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Investing.com -- Praxis Precision Medicines (NASDAQ:PRAX) stock surged 100% after the company announced positive topline results from two pivotal Phase 3 studies of its essential tremor treatment ulixacaltamide HCl.
The Boston-based biopharmaceutical company reported that patients treated with ulixacaltamide in the parallel-group study (Study 1) 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 Study 1 were also statistically significant.
In Study 2, a randomized-withdrawal trial, the drug met its pre-specified primary endpoint with 55% of patients maintaining treatment effect while on ulixacaltamide versus 33% on placebo. The rate of disease improvement during the randomized-withdrawal phase also demonstrated superior effect in ulixacaltamide-treated patients compared 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 consistent with previous trials 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 FDA and plans to submit the New Drug Application by early 2026, pending agreement with the agency. The company noted that patients in the Essential3 program had been living with essential tremor for an average of 30 years, highlighting the significant unmet need for effective treatments.
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