Disappointing results from phase 2 clinical trial for Duchenne muscular dystrophy

19 December 2024

Percheron Therapeutics has announced high level results from its phase 2 clinical trial of avicursen (ATL1102). The trial failed to show any differences between avicursen and the placebo suggesting it may not be beneficial for non-ambulant boys with Duchenne muscular dystrophy.

The phase 2 clinical trial tested to see if avicursen was safe and effective for non-ambulant boys (boys who are unable to walk independently) with Duchenne muscular dystrophy (DMD). Avicursen aims to reduce muscle damage found in DMD by reducing inflammation. While there were no concerns about its safety, no improvement was observed in participants who received avicursen compared to those who received the placebo (dummy drug) after six months.   

At the end of the six month trial, all participants were given the option to take avicursen for a further 6 months, regardless of whether they originally received the treatment or placebo. This is called an open label extension. Due to these disappointing results, the company have decided to stop the open label extension as soon as practical.

The company has committed to continue to analyse the data to help decide the next steps for the development of avicursen. While this is not the outcome we wanted, the learnings from this trial will help the search for effective treatments for DMD.

If your child has been involved in the trial, we recommend you contact the study site for further information. You can also find out more information in a press release.