What changed in this filing

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Item 1. Financial Statements

2026-05-07 compared with 2025-11-05 · 166 added, 187 removed, 358 unchanged (50% of the section changed)

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(Amounts in thousands, except share and per share data)

September 30, 2025 December 31, 2024

March 31, 2026 December 31, 2025

Current assets:

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Operating lease right-of-use assets 1,320 92

Other non-current assets — 416

Total assets $ 1,460,202 $ 937,907

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Total current liabilities 50,241 59,771

Long-term liabilities:

Non-current portion of operating lease liabilities — 110

Total liabilities 50,241 59,771

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Preferred stock, $ 0.0001 par value;

10,000,000 shares authorized and no shares issued or outstanding as of September 30, 2025 and December 31, 2024

10,000,000 shares authorized and no shares issued or outstanding as of March 31, 2026 and December 31, 2025

Common stock, $ 0.0001 par value;

150,000,000 shares authorized;

21,165,933 shares issued and outstanding as of September 30, 2025, and 19,422,358 shares issued and outstanding as of December 31, 2024

27,859,514 shares issued and outstanding as of March 31, 2026, and 25,195,092 shares issued and outstanding as of December 31, 2025

Additional paid-in capital 2,644,109 2,017,566

Accumulated other comprehensive gain 500 654

Accumulated deficit ( 1,232,569 ) ( 1,140,008 )

Accumulated other comprehensive (loss) gain ( 1,594 ) 563

Total stockholders’ equity 1,409,961 878,136

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Three Months Ended

September 30, Nine Months Ended

September 30,

2025 2024 2025 2024

Collaboration revenue $ — $ 302 $ — $ 1,090

Operating expenses:

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Three Months Ended

September 30, Nine Months Ended

September 30,

2025 2024 2025 2024

Net loss $ ( 92,561 ) $ ( 69,296 )

Change in unrealized gain (loss) on marketable securities, net of tax 105 1,402 ( 154 ) 1,331

Change in unrealized (loss) gain on marketable securities, net of tax ( 2,157 ) 5

Comprehensive loss $ ( 94,718 ) $ ( 69,291 )

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Deficit Accumulated Other

Comprehensive Gain Total

Comprehensive Gain (Loss) Total

Stockholders’

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Stock-based compensation expense — — 17,112 — — 17,112

Issuance of common stock from at-the-market public offerings, net of commission costs of $ 1,120

Issuance of common stock from follow-on public offering, net of underwriting discounts, commissions and offering costs of $ 40,153

2,543,800 — 621,235 — — 621,235

Issuance of common stock from exercise of pre-funded warrants 173,840 — — — — —

Vesting of restricted stock units 134,225 — — — — —

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Balance at March 31, 2026 27,859,514 $ 15 $ 2,644,109 $ ( 1,232,569 ) $ ( 1,594 ) $ 1,409,961

Stock-based compensation expense — — 7,770 — — 7,770

Issuance of common stock from at-the-market offerings, net of commission costs of $ 579

657,477 — 28,360 — — 28,360

Issuance of common stock from exercise of pre-funded warrants 26,509 — — — — —

Issuance of common stock under employee stock purchase plan 9,319 — 294 — — 294

Vesting of restricted stock units 5,860 — — — — —

Shares withheld for taxes for vesting of restricted stock units ( 608 ) — ( 23 ) — — ( 23 )

Change in unrealized gain on marketable securities, net of tax — — — — ( 264 ) ( 264 )

Net loss — — — ( 71,127 ) — ( 71,127 )

Balance at June 30, 2025 21,040,474 $ 14 $ 1,380,978 $ ( 977,163 ) $ 395 $ 404,224

Stock-based compensation expense — — 7,447 — — 7,447

Issuance of common stock from at-the-market offerings, net of issuance and commission costs of $ 359

102,364 — 4,907 — — 4,907

Vesting of restricted stock units 3,746 — — — — —

Shares withheld for taxes for vesting of restricted stock units ( 1,106 ) — ( 52 ) — — ( 52 )

Issuance of common stock upon exercise of stock options 20,455 — 808 — — 808

Change in unrealized gain on marketable securities, net of tax — — — — 105 105

Net loss — — — ( 73,934 ) — ( 73,934 )

Balance at September 30, 2025 21,165,933 $ 14 $ 1,394,088 $ ( 1,051,097 ) $ 500 $ 343,505

Common Stock Additional

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Stock-based compensation expense — — 8,786 — — 8,786

Issuance of common stock from follow-on public offering and accompanying pre-funded warrants, net of underwriting discounts, commissions and offering costs of $ 10,836

3,802,025 — 161,649 — — 161,649

Issuance of common stock from at-the-market public offerings, net of issuance and commission costs of $ 376

Issuance of common stock from at-the-market public offerings, net of commission costs of $ 1,120

694,212 — 54,904 — — 54,904

Issuance of common stock from a collaboration and license agreement 443,253 — 17,265 — — 17,265

Issuance of common stock from exercise of pre-funded warrants 173,840 — — — — —

Vesting of restricted stock units 55,850 — — — — —

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Balance at March 31, 2025 20,341,917 $ 14 $ 1,344,577 $ ( 906,036 ) $ 659 $ 439,214

Stock-based compensation expense — — 5,878 — — 5,878

Issuance of common stock from follow-on public offering and accompanying pre-funded warrants, net of underwriting discounts, commissions and offering costs of $ 14,013

3,849,558 1 215,987 — — 215,988

Issuance of common stock from exercise of pre-funded warrants 622,123 — — — — —

Issuance of common stock under employee stock purchase plan 25,146 — 311 — — 311

Vesting of restricted stock units 726 — — — — —

Shares withheld for taxes for vesting of restricted stock units ( 242 ) — ( 13 ) — — ( 13 )

Issuance of common stock upon exercise of stock options 148 — 4 — — 4

Change in unrealized loss on marketable securities, net of tax — — — — ( 74 ) ( 74 )

Net loss — — — ( 32,677 ) — ( 32,677 )

Balance at June 30, 2024 17,755,506 $ 14 $ 1,145,308 $ ( 726,151 ) $ ( 71 ) $ 419,100

Stock-based compensation expense — — 12,432 — — 12,432

Issuance of common stock from at-the-market public offerings, net of issuance and commission costs of $ 70

25,189 1,442 1,442

Issuance of common stock from exercise of stock options 4,971 201 201

Vesting of restricted stock units 51 — — — — —

Shares withheld for taxes for vesting of restricted stock units ( 20 ) — ( 1 ) — — ( 1 )

Change in unrealized loss on marketable securities, net of tax — — — — 1,402 1,402

Net loss — — — ( 51,910 ) — ( 51,910 )

Balance at September 30, 2024 17,785,697 $ 14 $ 1,159,382 $ ( 778,061 ) $ 1,331 $ 382,666

The accompanying notes are an integral part of these condensed consolidated financial statements.

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(Amounts in thousands)

Nine Months Ended

September 30,

Three Months Ended

Cash flows from operating activities:

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Amortization of premiums and discounts on marketable securities, net ( 655 ) ( 1,229 )

Non-cash collaboration and license agreement expense — 2,500

Changes in operating assets and liabilities:

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Operating lease liabilities ( 101 ) ( 303 )

Deferred revenue — ( 1,090 )

Other — ( 2 )

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Maturities of marketable securities 142,569 65,993

Net cash provided by (used in) investing activities 17,243 ( 237,576 )

Net cash used in investing activities ( 344,275 ) ( 51,064 )

Cash flows from financing activities:

Issuance of common stock from follow-on public offering and accompanying pre-funded warrants, net of underwriting discounts, commissions and offering costs — 377,636

Issuance of common stock from collaboration and license agreement — 14,765

Issuance of common stock from follow-on public offering, net of underwriting discounts, commissions and offering costs 621,235 —

Proceeds from at-the-market offerings, net of issuance and commission costs — 54,904

Payments of tax withholdings related to vesting of restricted stock units ( 12,923 ) ( 1,195 )

Proceeds from exercise of stock options and employee stock purchase plan purchases 1,662 659

Proceeds from exercise of stock options 1,119 560

Net cash provided by financing activities 609,431 54,269

(Decrease) increase in cash, cash equivalents and restricted cash ( 65,845 ) 87,345

Increase (decrease) in cash, cash equivalents and restricted cash 179,004 ( 49,805 )

Cash, cash equivalents and restricted cash, beginning of period 357,745 215,788

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Supplemental disclosures of non-cash activities:

Issuance costs from at-the-market offering included in accrued expenses $ 221 $ 36

Purchase of property and equipment included in accrued expenses $ — $ 64

Right-of-use assets obtained in exchange for lease obligations $ 1,425 $ —

Removal of right-of-use assets in connection with lease termination $ 4,086 $ —

The accompanying notes are an integral part of these condensed consolidated financial statements.

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Praxis Precision Medicines, Inc.

(“Praxis” or the “Company”) is a clinical-stage biopharmaceutical company translating insights from genetic epilepsies into the development of therapies for central nervous system ("CNS"), disorders characterized by neuronal excitation-inhibition imbalance.

(“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.

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• Cerebrum ™, the Company's small molecule platform, utilizes deep understanding of neuronal excitability and neuronal networks and applies a series of computational and experimental tools to develop orally available precision therapies

• Solidus ™, the Company's antisense oligonucleotide, or ASO, platform, is an efficient, targeted precision medicine discovery and development engine anchored on a proprietary, computational methodology

• 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.

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The Company has funded its operations primarily with proceeds from the issuance of redeemable convertible preferred stock, from the sale of common stock through an initial public offering, at-the-market offerings under its shelf registration statement, and follow-on public offerings of common stock and pre-funded warrants to purchase common stock.

From inception through September 30, 2025, the Company raised $ 1.2 billion in aggregate cash proceeds from these transactions, net of issuance costs.

The Company is subject to risks and uncertainties common to early-stage companies in the biotechnology industry, including but not limited to, risks associated with completing preclinical studies and clinical trials, receiving regulatory approvals for product candidates, development by competitors of new biopharmaceutical products, dependence on key personnel, protection of proprietary technology, compliance with government regulations and the ability to secure additional capital to fund operations.

Programs currently under development will require significant additional research and development efforts, including preclinical and clinical testing and regulatory approval, prior to commercialization.

These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities.

From inception through March 31, 2026, the Company raised $ 2.4 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.

In accordance with the Financial Accounting Standards Board (“FASB”) Accounting Standards Update (“ASU”) 2014-15, Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern (Subtopic 205-40) , the Company has evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date that these condensed consolidated financial statements are issued.

The Company has incurred recurring losses since its inception, including a net loss of $ 214.4 million for the nine months ended September 30, 2025.

In addition, as of September 30, 2025, the Company had an accumulated deficit of $ 1.1 billion.

The Company has incurred recurring losses since its inception, including a net loss of $ 92.6 million for the three months ended March 31, 2026.

In addition, as of March 31, 2026, the Company had an accumulated deficit of $ 1.2 billion.

The Company expects to continue to generate operating losses for the foreseeable future.

The Company expects that its cash, cash equivalents and marketable securities as of September 30, 2025 of $ 389.2 million, together with net proceeds of $ 567.0 million from the October 2025 financing, will be sufficient to fund its operating expenditures and capital expenditure requirements necessary to advance its program activities for at least one year from the date of issuance of these condensed consolidated financial statements.

The future viability of the Company is dependent on its ability to raise additional capital to finance its operations.

The Company expects that its cash, cash equivalents and marketable securities as of March 31, 2026 of $ 1.4 billion will be sufficient to fund its operating expenditures and capital expenditure requirements necessary to advance its research efforts and clinical trials for at least one year from the date of issuance of these condensed

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.

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Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification (“ASC”) and ASUs of the FASB.

The significant accounting policies used in preparation of these condensed consolidated financial statements for the three and nine months ended September 30, 2025 are consistent with those discussed in Note 2 to the consolidated financial statements included in the Company's 2024 Annual Report on Form 10-K, other than as noted below.

The significant accounting policies used in preparation of these condensed consolidated financial statements for the three months ended March 31, 2026 are consistent with those discussed in Note 2 to the consolidated financial statements included in the Company's 2025 Annual Report on Form 10-K, other than as noted below.

Unaudited Interim Condensed Consolidated Financial Information

The accompanying condensed consolidated balance sheet as of September 30, 2025, the condensed consolidated statements of operations for the three and nine months ended September 30, 2025 and 2024, the condensed consolidated statements of comprehensive loss for the three and nine months ended September 30, 2025 and 2024, the condensed consolidated statements of cash flows for the nine months ended September 30, 2025 and 2024 and the condensed consolidated statements of stockholders’ equity for the three and nine months ended September 30, 2025 and 2024 are unaudited.

The unaudited interim financial statements have been prepared on the same basis as the audited annual consolidated financial statements, and in the opinion of management reflect all adjustments, which include only normal recurring adjustments necessary for the fair statement of the Company’s financial position as of September 30, 2025, the results of its operations for the three and nine months ended September 30, 2025 and 2024 and its cash flows for the nine months ended September 30, 2025 and 2024.

Financial statement disclosures for the three and nine months ended September 30, 2025 and 2024 are condensed and do not include all disclosures required for an annual set of financial statements in accordance with GAAP.

The results for the three and nine months ended September 30, 2025 are not necessarily indicative of results to be expected for the year ended December 31, 2025, any other interim periods, or any future year or period.

The accompanying condensed consolidated balance sheet as of March 31, 2026, the condensed consolidated statements of operations for the three months ended March 31, 2026 and 2025, the condensed consolidated statements of comprehensive loss for the three months ended March 31, 2026 and 2025, the condensed consolidated statements of cash flows for the three months ended March 31, 2026 and 2025 and the condensed consolidated statements of stockholders’ equity for the three months ended March 31, 2026 and 2025 are unaudited.

The unaudited interim financial statements have been prepared on the same basis as the audited annual consolidated financial statements, and in the opinion of management reflect all adjustments, which include only normal recurring adjustments necessary for the fair statement of the Company’s financial position as of March 31, 2026, the results of its operations for the three months ended March 31, 2026 and 2025 and its cash flows for the three months ended March 31, 2026 and 2025.

Financial statement disclosures for the three months ended March 31, 2026 and 2025 are condensed and do not include all disclosures required for an annual set of financial statements in accordance with GAAP.

The results for the three months ended March 31, 2026 are not necessarily indicative of results to be expected for the year ended December 31, 2026, any other interim periods, or any future year or period.

Use of Estimates

The preparation of condensed 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 condensed consolidated financial statements and the reported amounts of expenses during the reporting periods.

Significant estimates and assumptions reflected in these condensed 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.

Common Stock Warrants

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The Company determines the classification for its warrants at the time of issuance and updates its assessment, as necessary.

Warrants that meet all of the criteria for equity classification are recorded as a component of additional paid-in capital.

Recently Enacted Tax Legislation

The One Big Beautiful Bill Act (“OBBBA”) was enacted in the U.S.

on July 4, 2025.

The OBBBA legislation provides for the permanent extension of certain expiring provisions of the Tax Cuts and Jobs Act, revisions to the international tax framework and the reinstatement of favorable tax treatment for certain business provisions.

The legislation has multiple effective dates, with certain provisions effective in 2025 and others implemented in future

While tax changes in the OBBBA do not have a material impact to the Company's effective tax rate, we anticipate that certain tax provisions in the OBBBA, namely the provision allowing for immediate expensing of certain research and development expenses, will result in accelerated deductions of previously capitalized expenses of approximately $ 203.9 million.

We continue to evaluate the tax and other provisions of the OBBBA and the potential effects on our financial position, results of operations, and cash flows.

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.

Performance Stock Units

For equity awards that do not vest unless the performance condition is met, the Company recognizes expense if, and to the extent that, it is determined that achievement of the performance conditions are probable.

If the Company concludes that vesting is probable, expense is recognized over the requisite service period.

Recent Accounting Pronouncements

Recently Issued Accounting Pronouncements

In December 2023, the FASB issued ASU No.

2023-09, Income Taxes (Topic 740) - Improvements to Income Tax Disclosures .

2023-09 provides more transparency about income tax information through improvements to income tax disclosures primarily related to the rate reconciliation and income taxes paid information.

For public companies, the amendments are effective for annual periods beginning after December 15, 2024 and can be applied prospectively or retrospectively.

The Company has determined that the effects of adopting the amendments in ASU 2023-09 will only impact its disclosures and will not have a material impact on its consolidated financial position and the results of its operations when such amendment is adopted.

In November 2024, the FASB issued ASU No.

2024-03, Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40):

Disaggregation of Income Statement Expenses , requiring public entities to disclose additional information about specific expense categories in the notes to the financial statements on an interim and annual basis.

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.

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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 using a prospective, retrospective, or modified transition approach.

The Company does not expect the adoption of ASU 2025-06 to have a material impact on its consolidated financial statements.

In December 2025, the FASB issued ASU No.

2025-11, Interim Reporting (Topic 270):

Narrow-Scope Improvements ("ASU 2025-11"), which clarifies the guidance in Topic 270 to improve the consistency of interim financial reporting.

The ASU provides a comprehensive list of required interim disclosures and introduces a disclosure principle requiring entities to disclose events since the end of the last annual reporting period that have material impact on the entity.

ASU 2025-11 is effective for fiscal years beginning after December 15, 2027, including interim periods within those fiscal years, with early adoption permitted.

The Company is currently evaluating the impact of adopting ASU 2025-11on its consolidated financial statements.

Cash Equivalents and Marketable Securities

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

As of September 30, 2025

The following is a summary of the Company's investment portfolio as of March 31, 2026 and December 31, 2025 (in thousands):

As of March 31, 2026

Gross Unrealized Estimated

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Corporate debt securities 311,749 — ( 643 ) 311,106

Asset backed securities 88,419 ( 31 ) 88,388

Commercial paper 6,552 — ( 15 ) 6,537

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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

Corporate debt securities 107,695 157 — 107,852

Asset backed securities 61,282 70 — 61,352

Commercial paper 7,714 4 — 7,718

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Total cash equivalents and marketable securities $ 770,494 $ 614 $ ( 51 ) $ 771,057

As of September 30, 2025, the Company had 4 securities with a total fair market value of $ 16.1 million in an unrealized loss position.

As of March 31, 2026, the Company had 178 securities with a total fair market value of $ 763.2 million in an unrealized loss position.

The Company believes that any unrealized losses associated with the decline in value of its securities is temporary and primarily related to the change in market interest rates since purchase, and believes that it is more likely than not that it will be able to hold its debt securities to maturity.

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

September 30, 2025 December 31, 2024

March 31, 2026 December 31, 2025

Within one year $ 250,532 $ 242,002

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Securities are evaluated for impairment at the end of each reporting period.

The Company did not record any impairment related to its available-for-sale securities during the three and nine months ended September 30, 2025 and 2024.

The Company did not record any impairment related to its available-for-sale securities during the three months ended March 31, 2026 and 2025.

Fair Value Measurements

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

The following table presents information about the Company’s financial assets measured at fair value on a recurring basis and indicates the level of the fair value hierarchy utilized to determine such fair values as of September 30, 2025 and December 31, 2024 (in thousands):

As of September 30, 2025

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 March 31, 2026 and December 31, 2025 (in thousands):

As of March 31, 2026

Level 1 Level 2 Level 3 Total

Money market funds $ 245,659 $ — $ — $ 245,659

Debt securities issued by US.

Debt securities issued by U.S.

government agencies 257,298 — — 257,298

Corporate debt securities — 311,106 — 311,106

Asset backed securities — 88,388 — 88,388

Commercial paper — 6,537 — 6,537

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Money market funds $ 195,296 $ — $ — $ 195,296

Debt securities issued by US.

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

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Accrued expenses consisted of the following (in thousands):

September 30, 2025 December 31, 2024

March 31, 2026 December 31, 2025

Accrued external research and development expenses $ 12,944 $ 14,291

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Commitments and Contingencies

In May 2021, the Company entered into a sublease agreement for office space located in Boston, Massachusetts that expires on January 31, 2026, with no option to renew or terminate early.

The base rent increases by approximately 2 % annually.

The Company issued a letter of credit to the landlord related to the security deposit, secured by restricted cash, which is reflected within prepaid expenses and other current assets and other non-current assets on the accompanying condensed consolidated balance sheets as of September 30, 2025 and December 31, 2024, respectively.

This lease qualifies as an operating lease.

In May 2021, the Company entered into a sublease agreement for office space located in Boston, Massachusetts that expired on January 31, 2026.

The Company issued a letter of credit to the landlord related to the security deposit, secured by restricted cash, which is reflected within prepaid expenses and other current assets on the accompanying condensed consolidated balance sheets as of March 31, 2026 and December 31, 2025.

In September 2025, the Company entered into a license agreement for its existing office space which commenced on

March 1, 2026 and expires on March 31, 2027, with no option to renew or terminate early.

At commencement, the Company recorded an operating lease right-of-use asset and operating lease liability of $ 1.4 million, which are reflected within the accompanying condensed consolidated balance sheet as of March 31, 2026.

This license agreement qualifies as an operating lease.

Collaboration and License Agreements

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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.

In December 2024, UCB exercised its option to in-license global development and commercialization rights for a KCNT1 development candidate as part of the Collaboration Agreement.

Upon notice of the exercise, the Company recognized the $ 6.0 million option exercise fee and the $ 2.6 million of remaining collaboration revenue associated with the $ 5.0 million up front payment as collaboration revenue within the December 31, 2024 consolidated statement of operations, as the Company had no further research service obligations under the terms of the Collaboration Agreement.

The Company did not recognize any collaboration revenue associated with this Collaboration Agreement during the three and nine months ended September 30, 2025.

As of September 30, 2025, there was no deferred revenue recorded in the condensed consolidated balance sheet.

During the three and nine months ended September 30, 2024, the Company recognized $ 0.3 million and $ 1.1 million, respectively, related to the Collaboration Agreement in the condensed consolidated statement of operations.

As of December 31, 2024, there was no deferred revenue recorded in the consolidated balance sheet.

Accordingly, the Company did not recognize any collaboration revenue associated with this Collaboration Agreement during the three months ended March 31, 2026 or March 31, 2025.

As of March 31, 2026, there was no deferred revenue recorded in the condensed consolidated balance sheet.

Tenacia Collaboration and License Agreement

On January 4, 2024, the Company entered into an exclusive collaboration and license Agreement (“the License Agreement”) with Tenacia Biotechnology (Shanghai) Co., Ltd.

On January 4, 2024, the Company entered into an exclusive collaboration and license Agreement (“the Tenacia License Agreement”) with Tenacia Biotechnology (Shanghai) Co., Ltd.

(“Tenacia”), a China-based portfolio company of Bain Capital, which provides Tenacia an exclusive license to use certain intellectual property for the development and commercialization of ulixacaltamide and products containing ulixacaltamide in China, Hong Kong, Macau and Taiwan.

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(“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.

Pursuant to the terms of the Tenacia 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.

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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.

Under the terms of the Tenacia 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 concluded that the Tenacia License Agreement and the Stock Purchase Agreement is a combined arrangement since they were executed at the same time and in contemplation of each other with the same counterparty or a related party thereof and the combined arrangement falls within the scope of Topic 606.

The Company’s obligations under the arrangement comprise a single promise, or one performance obligation, related to the exclusive development and commercialization license granted to Tenacia.

Total proceeds associated with the combined arrangement at inception were $ 14.8 million consisting of the following:

(i) $ 10.0 million gross proceeds from the sale of common stock under the Stock Purchase Agreement and (ii) $ 4.8 million, net of tax, related to the up-front payment under the License Agreement, both of which were received in January 2024.

During the three and nine months ended September 30, 2025 and 2024, the Company did not achieve any development or sales milestones or earn any royalties under the License Agreement.

(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 Tenacia License Agreement, both of which were received in January 2024.

During the three months ended March 31, 2026, the Company had not achieved any development or sales milestones or earned any royalties under the Tenacia 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.

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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.

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 Tenacia License Agreement in research and development expense in the accompanying consolidated statement of operations.

For agreements where the licenses are considered separate performance obligations or represent the only performance obligation, the Company recognizes revenue at the point in time that the Company effectively grants the license, as the licenses or assignments represent functional intellectual property.

As of September 30, 2025 and December 31, 2024, the Company did not have any receivables or deferred revenue related to the arrangement with Tenacia.

The Company did not recognize any contract assets related to costs to obtain a contract with a customer or costs to fulfill a contract with a customer through September 30, 2025 because no qualifying costs were incurred.

During the three and nine months ended September 30, 2025 and 2024, the Company did not recognize any revenue associated with the combined arrangement.

As of March 31, 2026, 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 March 31, 2026 because no qualifying costs were incurred.

During the three months ended March 31, 2026 and March 31, 2025, the Company did not recognize any revenue associated with the combined arrangement.

Common Stock and Preferred Stock

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

As of September 30, 2025 and December 31, 2024, the Company did no t hold any treasury shares.

As of March 31, 2026 and December 31, 2025, the authorized capital stock of the Company included 150,000,000 shares of common stock, $ 0.0001 par value.

As of March 31, 2026 and December 31, 2025, the Company did no t hold any treasury shares.

Follow-On Public Offerings

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The pre-funded warrants may be settled through either physical or net share settlement.

Following the occurrence of certain fundamental transactions, the holders of the pre-funded warrants have the right to receive upon exercise of the warrants the same amount and kind of securities, cash, or property as they would have been entitled to receive if they had been holders of the common shares issuable under the warrants immediately prior to such transaction.

During the year ended December 31, 2024, 152,145 pre-funded warrants were exercised via a cashless exercise, resulting in 152,142 shares of common stock issued.

No cash proceeds associated with the exercise were received by the Company.

During the nine months ended September 30, 2025, 200,355 pre-funded warrants were exercised via cashless exercise, resulting in 200,349 shares of common stock issued.

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, 2025, 200,355 pre-funded warrants were exercised via cashless exercise, resulting in 200,349 shares of common stock issued.

No cash proceeds associated with the exercise were received by the Company.

As of September 30, 2025, a total of 704,225 pre-funded warrants associated with this offering remained outstanding.

As of December 31, 2025, a total of 704,225 pre-funded warrants associated with this offering remained outstanding.

During the three months ended March 31, 2026, no pre-funded warrants were exercised.

As of March 31, 2026, a total of 704,225 pre-funded warrants associated with this offering remained outstanding.

April 2024 Public Offering

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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.

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.

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Following the occurrence of certain fundamental transactions, the holders of the pre-funded warrants have the right to receive upon exercise of the warrants the same amount and kind of securities, cash or property as they would have been entitled to receive if they had been holders of the common shares issuable under the warrants immediately prior to such transaction.

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

The Company determined that the pre-funded warrants related to the January 2024 and April 2024 public offerings are freestanding financial instruments because they are both legally detachable and separately exercisable from the common stock sold in the offering.

As of March 31, 2026, 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.

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.

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.

As of March 31, 2026, 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.

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.

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Accordingly, the Company has classified the pre-funded warrants as permanent equity.

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.

Shares Reserved for Future Issuance

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

September 30,

2026 December 31,

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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 October 2025 Financing 318,470 318,470

Shares reserved for future awards under the 2020 Stock Option and Incentive Plan 1,423,495 473,358

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Shares reserved for future awards under the 2024 Inducement Plan 944,629 945,280

Shares reserved for vesting of restricted stock units 516,119 224,473

Shares reserved for vesting of restricted stock units and performance stock units 636,178 501,853

Total shares of authorized common stock reserved for future issuance 6,727,981 5,322,382

Preferred Stock

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

As of March 31, 2026 and December 31, 2025, the authorized capital stock of the Company included 10,000,000 shares of undesignated preferred stock, $ 0.0001 par value.

Stock-Based Compensation

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In January 2025, the number of shares of common stock authorized for issuance under the Inducement Plan was increased by 870,000 shares.

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

The total number of shares of common stock authorized for issuance under the Inducement Plan as of March 31, 2026 and December 31, 2025 was 1,000,000 shares.

2020 Stock Option and Incentive Plan

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

The total number of shares of common stock authorized for issuance under the 2020 Stock Option and Incentive Plan (the "2020 Plan”) as of March 31, 2026 and December 31, 2025 was 4,201,704 shares and 2,941,950 shares, respectively.

2017 Stock Incentive Plan

The total number of shares of common stock authorized for issuance under the 2017 Stock Incentive Plan (the "2017 Plan") as of September 30, 2025 and December 31, 2024 was 395,850 shares.

Any authorization to issue new options under the 2017 Plan was cancelled upon the effectiveness of the 2020 Plan and no further awards will be granted under the 2017 Plan.

The total number of shares of common stock authorized for issuance under the 2017 Stock Incentive Plan (the "2017 Plan") as of March 31, 2026 and December 31, 2025 was 395,850 shares.

Any authorization to issue new

options under the 2017 Plan was cancelled upon the effectiveness of the 2020 Plan and no further awards will be granted under the 2017 Plan.

2020 Employee Stock Purchase Plan

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

The total number of shares of common stock authorized for issuance under the 2020 Employee Stock Purchase Plan (the "2020 ESPP”) as of March 31, 2026 and December 31, 2025 was 621,318 shares and 369,368 shares, respectively.

Restricted Stock Units

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Forfeited ( 15,791 ) 109.24

Unvested as of September 30, 2025 516,119 $ 61.24

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

Unvested as of March 31, 2026 504,376 $ 128.64

As of March 31, 2026, total unrecognized compensation cost related to unvested restricted stock units was $ 60.4 million, which is expected to be recognized over a weighted-average period of 3.43 years.

Performance Stock Units

In January 2026, the Company granted performance stock units ("PSUs") to its executive officers under the 2020 Plan.

Vesting of the PSUs is subject to the achievement of specified performance conditions, which include achieving objectively measurable clinical, regulatory and commercial revenue targets.

If these conditions are not met, the PSUs would be forfeited and returned to the plan.

The actual number of common shares issued that could potentially vest is calculated by multiplying the target number of PSUs granted by a multiplier determined for each performance goal, resulting in an aggregate payout ranging from 0 % to 250 % of the total target PSUs granted.

The performance conditions are required to be achieved during the performance period, which ends on December 31, 2027.

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

Shares Grant Date

Unvested as of December 31, 2025 — $ —

131,802 294.38

Forfeited — —

Unvested as of March 31, 2026 131,802 $ 294.38

(a) The number of shares issued during the period is the maximum amount of shares that could be payable under the terms of the PSU agreement.

As of March 31, 2026, total unrecognized compensation cost related to unvested PSUs that were determined to be probable of achievement was $ 24.1 million, which is expected to be recognized over a weighted-average period of 0.85 years.

Stock Options

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Cancelled or Forfeited ( 6,823 ) 81.11

Outstanding as of September 30, 2025 2,070,371 $ 87.20 7.94 $ 8,662

Exercisable as of September 30, 2025 1,197,365 $ 108.73 7.28 $ 6,571

Vested and expected to vest as of September 30, 2025 2,070,371 $ 87.20 7.94 $ 8,662

Outstanding as of March 31, 2026 1,946,616 $ 101.08 7.59 $ 454,075

Exercisable as of March 31, 2026 1,236,855 $ 107.19 7.03 $ 289,586

Vested and expected to vest as of March 31, 2026 1,946,616 $ 101.08 7.59 $ 454,075

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 and non-employees on the date of grant were as follows for the nine months ended September 30, 2025.

No stock options were granted during the three months ended September 30, 2025.

Nine Months Ended

September 30,

Three Months Ended

Risk-free interest rate 3.85 %

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Weighted average grant-date fair value per share $ 209.19

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

As of March 31, 2026, total unrecognized compensation cost related to unvested stock options was $ 46.4 million, which is expected to be recognized over a weighted-average period of 2.52 years.

Stock-Based Compensation

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Three Months Ended

September 30, Nine Months Ended

September 30,

2025 2024 2025 2024

Research and development $ 5,513 $ 3,780

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Three Months Ended

September 30, Nine Months Ended

September 30,

2025 2024 2025 2024

Outstanding stock options 1,946,616 2,050,354

Unvested restricted stock units 516,119 222,674 516,119 222,674

Unvested restricted stock units and performance stock units 636,178 522,257

Potential shares issuable under the 2020 ESPP 4,991 6,369

2,587,785 2,578,980

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

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 basic weighted average number of common shares outstanding for the three months ended March 31, 2026 and 2025.

Consistent with the guidance in ASC 260-10-45-13, the underlying common shares are issuable for little to no consideration and there are no vesting conditions or contingencies associated with the warrants.

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One of the founders of RogCon became the Company's General Counsel in June 2020.

The Company continues to reimburse RogCon for its out-of-pocket costs incurred for

activities performed under the License Agreement.

The Company continues to reimburse RogCon for its out-of-pocket costs incurred for activities performed under the License Agreement.

Expenses incurred during all periods presented were not material.

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Net loss is used by the CODM to make key strategic and operational decisions.

The operating segment's revenue is derived from a single domestic collaboration agreement from which the segment licenses certain development or product candidates and has performed research and development services.

The measure of segment assets is reported on the condensed consolidated balance sheet as total consolidated assets.

The majority of the Company's long-lived assets are held in the United States.

The following table presents selected financial information about the Company's single operating segment for the three and nine months ended September 30, 2025 and 2024 (in thousands):

The following table presents selected financial information about the Company's single operating segment for the three months ended March 31, 2026 and 2025 (in thousands):

Three Months Ended

September 30, Nine Months Ended

September 30,

2025 2024 2025 2024

Collaboration revenue $ — $ 302 $ — $ 1,090

Program-specific expenses:

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Other segment expenses (a)

5,908 5,730 21,037 15,913

Interest income 4,425 4,925 14,797 12,069

Net loss $ ( 73,934 ) $ ( 51,910 ) $ ( 214,357 ) $ ( 124,140 )

Other income, net 13,299 5,432

Consolidated net loss $ ( 92,561 ) $ ( 69,296 )

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

Subsequent Events

The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the consolidated financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure.

The Company has concluded that no subsequent events have occurred that require disclosure other than those noted below.

October 2025 Public Offering

On October 20, 2025, the Company completed a public offering of:

The purchase price per share of each pre-funded warrant represents the per share offering price for the common stock, less the $ 0.0001 per share exercise price of each underlying share.

Total net proceeds generated from the offering were approximately $ 567.0 million, after deducting underwriting discounts, commissions and other offering expenses payable by the Company.

The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the condensed consolidated financial statements to provide additional evidence for certain estimates or identify matters that require additional disclosure.

The Company has concluded that no subsequent events have occurred that require disclosure.

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-Q

Item 1. Financial Statements