Positive data from Essential3 program marks major step toward first targeted therapy for millions living with essential tremor
Praxis Precision Medicines (PRAX) has reported strong topline results from its Phase 3 Essential3 program evaluating ulixacaltamide as a treatment for essential tremor (ET), a chronic neurological disorder affecting an estimated 10 million people in the United States alone. The results bring new hope to patients who have long faced limited and often ineffective treatment options.
In Study 1, a randomized parallel-group trial, patients treated with ulixacaltamide achieved a mean improvement of 4.3 points on the Modified Activities of Daily Living 11 scale at Week 8, a statistically significant outcome (p<0.0001) and the study’s pre-specified primary endpoint. All key secondary endpoints were also met, including rate of disease improvement over 12 weeks, Patient Global Impression of Change (PGI-C), and Clinical Global Impression of Severity (CGI-S), each showing significance at p<0.001.
Study 2, which evaluated maintenance of treatment effect during a randomized withdrawal phase, also met its primary endpoint with ulixacaltamide demonstrating superior durability of effect versus placebo (p=0.0369). The first key secondary endpoint, measuring rate of disease worsening, again favored ulixacaltamide significantly (p=0.0042). Across both studies, the therapy was well tolerated with no drug-related serious adverse events, reinforcing the safety profile observed in prior trials.
Praxis has submitted a pre-NDA (New Drug Application) meeting request to the U.S. Food and Drug Administration, signaling momentum toward potential regulatory approval. President and CEO Marcio Souza emphasized the profound unmet medical need, noting that participants had lived with ET for an average of 30 years and that interest in the study exceeded 200,000 individuals.
Clinical investigators hailed the results as “life-changing” for patients. Dr. Salima Brillman called it the first therapy designed specifically for ET, offering meaningful functional improvements. The studies also featured a home-based virtual trial design, increasing accessibility for a diverse patient population.
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