Percheron Therapeutics (ASX:PER) has announced that its Phase 2b trial of avicursen (ATL-1102) for Duchenne muscular dystrophy (DMD) has failed to meet its primary endpoint. The announcement led to an 86% drop in the company's share price. The trial, involving 48 wheelchair-bound boys across 16 hospitals in five countries, aimed to assess the efficacy of avicursen in improving upper limb function.
DMD is a regressive, fatal disease affecting approximately 1 in 10,000 males, resulting from a gene mutation that impairs the production of dystrophin, a muscle protein. This deficiency leads to muscle damage, chronic inflammation, and progressive loss of function.
Trial Results and Implications
Avicursen (ATL-1102) was designed to inhibit the VLA-4 protein (CD49d), exerting an immune-modulatory effect. However, the trial results showed no statistically significant differences in efficacy on the primary endpoint, which measured upper limb function at week 25, compared to the placebo group. The company also reported no clear directional trend toward benefit associated with avicursen administration on available secondary endpoints.
According to Percheron, avicursen was safe and well-tolerated during the trial. The company is now considering other potential uses for the drug. The placebo group was re-allocated to the active treatment group after six months to address ethical concerns.
Strategic Review and Future Plans
Percheron Therapeutics will conduct a strategic review of its pipeline in the June half of 2025 to evaluate alternative sources of value within its current assets. The company aims to determine whether there are convincing reasons to continue avicursen development in DMD, while also exploring its potential in other conditions. The board has indicated that if an alternative indication is pursued, it will be crucial to ensure that the negative results in DMD do not negatively impact the chances of success in other illnesses.
Dr. James Garner, Percheron's chief, expressed disappointment with the results, stating, "In the more rigorous environment of a randomised, placebo-controlled, international study, avicursen has not performed in the way that we had been led to expect from earlier studies." Despite the setback, Garner affirmed that the company remains well-funded, with $8 million in cash on hand as of the end of September, following a recent $14.8 million capital raise.
Broader Industry Context
The failure of Percheron's DMD trial underscores the risks inherent in drug development, despite clinical successes observed across the sector in 2024. The company's board of directors acknowledged the disappointment for shareholders and outlined plans to rebuild and restore the company following this setback. They emphasized the importance of understanding the trial results and not prematurely abandoning avicursen, while also exploring alternative indications and diversifying the pipeline.
The board noted that analysis by the Biotechnology Innovation Organization calculates the probability that a drug entering phase I human trials will become a marketed product at 7.9%. They added that aggressive measures are being taken to conserve the company’s cash, including finalizing the termination of the clinical trial and making substantial reductions in operating expenditure.
