BOSTON, Massachusetts — October 16, 2025 — Leads & Copy — Praxis Precision Medicines, Inc. (NASDAQ: PRAX) reported positive topline results from its Phase 3 Essential3 program of ulixacaltamide for essential tremor (ET). Patients treated with ulixacaltamide in Study 1 showed a mean improvement of 4.3 points (p<0.0001) from baseline in the Modified Activities of Daily Living 11 at Week 8, the primary endpoint.
All key secondary endpoints in Study 1, including the rate of disease improvement over 12 weeks, PGI-C, and CGI-S, were statistically significant (p<0.001). Study 2 met its primary endpoint, with patients on ulixacaltamide showing superior maintenance of effect versus placebo during the randomized-withdrawal phase (p=0.0369). The first key secondary endpoint in Study 2, the rate of disease improvement during the randomized-withdrawal phase, also demonstrated a superior effect in ulixacaltamide-treated patients versus placebo (p=0.0042).
Ulixacaltamide was generally well-tolerated, with a safety profile consistent with previous trials and no drug-related serious adverse events. Praxis has submitted a pre-NDA meeting request to the FDA and will host an investor call today, October 16, at 8 a.m. EST.
Marcio Souza, president and chief executive officer of Praxis, expressed excitement about the results and gratitude to the patients who participated in the program. Salima Brillman, MD, co-lead investigator of the E3 program, highlighted the potential of ulixacaltamide to change lives and help people regain their independence. Jill Farmer, DO, MPH, FCPP, DipABLM, BoroNeuro, co-lead investigator of the E3 program, emphasized the trial’s innovative home-based design and its reflection of the diversity of the ET community.
Alexander Shtilbans, MD, PhD, FAAN, co-lead investigator, is encouraged by the clinically meaningful improvements in tremor control and daily function. Patrick McCartney, Executive Director of the International Essential Tremor Foundation, said the results mark an extraordinary step forward for the ET community.
Investor Contact: Praxis Precision Medicines investors@praxismedicines.com 857-702-9452
Media Contact: Dan Ferry Life Science Advisors Daniel@lifesciadvisors.com 617-430-7576
Source: Praxis Precision Medicines, Inc.
