Percheron Therapeutics Ends Avicursen Trial: What This Means for Investors
PER (PER) Share Update December 2024 Tuesday 17th
Percheron Therapeutics Halts Avicursen Trial After Disappointing Results
Percheron Therapeutics Limited (ASX: PER) has announced the termination of its phase IIb trial of avicursen in non-ambulant boys with Duchenne muscular dystrophy (DMD) following disappointing results.
Instant Summary:
- Trial did not meet primary endpoint for PUL2.0 score.
- No significant differences in secondary endpoints.
- Avicursen was safe and well-tolerated.
- Study to be terminated; strategic review planned for 2025.
Trial Results
Percheron Therapeutics' phase IIb trial for avicursen, aimed at treating non-ambulant boys with Duchenne muscular dystrophy, did not achieve its primary endpoint. The Performance of the Upper Limb 2.0 (PUL2.0) score showed no significant improvement over placebo. The least squares mean change in PUL2.0 score was -1.4 for placebo, -1.8 for 25mg avicursen, and -1.6 for 50mg avicursen, with p-values indicating no statistical significance.
Secondary endpoints also failed to show any significant efficacy, and there was no clear trend suggesting benefit from avicursen treatment. Despite these results, the drug was found to be safe and well-tolerated, with mild to moderate injection site reactions being the most common adverse event.
Decision to Terminate
In light of these findings, Percheron has decided to terminate the trial. The company, in consultation with investigators, determined that continuing the study was not in the best interests of patients or shareholders. They plan to conduct a strategic review in the first half of 2025 to explore future directions for the avicursen program, particularly considering its favorable safety profile.
Percheron's CEO, Dr. James Garner, expressed disappointment but emphasized the importance of understanding the data and exploring future opportunities. The company remains well-funded, allowing for a systematic approach to future planning.
The termination of the avicursen trial is a setback for Percheron Therapeutics, potentially impacting investor confidence and stock value. The lack of efficacy in the trial results may lead to a reevaluation of the company's pipeline and strategic focus. However, the drug's safety profile could offer future opportunities in other therapeutic areas.
Investor Reaction:
Analysts may express concern over the trial's failure to meet its endpoints, which could influence investor sentiment negatively. However, the company's financial stability and plans for a strategic review may provide some reassurance.
Conclusion:
Investors should closely follow Percheron Therapeutics' upcoming strategic review and any new data releases in 2025. Understanding the company's future plans and potential shifts in focus will be crucial for making informed investment decisions.