- Biohaven Pharmaceuticals said Thursday its experimental drug verdiperstat failed in a late-stage study of patients with amyotrophic lateral sclerosis, or ALS, marking the second setback for the neurological treatment in a year.
- Researchers found no benefit for the medicine compared to a placebo on disease progression and survival, or on a number of secondary goals evaluating function like muscle strength. Full results from the 24-week study will be presented at a medical meeting in the future, Biohaven said.
- The study failure follows a September 2021 announcement that the drug provided no benefit for patients with a neurological condition called multiple system atrophy. The company described the latest news as disappointing but vowed to continue researching treatments for neurodegenerative diseases.
Even before last year’s setback, analysts expected little from verdiperstat, which Biohaven licensed from AstraZeneca in 2018. The biotech’s top product is Nurtec ODT, a top-selling migraine pill that Pfizer markets outside the U.S. as part of a deal struck in 2021.
In May, Pfizer announced plans to buy Biohaven for $11.6 billion. Pfizer said it planned to hold onto the migraine business and spin off other assets, including verdiperstat, into a new company currently dubbed New Biohaven. The companies expect the transaction to close by early 2023.
The latest research is part of a study being run by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital. Known as the Healey ALS Platform Trial, it’s designed to test multiple experimental medicines at the same time using a common study protocol. Each arm is designed to enroll 160 patients, with 75% of patients getting the experimental drug and the rest a placebo.
Verdiperstat was one of the first drugs to be included in the trial. While verdiperstat wasn’t successful, the platform study offered “a decisive answer with a more efficient approach than traditional stand-alone trials,” Merit Cudkowicz, director of the Healey center, said Thursday. Researchers plan to keep adding experimental drugs to the trial until there are effective treatments found for all patients.
The announcement comes as the ALS community awaits news on another experimental drug from Amylyx Pharmaceuticals, for which the Food and Drug Administration is expected to decide on approval by Thursday. An FDA advisory committee recommended approval of the medicine earlier this month.