Praxis Precision Medicines Provides Corporate Update and Reports Third Quarter 2025 Financial Results

Announced positive results from two pivotal Phase 3 Essential3 studies of ulixacaltamide HCl in essential tremor (ET); Pre-NDA meeting with FDA scheduled for Q4 2025

Plans to accelerate the development of relutrigine in SCN2A and SCN8A DEE patients after comprehensive Type B meeting with FDA

Recruitment completed for POWER1 study of vormatrigine in focal onset seizures, with topline results expected in 1H 2026

POWER2 study started, with enrollment expected to complete in 2H 2026

Pro forma cash and investments of approximately $956 million, including proceeds from October 2025 public offering, fund operations into 2028

BOSTON, Nov. 05, 2025 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today provided a corporate update and reported financial results for the third quarter 2025.

“It’s been a monumental few months for us at Praxis, culminating in the first ever successful Phase 3 program of an investigational drug in ET, with both studies in the Essential3 program meeting their primary endpoints. Ulixacaltamide demonstrated significant improvements in symptoms that are not only clinically meaningful but also change patients’ lives. We have been granted a pre-NDA meeting with the FDA and look forward to discussing Praxis’ first NDA submission,” said Marcio Souza, president and chief executive officer. “Leveraging our breakthrough therapy designation for relutrigine, we completed comprehensive discussions with the FDA to perform an interim analysis of our EMBOLD study in Q4 which, if successful, supports another potential NDA submission for relutrigine in SCN2A/8A DEEs in early 2026. For our vormatrigine program, following the positive results from the first cohort of the RADIANT study, we have now completed recruitment for POWER1 with expected readout in the first half of 2026, and POWER2 is underway and planning to be fully enrolled in the second half of 2026. With the recently completed public offering, we are well-positioned to continue executing across our robust portfolio as we prepare for our first commercial launch, establishing Praxis as the leader in innovative therapies for CNS disorders.”

Recent Highlights and Anticipated Milestones

Cerebrum™ Small Molecule Platform

• Ulixacaltamide for Essential Tremor (ET): ET is the largest movement disorder affecting approximately seven million people in the U.S. In October 2025, Praxis announced the positive results from the Essential3 program – the first positive Phase 3 program for a drug in ET. During the recruitment phase of the trial, which started in November 2023, over 200,000 patients demonstrated interest in participating in the study, underscoring the unmet need and size of the opportunity.
  • Study 1 (placebo-controlled, parallel group) met its primary and all secondary endpoints:
    • Patients treated with ulixacaltamide in Study 1 showed a mean improvement from baseline in the modified Activities of Daily Living 11 (mADL11) at Week 8, the pre-specified primary endpoint, of 4.3 points (p