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Highlights
- Percheron Therapeutics (PER) announces Phase IIb trial results of avicursen in DMD.
- The trial did not meet its primary efficacy endpoint but confirmed safety and tolerability.
- Further data expected in early 2025 to guide the drug's development strategy.
Percheron Therapeutics (ASX:PER) recently revealed topline results from its six-month international Phase IIb randomised, placebo-controlled trial of avicursen (ATL1102), a potential treatment for Duchenne muscular dystrophy (DMD) in non-ambulant boys. Despite high expectations set by earlier studies, the latest trial did not show significant therapeutic benefits, marking a setback for the program.
The study primarily assessed the Performance of the Upper Limb (PUL2.0) score at week 25 compared to placebo. Unfortunately, no statistically significant differences were observed. Patients in the placebo group experienced a mean score change of -1.4, while those treated with avicursen at doses of 25 mg and 50 mg recorded changes of -1.8 and -1.6, respectively. The p-values of 0.695 and 0.919 for these results indicate a lack of statistical significance. Secondary endpoints also failed to demonstrate efficacy, and no directional trend favoring avicursen emerged.
Despite these results, the drug was well-tolerated, showing a favorable safety profile. Following consultations with investigators and advisors, Percheron has decided not to proceed further with the trial. The company, however, intends to conduct a detailed analysis of the existing data, as well as additional data expected in January 2025, to evaluate potential next steps for the avicursen program.
In a strategic review planned for the first half of 2025, Percheron aims to assess its drug development pipeline and chart the future course for avicursen. The company’s leadership emphasized the importance of using this opportunity to refine its approach while remaining committed to addressing the significant unmet needs in DMD treatment.
CEO and Managing Director Dr. James Garner expressed gratitude to the study’s investigators, healthcare professionals, and families for their trust and participation. He acknowledged the challenges posed by DMD and reiterated the company’s commitment to advancing therapeutic understanding for this complex disease.
Percheron also highlighted its robust financial position, enabling a systematic and methodical exploration of future opportunities. The company plans to share updated strategies and plans with stakeholders after analyzing the upcoming data in early 2025.
