0001689548-25-000111 | 10-Q | XBRL Viewer | Praxis Precision Medicines INC

Stock-Based Compensation (Tables)

9 Months Ended

Sep. 30, 2025

Share-Based Payment Arrangement [Abstract]

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


	Shares	Weighted Average Grant Date Fair Value
Unvested as of December 31, 2024	224,473	$	71.71
Issued	387,431	61.51
Vested	(65,339)	99.63
Forfeited	(30,446)	59.46
Unvested as of September 30, 2025	516,119	$	61.24

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, 2024	1,809,343	$	90.84
Granted	324,670	60.03
Exercised	(34,877)	39.21			$	738
Cancelled or Forfeited	(28,765)	67.76
Outstanding as of September 30, 2025	2,070,371	$	87.20	7.94	$	8,662
Exercisable as of September 30, 2025	1,197,365	$	108.73	7.28	$	6,571
Vested and expected to vest as of September 30, 2025	2,070,371	$	87.20	7.94	$	8,662

Schedule of Fair Value of Stock Option Awards on the Grant Date Using the Black-Scholes Option Valuation Model

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 and non-employees on the date of grant were as follows for the nine months ended September 30, 2025. No stock options were granted during the three months ended September 30, 2025.


	Nine Months Ended September 30,
	2025
Risk-free interest rate	4.45		%
Expected term (in years)	6.00
Expected volatility	93.08		%
Expected dividend yield	—		%
Weighted average grant-date fair value per share	$	46.74

Summary of Stock-Based Compensation Expense

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


	Three Months Ended September 30,				Nine Months Ended September 30,
	2025		2024		2025		2024
Research and development	$	2,959	$	4,640	$	9,969	$	12,298
General and administrative	4,488		7,792		14,034		20,487
Total stock-based compensation expense	$	7,447	$	12,432	$	24,003	$	32,785

Cash Equivalents and Marketable Securities

9 Months Ended

Sep. 30, 2025

Cash and Cash Equivalents [Abstract]

Cash, Cash Equivalent, and Marketable Security

Cash Equivalents and Marketable Securities

The following is a summary of the Company's investment portfolio as of September 30, 2025 and December 31, 2024 (in thousands):


	As of September 30, 2025
			Gross Unrealized				Estimated
	Cost		Gains		Losses		Fair Value
Cash equivalents:
Money market funds	$	76,033	$	—	$	—	$	76,033
Available-for-sale securities:
Debt securities issued by U.S. government agencies	98,033		204		—		98,237
Corporate debt securities	99,583		240		—		99,823
Commercial paper	1,928		1		—		1,929
Other debt securities	39,594		55		—		39,649
Total cash equivalents and marketable securities	$	315,171	$	500	$	—	$	315,671


	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

As of September 30, 2025, the Company had 4 securities with a total fair market value of $16.1 million in an unrealized loss position. The Company believes that any unrealized losses associated with the decline in value of its securities is temporary and primarily related to the change in market interest rates since purchase, and believes that it is more likely than not that it will be able to hold its debt securities to maturity. The Company anticipates a full recovery of the amortized cost basis of its debt securities at maturity and an allowance was not recognized.

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


	September 30, 2025		December 31, 2024
Within one year	$	117,630	$	177,195
After one year through five years	122,008		76,961
Total	$	239,638	$	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 three and nine months ended September 30, 2025 and 2024.

Cover Page - shares	9 Months Ended
Cover Page - shares	Sep. 30, 2025	Oct. 31, 2025
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Sep. 30, 2025
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 Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		25,007,171
Amendment Flag	false
Document Fiscal Year Focus	2025
Document Fiscal Period Focus	Q3
Entity Central Index Key	0001689548
Current Fiscal Year End Date	--12-31

Stock-Based Compensation - Narrative (Details) - USD ($) $ in Millions	1 Months Ended	9 Months Ended
	Jan. 31, 2025	Sep. 30, 2025	Dec. 31, 2024
Unvested Restricted Stock Units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total unrecognized compensation cost related to unvested restricted stock units		$ 25.0
Weighted-average period (in years)		3 years 1 month 2 days
Shares reserved for exercise of outstanding stock options
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Weighted-average period (in years)		2 years 2 months 26 days
Unrecognized compensation expense related to unvested stock based awards		$ 36.4
Inducement Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share-based payment award, increase Authorized (in shares)	870,000
Number of shares authorized (in shares)		1,000,000	130,000
2020 Employee Stock Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of shares authorized (in shares)		2,941,950	1,970,833
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
2020 Employee Stock Purchase Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of shares authorized (in shares)		369,368	175,145

Common Stock and Preferred Stock - Summary of Common Stock Reserved For Future Issuance (Details) - shares	Sep. 30, 2025	Dec. 31, 2024
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	5,219,293	3,473,399
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)	2,070,371	1,809,343
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)	704,225	904,580
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	221,238
Share-based payment arrangement \| 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)	462,188	91,343
Share-based payment arrangement \| 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)	294,552	109,648
Share-based payment arrangement \| 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)	950,600	112,774
Shares reserved for vesting of restricted stock units
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	516,119	224,473

Accrued Expenses

9 Months Ended

Sep. 30, 2025

Payables and Accruals [Abstract]

Accrued Expenses

Accrued expenses consisted of the following (in thousands):


	September 30, 2025		December 31, 2024
Accrued external research and development expenses	$	15,624	$	13,363
Accrued personnel-related expenses	3,683		9,365
Accrued other expenses	2,321		1,035
Total accrued expenses	$	21,628	$	23,763

Common Stock and Preferred Stock

9 Months Ended

Sep. 30, 2025

Stockholders' Equity Note [Abstract]

Common Stock and Preferred Stock

Common Stock

As of September 30, 2025 and December 31, 2024, the authorized capital stock of the Company included 150,000,000 shares of common stock, $0.0001 par value.

As of September 30, 2025 and December 31, 2024, the Company did not hold any treasury shares.

Follow-On Public Offerings

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.

During the nine months ended September 30, 2025, 200,355 pre-funded warrants were exercised via cashless exercise, resulting in 200,349 shares of common stock issued. No cash proceeds associated with the exercise were received by the Company. As of September 30, 2025, a total of 704,225 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.

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 September 30, 2025, none of the pre-funded warrants have been exercised and all remained outstanding.

The Company determined that the pre-funded warrants related to the 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 has reserved the following shares of common stock for future issuance:


	September 30, 2025	December 31, 2024
Shares reserved for exercise of outstanding stock options	2,070,371	1,809,343
Shares reserved for exercise of pre-funded warrants related to the January 2024 Financing	704,225	904,580
Shares reserved for exercise of pre-funded warrants related to the April 2024 Financing	221,238	221,238
Shares reserved for future awards under the 2020 Stock Option and Incentive Plan	462,188	91,343
Shares reserved for future awards under the 2020 Employee Stock Purchase Plan	294,552	109,648
Shares reserved for future awards under the 2024 Inducement Plan	950,600	112,774
Shares reserved for vesting of restricted stock units	516,119	224,473
Total shares of authorized common stock reserved for future issuance	5,219,293	3,473,399

Preferred Stock

As of September 30, 2025 and December 31, 2024, the authorized capital stock of the Company included 10,000,000 shares of undesignated preferred stock, $0.0001 par value.

- Definition

The entire disclosure for equity.

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+ Details

Summary of Significant Accounting Policies	9 Months Ended
Summary of Significant Accounting Policies	Sep. 30, 2025
Accounting Policies [Abstract]
Summary of Significant Accounting Policies	Summary of Significant Accounting Policies Basis of Presentation The accompanying condensed 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. The significant accounting policies used in preparation of these condensed consolidated financial statements for the three and nine months ended September 30, 2025 are consistent with those discussed in Note 2 to the consolidated financial statements included in the Company's 2024 Annual Report on Form 10-K, other than as noted below. Unaudited Interim Condensed Consolidated Financial Information The accompanying condensed consolidated balance sheet as of September 30, 2025, the condensed consolidated statements of operations for the three and nine months ended September 30, 2025 and 2024, the condensed consolidated statements of comprehensive loss for the three and nine months ended September 30, 2025 and 2024, the condensed consolidated statements of cash flows for the nine months ended September 30, 2025 and 2024 and the condensed consolidated statements of stockholders’ equity for the three and nine months ended September 30, 2025 and 2024 are unaudited. The unaudited interim financial statements have been prepared on the same basis as the audited annual consolidated financial statements, and in the opinion of management reflect all adjustments, which include only normal recurring adjustments necessary for the fair statement of the Company’s financial position as of September 30, 2025, the results of its operations for the three and nine months ended September 30, 2025 and 2024 and its cash flows for the nine months ended September 30, 2025 and 2024. Financial statement disclosures for the three and nine months ended September 30, 2025 and 2024 are condensed and do not include all disclosures required for an annual set of financial statements in accordance with GAAP. The results for the three and nine months ended September 30, 2025 are not necessarily indicative of results to be expected for the year ended December 31, 2025, any other interim periods, or any future year or period. 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. Recently Enacted Tax Legislation The One Big Beautiful Bill Act (“OBBBA”) was enacted in the U.S. on July 4, 2025. The OBBBA legislation provides for the permanent extension of certain expiring provisions of the Tax Cuts and Jobs Act, revisions to the international tax framework and the reinstatement of favorable tax treatment for certain business provisions. The legislation has multiple effective dates, with certain provisions effective in 2025 and others implemented in future periods. While tax changes in the OBBBA do not have a material impact to the Company's effective tax rate, we anticipate that certain tax provisions in the OBBBA, namely the provision allowing for immediate expensing of certain research and development expenses, will result in accelerated deductions of previously capitalized expenses of approximately $203.9 million. We continue to evaluate the tax and other provisions of the OBBBA and the potential effects on our financial position, results of operations, and cash flows. Recent Accounting Pronouncements Recently Issued Accounting Pronouncements In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740) - Improvements to Income Tax Disclosures. ASU No. 2023-09 provides more transparency about income tax information through improvements to income tax disclosures primarily related to the rate reconciliation and income taxes paid information. For public companies, the amendments are effective for annual periods beginning after December 15, 2024 and can be applied prospectively or retrospectively. The Company has determined that the effects of adopting the amendments in ASU 2023-09 will only impact its disclosures and will not have a material impact on its consolidated financial position and the results of its operations when such amendment is adopted. In November 2023, the FASB issued ASU No. 2024-03, Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses, 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.

Summary of Significant Accounting Policies (Policies)	9 Months Ended
Summary of Significant Accounting Policies (Policies)	Sep. 30, 2025
Accounting Policies [Abstract]
Basis of Presentation	Basis of Presentation The accompanying condensed 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. The significant accounting policies used in preparation of these condensed consolidated financial statements for the three and nine months ended September 30, 2025 are consistent with those discussed in Note 2 to the consolidated financial statements included in the Company's 2024 Annual Report on Form 10-K, other than as noted below.
Unaudited Interim Condensed Consolidated Financial Information	Unaudited Interim Condensed Consolidated Financial Information The accompanying condensed consolidated balance sheet as of September 30, 2025, the condensed consolidated statements of operations for the three and nine months ended September 30, 2025 and 2024, the condensed consolidated statements of comprehensive loss for the three and nine months ended September 30, 2025 and 2024, the condensed consolidated statements of cash flows for the nine months ended September 30, 2025 and 2024 and the condensed consolidated statements of stockholders’ equity for the three and nine months ended September 30, 2025 and 2024 are unaudited. The unaudited interim financial statements have been prepared on the same basis as the audited annual consolidated financial statements, and in the opinion of management reflect all adjustments, which include only normal recurring adjustments necessary for the fair statement of the Company’s financial position as of September 30, 2025, the results of its operations for the three and nine months ended September 30, 2025 and 2024 and its cash flows for the nine months ended September 30, 2025 and 2024. Financial statement disclosures for the three and nine months ended September 30, 2025 and 2024 are condensed and do not include all disclosures required for an annual set of financial statements in accordance with GAAP. The results for the three and nine months ended September 30, 2025 are not necessarily indicative of results to be expected for the year ended December 31, 2025, any other interim periods, or any future year or period.
Common Stock Warrants	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.
Recently Enacted Tax Legislation	Recently Enacted Tax Legislation The One Big Beautiful Bill Act (“OBBBA”) was enacted in the U.S. on July 4, 2025. The OBBBA legislation provides for the permanent extension of certain expiring provisions of the Tax Cuts and Jobs Act, revisions to the international tax framework and the reinstatement of favorable tax treatment for certain business provisions. The legislation has multiple effective dates, with certain provisions effective in 2025 and others implemented in future periods. While tax changes in the OBBBA do not have a material impact to the Company's effective tax rate, we anticipate that certain tax provisions in the OBBBA, namely the provision allowing for immediate expensing of certain research and development expenses, will result in accelerated deductions of previously capitalized expenses of approximately $203.9 million. We continue to evaluate the tax and other provisions of the OBBBA and the potential effects on our financial position, results of operations, and cash flows.
Recent Accounting Pronouncements	Recent Accounting Pronouncements Recently Issued Accounting Pronouncements In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740) - Improvements to Income Tax Disclosures. ASU No. 2023-09 provides more transparency about income tax information through improvements to income tax disclosures primarily related to the rate reconciliation and income taxes paid information. For public companies, the amendments are effective for annual periods beginning after December 15, 2024 and can be applied prospectively or retrospectively. The Company has determined that the effects of adopting the amendments in ASU 2023-09 will only impact its disclosures and will not have a material impact on its consolidated financial position and the results of its operations when such amendment is adopted. In November 2023, the FASB issued ASU No. 2024-03, Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses, 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.

- Definition

Disclosure of accounting policy for income taxes, which may include its accounting policies for recognizing and measuring deferred tax assets and liabilities and related valuation allowances, recognizing investment tax credits, operating loss carryforwards, tax credit carryforwards, and other carryforwards, methodologies for determining its effective income tax rate and the characterization of interest and penalties in the financial statements.

+ References + Details

Collaboration and License Agreements - Narrative (Details)		1 Months Ended		3 Months Ended		9 Months Ended
	Jan. 04, 2024 USD ($) obligation $ / shares shares	Dec. 31, 2024 USD ($)	Jan. 31, 2024 USD ($)	Sep. 30, 2025 USD ($) $ / shares	Sep. 30, 2024 USD ($)	Sep. 30, 2025 USD ($) $ / shares	Sep. 30, 2024 USD ($)	Dec. 31, 2022 USD ($) obligation
Disaggregation of Revenue [Line Items]
Number of performance obligations \| obligation	1							1
UCB \| License
Disaggregation of Revenue [Line Items]
Upfront payment		$ 0		$ 0		$ 0		$ 5,000,000.0
Milestone payments								98,500,000
Transaction price								$ 5,000,000.0
Contract with customer, liability, revenue recognized				$ 0	$ 300,000	$ 0	$ 1,100,000
UCB \| Option Exercise Fee
Disaggregation of Revenue [Line Items]
Contract with customer, liability, revenue recognized		6,000,000.0
UCB \| Upfront Payment
Disaggregation of Revenue [Line Items]
Contract with customer, liability, revenue recognized		$ 2,600,000
Tenacia
Disaggregation of Revenue [Line Items]
Number of shares issued in transaction (in shares) \| shares	443,253
Sale of stock (in USD per share) \| $ / shares	$ 22.5605			$ 38.95		$ 38.95
Proceeds from stock offerings, net of issuance costs	$ 10,000,000.0		$ 10,000,000
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			14,800,000
Sale of stock, value transferred						$ 17,300,000
Sale of stock, value transferred in excess of consideration received						$ 2,500,000
Tenacia \| License
Disaggregation of Revenue [Line Items]
Upfront payment	$ 5,000,000.0		$ 4,800,000
Milestone payments	$ 264,000,000.0

Condensed Consolidated Statements of Comprehensive Loss (Unaudited) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024
Statement of Comprehensive Income [Abstract]
Net loss	$ (73,934)	$ (51,910)	$ (214,357)	$ (124,140)
Change in unrealized gain (loss) on marketable securities, net of tax	105	1,402	(154)	1,331
Comprehensive loss	$ (73,829)	$ (50,508)	$ (214,511)	$ (122,809)

- 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

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+ Details

Summary of Significant Accounting Policies (Details) $ in Millions	3 Months Ended
	Sep. 30, 2025 USD ($)
Accounting Policies [Abstract]
One Big Beautiful Bill Act, research and development expense, accelerated deductions	$ 203.9

Common Stock and Preferred Stock - Narrative (Details) - USD ($) $ / shares in Units, $ in Millions			9 Months Ended	12 Months Ended
	Apr. 02, 2024	Jan. 16, 2024	Sep. 30, 2025	Dec. 31, 2024
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Common stock, authorized (in shares)			150,000,000	150,000,000
Common stock, par value (in dollars per share)			$ 0.0001	$ 0.0001
Treasury stock (in shares)			0	0
Preferred stock, authorized (in shares)			10,000,000	10,000,000
Preferred stock, par value (in dollars per share)			$ 0.0001	$ 0.0001
Pre-Funded Warrant \| Maximum
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Maximum percentage of voting power			4.99%
Percentage of ownership held by sole owner and affiliates			9.99%
Percentage change in ownership			19.99%
Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Change in ownership notice, term			61 days
January 2024 Follow-On Offering
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Sale of stock, shares, aggregate authorized amount (in shares)		3,802,025
Sale of stock (in USD per share)		$ 35.50
Proceeds from stock offerings, net of issuance costs		$ 161.6
January 2024 Follow-On Offering \| Pre-Funded Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Sale of stock (in USD per share)		$ 35.4999
Warrants (in shares)		1,056,725
Class of warrant or right, outstanding (in shares)			704,225
January 2024 Follow-On Offering \| Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Warrants (in dollars per share)		$ 0.0001
Over-Allotment Option
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Number of shares issued in transaction (in shares)		633,750
January 2024 Public Offering \| Pre-Funded Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Class of warrants or rights, exercised (in shares)			200,355	152,145
Issuance of common stock from exercise of pre-funded warrants (in shares)			200,349	152,142
April 2024 Public Offering
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Sale of stock, shares, aggregate authorized amount (in shares)	3,849,558
Sale of stock (in USD per share)	$ 56.50
Number of shares issued in transaction (in shares)	530,973
Proceeds from stock offerings, net of issuance costs	$ 216.0
April 2024 Public Offering \| Pre-Funded Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Sale of stock (in USD per share)	$ 56.4999
Warrants (in shares)	221,238
April 2024 Public Offering \| Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Warrants (in dollars per share)	$ 0.0001

Segment Information - Narrative (Details)	9 Months Ended
Segment Information - Narrative (Details)	Sep. 30, 2025 segment
Segment Reporting [Abstract]
Number of operating segments	1

Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2025	Sep. 30, 2024
Cash flows from operating activities:
Net loss	$ (214,357)	$ (124,140)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation expense	112	311
Stock-based compensation expense	24,003	32,785
Non-cash operating lease expense	769	690
Amortization of premiums and discounts on marketable securities, net	(2,921)	(3,620)
Non-cash collaboration and license agreement expense	0	2,500
Changes in operating assets and liabilities:
Prepaid expenses and other current assets	5,534	564
Accounts payable	18,291	9,195
Accrued expenses	(3,228)	8,005
Operating lease liabilities	(933)	(835)
Deferred revenue	0	(1,090)
Other	(13)	0
Net cash used in operating activities	(172,743)	(75,635)
Cash flows from investing activities:
Purchases of property and equipment	(56)	0
Purchases of marketable securities	(233,537)	(302,101)
Maturities of marketable securities	250,836	64,525
Net cash provided by (used in) investing activities	17,243	(237,576)
Cash flows from financing activities:
Payments of tax withholdings related to vesting of restricted stock units	(1,270)	(151)
Proceeds from exercise of stock options and employee stock purchase plan purchases	1,662	659
Net cash provided by financing activities	89,655	400,556
(Decrease) increase in cash, cash equivalents and restricted cash	(65,845)	87,345
Cash, cash equivalents and restricted cash, beginning of period	215,788	81,716
Cash, cash equivalents and restricted cash, end of period	149,943	169,061
Reconciliation of cash, cash equivalents and restricted cash:
Cash and cash equivalents	149,527	168,645
Restricted cash	416	416
Total cash, cash equivalents and restricted cash	149,943	169,061
Supplemental disclosures of non-cash activities:
Issuance costs from at-the-market offering included in accrued expenses	221	36
Follow-On Offering
Cash flows from financing activities:
Proceeds from stock offerings, net of issuance costs	0	377,636
License And Collaboration Agreement
Cash flows from financing activities:
Proceeds from stock offerings, net of issuance costs	0	14,765
At-The-Market Offering
Cash flows from financing activities:
Proceeds from stock offerings, net of issuance costs	$ 89,263	$ 7,647

Net Loss per Share

9 Months Ended

Sep. 30, 2025

Earnings Per Share [Abstract]

Net Loss 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:


	Three Months Ended September 30,		Nine Months Ended September 30,
	2025	2024	2025	2024
Outstanding stock options	2,070,371	1,829,324	2,070,371	1,829,324
Unvested restricted stock units	516,119	222,674	516,119	222,674
Potential shares issuable under the 2020 ESPP	13,795	7,391	13,795	7,391
	2,600,285	2,059,389	2,600,285	2,059,389

Common shares issuable upon exercise of the pre-funded warrants that were sold in connection with the January 2024 and April 2024 underwritten public offerings are included in the calculation of basic weighted average number of common shares outstanding for the three and nine months ended September 30, 2025. 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 basic net loss per share from the date of issuance.

Condensed Consolidated Statements of Operations (Unaudited) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024
Income Statement [Abstract]
Collaboration revenue	$ 0	$ 302	$ 0	$ 1,090
Operating expenses:
Research and development	65,797	41,881	189,609	96,125
General and administrative	12,562	15,256	39,545	41,174
Total operating expenses	78,359	57,137	229,154	137,299
Loss from operations	(78,359)	(56,835)	(229,154)	(136,209)
Other income:
Other income, net	4,425	4,925	14,797	12,069
Total other income	4,425	4,925	14,797	12,069
Net loss	$ (73,934)	$ (51,910)	$ (214,357)	$ (124,140)
Net loss per share attributable to common stockholders, basic (in dollars per share)	$ (3.36)	$ (2.75)	$ (9.97)	$ (7.21)
Net loss per share attributable to common stockholders, diluted (in dollars per share)	$ (3.36)	$ (2.75)	$ (9.97)	$ (7.21)
Weighted average common shares outstanding, basic (in shares)	21,977,268	18,884,562	21,506,021	17,210,604
Weighted average common shares outstanding, diluted (in shares)	21,977,268	18,884,562	21,506,021	17,210,604

Cash Equivalents and Marketable Securities - Contractual Maturity of Marketable Securities (Details) - USD ($) $ in Thousands	Sep. 30, 2025	Dec. 31, 2024
Cash and Cash Equivalents [Abstract]
Within one year	$ 117,630	$ 177,195
After one year through five years	122,008	76,961
Total	$ 239,638	$ 254,156

- 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/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 2: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 820
-SubTopic 10
-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 3: 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 4: http://www.xbrl.org/2003/role/exampleRef
-Name Accounting Standards Codification
-Section 55
-Paragraph 10
-SubTopic 10
-Topic 825
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482881/825-10-55-10

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 825
-SubTopic 10
-Name Accounting Standards Codification
-Section 45
-Paragraph 1A
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482736/825-10-45-1A

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 825
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 11
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482907/825-10-50-11

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 825
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 11
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482907/825-10-50-11

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 825
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 10
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482907/825-10-50-10

Reference 9: http://www.xbrl.org/2003/role/exampleRef
-Topic 825
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-Name Accounting Standards Codification
-Section 55
-Paragraph 12
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482881/825-10-55-12

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

Reference 11: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
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-Name Accounting Standards Codification
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-100

Reference 12: 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 13: 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 14: 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 15: 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 16: http://www.xbrl.org/2003/role/disclosureRef
-Topic 326
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-Name Accounting Standards Codification
-Section 45
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479130/326-30-45-1

+ Details

Commitments and Contingencies	9 Months Ended
Commitments and Contingencies	Sep. 30, 2025
Commitments and Contingencies Disclosure [Abstract]
Commitments and Contingencies	Commitments and Contingencies In May 2021, the Company entered into a sublease agreement for office space located in Boston, Massachusetts that 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, which is reflected within prepaid expenses and other current assets and other non-current assets on the accompanying condensed consolidated balance sheets as of September 30, 2025 and December 31, 2024, respectively. This lease qualifies as an operating lease.

Stock-Based Compensation

9 Months Ended

Sep. 30, 2025

Share-Based Payment Arrangement [Abstract]

Stock-Based Compensation

2024 Inducement Plan

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. In January 2025, the number of shares of common stock authorized for issuance under the Inducement Plan was increased by 870,000 shares. The total number of shares of common stock authorized for issuance under the Inducement Plan as of September 30, 2025 and December 31, 2024 was 1,000,000 shares and 130,000 shares, respectively.

2020 Stock Option and Incentive Plan

The total number of shares of common stock authorized for issuance under the 2020 Stock Option and Incentive Plan (the "2020 Plan”) as of September 30, 2025 and December 31, 2024 was 2,941,950 shares and 1,970,833 shares, respectively.

2017 Stock Incentive Plan

The total number of shares of common stock authorized for issuance under the 2017 Stock Incentive Plan (the "2017 Plan") as of September 30, 2025 and December 31, 2024 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.

2020 Employee Stock Purchase Plan

The total number of shares of common stock authorized for issuance under the 2020 Employee Stock Purchase Plan (the "2020 ESPP”) as of September 30, 2025 and December 31, 2024 was 369,368 shares and 175,145 shares, respectively.

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, 2024	224,473	$	71.71
Issued	387,431	61.51
Vested	(65,339)	99.63
Forfeited	(30,446)	59.46
Unvested as of September 30, 2025	516,119	$	61.24

As of September 30, 2025, total unrecognized compensation cost related to unvested restricted stock units was $25.0 million, which is expected to be recognized over a weighted-average period of 3.09 years.

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, 2024	1,809,343	$	90.84
Granted	324,670	60.03
Exercised	(34,877)	39.21			$	738
Cancelled or Forfeited	(28,765)	67.76
Outstanding as of September 30, 2025	2,070,371	$	87.20	7.94	$	8,662
Exercisable as of September 30, 2025	1,197,365	$	108.73	7.28	$	6,571
Vested and expected to vest as of September 30, 2025	2,070,371	$	87.20	7.94	$	8,662

Valuation of Stock Options


	Nine Months Ended September 30,
	2025
Risk-free interest rate	4.45		%
Expected term (in years)	6.00
Expected volatility	93.08		%
Expected dividend yield	—		%
Weighted average grant-date fair value per share	$	46.74

As of September 30, 2025, total unrecognized compensation cost related to unvested stock options was $36.4 million, which is expected to be recognized over a weighted-average period of 2.24 years.

Stock-Based Compensation

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


	Three Months Ended September 30,				Nine Months Ended September 30,
	2025		2024		2025		2024
Research and development	$	2,959	$	4,640	$	9,969	$	12,298
General and administrative	4,488		7,792		14,034		20,487
Total stock-based compensation expense	$	7,447	$	12,432	$	24,003	$	32,785

Segment Information (Tables)

9 Months Ended

Sep. 30, 2025

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 three and nine months ended September 30, 2025 and 2024 (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2025		2024		2025		2024
Collaboration revenue	$	—	$	302	$	—	$	1,090
Program-specific expenses:
Ulixacaltamide	11,644		19,976		53,542		43,160
Vormatrigine	20,899		6,613		52,589		9,205
Relutrigine	14,965		1,263		30,851		3,514
Elsunersen	2,219		380		4,297		1,586
Other early stage assets	3,142		1,179		5,664		3,242
Personnel-related expenses	12,116		9,472		37,059		27,582
Stock-based compensation expense	7,447		12,432		24,003		32,786
Depreciation expense	19		92		112		311
Other segment expenses(a)	5,908		5,730		21,037		15,913
Interest income	4,425		4,925		14,797		12,069
Net loss	$	(73,934)	$	(51,910)	$	(214,357)	$	(124,140)

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

Fair Value Measurements (Tables)

9 Months Ended

Sep. 30, 2025

Fair Value Disclosures [Abstract]

Summary of Financial Assets Measured At Fair Value On A 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 September 30, 2025 and December 31, 2024 (in thousands):


	As of September 30, 2025
	Level 1		Level 2		Level 3		Total
Assets:
Money market funds	$	76,033	$	—	$	—	$	76,033

Debt securities issued by US. government agencies	98,237		—		—		98,237
Corporate debt securities	—		99,823		—		99,823
Commercial paper	—		1,929		—		1,929
Other debt securities	—		39,649		—		39,649
	$	174,270	$	141,401	$	—	$	315,671


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

Debt securities issued by US. 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

Stock-Based Compensation - Summary of Restricted Stock Activity (Details) - Unvested restricted stock units	9 Months Ended
	Sep. 30, 2025 $ / shares shares
Shares
Beginning balance (in shares) \| shares	224,473
Issued (in shares) \| shares	387,431
Vested (in shares) \| shares	(65,339)
Forfeited (in shares) \| shares	(30,446)
Ending balance (in shares) \| shares	516,119
Weighted Average Grant Date Fair Value
Unvested beginning balance (in dollars per share) \| $ / shares	$ 71.71
Issued (in dollars per share) \| $ / shares	61.51
Vested (in dollars per share) \| $ / shares	99.63
Forfeitures (in dollars per shares) \| $ / shares	59.46
Unvested ending balance (in dollars per share) \| $ / shares	$ 61.24

Accrued Expenses - Summary of Accrued Expenses (Details) - USD ($) $ in Thousands	Sep. 30, 2025	Dec. 31, 2024
Payables and Accruals [Abstract]
Accrued external research and development expenses	$ 15,624	$ 13,363
Accrued personnel-related expenses	3,683	9,365
Accrued other expenses	2,321	1,035
Total accrued expenses	$ 21,628	$ 23,763

Accrued Expenses (Tables)

9 Months Ended

Sep. 30, 2025

Payables and Accruals [Abstract]

Summary of Accrued Expenses

Accrued expenses consisted of the following (in thousands):


	September 30, 2025		December 31, 2024
Accrued external research and development expenses	$	15,624	$	13,363
Accrued personnel-related expenses	3,683		9,365
Accrued other expenses	2,321		1,035
Total accrued expenses	$	21,628	$	23,763

Segment Information - Schedule of Segment Reporting Information, by Segment (Details) - USD ($) $ in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2025	Jun. 30, 2025	Mar. 31, 2025	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Sep. 30, 2025	Sep. 30, 2024
Segment Reporting Information [Line Items]
Collaboration revenue	$ 0			$ 302			$ 0	$ 1,090
Stock-based compensation expense	7,447			12,432			24,003	32,785
Depreciation expense							112	311
Net loss	(73,934)	$ (71,127)	$ (69,296)	(51,910)	$ (32,677)	$ (39,553)	(214,357)	(124,140)
Reportable Segment
Segment Reporting Information [Line Items]
Collaboration revenue	0			302			0	1,090
Personnel-related expenses	12,116			9,472			37,059	27,582
Stock-based compensation expense	7,447			12,432			24,003	32,786
Depreciation expense	19			92			112	311
Other segment expenses	5,908			5,730			21,037	15,913
Interest income	4,425			4,925			14,797	12,069
Net loss	(73,934)			(51,910)			(214,357)	(124,140)
Reportable Segment \| Ulixacaltamide
Segment Reporting Information [Line Items]
Program-specific expenses	11,644			19,976			53,542	43,160
Reportable Segment \| Vormatrigine
Segment Reporting Information [Line Items]
Program-specific expenses	20,899			6,613			52,589	9,205
Reportable Segment \| Relutrigine
Segment Reporting Information [Line Items]
Program-specific expenses	14,965			1,263			30,851	3,514
Reportable Segment \| Elsunersen
Segment Reporting Information [Line Items]
Program-specific expenses	2,219			380			4,297	1,586
Reportable Segment \| Other early stage assets
Segment Reporting Information [Line Items]
Program-specific expenses	$ 3,142			$ 1,179			$ 5,664	$ 3,242

Net Loss per Share (Tables)

9 Months Ended

Sep. 30, 2025

Earnings Per Share [Abstract]

Summary of Antidilutive Securities Excluded From Computation of Earnings Per Share


	Three Months Ended September 30,		Nine Months Ended September 30,
	2025	2024	2025	2024
Outstanding stock options	2,070,371	1,829,324	2,070,371	1,829,324
Unvested restricted stock units	516,119	222,674	516,119	222,674
Potential shares issuable under the 2020 ESPP	13,795	7,391	13,795	7,391
	2,600,285	2,059,389	2,600,285	2,059,389

Cash Equivalents and Marketable Securities - Investment Profile (Details) - USD ($) $ in Thousands	Sep. 30, 2025	Dec. 31, 2024
Debt Securities, Available-for-sale [Line Items]
Debt securities, available-for-sale, current	$ 117,630	$ 177,195
Cash equivalents, at carrying value and debt securities, available-for-sale, amortized cost	315,171	398,109
Cash equivalents and debt securities, available-for-sale, accumulated gross unrealized gains, current, before tax	500	654
Cash equivalents and debt securities, available-for-sale, accumulated gross unrealized losses, current, before tax	0	0
Cash equivalents and debt securities, available-for-sale, fair value disclosure	315,671	398,763
Money market funds
Debt Securities, Available-for-sale [Line Items]
Cash equivalents, at carrying value	76,033	128,652
Cash equivalents, accumulated gross unrealized gains, current, before tax	0	0
Cash equivalents, accumulated gross unrealized losses, current, before tax	0	0
Cash equivalents, fair value disclosure	76,033	128,652
Debt securities issued by US. government agencies
Debt Securities, Available-for-sale [Line Items]
Cash equivalents, at carrying value		15,953
Cash equivalents, accumulated gross unrealized gains, current, before tax		2
Cash equivalents, accumulated gross unrealized losses, current, before tax		0
Cash equivalents, fair value disclosure		15,955
Debt securities, available-for-sale, amortized cost, current	98,033	128,215
Debt securities, available-for-sale, accumulated gross unrealized gains, current, before tax	204	475
Debt securities, available-for-sale, accumulated gross unrealized losses, current, before tax	0	0
Debt securities, available-for-sale, current	98,237	128,690
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Debt securities, available-for-sale, amortized cost, current	99,583	107,695
Debt securities, available-for-sale, accumulated gross unrealized gains, current, before tax	240	157
Debt securities, available-for-sale, accumulated gross unrealized losses, current, before tax	0	0
Debt securities, available-for-sale, current	99,823	107,852
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Debt securities, available-for-sale, amortized cost, current	1,928	8,775
Debt securities, available-for-sale, accumulated gross unrealized gains, current, before tax	1	19
Debt securities, available-for-sale, accumulated gross unrealized losses, current, before tax	0	0
Debt securities, available-for-sale, current	1,929	8,794
Other debt securities
Debt Securities, Available-for-sale [Line Items]
Debt securities, available-for-sale, amortized cost, current	39,594	8,819
Debt securities, available-for-sale, accumulated gross unrealized gains, current, before tax	55	1
Debt securities, available-for-sale, accumulated gross unrealized losses, current, before tax	0	0
Debt securities, available-for-sale, current	$ 39,649	$ 8,820

Common Stock and Preferred Stock (Tables)

9 Months Ended

Sep. 30, 2025

Stockholders' Equity Note [Abstract]

Summary of Common Stock Reserved For Future Issuance

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


	September 30, 2025	December 31, 2024
Shares reserved for exercise of outstanding stock options	2,070,371	1,809,343
Shares reserved for exercise of pre-funded warrants related to the January 2024 Financing	704,225	904,580
Shares reserved for exercise of pre-funded warrants related to the April 2024 Financing	221,238	221,238
Shares reserved for future awards under the 2020 Stock Option and Incentive Plan	462,188	91,343
Shares reserved for future awards under the 2020 Employee Stock Purchase Plan	294,552	109,648
Shares reserved for future awards under the 2024 Inducement Plan	950,600	112,774
Shares reserved for vesting of restricted stock units	516,119	224,473
Total shares of authorized common stock reserved for future issuance	5,219,293	3,473,399

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Sep. 30, 2025
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

Cash Equivalents and Marketable Securities (Tables)

9 Months Ended

Sep. 30, 2025

Cash and Cash Equivalents [Abstract]

Schedule of Debt Securities, Available-for-Sale

The following is a summary of the Company's investment portfolio as of September 30, 2025 and December 31, 2024 (in thousands):


	As of September 30, 2025
			Gross Unrealized				Estimated
	Cost		Gains		Losses		Fair Value
Cash equivalents:
Money market funds	$	76,033	$	—	$	—	$	76,033
Available-for-sale securities:
Debt securities issued by U.S. government agencies	98,033		204		—		98,237
Corporate debt securities	99,583		240		—		99,823
Commercial paper	1,928		1		—		1,929
Other debt securities	39,594		55		—		39,649
Total cash equivalents and marketable securities	$	315,171	$	500	$	—	$	315,671


	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 date are as follows (in thousands):


	September 30, 2025		December 31, 2024
Within one year	$	117,630	$	177,195
After one year through five years	122,008		76,961
Total	$	239,638	$	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

Condensed Consolidated Statements of Stockholders’ Equity (Unaudited) - USD ($) $ in Thousands	Total	At-The-Market Offering	Follow-On Offering	License And Collaboration Agreement	Common Stock	Common Stock At-The-Market Offering	Common Stock Follow-On Offering	Common Stock License And Collaboration Agreement	Additional Paid-In Capital	Additional Paid-In Capital At-The-Market Offering	Additional Paid-In Capital Follow-On Offering	Additional Paid-In Capital License And Collaboration Agreement	Accumulated Deficit	Accumulated Other Comprehensive Gain (Loss)
Beginning balance (in shares) at Dec. 31, 2023					8,791,877
Beginning 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 (in shares)						209,852	3,802,025	443,253
Issuance of common stock		$ 6,169	$ 161,649	$ 17,265						$ 6,169	$ 161,649	$ 17,265
Vesting of restricted stock awards (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 upon exercise of stock options (in shares)					3,634
Issuance of common stock upon exercise of stock options	143								143
Change in unrealized gain 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
Beginning balance at Dec. 31, 2023	69,669				$ 13				723,577				(653,921)	0
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Change in unrealized gain on marketable securities, net of tax	1,331
Net loss	(124,140)
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
Beginning balance (in shares) at Dec. 31, 2023					8,791,877
Beginning balance at Dec. 31, 2023	$ 69,669				$ 13				723,577				(653,921)	0
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 (in shares)							3,849,558
Issuance of common stock			$ 215,988				$ 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
Issuance of common stock from exercise of pre-funded warrants (in shares)					622,123
Vesting of restricted stock awards (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 upon exercise of stock options (in shares)					148
Issuance of common stock upon exercise of stock options	4								4
Change in unrealized gain 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 (in shares)						25,189
Issuance of common stock		1,442								1,442
Vesting of restricted stock awards (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 upon exercise of stock options (in shares)					4,971
Issuance of common stock upon exercise of stock options	201								201
Change in unrealized gain 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
Beginning balance (in shares) at Dec. 31, 2024	19,422,358				19,422,358
Beginning balance at Dec. 31, 2024	$ 445,450				$ 14				1,281,522				(836,740)	654
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	8,786								8,786
Issuance of common stock (in shares)						694,212
Issuance of common stock		54,904								54,904
Issuance of common stock from exercise of pre-funded warrants (in shares)					173,840
Vesting of restricted stock awards (in shares)					55,850
Shares withheld for taxes for vesting of restricted stock units (in shares)					(18,765)
Shares withheld for taxes for vesting of restricted stock units	(1,195)								(1,195)
Issuance of common stock upon exercise of stock options (in shares)					14,422
Issuance of common stock upon exercise of stock options	560								560
Change in unrealized gain on marketable securities, net of tax	5													5
Net loss	(69,296)												(69,296)
Ending balance (in shares) at Mar. 31, 2025					20,341,917
Ending balance at Mar. 31, 2025	$ 439,214				$ 14				1,344,577				(906,036)	659
Beginning balance (in shares) at Dec. 31, 2024	19,422,358				19,422,358
Beginning balance at Dec. 31, 2024	$ 445,450				$ 14				1,281,522				(836,740)	654
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock upon exercise of stock options (in shares)	34,877
Change in unrealized gain on marketable securities, net of tax	$ (154)
Net loss	$ (214,357)
Ending balance (in shares) at Sep. 30, 2025	21,165,933				21,165,933
Ending balance at Sep. 30, 2025	$ 343,505				$ 14				1,394,088				(1,051,097)	500
Beginning balance (in shares) at Mar. 31, 2025					20,341,917
Beginning balance at Mar. 31, 2025	439,214				$ 14				1,344,577				(906,036)	659
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	7,770								7,770
Issuance of common stock (in shares)						657,477
Issuance of common stock		28,360								28,360
Issuance of common stock under employee stock purchase plan (in shares)					9,319
Issuance of common stock under employee stock purchase plan	294								294
Issuance of common stock from exercise of pre-funded warrants (in shares)					26,509
Vesting of restricted stock awards (in shares)					5,860
Shares withheld for taxes for vesting of restricted stock units (in shares)					(608)
Shares withheld for taxes for vesting of restricted stock units	(23)								(23)
Change in unrealized gain on marketable securities, net of tax	(264)													(264)
Net loss	(71,127)												(71,127)
Ending balance (in shares) at Jun. 30, 2025					21,040,474
Ending balance at Jun. 30, 2025	404,224				$ 14				1,380,978				(977,163)	395
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	7,447								7,447
Issuance of common stock (in shares)						102,364
Issuance of common stock		$ 4,907								$ 4,907
Vesting of restricted stock awards (in shares)					3,746
Shares withheld for taxes for vesting of restricted stock units (in shares)					(1,106)
Shares withheld for taxes for vesting of restricted stock units	(52)								(52)
Issuance of common stock upon exercise of stock options (in shares)					20,455
Issuance of common stock upon exercise of stock options	808								808
Change in unrealized gain on marketable securities, net of tax	105													105
Net loss	$ (73,934)												(73,934)
Ending balance (in shares) at Sep. 30, 2025	21,165,933				21,165,933
Ending balance at Sep. 30, 2025	$ 343,505				$ 14				$ 1,394,088				$ (1,051,097)	$ 500

Condensed Consolidated Balance Sheets (Unaudited) (Parenthetical) - $ / shares	Sep. 30, 2025	Dec. 31, 2024
Statement of Financial Position [Abstract]
Preferred stock, par value (in dollars 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 dollars per share)	$ 0.0001	$ 0.0001
Common stock, authorized (in shares)	150,000,000	150,000,000
Common stock, issued (in shares)	21,165,933	19,422,358
Common stock, outstanding (in shares)	21,165,933	19,422,358

Related Party Transactions	9 Months Ended
Related Party Transactions	Sep. 30, 2025
Related Party Transactions [Abstract]
Related Party Transactions	Related Party Transactions On September 11, 2019, the Company entered into a Cooperation and License Agreement (the “License Agreement”) with RogCon Inc. (“RogCon”). 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. Pursuant to the terms of the License Agreement, the Company will conduct, at its own cost and expense, the research and development activities assigned to it under the associated 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. One of the founders of RogCon became the Company's General Counsel in June 2020. The Company continues to reimburse RogCon for its out-of-pocket costs incurred for activities performed under the License Agreement. Expenses incurred during all periods presented were not material.

- 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

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483326/850-10-50-1

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480990/946-20-50-2

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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-Section 50
-Paragraph 5
-Publisher FASB
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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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-Subparagraph (c)
-Publisher FASB
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Section 50
-Paragraph 2
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.6-07(2)(g)(3))
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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-Section S99
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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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-Section 50
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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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-Section 50
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483326/850-10-50-1

+ Details

Collaboration and License Agreements	9 Months Ended
Collaboration and License Agreements	Sep. 30, 2025
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 Biopharma SRL (“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 was 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 recognized 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 and the $2.6 million of remaining collaboration revenue associated with the $5.0 million up front payment as collaboration revenue within the December 31, 2024 consolidated statement of operations, as the Company had no further research service obligations under the terms of the Collaboration Agreement. The Company did not recognize any collaboration revenue associated with this Collaboration Agreement during the three and nine months ended September 30, 2025. As of September 30, 2025, there was no deferred revenue recorded in the condensed consolidated balance sheet. During the three and nine months ended September 30, 2024, the Company recognized $0.3 million and $1.1 million, respectively, related to the Collaboration Agreement in the condensed consolidated statement of operations. As of December 31, 2024, there was no deferred revenue recorded 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 Biotechnology (Shanghai) Co., Ltd. (“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 three and nine months ended September 30, 2025 and 2024, the Company did not achieve any development or sales milestones or earn 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 September 30, 2025 and 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 September 30, 2025 because no qualifying costs were incurred. During the three and nine months ended September 30, 2025 and 2024, the Company did not recognize any revenue associated with the combined arrangement.

Condensed Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands	Sep. 30, 2025	Dec. 31, 2024
Current assets:
Cash and cash equivalents	$ 149,527	$ 215,372
Marketable securities	117,630	177,195
Prepaid expenses and other current assets	6,687	11,805
Total current assets	273,844	404,372
Long-term marketable securities	122,008	76,961
Property and equipment, net	174	230
Operating lease right-of-use assets	362	1,131
Other non-current assets	0	416
Total assets	396,388	483,110
Current liabilities:
Accounts payable	30,819	12,528
Accrued expenses	21,628	23,763
Operating lease liabilities	436	1,259
Total current liabilities	52,883	37,550
Long-term liabilities:
Non-current portion of operating lease liabilities	0	110
Total liabilities	52,883	37,660
Commitments and contingencies (Note 6)
Stockholders’ equity:
Preferred stock, $0.0001 par value; 10,000,000 shares authorized and no shares issued or outstanding as of September 30, 2025 and December 31, 2024	0	0
Common stock, $0.0001 par value; 150,000,000 shares authorized; 21,165,933 shares issued and outstanding as of September 30, 2025, and 19,422,358 shares issued and outstanding as of December 31, 2024	14	14
Additional paid-in capital	1,394,088	1,281,522
Accumulated other comprehensive gain	500	654
Accumulated deficit	(1,051,097)	(836,740)
Total stockholders’ equity	343,505	445,450
Total liabilities and stockholders’ equity	$ 396,388	$ 483,110

Subsequent Events (Details) - USD ($) $ / shares in Units, $ in Millions	Oct. 20, 2025	Jan. 16, 2024
Over-Allotment Option
Subsequent Event [Line Items]
Number of shares issued in transaction (in shares)		633,750
Subsequent Event \| October 2025 Public Offering
Subsequent Event [Line Items]
Number of shares issued in transaction (in shares)	3,527,072
Sale of stock (in USD per share)	$ 157.00
Sale of stock, consideration received on transaction	$ 567.0
Subsequent Event \| Over-Allotment Option
Subsequent Event [Line Items]
Number of shares issued in transaction (in shares)	501,592
Subsequent Event \| Pre-funded Warrants
Subsequent Event [Line Items]
Number of shares issued in transaction (in shares)	318,470
Sale of stock (in USD per share)	$ 156.9999
Warrants (in dollars per share)	$ 0.0001

Stock-Based Compensation - Summary of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]
Total stock-based compensation expense	$ 7,447	$ 12,432	$ 24,003	$ 32,785
Research and development
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]
Total stock-based compensation expense	2,959	4,640	9,969	12,298
General and administrative
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]
Total stock-based compensation expense	$ 4,488	$ 7,792	$ 14,034	$ 20,487

Fair Value Measurements

9 Months Ended

Sep. 30, 2025

Fair Value Disclosures [Abstract]

Fair Value Measurements

Fair value is defined as the price that would be received to sell an asset in an orderly transaction between market participants at the measurement date. The Company categorizes financial assets measured at fair value based on a fair value hierarchy. The following fair value hierarchy is used to classify financial assets based on observable inputs and unobservable inputs used to value the financial assets:

•Level 1: Unadjusted quoted prices in active markets that are accessible at the measurement date for identical assets;

•Level 2: Quoted prices for similar assets in active markets, quoted prices in markets that are not active, or inputs which are unobservable, either directly or indirectly, for substantially the full term of the asset; or

•Level 3: Prices or valuation techniques that require inputs that are both significant to the valuation of the asset and unobservable.


	As of September 30, 2025
	Level 1		Level 2		Level 3		Total
Assets:
Money market funds	$	76,033	$	—	$	—	$	76,033

Debt securities issued by US. government agencies	98,237		—		—		98,237
Corporate debt securities	—		99,823		—		99,823
Commercial paper	—		1,929		—		1,929
Other debt securities	—		39,649		—		39,649
	$	174,270	$	141,401	$	—	$	315,671


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

Debt securities issued by US. 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

- 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/disclosureRef
-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 2: 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 3: 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 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)(3)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

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

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (f)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6A
-Subparagraph (e)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-6A

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

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (g)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482106/820-10-50-2

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 940
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-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147478119/940-820-50-1

+ Details

Nature of the Business	9 Months Ended
Nature of the Business	Sep. 30, 2025
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, or 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, 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 September 30, 2025, the Company raised $1.2 billion in aggregate cash proceeds from these transactions, net of issuance costs. The Company is subject to risks and uncertainties common to early-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 these condensed consolidated financial statements are issued. The Company has incurred recurring losses since its inception, including a net loss of $214.4 million for the nine months ended September 30, 2025. In addition, as of September 30, 2025, the Company had an accumulated deficit of $1.1 billion. 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 September 30, 2025 of $389.2 million, together with net proceeds of $567.0 million from the October 2025 financing, will be sufficient to fund its operating expenditures and capital expenditure requirements necessary to advance its program activities for at least one year from the date of issuance of these condensed 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.

Subsequent Events	9 Months Ended
Subsequent Events	Sep. 30, 2025
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. October 2025 Public Offering On October 20, 2025, the Company completed a public offering of: (i) an aggregate of 3,527,072 shares of its common stock at a public offering price of $157.00 per share, including the underwriters' full exercise of their option to purchase 501,592 additional shares of common stock, and (ii) pre-funded warrants to purchase 318,470 shares of common stock at a public offering price of $156.9999 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 $567.0 million, after deducting underwriting discounts, commissions and other offering expenses payable by the Company.

Stock-Based Compensation - Summary of Stock Option Activity (Details) $ / shares in Units, $ in Thousands	3 Months Ended	9 Months Ended
	Sep. 30, 2025 USD ($) $ / shares shares	Sep. 30, 2025 USD ($) $ / shares shares
Number of Shares
Beginning balance (in shares) \| shares		1,809,343
Granted (in shares) \| shares	0	324,670
Exercised (in shares) \| shares		(34,877)
Canceled or forfeited (in shares) \| shares		(28,765)
Ending balance (in shares) \| shares	2,070,371	2,070,371
Number of shares exercisable (in shares) \| shares	1,197,365	1,197,365
Vested and expected to vest (in shares) \| shares	2,070,371	2,070,371
Weighted Average Exercise Price per Share
Beginning balance (in dollars per share) \| $ / shares		$ 90.84
Granted (in dollars per share) \| $ / shares		60.03
Exercised (in dollars per share) \| $ / shares		39.21
Cancelled or forfeited (in dollars per share) \| $ / shares		67.76
Ending balance (in dollars per share) \| $ / shares	$ 87.20	87.20
Exercisable (in dollars per share) \| $ / shares	108.73	108.73
Vested and expected to vest (in dollars per share) \| $ / shares	$ 87.20	$ 87.20
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract]
Weighted average remaining contractual term outstanding		7 years 11 months 8 days
Weighted average remaining contractual term exercisable		7 years 3 months 10 days
Weighted average remaining contractual term vested and expected to vest		7 years 11 months 8 days
Aggregate intrinsic value exercised \| $		$ 738
Aggregate intrinsic value outstanding \| $	$ 8,662	8,662
Aggregate intrinsic value exercisable \| $	6,571	6,571
Aggregate intrinsic value vested and expected to vest \| $	$ 8,662	$ 8,662

Net Loss per Share - Summary of antidilutive securities excluded from computation of earnings per share (Details) - shares	3 Months Ended		9 Months Ended
	Sep. 30, 2025	Sep. 30, 2024	Sep. 30, 2025	Sep. 30, 2024
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities (in shares)	2,600,285	2,059,389	2,600,285	2,059,389
Outstanding stock options
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities (in shares)	2,070,371	1,829,324	2,070,371	1,829,324
Unvested restricted stock units
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities (in shares)	516,119	222,674	516,119	222,674
Potential shares issuable under the 2020 ESPP
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities (in shares)	13,795	7,391	13,795	7,391

Condensed Consolidated Statements of Stockholders’ Equity (Unaudited) (Parenthetical) - USD ($) $ in Thousands	3 Months Ended
	Sep. 30, 2025	Jun. 30, 2025	Mar. 31, 2025	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024
Follow-On Offering
Stock issuance costs					$ 14,013	$ 10,836
At-The-Market Offering
Stock issuance costs	$ 359	$ 579	$ 1,120	$ 70		$ 376

Nature of the Business (Details) $ in Thousands		3 Months Ended						9 Months Ended
Nature of the Business (Details) $ in Thousands	Oct. 20, 2025 USD ($)	Sep. 30, 2025 USD ($) platform candidate	Jun. 30, 2025 USD ($)	Mar. 31, 2025 USD ($)	Sep. 30, 2024 USD ($)	Jun. 30, 2024 USD ($)	Mar. 31, 2024 USD ($)	Sep. 30, 2025 USD ($) platform candidate	Sep. 30, 2024 USD ($)	Dec. 31, 2024 USD ($)
Product Information [Line Items]
Number of platforms \| platform		2						2
Number of product candidates \| candidate		4						4
Proceeds from issuance of equity								$ 1,200,000
Net loss		$ 73,934	$ 71,127	$ 69,296	$ 51,910	$ 32,677	$ 39,553	214,357	$ 124,140
Accumulated deficit		1,051,097						1,051,097		$ 836,740
Cash, cash equivalents, and short-term investments		$ 389,200						$ 389,200
Subsequent Event \| October 2025 Public Offering
Product Information [Line Items]
Sale of stock, consideration received on transaction	$ 567,000

Segment Information

9 Months Ended

Sep. 30, 2025

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 condensed 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 condensed consolidated 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 three and nine months ended September 30, 2025 and 2024 (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2025		2024		2025		2024
Collaboration revenue	$	—	$	302	$	—	$	1,090
Program-specific expenses:
Ulixacaltamide	11,644		19,976		53,542		43,160
Vormatrigine	20,899		6,613		52,589		9,205
Relutrigine	14,965		1,263		30,851		3,514
Elsunersen	2,219		380		4,297		1,586
Other early stage assets	3,142		1,179		5,664		3,242
Personnel-related expenses	12,116		9,472		37,059		27,582
Stock-based compensation expense	7,447		12,432		24,003		32,786
Depreciation expense	19		92		112		311
Other segment expenses(a)	5,908		5,730		21,037		15,913
Interest income	4,425		4,925		14,797		12,069
Net loss	$	(73,934)	$	(51,910)	$	(214,357)	$	(124,140)

(a) 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.

+ References

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+ Details

Commitments and Contingencies - Narrative (Details)	May 31, 2021
Office Space in Boston Massachusetts
Loss Contingencies [Line Items]
Percentage increase in annual base rent	2.00%

- 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.

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Fair Value Measurements (Details) - Fair Value, Recurring - USD ($) $ in Thousands	Sep. 30, 2025	Dec. 31, 2024
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Assets	$ 315,671	$ 398,763
Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Assets	174,270	273,296
Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Assets	141,401	125,467
Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Assets	0	0
Money market funds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	76,033	128,652
Money market funds \| Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	76,033	128,652
Money market funds \| Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	0	0
Money market funds \| Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	0	0
Debt securities issued by US. government agencies
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	98,237	144,644
Debt securities issued by US. government agencies \| Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	98,237	144,644
Debt securities issued by US. government agencies \| Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0	0
Debt securities issued by US. government agencies \| Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0	0
Corporate debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	99,823	107,852
Corporate debt securities \| Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0	0
Corporate debt securities \| Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	99,823	107,852
Corporate debt securities \| Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0	0
Commercial paper
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	1,929	8,794
Commercial paper \| Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0	0
Commercial paper \| Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	1,929	8,794
Commercial paper \| Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0	0
Other debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	39,649	8,821
Other debt securities \| Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	0	0
Other debt securities \| Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	39,649	8,821
Other debt securities \| Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Available-for-sale marketable securities	$ 0	$ 0

Cash Equivalents and Marketable Securities - Narrative (Details) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2025 USD ($) security	Sep. 30, 2024 USD ($)	Sep. 30, 2025 USD ($) security	Sep. 30, 2024 USD ($)
Cash and Cash Equivalents [Abstract]
Number of securities \| security	4		4
Securities, unrealized loss position, fair value	$ 16,100		$ 16,100
Gain (loss) on available-for-sale securities	$ 0	$ 0	$ 0	$ 0

Stock-Based Compensation - Schedule of Fair Value of Stock Option Awards on the Grant Date Using the Black-Scholes Option Valuation Model (Details) - $ / shares	3 Months Ended	9 Months Ended
	Sep. 30, 2025	Sep. 30, 2025
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Granted (in shares)	0	324,670
Risk-free interest rate		4.45%
Expected term (in years)		6 years
Expected volatility		93.08%
Expected dividend yield		0.00%
Shares reserved for exercise of outstanding stock options
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Weighted average grant-date fair value per share (in dollars per share)		$ 46.74

- Definition

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+ Details