive Plan
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	91,343	148,264
Future Share-based Awards \| Shares reserved for future awards under the 2020 Employee Stock Purchase Plan
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	109,648	53,111
Future Share-based Awards \| Shares reserved for future awards under the 2024 Inducement Plan
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	112,774	0
Restricted Stock
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	224,473	47,145

Stock-Based Compensation

12 Months Ended

Dec. 31, 2024

Share-Based Payment Arrangement [Abstract]

Stock-Based Compensation

Equity Incentive Plans

In January 2024, the Board of Directors ("the Board") adopted the 2024 Inducement Plan (the "Inducement Plan") without stockholder approval pursuant to Rule 5635(c)(4) of the Nasdaq Stock Market LLC listing rules ("Rule 5635(c)(4)"). In accordance with Rule 5635(c)(4), the Inducement Plan allows the Company to grant awards only to a newly hired employee who was not previously an employee or non-employee director or to an employee who is being rehired following a bona fide period of non-employment if such award is a material inducement to such employee entering into employment. The total shares of common stock initially authorized for issuance under the Inducement Plan was 1,000,000. In June 2024, the shares of common stock authorized for issuance under the Inducement Plan was reduced by 870,000 shares following stockholder approval of an amendment and restatement of the 2020 Stock Option and Incentive Plan ("the 2020 Plan"). The total number of shares of common stock authorized for issuance under the Inducement Plan as of December 31, 2024 was 130,000 shares.

The total number of shares of common stock authorized for issuance under the 2020 Plan as of December 31, 2024 and December 31, 2023 was 1,970,833 shares and 661,240 shares, respectively. The total number of shares of common stock authorized for issuance under the 2017 Stock Incentive Plan (the "2017 Plan") as of December 31, 2024 and 2023 was 395,850 shares. Any authorization to issue new options under the 2017 Plan was cancelled upon the effectiveness of the 2020 Plan and no further awards will be granted under the 2017 Plan.

Stock options issued under the 2024 Inducement, 2020 Plan and 2017 Plan expire ten years from the date of grant. Shares that expire, are terminated, surrendered or canceled under the 2024 Inducement, 2020 Plan and 2017 Plan without having been fully exercised will be available for future awards. In addition, shares of common stock that are tendered to the Company by a participant to exercise an award are added to the number of shares of

common stock available for future awards. Upon stock option exercise, the Company issues new shares and delivers them to the participant.

The total shares authorized for issuance under the 2020 Employee Stock Purchase Plan (the "2020 ESPP") as of December 31, 2024 and 2023 was 175,145 shares and 87,227 shares, respectively. During the years ended December 31, 2024 and 2023, the Company issued 31,381 and 24,690 shares, respectively, under the 2020 ESPP.

Restricted Stock Units

The following table summarizes the Company’s restricted stock unit activity:


	Shares	Weighted Average Grant Date Fair Value
Unvested as of December 31, 2023	47,145	$	202.27
Issued	208,997	53.35
Vested	(19,576)	201.07
Forfeited	(12,093)	53.90
Unvested as of December 31, 2024	224,473	$	71.71

As of December 31, 2024, total unrecognized compensation cost related to unvested restricted stock units was $10.6 million, which is expected to be recognized over a weighted-average period of 2.72 years.

The total fair value of restricted stock units that vested during the years ended December 31, 2024 and 2023 was $1.0 million and $0.5 million, respectively.

Stock Options

The following table summarizes the Company’s stock option activity:


	Number of Shares	Weighted Average Exercise Price per Share		Weighted Average Remaining Contractual Term	Aggregate Intrinsic Value
				(In years)	(In thousands)
Outstanding as of December 31, 2023	666,163	$	160.19	7.79	$	254
Granted	1,221,746	51.59
Exercised	(45,177)	46.24			$	1,231
Cancelled or Forfeited	(33,389)	108.85
Outstanding as of December 31, 2024	1,809,343	$	90.84	8.47	$	39,989
Exercisable as of December 31, 2024	933,936	$	120.30	7.77	$	19,033
Vested and expected to vest as of December 31, 2024	1,809,343	$	90.84	8.47	$	39,989

The aggregate intrinsic value of stock options exercised for the years ended December 31, 2024 and 2023 was $1.2 million and $0.1 million, respectively. The aggregate intrinsic value of stock options outstanding, exercisable, and vested and expected to vest is calculated as the difference between the exercise price of the underlying stock options and the estimated fair value of the Company’s common stock for those stock options that had exercise prices lower than the estimated fair value of the Company’s common stock at December 31, 2024. The aggregate intrinsic value of stock options exercised is calculated as the difference between the exercise price of the underlying stock options and the estimated fair value of the Company’s common stock on the date of exercise for those stock options that had exercise prices lower than the fair value of the Company’s common stock on the exercise date.

Valuation of Stock Options

The weighted-average assumptions that the Company used in the Black-Scholes option pricing model to determine the grant-date fair value of stock options granted to employees, members of the Board, and non-employees on the date of grant were as follows for the for the years ended December 31, 2024 and 2023:


	Year Ended December 31,
	2024			2023
Risk-free interest rate	4.01		%	3.60		%
Expected term (in years)	6.03			6.00
Expected volatility	96.60		%	88.40		%
Expected dividend yield	—		%	—		%
Weighted average grant-date fair value per share	$	41.25		$	29.73

As of December 31, 2024, total unrecognized compensation cost related to unvested stock options was $38.4 million, which is expected to be recognized over a weighted-average period of 2.49 years.

Stock-Based Compensation

Stock-based compensation expense was allocated as follows (in thousands):


	Year Ended December 31,
	2024		2023
Research and development	$	15,620	$	8,055
General and administrative	25,740		16,802
Total stock-based compensation expense	$	41,360	$	24,857

Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Statement of Comprehensive Income [Abstract]
Net loss	$ (182,819)	$ (123,277)
Change in unrealized gain (loss) on marketable securities, net of tax	654	173
Comprehensive loss	$ (182,165)	$ (123,104)

- Definition

Amount after tax of increase (decrease) in equity from transactions and other events and circumstances from net income and other comprehensive income, attributable to parent entity. Excludes changes in equity resulting from investments by owners and distributions to owners.

+ References + Details

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 250
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483443/250-10-50-6

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 250
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483443/250-10-50-9

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 805
-SubTopic 60
-Name Accounting Standards Codification
-Section 65
-Paragraph 1
-Subparagraph (g)
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+ Details

Related Party Transactions	12 Months Ended
Related Party Transactions	Dec. 31, 2024
Related Party Transactions [Abstract]
Related Party Transactions	Related Party TransactionsOne of the founders of RogCon is the Company's General Counsel. During the years ended December 31, 2024 and 2023, the Company reimbursed RogCon for its out-of-pocket costs incurred for activities performed under the License Agreement (Note 12).

- Definition

The entire disclosure for related party transactions. Examples of related party transactions include transactions between (a) a parent company and its subsidiary; (b) subsidiaries of a common parent; (c) and entity and its principal owners; and (d) affiliates.

+ References

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+ Details

Consolidated Statements of Operations - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Income Statement [Abstract]
Collaboration revenue	$ 8,553	$ 2,447
Operating expenses:
Research and development	152,413	86,766
General and administrative	56,305	42,054
Total operating expenses	208,718	128,820
Loss from operations	(200,165)	(126,373)
Other income:
Other income, net	17,346	3,096
Total other income	17,346	3,096
Net loss	$ (182,819)	$ (123,277)
Net loss per share attributable to common stockholders, basic (in usd per share)	$ (10.21)	$ (18.69)
Net loss per share attributable to common stockholders, diluted (in usd per share)	$ (10.21)	$ (18.69)
Weighted average common shares outstanding, basic (in shares)	17,906,794	6,594,316
Weighted average common shares outstanding, diluted (in shares)	17,906,794	6,594,316

Accrued Expenses

12 Months Ended

Dec. 31, 2024

Payables and Accruals [Abstract]

Accrued Expenses

Accrued expenses consisted of the following (in thousands):


	December 31,
	2024		2023
Accrued external research and development expenses	$	13,363	$	2,957
Accrued personnel-related expenses	9,365		3,716
Accrued other expenses	1,035		743
Total accrued expenses	$	23,763	$	7,416

Commitments and Contingencies - Total Lease Costs (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Commitments and Contingencies Disclosure [Abstract]
Operating lease cost	$ 1,102	$ 1,102
Variable lease costs	33	14
Total lease costs	$ 1,135	$ 1,116

Stock-Based Compensation - Narrative (Details) - USD ($) $ in Thousands	1 Months Ended	12 Months Ended
	Jun. 30, 2024	Dec. 31, 2024	Dec. 31, 2023	Jan. 31, 2024
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total fair value of restricted stock units vested		$ 1,000	$ 500
Aggregate intrinsic value exercised		1,231	$ 100
Unvested Restricted Stock Units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized compensation cost related to unvested restricted stock units		$ 10,600
Unrecognized compensation expense expected to be recognized		2 years 8 months 19 days
Shares reserved for exercise of outstanding stock options
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized compensation expense expected to be recognized		2 years 5 months 26 days
Unrecognized compensation expense related to unvested stock based awards		$ 38,400
Shares reserved for future awards under the 2024 Inducement Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of shares authorized (in shares)		130,000		1,000,000
Share-based payment award, increase (decrease) Authorized (in shares)	(870,000)
2020 Employee Stock Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of shares authorized (in shares)		1,970,833	661,240
Award expiration period		10 years
2017 Stock Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of shares authorized (in shares)		395,850	395,850
Award expiration period		10 years
Shares reserved for future awards under the 2020 Employee Stock Purchase Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of shares authorized (in shares)		175,145	87,227
Issuance of common stock under employee stock purchase plan (in shares)		(31,381)	(24,690)
2024 Employee Stock Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Award expiration period		10 years

Segment Information (Tables)

12 Months Ended

Dec. 31, 2024

Segment Reporting [Abstract]

Schedule of Segment Reporting Information, by Segment

The following table presents selected financial information about the Company's single operating segment for the year ended December 31, 2024 (in thousands):


	December 31,
	2024		2023
Collaboration revenue	$	8,553	$	2,447
Program-specific expenses:
Ulixacaltamide(a)	69,172		19,464
Vormatrigine	18,360		6,275
Relutrigine	6,273		3,996
Elsunersen	2,135		14,595
Other early stage assets	4,634		7,055
Personnel-related expenses	42,853		33,160
Stock-based compensation expense	41,361		24,857
Depreciation expense	358		432
Other segment expenses(b)	23,572		18,986
Interest income	17,346		3,096
Consolidated net loss	$	(182,819)	$	(123,277)

(a) Includes non-cash research and development of $2.5 million in the year ended December 31, 2024 associated with the Company's collaboration with Tenacia

(b) Other segment expenses includes research and development and general and administrative costs not attributable to a specific program.

Stock-Based Compensation - Summary of Stock Option Activity (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Number of Shares
Number of shares outstanding beginning balance (in shares)	666,163
Number of shares granted (in shares)	1,221,746
Number of shares exercised (in shares)	(45,177)
Number of shares canceled or forfeited (in shares)	(33,389)
Number of shares outstanding ending balance (in shares)	1,809,343	666,163
Number of shares exercisable (in shares)	933,936
Number of shares vested and expected to vest (in shares)	1,809,343
Weighted Average Exercise Price per Share
Weighted average exercise price beginning balance (in dollars per share)	$ 160.19
Weighted average exercise price granted (in dollars per share)	51.59
Weighted average exercise price exercised (in dollars per share)	46.24
Weighted average exercise price cancelled or forfeited (in dollars per share)	108.85
Weighted average exercise price ending balance (in dollars per share)	90.84	$ 160.19
Weighted average exercise price exercisable (in dollars per share)	120.30
Weighted average exercise price vested and expected to vest (in dollars per share)	$ 90.84
Weighted Average Remaining Contractual Term and Aggregate Intrinsic Value
Weighted average remaining contractual term outstanding	8 years 5 months 19 days	7 years 9 months 14 days
Weighted average remaining contractual term exercisable	7 years 9 months 7 days
Weighted average remaining contractual term vested and expected to vest	8 years 5 months 19 days
Aggregate intrinsic value exercised	$ 1,231	$ 100
Aggregate intrinsic value outstanding	39,989	$ 254
Aggregate intrinsic value exercisable	19,033
Aggregate intrinsic value vested and expected to vest	$ 39,989

Nature of the Business (Details)	3 Months Ended								12 Months Ended
Nature of the Business (Details)	Dec. 31, 2024 USD ($) platform candidate	Sep. 30, 2024 USD ($)	Jun. 30, 2024 USD ($)	Mar. 31, 2024 USD ($)	Dec. 31, 2023 USD ($)	Sep. 30, 2023 USD ($)	Jun. 30, 2023 USD ($)	Mar. 31, 2023 USD ($)	Dec. 31, 2024 USD ($) platform candidate	Dec. 31, 2023 USD ($)
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Number of platforms \| platform	2								2
Number of product candidates \| candidate	4								4
Proceeds from issuance of equity									$ 1,100,000,000
Net loss	$ 58,679,000	$ 51,910,000	$ 32,677,000	$ 39,553,000	$ 26,878,000	$ 24,632,000	$ 34,312,000	$ 37,455,000	182,819,000	$ 123,277,000
Accumulated deficit	836,740,000				653,921,000				836,740,000	653,921,000
Cash, cash equivalents, and short-term investments	$ 469,500,000				$ 0				$ 469,500,000	$ 0

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References

+ Details

- Definition

Amount of accumulated undistributed earnings (deficit).

+ References + Details

Cover Page - USD ($) $ in Millions	12 Months Ended
Cover Page - USD ($) $ in Millions	Dec. 31, 2024	Feb. 26, 2025	Jun. 28, 2024
Cover [Abstract]
Document Type	10-K
Document Annual Report	true
Document Period End Date	Dec. 31, 2024
Current Fiscal Year End Date	--12-31
Document Transition Report	false
Entity File Number	001-39620
Entity Registrant Name	PRAXIS PRECISION MEDICINES, INC.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	47-5195942
Entity Address, Address Line One	99 High Street
Entity Address, Address Line Two	30th Floor
Entity Address, City or Town	Boston
Entity Address, State or Province	MA
Entity Address, Postal Zip Code	02110
City Area Code	617
Local Phone Number	300-8460
Title of 12(b) Security	Common Stock, par value $0.0001 per share
Trading Symbol	PRAX
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	true
Entity Emerging Growth Company	false
ICFR Auditor Attestation Flag	true
Document Financial Statement Error Correction [Flag]	false
Entity Shell Company	false
Entity Public Float			$ 733.0
Entity Common Stock, Shares Outstanding		20,163,250
Amendment Flag	false
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	FY
Entity Central Index Key	0001689548

Income Taxes

12 Months Ended

Dec. 31, 2024

Income Tax Disclosure [Abstract]

Income Taxes

The Company maintains a full valuation allowance on its U.S. net deferred tax assets due to the uncertainty of future taxable income. The Company did not recognize an income tax benefit in the years ended December 31, 2024 or 2023 related to its U.S. operations due to the uncertainty regarding future taxable income. In the years ended December 31, 2024 and 2023, the difference between the statutory tax rate in the U.S. and the Company’s effective tax rate was due primarily to the valuation allowance recorded to offset any potential tax benefit. The income tax benefit (provision) recognized for the years ended December 31, 2024 and 2023 was not material.

The reconciliation of the U.S. statutory income tax rate to the Company’s effective tax rate is as follows:


	Year Ended December 31,
	2024		2023
Federal statutory income tax rate	21.0	%	21.0	%
Federal and state research and development credits	3.9	%	3.5	%
State taxes, net of federal benefit	3.5	%	4.3	%
Changes in tax rates	(3.2)	%	2.9	%
Non-deductible executive compensation	(2.6)	%	(1.4)	%
Non-deductible items	(0.8)	%	—	%
Stock-based compensation	(0.9)	%	(1.3)	%
Change in valuation allowance	(20.7)	%	(29.7)	%
Other	(0.2)	%	0.7	%
Effective income tax rate	—	%	—	%

Net deferred tax assets consisted of the following (in thousands):


	December 31,
	2024		2023
Deferred tax assets:
Net operating loss carryforwards	$	90,108	$	74,528
Capitalized research expenditures	65,836		48,954
Research and development credits	30,582		23,451
Amortization	20,600		23,929
Stock-based compensation	8,784		7,697
Accrued expenses	2,333		1,147
Leases	344		676
Deferred revenue	—		692
Total gross deferred tax assets	218,587		181,074
Less: Valuation allowance	(218,303)		(180,515)
Net deferred tax assets	$	284	$	559
Deferred tax liabilities:
Operating lease right-of-use asset	(284)		(559)
Total gross deferred tax liabilities	(284)		(559)
Net deferred tax assets	$	—	$	—

As of December 31, 2024 and 2023, the Company had U.S. federal net operating loss carryforwards which may be able to offset future income tax liabilities of approximately $342.4 million and $282.8 million, respectively. Federal net operating loss carryforwards of $7.7 million will expire at various dates from 2035 through 2037 and approximately $334.7 million may be carried forward indefinitely. As of December 31, 2024 and 2023, the Company also had state net operating loss carryforwards of approximately $289.7 million and $239.4 million, respectively, which may be available to offset future income tax liabilities and expire at various dates from 2036 through 2044.

As of December 31, 2024 and 2023, the Company had federal research and development tax credit carryforwards of approximately $27.3 million and $20.4 million, respectively, available to reduce future tax liabilities which expire at various dates from 2039 through 2044. As of December 31, 2024 and 2023, the Company had state research and development tax credit carryforwards of approximately $4.1 million and $3.8 million, respectively, available to reduce future tax liabilities which expire at various dates from 2032 through 2039. The Company has generated research credits but has not conducted a study to document the qualified activity. This study may result in an adjustment to the Company’s research and development credit carryforwards; however, until a study is completed, and any adjustment is known, no amounts are being presented as an uncertain tax position. A full valuation allowance has been provided against the Company’s research and development credits and, if an adjustment is required, this adjustment would be offset by an adjustment to the deferred tax asset established for the research and development credit carryforwards and the valuation allowance.

Under the provisions of the Internal Revenue Code, 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. 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 the ownership change. Subsequent ownership changes may further affect the limitation in future years. The Company has not conducted a study to assess whether a change of control has occurred or whether there have been multiple changes of control since inception due to the significant complexity and cost associated with such a study. If the Company has experienced a change of control, as defined by Section 382, at any time since inception, utilization of

the net operating loss carryforwards or research and development tax credit carryforwards would be subject to an annual limitation under Section 382, which is determined by first multiplying the value of the Company’s stock at the time of the ownership change by the applicable long-term tax-exempt rate, and then could be subject to additional adjustments, as required. Any limitation may result in expiration of a portion of the net operating loss carryforwards or research and development tax credit carryforwards before utilization. Further, until a study is completed by the Company and any limitation is known, no amounts are being presented as an uncertain tax position.

ASC 740 requires a valuation allowance to reduce the deferred tax assets reported if, based on the weight of the 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 of the evidence, both positive and negative, the Company has recorded a valuation allowance against its deferred tax assets at December 31, 2024 and 2023 because management has determined that it is more likely than not that the Company will not recognize the benefits of its federal and state deferred tax assets, primarily due to its history of cumulative net losses incurred since inception and its lack of commercialization of any products or generation of any revenue from product sales since inception. As a result, a valuation allowance of $218.3 million and $180.5 million has been established at December 31, 2024 and 2023, respectively. Management reevaluates the positive and negative evidence at each reporting period. The valuation allowance increased by approximately $37.8 million and $36.6 million during the years ended December 31, 2024 and 2023, respectively, due primarily to the generation of net operating losses.

The Company has not recorded any amounts for unrecognized tax benefits as of December 31, 2024 and 2023. The Company’s policy is to record interest and penalties related to uncertain tax positions as part of its income tax provision. As of December 31, 2024 and 2023, the Company had no accrued interest or penalties related to uncertain tax positions and no such amounts have been recognized in the Company’s consolidated statement of operations for either year ended December 31, 2024 or 2023. The statute of limitations for federal and the majority of state tax authorities is open for tax years ended December 31, 2021 through December 31, 2024. The statute of limitation for the remaining state tax authorities is open for tax years ended December 31, 2020 through December 31, 2024. Since the Company is in a net loss carryforward position, the Company 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.

- Definition

The entire disclosure for income tax.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-12

Reference 2: http://www.xbrl.org/2003/role/exampleRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 231
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482663/740-10-55-231

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12C
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-12C

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12B
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-12B

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 270
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477891/740-270-50-1

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SAB Topic 6.I.5.Q1)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479360/740-10-S99-1

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480990/946-20-50-13

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 235
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(h)(2))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480678/235-10-S99-1

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-Name Accounting Standards Codification
-Publisher FASB
-URI https://asc.fasb.org/740/tableOfContent

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 14
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-14

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 21
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-21

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 17
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-17

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 2
-Subparagraph (SAB Topic 11.C)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479360/740-10-S99-2

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 30
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482603/740-30-50-2

+ Details

Common Stock and Preferred Stock (Tables)

12 Months Ended

Dec. 31, 2024

Stockholders' Equity Note [Abstract]

Summary of common stock reserved for future issuance

The Company had reserved the following shares of common stock for future issuance:


	December 31,
	2024	2023
Shares reserved for exercise of outstanding stock options	1,809,343	666,163
Shares reserved for exercise of pre-funded warrants related to the June 2023 Financing	—	470,000
Shares reserved for exercise of pre-funded warrants related to the January 2024 Financing	904,580	—
Shares reserved for exercise of pre-funded warrants related to the April 2024 Financing	221,238	—
Shares reserved for future awards under the 2020 Stock Option and Incentive Plan	91,343	148,264
Shares reserved for future awards under the 2020 Employee Stock Purchase Plan	109,648	53,111
Shares reserved for future awards under the 2024 Inducement Plan	112,774	—
Shares reserved for vesting of restricted stock units	224,473	47,145
Total shares of authorized common stock reserved for future issuance	3,473,399	1,384,683

Commitments and Contingencies - Future Lease Payments (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Commitments and Contingencies Disclosure [Abstract]
2025	$ 1,321
2026	110
Total future lease payments	1,431
Less: interest	(62)
Total lease liabilities	$ 1,369	$ 2,495

Cash Equivalents and Marketable Securities - Additional Information (Details) - USD ($)	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Cash and Cash Equivalents [Abstract]
Cash, cash equivalents, and short-term investments	$ 469,500,000	$ 0
Loss on available-for-sale securities	$ 0	$ 0

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended								12 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Dec. 31, 2024	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Dec. 31, 2023	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Dec. 31, 2024	Dec. 31, 2023
Pay vs Performance Disclosure
Net loss	$ (58,679)	$ (51,910)	$ (32,677)	$ (39,553)	$ (26,878)	$ (24,632)	$ (34,312)	$ (37,455)	$ (182,819)	$ (123,277)

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References

+ Details

Fair Value of Financial Assets (Tables)

12 Months Ended

Dec. 31, 2024

Fair Value Disclosures [Abstract]

Fair Value, Assets Measured on Recurring Basis

The following table presents information about the Company's financial assets measured at fair value on a recurring basis and indicates the level of the fair value hierarchy utilized to determine such fair values as of December 31, 2024 (in thousands). The Company did not hold any financial assets measured at fair value on a recurring basis as of December 31, 2023.


	As of December 31, 2024
	Level 1		Level 2		Level 3		Total
Assets:
Money market funds	$	128,652	$	—	$	—	$	128,652

Debt securities issued by U.S. government agencies	144,644		—		—		144,644
Corporate debt securities	—		107,852		—		107,852
Commercial paper	—		8,794		—		8,794
Other debt securities	—		8,821		—		8,821
	$	273,296	$	125,467	$	—	$	398,763

Consolidated Statements of Stockholders' Equity (Parenthetical) - USD ($) $ in Thousands	3 Months Ended
	Dec. 31, 2024	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Dec. 31, 2023	Jun. 30, 2023	Mar. 31, 2023
Stock issuance costs					$ 123	$ 4,484	$ 560
Follow-On Offering
Stock issuance costs			$ 14,013	$ 10,836
At-The-Market Offering
Stock issuance costs	$ 2,812	$ 70		$ 376

Summary of Significant Accounting Policies (Tables)

12 Months Ended

Dec. 31, 2024

Accounting Policies [Abstract]

Summary of Cash, Cash Equivalents and Restricted Cash

The following table presents cash and cash equivalents and restricted cash as reported on the consolidated balance sheets that equal the total amounts on the consolidated statements of cash flows (in thousands):


	December 31,
	2024		2023
Cash and cash equivalents	$	215,372	$	81,300
Restricted cash	416		416
Total cash, cash equivalents and restricted cash as shown on the consolidated statements of cash flows	$	215,788	$	81,716

Schedule of Restricted Cash and Cash Equivalents


	December 31,
	2024		2023
Cash and cash equivalents	$	215,372	$	81,300
Restricted cash	416		416
Total cash, cash equivalents and restricted cash as shown on the consolidated statements of cash flows	$	215,788	$	81,716

Schedule of Property, Plant and Equipment

Property and equipment are recorded at cost. Depreciation is calculated using the straight-line method over the following estimated useful lives of the assets:


			Estimated Useful Life
Office furniture and equipment			5 years
Laboratory equipment			3 years
Computer equipment			3 years

Property and equipment, net consisted of the following (in thousands):


	December 31,
	2024		2023
Computer equipment	$	689	$	689
Office furniture and equipment	545		545
Laboratory equipment	359		359
Total property and equipment	1,593		1,593
Less: Accumulated depreciation	(1,363)		(1,005)
Property and equipment, net	$	230	$	588

Commitments and Contingencies

12 Months Ended

Dec. 31, 2024

Commitments and Contingencies Disclosure [Abstract]

Commitments and Contingencies

Leases

In May 2021, the Company entered into a sublease agreement for office space located in Boston, Massachusetts. The sublease expires on January 31, 2026, with no option to renew or terminate early. The base rent increases by approximately 2% annually. The Company issued a letter of credit to the landlord related to the security deposit, secured by restricted cash (Note 3). This lease qualifies as an operating lease. At inception, the Company recorded an operating lease right-of-use asset and operating lease liability of $4.1 million.

The following table summarizes the presentation of the operating lease in the Company’s consolidated balance sheets (in thousands):


	As of December 31,
	2024		2023
Assets:
Operating lease right-of-use assets	$	1,131	$	2,064
Liabilities:
Current operating lease liabilities	$	1,259	$	1,126
Non-current portion of operating lease liabilities	110		1,369
Total lease liabilities	$	1,369	$	2,495

The following table summarizes total lease costs recognized in the Company’s consolidated statements of operations (in thousands):


	For the year ended December 31,
	2024		2023
Operating lease cost	$	1,102	$	1,102
Variable lease costs	33		14
Total lease costs	$	1,135	$	1,116

Variable lease costs were primarily related to operating expenses, taxes and insurance associated with the operating leases, which were assessed based on the Company’s proportionate share of such costs for the leased premises. As these costs are generally variable in nature, they were not included in the measurement of the operating lease right-of-use asset and related lease liability. Total lease costs are included as operating expenses in the Company’s consolidated statements of operations and comprehensive loss.

Future lease payments under non-cancelable lease agreements as of December 31, 2024 were as follows (in thousands):


Year Ended December 31,	Future Lease Payments
2025	$	1,321
2026	110
Total future lease payments	1,431
Less: interest	(62)
Present value of operating lease liabilities	$	1,369

The weighted average remaining lease term and weighted average incremental borrowing rate of the Company’s operating lease were as follows:


	As of December 31,
	2024		2023
Weighted average remaining lease term (in years)	1.1		2.1
Weighted average incremental borrowing rate	9.0	%	9.0	%

Legal Proceedings

The Company, from time to time, may be party to litigation arising in the ordinary course of business. The Company was not subject to any material legal proceedings during the years ended December 31, 2024 and 2023, and no material legal proceedings are currently pending or threatened.

Purchase Orders

The Company has agreements with third parties for various services, including services related to research, preclinical and clinical operations and support, for which the Company is not contractually able to terminate for convenience to avoid future obligations to the vendors. Certain agreements provide for termination rights subject to termination fees or wind down costs. Under such agreements, the Company is contractually obligated to make certain payments to vendors, primarily to reimburse them for their unrecoverable outlays incurred prior to cancellation. The actual amounts the Company could pay in the future to the vendors under such agreements may differ from the purchase order amounts due to cancellation provisions.

Indemnification Agreements

The Company enters into indemnification agreements and agreements containing indemnification provisions in the ordinary course of business. Pursuant to these agreements, the Company agrees to indemnify, hold

harmless, and to reimburse the indemnified party for losses suffered or incurred by the indemnified party, generally the Company’s business partners, in connection with any U.S. patent or any copyright or other intellectual property infringement claim by any third party with respect to the Company’s products. The term of these indemnification agreements is generally perpetual upon execution of the agreement. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is unlimited. The Company has never incurred costs to defend lawsuits or settle claims related to these indemnification agreements.

Agreements

The Company has entered into multiple agreements with third parties under which it may be obligated to make future development, regulatory and commercial milestone payments and royalty payments on future sales of specified product candidates. Payments under these agreements generally become due and payable upon achievement of such milestones and sales. When the achievement of these milestones or sales have not occurred, such contingencies are not recorded in the Company's financial statements.

Collaboration and License Agreements	12 Months Ended
Collaboration and License Agreements	Dec. 31, 2024
Revenue from Contract with Customer [Abstract]
Collaboration and License Agreements	Collaboration and License Agreements UCB Option and License Agreement In December 2022, the Company entered into an Option and License Agreement (“the Collaboration Agreement”) with UCB for the discovery of small molecule therapeutics as potential treatments of KCNT1-related epilepsies. Under the terms of the Collaboration Agreement, the Company has agreed to perform general biology-related research services as part of a mutually agreed upon research plan in exchange for a $5.0 million upfront payment. In addition, the Company provided UCB an exclusive option to in-license global development and commercialization rights to any resulting KCNT1 small molecule development candidate identified as part of the research plan. If UCB exercises its option to in-license global development and commercialization rights, the Collaboration Agreement stipulates that UCB will assume research, development, manufacturing and commercialization responsibilities and costs. Under the terms of the Collaboration Agreement, the Company will be eligible to receive an option fee and future success-based development and commercialization milestone payments, totaling up to $98.5 million, in addition to tiered royalties on net sales of any resulting products from the Collaboration Agreement. The Company concluded that UCB is a customer, and as such, the arrangement falls within the scope of Topic 606. At the commencement of the Collaboration Agreement, the Company identified one performance obligation, which was to perform the research services for UCB. The Company determined the transaction price to be $5.0 million, comprised of the upfront payment it received. The option provided to UCB was determined not to be a material right. The Company recognizes revenue for its research services performance obligation over time using an input method over the duration of the research services. In December 2024, UCB exercised its option to in-license global development and commercialization rights for a KCNT1 development candidate as part of the Collaboration Agreement. Upon notice of the exercise, the Company recognized the $6.0 million option exercise fee as collaboration revenue within the consolidated statement of operations. The Company also recognized the $2.6 million of remaining collaboration revenue associated with the $5.0 million up front payment, as the Company will have no further research service obligations under the terms of the Collaboration Agreement. During the years ended December 31, 2024 and 2023, the Company recognized $8.6 million and $2.4 million respectively, in collaboration revenue related to the Collaboration Agreement in the consolidated statement of operations. As of December 31, 2024, there was no deferred revenue included in the consolidated balance sheet. Tenacia Collaboration and License Agreement On January 4, 2024, the Company entered into an exclusive collaboration and license Agreement (“the License Agreement”) with Tenacia, a China-based portfolio company of Bain Capital, which provides Tenacia an exclusive license to use certain intellectual property for the development and commercialization of ulixacaltamide and products containing ulixacaltamide in China, Hong Kong, Macau and Taiwan. Tenacia is solely responsible for the development and commercialization under the arrangement, with the exception of the associated manufacturing. The Company also entered into a Stock Purchase Agreement (“the Stock Purchase Agreement”) with BCPE Tenet Holdings Cayman, Ltd. (“BCPE”), a related party of Tenacia. Pursuant to the terms of the License Agreement, the Company was entitled to an up-front, non-refundable and non-creditable cash payment of $5.0 million, net of certain tax withholdings. In addition, the Company is eligible to receive $264.0 million in success-based development and commercialization milestone payments as well as tiered royalties on net sales. Pursuant to the terms of the Stock Purchase Agreement, the Company issued and sold 443,253 shares of its common stock to BCPE at a price per share of $22.5605 for aggregate gross proceeds of $10.0 million. The per share price was based on a 20% premium over the 30-day volume-weighted average price. Under the terms of the License Agreement, the Company granted to Tenacia an exclusive license to use certain intellectual property for the development and commercialization of ulixacaltamide and products containing ulixacaltamide in China, Hong Kong, Macau and Taiwan. Tenacia is solely responsible for the development and commercialization under the arrangement, with the exception of the associated manufacturing. The Company concluded that the License Agreement and the Stock Purchase Agreement is a combined arrangement since they were executed at the same time and in contemplation of each other with the same counterparty or a related party thereof and the combined arrangement falls within the scope of Topic 606. The Company’s obligations under the arrangement comprise a single promise, or one performance obligation, related to the exclusive development and commercialization license granted to Tenacia. Total proceeds associated with the combined arrangement at inception were $14.8 million consisting of the following: (i) $10.0 million gross proceeds from the sale of common stock under the Stock Purchase Agreement and (ii) $4.8 million, net of tax, related to the up-front payment under the License Agreement, both of which were received in January 2024. During the year ended December 31, 2024, the Company had not achieved any development or sales milestones or earned any royalties under the License Agreement. The Company recorded the common stock sold to BCPE at its issuance date fair value of $38.95 per share, or $17.3 million in the aggregate, which exceeded the proceeds received which were calculated based on the 20% premium over the prior 30-day volume-weighted average price. Accordingly, there is no transaction price allocable to the performance obligation. The Company accounted for the excess of the fair value of the equity securities issued over the total proceeds received as consideration paid to a customer for which no distinct good or service was transferred in exchange. As a result, the transaction gives rise to negative revenue on a cumulative basis in the amount of $2.5 million, which was recorded at the inception of the collaboration and license agreement in research and development expense in the accompanying consolidated statement of operations. For agreements where the licenses are considered separate performance obligations or represent the only performance obligation, the Company recognizes revenue at the point in time that the Company effectively grants the license, as the licenses or assignments represent functional intellectual property. As of December 31, 2024, the Company did not have any receivables or deferred revenue related to the arrangement with Tenacia. The Company did not recognize any contract assets related to costs to obtain a contract with a customer or costs to fulfill a contract with a customer through December 31, 2024 because no qualifying costs were incurred. During the year ended December 31, 2024, the Company did not recognize any revenue associated with the combined arrangement.

Income Taxes - Net Deferred Tax Assets (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Deferred tax assets:
Net operating loss carryforwards	$ 90,108	$ 74,528
Capitalized research expenditures	65,836	48,954
Research and development credits	30,582	23,451
Amortization	20,600	23,929
Stock-based compensation	8,784	7,697
Accrued expenses	2,333	1,147
Leases	344	676
Deferred revenue	0	692
Total gross deferred tax assets	218,587	181,074
Less: Valuation allowance	(218,303)	(180,515)
Net deferred tax assets	284	559
Deferred tax liabilities:
Operating lease right-of-use asset	(284)	(559)
Total gross deferred tax liabilities	(284)	(559)
Net deferred tax assets	$ 0	$ 0

Significant Agreements (Details) - USD ($) $ in Thousands	12 Months Ended
Significant Agreements (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023	Jul. 31, 2023	Dec. 31, 2022	Sep. 11, 2019
Other Commitments [Line Items]
Research and development expense	$ 152,413	$ 86,766
Ionis
Other Commitments [Line Items]
Research and development expense	0	$ 0
License agreement \| RogCon
Other Commitments [Line Items]
Commitment					$ 2,100
License agreement \| Ionis
Other Commitments [Line Items]
Commitment				$ 2,000
License agreement, profit share payments \| RogCon
Other Commitments [Line Items]
Commitment	$ 3,000
Milestone Payment \| Ionis
Other Commitments [Line Items]
Commitment			$ 6,900

Income Taxes - Schedule of Effective Income Tax Rate Reconciliation (Details)	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Income Tax Disclosure [Abstract]
Federal statutory income tax rate	21.00%	21.00%
Federal and state research and development credits	3.90%	3.50%
State taxes, net of federal benefit	3.50%	4.30%
Changes in tax rates	(3.20%)	2.90%
Non-deductible executive compensation	(2.60%)	(1.40%)
Non-deductible items	(0.80%)	0.00%
Stock-based compensation	(0.90%)	(1.30%)
Change in valuation allowance	(20.70%)	(29.70%)
Other	(0.20%)	0.70%
Effective income tax rate	0.00%	0.00%

Significant Agreements	12 Months Ended
Significant Agreements	Dec. 31, 2024
Commitments and Contingencies Disclosure [Abstract]
Significant Agreements	Significant Agreements RogCon and Ionis Agreements On September 11, 2019, the Company entered into both a Cooperation and License Agreement (the “License Agreement”) with RogCon, Inc. ("RogCon"), and a Research, Collaboration, Option and License Agreement (the “Ionis Collaboration Agreement”) with Ionis Pharmaceuticals, Inc. ("Ionis"). The agreements were entered into contemporaneously to enable the parties to advance their collective efforts related to SCN2A. RogCon Agreement Under the License Agreement, RogCon granted to the Company an exclusive, worldwide license under RogCon’s intellectual property to research, develop and commercialize products for the treatment of all forms of epilepsy and/or neurodevelopmental disorders in each case caused by any mutation of the SCN2A gene. Under the License Agreement, the Company will conduct, at its own cost and expense, the research and development activities assigned to it under the research plan. In addition, the Company is responsible for reimbursing RogCon for any costs associated with research and development activities RogCon performs at the request of the Company. As part of the agreement, the Company agreed to provide up-front consideration of $2.1 million. Subsequent to September 11, 2019, the Company will reimburse RogCon for its out-of-pocket costs incurred for activities performed under the License Agreement. The Company expenses these costs as incurred as research and development. Expenses incurred for all periods presented were not material. Additionally, the Company may pay RogCon a milestone payment of $3.0 million and profit share payments. The $3.0 million milestone payment will become due when the first profit share payment has become due and certain contingent payments have become payable to Ionis under the Ionis Collaboration Agreement, which are subject to the Company exercising its option to obtain license rights to a development candidate, as well as other contingent events. The profit share payments will be based on a low-double-digit percentage of net profits, depending on sales volume. The License Agreement, unless earlier terminated, will continue until the latest of: (i) expiration of all patent rights within RogCon patents, (ii) the Company and its affiliates certify they have abandoned the research, development and commercialization of product with no intention to re-establish such activities, and (iii) no third party is obligated to pay the Company or its affiliates any amounts that comprise net sublicense revenue. Either party may terminate the License Agreement for material breach or insolvency of the other party. Additionally, the Company may terminate for convenience with prior written notice to RogCon. Upon termination by either party, all rights and licenses granted by RogCon to the Company will revert back to RogCon. Ionis Collaboration Agreement Under the Ionis Collaboration Agreement, both parties participated in research activities related to the downregulation of SCN2A gene products associated with the treatment of any and all forms of epilepsy and/or neurodevelopmental disorders in each case caused by any mutation of the SCN2A gene, other than one severe type of epilepsy. Ionis was also responsible for identifying a development candidate and conducting an IND-enabling toxicology study. The Company reimbursed Ionis for out-of-pocket costs incurred related to the research activities, identification of a development candidate and conducting an IND-enabling toxicology study. The reimbursement of out-of-pocket costs was recognized as research and development expense as incurred. Expense for the years ended December 31, 2024 and 2023 was immaterial. Ionis granted the Company an exclusive option to obtain the rights and license to further develop and commercialize a development candidate in the field of epilepsy and neurodevelopmental disorders, other than Dravet Syndrome, following the results of the investigational new drug application-enabling toxicology study. The Company exercised this exclusive option in January 2022, paid a $2.0 million license fee and recognized the license fee in research and development expenses in the consolidated statement of operations during the year ended December 31, 2022. After option exercise, the Company is responsible for clinical development and commercialization of the development candidate. Additionally, in July 2023, the Company paid a milestone payment of $6.9 million to Ionis, which was earned in the second quarter of 2023 upon initiation of the Company's elsunersen EMBRAVE study, and was recognized in research and development expenses in the consolidated statement of operations during the year ended December 31, 2023. Ionis may be entitled to additional development milestone payments, additional milestone payments, and sales royalties or sublicense fees. The Ionis Collaboration Agreement will continue until the expiration of all payment obligations to Ionis, unless earlier terminated. Either party may terminate the Ionis Collaboration Agreement upon material breach or insolvency of the other party. Ionis may terminate if the Company fails to achieve a performance milestone. The Company may terminate for convenience with prior written notice to Ionis. Upon termination by the Company for convenience, the Company will stop selling all products, subject to certain wind-down provisions, and all products will revert back to Ionis.

Fair Value of Financial Assets

12 Months Ended

Dec. 31, 2024

Fair Value Disclosures [Abstract]

Fair Value of Financial Assets


	As of December 31, 2024
	Level 1		Level 2		Level 3		Total
Assets:
Money market funds	$	128,652	$	—	$	—	$	128,652

Debt securities issued by U.S. government agencies	144,644		—		—		144,644
Corporate debt securities	—		107,852		—		107,852
Commercial paper	—		8,794		—		8,794
Other debt securities	—		8,821		—		8,821
	$	273,296	$	125,467	$	—	$	398,763

The Company estimates the fair value of its marketable securities classified as Level 2 by taking into consideration valuations obtained from third-party pricing sources. These pricing sources utilize industry standard valuation models, including both income and market-based approaches, for which all significant inputs are observable, either directly or indirectly, to estimate fair value. These inputs include market pricing based on real-time trade data for the same or similar securities, issuer credit spreads, benchmark yields, and other observable inputs. The Company validates the prices provided by its third-party pricing sources by understanding the models used, obtaining market values from other pricing sources and analyzing pricing data in certain instances.

- Definition

The entire disclosure for the fair value of financial instruments (as defined), including financial assets and financial liabilities (collectively, as defined), and the measurements of those instruments as well as disclosures related to the fair value of non-financial assets and liabilities. Such disclosures about the financial instruments, assets, and liabilities would include: (1) the fair value of the required items together with their carrying amounts (as appropriate); (2) for items for which it is not practicable to estimate fair value, disclosure would include: (a) information pertinent to estimating fair value (including, carrying amount, effective interest rate, and maturity, and (b) the reasons why it is not practicable to estimate fair value; (3) significant concentrations of credit risk including: (a) information about the activity, region, or economic characteristics identifying a concentration, (b) the maximum amount of loss the entity is exposed to based on the gross fair value of the related item, (c) policy for requiring collateral or other security and information as to accessing such collateral or security, and (d) the nature and brief description of such collateral or security; (4) quantitative information about market risks and how such risks are managed; (5) for items measured on both a recurring and nonrecurring basis information regarding the inputs used to develop the fair value measurement; and (6) for items presented in the financial statement for which fair value measurement is elected: (a) information necessary to understand the reasons for the election, (b) discussion of the effect of fair value changes on earnings, (c) a description of [similar groups] items for which the election is made and the relation thereof to the balance sheet, the aggregate carrying value of items included in the balance sheet that are not eligible for the election; (7) all other required (as defined) and desired information.

+ References

Reference 1: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 107
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-107

Reference 2: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
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-Name Accounting Standards Codification
-Section 55
-Paragraph 100
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-100

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
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-Section 50
-Paragraph 2
-Subparagraph (c)(3)
-Publisher FASB
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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-Section 50
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-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2E

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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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Reference 10: http://www.xbrl.org/2003/role/disclosureRef
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Reference 11: http://www.xbrl.org/2003/role/disclosureRef
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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 940
-SubTopic 820
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147478119/940-820-50-1

+ Details

Cash Equivalents and Marketable Securities - Investment Profile (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Debt Securities, Available-for-sale [Line Items]
Marketable securities	$ 177,195	$ 0
Cash equivalents, at carrying value and debt securities, available-for-sale, amortized cost	398,109
Cash equivalents and debt securities, available-for-sale, accumulated gross unrealized gain, current, before tax	654
Cash equivalents and debt securities, available-for-sale, accumulated gross unrealized loss, current, before tax	0
Cash equivalents and debt securities, available-for-sale, fair value disclosure	398,763
Money market funds
Debt Securities, Available-for-sale [Line Items]
Cash equivalents, at carrying value	128,652
Cash equivalents, accumulated gross unrealized gain, current, before tax	0
Cash equivalents, accumulated gross unrealized loss, current, before tax	0
Cash equivalents, fair value disclosure	128,652
Debt securities issued by U.S. government agencies
Debt Securities, Available-for-sale [Line Items]
Cash equivalents, at carrying value	15,953
Cash equivalents, accumulated gross unrealized gain, current, before tax	2
Cash equivalents, accumulated gross unrealized loss, current, before tax	0
Cash equivalents, fair value disclosure	15,955
Debt securities, available-for-sale, amortized cost, current	128,215
Debt securities, available-for-sale, accumulated gross unrealized gain, current, before tax	475
Debt securities, available-for-sale, accumulated gross unrealized loss, current, before tax	0
Marketable securities	128,690
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Debt securities, available-for-sale, amortized cost, current	107,695
Debt securities, available-for-sale, accumulated gross unrealized gain, current, before tax	157
Debt securities, available-for-sale, accumulated gross unrealized loss, current, before tax	0
Marketable securities	107,852
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Debt securities, available-for-sale, amortized cost, current	8,775
Debt securities, available-for-sale, accumulated gross unrealized gain, current, before tax	19
Debt securities, available-for-sale, accumulated gross unrealized loss, current, before tax	0
Marketable securities	$ 8,794
Other debt securities
Debt Securities, Available-for-sale [Line Items]
Debt securities, available-for-sale, amortized cost, current		8,819
Debt securities, available-for-sale, accumulated gross unrealized gain, current, before tax		1
Debt securities, available-for-sale, accumulated gross unrealized loss, current, before tax		0
Marketable securities		$ 8,820

Property and Equipment, Net (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Property, Plant and Equipment [Line Items]
Property and equipment, gross	$ 1,593	$ 1,593
Less: Accumulated depreciation	(1,363)	(1,005)
Property and equipment, net	230	588
Depreciation	358	432
Computer equipment
Property, Plant and Equipment [Line Items]
Property and equipment, gross	689	689
Office furniture and equipment
Property, Plant and Equipment [Line Items]
Property and equipment, gross	545	545
Laboratory equipment
Property, Plant and Equipment [Line Items]
Property and equipment, gross	$ 359	$ 359

Cash Equivalents and Marketable Securities

12 Months Ended

Dec. 31, 2024

Cash and Cash Equivalents [Abstract]

Cash Equivalents and Marketable Securities

The following is a summary of the Company's investment portfolio as of December 31, 2024 (in thousands). The Company did not hold any cash equivalents or marketable securities as of December 31, 2023.


	As of December 31, 2024
			Gross Unrealized					Estimated
	Cost		Gains		Losses			Fair Value
Cash equivalents:
Money market funds	$	128,652	$	—		$	—	$	128,652
Debt securities issued by U.S. government agencies	15,953		2			—		15,955
Available-for-sale securities:
Debt securities issued by U.S. government agencies	128,215		475			—		128,690
Corporate debt securities	107,695		157			—		107,852
Commercial paper	8,775		19			—		8,794
Other debt securities	8,819		1			—		8,820
Total cash equivalents and marketable securities	$	398,109	$	654		$	—	$	398,763

Contractual maturities of the marketable securities at each balance sheet are as follows (in thousands):


	December 31, 2024		December 31, 2023
Within one year	$	177,195	$	—
After one year through five years	76,961		—
Total	$	254,156	$	—

Securities are evaluated for impairment at the end of each reporting period. The Company did not record any impairment related to its available-for-sale securities during the years ended December 31, 2024 and 2023.

Stock-Based Compensation - Summary of Restricted Stock Activity (Details) - Restricted Stock	12 Months Ended
	Dec. 31, 2024 $ / shares shares
Shares
Beginning balance (in shares) \| shares	47,145
Issued (in shares) \| shares	208,997
Vested (in shares) \| shares	(19,576)
Forfeited (in shares) \| shares	(12,093)
Ending balance (in shares) \| shares	224,473
Weighted Average Grant Date Fair Value
Weighted average grant date fair value beginning balance (in dollars per share) \| $ / shares	$ 202.27
Issued (in dollars per share) \| $ / shares	53.35
Vested (in dollars per share) \| $ / shares	201.07
Forfeited (in dollars per share) \| $ / shares	53.90
Weighted average grant date fair value ending balance (in dollars per share) \| $ / shares	$ 71.71

Employee Benefit Plan	12 Months Ended
Employee Benefit Plan	Dec. 31, 2024
Retirement Benefits [Abstract]
Employee Benefit Plan	Employee Benefit Plan The Company has a defined contribution savings plan under Section 401(k) of the Internal Revenue Code for eligible employees. The plan covers substantially all employees who meet a minimum age requirement and allows participants to defer a portion of their annual compensation on a pre-tax basis. Under the plan, the Company is not obligated to match any participant contributions. The Company made contributions of $1.2 million and $1.0 million during the years ended December 31, 2024 and 2023, respectively.

- Definition

The entire disclosure for retirement benefits.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 715
-SubTopic 70
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480794/715-70-50-1

Reference 2: http://www.xbrl.org/2003/role/exampleRef
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-Section 55
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-URI https://asc.fasb.org/1943274/2147480482/715-20-55-17

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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Reference 14: http://www.xbrl.org/2003/role/disclosureRef
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480266/715-60-50-3

+ Details

Stock-Based Compensation - Schedule of Fair Value of Stock Option Awards on the Grant Date Using the Black-Scholes Option Valuation Model (Details) - $ / shares	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Expected dividend yield	0.00%
Shares reserved for exercise of outstanding stock options
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Risk-free interest rate	4.01%	3.60%
Expected term (in years)	6 years 10 days	6 years
Expected volatility	96.60%	88.40%
Expected dividend yield	0.00%	0.00%
Weighted average grant-date fair value per share (in dollars per share)	$ 41.25	$ 29.73

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

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-Topic 718
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-Section 35
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+ Details

Segment Information

12 Months Ended

Dec. 31, 2024

Segment Reporting [Abstract]

Segment Information

The Company has one operating segment. The Company's operating segment discovers and develops therapies for CNS disorders. The Company's chief operating decision maker ("CODM"), its Chief Executive Officer, manages the Company's operations on a consolidated basis for the purposes of assessing performance and allocating resources based on net loss that also is reported on the consolidated statement of operations as consolidated net loss. Net loss is used by the CODM to make key strategic and operational decisions. The operating segment's revenue is derived from a single domestic collaboration agreement from which the segment licenses certain development or product candidates and has performed research and development services. The measure of segment assets is reported on the balance sheet as total consolidated assets. The majority of the Company's long-lived assets are held in the United States.

The following table presents selected financial information about the Company's single operating segment for the year ended December 31, 2024 (in thousands):


	December 31,
	2024		2023
Collaboration revenue	$	8,553	$	2,447
Program-specific expenses:
Ulixacaltamide(a)	69,172		19,464
Vormatrigine	18,360		6,275
Relutrigine	6,273		3,996
Elsunersen	2,135		14,595
Other early stage assets	4,634		7,055
Personnel-related expenses	42,853		33,160
Stock-based compensation expense	41,361		24,857
Depreciation expense	358		432
Other segment expenses(b)	23,572		18,986
Interest income	17,346		3,096
Consolidated net loss	$	(182,819)	$	(123,277)

(a) Includes non-cash research and development of $2.5 million in the year ended December 31, 2024 associated with the Company's collaboration with Tenacia

(b) Other segment expenses includes research and development and general and administrative costs not attributable to a specific program.

- Definition

The entire disclosure for reporting segments including data and tables. Reportable segments include those that meet any of the following quantitative thresholds a) it's reported revenue, including sales to external customers and intersegment sales or transfers is 10 percent or more of the combined revenue, internal and external, of all operating segments b) the absolute amount of its reported profit or loss is 10 percent or more of the greater, in absolute amount of 1) the combined reported profit of all operating segments that did not report a loss or 2) the combined reported loss of all operating segments that did report a loss c) its assets are 10 percent or more of the combined assets of all operating segments.

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+ Details

Income Taxes (Tables)

12 Months Ended

Dec. 31, 2024

Income Tax Disclosure [Abstract]

Schedule of Effective Income Tax Rate Reconciliation

The reconciliation of the U.S. statutory income tax rate to the Company’s effective tax rate is as follows:


	Year Ended December 31,
	2024		2023
Federal statutory income tax rate	21.0	%	21.0	%
Federal and state research and development credits	3.9	%	3.5	%
State taxes, net of federal benefit	3.5	%	4.3	%
Changes in tax rates	(3.2)	%	2.9	%
Non-deductible executive compensation	(2.6)	%	(1.4)	%
Non-deductible items	(0.8)	%	—	%
Stock-based compensation	(0.9)	%	(1.3)	%
Change in valuation allowance	(20.7)	%	(29.7)	%
Other	(0.2)	%	0.7	%
Effective income tax rate	—	%	—	%

Schedule of Deferred Tax Assets and Liabilities

Net deferred tax assets consisted of the following (in thousands):


	December 31,
	2024		2023
Deferred tax assets:
Net operating loss carryforwards	$	90,108	$	74,528
Capitalized research expenditures	65,836		48,954
Research and development credits	30,582		23,451
Amortization	20,600		23,929
Stock-based compensation	8,784		7,697
Accrued expenses	2,333		1,147
Leases	344		676
Deferred revenue	—		692
Total gross deferred tax assets	218,587		181,074
Less: Valuation allowance	(218,303)		(180,515)
Net deferred tax assets	$	284	$	559
Deferred tax liabilities:
Operating lease right-of-use asset	(284)		(559)
Total gross deferred tax liabilities	(284)		(559)
Net deferred tax assets	$	—	$	—

Net Loss per Share (Tables)

12 Months Ended

Dec. 31, 2024

Earnings Per Share [Abstract]

Summary of antidilutive securities excluded from computation of earnings per share

The following potential common shares, presented based on amounts outstanding at each period end, were excluded from the calculation of diluted net loss per share attributable to common stockholders for the periods indicated because including them would have been anti-dilutive:


	Year Ended December 31,
	2024	2023
Outstanding stock options	1,809,343	666,163
Unvested restricted stock units	224,473	47,145
Potential shares issuable under the ESPP	7,315	24,972
	2,041,131	738,280

Audit Information	12 Months Ended
Audit Information	Dec. 31, 2024
Audit Information [Abstract]
Auditor Firm ID	42
Auditor Name	Ernst & Young LLP
Auditor Location	Boston, Massachusetts

Commitments and Contingencies - Narrative (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023	May 31, 2021
Loss Contingencies [Line Items]
Operating lease right-of-use assets	$ 1,131	$ 2,064
Present value of operating lease liabilities	$ 1,369	$ 2,495
Office Space In Boston, Massachusetts
Loss Contingencies [Line Items]
Percentage increase in annual base rent			2.00%
Operating lease right-of-use assets			$ 4,100
Present value of operating lease liabilities			$ 4,100

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References + Details

Income Taxes - Narrative (Details) - USD ($) $ in Thousands	12 Months Ended
Income Taxes - Narrative (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Operating Loss Carryforwards [Line Items]
Operating loss carryforwards, domestic	$ 342,400	$ 282,800
Operating loss carryforwards, subject to expiration	7,700
Operating loss carryforwards, not subject to expiration	334,700
Operating loss carryforwards, state and local	289,700	239,400
Research and development credits	30,582	23,451
Valuation allowance	218,303	180,515
Valuation allowance, increase (decrease), amount	37,800	36,600
Federal
Operating Loss Carryforwards [Line Items]
Research and development credits	27,300	20,400
State
Operating Loss Carryforwards [Line Items]
Research and development credits	$ 4,100	$ 3,800

Subsequent Events	12 Months Ended
Subsequent Events	Dec. 31, 2024
Subsequent Events [Abstract]
Subsequent Events	Subsequent Events The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the consolidated financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. The Company has concluded that no subsequent events have occurred that require disclosure other than those noted below. At-the-Market Offerings Subsequent to December 31, 2024, the Company issued and sold a total of 694,212 shares under the amended March 2024 Sales Agreement for aggregate net proceeds of $54.9 million after deducting commissions and offering expenses payable by the Company.

Property and Equipment, Net

12 Months Ended

Dec. 31, 2024

Property, Plant and Equipment [Abstract]

Property and Equipment, Net

Property and equipment, net consisted of the following (in thousands):


	December 31,
	2024		2023
Computer equipment	$	689	$	689
Office furniture and equipment	545		545
Laboratory equipment	359		359
Total property and equipment	1,593		1,593
Less: Accumulated depreciation	(1,363)		(1,005)
Property and equipment, net	$	230	$	588

Depreciation expense was $0.4 million and $0.4 million for the years ended December 31, 2024 and 2023, respectively.

Summary of Significant Accounting Policies

12 Months Ended

Dec. 31, 2024

Accounting Policies [Abstract]

Summary of Significant Accounting Policies

Basis of Presentation

The accompanying consolidated financial statements have been prepared in conformity with generally accepted accounting principles in the United States of America (“GAAP”). Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification (“ASC”) and ASUs of the FASB.

Principles of Consolidation

The consolidated financial statements include the accounts of the Company and its wholly owned subsidiary, Praxis Security Corporation. All intercompany transactions have been eliminated in consolidation.

Use of Estimates

The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the consolidated financial statements and the reported amounts of expenses during the reporting periods. Significant estimates and assumptions reflected in these consolidated financial statements include, but are not limited to, accrued and prepaid research and development expense, collaboration revenue, stock-based compensation expense and the recoverability of the Company’s net deferred tax assets and related valuation allowance. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ materially from those estimates.

Cash, Cash Equivalents and Restricted Cash

The Company considers all highly liquid investments purchased with original maturities of 90 days or less at acquisition to be cash equivalents. Cash and cash equivalents include cash held in banks and amounts held in interest-bearing insured liquid deposit funds and interest-bearing money market funds. Cash equivalents are carried at cost, which approximates their fair market value.

Restricted cash comprises letters of credit for the benefit of the landlord in connection with the Company’s lease facilities. Restricted cash is classified as either current or non-current based on the terms of the underlying lease arrangement.


	December 31,
	2024		2023
Cash and cash equivalents	$	215,372	$	81,300
Restricted cash	416		416
Total cash, cash equivalents and restricted cash as shown on the consolidated statements of cash flows	$	215,788	$	81,716

Marketable Securities

The Company may invest its excess cash in money market funds and debt instruments of the U.S. Treasury, financial institutions, corporations and U.S. government agencies with strong credit ratings and an investment grade rating at or above A-1 or P-1 by two of the three nationally recognized statistical rating organizations. The Company does not believe that it is exposed to more than a nominal amount of credit risk related to any marketable securities it may invest in. The Company has established guidelines relative to diversification and maturities that maintain safety and liquidity, and periodically reviews and modifies these guidelines to maximize trends in yields and interest rates without compromising safety and liquidity.

The Company classifies its investments in debt instruments as available-for-sale. Available-for-sale investments are reported at fair value at each balance sheet date, and include any unrealized holding gains and losses in accumulated other comprehensive loss, a component of stockholders’ equity. Realized gains and losses are included in the Company's consolidated statements of operations. All of the Company's available-for-sale securities are available for use in its current operations. The Company classifies marketable securities as either cash equivalents, short-term or long-term based on their stated maturity dates as it is more-likely-than-not that the Company will hold these assets through to their maturity dates. The Company held $254.2 million of marketable securities as of December 31, 2024. The Company did not hold any marketable securities as of December 31, 2023.

The Company evaluates securities for impairment at the end of each reporting period. Impairment is evaluated considering numerous factors, and their relative significance varies depending on the situation. Factors considered include whether a decline in fair value below the amortized cost basis is due to credit-related factors or non-credit-related factors, the financial condition and near-term prospects of the Company, and the Company's intent and ability to hold the investment to allow for an anticipated recovery in fair value. A credit-related impairment is recognized as an allowance on the balance sheet with a corresponding adjustment to earnings. Any impairment that is not credit-related is recognized in other comprehensive loss, net of applicable taxes unless deemed other than temporary.

Concentrations of Credit Risk and Significant Suppliers and License Agreements

Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of cash, cash equivalents, restricted cash and marketable securities. The Company's investment portfolio may include money market funds, marketable debt securities, including debt securities issued by U.S. government agencies and corporations, and commercial paper. The Company's investments are limited to investment-grade securities with strong credit ratings with the objective of maintaining safety and liquidity. The Company also maintains deposits in accredited financial institutions in excess of federally insured limits. Bank accounts in the United States are insured by the Federal Deposit Insurance Corporation (the “FDIC”) up to $250,000. As of December 31, 2024 and 2023, the Company’s primary operating accounts significantly exceeded the FDIC limits. The Company deposits its cash in financial institutions that it believes have high credit quality, and has not experienced any losses on such accounts and does not believe it is exposed to any unusual credit risk beyond the normal credit risk associated with commercial banking relationships.

The Company is dependent on third-party manufacturers to supply materials for research and development activities of its programs, including preclinical and clinical testing. In particular, the Company relies, and expects to continue to rely, on a small number of third-party manufacturers to produce and process its current and potential product candidates and to manufacture supply of its current and potential product candidates for preclinical and clinical activities. These programs could be adversely affected by a significant interruption in the supply of the necessary materials. The Company is also dependent on third parties who provide license rights used in the development of certain programs. The Company could experience delays in the development of its programs if any of these license agreements are terminated, if the Company fails to meet the obligations required under its arrangements, or if the Company is unable to successfully secure new strategic alliances or licensing agreements.

Off-Balance Sheet Risk

As of December 31, 2024 and 2023, the Company had no off-balance sheet risk such as foreign exchange contracts, option contracts or other hedging arrangements.

Fair Value Measurements

Certain assets and liabilities of the Company are carried at fair value under GAAP. The fair values of the Company’s financial assets and liabilities reflect the Company’s estimate of 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 must maximize the use of observable inputs and minimize the use of unobservable inputs. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable:

•Level 1: Quoted prices in active markets for identical assets or liabilities.

•Level 2: Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data.

•Level 3: Unobservable inputs that are supported by little or no market activity that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques.

To the extent the valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement.

An entity may choose to measure many financial instruments and certain other items at fair value at specified election dates. Subsequent unrealized gains and losses on items for which the fair value option has been elected will be reported in earnings.

Items measured at fair value on a recurring basis include cash equivalents and marketable securities (Note 4 and Note 5). The carrying amounts reflected in the consolidated balance sheets for cash, prepaid expenses and other current assets, accounts payable and accrued expenses approximate their fair values due to the short-term nature of these assets and liabilities.

Property and Equipment

Property and equipment are recorded at cost. Depreciation is calculated using the straight-line method over the following estimated useful lives of the assets:


			Estimated Useful Life
Office furniture and equipment			5 years
Laboratory equipment			3 years
Computer equipment			3 years

Upon disposal, retirement or sale, the cost of assets disposed of and the related accumulated depreciation are removed from the accounts and any resulting gain or loss is included in the results of operations. Expenditures for repairs and maintenance that do not improve or extend the lives of the respective assets are charged to expense as incurred.

Impairment of Long-Lived Assets

Long-lived assets consist of property and equipment. Long-lived assets to be held and used are tested for recoverability whenever events or changes in business circumstances indicate that the carrying amount of the assets may not be fully recoverable. Factors that the Company considers in deciding when to perform an impairment review include significant underperformance of the business in relation to expectations, significant

negative industry or economic trends and significant changes or planned changes in the use of the assets. If an impairment review is performed to evaluate a long-lived asset group for recoverability, the Company compares forecasts of undiscounted cash flows expected to result from the use and eventual disposition of the long-lived asset group to its carrying value. An impairment loss would be recognized when estimated undiscounted future cash flows expected to result from the use of an asset group are less than its carrying amount. The impairment loss would be based on the excess of the carrying value of the impaired asset group over its fair value, determined based on discounted cash flows. The Company did not record any impairment losses on long-lived assets during the years ended December 31, 2024 and 2023.

Leases

The Company accounts for leases in accordance with ASC 842. The Company determines if an arrangement is a lease at contract inception. Operating lease right-of-use assets represent the Company’s right to use an underlying asset for the lease term and operating lease liabilities represent its obligation to make lease payments arising from the lease. Operating right-of-use assets and liabilities are recognized at the commencement date of the lease based upon the present value of lease payments over the lease term. When determining the lease term, the Company includes options to extend or terminate the lease when it is reasonably certain that the option will be exercised.

The Company uses the implicit rate when readily determinable and uses its incremental borrowing rate when the implicit rate is not readily determinable, based upon the information available at the commencement date in determining the present value of the lease payments. The incremental borrowing rate is determined using a secured borrowing rate for the same currency and term as the associated lease.

The lease payments used to determine operating lease right-of-use assets may include lease incentives and stated rent increases. The Company’s lease agreements may include both lease and non-lease components, which the Company accounts for as a single lease component when the payments are fixed, for all classes of underlying assets. Variable payments included in the lease agreement are expensed as incurred.

The Company’s operating leases are reflected in operating lease right-of-use assets and in current operating lease liabilities and long-term operating lease liabilities in its consolidated balance sheets. The Company’s operating lease right-of-use asset as of December 31, 2024 and 2023 did not include any lease incentives. Lease expense for future lease payments is recognized on a straight-line basis over the lease term.

Collaboration Agreements

The Company analyzes its collaboration arrangements to assess whether they are within the scope of ASC Topic 808, Collaborative Arrangements ("ASC 808"), to determine whether such arrangements involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards that are dependent on the commercial success of such activities. To the extent the arrangement is within the scope of ASC 808, the Company assesses whether aspects of the arrangement between the Company and its collaboration partner are within the scope of other accounting literature, including ASC Topic 606, Revenue from Contracts with Customers ("ASC 606"). If it is concluded that some or all aspects of the arrangement represent a transaction with a customer, the Company will account for those aspects of the arrangement within the scope of ASC 606.

ASC 808 provides guidance for the presentation and disclosure of transactions in collaborative arrangements, but it does not provide recognition or measurement guidance. Therefore, if the Company concludes a counterparty to a transaction is not a customer or otherwise not within the scope of ASC 606, the Company considers the guidance in other accounting literature as applicable or by analogy to account for such transaction. The classification of transactions under the Company’s arrangements is determined based on the nature and contractual terms of the arrangement along with the nature of the operations of the participants.

Revenue Recognition

The Company enters into collaboration agreements which are within the scope of ASC 606 under which the Company may license or provide options to license certain development or product candidates and, in certain

cases, may perform research and development services. The terms of these agreements may include payment of non-refundable, upfront fees, option exercise fees, development and commercial milestone payments and royalties on net sales of licensed products.

Under ASC 606, the Company recognizes revenue when its customer obtains control of promised goods or services in an amount that reflects the consideration which the Company expects to receive in exchange for those goods or services. To determine the appropriate amount of revenue to be recognized for agreements determined to be within the scope of ASC 606, the Company performs the following five steps: (1) identify the contract with a customer; (2) identify the performance obligations in the contract; (3) determine the transaction price; (4) allocate the transaction price to the performance obligations in the contract; and (5) recognize revenue when or as the Company satisfies each performance obligation. The Company only applies the five-step model to contracts when it is probable that the Company will collect consideration it is entitled to in exchange for the goods or services it transfers to the customer.

At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations. The Company then assesses whether each promised good or service is distinct. When the Company offers options for additional goods or services, such as an option to obtain a license, it evaluates whether such options are material rights that should be treated as separate performance obligations.

The Company estimates the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract. The consideration may include fixed consideration or variable consideration. At the inception of each agreement that includes variable consideration, the Company evaluates the amount of potential payments and the likelihood that the payments will be received to estimate the amount expected to be received. The amount of variable consideration included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal of the cumulative revenue recognized will not occur in a future period.

The Company recognizes as revenue the amount of the transaction price that it allocated to the respective performance obligation when or as each performance obligation is satisfied, either at a point in time or over time. If the performance obligation is satisfied over time, the Company recognizes revenue based on the use of an output or input method. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. For example, for research and development services that are recognized over time, the Company measures its progress using an input method. The input methods used are based on the effort the Company expends or costs it incurs toward the satisfaction of its performance obligation. The Company estimates the amount of effort it expends, including the time it estimates it will take to complete the activities, or costs it incurs in a given period, relative to the estimated total effort or costs to satisfy the performance obligation. This results in a percentage that is multiplied by the transaction price to determine the amount of revenue recognized each period. If the estimates or judgements change over the course of the collaboration, they may affect the timing and amount of revenue that is recognized in the current and future periods.

Upfront payments are recorded as contract liabilities within deferred revenue on the consolidated balance sheets until the Company performs its obligations under these agreements. Amounts are recorded as accounts receivable when the Company’s right to consideration is unconditional.

As of December 31, 2024, the Company had an option and license agreement with UCB Biopharma SRL ("UCB"), which it entered into in December 2022. Additionally, it had a collaboration and license agreement with Tenacia Biotechnology (Shanghai) Co., Ltd ("Tenacia"), which it entered into in January 2024. The accounting related to these agreements is outlined in Note 9, Collaboration and License Agreements.

Research and Development Costs

Research and development costs are expensed as incurred. Research and development expenses consist of costs incurred in performing research and development activities, including salaries, stock-based compensation and benefits, facilities costs, depreciation, third-party license fees and external costs of outside vendors engaged to conduct preclinical development activities and clinical trials as well as to manufacture research and development

materials. Non-refundable prepayments for goods or services that will be used or rendered for future research and development activities are deferred and capitalized. Such amounts are recognized as an expense as the goods are delivered or the related services are performed or until it is no longer expected that the goods will be delivered or the services rendered. Costs incurred to obtain licenses are recognized as research and development expense as incurred if the technology licensed has no alternative future uses.

The Company has entered into various research and development related contracts with parties both inside and outside of the United States. These agreements are cancellable, and related fees are recorded as research and development expenses as incurred. The Company records accrued liabilities and prepaid expenses for estimated ongoing research costs. When evaluating the adequacy of the accrued liabilities and prepaid expenses, the Company analyzes progress of the studies or clinical trials, including the phase or completion of events, invoices received and contracted costs. Significant judgments and estimates are made in determining the accrued and prepaid balances at the end of any reporting period. Actual results could differ from the Company’s estimates. The Company’s historical estimates have not been materially different from the actual costs.

Patent-Related Costs

Patent-related costs incurred in connection with patent applications are expensed as incurred due to the uncertainty about the recovery of the expenditure. Amounts incurred are classified as general and administrative expenses in the accompanying consolidated statement of operations.

Stock-Based Compensation

The Company accounts for all stock-based awards granted to employees and non-employees as stock-based compensation expense at fair value in accordance with FASB ASC Topic 718, Compensation—Stock Compensation ("ASC 718"). For stock-based awards issued to employees, non-employees and members of the Company’s board of directors (the “Board”) for their services on the Board, the Company measures the estimated fair value of the stock-based award on the date of the grant. The Company recognizes compensation expense for those awards granted to employees and members of the Board over the requisite service period, which is generally the vesting period of the respective award. For non-employee awards, compensation expense is recognized as the services are provided, which is generally ratably over the vesting period. The Company issues stock-based awards with service-based vesting conditions and records the expense for these awards on a straight-line basis over the vesting period. To date, the Company has not issued any stock-based awards with performance or market-based vesting conditions. The Company accounts for forfeitures as they occur.

The Company classifies stock-based compensation expense in its consolidated statement of operations in the same manner in which the award recipient’s salary or service payments are classified.

The fair value of each stock option award is estimated on the date of grant using the Black-Scholes option pricing model, which requires inputs of subjective assumptions, including: (i) the expected volatility of the Company’s stock; (ii) the expected term of the award; (iii) the risk-free interest rate; (iv) expected dividends; and (v) the fair value of common stock. Due to the lack of Company-specific historical and implied volatility data, the Company determines the volatility for awards granted based on an analysis of reported data for a group of guideline publicly-traded companies that issued options with substantially similar terms. For this analysis, the Company selects companies with comparable characteristics including enterprise value, risk profiles, and with historical share price information sufficient to meet the expected life of the stock-based awards. The Company determines expected volatility using a weighted average of the historical volatilities of the guideline group of companies. The Company expects to continue to apply this process until such time as it has adequate historical data regarding the volatility of its own traded stock price. As permitted under ASC 718, the Company has elected to use the contractual term as the expected term for certain non-employee awards, on an award-by-award basis. For all other awards, the expected term of the Company’s stock options has been determined utilizing the “simplified” method whereby the expected term equals the average of the vesting term and the original contractual term of the option, on a weighted basis based on the vesting of each tranche. The Company utilizes this method as it has insufficient historical exercise data to provide a reasonable basis upon which to estimate the expected term. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for instruments with a term commensurate with the expected term assumption. The expected dividend yield is assumed to be zero

as the Company has never paid dividends, and does not have current plans to pay any dividends on its common stock. Subsequent to the IPO, the fair value of the common stock underlying the Company's stock-based awards is the closing price of the Company's common stock on the date of grant.

The grant date fair value of restricted stock units is estimated based on the fair value of the Company's underlying common stock on the date of grant.

Foreign Currency

Monetary assets and liabilities resulting from transactions denominated in currencies other than the functional currency are remeasured into the functional currency at exchange rates prevailing at the balance sheet date. Non-monetary assets and liabilities denominated in foreign currencies are measured using historical exchange rates prevailing at the date of the transaction and are not subsequently remeasured. Exchange gains or losses arising from foreign currency transactions are included in the determination of net loss. There were no material foreign currency gains or losses for the years ended December 31, 2024 and 2023.

Income Taxes

Deferred tax assets and liabilities are determined on the basis of the differences between the consolidated financial statements and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Changes in deferred tax assets and liabilities are recorded in the provision for income taxes. The Company assesses the likelihood that its deferred tax assets will be recovered from future taxable income and, to the extent that it believes based upon the weight of available evidence it is more likely than not that all or a portion of the deferred tax assets will not be realized, a valuation allowance is established.

The Company accounts for uncertain tax positions recognized in the consolidated financial statements by prescribing a more-likely-than-not threshold for financial statement recognition and measurement of a tax position taken or expected to be taken in a tax return. The provision for income taxes includes the effects of any resulting tax reserves, or unrecognized tax benefits, that are considered appropriate as well as the related net interest and penalties.

Comprehensive Loss

Comprehensive loss includes net loss and certain changes in stockholders’ equity that are excluded from net loss. For the years ended December 31, 2024 and 2023, comprehensive loss consists of net loss and changes in unrealized gains and losses on marketable securities.

Common Stock Warrants

The Company accounts for warrants to purchase shares of its common stock in accordance with the guidance in FASB ASC No. 480, Distinguishing Liabilities from Equity ("ASC 480") and ASC No. 815, Derivatives and Hedging ("ASC 815"). The Company classifies warrants issued for the purchase of shares of its common stock as either equity or liability instruments based on an assessment of the specific terms and conditions of each respective contract. Such assessment includes determining whether the warrants are freestanding financial instruments or embedded in a host instrument, whether the warrants are liabilities within the scope of ASC 480, whether the warrants meet the definition of a derivative in ASC 815 and whether the warrants meet the requirements for equity classification pursuant to the indexation and equity classification criteria in ASC 815. The Company determines the classification for its warrants at the time of issuance and updates its assessment, as necessary. Warrants that meet all of the criteria for equity classification are recorded as a component of additional paid-in capital. Equity classified warrants are accounted for at fair value on the issuance date with no changes in fair value recognized after the issuance date.

Net Loss per Share

Basic net loss per share is computed by dividing net loss by the weighted average number of common shares outstanding for the period. The calculation of weighted average number of common shares outstanding excludes shares of restricted common stock that are not vested but includes shares of common stock underlying pre-funded warrants. Diluted net loss per share is computed by dividing net loss by the weighted average number of common

shares outstanding for the period, after giving consideration to the dilutive effect of potentially dilutive common shares. For purposes of this calculation, outstanding options to purchase shares of common stock, unvested shares of restricted common stock and potential shares issuable under the 2020 ESPP are considered potentially dilutive common shares. The Company has generated a net loss in all periods presented so the basic and diluted net loss per share are the same, as the inclusion of the potentially dilutive securities would be anti-dilutive.

Recent Accounting Pronouncements

Recently Adopted Accounting Pronouncements

In November 2023, the FASB issued ASU No. 2023-07, Segment Reporting (Topic 280) - Improvements to Reportable Segment Disclosures ("ASU 2023-07"). ASU 2023-07 improves reportable segment disclosure requirements, primarily through enhanced disclosures about significant segment expenses. The amendments included in this ASU apply to all public entities that are required to report segment information in accordance with Topic 280, including those with a single reportable segment. The amendments are effective for fiscal years beginning after December 15, 2023, and interim periods within fiscal years beginning after December 15, 2024. The Company adopted ASU 2023-07 as of December 31, 2024. The amendments were applied retroactively to all prior periods presented in the consolidated financial statements and are reflected within Note 17.

Recently Issued Accounting Pronouncements

In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740) - Improvements to Income Tax Disclosures ("ASU 2023-09"). ASU 2023-09 provides more transparency about income tax information through improvements to income tax disclosures primarily related to the rate reconciliation and incomes taxes paid information. For public companies, the amendments are effective for annual periods beginning after December 15, 2024 and should be applied prospectively. The Company has determined that the effects of adopting the amendments in ASU 2023-09 will only impact its disclosures and not have a material impact on its consolidated financial position and the results of its operations when such amendment is adopted.

In November 2024, the FASB issued ASU No. 2024-03, Income Statement-Reporting Comprehensive Income-Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses ("ASU 2024-03"), requiring public entities to disclose additional information about specific expense categories in the notes to the financial statements on an interim and annual basis. ASU 2024-03 is effective for fiscal years beginning after December 15, 2026, and for interim periods beginning after December 15, 2027, with early adoption permitted. The amendments in this update should be applied either prospectively to financial statements issued for reporting periods after the effective date of this update, or retrospectively to any or all prior periods presented in the financial statements. The Company is currently evaluating the impact of adopting ASU 2024-03 on its consolidated financial statements.

Net Loss per Share

12 Months Ended

Dec. 31, 2024

Earnings Per Share [Abstract]

Net Loss per Share


	Year Ended December 31,
	2024	2023
Outstanding stock options	1,809,343	666,163
Unvested restricted stock units	224,473	47,145
Potential shares issuable under the ESPP	7,315	24,972
	2,041,131	738,280

Common shares issuable upon exercise of the pre-funded warrants that were sold and remain outstanding in connection with the January 2024 and April 2024 underwritten public offerings are included in the calculation of weighted average number of common shares outstanding for the year ended December 31, 2024. Consistent with the guidance in ASC 260-10-45-13, the underlying common shares are issuable for little to no consideration and there are no vesting conditions or contingencies associated with the warrants. Accordingly, the aggregate number of common shares underlying the pre-funded warrants have been considered outstanding for purposes of the calculation of net loss per share from the date of issuance.

Cash Equivalents and Marketable Securities (Tables)

12 Months Ended

Dec. 31, 2024

Cash and Cash Equivalents [Abstract]

Schedule of Debt Securities, Available-for-Sale


	As of December 31, 2024
			Gross Unrealized					Estimated
	Cost		Gains		Losses			Fair Value
Cash equivalents:
Money market funds	$	128,652	$	—		$	—	$	128,652
Debt securities issued by U.S. government agencies	15,953		2			—		15,955
Available-for-sale securities:
Debt securities issued by U.S. government agencies	128,215		475			—		128,690
Corporate debt securities	107,695		157			—		107,852
Commercial paper	8,775		19			—		8,794
Other debt securities	8,819		1			—		8,820
Total cash equivalents and marketable securities	$	398,109	$	654		$	—	$	398,763

Contractual maturities of the marketable securities at each balance sheet are as follows (in thousands):


	December 31, 2024		December 31, 2023
Within one year	$	177,195	$	—
After one year through five years	76,961		—
Total	$	254,156	$	—

- Definition

Tabular disclosure of investment in debt security measured at fair value with change in fair value recognized in other comprehensive income (available-for-sale).

+ References

Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-9

Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 5: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 6: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (aa)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 7: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (aaa)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 9: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 10: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 11: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 12: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

+ Details

Collaboration and License Agreements (Details) $ / shares in Units, $ in Thousands		1 Months Ended		12 Months Ended
	Jan. 04, 2024 USD ($) obligation $ / shares shares	Dec. 31, 2024 USD ($) $ / shares	Jan. 31, 2024 USD ($)	Dec. 31, 2024 USD ($) $ / shares	Dec. 31, 2023 USD ($)	Dec. 31, 2022 USD ($) obligation
Disaggregation of Revenue [Line Items]
Number of performance obligations \| obligation	1					1
Collaboration revenue				$ 8,553	$ 2,447
UCB \| License
Disaggregation of Revenue [Line Items]
Upfront payment						$ 5,000
Milestone payments						98,500
Upfront payment						$ 5,000
Collaboration revenue				8,600	$ 2,400
UCB \| Upfront Payment
Disaggregation of Revenue [Line Items]
Contract with customer, liability, revenue recognized				$ 2,600
UCB \| Option Exercise Fee
Disaggregation of Revenue [Line Items]
Contract with customer, liability, revenue recognized		$ 6,000
Tenacia
Disaggregation of Revenue [Line Items]
Shares sold in follow-on public offering (in shares) \| shares	443,253
Sale of stock, price (in USD per share) \| $ / shares	$ 22.5605	$ 38.95		$ 38.95
Proceeds from stock offerings, net of issuance costs	$ 10,000		$ 10,000
Sale of stock, volume-weighted average price, premium	20.00%			20.00%
Sale of stock, volume-weighted average price, duration	30 days			30 days
Proceeds from collaboration arrangement			14,800
Sale of Stock, value transferred				$ 17,300
Sale of stock, value transferred in excess of consideration received				$ 2,500
Tenacia \| License
Disaggregation of Revenue [Line Items]
Upfront payment	$ 5,000		$ 4,800
Milestone payments	$ 264,000

Cash Equivalents and Marketable Securities - Contractual Maturity of Marketable Securities (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Cash and Cash Equivalents [Abstract]
Within one year	$ 177,195	$ 0
After one year through five years	76,961	0
Total	$ 254,156	$ 0

- Definition

Amount of investment in debt security measured at fair value with change in fair value recognized in other comprehensive income (available-for-sale).

+ References

+ Details

Summary of Significant Accounting Policies (Policies)

12 Months Ended

Dec. 31, 2024

Accounting Policies [Abstract]

Basis of Presentation

Principles of Consolidation

Use of Estimates

Cash, Cash Equivalents and Restricted Cash

Marketable Securities

Concentrations of Credit Risk and Significant Suppliers and License Agreements

Off-Balance Sheet Risk

As of December 31, 2024 and 2023, the Company had no off-balance sheet risk such as foreign exchange contracts, option contracts or other hedging arrangements.

Fair Value Measurements

•Level 1: Quoted prices in active markets for identical assets or liabilities.

Property and Equipment

Property and equipment are recorded at cost. Depreciation is calculated using the straight-line method over the following estimated useful lives of the assets:


			Estimated Useful Life
Office furniture and equipment			5 years
Laboratory equipment			3 years
Computer equipment			3 years

Impairment of Long-Lived Assets

Leases

Collaboration Agreements and Revenue Recognition

Collaboration Agreements

Revenue Recognition

Research and Development Costs and Patent-Related Costs

Research and Development Costs

Patent-Related Costs

Stock-Based Compensation

The Company classifies stock-based compensation expense in its consolidated statement of operations in the same manner in which the award recipient’s salary or service payments are classified.

The grant date fair value of restricted stock units is estimated based on the fair value of the Company's underlying common stock on the date of grant.

Foreign Currency

Income Taxes

Comprehensive Loss

Common Stock Warrants

Net Loss per Share

Recent Accounting Pronouncements

Recently Adopted Accounting Pronouncements

Recently Issued Accounting Pronouncements

Subsequent Events (Details) - Subsequent Event $ in Millions	2 Months Ended
	Feb. 28, 2025 USD ($) shares
Subsequent Event [Line Items]
Shares sold in follow-on public offering (in shares) \| shares	694,212
Sale of stock, gross proceeds \| $	$ 54.9

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Cash flows from operating activities:
Net loss	$ (182,819)	$ (123,277)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation expense	358	432
Stock-based compensation expense	41,360	24,857
Non-cash operating lease expense	933	837
Amortization of premiums and discounts on marketable securities, net	(5,010)	47
Non-cash collaboration and license agreement expense	2,500	0
Changes in operating assets and liabilities:
Prepaid expenses and other current assets	(8,225)	6,772
Accounts payable	6,713	(8,855)
Accrued expenses	16,110	(8,497)
Operating lease liabilities	(1,126)	(1,005)
Deferred revenue	(2,553)	(2,447)
Other	2	0
Net cash used in operating activities	(131,757)	(111,136)
Cash flows from investing activities:
Purchases of property and equipment	0	(50)
Purchases of marketable securities	(371,519)	0
Maturities of marketable securities	123,025	39,000
Net cash (used in) provided by investing activities	(248,494)	38,950
Cash flows from financing activities:
Payments of tax withholdings related to vesting of restricted stock units	(308)	(137)
Proceeds from exercise of options and employee stock purchase plan purchases	2,633	416
Net cash provided by financing activities	514,323	91,871
Increase in cash, cash equivalents and restricted cash	134,072	19,685
Cash, cash equivalents and restricted cash, beginning of period	81,716	62,031
Cash, cash equivalents and restricted cash, end of period	215,788	81,716
Reconciliation of cash, cash equivalents and restricted cash:
Cash and cash equivalents	215,372	81,300
Restricted cash	416	416
Total cash, cash equivalents and restricted cash	215,788	81,716
At-The-Market Offering
Cash flows from financing activities:
Proceeds from stock offerings, net of issuance costs	119,597	28,153
Supplemental disclosures of non-cash activities:
Offering costs from in accounts payable / accrued expenses	238	63
Follow-On Offering
Cash flows from financing activities:
Proceeds from stock offerings, net of issuance costs	377,636	63,439
Supplemental disclosures of non-cash activities:
Offering costs from in accounts payable / accrued expenses	0	184
License And Collaboration Agreement
Cash flows from financing activities:
Proceeds from stock offerings, net of issuance costs	$ 14,765	$ 0

Consolidated Balance Sheets (Parenthetical) - $ / shares	Dec. 31, 2024	Dec. 31, 2023
Statement of Financial Position [Abstract]
Preferred stock, par value (in USD per share)	$ 0.0001	$ 0.0001
Preferred stock, authorized (in shares)	10,000,000	10,000,000
Preferred stock, issued (in shares)	0	0
Preferred stock, outstanding (in shares)	0	0
Common stock, par value (in USD per share)	$ 0.0001	$ 0.0001
Common stock, authorized (in shares)	150,000,000	150,000,000
Common stock, outstanding (in shares)	19,422,358	8,791,877
Common stock, issued (in shares)	19,422,358	8,791,877

Stock-Based Compensation (Tables)

12 Months Ended

Dec. 31, 2024

Share-Based Payment Arrangement [Abstract]

Summary of restricted stock activity

The following table summarizes the Company’s restricted stock unit activity:


	Shares	Weighted Average Grant Date Fair Value
Unvested as of December 31, 2023	47,145	$	202.27
Issued	208,997	53.35
Vested	(19,576)	201.07
Forfeited	(12,093)	53.90
Unvested as of December 31, 2024	224,473	$	71.71

Summary of stock option activity

The following table summarizes the Company’s stock option activity:


	Number of Shares	Weighted Average Exercise Price per Share		Weighted Average Remaining Contractual Term	Aggregate Intrinsic Value
				(In years)	(In thousands)
Outstanding as of December 31, 2023	666,163	$	160.19	7.79	$	254
Granted	1,221,746	51.59
Exercised	(45,177)	46.24			$	1,231
Cancelled or Forfeited	(33,389)	108.85
Outstanding as of December 31, 2024	1,809,343	$	90.84	8.47	$	39,989
Exercisable as of December 31, 2024	933,936	$	120.30	7.77	$	19,033
Vested and expected to vest as of December 31, 2024	1,809,343	$	90.84	8.47	$	39,989

Schedule of fair value of stock option awards on the grant date using the Black-Scholes option valuation model


	Year Ended December 31,
	2024			2023
Risk-free interest rate	4.01		%	3.60		%
Expected term (in years)	6.03			6.00
Expected volatility	96.60		%	88.40		%
Expected dividend yield	—		%	—		%
Weighted average grant-date fair value per share	$	41.25		$	29.73

Summary of stock-based compensation expense

Stock-based compensation expense was allocated as follows (in thousands):


	Year Ended December 31,
	2024		2023
Research and development	$	15,620	$	8,055
General and administrative	25,740		16,802
Total stock-based compensation expense	$	41,360	$	24,857

Commitment and Contingencies (Tables)

12 Months Ended

Dec. 31, 2024

Commitments and Contingencies Disclosure [Abstract]

Schedule of Operating Lease, Consolidated Balance Sheets

The following table summarizes the presentation of the operating lease in the Company’s consolidated balance sheets (in thousands):


	As of December 31,
	2024		2023
Assets:
Operating lease right-of-use assets	$	1,131	$	2,064
Liabilities:
Current operating lease liabilities	$	1,259	$	1,126
Non-current portion of operating lease liabilities	110		1,369
Total lease liabilities	$	1,369	$	2,495

Schedule of Lease cost

The following table summarizes total lease costs recognized in the Company’s consolidated statements of operations (in thousands):


	For the year ended December 31,
	2024		2023
Operating lease cost	$	1,102	$	1,102
Variable lease costs	33		14
Total lease costs	$	1,135	$	1,116

The weighted average remaining lease term and weighted average incremental borrowing rate of the Company’s operating lease were as follows:


	As of December 31,
	2024		2023
Weighted average remaining lease term (in years)	1.1		2.1
Weighted average incremental borrowing rate	9.0	%	9.0	%

Schedule of Lessee, Operating Lease, Liability, Maturity

Future lease payments under non-cancelable lease agreements as of December 31, 2024 were as follows (in thousands):


Year Ended December 31,	Future Lease Payments
2025	$	1,321
2026	110
Total future lease payments	1,431
Less: interest	(62)
Present value of operating lease liabilities	$	1,369

Restricted Cash (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Cash and Cash Equivalents [Abstract]
Restricted cash	$ 416	$ 416
Letters of credit receivable, noncurrent	$ 400	$ 400

Property and Equipment, Net (Tables)

12 Months Ended

Dec. 31, 2024

Property, Plant and Equipment [Abstract]

Schedule of Property, Plant and Equipment

Property and equipment are recorded at cost. Depreciation is calculated using the straight-line method over the following estimated useful lives of the assets:


			Estimated Useful Life
Office furniture and equipment			5 years
Laboratory equipment			3 years
Computer equipment			3 years

Property and equipment, net consisted of the following (in thousands):


	December 31,
	2024		2023
Computer equipment	$	689	$	689
Office furniture and equipment	545		545
Laboratory equipment	359		359
Total property and equipment	1,593		1,593
Less: Accumulated depreciation	(1,363)		(1,005)
Property and equipment, net	$	230	$	588

Segment Reporting - Narrative (Details)	12 Months Ended
Segment Reporting - Narrative (Details)	Dec. 31, 2024 segment
Segment Reporting [Abstract]
Number of operating segments	1

Accrued Expenses (Tables)

12 Months Ended

Dec. 31, 2024

Payables and Accruals [Abstract]

Schedule of Accrued Expenses

Accrued expenses consisted of the following (in thousands):


	December 31,
	2024		2023
Accrued external research and development expenses	$	13,363	$	2,957
Accrued personnel-related expenses	9,365		3,716
Accrued other expenses	1,035		743
Total accrued expenses	$	23,763	$	7,416

Fair Value of Financial Assets - Summary of Financial Assets Measured at Fair Value on a Recurring Basis (Details) - Fair Value, Recurring - USD ($)	Dec. 31, 2024	Dec. 31, 2023
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Assets	$ 398,763,000	$ 0
Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Assets	273,296,000
Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Assets	125,467,000
Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Assets	0
Money market funds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	128,652,000
Money market funds \| Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	128,652,000
Money market funds \| Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	0
Money market funds \| Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	0
Debt securities issued by U.S. government agencies
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	144,644,000
Debt securities issued by U.S. government agencies \| Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	144,644,000
Debt securities issued by U.S. government agencies \| Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0
Debt securities issued by U.S. government agencies \| Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0
Corporate debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	107,852,000
Corporate debt securities \| Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0
Corporate debt securities \| Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	107,852,000
Corporate debt securities \| Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0
Commercial paper
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	8,794,000
Commercial paper \| Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0
Commercial paper \| Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	8,794,000
Commercial paper \| Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0
Other debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	8,821,000
Other debt securities \| Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0
Other debt securities \| Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	8,821,000
Other debt securities \| Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	$ 0