Key Takeaways
- Praxis Precision Medicines has received a recommendation to stop a phase 3 essential tremor trial due to futility, with interim analysis showing no significant difference between ulixacaltamide and placebo.
- Despite the setback, Praxis plans to continue both late-stage trials, citing advanced enrollment and feedback from the monitoring committee regarding alternative analysis options.
- The company’s stock price fell more than 40% following the announcement, reflecting investor concerns over the trial’s viability.
Investment in Research Continues Despite Setbacks
Praxis Precision Medicines has faced unexpected challenges with its phase 3 trial for ulixacaltamide, a treatment for essential tremor. An independent data monitoring committee conducted an interim analysis and advised Praxis to halt the trial due to concerns that the study would not meet its primary endpoint. This development surfaces two months after CEO Marcio Souza confidently stated that the probability of futility was “less than 3%.”
The phase 3 trial is one of two late-stage studies being conducted on ulixacaltamide. In the trial, participants were assigned to either the study drug or a placebo for a duration of 12 weeks. The primary endpoint was chosen based on a failed phase 2 trial and focuses on a daily living scale, which assesses how essential tremor affects a patient’s ability to carry out routine tasks like dressing.
Initial findings from the analysis indicated that patients treated with ulixacaltamide showed no improvement compared to those receiving the placebo. As a result, the monitoring committee recommended discontinuing the trial for futility. However, Praxis has decided to proceed with both late-stage studies. The company argues that enrollment in these trials is already advanced and noted the committee’s feedback, suggesting that “some underlying assumptions of the statistical model might have influenced” the futility assessment. The committee also encouraged Praxis to explore alternative statistical methods for analysis.
Looking ahead, Praxis anticipates reporting topline data from both trials in the third quarter of the year. After the final results are analyzed, the company will determine whether to file for regulatory approval. The second trial has a different design that allows for an evaluation of patients’ ability to maintain their responses after being randomized to receive either ulixacaltamide or placebo. This follows a previous trial wherein patients experienced a relapse after discontinuation of the therapy.
Prior to the interim analysis, Praxis was optimistic about the trial’s potential, with Souza and CFO Tim Kelly emphasizing the low probability of futility at various events. Their confidence stemmed from awareness of the options available to the monitoring committee, which could have included adding more participants if needed to strengthen the study.
In the wake of the committee’s recommendation, investor reactions were swift, with shares of Praxis dropping over 40%, now trading around $38 in premarket trading. As of late 2024, Praxis holds $469.5 million in cash, positioning the company to navigate these trial challenges and continue its research efforts.
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