Percheron Therapeutics Ltd (ASX:PER, OTC:PERCF) has announced topline six-month results from the company’s ongoing international phase IIb randomised, placebo-controlled trial of avicursen (ATL1102) in non-ambulant boys with Duchenne muscular dystrophy (DMD).
“We are of course disappointed by these results”, said Percheron CEO and MD Dr James Garner.
“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 by earlier studies.
“The trial has not demonstrated therapeutic benefit in non-ambulant DMD.”
Trial results
The trial determined that 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. However, the drug was safe and well-tolerated.
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.
Percheron advises that after careful consideration of the results, and in consultation with investigators, it has decided to not continue the study.
Working closely with investigators and advisors, Percheron says it will examine the data received and further data expected in January, to determine the best path forward for the avicursen program — noting the drug’s favourable safety profile in particular.
The company expects to hold a strategic review of its pipeline in 1H CY2025 and advises that it will share further information with investors once available.
More information to come
“We anticipate further data from this study in January 2025 and will be examining it closely to better understand this outcome and to assess future opportunities,” said Garner.
“The company is well-funded and so we can approach these discussions systematically and methodically. We expect to be able to share further information and plans with investors in the new year.
“In the meantime, I want to pay tribute to the investigators, healthcare professionals and families that have placed their trust in us by participating in this study.
“Duchenne muscular dystrophy is an immensely challenging illness, and the need for new therapeutic options remains substantial. Although these results may not be as we had hoped, we are proud to have played a small role in advancing understanding of the disease, and in bringing hope to those affected by it.”