Praxis Precision Medicines (NASDAQ: PRAX) posts strong seizure drug results

Rhea-AI Filing Summary

Praxis Precision Medicines reported multiple advances across its neurology pipeline. For ulixacaltamide, the company completed a successful pre-New Drug Application meeting with the U.S. Food and Drug Administration and gained alignment on NDA content, and now expects to finish its NDA submission in early 2026.

For relutrigine, Praxis announced positive registrational EMBOLD results in patients with SCN2A and SCN8A developmental and epileptic encephalopathies. Patients on relutrigine (n=51) achieved a 53% placebo-adjusted reduction in seizures over 16 weeks and a 66% increase in motor seizure-free days, with statistically significant improvements in clinician and caregiver global impression scores and no drug-related serious adverse events. Praxis plans to meet with the FDA and set NDA timing afterward.

For vormatrigine, full RADIANT data in focal onset seizures showed an 8-week 54% median seizure reduction, with FOS patients reaching 100% median weekly seizure reduction by week 8 and maintaining this through 16 weeks; more than 11% were seizure-free for the entire 8-week period and roughly one third for a consecutive 28-day period. Recruitment is complete for the POWER1 pivotal study in focal onset seizures, POWER2 is on track to finish in the second half of 2026, and the POWER3 monotherapy study is expected to begin in the first half of 2026.

Positive

• Positive registrational data for relutrigine, including a 53% placebo-adjusted seizure reduction over 16 weeks and a 66% increase in motor seizure-free days in SCN2A and SCN8A DEE patients, with no drug-related serious adverse events.
• Encouraging seizure control with vormatrigine, with a 54% median seizure reduction in focal onset seizures, 100% median weekly seizure reduction by week 8 maintained through 16 weeks, and completion of recruitment for the POWER1 pivotal study.
• Regulatory progress for ulixacaltamide, with a successful pre-NDA meeting, alignment with the FDA on NDA content, and an expected NDA submission completion in early 2026.

Negative

• None.

Insights

Biotech clinical-development analyst

Praxis reported positive late-stage epilepsy data and clear regulatory paths for three CNS programs.

Praxis Precision Medicines disclosed coordinated progress across ulixacaltamide, relutrigine and vormatrigine. Ulixacaltamide has completed a pre-NDA meeting with the U.S. Food and Drug Administration, with alignment on the content of the NDA and an expected submission completion in early 2026, which moves this program toward a potential marketing application.

Relutrigine’s registrational EMBOLD cohort in SCN2A and SCN8A developmental and epileptic encephalopathies showed a 53% placebo-adjusted seizure reduction over 16 weeks and a 66% increase in motor seizure-free days, alongside statistically significant clinician and caregiver impression scores. The absence of drug-related serious adverse events and predominantly mild or moderate treatment-related adverse events supports the tolerability profile described here. Praxis intends to meet with the FDA and then decide on NDA timing, so regulatory strategy for this program hinges on that interaction.

Vormatrigine’s RADIANT data in focal onset seizures showed a 54% median seizure reduction over 8 weeks, with FOS patients reaching 100% median weekly seizure reduction by week 8 and maintaining it through 16 weeks; more than 11% achieved seizure freedom across the entire 8-week period. Completion of recruitment for the POWER1 pivotal study, and defined timelines for POWER2 and POWER3 into 2026, outline a multi-study path intended to further characterize efficacy and safety in focal onset and generalized epilepsy settings.

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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d)

of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): December 4, 2025

PRAXIS PRECISION MEDICINES, INC.

(Exact name of registrant as specified in its charter)

Delaware			001-39620			47-5195942
(State or other jurisdiction of incorporation)			(Commission File Number)			(I.R.S. Employer Identification No.)

Praxis Precision Medicines, Inc.

99 High Street, 30th Floor

Boston, Massachusetts 02110

(Address of principal executive offices, including zip code)

(617) 300-8460

(Registrant’s telephone number, including area code)

Not Applicable

(Former Name or Former Address, if Changed Since Last Report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

☐ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class

Trade Symbol(s)

Name of each exchange on which registered

Common Stock, $0.0001 par value per share

PRAX

The Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

Emerging growth company  ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.  ☐

Item 8.01. Other Events.

Ulixacaltamide

On December 4, 2025, Praxis Precision Medicines, Inc. (the “Company”) announced the successful completion of its pre-New Drug Application (“NDA”) meeting with the U.S. Food and Drug Administration (the “FDA”), including receipt of written feedback and an in-person meeting. The Company has gained alignment from the FDA on the content of the NDA and expects to complete its NDA submission in early 2026.

Relutrigine

On December 4, 2025, the Company announced positive results from the registrational cohort of the EMBOLD study evaluating relutrigine for the treatment of patients with SCN2A and SCN8A developmental and epileptic encephalopathies (“DEEs”), following a recommendation by the Data Monitoring Committee to stop the study early for efficacy.

On December 6, 2025, the Company shared the results of the EMBOLD study, demonstrating relutrigine was well-tolerated with rapid, significant and increasing seizure reduction over time with broad functional improvements across behavior, alertness, communication and overall status. The Company will meet with the FDA in the coming weeks to discuss next steps. The Company will make a determination of the timing for filing the NDA after the meeting.

The topline results for the EMBOLD study were as follows:

•Patients receiving relutrigine (n=51) experienced a 53% placebo-adjusted reduction in seizures over 16-weeks (p<0.0002).

•Patients achieved a 66% increase in motor seizure-free days (p=0.034).

•Both clinician and caregiver global impression scores showed statistically significant improvements, with most patients improving across both scales in alertness, communication, and seizure severity.

•There were no drug-related serious adverse events and treatment-related adverse events were predominantly mild and moderate.

Vormatrigine

On December 6, 2025, the Company shared the full results of the RADIANT study evaluating vormatrigine in patients with focal onset seizures (“FOS”) or generalized epilepsy. The key results were as follows:

Focal Onset Seizures (n=62)

•Patients taking vormatrigine for 8 weeks on background anti-seizure medications saw a 54% median reduction in seizures.

•In week 1, 58% of patients achieved at least a 50% reduction in seizures, which increased to 61% by week 8.

•Increasing and sustained effect was observed, with FOS patients reaching 100% median weekly seizure reduction after 8 weeks and maintained through 16 weeks.

•Over 11% of patients experienced seizure freedom for the entire 8-week period and roughly one third of patients experienced seizure freedom for a consecutive 28-day period.

Generalized Epilepsy (n=3)

•Three patients with generalized epilepsy included in the cohort experienced a similar treatment effect as FOS patients, with rapid, durable seizure reduction.

The Company has completed recruiting for the POWER1 pivotal study in FOS and is on track to complete the POWER2 study in the second half of 2026. The monotherapy study, POWER3, is on track to begin in the first half of 2026.

Forward-Looking Statements

This Current Report contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the clinical development of relutrigine and vormatrigine and the anticipated timing of regulatory submissions and interactions for ulixacaltamide and relutrigine. The forward-looking statements included in this Current Report are subject to a number of risks, uncertainties and assumptions, including, without limitation, uncertainties inherent in clinical trials, the expected timing of submission for regulatory approval or review by governmental authorities and other risks as described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024, its Quarterly Report on Form 10-Q for the quarter ended June 30, 2025 and its other filings with the Securities and Exchange Commission. These statements are based only on facts currently known by the Company and speak only as of the date of this Current Report. As a result, you are cautioned not to rely on these forward-looking statements and the Company undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise.

SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

			PRAXIS PRECISION MEDICINES, INC.
Date: December 8, 2025			By:						/s/ Marcio Souza
									Marcio Souza
									Chief Executive Officer

FAQ

What did Praxis Precision Medicines (PRAX) announce about ulixacaltamide?

Praxis reported a successful pre-New Drug Application meeting with the U.S. Food and Drug Administration for ulixacaltamide, gaining alignment on the content of the NDA and stating it expects to complete its NDA submission in early 2026.

What were the key results from the EMBOLD study of relutrigine disclosed by PRAX?

In the EMBOLD registrational cohort for relutrigine in SCN2A and SCN8A developmental and epileptic encephalopathies, patients (n=51) achieved a 53% placebo-adjusted reduction in seizures over 16 weeks and a 66% increase in motor seizure-free days, with statistically significant improvements in clinician and caregiver global impression scores.

How well was relutrigine tolerated in Praxis Precision Medicines' EMBOLD study?

Praxis stated that relutrigine was well-tolerated in the EMBOLD study, reporting no drug-related serious adverse events and predominantly mild and moderate treatment-related adverse events.

What did the RADIANT study show about vormatrigine in focal onset seizures?

In focal onset seizures (n=62), vormatrigine produced a 54% median reduction in seizures over 8 weeks, with 58% of patients achieving at least a 50% reduction in week 1, increasing to 61% by week 8, and FOS patients reaching 100% median weekly seizure reduction by week 8 and maintaining this effect through 16 weeks.

Did Praxis report seizure freedom outcomes for vormatrigine in the RADIANT study?

Yes. Praxis noted that over 11% of patients in the focal onset seizure cohort experienced seizure freedom for the entire 8-week period and roughly one third experienced seizure freedom for a consecutive 28-day period while on vormatrigine.

What are the next clinical steps for vormatrigine in PRAX's POWER program?

Praxis has completed recruiting for the POWER1 pivotal study in focal onset seizures. It reported that POWER2 is on track to complete in the second half of 2026 and that the monotherapy study POWER3 is on track to begin in the first half of 2026.

When does Praxis Precision Medicines expect to file NDAs for ulixacaltamide and relutrigine?

Praxis expects to complete the ulixacaltamide NDA submission in early 2026. For relutrigine, the company plans to meet with the U.S. Food and Drug Administration and will determine the timing for filing the NDA after that meeting.