Ulixacaltamide Shows Robust Efficacy and Tolerability in Phase 3 Essential Tremor Trials—Praxis Targets FDA Submission in 2026
Key Takeaway: Both Primary and Secondary Endpoints Exceeded Expectations in Over 700 Patients
Praxis Precision Medicines (NASDAQ: PRAX) revealed today that ulixacaltamide, its lead investigational treatment for essential tremor (ET), met all primary and key secondary endpoints in two pivotal Phase 3 studies. These results, coming from one of the largest-ever decentralized clinical programs for ET, support Praxis’s ambition to file for FDA approval in early 2026.
Statistically Significant Benefits for Essential Tremor—What the Data Reveals
Study 1 enrolled 473 patients, using a double-blind, placebo-controlled design to assess ulixacaltamide over 12 weeks. The main outcome was the mean improvement in the Modified Activities of Daily Living 11 (mADL11) at Week 8, where patients receiving ulixacaltamide achieved a 4.3-point mean improvement—compared to 1.7 points for placebo (p<0.0001). Every key secondary endpoint, including the rate of disease improvement and global impression scores, also showed statistically significant improvements favoring ulixacaltamide.
| Endpoint | Ulixacaltamide | Placebo | p-value |
|---|---|---|---|
| Mean Improvement mADL11 (Day 56) | -4.3 | -1.7 | <0.0001 |
| Rate of Disease Improvement | -4.0 | -1.7 | <0.0001 |
| PGI-C (Day 56, lower better) | 3.3 | 3.9 | <0.0001 |
| CGI-S Change (to Day 56) | -0.41 | -0.12 | 0.0007 |
Study 2 adopted a randomized-withdrawal design to examine if improvements were maintained over an additional four weeks. Ulixacaltamide-treated patients were nearly twice as likely as placebo to sustain benefits: 55% vs. 33% (p=0.037). Secondary endpoints continued to trend in favor of active treatment.
| Endpoint | Ulixacaltamide | Placebo | p-value |
|---|---|---|---|
| Maintenance of Response | 55% | 33% | 0.037 |
| Rate of Disease Improvement | 2.8 | 5.2 | 0.004 |
| PGI-C (Day 84) | 3.24 | 3.67 | 0.087 |
| CGI-S (Day 56–84) | 0.39 | 0.73 | 0.055 |
Tolerability Profile Remains Consistent With Previous Studies
Ulixacaltamide’s safety was broadly reassuring. Across both studies, the drug was generally well tolerated. Common adverse events were mostly mild to moderate and included constipation, dizziness, euphoric mood, brain fog, headache, paraesthesia, and insomnia. Crucially, there were no drug-related serious adverse events and no deaths. Most discontinuations were attributed to known, non-life-threatening side effects like dizziness and brain fog.
| Category | Ulixacaltamide Study 1 | Placebo Study 1 | Ulixacaltamide Study 2 |
|---|---|---|---|
| Any TEAE | 94.9% | 75.6% | 90.5% |
| Mild TEAE | 42.0% | 38.0% | 37.7% |
| Moderate TEAE | 46.8% | 33.3% | 45.5% |
| Severe TEAE | 6.0% | 4.3% | 7.4% |
| Any SAE* | 0.86% | 3.4% | 1.73% |
| Discontinuations (drug-related) | 27.0% | 1.7% | 28.1% |
*SAEs were not considered related to study drug
Patient Need, Market Potential, and What’s Next
Essential tremor impacts nearly seven million Americans, and treatment options remain limited—propranolol is currently the only approved therapy, but with poor tolerability and modest efficacy. Praxis’s approach, leveraging a selective T-type calcium channel blocker designed to target the underlying neural circuit, represents the first drug developed specifically for ET. With up to 85% of neurologist visits related to seeking treatment and 40% of patients currently not receiving therapy, market demand for effective options is substantial.
Praxis has already initiated discussions with the FDA through a pre-NDA meeting request, with full submission anticipated in early 2026. Additional data is expected to be presented at upcoming conferences, with the company emphasizing its patient-centered clinical design and large trial enrollment (over 700 patients) as signs of broad market relevance.
Bottom Line: Pivotal Results Set Up Ulixacaltamide as a Major Contender in Essential Tremor
The topline Phase 3 data puts Praxis in a strong position as it eyes regulatory submission for ulixacaltamide. Statistically robust efficacy, maintained benefits, and a favorable safety profile could set a new standard of care for ET—potentially unlocking a multi-billion dollar commercial opportunity in a field hungry for new solutions.
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