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Praxis Precision Medicines, Inc.

For the period ended Mar 31, 2026. Filed May 7, 2026.

View on SEC.gov

About this filing

A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.

Mar 31, 2026 · Filed May 7, 2026

PRAX$277.62+5.7%Stored close · as of Nov 6, 2026

What changed in Management’s Discussion and Analysis?

Read the current filing

Previous report: 10-Q

Period ended 2025-09-30; filed 2025-11-05.

0001689548-25-000111

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

Current report: 10-Q

Period ended 2026-03-31; filed 2026-05-07.

0001689548-26-000047

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.

Compared 167 previous paragraphs and 142 current paragraphs.

85 unchanged; 48 reworded; 9 added; 24 removed; 10 previous and 0 current paragraphs rearranged.

Tables are not compared: 6 previous and 4 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.

A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.

Added paragraphs (9)

Current report

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.

Current report

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.

Current report

•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;

Current report

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.

Current report

◦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.

Current report

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.
Removed paragraphs (24)

Previous report

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.

Previous report

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.

Previous report

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

Previous report

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.

Previous report

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.

Previous report

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

Previous report

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

Previous report

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

Previous report

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

Previous report

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

Previous report

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.

Previous report

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

Previous report

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.

Previous report

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

Previous report

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

Previous report

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

Previous report

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

Previous report

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.

Previous report

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.
Reworded paragraphs (48)

Previous wording

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. 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.

Current wording

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” 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.

Previous wording

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. 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. We are applying genetic insights to the discovery and development of therapies for neurological disorders through two proprietary platforms, using our understanding of shared biological targets and circuits in the brain. Each platform currently has multiple programs, with significant potential for additional program and indication expansion:

Current wording

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. We are applying genetic insights to the discovery and development of therapies for neurological disorders through two proprietary platforms, using our understanding of shared biological targets and circuits in the brain. Each platform currently has multiple programs, with significant potential for additional program and indication expansion:

Previous wording

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. 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.

Current wording

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. 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. POWER2, 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.

Previous wording

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.

Current wording

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.

Previous wording

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

Current wording

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 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.

Previous wording

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. 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. 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:

Current wording

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 $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:

Previous wording

•advance relutrigine in the EMBOLD and EMERALD clinical trials;

Current wording

•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;

Current wording

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

Previous wording

•advance elsunersen in the EMBRAVE and EMBRAVE3 clinical trials;

Current wording

•advance elsunersen in the EMBRAVE3 pivotal clinical trial;

Current wording

•continue to seek regulatory approval for our product candidates;

Previous wording

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

Current wording

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

Previous wording

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

Current wording

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

Previous wording

As a result, we will need substantial additional funding 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. We may be unable to raise additional funds or enter into such other agreements or arrangements when needed on favorable terms, or at all. If we fail to raise capital or enter into such agreements as and when needed, we may have to significantly delay, scale back or discontinue the development and commercialization of one or more of our product candidates or delay our pursuit of potential in-licenses or acquisitions.

Current wording

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. We may be unable to raise additional funds or enter into such other agreements or arrangements when needed on favorable terms, or at all. If we fail to raise capital or enter into such agreements as and when needed, we may have to significantly delay, scale back or discontinue the development and commercialization of one or more of our product candidates or delay our pursuit of potential in-licenses or acquisitions.

Previous wording

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. See “—Liquidity and Capital Resources.”

Current wording

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.”

Previous wording

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):

Current wording

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 the periods presented below (in thousands):

Previous wording

Research and development activities are central to our business model. 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.

Current wording

Research and development activities are central to our business model. 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, build manufacturing capabilities and expand into additional therapeutic areas.

Previous wording

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

Current wording

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, 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:

Previous wording

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

Current wording

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

Previous wording

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

Current wording

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

Previous wording

General and administrative expenses consist primarily of personnel-related costs, including salaries, benefits and stock-based compensation, for personnel in our executive, finance, legal, commercial and administrative functions. General and administrative expenses also include legal fees relating to corporate matters; professional fees for accounting, auditing, tax and administrative consulting services; commercial-related costs to support market assessments and scenario planning; insurance costs; administrative travel expenses; and facility-related expenses, which include direct depreciation costs and allocated expenses for office rent and other operating costs, such as information technology. Costs to secure and defend our IP are expensed as incurred and are classified as general and administrative expenses. These costs relate to the operation of the business and are unrelated to the research and development function or any individual platform or product candidate.

Current wording

General and administrative expenses consist primarily of personnel-related costs, including salaries, benefits and stock-based compensation, for personnel in our executive, finance, legal, commercial and administrative functions. General and administrative expenses also include legal fees relating to corporate matters; professional fees for accounting, auditing, tax and administrative consulting services; commercial-related costs as we prepare for the potential launch of our product candidates; insurance costs; administrative travel expenses; and facility-related expenses, which include direct depreciation costs and allocated expenses for office rent and other operating costs, such as information technology. Costs to secure and defend our IP are expensed as incurred and are classified as general and administrative expenses. These costs relate to the operation of the business and are unrelated to the research and development function or any individual platform or product candidate.

Previous wording

Since our inception, we have not recorded any U.S. federal or state income tax benefits for the net losses we have incurred in each year or for our earned research and development tax credits due to our uncertainty of realizing a benefit from those items. Income taxes are determined at the applicable tax rates adjusted for non-deductible expenses, research and development tax credits and other permanent differences. 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.

Current wording

Since our inception, we have not recorded any U.S. federal or state income tax benefits for the net losses we have incurred in each year or for our earned research and development tax credits due to our uncertainty of realizing a benefit from those items. Income taxes are determined at the applicable tax rates adjusted for non-deductible expenses, research and development tax credits and other permanent differences. Our income tax provision may be significantly affected by changes to our estimates. There was no income tax provision recognized for the three months ended March 31, 2026 and 2025.

Previous wording

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

Current wording

The $17.2 million increase in research and development expenses was primarily attributable to the following:

Previous wording

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

Current wording

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

Previous wording

◦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;

Current wording

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

Previous wording

◦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;

Current wording

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

Previous wording

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

Current wording

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

Previous wording

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

Current wording

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

Previous wording

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

Current wording

•$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.

Previous wording

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

Current wording

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

Previous wording

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

Current wording

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

Current wording

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

Previous wording

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.

Current wording

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 or awaiting regulatory review, and we may not generate meaningful revenue from product sales in the near term, if at all.

Previous wording

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.

Current wording

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 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.

Previous wording

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.

Current wording

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.

Previous wording

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.

Current wording

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

Previous wording

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.

Current wording

In October 2025, we completed a public offering of: (i) an aggregate of 3,527,072 shares of our 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 $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.

Previous wording

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. 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.

Current wording

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.

Previous wording

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.

Current wording

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.

Previous wording

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.

Current wording

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.

Previous wording

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.

Current wording

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.

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Previous report — wording carried elsewhere

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

Previous report — wording carried elsewhere

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

Previous report — wording carried elsewhere

The $93.5 million increase in research and development expenses was primarily attributable to the following:

Previous report — wording carried elsewhere

•$83.8 million increase in expense related to our Cerebrum™ platform, driven primarily by:

Previous report — wording carried elsewhere

◦a $2.8 million increase in activities for our earlier stage assets as we advance our portfolio.

Previous report — wording carried elsewhere

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

Previous report — wording carried elsewhere

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

Previous report — wording carried elsewhere

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.

Previous report — wording carried elsewhere

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. The amended 2024 Sales Agreement was terminated in September 2025.

Previous report — wording carried elsewhere

In October 2025, we completed a public offering of: (i) an aggregate of 3,527,072 shares of our common stock at a public offering price of $157.00 per share, including the underwriters' full exercise of their option to purchase 501,592 additional shares of common stock, and (ii) pre-funded warrants to purchase 318,470 shares of common stock at a public offering price of $156.9999 per share of common stock underlying the warrants. The purchase price per share of each pre-funded warrant represents the per share offering price for the common stock, less the $0.0001 per share exercise price of each underlying share. Total net proceeds generated from the offering were approximately $567.0 million, after deducting underwriting discounts, commissions and other offering expenses payable by us.