Percheron Therapeutics has announced the termination of its Phase 2b clinical trial of avicursen (ATL1102) for Duchenne muscular dystrophy (DMD) after the drug failed to meet its primary endpoint. The trial, conducted in Australia and Europe, involved non-ambulatory individuals with DMD. Topline 6-month data indicated no clear trends toward benefits on secondary outcomes, leading to the decision to discontinue the trial.
The study assessed avicursen's efficacy and safety in patients with Duchenne muscular dystrophy. Avicursen, an antisense oligonucleotide, targets the insulin-like growth factor-1 receptor (IGF-1R). The primary endpoint was not met, and further analysis revealed no significant efficacy benefits. Despite the lack of efficacy, avicursen demonstrated a favorable safety profile throughout the trial.
"The company did not observe any significant efficacy benefits," Percheron Therapeutics stated in a press release. The trial's failure represents a setback in the development of new treatments for Duchenne muscular dystrophy, a progressive and debilitating genetic disorder affecting primarily males.
Percheron Therapeutics plans to share more detailed analyses from the trial in 2025. The company will also re-evaluate its pipeline and strategic direction in light of these results. Investors and the Duchenne community await further updates as Percheron determines its next steps.
Duchenne muscular dystrophy is characterized by progressive muscle degeneration and weakness due to alterations in the dystrophin gene. Current treatments focus on managing symptoms and slowing disease progression, highlighting the urgent need for more effective therapies. The discontinuation of the avicursen trial underscores the challenges in developing successful treatments for this complex condition.
