Percheron Therapeutics - Clinical Trial Update on Avicursen for Duchenne Muscular Dystrophy
PER (PER) Share Update December 2024 Sunday 1st
Percheron Therapeutics Awaits Key Data from Duchenne Muscular Dystrophy Trial
Percheron Therapeutics Limited (ASX: PER) is on the brink of a significant milestone as it prepares to release initial topline data from its phase IIb clinical trial of avicursen (ATL1102) in patients with Duchenne muscular dystrophy.
Instant Summary:
- All 48 patients completed week 25 visit for primary endpoint assessment.
- Initial topline data expected during the week of 16 December 2024.
- Twelve-month data anticipated mid-2025, with final data in 2H 2025.
- Phase IIb trial involves non-ambulant boys across 13 international sites.
Clinical Trial Progress
Percheron Therapeutics has announced that all 48 patients enrolled in its phase IIb clinical trial for avicursen in Duchenne muscular dystrophy have completed their week 25 visit. This visit marks the point at which the primary endpoint of the study is assessed. The company is currently working on confirming and validating the data, as well as performing the necessary statistical analyses.
The company expects to release initial topline data, representing the first six months of study for all patients, during the week beginning 16 December 2024. This data will provide the first indication of avicursen's activity in treating Duchenne muscular dystrophy, a rare and severe muscle-wasting disease.
Study Design and Expectations
The ongoing phase IIb study is a randomized, placebo-controlled trial, considered the 'gold standard' in clinical research. It involves non-ambulant boys whose disease has progressed to a stage where they are substantially unable to walk. These patients represent about half of the total Duchenne population. The trial is being conducted at 13 sites across Australia, the United Kingdom, Turkey, Bulgaria, and Serbia.
Percheron previously conducted a phase IIa pilot study, which reported encouraging results and was published in a peer-reviewed journal. The company has received orphan drug designation and rare pediatric disease designation from the US FDA for avicursen.
The upcoming data release is a critical milestone for Percheron Therapeutics and could significantly impact the company's stock. Positive results could boost investor confidence and drive the stock price higher, while negative or inconclusive results might lead to a decline. The broader market may also react to the news, given the potential implications for treatments of rare diseases.
Investor Reaction:
Analysts are likely to closely watch the data release, as it will provide crucial insights into avicursen's efficacy and safety profile. A positive readout could lead to increased interest from investors and potential partners, while any setbacks may prompt caution.
Conclusion:
Investors should stay tuned for the initial topline data release in mid-December 2024, as it could have significant implications for Percheron Therapeutics' future. Monitoring the company's progress and strategic decisions will be essential for those invested in or considering investment in the biotechnology sector.