What changed in this filing

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Item 2. Management’s Discussion and Analysis

2026-05-07 compared with 2025-11-05 · 85 added, 160 removed, 178 unchanged (58% of the section changed)

Management’s Discussion and Analysis of Financial Condition and Results of Operations.

You should read the following discussion and analysis of our financial condition and results of operations together with our condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and the audited financial information and the notes thereto included in our Annual Report on Form 10-K, which was filed with the Securities and Exchange Commission, or the SEC, on February 28, 2025.

You should read the following discussion and analysis of our financial condition and results of operations together with our condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and the audited financial information and the notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2025, which was filed with the Securities and Exchange Commission, or the SEC, on February 19, 2026.

Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business and related financing, includes forward-looking statements that involve risks and uncertainties.

As a result of many factors, including those factors set forth in the “Risk Factors” sections of our Annual Report on Form 10-K for the year ended December 31, 2024 and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, our actual results could differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.

We are a clinical-stage biopharmaceutical company translating insights from genetic epilepsies into the development of therapies for central nervous system, or CNS, disorders characterized by neuronal excitation-inhibition imbalance.

As a result of many factors, including those factors set forth in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2025, our actual results could differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.

We are a fully integrated, leading central nervous system, or 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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Through this approach, we have established a diversified, multimodal CNS portfolio with four clinical-stage product candidates across movement disorders and epilepsy.

For our ulixacaltamide program, we announced positive topline results for the two studies in the Phase 3 Essential3 program in essential tremor in October 2025.

Following the FDA's receipt of the Essential3 topline results, the FDA granted a Type B pre-NDA meeting that is scheduled to take place in the fourth quarter of 2025.

For our vormatrigine program (formerly known as PRAX-628) within the Cerebrum™ platform, we announced positive results from our Photo-Paroxysmal Response, or PPR, study in the first quarter of 2024, and initiated the ENERGY program to generate patient eligibility, efficacy, safety and pharmacokinetics data.

The ENERGY program includes five studies:

EMPOWER, RADIANT, POWER1, POWER2 and POWER3.

For our ulixacaltamide program within the Cerebrum™ platform, we announced positive topline results for the two studies in the Phase 3 Essential3 program in essential tremor, or ET, in October 2025.

We submitted a New Drug Application, or NDA, for ulixacaltamide HCl for the treatment of ET to the FDA, and it was accepted for review with a target action date under the Prescription Drug User Fee Act, or PDUFA, of January 29, 2027.

In anticipation of potential approval, commercial preparations and pre-launch activities are ongoing.

For our relutrigine program within the Cerebrum™ platform, in December 2025, we announced positive results from the registrational cohort of the EMBOLD study in SCN2A and SCN8A developmental and epileptic encephalopathies, or DEEs, after receiving a recommendation from the Data Monitoring Committee to stop the study early for efficacy.

We submitted an NDA for relutrigine for the treatment of SCN2A-DEE and SCN8A-DEE to the FDA, and it was accepted for priority review with a target PDUFA action date of September 27, 2026.

In anticipation of potential approval, commercial preparations and pre-launch activities are ongoing.

The EMERALD study, which is evaluating relutrigine in patients with broad DEEs, has completed recruitment, with topline results anticipated in the fourth quarter of 2026.

Assuming approval of the NDA for relutrigine, we believe the EMERALD study, if positive, could serve as the basis for a supplemental NDA, or sNDA, submission in 2027.

For our vormatrigine program within the Cerebrum™ platform, our ENERGY program is ongoing, which consists of five studies to generate patient eligibility, efficacy, safety and pharmacokinetics, or PK, data in patients with focal onset seizures, or FOS, or generalized epilepsy.

Studies under the ENERGY program include EMPOWER, RADIANT, POWER1, POWER2 and POWER3.

In the second half of 2025, we announced positive results from our RADIANT Phase 2 open-label study evaluating the PK, safety and efficacy of vormatrigine in patients with FOS or generalized epilepsy.

We initiated the EMPOWER study, an observational study of vormatrigine in patients with epilepsy, in the third quarter of 2024.

In August 2025, we announced positive topline results from the first cohort of our first efficacy study, RADIANT, an open label study in patients with focal onset seizures or generalized epilepsy.

We plan to share additional RADIANT study results at the American Epilepsy Society Annual Meeting in December 2025.

We have completed recruitment of the POWER1 study, a double-blind, placebo-controlled, 12-week study in focal onset seizures, and expect to announce topline results in the first half of 2026.

POWER2, our third efficacy study, has started, with enrollment expected to be completed in the second half of 2026.

We intend to initiate POWER3, a clinical trial evaluating vormatrigine as a single-agent treatment for focal onset seizures, in the first half of 2026.

For our relutrigine program (formerly known as PRAX-562), we announced positive topline results from the first cohort of our EMBOLD study in the third quarter of 2024, and disclosed updated data from the study's open-label extension through 11 months in May 2025.

In July 2025, the FDA granted breakthrough therapy designation for relutrigine for the treatment of seizures associated with SCN2A and SCN8A developmental and epileptic encephalopathies, or DEEs.

Following a comprehensive Type B meeting with the FDA, we will be performing an interim analysis of the second cohort of the ongoing EMBOLD study in the fourth quarter of 2025.

If positive, these

study results may support an NDA filing in early 2026.

We have also started enrollment of the EMERALD study that is investigating relutrigine in a broader DEE patient population, and expect to be completed in the second half of 2026.

For our most advanced product candidate under the Solidus™ platform, elsunersen (formerly known as PRAX-222), we shared results from Part 1 of the EMBRAVE study in the fourth quarter of 2023.

We are currently enrolling the second cohort of the EMBRAVE study in Brazil, with topline results expected in the first half of 2026.

We have initiated EMBRAVE3, a Phase 3 registrational study for SCN2A gain-of-function DEE, and expect to complete in 2026.

For our POWER1 study, a double-blind, placebo-controlled, 12-week study in FOS, we expect to announce topline results in the second quarter of 2026.

our third efficacy study, is enrolling patients, with completion expected in the second half of 2026 and topline results expected in 2027.

We intend to initiate POWER3, a clinical trial evaluating vormatrigine as a single-agent treatment for FOS, in the first half of 2026.

For our most advanced product candidate under the Solidus™ platform, elsunersen, we announced positive topline results from the EMBRAVE Part A study evaluating the safety and efficacy of elsunersen versus sham procedure in April 2026 for treating SCN2A gain-of-function DEE.

We are currently enrolling EMBRAVE3, a Phase 3 registrational study for SCN2A gain-of-function DEE, and expect to announce topline results in 2027.

We also have three earlier stage novel ASOs with preclinical proof of mechanism.

PRAX-080 is focused on PCDH19 mosaic expression, PRAX-090 is targeting SYNGAP1 loss-of-function, or LoF, mutations which is a leading cause of severe intellectual disability and epilepsy in DEEs, and PRAX-100 is targeting SCN2A LoF mutations, the predominant generic link to de novo autism spectrum disorders, or ASD.

PRAX-080 is focused on PCDH19 mosaic expression, PRAX-090 is targeting SYNGAP1 loss-of-function, or LoF, mutations which is a leading cause of severe intellectual disability and epilepsy in DEEs, and PRAX-100 is targeting SCN2A LoF mutations, the predominant genetic link to de novo autism spectrum disorders, or ASD.

We anticipate nominating a development candidate for PRAX-080, PRAX-090 and PRAX-100 in the first half of 2026.

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Since inception, we have financed our operations primarily with proceeds from the sale and issuance of equity securities.

We are a development stage company and we have not generated any revenue from product sales, and do not expect to do so for several years, if at all.

All of our product candidates are still in preclinical and clinical development.

We are a development stage company and we have not generated any revenue from product sales.

All of our product candidates are in preclinical and clinical development or awaiting regulatory approval.

Our ability to generate product revenue sufficient to achieve profitability will depend heavily on the successful development and eventual commercialization of one or more of our product candidates, if approved.

We have incurred recurring operating losses since inception, including a net loss of $214.4 million for the nine months ended September 30, 2025.

As of September 30, 2025, we had an accumulated deficit of $1.1 billion.

We expect to incur significant expenses and operating losses for the foreseeable future as we expand our research and development activities.

We have incurred recurring operating losses since inception, including a net loss of $92.6 million for the three months ended March 31, 2026.

As of March 31, 2026, we had an accumulated deficit of $1.2 billion.

We expect to incur significant expenses for the foreseeable future as we expand our research and development activities and prepare for commercialization of our product candidates.

In addition, our losses from operations may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and our expenditures on other research and development activities.

We anticipate that our expenses will be maintained or increased in connection with our ongoing activities, as we:

• prepare for potential commercialization of ulixacaltamide, following our positive Phase 3 results in the Essential3 clinical trial program for ET;

• advance vormatrigine in the ENERGY program;

• advance relutrigine in the EMBOLD and EMERALD clinical trials;

• advance elsunersen in the EMBRAVE and EMBRAVE3 clinical trials;

• prepare for the potential commercialization of ulixacaltamide, for which we have submitted an NDA to the FDA that has been accepted for review, after successful completion of our Phase 3 Essential3 program for ET;

• prepare for the potential commercialization of relutrigine, for which we have submitted an NDA to the FDA which has been accepted for review after a successful interim analysis in the EMBOLD clinical trial, and advance relutrigine in the EMERALD clinical trial;

• advance vormatrigine in the ENERGY clinical trials for FOS or generalized epilepsy;

• advance elsunersen in the EMBRAVE3 pivotal clinical trial;

• advance our preclinical candidates to clinical trials;

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• further invest in our manufacturing capabilities;

• seek regulatory approval for our product candidates;

• continue to seek regulatory approval for our product candidates;

• maintain, expand, protect and defend our IP portfolio;

• acquire or in-license technology;

• establish a sales, marketing and distribution infrastructure to commercialize any products for which we may obtain marketing approval;

• when needed, increase our headcount to support our development efforts and any future commercialization efforts.

As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy.

• continue building our sales, marketing and distribution infrastructure to commercialize any products for which we may obtain marketing approval;

• increase our headcount to support our development and commercialization efforts.

As a result, we will need substantial capital to support our continuing operations and pursue our growth strategy.

Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through the sale of equity, debt financings or other capital sources, including potential collaborations with other companies or other strategic transactions.

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If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be unable to continue our operations at planned levels and be forced to reduce or terminate our operations.

As of September 30, 2025, we had cash, cash equivalents and marketable securities of $389.2 million.

We expect that our cash, cash equivalents, and marketable securities as of September 30, 2025, together with proceeds from our follow-on pubic offering in October 2025, will be sufficient to fund our operating expenditures and capital expenditure requirements necessary to advance our program activities into 2028.

The analysis included consideration of our current financial needs and ongoing research, development and commercialization plans.

We have based this estimate on assumptions that may provide to be wrong, and we could exhaust our available capital resources sooner than we expect.

As of March 31, 2026, we had cash, cash equivalents and marketable securities of $1.4 billion.

We expect that our cash, cash equivalents, and marketable securities as of March 31, 2026 will be sufficient to fund our operating expenditures and capital expenditure requirements necessary to advance our operating activities into 2028.

The analysis included consideration of our current financial needs and ongoing research and development and commercialization plans.

We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect.

See “—Liquidity and Capital Resources.”

Financial Operations Overview

We have not generated any revenue from the sale of products since inception and do not expect to generate any revenue from the sale of products for several years, if at all.

As discussed in Note 7 to our condensed consolidated financial statements, we entered into an Option and License Agreement, or the Collaboration Agreement, with UCB Biopharma SRL, or UCB, in December 2022.

During the three and nine months ended September 30, 2024, we recognized $0.3 million and $1.1 million of collaboration revenue, respectively.

In December 2024, UCB exercised its option to in-license global development and commercialization rights under the terms of the Collaboration Agreement.

Accordingly, we did not recognize any collaboration revenue from the Collaboration Agreement during the three and nine months ended September 30, 2025.

We have no further research service obligations under the terms of the Collaboration Agreement.

We have not generated any revenue from the sale of products since inception and our ability to generate revenue from the sale of products in the future will be dependent on regulatory approval of our product candidates.

If our development efforts for our current or future product candidates are successful and result in marketing approval or collaboration or license agreements with third parties, we may generate revenue in the future from a combination of product sales or payments from such collaboration or license agreements.

Operating Expenses

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Due to the number of ongoing studies and our ability to use resources across platforms, indirect or shared operating costs incurred for our research and development platforms, such as personnel, facility costs and certain consulting costs, are not recorded or maintained on a platform-specific basis.

The following table reflects our research and development expenses, including direct expenses summarized by platform and indirect or shared operating costs recognized as research and development expenses during each period presented (in thousands):

Three Months Ended

September 30, Nine Months Ended

September 30,

$ 51,989 $ 42,832

$ 49,182 $ 27,474 $ 138,991 $ 55,226

3,244 1,327 6,571 4,197

Personnel-related (including stock-based compensation) 16,651 12,851

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Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials.

We expect that our research and development expenses will be maintained or increase in the foreseeable future as we advance our product candidates through the development phase, and as we continue to discover and develop additional product candidates and expand into additional therapeutic areas.

At this time, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete the development of, and obtain regulatory approval for, any of our product candidates.

We are also unable to predict when, if ever, material net cash inflows will commence from sales or licensing of our product candidates.

We are also unable to predict when, if ever, material net cash inflows will commence from sales, if approved, or licensing of our product candidates.

This is due to the numerous risks and uncertainties associated with drug development, including the uncertainty of:

• our ability to successfully develop, obtain regulatory approval for, and then successfully commercialize, our product candidates;

• our receipt of marketing approvals from applicable regulatory authorities;

• our ability to commercialize products, if approved, whether alone or in collaboration with others;

• our ability to add and retain key research and development personnel;

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• our ability to successfully complete clinical trials with safety, tolerability and efficacy profiles that are satisfactory to the FDA or any comparable foreign regulatory authority;

• our ability to successfully develop, obtain regulatory approval for, and then successfully commercialize, our product candidates;

• our successful enrollment in and completion of clinical trials;

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• our ability to obtain and maintain patent, trade secret and other IP protection and regulatory exclusivity for our product candidates, if approved;

• our receipt of marketing approvals from applicable regulatory authorities;

• our ability to commercialize products, if approved, whether alone or in collaboration with others;

• the continued acceptable safety profiles of our product candidates.

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professional fees for accounting, auditing, tax and administrative consulting services;

commercial-related costs to support market assessments and scenario planning;

commercial-related costs as we prepare for the potential launch of our product candidates;

insurance costs;

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Our income tax provision may be significantly affected by changes to our estimates.

There was no income tax provision recognized for the three and nine months ended September 30, 2025 and 2024.

There was no income tax provision recognized for the three months ended March 31, 2026 and 2025.

Results of Operations

Comparison of the Three Months Ended September 30, 2025 and 2024

Comparison of the Three Months Ended March 31, 2026 and 2025

The following table summarizes our condensed consolidated statements of operations for each period presented (in thousands):

Three Months Ended

September 30, Change

Collaboration revenue $ — $ 302 $ (302)

March 31, Change

Operating expenses:

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Net loss $ (92,561) $ (69,296) $ (23,265)

Collaboration Revenue

The $0.3 million decrease in collaboration revenue is associated with a decrease in the revenue recorded under the Collaboration Agreement with UCB that was executed in December 2022.

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

As such, we have no further research service obligations under the terms of the Collaboration Agreement and did not record any associated revenue during the three months ended September 30, 2025.

Research and Development Expense

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Three Months Ended

September 30, Change

$ 49,182 $ 27,474 $ 21,708

3,244 1,327 1,917

Personnel-related (including stock-based compensation) 11,069 10,863 206

Other indirect research and development expenses 2,302 2,217 85

Total research and development expenses $ 65,797 $ 41,881 $ 23,916

The $23.9 million increase in research and development expenses was primarily attributable to the following:

• $21.7 million increase in expense related to our Cerebrum™ platform, driven primarily by:

◦ a $14.6 million increase for our vormatrigine program, primarily driven by POWER1 and RADIANT study activities;

◦ a $13.4 million increase in spend for our relutrigine program, primarily related to EMBOLD and EMERALD study activities and manufacturing spend;

◦ a $1.9 million increase in development activities for our earlier stage assets as we advance our portfolio, partially offset by;

◦ a $8.2 million decrease in spend for our ulixacaltamide program, primarily due to decreased activity for Essential3 program and phase 1 spending.

• $1.9 million increase in expense related to our Solidus™ platform, driven primarily by elsunersen program spend for EMBRAVE3;

• $0.3 million increase in personnel-related costs and indirect expenses.

General and Administrative Expense

The $2.7 million decrease in general and administrative expenses was primarily attributable to the following:

• $2.6 million decrease in personnel-related costs due to decreased stock compensation expense;

• $0.6 million decrease in indirect expenses;

• $0.5 million increase in professional expenses.

Other income for the three months ended September 30, 2025 and 2024 was comprised of interest income on our cash, cash equivalents and marketable securities and investment premium and discount amortization.

Results of Operations

Comparison of the Nine Months Ended September 30, 2025 and 2024

The following table summarizes our condensed consolidated statements of operations for each period presented (in thousands):

Nine Months Ended

September 30, Change

Collaboration revenue $ — $ 1,090 $ (1,090)

Operating expenses:

Research and development 189,609 96,125 93,484

General and administrative 39,545 41,174 (1,629)

Total operating expenses 229,154 137,299 91,855

Loss from operations (229,154) (136,209) (92,945)

Other income:

Other income, net 14,797 12,069 2,728

Total other income 14,797 12,069 2,728

Net loss $ (214,357) $ (124,140) $ (90,217)

Collaboration Revenue

The $1.1 million decrease in collaboration revenue is associated with a decrease in the revenue recorded under the Collaboration Agreement with UCB that was executed in December 2022.

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

As such, we have no further research service obligations under the terms of the Collaboration Agreement and did not record any associated revenue during the nine months ended September 30, 2025.

Research and Development Expense

The following table summarizes our research and development expenses for each period presented, along with the changes in those items (in thousands):

Nine Months Ended

September 30, Change

March 31, Change

$ 51,989 $ 42,832 $ 9,157

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• $9.2 million increase in expense related to our Cerebrum™ platform, driven primarily by:

◦ a $43.5 million increase in spend for our vormatrigine program, primarily driven by POWER1 and RADIANT study spend as well as phase 1 study activities;

◦ a $27.0 million increase in spend for our relutrigine program, primarily related to our EMBOLD and EMERALD trial activities, manufacturing spend and Phase 1 trial spending;

◦ a $10.5 million increase in spend for our ulixacaltamide program, primarily due to Essential3 study activity;

◦ a $6.9 million increase in spend for our relutrigine program, primarily related to our EMERALD trial activities and manufacturing spend to support commercialization;

◦ a $6.9 million increase in spend for our vormatrigine program, primarily driven by spend for our ENERGY program;

◦ a $1.7 million increase in activities for our earlier stage assets as we advance our portfolio;

• $5.1 million increase in personnel-related costs due to increased headcount;

• $2.4 million increase in expense related to our Solidus™ platform, driven by elsunersen program spend;

• $2.2 million increase in indirect expenses driven primarily by software and consulting spend to support operations.

partially offset by,

◦ a $6.3 million decrease in spend for our ulixacaltamide program, driven primarily by decreased activity in the Essential3 study, partially offset by increased manufacturing costs to support commercialization and the PDUFA fee paid in the first quarter of 2026.

• $3.8 million increase in personnel-related costs mainly due to increased headcount;

• $3.0 million increase in expense related to our Solidus™ platform, driven by elsunersen program spend for EMBRAVE Part A clinical trial and our EMBRAVE3 pivotal clinical trial.

General and Administrative Expense

The $1.6 million decrease in general and administrative expenses was primarily attributable to the following:

• $3.8 million decrease in personnel-related costs mainly due to decreased stock-based compensation expense;

• $1.3 million increase in professional expenses primarily related to legal and audit expenses;

• $0.9 million increase in indirect expenses, including increased sponsorships and advocacy activities.

Other income for the nine months ended September 30, 2025 and 2024 was comprised of interest income on our cash, cash equivalents, and marketable securities and investment premium and discount amortization.

The $14.0 million increase in general and administrative expenses was primarily attributable to the following:

• $9.8 million increase in personnel-related costs mainly due to increased stock-based compensation expense and increased headcount;

• $3.5 million increase in professional expenses, primarily attributable to increased commercial spend as we prepare for the potential launch of ulixacaltamide and relutrigine.

Liquidity and Capital Resources

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Since our inception, we have incurred significant losses in each period.

We have not yet commercialized any of our product candidates, which are in various phases of preclinical and clinical development, and we may not generate revenue from sales of any products for several years, if at all.

We have not yet commercialized any of our product candidates, which are in various phases of preclinical and clinical development or awaiting regulatory review, and we may not generate meaningful revenue from product sales in the near term, if at all.

To date, we have financed our operations primarily with proceeds from the issuance of redeemable convertible preferred stock and from the sale of common stock through an initial public offering, common stock and pre-funded warrants through follow-on public offerings and common stock from at-the-market offerings under our shelf registration statement.

From inception through September 30, 2025, we have raised $1.2 billion in aggregate cash

proceeds from such transactions, net of issuance costs.

As of September 30, 2025, we had cash, cash equivalents and marketable securities of $389.2 million.

In December 2023, we entered into an Open Market Sale Agreement, or 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 nine months ended September 30, 2024, we 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 us.

In January 2024, we completed a public offering of:

(i) an aggregate of 3,802,025 shares of our 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 us.

As of September 30, 2025, 352,500 warrants associated with this offering were exercised on a cashless basis with no cash proceeds received by us.

In March 2024, we entered into an Open Market Sale Agreement, or the 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 in at-the-market offerings.

During the nine months ended September 30, 2024, we issued and sold an aggregate of 42,851 shares under the 2024 Sales Agreement for aggregate net proceeds of $2.3 million, after deducting commissions and offering expenses payable by us.

In April 2024, we completed a public offering of:

(i) an aggregate of 3,849,558 shares of our 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 us.

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

In December 2024, we entered into an amendment to the 2024 Sales Agreement with Jefferies 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 nine months ended September 30, 2025, we issued and sold an aggregate of 1,351,689 shares under the amended 2024 Sales Agreement for aggregate net proceeds of $83.3 million, after deducting commissions and offering expenses payable by us.

From inception through March 31, 2026, we have raised $2.4 billion in aggregate cash proceeds from such transactions, net of issuance costs.

As of March 31, 2026, we had cash, cash equivalents, and marketable securities of $1.4 billion.

In March 2024, we entered into an Open Market Sale Agreement, or the 2024 Sales Agreement, with Jefferies LLC, or Jefferies, to provide for the offering, issuance, and sale of up to an aggregate amount of $150.0 million of common stock in at-the-market offerings.

In December 2024, we entered into an amendment to the 2024 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 three months ended March 31, 2025, we issued and sold an aggregate of 694,212 shares under the amended 2024 Sales Agreement for aggregate net proceeds of $54.9 million, after deducting commissions and offering expenses payable by us.

The amended 2024 Sales Agreement was terminated in September 2025.

In September 2025, we 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 nine months ended September 30, 2025, we issued and sold an aggregate of 119,364 shares under the 2025 Sales Agreement for aggregate net proceeds of $5.8 million, after deducting commissions and offering expenses payable by us.

During the three months ended March 31, 2026, we did not issue or sell any shares of common stock under the 2025 Sales Agreement.

In October 2025, we completed a public offering of:

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

As of March 31, 2026, none of the pre-funded warrants had been exercised and all remained outstanding.

In January 2026, we completed a public offering of 2,543,800 shares of our 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 us.

The following table provides information regarding our cash flows for each period presented (in thousands):

Nine Months Ended

September 30,

Three Months Ended

Net cash (used in) provided by:

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Financing activities 609,431 54,269

Net (decrease) increase in cash, cash equivalents and restricted cash $ (65,845) $ 87,345

Net increase (decrease) in cash, cash equivalents and restricted cash $ 179,004 $ (49,805)

Operating Activities

Our cash flows from operating activities are greatly influenced by our use of cash for operating expenses and working capital requirements to support our business.

Our cash flows from operating activities are greatly influenced by our use of cash for operating expenses and working capital requirements to support the business.

We have historically experienced negative cash flows from operating activities as we have invested in developing our portfolio, drug discovery efforts and related infrastructure.

The cash used in operating activities resulted primarily from our net losses adjusted for non-cash charges and changes in operating assets and liabilities, which are primarily the result of increased expenses and timing of vendor payments.

During the nine months ended September 30, 2025, net cash used in operating activities of $172.7 million was primarily due to our $214.4 million net loss, partially offset by $19.7 million in changes in operating assets and liabilities primarily related to an increase in accounts payable and $22.0 million of non-cash charges primarily related to stock-based compensation.

During the nine months ended September 30, 2024, net cash used in operating activities of $75.6 million was primarily due to our $124.1 million net loss, partially offset by $15.8 million in changes in operating assets and liabilities primarily related to increases in accounts payable and accrued expenses, and $32.7 million of non-cash charges primarily related to stock-based compensation.

The cash used in operating activities resulted primarily from our net losses adjusted for non-cash charges and changes in operating assets and liabilities.

During the three months ended March 31, 2026, net cash used in operating activities of $86.2 million was primarily due to our $92.6 million net loss and $10.3 million in changes in operating assets and liabilities primarily related to a decrease in accrued expenses, partially offset by $16.7 million of non-cash charges primarily related to stock-based compensation.

During the three months ended March 31, 2025, net cash used in operating activities of $53.0 million was primarily due to our $69.3 million net loss, partially offset by $8.4 million in changes in operating assets and liabilities primarily related to an increase in accounts payable and $7.9 million of non-cash charges primarily related to stock-based compensation.

Investing Activities

During the nine months ended September 30, 2025, net cash provided by investing activities of $17.2 million was primarily related to maturities of marketable securities offset by purchases of marketable securities.

During the nine months ended September 30, 2024, net cash used in investing activities of $237.6 million was primarily related to purchases of marketable securities partially offset by maturities of marketable securities.

During the three months ended March 31, 2026, net cash used in investing activities of $344.3 million was primarily related to purchases of marketable securities offset by maturities of marketable securities.

During the three months ended March 31, 2025, net cash used in investing activities of $51.1 million was primarily related to purchases of marketable securities offset by maturities of marketable securities.

Financing Activities

During the nine months ended September 30, 2025, net cash provided by financing activities of $89.7 million consisted primarily of net proceeds from our at-the-market offerings.

During the nine months ended September 30, 2024, net cash provided by financing activities of $400.6 million consisted primarily of net proceeds from our January 2024 and April 2024 follow-on public offerings, our at-the-market offerings and our collaboration and license agreement with Tenacia.

During the three months ended March 31, 2026, net cash provided by financing activities of $609.4 million consisted primarily of net proceeds from our January 2026 follow-on offering.

During the three months ended March 31, 2025, net cash provided by financing activities of $54.3 million consisted primarily of net proceeds from our at-the-market offerings.

Plan of Operation and Future Funding Requirements

As a result, we expect to incur substantial operating losses and negative operating cash flows for the foreseeable future.

We expect our expenses to increase substantially in connection with our ongoing research and development activities, particularly as we advance the preclinical activities and clinical trials of our product candidates, and our commercialization activities as we prepare for the potential approval of our product candidates.

As a result, we expect to incur substantial operating expenses for the foreseeable future.

We anticipate that our expenses will increase substantially if and as we:

• continue to build a sales, marketing, technology and distribution infrastructure and scale-up manufacturing capabilities to commercialize any products for which we may obtain regulatory approval;

• advance the clinical development of our clinical-stage product candidates within our Cerebrum™ and Solidus™ platforms;

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• seek to obtain regulatory approvals for our product candidates;

• establish a sales, marketing and distribution infrastructure and scale-up manufacturing capabilities to commercialize any products for which we may obtain regulatory approval;

• when needed, add clinical, scientific, operational, financial and management information systems and personnel, including personnel to support our product development and potential future commercialization efforts and to support our operations as a public company;

• when needed, add clinical, scientific, operational, financial and management information systems and personnel, including personnel to support our product development and commercialization efforts and to support our operations as a public company;

• experience any delays or encounter any issues with any of the above, including but not limited to failed studies, complex results, safety issues or other regulatory challenges.

We are unable to estimate the exact amount of our working capital requirements, but based on our current operating plan, we believe that our cash, cash equivalents, and marketable securities as of September 30, 2025, together with proceeds from our follow-on public offering in October 2025, will be sufficient to fund our operating expenses and capital expenditure requirements into 2028.

We are unable to estimate the exact amount of our working capital requirements, but based on our current operating plan, we believe that our cash, cash equivalents, and marketable securities as of March 31, 2026 will be sufficient to fund our operating expenditures and capital expenditure requirements into 2028.

However, we have based this estimate on assumptions that may prove to be wrong and we could exhaust our capital resources sooner than we expect.

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In addition, our product candidates, if approved, may not achieve commercial success.

Our commercial revenues, if any, will be derived

from sales of products that we do not expect to be commercially available for many years, if ever.

Accordingly, we will need to obtain substantial additional funds to achieve our business objectives.

Accordingly, we may need to obtain substantial additional funds to achieve our business objectives.

Adequate additional funds may not be available to us on acceptable terms, or at all.

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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 2. Management’s Discussion and Analysis