Praxis Achieves Breakthrough in Essential Tremor Treatment with Phase 3 Success

Praxis Precision Medicines has announced positive results from its Essential3 phase 3 program for ulixacaltamide, a potential treatment for essential tremor (ET). The company's persistence in continuing the trials despite earlier setbacks has paid off, with two pivotal studies demonstrating statistically significant improvements in patients' daily activities.

Ulixacaltamide Shows Promising Efficacy in Dual Studies

In Study 1, which involved 473 patients, ulixacaltamide demonstrated a statistically significant and clinically meaningful 4.3-point mean improvement in the modified Activities of Daily Living 11 (mADL11) score at week 8. This improvement was sustained from week 2 through the 12-week dosing period.

Study 2, with 238 patients, focused on maintaining the response achieved by week 8. Results showed that 55% of patients continuing ulixacaltamide maintained their response, compared to only 33% in the placebo group. Additionally, the rate of disease improvement, a key secondary endpoint, met statistical significance.

Innovative Trial Design and Market Implications

Praxis employed a decentralized, home-based study design for both trials, primarily measuring the drug's effects through the mADL11 scale. This approach, adapted from lessons learned in a previous phase 2 trial, appears to have been crucial in demonstrating the drug's efficacy.

The success of ulixacaltamide could have significant market implications. Essential tremor affects approximately seven million people in the U.S., with limited treatment options currently available. The only approved pharmaceutical treatment, propranolol, offers limited efficacy and is contraindicated for many patients.

Financial Impact and Future Plans

The announcement of these positive results had an immediate and dramatic effect on Praxis' stock, which soared more than 220% following the news. This marks a significant turnaround for the company, whose shares had previously plummeted after a grim interim analysis earlier in the year.

Praxis is now moving swiftly to capitalize on these results. 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.

CEO Marcio Souza expressed enthusiasm about the results and the potential impact of ulixacaltamide, stating, "In just 15 months of recruitment, we had over 200,000 people interested in participating in this study, which is a powerful reflection of the large unmet need for a therapy like ulixacaltamide."