Phase IIb Results: Avicursen in Duchenne Muscular Dystrophy

Key Points
• The trial did not meet its primary endpoint, which was Performance of the Upper Limb 2.0 (PUL2.0) score at week 25 compared to placebo. The least squares mean change in PUL2.0 score for patients receiving placebo was -1.4, for patients receiving 25mg of avicursen was -1.8 (p=0.695), and for patients receiving 50mg of avicursen was -1.6 (p=0.919). A p-value above 0.05 means that any numerical difference observed is not statistically significant.
• There were no statistically significant differences in efficacy on available secondary endpoints, nor was there a clear directional trend toward benefit associated with administration of avicursen.                                                       • The drug was safe and well-tolerated, with injection site reactions the most common treatment-emergent adverse event. Injection site reactions were more common at the 50mg dose than at the 25mg dose, but all were considered mild or moderate by investigators.
• After careful consideration of these results, and in consultation with investigators, the Company has determined that it is not in the best interests of patients or shareholders for the study to continue and has therefore resolved to terminate it as soon as practicable.
• The Percheron team, working closely with investigators and advisors, will examine this data, along with further data expected in January 2025, to determine the best path forward for the avicursen program, noting in particular the drug’s favourable safety profile. The Company expects to hold a strategic review of its pipeline in H1 CY2025 and will share further information with investors as it becomes available