Rare Daily Staff
Wave Life Sciences said it will discontinue development of suvodirsen for patients with Duchenne muscular dystrophy after an interim analysis of a phase 1 study showed no indication of efficacy.
The news sent shares of Wave tumbling as they fell more than 50 percent on Monday.
Duchenne muscular dystrophy (DMD) is a fatal, genetic, neuromuscular disorder caused by genetic mutations that result in absent or defective dystrophin protein. Dystrophin protein is needed for normal muscle maintenance and operation. Because of the genetic mutations in DMD, the body cannot produce functional dystrophin, which results in progressive and irreversible loss of muscle function, including the heart and lungs.
Suvodirsen is an experimental stereopure oligonucleotide that was in development for DMD patients who have mutations amenable to exon 51 skipping. The results showed no change from baseline in dystrophin expression, as measured by western blot, with either of two doses. The company said there were no safety concerns or emerging safety signals.
As a result of this decision, the company is immediately discontinuing the two suvodirsen trials, the OLE study and the phase 2/3 DYSTANCE 51 trial. Patients will have a final follow-up visit, but no further doses will be administered, and patients will no longer undergo muscle biopsies. In addition, Wave is suspending further development of WVE-N531 for patients with mutations amenable to exon 53 skipping.
“We set out to restore meaningful levels of dystrophin in patients with Duchenne, and we failed to achieve this goal,” said Michael Panzara, chief medical officer of Wave Life Sciences. “These results are not what we expected, particularly given the promising data from our preclinical models, and we commit to further analyzing and understanding the results to aid in future research.”
Wave said its existing cash and cash equivalents, together with expected and committed cash from existing collaborations, will enable it to fund its operating and capital expenditure requirements into the third quarter of 2021.
The company also has a Huntington’s disease therapy in development and expects to report topline results before the end of 2019.
Photo: Michael Panzara, chief medical officer of Wave Life Sciences