Praxis Precision Medicines (NASDAQ:PRAX) saw its stock soar 100% on Thursday after announcing strong topline data from two pivotal Phase 3 studies of ulixacaltamide HCl, its investigational treatment for essential tremor.
In the parallel-group study (Study 1), patients receiving ulixacaltamide showed a statistically significant mean improvement of 4.3 points from baseline in the Modified Activities of Daily Living 11 at Week 8, meeting the pre-specified primary endpoint. All key secondary endpoints in the study were also statistically significant.
The randomized-withdrawal trial (Study 2) further supported the drug’s efficacy. Fifty-five percent of patients maintained their treatment effect on ulixacaltamide, compared to 33% on placebo, achieving the primary endpoint. The treatment group also demonstrated superior improvement during the withdrawal phase relative to placebo.
“For the first time we have a medication designed specifically for our ET patients,” said Dr. Salima Brillman, co-lead investigator of the program. “Ulixacaltamide represents more than data on a chart – it is a real opportunity to help people regain their independence and improve their daily functioning in meaningful ways.”
The company reported that ulixacaltamide was generally well tolerated, with a safety profile in line with earlier studies and no drug-related serious adverse events. The most common side effects included constipation, dizziness, euphoric mood, brain fog, headache, paraesthesia, and insomnia.
Praxis has submitted a pre-NDA meeting request to the U.S. Food and Drug Administration and expects to file a New Drug Application (NDA) by early 2026, pending agency feedback. The company highlighted that participants in the Essential3 program had been living with essential tremor for an average of 30 years, underscoring the significant unmet medical need.
Praxis Precision Medicines stock price
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