Praxis Precision Medicines (PRAX) Company Report

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.

13 hours ago

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.

Nov 18, 2025, 1:30 PM

Transcript

Praxis Provides Updates on Essential Tremor and Epilepsy Programs

PRAX

Product Launch

New Projects/Investments

Guidance Update

Praxis anticipates filing a New Drug Application (NDA) for its essential tremor program early in 2026, following successful study readouts and a scheduled pre-NDA meeting with the FDA in Q4 2025.
The essential tremor drug targets a market of 7 million Americans, with an estimated $8 billion to $10 billion in peak sales, and is projected to launch in approximately 12 months.
An interim analysis for the relutrigine program, targeting rare epilepsy indications SCN2A and SCN8A, is being conducted in Q4 2025, with a potential NDA filing if successful.
The larger Emerald study for relutrigine, addressing a broader DEE indication, is expected to read out in 2026, representing a $3 billion to $4 billion opportunity and potentially becoming the first approved drug for DEEs in the U.S..

Nov 18, 2025, 1:30 PM

Transcript

Praxis Precision Medicines Prepares for Two Potential NDA Filings and Highlights Significant Market Opportunities

PRAX

Product Launch

New Projects/Investments

Guidance Update

Praxis Precision Medicines is preparing for two potential New Drug Application (NDA) filings: one for ulixacaltamide (Essential Tremor program) in the next few months, and another for SCN2A (EMBOLD program) early next year, potentially within three months.
The company has an upcoming FDA meeting for ulixacaltamide to confirm the adequacy of the data package for NDA submission. For the SCN2A program, the FDA has agreed to an interim analysis with a 4% alpha level, with the intent to file an NDA based on positive results.
Praxis highlights a substantial market opportunity for ulixacaltamide, estimating 7 million Americans live with essential tremor, with 2 million actively seeking treatment. Peak sales are projected to be at least $10 billion in the US, potentially $13 billion-$15 billion including ex-US.
The market for SCN2A and 8A is estimated to be at least $500 million at launch, with a most likely scenario of over $1 billion.
Commercial preparations for ulixacaltamide include building a database of 1 million patients before launch, with a projected $1 billion run rate in the first six months to one year post-launch.

Nov 10, 2025, 2:30 PM

Transcript

Praxis discusses accelerated Relutrigine pathway and Ulixacaltamide Phase 3 success

PRAX

New Projects/Investments

Guidance Update

Revenue Acceleration/Inflection

Praxis is pursuing an accelerated pathway for Relutrigine, with an interim analysis for the Evolve study expected this quarter, potentially leading to a quick New Drug Application (NDA) submission.
This accelerated pathway for Relutrigine makes a pediatric review voucher (PRV) "more than ever possible" and shortens the timeline for the broader multi-billion dollar market opportunity for the Emerald population.
Management believes the Relutrigine opportunity for the SCN2A and SCN8A populations is a multi-billion dollar opportunity, with a starting estimate of $500 million to $1 billion.
Ulixacaltamide had a successful Phase 3, and the company is awaiting meeting minutes from a pre-NDA meeting to provide perspectives on the outcome.

Nov 7, 2025, 5:30 PM

Transcript

Praxis Precision Medicines Reports Q3 2025 Financial Results and Positive Phase 3 Ulixacaltamide Data

PRAX

Earnings

Guidance Update

New Projects/Investments

Praxis Precision Medicines reported a net loss of $73.9 million for the three months ended September 30, 2025, with research and development expenses increasing to $65.8 million.
The company announced positive results from two pivotal Phase 3 Essential3 studies of ulixacaltamide HCl in essential tremor, with a pre-NDA meeting with the FDA scheduled for Q4 2025.
Praxis completed a public offering in October 2025, generating approximately $567.0 million in net proceeds, resulting in pro forma cash and investments of approximately $956 million expected to fund operations into 2028.
Recruitment for the POWER1 study of vormatrigine in focal onset seizures is complete, with topline results expected in 1H 2026, and an interim analysis for relutrigine's EMBOLD study is planned for Q4 2025.

Nov 5, 2025, 1:03 PM

8-K

Praxis Precision Medicines Announces FDA Meeting for Ulixacaltamide and Pipeline Progress

PRAX

New Projects/Investments

Guidance Update

Praxis Precision Medicines, Inc. announced that the FDA granted a Type B meeting for ulixacaltamide to take place in the fourth quarter of 2025, following the review of Essential3 topline results.
The ulixacaltamide Essential3 Study 1 met its primary and all key secondary efficacy endpoints, demonstrating significant improvement in mADL11. However, 27.0% of ulixacaltamide participants in Study 1 experienced drug-related treatment-emergent adverse events (TEAEs) leading to discontinuation, compared to 1.7% for placebo.
Key upcoming clinical catalysts include full cohort topline results for vormatrigine's RADIANT Phase 2 study in Q4 2025, relutrigine's EMBOLD Cohort 2 topline results in 1H 2026, and elsunersen's EMBRAVE Phase 1/2 topline results in 1H 2026.
The company maintains a cash runway into 2028.

Oct 21, 2025, 12:02 PM

8-K

Earnings Transcripts

Quarterly earnings call transcripts for Praxis Precision Medicines.

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