Wave Life Sciences saw its stock price plunge nearly 60 percent Monday following the company’s decision to discontinue development of its investigational Duchenne muscular dystrophy drug, suvodirsen, after an interim analysis from a Phase 1 study.
Cambridge, MA-based Wave (NASDAQ: [[ticker:WVE]]) was testing suvodirsen as a potential treatment for Duchenne, a genetic neuromuscular disorder that affects approximately one in every 5,000 newborn boys worldwide. The rare disease, which causes progressive muscle weakness, is typically fatal by age 30 as a result of its impact on the heart and lungs.
The news comes in the wake of the FDA’s unexpected approval of the Sarepta Therapeutics (NASDAQ: [[ticker:SRPT]]) Duchenne drug golodirsen (Vyondys 53), which the agency OK’d Friday. The Wave drug was targeting a subgroup of Duchenne patients for whom another Sarepta drug, eteplirsen (Exondys 51), the first-ever approved to treat the disease, was approved for back in 2016.
Wave Life Sciences CEO Paul Bolno called the results “unexpected and deeply disappointing” in a prepared statement issued before markets opened Monday. The company’s stock price, which closed at $37.50 per share Friday, opened at $17.95 apiece.
Like other Duchenne drugs, Wave’s goal with suvodirsen was to raise the levels of a protein called dystrophin in patients with the condition. The mutation that causes the disease results in their making little or none of the protein, which is essential for proper muscle function.
Clinical development of suvodirsen was initiated in November 2017. The results that prompted Wave to end its trials of the drug came from an open-label extension of a Phase 1 trial, which didn’t show any change in dystrophin levels in patients receiving the drug. Wave is also ending a Phase 2/3 trial of the experimental drug that it started in June.
The analysis prompted the company to also cut its preclinical program studying another potential Duchenne drug, WVE-N531, which was also being developed for another subgroup of patients—the same as that targeted by Sarepta’s latest Duchenne drug.
With those programs stopped, Wave said it has enough cash to carry it through the third quarter of 2021. The company is also studying potential treatments for other neurological diseases, including Huntington’s disease (HD). Bolno said Wave anticipates reporting data from a Phase 1b/2a trial of its investigational HD drug by year’s end.
The company also has lead programs in amyotrophic lateral sclerosis and frontotemporal dementia, though it is targeting various genetically defined diseases in multiple therapeutic areas.
(Photo by Jeremy Bishop on Unsplash)