Praxis Precision Medicines (PRAX) Company Report

Praxis Precision Medicines, Inc. announced FDA agreement to convert the EMBRAVE3 registrational study of elsunersen into a single-arm study, reducing patient enrollment from 40 to 30 patients.
The company expects to disclose topline results for Part A of the EMBRAVE Study in the first half of 2026.
The FDA has confirmed that Praxis Precision Medicines, Inc. can file a New Drug Application for relutrigine in early 2026 for SCN2A and SCN8A DEEs.

Dec 11, 2025, 9:23 PM

8-K

Praxis Precision Medicines Confirms Relutrigine NDA Submission in Early 2026

PRAX

Product Launch

New Projects/Investments

Praxis Precision Medicines has successfully completed discussions with the FDA, confirming its plan to file a New Drug Application (NDA) for relutrigine in early 2026.
The NDA for relutrigine, a treatment for developmental and epileptic encephalopathies (DEEs) including SCN2A and SCN8A, is supported by statistically significant and clinically meaningful improvements observed in the EMBOLD study.
Relutrigine has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, and Breakthrough Therapy Designation from the European Medicines Agency for SCN2A-DEE and SCN8A-DEE.

Dec 11, 2025, 1:00 PM

Press Release

Praxis Precision Medicines Announces FDA Alignment on Elsunersen Study Design

PRAX

New Projects/Investments

Product Launch

Praxis Precision Medicines announced FDA alignment to convert the EMBRAVE3 registrational study for elsunersen into a single-arm, baseline-controlled study, reducing patient enrollment from 40 to 30.
This change ensures all patients in the EMBRAVE3 trial will receive active treatment from day one, with the primary analysis focusing on the change from baseline in countable motor seizures.
Praxis anticipates topline results for EMBRAVE3 in 2026 and for the EMBRAVE study (Part A) in the first half of 2026.

Dec 9, 2025, 9:01 PM

Press Release

Praxis Precision Medicines, Inc. Reports Positive Clinical Trial Results and Regulatory Progress for Multiple Programs

PRAX

Product Launch

New Projects/Investments

Praxis Precision Medicines, Inc. successfully completed its pre-New Drug Application (NDA) meeting with the FDA for ulixacaltamide, with the NDA submission anticipated in early 2026.
The company announced positive results from the registrational EMBOLD study for relutrigine, which was stopped early for efficacy, demonstrating a 53% placebo-adjusted reduction in seizures over 16 weeks.
Full results from the RADIANT study for vormatrigine showed a 54% median reduction in seizures for focal onset seizure patients, with over 11% achieving seizure freedom for 8 weeks. Recruitment for the POWER1 pivotal study is complete, and the POWER3 monotherapy study is on track to begin in the first half of 2026.

Dec 8, 2025, 9:18 PM

8-K

Praxis Precision Medicines Plans Early 2026 NDA Submission for Ulixacaltamide

PRAX

Product Launch

New Projects/Investments

Praxis Precision Medicines successfully completed a pre-New Drug Application (NDA) meeting with the FDA for ulixacaltamide, an investigational treatment for essential tremors, with plans to submit the NDA in early 2026.
The FDA provided written feedback and alignment on the NDA content, moving Praxis closer to offering a new therapy for the seven million people in the U.S. affected by essential tremor.
Ulixacaltamide demonstrated statistically significant and clinically meaningful improvements in daily functioning in the Essential3 clinical program.
Following the positive FDA meeting, Praxis shares rose 25% in premarket trading to $237.10 per share.
Additionally, the company reported positive Phase 2 results for relutrigine in treating certain developmental and epileptic encephalopathies and has a meeting scheduled with the FDA to review this data.

Dec 5, 2025, 1:39 AM

Morningstar

Praxis Precision Medicines Announces Positive EMBOLD Study Results for Relutrigine

PRAX

New Projects/Investments

Praxis Precision Medicines 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).
The study was stopped early for efficacy following a recommendation by the Data Monitoring Committee.
Relutrigine is a first-in-class small molecule in development for DEEs, which currently have no approved treatment options.
Praxis has an upcoming meeting with the FDA to discuss the results and the New Drug Application (NDA) path.
Topline study results are scheduled to be shared at the American Epilepsy Society Annual Meeting on December 6, 2025.

Dec 4, 2025, 9:10 PM

Press Release

Praxis Discusses Essential One Study Results and Statistical Robustness

PRAX

New Projects/Investments

Product Launch

The Essential One study involved a mid-study change in its primary endpoint, which was submitted and approved by the FDA before the database was locked.
The study demonstrated highly significant results, with the p-value for the primary endpoint at day 56 being in the order of 10^-6. All assessed time points, including the original day 84 endpoint (p-value of 10^-3), also showed significance.
Statistical analyses, including mixed model for repeated measures (MMRM) and various sensitivity tests (tipping point analysis, jump to reference, baseline carried forward), consistently yielded robust and highly significant results, even under stress testing for missing data.
The FDA had previously insisted on using a modified ADL scale for assessment and did not object to the pre-specified maximum penalty of two and a half points in the tipping point analysis.
An integrated analysis of Essential One and Essential Three revealed incredibly consistent effects across studies, resulting in an "insanely small" p-value when combined, reinforcing the drug's benefit.

Nov 24, 2025, 6:00 PM

Transcript

PRAX Discusses Robustness and Statistical Significance of Clinical Trial Results

PRAX

The Essential One study demonstrated highly statistically significant positive results across all assessed time points, with the primary endpoint at day 56 showing a p-value of 10 to the minus 6.
A mid-study change to the primary endpoint in Essential One was made before database lock and unblinding, and was considered a minor shift in assessment time rather than a change in metric. The FDA did not criticize the maximum penalty specified for the tipping point analysis.
Pre-specified sensitivity analyses, including a tipping point analysis for missing data, confirmed the robustness of the results, with the study remaining statistically significant even with a 2.5-point penalty (p-value of 0.0026), which is considered a substantial decrement.
Despite an earlier futility analysis suggesting a potential reason to stop the study, the final results were overwhelmingly statistically significant, which can occur if later data is more optimistic than interim projections.

Nov 24, 2025, 6:00 PM

Transcript

Praxis's Essential Three Program Demonstrates Robust Clinical Trial Results

PRAX

New Projects/Investments

Praxis's Essential Three program utilized a Mixed Model Repeated Measures (MMRM) analysis, confirmed by an independent biostatistics expert as the most appropriate for its longitudinal data and handling of missing data.
The program achieved overwhelming statistical significance with very small p-values across all assessed time points, indicating strong evidence of efficacy.
Sensitivity analyses, including a pre-specified tipping point analysis and jump to reference, demonstrated the robustness of the results even under conservative assumptions for "missing not at random" data, with the tipping point not being reached at a moderate-sized effect.
Despite a prior futility analysis and a last-minute change in the primary endpoint assessment time (made before database lock), the final results were highly positive and statistically significant, with secondary endpoints also consistently positive.

Nov 24, 2025, 6:00 PM

Transcript

Praxis Provides Updates on Essential Tremor and Relutrigine Programs

PRAX

Product Launch

Guidance Update

Praxis anticipates filing an NDA for its essential tremor program early in 2026, following successful readouts from both studies and confirmed FDA alignment on the regulatory pathway, including the use of mADL 11. A pre-NDA meeting with the FDA is scheduled for Q4 2025, with communication on the NDA filing expected approximately 30 days after receiving the meeting minutes.
The company is conducting an interim analysis in Q4 2025 for its relutrigine program for SCN2A and SCN8A, with a potential NDA approval in 2026 and a subsequent sNDA filing for a broader DEE indication based on the Emerald study, which is also expected to read out in 2026.
Praxis estimates the essential tremor market opportunity to be 7 million Americans, with 2-3 million actively seeking treatment, and projects $8 billion-$10 billion in peak sales for its drug, requiring approximately 300 sales representatives for launch. The DEE market opportunity is estimated at $3 billion-$4 billion.