Percheron Therapeutics Ltd (ASX:PER, OTC:PERCF) is preparing to publish initial topline data from its Phase 2b, placebo-controlled trial of avicursen (ATL1102) in Duchenne muscular dystrophy after all 48 patients reached the primary endpoint of the study, completing their week-25 visit.
The company is now working to validate and confirm the trial’s data, performing the requisite statistical analysis before results for all patients from the first six months can be released – PER is targeting December 16 as a release date.
Major development milestone
“We eagerly await the six-month data,” Percheron CEO Dr James Garner said.
“This readout will provide the first indication of avicursen’s activity in Duchenne and will help us to better chart its path forward toward registration and commercialisation.
“This is only the first of three major readouts from the study and we will learn much more as we progress through CY2025, but we nevertheless see this as a major milestone in the development of the drug.
“The team has worked hard to generate results as quickly as possible and this is reflected in the fact that the data is currently anticipated just a few weeks after the final patient attended the relevant hospital visit.
“A significant amount of work remains to be done and we will update the market if our expectations change, but we currently expect to be able to discuss data during the week beginning December 16, 2024.”
Percheron expects the full 12-month results to be available by the middle of 2025 and the final 16-month data in the second half of 2025.
PER’s Phase 2b study is focused on non-ambulant boys, whose disease has progressed to the stage where they are substantially unable to walk, and who represent approximately half of the total Duchenne population.
High unmet need
Duchenne muscular dystrophy is a rare, inherited muscle disorder that causes progressive muscle weakness and wasting, eventually resulting in the deterioration of the skeletal, heart and lung muscles.
The disease mostly effects those with only one X chromosome (predominantly male children), as it’s passed via that route.
Life expectancy for someone born with the disease is between 22 and 28 years of age, with outcomes slowly improving over time – about 6 in 100,000 individuals are diagnosed with Duchenne in the US each year.
Percheron is advancing its lead program, avicursen (ATL1102), an antisense oligonucleotide targeting the CD49d receptor, to treat the disease.
PER previously reported promising results from an exploratory Phase 2a study of avicursen in non-ambulant boys and has been awarded orphan drug designation (ODD) and rare pediatric disease designation (RPDD) by the US FDA.