Shares in Wave Life Sciences sunk 50% Monday morning on the news it is throwing out suvodirsen in Duchenne muscular dystrophy after it failed to move the needle in a key measurement.
The drug, which focused on patients whose mutations are amenable to exon 51 skipping, is a stereopure oligonucleotide and was being tested in two phase 1 and phase 2/3 trials for patients with DMD, which dramatically reduces muscle use and is usually terminal.
A genetic condition, DMD typically occurs with absent or defective dystrophin protein (which is needed for normal muscle maintenance and operation). Alongside other drugs both approved and in testing, Wave was looking to boost this protein, but said in a brief update that: “The results showed no change from baseline in dystrophin expression, as measured by western blot, with either the 3.5 mg/kg or 5 mg/kg doses of suvodirsen.”
Testing of the drug will now be canned. “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,” the company said.
“The suvodirsen results are unexpected and deeply disappointing to us, and undoubtedly will be to the patients we aim to serve. We are grateful to be part of the Duchenne community and our organization has been shaped by their strength and resilience,” said chief Paul Bolno, M.D.
“While we did not achieve dystrophin restoration in this study, there is a rising tide in nucleic acid therapeutics, and we are fully committed to advancing genetic medicines in diseases of the central nervous system, eye and liver. We will work to rapidly incorporate our learnings, so that we can seek to deliver on the promise of our current and future pipeline.”
The next big data report is mere days away, as biotech is slated to report topline results from its so-called PRECISION-HD2 trial in Huntington’s disease “by the end of 2019.” It will be hoping for better news as today, its shares were more than halved on the DMD flops and termination.
Analysts at Jefferies said in a note to clients that this was “a surprise as the Street debate was largely on the level of efficacy.” The firm said that while “a clear negative,” if the HD programs succeed this could help reverse it misfortune.