0001689548-26-000029 | 10-K | XBRL Viewer | Praxis Precision Medicines INC

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Stock-Based Compensation - Summary of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]
Total stock-based compensation expense	$ 33,941	$ 41,360	$ 24,857
Research and development
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]
Total stock-based compensation expense	15,286	15,620	8,055
General and administrative
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]
Total stock-based compensation expense	$ 18,655	$ 25,740	$ 16,802

Insider Trading Policies and Procedures	12 Months Ended
Insider Trading Policies and Procedures	Dec. 31, 2025
Insider Trading Policies and Procedures [Line Items]
Insider Trading Policies and Procedures Adopted	true

Summary of Significant Accounting Policies

12 Months Ended

Dec. 31, 2025

Accounting Policies [Abstract]

Summary of Significant Accounting Policies

Basis of Presentation

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

Principles of Consolidation

The consolidated financial statements include the accounts of the Company and its wholly owned subsidiaries, Praxis Security Corporation, Praxis Medicines International Holdings LLC and Praxis Precision Medicines India Private Limited. All intercompany transactions have been eliminated in consolidation.

Use of Estimates

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

Cash, Cash Equivalents and Restricted Cash

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

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

As of December 31, 2025 and 2024, the Company had restricted cash of $0.4 million and $0.4 million, respectively, held as letters of credit for the benefit of its current landlord. The Company's Boston, Massachusetts sublease expires on February 28, 2026, and the related letter of credit of $0.4 million was classified in current assets on the consolidated balance sheet as of December 31, 2025. The related letter of credit of $0.4 million was classified within other non-current assets on the consolidated balance sheet as of December 31, 2024.

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


	December 31,
	2025		2024
Cash and cash equivalents	$	357,329	$	215,372
Restricted cash	416		416
Total cash, cash equivalents and restricted cash as shown on the consolidated statements of cash flows	$	357,745	$	215,788

Marketable Securities

The Company may invest its excess cash in money market funds and debt instruments of the U.S. Treasury, financial institutions, corporations and U.S. government agencies with strong credit ratings and an investment grade rating at or above A- or A3 by one of the three nationally recognized statistical rating organizations. The Company does not believe that it is exposed to more than a nominal amount of credit risk related to any marketable securities it may invest in. The Company has established guidelines relative to diversification and maturities that maintain safety and liquidity, and periodically reviews and modifies these guidelines to maximize trends in yields and interest rates without compromising safety and liquidity.

The Company classifies its investments in debt instruments as available-for-sale. Available-for-sale investments are reported at fair value at each balance sheet date, and include any unrealized holding gains and losses in accumulated other comprehensive loss, a component of stockholders’ equity. Realized gains and losses are included in the Company's consolidated statements of operations. All of the Company's available-for-sale securities are available for use in its current operations. The Company classifies marketable securities as either cash equivalents, short-term or long-term based on their stated maturity dates as it is more-likely-than-not that the Company will hold these assets through to their maturity dates. The Company held $568.8 million and $254.2 million of marketable securities as of December 31, 2025 and 2024, respectively.

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

Concentrations of Credit Risk and Significant Suppliers and License Agreements

Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of cash, cash equivalents, restricted cash and marketable securities. The Company's investment portfolio may include money market funds, marketable debt securities, including debt securities issued by U.S. government agencies and corporations, and commercial paper. The Company's investments are limited to investment-grade securities with strong credit ratings with the objective of maintaining safety and liquidity. The Company also maintains deposits in accredited financial institutions in excess of federally insured limits. Bank accounts in the United States are insured by the Federal Deposit Insurance Corporation (the “FDIC”) up to $250,000. As of December 31, 2025 and 2024, the Company’s primary operating accounts significantly exceeded the FDIC limits. The Company deposits its cash in financial institutions that it believes have high credit quality, and has not experienced any losses on such accounts and does not believe it is exposed to any unusual credit risk beyond the normal credit risk associated with commercial banking relationships.

The Company is dependent on third-party manufacturers to supply materials for research and development activities of its programs, including preclinical and clinical testing. In particular, the Company relies, and expects to continue to rely, on a small number of third-party manufacturers to produce and process its current and potential product candidates and to manufacture supply of its current and potential product candidates for preclinical and clinical activities. These programs could be adversely affected by a significant interruption in the supply of the necessary materials. The Company is also dependent on third parties who provide license rights used in the development of certain programs. The Company could experience delays in the development of its programs if any

of these license agreements are terminated, if the Company fails to meet the obligations required under its arrangements, or if the Company is unable to successfully secure new strategic alliances or licensing agreements.

Off-Balance Sheet Risk

As of December 31, 2025 and 2024, the Company had no off-balance sheet risk such as foreign exchange contracts, option contracts or other hedging arrangements.

Fair Value Measurements

Certain assets and liabilities of the Company are carried at fair value under GAAP. The fair values of the Company’s financial assets and liabilities reflect the Company’s estimate of the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable:

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

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

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

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

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

Items measured at fair value on a recurring basis include cash equivalents and marketable securities (Note 3 and Note 4). The carrying amounts reflected in the consolidated balance sheets for cash, prepaid expenses and other current assets, accounts payable and accrued expenses approximate their fair values due to the short-term nature of these assets and liabilities.

Property and Equipment

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


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

Upon disposal, retirement or sale, the cost of assets disposed of and the related accumulated depreciation are removed from the accounts and any resulting gain or loss is included in the results of operations. Expenditures for

repairs and maintenance that do not improve or extend the lives of the respective assets are charged to expense as incurred.

Impairment of Long-Lived Assets

Long-lived assets consist of property and equipment. Long-lived assets to be held and used are tested for recoverability whenever events or changes in business circumstances indicate that the carrying amount of the assets may not be fully recoverable. Factors that the Company considers in deciding when to perform an impairment review include significant underperformance of the business in relation to expectations, significant negative industry or economic trends and significant changes or planned changes in the use of the assets. If an impairment review is performed to evaluate a long-lived asset group for recoverability, the Company compares forecasts of undiscounted cash flows expected to result from the use and eventual disposition of the long-lived asset group to its carrying value. An impairment loss would be recognized when estimated undiscounted future cash flows expected to result from the use of an asset group are less than its carrying amount. The impairment loss would be based on the excess of the carrying value of the impaired asset group over its fair value, determined based on discounted cash flows. The Company did not record any impairment losses on long-lived assets during the years ended December 31, 2025, 2024, and 2023.

Leases

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

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

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

The Company’s operating leases are reflected in operating lease right-of-use assets and in current operating lease liabilities and long-term operating lease liabilities in its consolidated balance sheets. The Company’s operating lease right-of-use asset as of December 31, 2025 and 2024 did not include any lease incentives. Lease expense for future lease payments is recognized on a straight-line basis over the lease term.

Collaboration Agreements

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

ASC 808 provides guidance for the presentation and disclosure of transactions in collaborative arrangements, but it does not provide recognition or measurement guidance. Therefore, if the Company concludes a counterparty

to a transaction is not a customer or otherwise not within the scope of ASC 606, the Company considers the guidance in other accounting literature as applicable or by analogy to account for such transaction. The classification of transactions under the Company’s arrangements is determined based on the nature and contractual terms of the arrangement along with the nature of the operations of the participants.

Revenue Recognition

The Company enters into collaboration agreements which are within the scope of ASC 606 under which the Company may license or provide options to license certain development or product candidates and, in certain cases, may perform research and development services. The terms of these agreements may include payment of non-refundable, upfront fees, option exercise fees, development and commercial milestone payments and royalties on net sales of licensed products.

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

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

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

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

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

As of December 31, 2025, the Company had a collaboration and license agreement with Tenacia Biotechnology (Shanghai) Co., Ltd ("Tenacia"), which it entered into in January 2024. Additionally, the Company had

an option and license agreement with UCB Biopharma SRL ("UCB"), which it entered into in December 2022. In December 2024, UCB exercised its option to in-license global development and commercialization rights under the terms of the Collaboration Agreement. The Company has no further research service obligations under the terms of the Collaboration Agreement. The accounting related to these agreements is outlined in Note 8, Collaboration and License Agreements.

Research and Development Costs

Research and development costs are expensed as incurred. Research and development expenses consist of costs incurred in performing research and development activities, including salaries, stock-based compensation and benefits, facilities costs, depreciation, third-party license fees and external costs of outside vendors engaged to conduct preclinical development activities and clinical trials as well as to manufacture research and development materials. Non-refundable prepayments for goods or services that will be used or rendered for future research and development activities are deferred and capitalized. Such amounts are recognized as an expense as the goods are delivered or the related services are performed or until it is no longer expected that the goods will be delivered or the services rendered. Costs incurred to obtain licenses are recognized as research and development expense as incurred if the technology licensed has no alternative future uses.

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

Patent-Related Costs

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

Stock-Based Compensation

The Company accounts for all stock-based awards granted to employees and non-employees as stock-based compensation expense at fair value in accordance with FASB ASC Topic 718, Compensation—Stock Compensation ("ASC 718"). For stock-based awards issued to employees, non-employees and members of the Company’s board of directors (the “Board”) for their services on the Board, the Company measures the estimated fair value of the stock-based award on the date of the grant. The Company recognizes compensation expense for those awards granted to employees and members of the Board over the requisite service period, which is generally the vesting period of the respective award. For non-employee awards, compensation expense is recognized as the services are provided, which is generally ratably over the vesting period. The Company issues stock-based awards with service-based vesting conditions and records the expense for these awards on a straight-line basis over the vesting period. As of December 31, 2025, the Company had not issued any stock-based awards with performance or market-based vesting conditions. The Company accounts for forfeitures as they occur.

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

The fair value of each stock option award is estimated on the date of grant using the Black-Scholes option pricing model, which requires inputs of subjective assumptions, including: (i) the expected volatility of the Company’s stock; (ii) the expected term of the award; (iii) the risk-free interest rate; (iv) expected dividends; and (v) the fair value of common stock. Due to the lack of Company-specific historical and implied volatility data, the Company determines the volatility for awards granted based on an analysis of reported data for a group of guideline publicly-traded companies that issued options with substantially similar terms. For this analysis, the Company selects companies with comparable characteristics including enterprise value, risk profiles, and with historical share

price information sufficient to meet the expected life of the stock-based awards. The Company determines expected volatility using a weighted average of the historical volatilities of the guideline group of companies. The Company expects to continue to apply this process until such time as it has adequate historical data regarding the volatility of its own traded stock price. As permitted under ASC 718, the Company has elected to use the contractual term as the expected term for certain non-employee awards, on an award-by-award basis. For all other awards, the expected term of the Company’s stock options has been determined utilizing the “simplified” method whereby the expected term equals the average of the vesting term and the original contractual term of the option, on a weighted basis based on the vesting of each tranche. The Company utilizes this method as it has insufficient historical exercise data to provide a reasonable basis upon which to estimate the expected term. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for instruments with a term commensurate with the expected term assumption. The expected dividend yield is assumed to be zero as the Company has never paid dividends, and does not have current plans to pay any dividends on its common stock. Subsequent to the IPO, the fair value of the common stock underlying the Company's stock-based awards is the closing price of the Company's common stock on the date of grant.

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

Foreign Currency

Monetary assets and liabilities resulting from transactions denominated in currencies other than the functional currency are remeasured into the functional currency at exchange rates prevailing at the balance sheet date. Non-monetary assets and liabilities denominated in foreign currencies are measured using historical exchange rates prevailing at the date of the transaction and are not subsequently remeasured. Exchange gains or losses arising from foreign currency transactions are included in the determination of net loss. There were no material foreign currency gains or losses for the years ended December 31, 2025, 2024, and 2023.

Income Taxes

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

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

Comprehensive Loss

Comprehensive loss includes net loss and certain changes in stockholders’ equity that are excluded from net loss. For the years ended December 31, 2025, 2024, and 2023, comprehensive loss consists of net loss and changes in unrealized gains and losses on marketable securities.

Common Stock Warrants

The Company accounts for warrants to purchase shares of its common stock in accordance with the guidance in FASB ASC No. 480, Distinguishing Liabilities from Equity ("ASC 480") and ASC No. 815, Derivatives and Hedging ("ASC 815"). The Company classifies warrants issued for the purchase of shares of its common stock as either equity or liability instruments based on an assessment of the specific terms and conditions of each respective contract. Such assessment includes determining whether the warrants are freestanding financial instruments or embedded in a host instrument, whether the warrants are liabilities within the scope of ASC 480, whether the warrants meet the definition of a derivative in ASC 815 and whether the warrants meet the requirements for equity

classification pursuant to the indexation and equity classification criteria in ASC 815. The Company determines the classification for its warrants at the time of issuance and updates its assessment, as necessary. Warrants that meet all of the criteria for equity classification are recorded as a component of additional paid-in capital. Equity classified warrants are accounted for at fair value on the issuance date with no changes in fair value recognized after the issuance date.

Net Loss per Share

Basic net loss per share is computed by dividing net loss by the weighted average number of common shares outstanding for the period. The calculation of weighted average number of common shares outstanding excludes shares of restricted common stock that are not vested but includes shares of common stock underlying pre-funded warrants. Diluted net loss per share is computed by dividing net loss by the weighted average number of common shares outstanding for the period, after giving consideration to the dilutive effect of potentially dilutive common shares. For purposes of this calculation, outstanding options to purchase shares of common stock, unvested shares of restricted common stock and potential shares issuable under the 2020 ESPP are considered potentially dilutive common shares. The Company has generated a net loss in all periods presented so the basic and diluted net loss per share are the same, as the inclusion of the potentially dilutive securities would be anti-dilutive.

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. The tax changes in the OBBBA did not have a material impact to the Company's income tax provision for the year ended December 31, 2025. 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 Adopted Accounting Pronouncements

In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740) - Improvements to Income Tax Disclosures ("ASU 2023-09"). ASU 2023-09 provides more transparency about income tax information through improvements to income tax disclosures primarily related to the rate reconciliation and income taxes paid information. For public companies, the amendments are effective for annual periods beginning after December 15, 2024 and may be applied prospectively or retrospectively. The Company has determined that the effects of adopting the amendments in ASU 2023-09 only impact its disclosures and do not have a material impact on its consolidated financial position and the results of its operations. The amendments were applied prospectively and are reflected within Note 13.

Recently Issued Accounting Pronouncements

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

In September 2025, the FASB issued ASU No. 2025-06, Intangibles - Goodwill and Other - Internal-Use Software (Subtopic 350-40): Targeted Improvements to the Accounting for Internal-Use Software ("ASU 2025-06"), which amends certain aspects of the accounting for and disclosure of software costs under ASC 350-40. The ASU makes targeted improvements to ASC 350-40 to modernize the guidance for internal-use software and better reflect current software development practices. ASU 2025-06 is effective for fiscal periods beginning after December 15,

2027 and interim periods within those fiscal years. The amendments in this update can be applied prospective, retrospective, or a modified transition approach. The Company does not expect the adoption of ASU 2025-06 to have a material impact on its consolidated financial statements.

Common Stock and Preferred Stock - Summary of Common Stock Reserved For Future Issuance (Details) - shares	Dec. 31, 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)	5,322,382	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)	1,876,778	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
Pre-Funded Warrant \| October 2025 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)	318,470	0
Future Share-based Awards \| Shares reserved for future awards under the 2020 Stock Option and Incentive Plan
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	473,358	91,343
Future Share-based Awards \| Shares reserved for future awards under the 2020 Employee Stock Purchase Plan
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	281,180	109,648
Future Share-based Awards \| Shares reserved for future awards under the 2024 Inducement Plan
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	945,280	112,774
Restricted Stock
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Total shares of authorized common stock reserved for future issuance (in shares)	501,853	224,473

Cover Page - USD ($) $ in Millions	12 Months Ended
Cover Page - USD ($) $ in Millions	Dec. 31, 2025	Feb. 16, 2026	Jun. 30, 2025
Cover [Abstract]
Document Type	10-K
Document Annual Report	true
Document Period End Date	Dec. 31, 2025
Current Fiscal Year End Date	--12-31
Document Transition Report	false
Entity File Number	001-39620
Entity Registrant Name	PRAXIS PRECISION MEDICINES, INC.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	47-5195942
Entity Address, Address Line One	99 High Street
Entity Address, Address Line Two	30th Floor
Entity Address, City or Town	Boston
Entity Address, State or Province	MA
Entity Address, Postal Zip Code	02110
City Area Code	617
Local Phone Number	300-8460
Title of 12(b) Security	Common Stock, par value $0.0001 per share
Trading Symbol	PRAX
Security Exchange Name	NASDAQ
Entity Well-known Seasoned Issuer	Yes
Entity Voluntary Filers	No
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
ICFR Auditor Attestation Flag	true
Document Financial Statement Error Correction [Flag]	false
Entity Shell Company	false
Entity Public Float			$ 883.2
Entity Common Stock, Shares Outstanding		27,850,833
Amendment Flag	false
Document Fiscal Year Focus	2025
Document Fiscal Period Focus	FY
Entity Central Index Key	0001689548

Nature of the Business (Details) $ in Thousands	12 Months Ended
Nature of the Business (Details) $ in Thousands	Dec. 31, 2025 USD ($) platform candidate	Dec. 31, 2024 USD ($)	Dec. 31, 2023 USD ($)
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Number of platforms \| platform	2
Number of product candidates \| candidate	4
Proceeds from issuance of equity	$ 1,800,000
Net loss	303,268	$ 182,819	$ 123,277
Accumulated deficit	1,140,008	$ 836,740
Cash, cash equivalents, and short-term investments	$ 926,100

- Definition

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

+ References

Reference 1: http://www.xbrl.org/2003/role/exampleRef
-Topic 946
-SubTopic 830
-Name Accounting Standards Codification
-Section 55
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+ Details

- Definition

Amount of accumulated undistributed earnings (deficit).

+ References + Details

Common Stock and Preferred Stock - Narrative (Details)					1 Months Ended				12 Months Ended
Common Stock and Preferred Stock - Narrative (Details)	Oct. 20, 2025 USD ($) $ / shares shares	Apr. 02, 2024 USD ($) $ / shares shares	Jan. 16, 2024 USD ($) $ / shares shares	Jun. 21, 2023 USD ($) $ / shares shares	Sep. 30, 2025 USD ($)	Dec. 31, 2024 USD ($) $ / shares shares	Mar. 31, 2024 USD ($)	Dec. 31, 2023 USD ($)	Dec. 31, 2025 USD ($) vote $ / shares shares	Dec. 31, 2024 USD ($) $ / shares shares
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Common stock, authorized (in shares)						150,000,000			150,000,000	150,000,000
Common stock, par value (in dollars per share) \| $ / shares						$ 0.0001			$ 0.0001	$ 0.0001
Number of votes per share \| vote									1
Treasury shares (in shares)									0
Preferred stock, authorized (in shares)						10,000,000			10,000,000	10,000,000
Preferred stock, par value (in dollars per share) \| $ / shares						$ 0.0001			$ 0.0001	$ 0.0001
Preferred stock, outstanding (in shares)						0			0	0
Preferred stock, issued (in shares)						0			0	0
Pre-Funded Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Change in ownership notice, term									61 days
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%
2023 Sales Agreement
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Sale of stock, aggregate authorized amount \| $								$ 75,000,000.0
Number of shares issued in transaction (in shares)										192,190
Proceeds from stock offerings, net of issuance costs \| $										$ 5,300,000
2024 Sales Agreement
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Sale of stock, aggregate authorized amount \| $							$ 150,000,000.0
Number of shares issued in transaction (in shares)										1,614,975
Proceeds from stock offerings, net of issuance costs \| $										$ 113,100,000
March Sales Agreement with Jeffries
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Sale of stock, aggregate authorized amount \| $						$ 250,000,000.0
Number of shares issued in transaction (in shares)									1,351,689	16,487
Proceeds from stock offerings, net of issuance costs \| $									$ 83,300,000	$ 1,000,000.0
2025 Sales Agreement
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Sale of stock, aggregate authorized amount \| $					$ 250,000,000.0
Number of shares issued in transaction (in shares)									403,947
Proceeds from stock offerings, net of issuance costs \| $									$ 40,500,000
June 2023 Public Offering
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Proceeds from stock offerings, net of issuance costs \| $				$ 63,400,000
Sale of stock, shares, aggregate authorized amount (in shares)				4,296,646
Sale of stock, price (in dollars per share) \| $ / shares				$ 14.25
June 2023 Public Offering \| Pre-Funded Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Issuance of common stock from exercise of pre-funded warrants (in shares)										469,981
Sale of stock, price (in dollars per share) \| $ / shares				$ 14.2485
Warrants (in shares)				470,000
Warrants (in dollars per share) \| $ / shares				$ 0.0015
January 2024 Follow-On Offering
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Proceeds from stock offerings, net of issuance costs \| $			$ 161,600,000
Sale of stock, shares, aggregate authorized amount (in shares)			3,802,025
Sale of stock, price (in dollars per share) \| $ / shares			$ 35.50
January 2024 Follow-On Offering \| Pre-Funded Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Proceeds from stock offerings, net of issuance costs \| $									$ 0
Sale of stock, price (in dollars per share) \| $ / shares			$ 35.4999
Warrants (in shares)			1,056,725
Warrants (in dollars per share) \| $ / shares			$ 0.0001
Class of warrant or right, outstanding (in shares)									704,225
Over-Allotment Option
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Number of shares issued in transaction (in shares)	501,592	530,973	633,750	619,979
January 2024 Public Offering \| Pre-Funded Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Proceeds from stock offerings, net of issuance costs \| $									$ 0
Issuance of common stock from exercise of pre-funded warrants (in shares)									200,349	152,142
Class of warrants or rights, exercised (in shares)									200,355	152,145
April 2024 Public Offering
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Proceeds from stock offerings, net of issuance costs \| $		$ 216,000,000.0
Sale of stock, shares, aggregate authorized amount (in shares)		3,849,558
Sale of stock, price (in dollars per share) \| $ / shares		$ 56.50
April 2024 Public Offering \| Pre-Funded Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Sale of stock, price (in dollars per share) \| $ / shares		$ 56.4999
Warrants (in shares)		221,238
Warrants (in dollars per share) \| $ / shares		$ 0.0001
Change in ownership notice, term									61 days
April 2024 Public Offering \| 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%
October 2025 Public Offering
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Sale of stock, shares, aggregate authorized amount (in shares)	3,527,072
Sale of stock, price (in dollars per share) \| $ / shares	$ 157.00
Sale of stock, net of discounts, commissions and offering expenses \| $	$ 567,100,000
October 2025 Public Offering \| Pre-Funded Warrant
Schedule Of Common Stock Reserved For Future Issuance [Line Items]
Sale of stock, price (in dollars per share) \| $ / shares	$ 156.9999
Warrants (in shares)	318,470
Warrants (in dollars per share) \| $ / shares	$ 0.0001
Change in ownership notice, term									61 days
October 2025 Public Offering \| 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%

Significant Agreements (Details) - USD ($)	12 Months Ended
Significant Agreements (Details) - USD ($)	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023	Jul. 31, 2023	Dec. 31, 2022	Sep. 11, 2019
Other Commitments [Line Items]
Research and development expense	$ 267,115,000	$ 152,413,000	$ 86,766,000
Ionis
Other Commitments [Line Items]
Research and development expense	0	$ 0	$ 0
License agreement \| RogCon
Other Commitments [Line Items]
Commitment						$ 2,100,000
License agreement \| Ionis
Other Commitments [Line Items]
Commitment					$ 2,000,000.0
License agreement, profit share payments \| RogCon
Other Commitments [Line Items]
Commitment	$ 3,000,000.0
Milestone Payment \| Ionis
Other Commitments [Line Items]
Commitment				$ 6,900,000

Segment Information (Tables)

12 Months Ended

Dec. 31, 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 years ended December 31, 2025, 2024, and 2023 (in thousands):


	December 31,
	2025		2024		2023
Collaboration revenue	$	—	$	8,553	$	2,447
Program-specific expenses:
Ulixacaltamide(a)	63,207		69,172		19,464
Vormatrigine	74,059		18,360		6,275
Relutrigine	45,951		6,273		3,996
Elsunersen	6,833		2,135		14,595
Other early stage assets	8,019		4,634		7,055
Personnel-related expenses	64,719		42,853		33,160
Stock-based compensation expense	33,941		41,361		24,857
Depreciation expense	140		358		432
Other segment expenses(b)	29,329		23,572		18,986
Other income, net	22,930		17,346		3,096
Consolidated net loss	$	(303,268)	$	(182,819)	$	(123,277)

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

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

Income Taxes - Schedule of Income Before Provision For Income Taxes (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Income Tax Disclosure [Abstract]
Domestic	$ (302,839)	$ (182,743)	$ (123,013)
Foreign	42	0	0
Income before income taxes	$ (302,797)	$ (182,743)	$ (123,013)

- Definition

Amount of income (loss) from continuing operations, including income (loss) from equity method investments, before deduction of income tax expense (benefit), and income (loss) attributable to noncontrolling interest.

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-URI https://asc.fasb.org/1943274/2147478671/942-235-S50-1

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

Fair Value of Financial Assets

12 Months Ended

Dec. 31, 2025

Fair Value Disclosures [Abstract]

Fair Value of Financial Assets

The following table presents information about the Company's financial assets measured at fair value on a recurring basis and indicates the level of the fair value hierarchy utilized to determine such fair values as of December 31, 2025 and 2024 (in thousands).


	As of December 31, 2025
	Level 1		Level 2		Level 3		Total
Assets:
Money market funds	$	195,296	$	—	$	—	$	195,296

Debt securities issued by U.S. government agencies	190,999		—		—		190,999
Corporate debt securities	—		198,318		—		198,318
Asset backed securities	—		61,352		—		61,352
Commercial paper	—		7,718		—		7,718
Other debt securities	—		117,374		—		117,374
	$	386,295	$	384,762	$	—	$	771,057


	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

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

- Definition

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

+ References

Reference 1: http://www.xbrl.org/2003/role/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
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-Section 55
-Paragraph 107
-Publisher FASB
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Reference 3: http://www.xbrl.org/2003/role/exampleRef
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-Section 55
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482078/820-10-55-100

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

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 820
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-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
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-Name Accounting Standards Codification
-Section 50
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-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)
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-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
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-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
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-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
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-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
-SubTopic 820
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147478119/940-820-50-1

+ Details

Income Taxes - Schedule of Income Taxes Paid (Details) $ in Thousands	12 Months Ended
	Dec. 31, 2025 USD ($)
Income Tax Disclosure [Abstract]
State	$ 224
Total	$ 224

Commitments and Contingencies

12 Months Ended

Dec. 31, 2025

Commitments and Contingencies Disclosure [Abstract]

Commitments and Contingencies

Leases

In May 2021, the Company entered into a sublease agreement for office space located in Boston, Massachusetts. The sublease expires on February 28, 2026. The base rent increases by approximately 2% annually. The Company issued a letter of credit to the landlord related to the security deposit, secured by restricted cash (Note 2). This lease qualifies as an operating lease. At inception, the Company recorded an operating lease right-of-use asset and operating lease liability of $4.1 million.

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


	As of December 31,
	2025		2024
Assets:
Operating lease right-of-use assets	$	92	$	1,131
Liabilities:
Current operating lease liabilities	$	110	$	1,259
Non-current portion of operating lease liabilities	—		110
Total lease liabilities	$	110	$	1,369

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


	For the year ended December 31,
	2025		2024		2023
Operating lease cost	$	1,102	$	1,102	$	1,102
Variable lease costs	51		33		14
Total lease costs	$	1,153	$	1,135	$	1,116

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

Future lease payments under non-cancelable lease agreements as of December 31, 2025 were as follows (in thousands):


Year Ended December 31,	Future Lease Payments
2026	$	110
2027	—
Total future lease payments	110
Less: interest	—
Present value of operating lease liabilities	$	110

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


	As of December 31,
	2025		2024
Weighted average remaining lease term (in years)	0.1		1.1
Weighted average incremental borrowing rate	9.0	%	9.0	%

In September 2025, the Company entered into a license agreement with the building owner for its existing office space located in Boston, Massachusetts, which will commence on March 1, 2026 and expire on March 31, 2027. As of December 31, 2025, the license agreement had not yet commenced and, accordingly, no right-of-use asset or lease liability or related lease payments had been recorded. Upon commencement, the license agreement will be accounted for as an operating lease under ASC 842.

Legal Proceedings

The Company, from time to time, may be party to litigation arising in the ordinary course of business. The Company was not subject to any material legal proceedings during the years ended December 31, 2025, 2024, and 2023, and no material legal proceedings are currently pending or threatened.

Purchase Orders

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

Indemnification Agreements

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

Agreements

The Company has entered into multiple agreements with third parties under which it may be obligated to make future development, regulatory and commercial milestone payments and royalty payments on future sales of

specified product candidates. Payments under these agreements generally become due and payable upon achievement of such milestones and sales. When the achievement of these milestones or sales have not occurred, such contingencies are not recorded in the Company's financial statements.

Nature of the Business	12 Months Ended
Nature of the Business	Dec. 31, 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 fully integrated, leading central nervous system ("CNS") precision neuroscience biopharmaceutical company translating insights from genetic epilepsies into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance. Normal brain function requires a delicate balance of excitation and inhibition in neuronal circuits, which, when dysregulated, can lead to abnormal function and both rare and more prevalent neurological disorders. The Company is applying genetic insights to the discovery and development of therapies for neurological disorders through two proprietary platforms, using its understanding of shared biological targets and circuits in the brain. Each platform has multiple programs currently, with significant potential for additional program and indication expansion: •Cerebrum™, the Company's small molecule platform, utilizes deep understanding of neuronal excitability and neuronal networks and applies a series of computational and experimental tools to develop orally available precision therapies •Solidus™, the Company's antisense oligonucleotide ("ASO"), platform, is an efficient, targeted precision medicine discovery and development engine anchored on a proprietary, computational methodology The Company's platforms utilize a deliberate, pragmatic and patient-guided approach, leveraging a suite of translational tools, including novel transgenic and predictive translational animal models and electrophysiology markers, to enable an efficient path to proof-of-concept in patients. Through this approach, the Company has established a diversified, multimodal CNS portfolio with four clinical-stage product candidates across movement disorders and epilepsy. Praxis was incorporated in 2015 and commenced operations in 2016. The Company has funded its operations primarily with proceeds from the issuance of redeemable convertible preferred stock, from the sale of common stock through an initial public offering ("IPO"), at-the-market offerings under its shelf registration statement, and follow-on public offerings of common stock and pre-funded warrants to purchase common stock. From inception through December 31, 2025, the Company raised $1.8 billion in aggregate cash proceeds from these transactions, net of issuance costs. The Company is subject to risks and uncertainties common to clinical-stage companies in the biotechnology industry, including but not limited to, risks associated with completing preclinical studies and clinical trials, receiving regulatory approvals for product candidates, development by competitors of new biopharmaceutical products, dependence on key personnel, protection of proprietary technology, compliance with government regulations and the ability to secure additional capital to fund operations. Programs currently under development may require significant additional research and development efforts, including preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will realize revenue from product sales. Liquidity In accordance with the Financial Accounting Standards Board ("FASB") Accounting Standards Update ("ASU") 2014-15, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern (Subtopic 205-40), the Company has evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date that the consolidated financial statements are issued. The Company has incurred recurring losses since its inception, including a net loss of $303.3 million for the year ended December 31, 2025. In addition, as of December 31, 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 December 31, 2025 of $926.1 million, together with net proceeds of $621.2 million from the January 2026 financing (Note 17), 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 consolidated financial statements. The future viability of the Company is dependent on its ability to achieve regulatory approvals of its new drug applications, which are subject to risk and timing uncertainty, and raising additional capital to finance its operations, if needed. 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.

Related Party Transactions	12 Months Ended
Related Party Transactions	Dec. 31, 2025
Related Party Transactions [Abstract]
Related Party Transactions	Related Party Transactions One of the founders of RogCon is the Company's General Counsel. During the years ended December 31, 2025, 2024, and 2023, the Company reimbursed RogCon for its out-of-pocket costs incurred for activities performed under the License Agreement (Note 11). 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
-SubTopic 20
-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
-Topic 946
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 5
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480990/946-20-50-5

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 6
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480990/946-20-50-6

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 235
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477968/946-235-50-2

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Name Accounting Standards Codification
-Section 50
-Paragraph 2
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477968/946-235-50-2

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.6-07(2)(g)(3))
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-URI https://asc.fasb.org/1943274/2147479134/946-220-S99-1

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 220
-Name Accounting Standards Codification
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org/1943274/2147479134/946-220-S99-1

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
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-URI https://asc.fasb.org/850/tableOfContent

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
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-Subparagraph (a)
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-URI https://asc.fasb.org/1943274/2147483326/850-10-50-1

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483326/850-10-50-1

+ Details

Commitments and Contingencies - Total Lease Costs (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Commitments and Contingencies Disclosure [Abstract]
Operating lease cost	$ 1,102	$ 1,102	$ 1,102
Variable lease costs	51	33	14
Total lease costs	$ 1,153	$ 1,135	$ 1,116

Net Loss per Share (Tables)

12 Months Ended

Dec. 31, 2025

Earnings Per Share [Abstract]

Summary of Antidilutive Securities Excluded From Computation of Earnings Per Share

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


	Year Ended December 31,
	2025	2024	2023
Outstanding stock options	1,876,778	1,809,343	666,163
Unvested restricted stock units	501,853	224,473	47,145
Potential shares issuable under the ESPP	4,955	7,315	24,972
	2,383,586	2,041,131	738,280

Stock-Based Compensation - Schedule of Fair Value of Stock Option Awards on the Grant Date Using the Black-Scholes Option Valuation Model (Details) - $ / shares	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Share-Based Payment Arrangement [Abstract]
Risk-free interest rate	4.45%	4.01%	3.60%
Expected term (in years)	6 years	6 years 10 days	6 years
Expected volatility	93.08%	96.60%	88.40%
Expected dividend yield	0.00%	0.00%	0.00%
Weighted average grant-date fair value per share (in dollars per share)	$ 46.74	$ 41.25	$ 29.73

Consolidated Statements of Operations - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Income Statement [Abstract]
Collaboration revenue	$ 0	$ 8,553	$ 2,447
Operating expenses:
Research and development	267,115	152,413	86,766
General and administrative	59,083	56,305	42,054
Total operating expenses	326,198	208,718	128,820
Loss from operations	(326,198)	(200,165)	(126,373)
Other income:
Other income, net	22,930	17,346	3,096
Total other income	22,930	17,346	3,096
Net loss	$ (303,268)	$ (182,819)	$ (123,277)
Net loss per share attributable to common stockholders, basic (in dollars per share)	$ (13.48)	$ (10.21)	$ (18.69)
Net loss per share attributable to common stockholders, diluted (in dollars per share)	$ (13.48)	$ (10.21)	$ (18.69)
Weighted average common shares outstanding, basic (in shares)	22,504,676	17,906,794	6,594,316
Weighted average common shares outstanding, diluted (in shares)	22,504,676	17,906,794	6,594,316

Cash Equivalents and Marketable Securities (Tables)

12 Months Ended

Dec. 31, 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 December 31, 2025 and 2024 (in thousands).


	As of December 31, 2025
			Gross Unrealized				Estimated
	Cost		Gains		Losses		Fair Value
Cash equivalents:
Money market funds	$	195,296	$	—	$	—	$	195,296
Debt securities issued by U.S. government agencies	7,003		—		(1)		7,002
Available-for-sale securities:
Corporate debt securities	198,036		282		—		198,318
Debt securities issued by U.S. government agencies	183,739		258		—		183,997
Asset backed securities	61,282		70		—		61,352
Commercial paper	7,714		4		—		7,718
Other debt securities	117,424		—		(50)		117,374
Total cash equivalents and marketable securities	$	770,494	$	614	$	(51)	$	771,057


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

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


	December 31, 2025		December 31, 2024
Within one year	$	242,002	$	177,195
After one year through five years	326,757		76,961
Total	$	568,759	$	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

Income Taxes (Tables)

12 Months Ended

Dec. 31, 2025

Income Tax Disclosure [Abstract]

Schedule of Income Before Provision For Income Taxes

Income before provision for income taxes was as follows:


	Year Ended December 31,
	2025		2024		2023
Domestic	$	(302,839)	$	(182,743)	$	(123,013)
Foreign	42		—		—
Income before income taxes	$	(302,797)	$	(182,743)	$	(123,013)

Schedule of Components of Provision For Income Taxes

The components of provision for income taxes for all periods presented were as follows:


	Year Ended December 31,
	2025		2024		2023
Current:
State	$	471	$	76	$	45
Total provision	$	471	$	76	$	45

Schedule of Effective Income Tax Rate Reconciliation

A reconciliation of the provision for income taxes to the amount computed by applying the 21% statutory U.S. federal income tax rate to income before income taxes after the adoption of ASU 2023-09 is as follows:


	Year Ended December 31,
	2025
Federal statutory income tax rate	(63,587)		21.0	%
State and local income tax, net of federal income tax effect (1)	417		(0.1)	%
Foreign tax effects
Other foreign jurisdictions	(9)		—	%
Non-taxable or non-deductible items	7		—	%
Stock-based compensation	(3,337)		1.1	%
Limitation on executive compensation	5,508		(1.8)	%
Tax credits	(12,853)		4.2	%
Change in valuation allowance	74,260		(24.5)	%
Other items	65		—	%
Effective income tax rate	$	471	(0.2)	%

(1) The state that makes up the majority of the effect of the state and local income tax line item is Massachusetts.

A reconciliation of the provision for income taxes to the amount computed by applying the 21% statutory U.S. federal income tax rate to income before income taxes for years prior to the adoption of ASU 2023-09 is as follows:


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

Schedule of Deferred Tax Assets and Liabilities

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


	Year Ended December 31,
	2025		2024
Deferred tax assets:
Net operating loss carryforwards	$	170,157	$	90,108
Capitalized research expenditures	59,250		65,836
Tax credits	44,210		30,582
Amortization	19,035		20,600
Stock-based compensation	9,508		8,784
Accrued expenses	4,441		2,333
Leases	28		344
Total gross deferred tax assets	306,629		218,587
Less: Valuation allowance	(306,606)		(218,303)
Net deferred tax assets	$	23	$	284
Deferred tax liabilities:
Operating lease right-of-use asset	(23)		(284)
Total gross deferred tax liabilities	(23)		(284)
Net deferred tax assets	$	—	$	—

Schedule of Income Taxes Paid

The amounts of cash income taxes paid by the Company were as follows:


	Year Ended December 31,
	2025
State	$	224
Total	$	224

Subsequent Events (Details) - Subsequent Event - January 2026 Public Offering $ / shares in Units, $ in Millions	Jan. 07, 2026 USD ($) $ / shares shares
Subsequent Event [Line Items]
Sale of stock, shares, aggregate authorized amount (in shares)	2,543,800
Sale of stock, price (in dollars per share) \| $ / shares	$ 260.00
Number of shares issued in transaction (in shares)	331,800
Proceeds from stock offerings, net of issuance costs \| $	$ 621.2

Cash Equivalents and Marketable Securities - Narrative (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Cash and Cash Equivalents [Abstract]
Cash, cash equivalents, and short-term investments	$ 926,100
Gain (loss) on available-for-sale securities	$ 0	$ 0	$ 0

Income Taxes - Narrative (Details) - USD ($) $ in Thousands	12 Months Ended
Income Taxes - Narrative (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Operating Loss Carryforwards [Line Items]
Operating loss carryforwards, domestic	$ 657,200	$ 342,400
Operating loss carryforwards, subject to expiration	7,700
Operating loss carryforwards, not subject to expiration	649,500
Operating loss carryforwards, state and local	515,600	289,700
Valuation allowance	306,606	218,303
Valuation allowance, increase (decrease), amount	88,300	37,800
UNITED STATES
Operating Loss Carryforwards [Line Items]
Tax credits	40,200	27,300
State
Operating Loss Carryforwards [Line Items]
Tax credits	$ 5,100	$ 4,100

Consolidated Statements of Stockholders' Equity (Parenthetical) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Follow-On Offering
Stock issuance costs	$ 36,635	$ 24,849	$ 4,484
At-The-Market Offering
Stock issuance costs	$ 2,767	$ 3,258	$ 683

Commitments and Contingencies - Operating Lease, Consolidated Balance Sheet (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Commitments and Contingencies Disclosure [Abstract]
Operating lease right-of-use assets	$ 92	$ 1,131
Current operating lease liabilities	110	1,259
Non-current portion of operating lease liabilities	0	110
Total lease liabilities	$ 110	$ 1,369

Common Stock and Preferred Stock

12 Months Ended

Dec. 31, 2025

Stockholders' Equity Note [Abstract]

Common Stock and Preferred Stock

Common Stock

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

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

In December 2024, the Company entered into an amendment to the March Sales Agreement with Jefferies to provide for the offering, issuance, and sale of up to an aggregate amount of $250.0 million of common stock from time to time in at-the-market offerings. During the year ended December 31, 2025, the Company issued and sold an

aggregate of 1,351,689 shares under the amended March 2024 Sales Agreement for aggregate net proceeds of $83.3 million, after deducting commissions and offering expenses payable by the Company. During the year ended December 31, 2024, the Company issued and sold an aggregate of 16,487 shares under the amended March 2024 Sales Agreement for aggregate net proceeds of $1.0 million, after deducting commissions and offering expenses payable by the Company. The amended 2024 Sales Agreement was terminated in September 2025.

In September 2025, the Company entered into a Sales Agreement, or the 2025 Sales Agreement, with TD Securities (USA) LLC, or TD Cowen, to provide for the offering, issuance and sale of up to an aggregate of $250.0 million of common stock from time to time in at-the-market offerings. During the year ended December 31, 2025, the Company issued and sold an aggregate of 403,947 shares under the 2025 Sales Agreement for aggregate net proceeds of $40.5 million, after deducting commissions and offering expenses payable by the Company.

As of December 31, 2025 and 2024, the authorized capital stock of the Company included 150,000,000 shares of common stock, $0.0001 par value, pursuant to the Amended and Restated Certificate of Incorporation effective upon the completion of the IPO. Holders of such shares of common stock have the exclusive right to vote for the election of the Company's directors and are entitled to one vote per share. In the event of the voluntary or involuntary liquidation, dissolution or winding up of the Company, the holders of common stock are entitled to a pro rata distribution of the Company's net assets. Dividends may be declared and paid to such holders only when, as, and if declared by the Board or an authorized committee thereof.

As of December 31, 2025, the Company did not hold any treasury shares.

Follow-On Public Offerings

June 2023 Public Offering

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

During the year ended December 31, 2024, all pre-funded warrants associated with the June 2023 Public Offering were exercised via a cashless exercise, resulting in 469,981 shares of common stock issued. No cash proceeds associated with the exercise were received by the Company. As of December 31, 2025, there were no pre-funded warrants associated with this offering outstanding.

January 2024 Public Offering

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

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 year ended December 31, 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 December 31, 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.

The pre-funded warrants are exercisable at any time on or after the date of issuance at the option of the holder, subject to a beneficial ownership blocker that may limit exercisability. No holder may exercise any portion of the warrants that would cause the aggregate number of shares of common stock beneficially owned by such holder, together with its affiliates, to exceed 4.99% (or 9.99%) of the issued and outstanding common stock. A holder of a pre-funded warrant may increase or decrease this percentage up to 19.99% by providing at least 61 days’ prior notice to the Company. The pre-funded warrants do not expire. The pre-funded warrants may be settled through either physical or net share settlement. Following the occurrence of certain fundamental transactions, the holders of the pre-funded warrants have the right to receive upon exercise of the warrants the same amount and kind of securities, cash, or property as they would have been entitled to receive if they had been holders of the common shares issuable under the warrants immediately prior to such transaction. As of December 31, 2025, none of the pre-funded warrants had been exercised and all remained outstanding.

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 $567.1 million, after deducting underwriting discounts, commissions and other offering expenses payable by the Company.

shares issuable under the warrants immediately prior to such transaction. As of December 31, 2025, none of the pre-funded warrants had been exercised and all remained outstanding.

The Company determined that the pre-funded warrants related to the January 2024, April 2024, and October 2025 public offerings are freestanding financial instruments because they are both legally detachable and separately exercisable from the common stock sold in the offering. As such, the Company evaluated the pre-funded warrants to determine whether they represent instruments that require liability classification pursuant to the guidance in ASC 480. However, the Company concluded that the pre-funded warrants are not a liability within the scope of ASC 480 due to their characteristics. Further, the Company determined that the pre-funded warrants do not meet the definition of a derivative under ASC 815 because they do not meet the criteria regarding no or little initial net investment. Accordingly, the Company assessed the pre-funded warrants relative to the guidance in ASC No. 815-40, Contracts in Entity’s Own Equity, to determine the appropriate treatment. The Company concluded that the pre-funded warrants are both indexed to its own stock and meet all other conditions for equity classification. Accordingly, the Company has classified the pre-funded warrants as permanent equity.

Shares Reserved for Future Issuance

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


	December 31,
	2025	2024
Shares reserved for exercise of outstanding stock options	1,876,778	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 exercise of pre-funded warrants related to the October 2025 Financing	318,470	—
Shares reserved for future awards under the 2020 Stock Option and Incentive Plan	473,358	91,343
Shares reserved for future awards under the 2020 Employee Stock Purchase Plan	281,180	109,648
Shares reserved for future awards under the 2024 Inducement Plan	945,280	112,774
Shares reserved for vesting of restricted stock units	501,853	224,473
Total shares of authorized common stock reserved for future issuance	5,322,382	3,473,399

Preferred Stock

As of December 31, 2025 and 2024, the Company was authorized to issue 10,000,000 shares of undesignated preferred stock, $0.0001 par value, in one or more series, and is authorized to fix the powers, designations, preferences and relative participating option or other rights thereof, including dividend rights, conversion rights, voting rights, redemption rights, liquidation preferences and the number of shares constituting any series, without any further vote or action by the Company's shareholders. As of December 31, 2025 and 2024, the Company had no shares of undesignated preferred stock issued or outstanding.

- Definition

The entire disclosure for equity.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 815
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 8A
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480434/815-10-50-8A

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

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Paragraph 13
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org/1943274/2147481112/505-10-50-14

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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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-Topic 946
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-Name Accounting Standards Codification
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org/505/tableOfContent

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Reference 18: http://www.xbrl.org/2003/role/disclosureRef
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Reference 19: http://www.xbrl.org/2003/role/disclosureRef
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-18

+ Details

Consolidated Balance Sheets (Parenthetical) - $ / shares	Dec. 31, 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, outstanding (in shares)	25,195,092	19,422,358
Common stock, issued (in shares)	25,195,092	19,422,358

Accrued Expenses (Tables)

12 Months Ended

Dec. 31, 2025

Payables and Accruals [Abstract]

Summary of Accrued Expenses

Accrued expenses consisted of the following (in thousands):


	December 31,
	2025		2024
Accrued personnel-related expenses	$	18,713	$	9,365
Accrued external research and development expenses	14,291		13,363
Accrued other expenses	2,029		1,035
Total accrued expenses	$	35,033	$	23,763

Property and Equipment, Net

12 Months Ended

Dec. 31, 2025

Property, Plant and Equipment [Abstract]

Property and Equipment, Net

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


	December 31,
	2025		2024
Computer equipment	$	689	$	689
Office furniture and equipment	545		545
Laboratory equipment	415		359
Total property and equipment	1,649		1,593
Less: Accumulated depreciation	(1,502)		(1,363)
Property and equipment, net	$	147	$	230

Depreciation expense was $0.1 million, $0.4 million, and $0.4 million for the years ended December 31, 2025, 2024, and 2023, respectively.

Collaboration and License Agreements	12 Months Ended
Collaboration and License Agreements	Dec. 31, 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 for the discovery of small molecule therapeutics as potential treatments of KCNT1-related epilepsies. Under the terms of the Collaboration Agreement, the Company 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 exercised its option to in-license global development and commercialization rights, the Collaboration Agreement stipulated that UCB would 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. Accordingly, the Company did not recognize any collaboration revenue associated with this Collaboration Agreement during the year ended December 31, 2025. During the years ended December 31, 2024 and 2023, the Company recognized $8.6 million and $2.4 million, respectively, in collaboration revenue related to the Collaboration Agreement in the consolidated statement of operations. As of December 31, 2025, there was no deferred revenue included in the consolidated balance sheet. Tenacia Collaboration and License Agreement On January 4, 2024, the Company entered into an exclusive collaboration and license Agreement (“the License Agreement”) with Tenacia, a China-based portfolio company of Bain Capital, which provides Tenacia an exclusive license to use certain intellectual property for the development and commercialization of ulixacaltamide and products containing ulixacaltamide in China, Hong Kong, Macau and Taiwan. Tenacia is solely responsible for the development and commercialization under the arrangement, with the exception of the associated manufacturing. The Company also entered into a Stock Purchase Agreement (“the Stock Purchase Agreement”) with BCPE Tenet Holdings Cayman, Ltd. (“BCPE”), a related party of Tenacia. Pursuant to the terms of the License Agreement, the Company was entitled to an up-front, non-refundable and non-creditable cash payment of $5.0 million, net of certain tax withholdings. In addition, the Company is eligible to receive $264.0 million in success-based development and commercialization milestone payments as well as tiered royalties on net sales. Pursuant to the terms of the Stock Purchase Agreement, the Company issued and sold 443,253 shares of its common stock to BCPE at a price per share of $22.5605 for aggregate gross proceeds of $10.0 million. The per share price was based on a 20% premium over the 30-day volume-weighted average price. Under the terms of the License Agreement, the Company granted to Tenacia an exclusive license to use certain intellectual property for the development and commercialization of ulixacaltamide and products containing ulixacaltamide in China, Hong Kong, Macau and Taiwan. Tenacia is solely responsible for the development and commercialization under the arrangement, with the exception of the associated manufacturing. The Company concluded that the License Agreement and the Stock Purchase Agreement is a combined arrangement since they were executed at the same time and in contemplation of each other with the same counterparty or a related party thereof and the combined arrangement falls within the scope of Topic 606. The Company’s obligations under the arrangement comprise a single promise, or one performance obligation, related to the exclusive development and commercialization license granted to Tenacia. Total proceeds associated with the combined arrangement at inception were $14.8 million consisting of the following: (i) $10.0 million gross proceeds from the sale of common stock under the Stock Purchase Agreement and (ii) $4.8 million, net of tax, related to the up-front payment under the License Agreement, both of which were received in January 2024. During the years ended December 31, 2025 and 2024, the Company had not achieved any development or sales milestones or earned any royalties under the License Agreement. The Company recorded the common stock sold to BCPE at its issuance date fair value of $38.95 per share, or $17.3 million in the aggregate, which exceeded the proceeds received which were calculated based on the 20% premium over the prior 30-day volume-weighted average price. Accordingly, there is no transaction price allocable to the performance obligation. The Company accounted for the excess of the fair value of the equity securities issued over the total proceeds received as consideration paid to a customer for which no distinct good or service was transferred in exchange. As a result, the transaction gives rise to negative revenue on a cumulative basis in the amount of $2.5 million, which was recorded at the inception of the collaboration and license agreement in research and development expense in the accompanying consolidated statement of operations. For agreements where the licenses are considered separate performance obligations or represent the only performance obligation, the Company recognizes revenue at the point in time that the Company effectively grants the license, as the licenses or assignments represent functional intellectual property. As of December 31, 2025 and 2024, the Company did not have any receivables or deferred revenue related to the arrangement with Tenacia. The Company did not recognize any contract assets related to costs to obtain a contract with a customer or costs to fulfill a contract with a customer through December 31, 2025 because no qualifying costs were incurred. During the years ended December 31, 2025, 2024, and 2023, the Company did not recognize any revenue associated with the combined arrangement.

Summary of Significant Accounting Policies (Policies)

12 Months Ended

Dec. 31, 2025

Accounting Policies [Abstract]

Basis of Presentation

Principles of Consolidation

Use of Estimates

Cash, Cash Equivalents and Restricted Cash

Marketable Securities

Concentrations of Credit Risk and Significant Suppliers and License Agreements

Off-Balance Sheet Risk

As of December 31, 2025 and 2024, the Company had no off-balance sheet risk such as foreign exchange contracts, option contracts or other hedging arrangements.

Fair Value Measurements

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

Property and Equipment

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


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

repairs and maintenance that do not improve or extend the lives of the respective assets are charged to expense as incurred.

Impairment of Long-Lived Assets

Leases

Collaboration Agreements and Revenue Recognition

Collaboration Agreements

Revenue Recognition

Research and Development Costs and Patent-Related Costs

Research and Development Costs

Patent-Related Costs

Stock-Based Compensation

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

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

Foreign Currency

Net Loss per Share

Income Taxes / Recently Enacted Tax Legislation

Income Taxes

Recently Enacted Tax Legislation

Comprehensive Loss

Common Stock Warrants

Recent Accounting Pronouncements

Recently Adopted Accounting Pronouncements

Recently Issued Accounting Pronouncements

Segment Information

12 Months Ended

Dec. 31, 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 consolidated statement of operations as consolidated net loss. Net loss is used by the CODM to make key strategic and operational decisions. The operating segment's revenue is derived from a single domestic collaboration agreement from which the segment licenses certain development or product candidates and has performed research and development services. The measure of segment assets is reported on the balance sheet as total consolidated assets. The majority of the Company's long-lived assets are held in the United States.

The following table presents selected financial information about the Company's single operating segment for the years ended December 31, 2025, 2024, and 2023 (in thousands):


	December 31,
	2025		2024		2023
Collaboration revenue	$	—	$	8,553	$	2,447
Program-specific expenses:
Ulixacaltamide(a)	63,207		69,172		19,464
Vormatrigine	74,059		18,360		6,275
Relutrigine	45,951		6,273		3,996
Elsunersen	6,833		2,135		14,595
Other early stage assets	8,019		4,634		7,055
Personnel-related expenses	64,719		42,853		33,160
Stock-based compensation expense	33,941		41,361		24,857
Depreciation expense	140		358		432
Other segment expenses(b)	29,329		23,572		18,986
Other income, net	22,930		17,346		3,096
Consolidated net loss	$	(303,268)	$	(182,819)	$	(123,277)

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

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

- Definition

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

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

Accrued Expenses (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Payables and Accruals [Abstract]
Accrued personnel-related expenses	$ 18,713	$ 9,365
Accrued external research and development expenses	14,291	13,363
Accrued other expenses	2,029	1,035
Total accrued expenses	$ 35,033	$ 23,763

Summary of Significant Accounting Policies - Narrative (Details) - USD ($)	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Accounting Policies [Abstract]
Restricted cash	$ 416,000	$ 416,000	$ 416,000
Letters of credit receivable, noncurrent	400,000	400,000
Marketable securities	568,800,000	254,200,000
Impairment losses	$ 0	$ 0	$ 0
Expected dividend yield	0.00%	0.00%	0.00%

Common Stock and Preferred Stock (Tables)

12 Months Ended

Dec. 31, 2025

Stockholders' Equity Note [Abstract]

Summary of Common Stock Reserved For Future Issuance

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


	December 31,
	2025	2024
Shares reserved for exercise of outstanding stock options	1,876,778	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 exercise of pre-funded warrants related to the October 2025 Financing	318,470	—
Shares reserved for future awards under the 2020 Stock Option and Incentive Plan	473,358	91,343
Shares reserved for future awards under the 2020 Employee Stock Purchase Plan	281,180	109,648
Shares reserved for future awards under the 2024 Inducement Plan	945,280	112,774
Shares reserved for vesting of restricted stock units	501,853	224,473
Total shares of authorized common stock reserved for future issuance	5,322,382	3,473,399

Property and Equipment, Net - Narrative (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Property, Plant and Equipment [Abstract]
Depreciation expense	$ 140	$ 358	$ 432

Subsequent Events	12 Months Ended
Subsequent Events	Dec. 31, 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. January 2026 Public Offering On January 7, 2026, the Company completed a public offering of 2,543,800 shares of its common stock at a public offering price of $260.00 per share, including the underwriters' full exercise of their option to purchase 331,800 additional shares of common stock. Total net proceeds generated from the offering were approximately $621.2 million, after deducting underwriting discounts, commissions and other offering expenses payable by the Company.

Fair Value of Financial Assets (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	$ 195,296	$ 128,652
Assets	771,057	398,763
Debt securities issued by U.S. government agencies
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	190,999	144,644
Corporate debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	198,318	107,852
Asset backed securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	61,352
Commercial paper
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	7,718	8,794
Other debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	117,374	8,821
Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	195,296	128,652
Assets	386,295	273,296
Level 1 \| Debt securities issued by U.S. government agencies
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	190,999	144,644
Level 1 \| Corporate debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	0	0
Level 1 \| Asset backed securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	0
Level 1 \| Commercial paper
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	0	0
Level 1 \| Other debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	0	0
Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	0	0
Assets	384,762	125,467
Level 2 \| Debt securities issued by U.S. government agencies
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	0	0
Level 2 \| Corporate debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	198,318	107,852
Level 2 \| Asset backed securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	61,352
Level 2 \| Commercial paper
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	7,718	8,794
Level 2 \| Other debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	117,374	8,821
Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	0	0
Assets	0	0
Level 3 \| Debt securities issued by U.S. government agencies
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	0	0
Level 3 \| Corporate debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	0	0
Level 3 \| Asset backed securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	0
Level 3 \| Commercial paper
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	0	0
Level 3 \| Other debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total available-for-sale securities, estimated fair value	$ 0	$ 0

Cybersecurity Risk Management and Strategy Disclosure	12 Months Ended
Cybersecurity Risk Management and Strategy Disclosure	Dec. 31, 2025
Cybersecurity Risk Management, Strategy, and Governance [Line Items]
Cybersecurity Risk Management Processes for Assessing, Identifying, and Managing Threats [Text Block]	We have developed and implemented a cybersecurity risk management program intended to protect the confidentiality, integrity and availability of our critical systems and information. We design and assess our cybersecurity risk management program based on the National Institute of Standards and Technology Cybersecurity Framework, or NIST CSF, and integrate recognized security best practices relevant to our business. This does not imply that we meet any particular technical standards, specifications, or requirements, only that we use the NIST CSF as a guide to help us identify, assess, and manage cybersecurity risks relevant to our business. As our operations scale and we transition to commercialization, our cybersecurity risk management program is designed to scale accordingly, including through enhanced governance, layered security controls, third-party oversight and continuous monitoring. Our cybersecurity risk management program is integrated into our overall risk management framework, and shares common methodologies, reporting channels and governance processes across legal, compliance, strategic, operational and financial risk areas. Key elements of our cybersecurity risk management program include but are not limited to: •risk assessments designed to help identify material risks from cybersecurity threats to our critical systems and information; •adoption of layered cybersecurity controls, including enhanced identity and access controls, automated detection and remediation for email threats, endpoint and network protections, centralized security monitoring and retention of security logs sufficient to support investigations and audit evidence; •a security team principally responsible for managing (1) our cybersecurity risk-assessment processes, (2) our security controls, (3) our response to cybersecurity incidents, and (4) the ongoing improvement of our security posture through measurement, testing, threat-hunting and lessons-learned activities; •the use of external service providers, where appropriate, to assess, test or otherwise assist with aspects of our security processes, including 24/7 monitoring and additional technical expertise, and periodic penetration and vulnerability testing; •cybersecurity awareness training for our employees, including incident response personnel and senior management; •a cybersecurity incident response plan that includes procedures for responding to cybersecurity incidents; and •security audits and a third-party risk assessment process for key service providers, suppliers and vendors based on our assessment of their criticality to our operations and respective risk profile.
Cybersecurity Risk Management Processes Integrated [Flag]	true
Cybersecurity Risk Management Processes Integrated [Text Block]	Our cybersecurity risk management program is integrated into our overall risk management framework, and shares common methodologies, reporting channels and governance processes across legal, compliance, strategic, operational and financial risk areas.
Cybersecurity Risk Management Third Party Engaged [Flag]	true
Cybersecurity Risk Third Party Oversight and Identification Processes [Flag]	true
Cybersecurity Risk Materially Affected or Reasonably Likely to Materially Affect Registrant [Flag]	false
Cybersecurity Risk Board of Directors Oversight [Text Block]	Our Board of Directors considers cybersecurity risk as part of its risk oversight function and has delegated to the Audit Committee oversight of cybersecurity and risks, including oversight of management's implementation of our cybersecurity risk management program. The Audit Committee receives regular updates from management on cybersecurity risks, control maturity and significant cybersecurity matters, including those related to systems surrounding commercial operations. In addition, management updates the Audit Committee, when it deems appropriate, regarding cybersecurity incidents it considers to be significant or potentially significant. The Audit Committee reports to the full Board regarding its activities, including those related to cybersecurity.
Cybersecurity Risk Board Committee or Subcommittee Responsible for Oversight [Text Block]	Our Board of Directors considers cybersecurity risk as part of its risk oversight function and has delegated to the Audit Committee oversight of cybersecurity and risks, including oversight of management's implementation of our cybersecurity risk management program. The Audit Committee receives regular updates from management on cybersecurity risks, control maturity and significant cybersecurity matters, including those related to systems surrounding commercial operations. In addition, management updates the Audit Committee, when it deems appropriate, regarding cybersecurity incidents it considers to be significant or potentially significant. The Audit Committee reports to the full Board regarding its activities, including those related to cybersecurity.
Cybersecurity Risk Process for Informing Board Committee or Subcommittee Responsible for Oversight [Text Block]	The Audit Committee receives regular updates from management on cybersecurity risks, control maturity and significant cybersecurity matters, including those related to systems surrounding commercial operations. In addition, management updates the Audit Committee, when it deems appropriate, regarding cybersecurity incidents it considers to be significant or potentially significant. The Audit Committee reports to the full Board regarding its activities, including those related to cybersecurity.
Cybersecurity Risk Role of Management [Text Block]	We maintain an internal cybersecurity team, reporting to our Chief Financial Officer, who is responsible for managing our cybersecurity risk-management program, assessing and mitigating cyber risks, and overseeing retained cybersecurity consultants and providers for additional external expertise. This internal team is led by our Vice President of Information Technology, who has over 20 years of experience managing cybersecurity, systems architecture and complex data operations for biotechnology and pharmaceutical companies. Additional team members have between 12 to 15 years of experience each in cybersecurity, IT management and cloud architecture. The internal team works closely with external network operations and cybersecurity teams who provide 24/7 network operations and cybersecurity review and response capabilities. We have an Executive Compliance Committee consisting of executive officers and members from senior management across the regulatory, quality, legal, finance and people operations functions that takes steps to stay informed about and monitor efforts to prevent, detect, mitigate and remediate cybersecurity risks and incidents through various means, which includes updates from our internal cybersecurity team, as relevant; review of any threat intelligence and other information obtained from governmental, public or private sources, including external consultants engaged by us; and alerts and reports produced by security tools deployed in our IT environment.
Cybersecurity Risk Management Positions or Committees Responsible [Flag]	true
Cybersecurity Risk Management Positions or Committees Responsible [Text Block]	We maintain an internal cybersecurity team, reporting to our Chief Financial Officer, who is responsible for managing our cybersecurity risk-management program, assessing and mitigating cyber risks, and overseeing retained cybersecurity consultants and providers for additional external expertise. This internal team is led by our Vice President of Information Technology, who has over 20 years of experience managing cybersecurity, systems architecture and complex data operations for biotechnology and pharmaceutical companies. Additional team members have between 12 to 15 years of experience each in cybersecurity, IT management and cloud architecture. The internal team works closely with external network operations and cybersecurity teams who provide 24/7 network operations and cybersecurity review and response capabilities. We have an Executive Compliance Committee consisting of executive officers and members from senior management across the regulatory, quality, legal, finance and people operations functions that takes steps to stay informed about and monitor efforts to prevent, detect, mitigate and remediate cybersecurity risks and incidents through various means, which includes updates from our internal cybersecurity team, as relevant; review of any threat intelligence and other information obtained from governmental, public or private sources, including external consultants engaged by us; and alerts and reports produced by security tools deployed in our IT environment.
Cybersecurity Risk Management Expertise of Management Responsible [Text Block]	This internal team is led by our Vice President of Information Technology, who has over 20 years of experience managing cybersecurity, systems architecture and complex data operations for biotechnology and pharmaceutical companies. Additional team members have between 12 to 15 years of experience each in cybersecurity, IT management and cloud architecture. The internal team works closely with external network operations and cybersecurity teams who provide 24/7 network operations and cybersecurity review and response capabilities.
Cybersecurity Risk Process for Informing Management or Committees Responsible [Text Block]	The Audit Committee receives regular updates from management on cybersecurity risks, control maturity and significant cybersecurity matters, including those related to systems surrounding commercial operations. In addition, management updates the Audit Committee, when it deems appropriate, regarding cybersecurity incidents it considers to be significant or potentially significant.
Cybersecurity Risk Management Positions or Committees Responsible Report to Board [Flag]	true

Stock-Based Compensation - Summary of Stock Option Activity (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024
Number of Shares
Beginning balance (in shares)	1,809,343
Granted (in shares)	324,670
Exercised (in shares)	(220,261)
Canceled or forfeited (in shares)	(36,974)
Ending balance (in shares)	1,876,778	1,809,343
Exercisable (in shares)	1,101,369
Vested and expected to vest (in shares)	1,876,778
Weighted Average Exercise Price per Share
Beginning balance (in dollars per share)	$ 90.84
Granted (in dollars per share)	60.03
Exercised (in dollars per share)	57.01
Cancelled or forfeited (in dollars per share)	74.65
Ending balance (in dollars per share)	89.80	$ 90.84
Exercisable (in dollars per share)	112.82
Vested and expected to vest (in dollars per share)	$ 89.80
Weighted Average Remaining Contractual Term and Aggregate Intrinsic Value
Weighted average remaining contractual term outstanding	7 years 8 months 19 days	8 years 5 months 19 days
Weighted average remaining contractual term exercisable	7 years 1 month 9 days
Weighted average remaining contractual term vested and expected to vest	7 years 8 months 19 days
Aggregate intrinsic value exercised	$ 27,608	$ 1,200
Aggregate intrinsic value outstanding	409,936	$ 39,989
Aggregate intrinsic value exercisable	225,671
Aggregate intrinsic value vested and expected to vest	$ 409,936

Fair Value of Financial Assets (Tables)

12 Months Ended

Dec. 31, 2025

Fair Value Disclosures [Abstract]

Summary of Financial Assets Measured At Fair Value On A Recurring Basis


	As of December 31, 2025
	Level 1		Level 2		Level 3		Total
Assets:
Money market funds	$	195,296	$	—	$	—	$	195,296

Debt securities issued by U.S. government agencies	190,999		—		—		190,999
Corporate debt securities	—		198,318		—		198,318
Asset backed securities	—		61,352		—		61,352
Commercial paper	—		7,718		—		7,718
Other debt securities	—		117,374		—		117,374
	$	386,295	$	384,762	$	—	$	771,057


	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

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

Income Taxes

12 Months Ended

Dec. 31, 2025

Income Tax Disclosure [Abstract]

Income Taxes

The Company maintains a full valuation allowance on its U.S. net deferred tax assets due to the uncertainty of future taxable income. The Company did not recognize any income tax benefit in the years ended December 31, 2025, 2024, and 2023 related to its U.S. operations due to the uncertainty regarding future taxable income. In the years ended December 31, 2025, 2024, and 2023, the difference between the statutory tax rate in the U.S. and the Company’s effective tax rate was due primarily to the valuation allowance recorded to offset any potential tax benefit.

Income before provision for income taxes was as follows:


	Year Ended December 31,
	2025		2024		2023
Domestic	$	(302,839)	$	(182,743)	$	(123,013)
Foreign	42		—		—
Income before income taxes	$	(302,797)	$	(182,743)	$	(123,013)

The components of provision for income taxes for all periods presented were as follows:


	Year Ended December 31,
	2025		2024		2023
Current:
State	$	471	$	76	$	45
Total provision	$	471	$	76	$	45


	Year Ended December 31,
	2025
Federal statutory income tax rate	(63,587)		21.0	%
State and local income tax, net of federal income tax effect (1)	417		(0.1)	%
Foreign tax effects
Other foreign jurisdictions	(9)		—	%
Non-taxable or non-deductible items	7		—	%
Stock-based compensation	(3,337)		1.1	%
Limitation on executive compensation	5,508		(1.8)	%
Tax credits	(12,853)		4.2	%
Change in valuation allowance	74,260		(24.5)	%
Other items	65		—	%
Effective income tax rate	$	471	(0.2)	%

(1) The state that makes up the majority of the effect of the state and local income tax line item is Massachusetts.


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

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


	Year Ended December 31,
	2025		2024
Deferred tax assets:
Net operating loss carryforwards	$	170,157	$	90,108
Capitalized research expenditures	59,250		65,836
Tax credits	44,210		30,582
Amortization	19,035		20,600
Stock-based compensation	9,508		8,784
Accrued expenses	4,441		2,333
Leases	28		344
Total gross deferred tax assets	306,629		218,587
Less: Valuation allowance	(306,606)		(218,303)
Net deferred tax assets	$	23	$	284
Deferred tax liabilities:
Operating lease right-of-use asset	(23)		(284)
Total gross deferred tax liabilities	(23)		(284)
Net deferred tax assets	$	—	$	—

As of December 31, 2025 and 2024, the Company had U.S. federal net operating loss carryforwards which may be able to offset future income tax liabilities of approximately $657.2 million and $342.4 million, respectively. Federal net operating loss carryforwards of $7.7 million will expire at various dates from 2035 through 2037 and approximately $649.5 million may be carried forward indefinitely. As of December 31, 2025 and 2024, the Company also had state net operating loss carryforwards of approximately $515.6 million and $289.7 million, respectively, which may be available to offset future income tax liabilities and expire at various dates from 2031 through 2045.

As of December 31, 2025 and 2024, the Company had federal research and development tax credit and orphan drug tax credit carryforwards of approximately $40.2 million and $27.3 million, respectively, available to reduce future tax liabilities which expire at various dates from 2039 through 2045. As of December 31, 2025 and 2024, the Company had state research and development tax credit carryforwards of approximately $5.1 million and $4.1 million, respectively, available to reduce future tax liabilities which expire at various dates from 2032 through 2040. The Company has generated research and orphan drug tax credits but has not conducted a study to document the qualified activity. This study may result in an adjustment to the Company’s research and development credit carryforwards; however, until a study is completed, and any adjustment is known, no amounts are being presented as an uncertain tax position. A full valuation allowance has been provided against the Company’s research and development credits and, if a deferred tax asset adjustment is required, this adjustment would be offset by the valuation allowance.

Under the provisions of the Internal Revenue Code, the net operating loss and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities. Net operating loss and tax credit carryforwards may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant shareholders over a three-year period in excess of 50%, as defined under Sections 382 and 383 of the Internal Revenue Code, respectively, as well as similar state provisions. This could limit the amount of tax attributes that can be utilized annually to offset future taxable income or tax liabilities. The amount of the annual limitation is determined based on the value of the Company immediately prior to the ownership change. Subsequent ownership changes may further affect the limitation in future years. The Company has not conducted a study to assess whether a change of control has occurred or whether there have been multiple changes of control since inception due to the significant complexity and cost associated with such a study. If the Company has experienced a change of control, as defined by Section 382, at any time since inception, utilization of the net operating loss carryforwards or research and development tax credit carryforwards would be subject to an annual limitation under Section 382, which is determined by first multiplying the value of the Company’s stock at the

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

ASC 740 requires a valuation allowance to reduce the deferred tax assets reported if, based on the weight of the evidence, it is more likely than not that some portion or all of the deferred tax assets will not be realized. After consideration of all of the evidence, both positive and negative, the Company has recorded a valuation allowance against its deferred tax assets at December 31, 2025 and 2024 because management has determined that it is more likely than not that the Company will not recognize the benefits of its federal and state deferred tax assets, primarily due to its history of cumulative net losses incurred since inception and its lack of commercialization of any products or generation of any revenue from product sales since inception. As a result, a valuation allowance of $306.6 million and $218.3 million has been established at December 31, 2025 and 2024, respectively. Management reevaluates the positive and negative evidence at each reporting period. The valuation allowance increased by approximately $88.3 million and $37.8 million during the years ended December 31, 2025 and 2024, respectively, due primarily to the generation of net operating losses.

The Company has not recorded any amounts for unrecognized tax benefits as of December 31, 2025 and 2024. The Company’s policy is to record interest and penalties related to uncertain tax positions as part of its income tax provision. As of December 31, 2025 and 2024, the Company had no accrued interest or penalties related to uncertain tax positions and no such amounts have been recognized in the Company’s consolidated statement of operations for the years ended December 31, 2025, 2024, and 2023. The statute of limitations for federal and the majority of state tax authorities is open for tax years ended December 31, 2022 through December 31, 2025. The statute of limitation for the remaining state tax authorities is open for tax years ended December 31, 2021 through December 31, 2025. Since the Company is in a net loss carryforward position, the Company is generally subject to examination by the U.S. federal, state and local income tax authorities for all tax years in which a loss carryforward is available.

The amounts of cash income taxes paid by the Company were as follows:


	Year Ended December 31,
	2025
State	$	224
Total	$	224

- Definition

The entire disclosure for income tax.

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+ Details

Employee Benefit Plan	12 Months Ended
Employee Benefit Plan	Dec. 31, 2025
Retirement Benefits [Abstract]
Employee Benefit Plan	Employee Benefit Plan The Company has a defined contribution savings plan under Section 401(k) of the Internal Revenue Code for eligible employees. The plan covers substantially all employees who meet a minimum age requirement and allows participants to defer a portion of their annual compensation. Under the plan, the Company is not obligated to match any participant contributions. The Company made contributions of $1.6 million, $1.2 million, and $1.0 million during the years ended December 31, 2025, 2024, and 2023, respectively.

- Definition

The entire disclosure for retirement benefits.

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+ Details

Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Statement of Comprehensive Income [Abstract]
Net loss	$ (303,268)	$ (182,819)	$ (123,277)
Change in unrealized gain (loss) on marketable securities, net of tax	(91)	654	173
Comprehensive loss	$ (303,359)	$ (182,165)	$ (123,104)

- Definition

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

+ References + Details

- Definition

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

+ References

+ Details

Income Taxes - Schedule of Components of Provision For Income Taxes (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Current:
State	$ 471	$ 76	$ 45
Total provision	$ 471	$ 76	$ 45

Consolidated Balance Sheets - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Current assets:
Cash and cash equivalents	$ 357,329	$ 215,372
Marketable securities	242,002	177,195
Prepaid expenses and other current assets	11,580	11,805
Total current assets	610,911	404,372
Long-term marketable securities	326,757	76,961
Property and equipment, net	147	230
Operating lease right-of-use assets	92	1,131
Other non-current assets	0	416
Total assets	937,907	483,110
Current liabilities:
Accounts payable	24,628	12,528
Accrued expenses	35,033	23,763
Operating lease liabilities	110	1,259
Total current liabilities	59,771	37,550
Long-term liabilities:
Non-current portion of operating lease liabilities	0	110
Total liabilities	59,771	37,660
Commitments and contingencies (Note 7)
Stockholders’ equity:
Preferred stock, $0.0001 par value; 10,000,000 shares authorized and no shares issued or outstanding as of December 31, 2025 and December 31, 2024	0	0
Common stock, $0.0001 par value; 150,000,000 shares authorized; 25,195,092 shares issued and outstanding as of December 31, 2025, and 19,422,358 shares issued and outstanding as of December 31, 2024	15	14
Additional paid-in capital	2,017,566	1,281,522
Accumulated deficit	(1,140,008)	(836,740)
Accumulated other comprehensive gain	563	654
Total stockholders’ equity	878,136	445,450
Total liabilities and stockholders’ equity	$ 937,907	$ 483,110

Stock-Based Compensation

12 Months Ended

Dec. 31, 2025

Share-Based Payment Arrangement [Abstract]

Stock-Based Compensation

Equity Incentive Plans

In January 2024, the Board of Directors ("the Board") adopted the 2024 Inducement Plan (the "Inducement Plan") without stockholder approval pursuant to Rule 5635(c)(4) of the Nasdaq Stock Market LLC listing rules ("Rule 5635(c)(4)"). In accordance with Rule 5635(c)(4), the Inducement Plan allows the Company to grant awards only to a newly hired employee who was not previously an employee or non-employee director or to an employee who is being rehired following a bona fide period of non-employment if such award is a material inducement to such employee entering into employment. 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 December 31, 2025 and 2024 was 1,000,000 and 130,000 shares, respectively.

The total number of shares of common stock authorized for issuance under the 2020 Plan as of December 31, 2025 and December 31, 2024 was 2,941,950 shares and 1,970,833 shares, respectively. The total number of shares of common stock authorized for issuance under the 2017 Stock Incentive Plan (the "2017 Plan") as of December 31, 2025 and 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.

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

The total shares authorized for issuance under the 2020 Employee Stock Purchase Plan (the "2020 ESPP") as of December 31, 2025 and 2024 was 369,368 shares and 175,145 shares, respectively. During the years ended December 31, 2025, 2024, and 2023, the Company issued 22,691, 31,381, and 24,690 shares, respectively, under the 2020 ESPP.

Restricted Stock Units

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


	Shares	Weighted Average Grant Date Fair Value
Unvested as of December 31, 2024	224,473	$	71.71
Issued	414,151	67.60
Vested	(86,005)	105.57
Forfeited	(50,766)	55.89
Unvested as of December 31, 2025	501,853	$	64.12

As of December 31, 2025, total unrecognized compensation cost related to unvested restricted stock units was $23.7 million, which is expected to be recognized over a weighted-average period of 2.97 years.

The total fair value of restricted stock units that vested during the years ended December 31, 2025 and 2024 was $8.7 million and $1.0 million, respectively.

Stock Options

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


	Number of Shares	Weighted Average Exercise Price per Share		Weighted Average Remaining Contractual Term	Aggregate Intrinsic Value
				(In years)	(In thousands)
Outstanding as of December 31, 2024	1,809,343	$	90.84	8.47	$	39,989
Granted	324,670	60.03
Exercised	(220,261)	57.01			$	27,608
Cancelled or Forfeited	(36,974)	74.65
Outstanding as of December 31, 2025	1,876,778	$	89.80	7.72	$	409,936
Exercisable as of December 31, 2025	1,101,369	$	112.82	7.11	$	225,671
Vested and expected to vest as of December 31, 2025	1,876,778	$	89.80	7.72	$	409,936

The aggregate intrinsic value of stock options exercised for the years ended December 31, 2025 and 2024 was $27.6 million and $1.2 million, respectively. The aggregate intrinsic value of stock options outstanding, exercisable and vested and expected to vest is calculated as the difference between the exercise price of the underlying stock options and the estimated fair value of the Company’s common stock for those stock options that had exercise prices lower than the estimated fair value of the Company’s common stock at December 31, 2025. The aggregate intrinsic value of stock options exercised is calculated as the difference between the exercise price of the underlying stock options and the estimated fair value of the Company’s common stock on the date of exercise for those stock options that had exercise prices lower than the fair value of the Company’s common stock on the exercise date.

Valuation of Stock Options

The weighted-average assumptions that the Company used in the Black-Scholes option pricing model to determine the grant-date fair value of stock options granted to employees, members of the Board, and non-employees on the date of grant were as follows for the for the years ended December 31, 2025, 2024, and 2023:


	Year Ended December 31,
	2025			2024			2023
Risk-free interest rate	4.45		%	4.01		%	3.60		%
Expected term (in years)	6.00			6.03			6.00
Expected volatility	93.08		%	96.60		%	88.40		%
Expected dividend yield	—		%	—		%	—		%
Weighted average grant-date fair value per share	$	46.74		$	41.25		$	29.73

As of December 31, 2025, total unrecognized compensation cost related to unvested stock options was $30.8 million, which is expected to be recognized over a weighted-average period of 2.10 years.

Stock-Based Compensation

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


	Year Ended December 31,
	2025		2024		2023
Research and development	$	15,286	$	15,620	$	8,055
General and administrative	18,655		25,740		16,802
Total stock-based compensation expense	$	33,941	$	41,360	$	24,857

Accrued Expenses

12 Months Ended

Dec. 31, 2025

Payables and Accruals [Abstract]

Accrued Expenses

Accrued expenses consisted of the following (in thousands):


	December 31,
	2025		2024
Accrued personnel-related expenses	$	18,713	$	9,365
Accrued external research and development expenses	14,291		13,363
Accrued other expenses	2,029		1,035
Total accrued expenses	$	35,033	$	23,763

Audit Information	12 Months Ended
Audit Information	Dec. 31, 2025
Audit Information [Abstract]
Auditor Firm ID	42
Auditor Name	Ernst & Young LLP
Auditor Location	Boston, Massachusetts

Income Taxes - Schedule of Effective Income Tax Rate Reconciliation For Prior Periods (Details)	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Income Tax Disclosure [Abstract]
Federal statutory income tax rate	21.00%	21.00%	21.00%
Federal and state research and development credits		3.90%	3.50%
State taxes, net of federal benefit	(0.10%)	3.50%	4.30%
Changes in tax rates		(3.20%)	2.90%
Non-deductible executive compensation	(1.80%)	(2.60%)	(1.40%)
Non-taxable or non-deductible items	0.00%	(0.80%)	0.00%
Stock-based compensation	1.10%	(0.90%)	(1.30%)
Change in valuation allowance	(24.50%)	(20.70%)	(29.70%)
Other	0.00%	(0.20%)	0.70%
Effective income tax rate	(0.20%)	0.00%	0.00%

Property and Equipment, Net - Schedule of Property, Plant and Equipment (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Property, Plant and Equipment [Line Items]
Property and equipment, gross	$ 1,649	$ 1,593
Less: Accumulated depreciation	(1,502)	(1,363)
Property and equipment, net	147	230
Computer equipment
Property, Plant and Equipment [Line Items]
Property and equipment, gross	689	689
Office furniture and equipment
Property, Plant and Equipment [Line Items]
Property and equipment, gross	545	545
Laboratory equipment
Property, Plant and Equipment [Line Items]
Property and equipment, gross	$ 415	$ 359

Commitments and Contingencies - Future Lease Payments (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Commitments and Contingencies Disclosure [Abstract]
2026	$ 110
2027	0
Total future lease payments	110
Less: interest	0
Total lease liabilities	$ 110	$ 1,369

Segment Information - Schedule of Segment Reporting Information, by Segment (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Segment Reporting Information [Line Items]
Collaboration revenue	$ 0	$ 8,553	$ 2,447
Stock-based compensation expense	33,941	41,360	24,857
Depreciation expense	140	358	432
Net loss	(303,268)	(182,819)	(123,277)
Tenacia
Segment Reporting Information [Line Items]
Contract with customer, liability, cumulative catch-up adjustment to revenue, modification of contract	2,500	2,500
Reportable Segment
Segment Reporting Information [Line Items]
Collaboration revenue	0	8,553	2,447
Personnel-related expenses	64,719	42,853	33,160
Stock-based compensation expense	33,941	41,361	24,857
Depreciation expense	140	358	432
Other segment expenses	29,329	23,572	18,986
Other income, net	22,930	17,346	3,096
Net loss	(303,268)	(182,819)	(123,277)
Reportable Segment \| Ulixacaltamide
Segment Reporting Information [Line Items]
Program-specific expenses	63,207	69,172	19,464
Reportable Segment \| Vormatrigine
Segment Reporting Information [Line Items]
Program-specific expenses	74,059	18,360	6,275
Reportable Segment \| Relutrigine
Segment Reporting Information [Line Items]
Program-specific expenses	45,951	6,273	3,996
Reportable Segment \| Elsunersen
Segment Reporting Information [Line Items]
Program-specific expenses	6,833	2,135	14,595
Reportable Segment \| Other Early State Assets
Segment Reporting Information [Line Items]
Program-specific expenses	$ 8,019	$ 4,634	$ 7,055

Cash Equivalents and Marketable Securities - Investment Profile (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Debt Securities, Available-for-sale [Line Items]
Cash equivalents, fair value disclosure	$ 195,296	$ 128,652
Long-term marketable securities	568,759	254,156
Cash equivalents, at carrying value and debt securities, available-for-sale, amortized cost	770,494	398,109
Cash equivalents and debt securities, available-for-sale, accumulated gross unrealized gain, current, before tax	614	654
Cash equivalents and debt securities, available-for-sale, accumulated gross unrealized loss, current, before tax	(51)	0
Cash equivalents and debt securities, available-for-sale, fair value disclosure	771,057	398,763
Money market funds
Debt Securities, Available-for-sale [Line Items]
Cash equivalents, at carrying value	195,296	128,652
Cash equivalents, accumulated gross unrealized gain, current, before tax	0	0
Cash equivalents, accumulated gross unrealized loss, current, before tax	0	0
Cash equivalents, fair value disclosure	195,296	128,652
Debt securities issued by U.S. government agencies
Debt Securities, Available-for-sale [Line Items]
Debt securities, available-for-sale, amortized cost, current	7,003	15,953
Debt securities, available-for-sale, accumulated gross unrealized gain, current, before tax	0	2
Debt securities, available-for-sale, accumulated gross unrealized loss, current, before tax	(1)	0
Debt securities, available-for-sale, current	7,002	15,955
Debt securities, available-for-sale, amortized cost, noncurrent	183,739	128,215
Debt securities, available-for-sale, accumulated gross unrealized gain, noncurrent, before tax	258	475
Debt securities, available-for-sale, accumulated gross unrealized loss, noncurrent, before tax	0	0
Long-term marketable securities	183,997	128,690
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Debt securities, available-for-sale, amortized cost, noncurrent	198,036	107,695
Debt securities, available-for-sale, accumulated gross unrealized gain, noncurrent, before tax	282	157
Debt securities, available-for-sale, accumulated gross unrealized loss, noncurrent, before tax	0	0
Long-term marketable securities	198,318	107,852
Asset backed securities
Debt Securities, Available-for-sale [Line Items]
Debt securities, available-for-sale, amortized cost, noncurrent	61,282
Debt securities, available-for-sale, accumulated gross unrealized gain, noncurrent, before tax	70
Debt securities, available-for-sale, accumulated gross unrealized loss, noncurrent, before tax	0
Long-term marketable securities	61,352
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Debt securities, available-for-sale, amortized cost, noncurrent	7,714	8,775
Debt securities, available-for-sale, accumulated gross unrealized gain, noncurrent, before tax	4	19
Debt securities, available-for-sale, accumulated gross unrealized loss, noncurrent, before tax	0	0
Long-term marketable securities	7,718	8,794
Other debt securities
Debt Securities, Available-for-sale [Line Items]
Debt securities, available-for-sale, amortized cost, noncurrent	117,424	8,819
Debt securities, available-for-sale, accumulated gross unrealized gain, noncurrent, before tax	0	1
Debt securities, available-for-sale, accumulated gross unrealized loss, noncurrent, before tax	(50)	0
Long-term marketable securities	$ 117,374	$ 8,820

Cash Equivalents and Marketable Securities

12 Months Ended

Dec. 31, 2025

Cash and Cash Equivalents [Abstract]

Cash Equivalents and Marketable Securities

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


	As of December 31, 2025
			Gross Unrealized				Estimated
	Cost		Gains		Losses		Fair Value
Cash equivalents:
Money market funds	$	195,296	$	—	$	—	$	195,296
Debt securities issued by U.S. government agencies	7,003		—		(1)		7,002
Available-for-sale securities:
Corporate debt securities	198,036		282		—		198,318
Debt securities issued by U.S. government agencies	183,739		258		—		183,997
Asset backed securities	61,282		70		—		61,352
Commercial paper	7,714		4		—		7,718
Other debt securities	117,424		—		(50)		117,374
Total cash equivalents and marketable securities	$	770,494	$	614	$	(51)	$	771,057


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

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


	December 31, 2025		December 31, 2024
Within one year	$	242,002	$	177,195
After one year through five years	326,757		76,961
Total	$	568,759	$	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 periods presented.

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Cash flows from operating activities:
Net loss	$ (303,268)	$ (182,819)	$ (123,277)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation expense	140	358	432
Stock-based compensation expense	33,941	41,360	24,857
Non-cash operating lease expense	1,040	933	837
Amortization of premiums and discounts on marketable securities, net	(3,592)	(5,010)	47
Non-cash collaboration and license agreement expense	0	2,500	0
Other non-cash adjustments	(7)	0	0
Changes in operating assets and liabilities:
Prepaid expenses and other current assets	640	(8,225)	6,772
Accounts payable	12,097	6,713	(8,855)
Accrued expenses	11,200	16,110	(8,497)
Operating lease liabilities	(1,259)	(1,126)	(1,005)
Deferred revenue	0	(2,553)	(2,447)
Other	0	2	0
Net cash used in operating activities	(249,068)	(131,757)	(111,136)
Cash flows from investing activities:
Purchases of property and equipment	(56)	0	(50)
Purchases of marketable securities	(623,812)	(371,519)	0
Maturities of marketable securities	312,720	123,025	39,000
Net cash (used in) provided by investing activities	(311,148)	(248,494)	38,950
Cash flows from financing activities:
Payments of tax withholdings related to vesting of restricted stock units	(2,089)	(308)	(137)
Proceeds from exercise of options and employee stock purchase plan purchases	13,274	2,633	416
Net cash provided by financing activities	702,173	514,323	91,871
Increase in cash, cash equivalents and restricted cash	141,957	134,072	19,685
Cash, cash equivalents and restricted cash, beginning of period	215,788	81,716	62,031
Cash, cash equivalents and restricted cash, end of period	357,745	215,788	81,716
Reconciliation of cash, cash equivalents and restricted cash:
Cash and cash equivalents	357,329	215,372	81,300
Restricted cash	416	416	416
Total cash, cash equivalents and restricted cash	357,745	215,788	81,716
Follow-On Offering
Cash flows from financing activities:
Proceeds from stock offerings, net of issuance costs	567,115	377,636	63,439
Supplemental disclosures of non-cash activities:
Offering costs from in accounts payable / accrued expenses	0	0	184
At-The-Market Offering
Cash flows from financing activities:
Proceeds from stock offerings, net of issuance costs	123,873	119,597	28,153
Supplemental disclosures of non-cash activities:
Offering costs from in accounts payable / accrued expenses	70	238	63
License And Collaboration Agreement
Cash flows from financing activities:
Proceeds from stock offerings, net of issuance costs	$ 0	$ 14,765	$ 0

Segment Reporting - Narrative (Details)	12 Months Ended
Segment Reporting - Narrative (Details)	Dec. 31, 2025 segment
Segment Reporting [Abstract]
Number of operating segments	1
Number of reportable segments	1

Net Loss per Share

12 Months Ended

Dec. 31, 2025

Earnings Per Share [Abstract]

Net Loss per Share


	Year Ended December 31,
	2025	2024	2023
Outstanding stock options	1,876,778	1,809,343	666,163
Unvested restricted stock units	501,853	224,473	47,145
Potential shares issuable under the ESPP	4,955	7,315	24,972
	2,383,586	2,041,131	738,280

Common shares issuable upon exercise of the pre-funded warrants that were sold in connection with the January 2024, April 2024, and October 2025 underwritten public offerings are included in the calculation of weighted average number of common shares outstanding for the years ended December 31, 2025 and 2024. Consistent with the guidance in ASC 260-10-45-13, the underlying common shares are issuable for little to no consideration and there are no vesting conditions or contingencies associated with the warrants. Accordingly, the aggregate number of common shares underlying the pre-funded warrants have been considered outstanding for purposes of the calculation of net loss per share from the date of issuance.

Summary of Significant Accounting Policies - Summary of Cash, Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Accounting Policies [Abstract]
Cash and cash equivalents	$ 357,329	$ 215,372	$ 81,300
Restricted cash	416	416	416
Total cash, cash equivalents and restricted cash	$ 357,745	$ 215,788	$ 81,716	$ 62,031

Cash Equivalents and Marketable Securities - Contractual Maturity of Marketable Securities (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Cash and Cash Equivalents [Abstract]
Within one year	$ 242,002	$ 177,195
After one year through five years	326,757	76,961
Long-term marketable securities	$ 568,759	$ 254,156

Income Taxes - Net Deferred Tax Assets (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Deferred tax assets:
Net operating loss carryforwards	$ 170,157	$ 90,108
Capitalized research expenditures	59,250	65,836
Tax credits	44,210	30,582
Amortization	19,035	20,600
Stock-based compensation	9,508	8,784
Accrued expenses	4,441	2,333
Leases	28	344
Total gross deferred tax assets	306,629	218,587
Less: Valuation allowance	(306,606)	(218,303)
Net deferred tax assets	23	284
Deferred tax liabilities:
Operating lease right-of-use asset	(23)	(284)
Total gross deferred tax liabilities	(23)	(284)
Net deferred tax assets	$ 0	$ 0

Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands	Total	Follow-On Offering	At-The-Market Offering	License And Collaboration Agreement	Common Stock	Common Stock Follow-On Offering	Common Stock At-The-Market Offering	Common Stock License And Collaboration Agreement	Additional Paid-In Capital	Additional Paid-In Capital Follow-On Offering	Additional Paid-In Capital At-The-Market Offering	Additional Paid-In Capital License And Collaboration Agreement	Accumulated Deficit	Accumulated Other Comprehensive Gain (Loss)
Beginning balance (in shares) at Dec. 31, 2022					3,292,163
Beginning balance at Dec. 31, 2022	$ 76,106				$ 5				$ 606,918				$ (530,644)	$ (173)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	24,857								24,857
Issuance of common stock (in shares)						4,296,646	1,165,247
Issuance of common stock		$ 63,439	$ 28,092			$ 6	$ 2			$ 63,433	$ 28,090
Issuance of common stock under employee stock purchase plan (in shares)					24,690
Issuance of common stock under employee stock purchase plan	314								314
Vesting of restricted stock units (in shares)					13,293
Shares withheld for taxes for vesting of restricted stock units (in shares)					(3,443)
Shares withheld for taxes for vesting of restricted stock units	(137)								(137)
Issuance of common stock upon exercise of stock options (in shares)					3,281
Issuance of common stock upon exercise of stock options	102								102
Change in unrealized gain (loss) on marketable securities, net of tax	173													173
Net loss	(123,277)												(123,277)
Ending balance (in shares) at Dec. 31, 2023					8,791,877
Ending balance at Dec. 31, 2023	69,669				$ 13				723,577				(653,921)	0
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	41,360								41,360
Issuance of common stock (in shares)						7,651,583	1,823,652	443,253
Issuance of common stock		377,637	119,359	$ 17,265		$ 1				377,636	119,359	$ 17,265
Issuance of common stock under employee stock purchase plan (in shares)					31,381
Issuance of common stock under employee stock purchase plan	544								544
Issuance of common stock from exercise of pre-funded warrants (in shares)					622,123
Vesting of restricted stock units (in shares)					19,475
Shares withheld for taxes for vesting of restricted stock units (in shares)					(6,163)
Shares withheld for taxes for vesting of restricted stock units	(308)								(308)
Issuance of common stock upon exercise of stock options (in shares)					45,177
Issuance of common stock upon exercise of stock options	2,089								2,089
Change in unrealized gain (loss) on marketable securities, net of tax	654													654
Net loss	$ (182,819)												(182,819)
Ending balance (in shares) at Dec. 31, 2024	19,422,358				19,422,358
Ending balance at Dec. 31, 2024	$ 445,450				$ 14				1,281,522				(836,740)	654
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Stock-based compensation expense	33,941								33,941
Issuance of common stock (in shares)						3,527,072	1,755,636
Issuance of common stock		$ 567,116	$ 123,803			$ 1				$ 567,115	$ 123,803
Issuance of common stock under employee stock purchase plan (in shares)					22,691
Issuance of common stock under employee stock purchase plan	717								717
Issuance of common stock from exercise of pre-funded warrants (in shares)					200,349
Vesting of restricted stock units (in shares)					78,281
Shares withheld for taxes for vesting of restricted stock units (in shares)					(24,333)
Shares withheld for taxes for vesting of restricted stock units	$ (2,089)								(2,089)
Issuance of common stock upon exercise of stock options (in shares)	220,261				213,038
Issuance of common stock upon exercise of stock options	$ 12,557								12,557
Change in unrealized gain (loss) on marketable securities, net of tax	(91)													(91)
Net loss	$ (303,268)												(303,268)
Ending balance (in shares) at Dec. 31, 2025	25,195,092				25,195,092
Ending balance at Dec. 31, 2025	$ 878,136				$ 15				$ 2,017,566				$ (1,140,008)	$ 563

Summary of Significant Accounting Policies (Tables)

12 Months Ended

Dec. 31, 2025

Accounting Policies [Abstract]

Summary of Cash, Cash Equivalents and Restricted Cash


	December 31,
	2025		2024
Cash and cash equivalents	$	357,329	$	215,372
Restricted cash	416		416
Total cash, cash equivalents and restricted cash as shown on the consolidated statements of cash flows	$	357,745	$	215,788

Schedule of Restricted Cash and Cash Equivalents


	December 31,
	2025		2024
Cash and cash equivalents	$	357,329	$	215,372
Restricted cash	416		416
Total cash, cash equivalents and restricted cash as shown on the consolidated statements of cash flows	$	357,745	$	215,788

Schedule of Property, Plant and Equipment

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


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

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


	December 31,
	2025		2024
Computer equipment	$	689	$	689
Office furniture and equipment	545		545
Laboratory equipment	415		359
Total property and equipment	1,649		1,593
Less: Accumulated depreciation	(1,502)		(1,363)
Property and equipment, net	$	147	$	230

Stock-Based Compensation - Narrative (Details) - USD ($) $ in Thousands	1 Months Ended	12 Months Ended
	Jan. 31, 2025	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total fair value of restricted stock units vested		$ 8,700	$ 1,000
Aggregate intrinsic value exercised		27,608	$ 1,200
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		$ 23,700
Weighted-average period (in years)		2 years 11 months 19 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 1 month 6 days
Unrecognized compensation expense related to unvested stock based awards		$ 30,800
Shares reserved for future awards under the 2024 Inducement Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Share-based payment award, increase (decrease) 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
Award expiration period		10 years
2017 Stock Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of shares authorized (in shares)		395,850	395,850
Award expiration period		10 years
2024 Employee Stock Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Award expiration period		10 years
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
Issuance of common stock under employee stock purchase plan (in shares)		22,691	31,381	24,690

Collaboration and License Agreements (Details)		1 Months Ended		12 Months Ended
Collaboration and License Agreements (Details)	Jan. 04, 2024 USD ($) obligation $ / shares shares	Dec. 31, 2024 USD ($)	Jan. 31, 2024 USD ($)	Dec. 31, 2025 USD ($) $ / shares	Dec. 31, 2024 USD ($)	Dec. 31, 2023 USD ($)	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			$ 5,000,000.0
Milestone payments							98,500,000
Transaction price							$ 5,000,000.0
Contract with customer, liability, revenue recognized				$ 0	$ 8,600,000	$ 2,400,000
UCB \| Upfront Payment
Disaggregation of Revenue [Line Items]
Contract with customer, liability, revenue recognized		$ 2,600,000
UCB \| Option Exercise Fee
Disaggregation of Revenue [Line Items]
Contract with customer, liability, revenue recognized		$ 6,000,000.0
Tenacia
Disaggregation of Revenue [Line Items]
Number of shares issued in transaction (in shares) \| shares	443,253
Sale of stock, price (in dollars per share) \| $ / shares	$ 22.5605			$ 38.95
Proceeds from stock offerings, net of issuance costs	$ 10,000,000.0		$ 10,000,000.0
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
Contract with customer, liability, cumulative catch-up adjustment to revenue, modification of contract				$ 2,500,000	$ 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

Significant Agreements	12 Months Ended
Significant Agreements	Dec. 31, 2025
Commitments and Contingencies Disclosure [Abstract]
Significant Agreements	Significant Agreements RogCon and Ionis Agreements On September 11, 2019, the Company entered into both a Cooperation and License Agreement (the “License Agreement”) with RogCon, Inc. ("RogCon"), and a Research, Collaboration, Option and License Agreement (the “Ionis Collaboration Agreement”) with Ionis Pharmaceuticals, Inc. ("Ionis"). The agreements were entered into contemporaneously to enable the parties to advance their collective efforts related to SCN2A. RogCon Agreement Under the License Agreement, RogCon granted to the Company an exclusive, worldwide license under RogCon’s intellectual property to research, develop and commercialize products for the treatment of all forms of epilepsy and/or neurodevelopmental disorders in each case caused by any mutation of the SCN2A gene. Under the License Agreement, the Company will conduct, at its own cost and expense, the research and development activities assigned to it under the research plan. In addition, the Company is responsible for reimbursing RogCon for any costs associated with research and development activities RogCon performs at the request of the Company. As part of the agreement, the Company agreed to provide up-front consideration of $2.1 million. Subsequent to September 11, 2019, the Company will reimburse RogCon for its out-of-pocket costs incurred for activities performed under the License Agreement. The Company expenses these costs as incurred as research and development. Expenses incurred for all periods presented were not material. Additionally, the Company may pay RogCon a milestone payment of $3.0 million and profit share payments. The $3.0 million milestone payment will become due when the first profit share payment has become due and certain contingent payments have become payable to Ionis under the Ionis Collaboration Agreement, which are subject to the Company exercising its option to obtain license rights to a development candidate, as well as other contingent events. The profit share payments will be based on a low-double-digit percentage of net profits, depending on sales volume. The License Agreement, unless earlier terminated, will continue until the latest of: (i) expiration of all patent rights within RogCon patents, (ii) the Company and its affiliates certify they have abandoned the research, development and commercialization of product with no intention to re-establish such activities, and (iii) no third party is obligated to pay the Company or its affiliates any amounts that comprise net sublicense revenue. Either party may terminate the License Agreement for material breach or insolvency of the other party. Additionally, the Company may terminate for convenience with prior written notice to RogCon. Upon termination by either party, all rights and licenses granted by RogCon to the Company will revert back to RogCon. Ionis Collaboration Agreement Under the Ionis Collaboration Agreement, both parties participated in research activities related to the downregulation of SCN2A gene products associated with the treatment of any and all forms of epilepsy and/or neurodevelopmental disorders in each case caused by any mutation of the SCN2A gene, other than one severe type of epilepsy. Ionis was also responsible for identifying a development candidate and conducting an IND-enabling toxicology study. The Company reimbursed Ionis for out-of-pocket costs incurred related to the research activities, identification of a development candidate and conducting an IND-enabling toxicology study. The reimbursement of out-of-pocket costs was recognized as research and development expense as incurred. No expenses were incurred during the year ended December 31, 2025. Expense for the years ended December 31, 2024 and 2023 was immaterial. Ionis granted the Company an exclusive option to obtain the rights and license to further develop and commercialize a development candidate in the field of epilepsy and neurodevelopmental disorders, other than Dravet Syndrome, following the results of the investigational new drug application-enabling toxicology study. The Company exercised this exclusive option in January 2022, paid a $2.0 million license fee and recognized the license fee in research and development expenses in the consolidated statement of operations during the year ended December 31, 2022. After option exercise, the Company is responsible for clinical development and commercialization of the development candidate. Additionally, in July 2023, the Company paid a milestone payment of $6.9 million to Ionis, which was earned in the second quarter of 2023 upon initiation of the Company's elsunersen EMBRAVE study, and was recognized in research and development expenses in the consolidated statement of operations during the year ended December 31, 2023. Ionis may be entitled to additional development milestone payments, additional milestone payments, and sales royalties or sublicense fees. The Ionis Collaboration Agreement will continue until the expiration of all payment obligations to Ionis, unless earlier terminated. Either party may terminate the Ionis Collaboration Agreement upon material breach or insolvency of the other party. Ionis may terminate if the Company fails to achieve a performance milestone. The Company may terminate for convenience with prior written notice to Ionis. Upon termination by the Company for convenience, the Company will stop selling all products, subject to certain wind-down provisions, and all products will revert back to Ionis.

Commitments and Contingencies - Weighted Average Remaining Lease Term and Weighted Average Incremental Borrowing Rate (Details)	Dec. 31, 2025	Dec. 31, 2024
Commitments and Contingencies Disclosure [Abstract]
Weighted average remaining lease term (in years)	1 month 6 days	1 year 1 month 6 days
Weighted average incremental borrowing rate	9.00%	9.00%

Stock-Based Compensation (Tables)

12 Months Ended

Dec. 31, 2025

Share-Based Payment Arrangement [Abstract]

Summary of Restricted Stock Activity

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


	Shares	Weighted Average Grant Date Fair Value
Unvested as of December 31, 2024	224,473	$	71.71
Issued	414,151	67.60
Vested	(86,005)	105.57
Forfeited	(50,766)	55.89
Unvested as of December 31, 2025	501,853	$	64.12

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	8.47	$	39,989
Granted	324,670	60.03
Exercised	(220,261)	57.01			$	27,608
Cancelled or Forfeited	(36,974)	74.65
Outstanding as of December 31, 2025	1,876,778	$	89.80	7.72	$	409,936
Exercisable as of December 31, 2025	1,101,369	$	112.82	7.11	$	225,671
Vested and expected to vest as of December 31, 2025	1,876,778	$	89.80	7.72	$	409,936

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


	Year Ended December 31,
	2025			2024			2023
Risk-free interest rate	4.45		%	4.01		%	3.60		%
Expected term (in years)	6.00			6.03			6.00
Expected volatility	93.08		%	96.60		%	88.40		%
Expected dividend yield	—		%	—		%	—		%
Weighted average grant-date fair value per share	$	46.74		$	41.25		$	29.73

Summary of Stock-Based Compensation Expense

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


	Year Ended December 31,
	2025		2024		2023
Research and development	$	15,286	$	15,620	$	8,055
General and administrative	18,655		25,740		16,802
Total stock-based compensation expense	$	33,941	$	41,360	$	24,857

Stock-Based Compensation - Summary of Restricted Stock Activity (Details) - Unvested restricted stock units	12 Months Ended
	Dec. 31, 2025 $ / shares shares
Shares
Unvested beginning balance (in shares) \| shares	224,473
Issued (in shares) \| shares	414,151
Vested (in shares) \| shares	(86,005)
Forfeited (in shares) \| shares	(50,766)
Unvested ending balance (in shares) \| shares	501,853
Weighted Average Grant Date Fair Value
Unvested beginning balance (in dollars per share) \| $ / shares	$ 71.71
Issued (in dollars per share) \| $ / shares	67.60
Vested (in dollars per share) \| $ / shares	105.57
Forfeited (in dollars per share) \| $ / shares	55.89
Unvested ending balance (in dollars per share) \| $ / shares	$ 64.12

Net Loss per Share - Summary of Antidilutive Securities Excluded From Computation of Earnings Per Share (Details) - shares	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities (in shares)	2,383,586	2,041,131	738,280
Outstanding stock options
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities (in shares)	1,876,778	1,809,343	666,163
Unvested restricted stock units
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities (in shares)	501,853	224,473	47,145
Potential shares issuable under the ESPP
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities (in shares)	4,955	7,315	24,972

Commitment and Contingencies (Tables)

12 Months Ended

Dec. 31, 2025

Commitments and Contingencies Disclosure [Abstract]

Schedule of Operating Lease, Consolidated Balance Sheets

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


	As of December 31,
	2025		2024
Assets:
Operating lease right-of-use assets	$	92	$	1,131
Liabilities:
Current operating lease liabilities	$	110	$	1,259
Non-current portion of operating lease liabilities	—		110
Total lease liabilities	$	110	$	1,369

Schedule of Lease cost

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


	For the year ended December 31,
	2025		2024		2023
Operating lease cost	$	1,102	$	1,102	$	1,102
Variable lease costs	51		33		14
Total lease costs	$	1,153	$	1,135	$	1,116

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


	As of December 31,
	2025		2024
Weighted average remaining lease term (in years)	0.1		1.1
Weighted average incremental borrowing rate	9.0	%	9.0	%

Schedule of Lessee, Operating Lease, Liability, Maturity

Future lease payments under non-cancelable lease agreements as of December 31, 2025 were as follows (in thousands):


Year Ended December 31,	Future Lease Payments
2026	$	110
2027	—
Total future lease payments	110
Less: interest	—
Present value of operating lease liabilities	$	110

Income Taxes - Schedule of Effective Income Tax Rate Reconciliation For Current Year (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Income Tax Expense (Benefit), Effective Income Tax Rate Reconciliation, Amount [Abstract]
Federal statutory income tax rate	$ (63,587)
State and local income tax, net of federal income tax effect	417
Non-taxable or non-deductible items	7
Stock-based compensation	(3,337)
Limitation on executive compensation	5,508
Tax credits	(12,853)
Change in valuation allowance	74,260
Other items	65
Effective income tax rate	$ 471
Effective Income Tax Rate Reconciliation, Percent [Abstract]
Federal statutory income tax rate	21.00%	21.00%	21.00%
State taxes, net of federal benefit	(0.10%)	3.50%	4.30%
Non-taxable or non-deductible items	0.00%	(0.80%)	0.00%
Stock-based compensation	1.10%	(0.90%)	(1.30%)
Non-deductible executive compensation	(1.80%)	(2.60%)	(1.40%)
Tax credits	4.20%
Change in valuation allowance	(24.50%)	(20.70%)	(29.70%)
Other	0.00%	(0.20%)	0.70%
Effective income tax rate	(0.20%)	0.00%	0.00%
Other foreign jurisdictions
Income Tax Expense (Benefit), Effective Income Tax Rate Reconciliation, Amount [Abstract]
Other foreign jurisdictions	$ (9)
Effective Income Tax Rate Reconciliation, Percent [Abstract]
Other foreign jurisdictions	0.00%

Employee Benefit Plan (Details) - USD ($) $ in Millions	12 Months Ended
Employee Benefit Plan (Details) - USD ($) $ in Millions	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Retirement Benefits [Abstract]
Defined contribution plan, employer discretionary contribution amount	$ 1.6	$ 1.2	$ 1.0

Commitments and Contingencies - Narrative (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024	May 31, 2021
Loss Contingencies [Line Items]
Operating lease right-of-use assets	$ 92	$ 1,131
Present value of operating lease liabilities	$ 110	$ 1,369
Office Space In Boston, Massachusetts
Loss Contingencies [Line Items]
Percentage increase in annual base rent			2.00%
Operating lease right-of-use assets			$ 4,100
Present value of operating lease liabilities			$ 4,100

- Definition

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

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 275
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482861/275-10-50-9

Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 450
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 4
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483076/450-20-50-4

Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 450
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483076/450-20-50-9

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 720
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483359/720-20-50-1

Reference 5: http://www.xbrl.org/2003/role/exampleRef
-Topic 460
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 27
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482395/460-10-55-27

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 450
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483076/450-20-50-9

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 450
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 4
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483076/450-20-50-4

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 460
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482425/460-10-50-3

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 450
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483076/450-20-50-1

+ Details

Property and Equipment, Net (Tables)

12 Months Ended

Dec. 31, 2025

Property, Plant and Equipment [Abstract]

Schedule of Property, Plant and Equipment

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


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

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


	December 31,
	2025		2024
Computer equipment	$	689	$	689
Office furniture and equipment	545		545
Laboratory equipment	415		359
Total property and equipment	1,649		1,593
Less: Accumulated depreciation	(1,502)		(1,363)
Property and equipment, net	$	147	$	230

Summary of Significant Accounting Policies - Property and Equipment Estimated Useful Life (Details)	Dec. 31, 2025
Office furniture and equipment
Property, Plant and Equipment [Line Items]
Estimated Useful Life	5 years
Laboratory equipment
Property, Plant and Equipment [Line Items]
Estimated Useful Life	3 years
Computer equipment
Property, Plant and Equipment [Line Items]
Estimated Useful Life	3 years