Praxis Precision Medicines Reports Significant Phase 3 Success in Essential Tremor—Ulixacaltamide Achieves All Key Endpoints
Ulixacaltamide Demonstrates Robust Efficacy in Both Pivotal Phase 3 Trials
Praxis Precision Medicines (NASDAQ: PRAX) has unveiled compelling topline results from two pivotal Phase 3 studies evaluating ulixacaltamide in the Essential3 program for essential tremor (ET). Both trials met all primary endpoints, underscoring the therapy’s potential to fill a major treatment gap for the estimated seven million people living with ET in the U.S.
Essential3 Study Results Highlight Meaningful Clinical Benefit
In Study 1, patients treated with ulixacaltamide experienced a statistically significant mean improvement of 4.3 points in the Modified Activities of Daily Living 11 (mADL11) scale at Week 8, compared to a 1.7-point improvement in the placebo group (p < 0.0001). All key secondary endpoints, including rate of disease improvement and global impression scales, also reached statistical significance, with results sustained through 12 weeks.
Study 2 reinforced ulixacaltamide’s effectiveness in maintaining benefit, as 55% of patients continued to respond versus 33% for placebo after randomized withdrawal (p = 0.037). These effects were seen alongside a favorable trend across additional efficacy and patient-reported outcomes.
| Endpoint | Ulixacaltamide | Placebo | p-value |
|---|---|---|---|
| Primary Endpoint: Day 56 CFB mADL11 (Study 1) | -4.3 | -1.7 | <0.0001 |
| Rate of Disease Improvement (Study 1) | -4.0 | -1.7 | <0.0001 |
| PGI-C Day 56 (Study 1) | 3.3 | 3.9 | <0.0001 |
| CGI-S CFB to Day 56 (Study 1) | -0.41 | -0.12 | 0.0007 |
| Maintenance of Response (Study 2) | 55% | 33% | 0.037 |
Safety Profile Consistent with Prior Trials—Well Tolerated Across Studies
Ulixacaltamide’s safety profile was in line with previous studies, with most adverse events classified as mild to moderate. There were no drug-related serious adverse events or deaths reported in either trial arm. Common side effects included dizziness, brain fog, and headache, and rates of discontinuation due to side effects were notably higher in the ulixacaltamide group, primarily from dizziness and brain fog.
| Adverse Event Category | Ulixacaltamide (n = 233/231) | Placebo (n = 234) |
|---|---|---|
| Any TEAE | 94.90% / 90.52% | 75.64% |
| Mild TEAEs | 42.06% / 37.66% | 38.03% |
| Moderate TEAEs | 46.78% / 45.45% | 33.33% |
| Severe TEAEs | 6.01% / 7.36% | 4.27% |
| Any SAE* | 0.86% / 1.73% | 3.42% |
| Discontinued from study | 35.62% / 38.10% | 5.56% |
*Serious adverse events not related to study drug
Broad Implications: Addressing Unmet Needs in Essential Tremor
The results underscore ulixacaltamide’s potential as a transformative option for ET patients, many of whom lack effective or tolerable treatments. Currently, only one pharmacotherapy (propranolol) is approved, and its utility is limited. Praxis aims to submit an NDA by early 2026, following the upcoming pre-NDA meeting with the FDA.
The Essential3 trials also broke ground in decentralized study design, enrolling patients across all 50 states with at-home participation—broadening access and generalizability of the data. In a condition where up to half of patients remain untreated, these positive results carry significance beyond numbers.
Key Takeaway: Ulixacaltamide Could Change the Standard of Care for Essential Tremor
The Phase 3 Essential3 program represents a significant step for Praxis and the ET community. Statistically robust, clinically meaningful benefits, and a safety profile consistent with expectations all point to ulixacaltamide’s potential to redefine care for millions of patients living with essential tremor. Investors and healthcare stakeholders will be watching closely as Praxis progresses toward regulatory milestones.
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