Drisapersen fails in Phase III – what’s next for DMD?

In Clinical Development by Tim Peplow

muscle generating stem cells

It’s news that has stunned many. GSK and Prosensa’s experimental orphan drug drisapersen has failed to meet its goal in a Phase III trial. The hugely promising orphan drug for the treatment of Duchenne muscular dystrophy did not improve the distance that the boys could walk in a six minute test compared to placebo.

In the race to develop a drug for the condition, the exon 51 skipping drug drisapersen had been seen as the lead candidate. Following the devastating news, however, Prosensa’s shares crashed by 75%.

Piecing this all back together, what’s next for Duchenne muscular dystrophy?

Well, there still could be a future for drisapersen. GSK and Prosensa reportedly now plan to study the clinical trial results in detail: “We are committed to evaluating the outcome of this study in the context of the overall development program with experts in the field and we expect such evaluation to help inform our next steps for drisapersen,” said Carlo Russo, head of rare diseases research at GSK.

The next drug to look at is Sarepta’s eteplirsen. Also an exon 51 skipping drug, eteplirsen has recently delivered some impressive data in phase IIb trials. But could it be the case that exon 51 skipping doesn’t work for DMD? Does the drisapersen failure work in Sarepta’s favour, or is this bad news for eteplirsen too?

According to the Duchenne Muscular Dystrophy eBook, alongside 11 other organisations with orphan drug designations for DMD, Prosensa does have a number of other experimental therapies for DMD in its pipeline. These include drugs for exon 44, 45, 52, 53 and 55.

Also to look at is PTC Therapeutics’ Ataluren. The candidate is in late stage clinical development for DMD caused by nonsense mutations (nmDMD). The company have initiated a Phase III trial with the drug for the treatment of nmDMD.

Let’s hope for the sake of patients and families affected by DMD that future news is more promising.

You can read more about this story at Reuters >