Praxis Precision Medicines reported that its drug ulixacaltamide failed to meet the primary efficacy endpoint in a Phase III clinical trial for essential tremor28.
The company's stock price plummeted by approximately 40-48% following the announcement of the trial results18.
An Independent Data Monitoring Committee (IDMC) recommended stopping Study 1 of the Essential3 program due to the unlikelihood of meeting the primary efficacy endpoint8.
Despite the setback, Praxis plans to complete both studies in the Essential3 program, with topline results expected in the third quarter of 202528.
Ulixacaltamide is a selective T-type calcium channel blocker designed to treat essential tremor, the most common movement disorder affecting approximately 7 million people in the U.S.47.
The company maintains a strong financial position with $469.5 million in cash, which is expected to fund operations into 202889.
Praxis is also exploring alternative analysis methods for the final dataset, as suggested by the IDMC89.
The decision about whether to submit a New Drug Application (NDA) to the FDA will be made after analyzing the final results of both Study 1 and Study 28.
Sources:
1. https://www.biospace.com/praxis-shares-fall-42-percent-following-mixed-data-for-essential-tremor-candidate
2. https://www.clinicaltrialsarena.com/news/praxis-precision-continues-with-et-trial-despite-likely-failure-warning/
4. https://www.biopharmadive.com/news/praxis-tremor-trial-results/644117/
7. https://www.fiercebiotech.com/biotech/praxis-shaken-essential-tremor-fail-still-plans-phase-3
8. https://www.benzinga.com/25/03/44087912/analyst-cut-price-targets-for-praxis-precision-after-trial-setback-for-essential-tremor-candidate
9. https://medcitynews.com/2025/03/essential-tremor-praxis-precision-medicines-ulixacaltamide-neurological-prax/