Praxis Precision Medicines Achieves Breakthrough in Essential Tremor Treatment

Praxis Precision Medicines has reported significant success in its phase 3 clinical trials for ulixacaltamide, a potential treatment for essential tremor (ET). The company's perseverance in the face of earlier setbacks has paid off, with positive results from two pivotal studies in its Essential3 phase 3 program.

Successful Phase 3 Results

The trials, which utilized a decentralized, home-based study design, primarily assessed the drug's efficacy using the modified activities of daily living 11 (mADL11) scale. This measure evaluates tremor impact on 11 specific daily activities.

In Study 1, involving 473 patients, ulixacaltamide demonstrated a statistically significant and clinically meaningful 4.3-point mean improvement in the mADL11 score at week 8. This improvement was sustained from week 2 through the 12-week dosing period.

Study 2, which enrolled 238 patients, showed that 55% of patients continuing ulixacaltamide treatment maintained their response at week 12, compared to 33% in the placebo group. Additionally, the rate of disease improvement, a key secondary endpoint, met statistical significance.

Market Impact and Future Plans

The positive trial outcomes have had a dramatic effect on Praxis' stock, which surged more than 220% following the announcement. This marks a significant turnaround from the company's previously grim outlook earlier in the year.

Praxis CEO Marcio Souza expressed enthusiasm about the results, highlighting the substantial unmet need for ET treatment. The company has submitted a meeting request with the FDA to discuss a New Drug Application (NDA), which it plans to file by early 2026, pending agreement with the agency.

Addressing an Unmet Medical Need

Essential tremor, affecting approximately seven million people in the U.S., is the most common movement disorder. Currently, the only approved pharmaceutical treatment is the beta-blocker propranolol, which has limited efficacy and is contraindicated for many patients.

Ulixacaltamide, a selective inhibitor of T-type calcium channels, is designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical (CTC) circuit associated with tremor activity. If approved, it could provide a much-needed alternative for ET patients.