- Wave Life Sciences has announced initiation of animal trials for a Duchenne's muscular dystrophy (DMD) drug candidate. Clinicals trials are expected to begin sometime in the second half of 2017.
- Like Sarepta's eteplirsen, Wave's investigational drug is designed to facilitate production of dystrophin -- except Wave claims its compound is significantly more efficient. Wave's drug also targets boys with the exon 51 mutation, roughly 13% of the DMD patient population.
- Wave's announcement comes days after the Cambridge, MA-based company signed its first big pharma collaboration deal to develop genetic therapies for metabolic conditions. The partnership with Pfizer will give it $40 million upfront, with $871 in potential milestones.
Although BioMarin's DMD drug drisapersen was rejected by the FDA in January and Sarepta's quest to gain approval for eteplirsen hit a major regulatory setback recently, DMD is now attracting other companies.
Cambridge-based Wave focuses on neurological and neuromuscular diseases, with a specific focus on rare diseases. According to the company, Wave's DMD drug candidate was found to be 25 times more efficient in boosting dystrophin production than eteplirsen and drisapersen. However, that finding is based only on preclinical data. Any clinical trial studying the drug's effect in boys with DMD is likely more than a year away.
Wave will first focus on developing an exon-51 skipping treatment, a similar approach to Sarepta's. DMD is linked to specific errors in the gene for production of dystrophin, a key protein in muscle function. By skipping specific exons in the mutated gene, Wave hopes to boost dystrophin production. But the company also hopes its stereopure nucleic acid approach will enable it broaden the reach of its future development work.
“We recognize the acute need of the Duchenne community for therapeutic options to address this devastating disease, and also appreciate regulators’ requirements for strong, well-validated, scientific evidence. Our goal is to fulfill both of these needs," said Paul Bolno, CEO of Wave.
DMD affects roughly 1 in every 3,500 boys, who progressively lose muscle strength, develop heart and lung problems, and eventually become wheelchair bound. BioMarin's drispersen and Sarepta's eteplirsen had been seen as two promising new treatments for the disease. But the Food and Drug Administration rejected drispersen and an approval of eteplirsen looks unlikely after an advisory panel voted against recommending the drug.
Patient- and parent-based testimony of the experiences of boys who have taken Sarepta's eteplirsen suggest the drug helped strengthen the boys' muscles and control progression of the disease during clinical trials.
Hundreds of parents, patient advocates, and boys with DMD packed the panel meeting to make an impassioned case for approval. But the independent panel criticized Sarepta's trial methodology, questioning the reliability of a measure used to gauge effectiveness and the small size (12 boys) of the study.
The FDA is slated to make a final decision by May 26.