- An experimental drug being developed by Biohaven Pharmaceutical did not benefit patients with a rare neurodegenerative disease any more than placebo in a late-stage clinical trial, the Connecticut-based drugmaker said in a Monday statement.
- According to Biohaven, results showed the drug, known as verdiperstat, missed its primary and secondary goals in the Phase 3 study, which enrolled more than 330 people with multiple system atrophy, a rapidly progressing Parkinsonian disease that has no approved therapies.
- Biohaven did not disclose any specific data in its statement on the trial, noting that "additional analyses" are still pending and indicating full results would be presented at an upcoming medical meeting. Shares in the biotech fell by as much as 2.5% Monday morning, before climbing back to trade up nearly 1%.
Biohaven has built itself into a nearly $10 billion company on the strength of its migraine drug Nurtec, which surprised Wall Street with second-quarter sales that were nearly twice as high as expected. The drug, now approved for preventive use as well as treatment, competes with medicines sold by large pharmaceutical companies like AbbVie, Eli Lilly, Amgen and Teva Pharmaceutical.
"A drug like Nurtec gives you the opportunity to fund your future assets and pipeline once you hit profitability, without having to go back to the equity markets," said Biohaven CEO Vlad Coric in a July interview with BioPharma Dive. "This is the kind of drug that can go on to fund a lot more innovation within human neuroscience."
Expectations among analysts generally weren't high for verdiperstat, which Biohaven licensed from AstraZeneca in 2018. Still, Marc Goodman, an analyst at SVB Leerink, recently described the drug as an "underappreciated yet significant" opportunity for the company.
The results disclosed Monday likely close off that opportunity, however. Biohaven said verdiperstat did not "statistically differentiate" from placebo on both the trial's primary efficacy measure and its secondary endpoints.
"While we are disappointed that verdiperstat did not demonstrate efficacy for the treatment of MSA, Biohaven remains committed to fighting on behalf of people living with neurodegenerative diseases," said Irfan Qureshi, the company's head of neurology, in a statement.
Verdiperstat was designed to work by blocking an enzyme thought to be involved in neuroinflammation and cell death. Biohaven sought to prove that it could slow the progression of multiple system atrophy by helping preserve neuronal health.
While that hypothesis did not pan out in multiple system atrophy, the company is also studying verdiperstat as a treatment for ALS, or Lou Gehrig's disease. The drug is one of several being tested in a closely followed "platform trial" of ALS treatments that's being run at Massachusetts General Hospital. Enrollment in the study is expected to be completed in the fourth quarter, Biohaven said.
Several companies, including Biogen, are also testing therapies for multiple system atrophy but only one other drug is currently in Phase 3 trials, according to SVB Leerink's Goodman.