In a press release published earlier, Wave Life Sciences has announced positive results of the phase 1 trial of the WVE-210201 – a potential drug that induces skipping of exon 51 of the dystrophin gene and is designed to restore production of a smaller, but functional dystrophin protein. 

The early-stage phase 1 trial was designed to test the safety and tolerability of different doses of WVE-210201 in people living with Duchenne. The results show the potential drug is safe and well tolerated and importantly, the trial's independent Safety Monitoring Committee has approved the testing of a higher dose.

Wave has used these results to select a dose for the larger phase 2/3 clinical trial they plan to start in 2019. The trial will be a global, placebo-controlled study that is designed to test both the effectiveness and safety of the potential drug in a larger number of people living with Duchenne. It will include sites in the UK and we'll bring more news when the trial starts.

WVE-210201 is also currently being studied in an ongoing open-label extension study, which started in August. Participants who complete the phase 1 trial are able to join the extension study (which has no placebo group) and the company hopes to announce further results from that study in the second half of 2019.

Paul Bolno, President and Chief Executive Officer of Wave Life Sciences said earlier “These results mark an important milestone for the Duchenne community and toward our goal of inducing meaningful, natural dystrophin expression in boys with DMD who are amenable to exon 51 skipping”.

Find out more

Contact Neil Bennett, Director of Research, to find out more on 020 7250 8240

More about potential therapies for Duchenne