Biohaven's Nurtec-less pipeline takes another gut punch, fails ALS trial

When Biohaven sold its migraine franchise to Pfizer for more than $11 billion in May, there was momentum-fueled hope that another gem was tucked somewhere in the Connecticut biotech’s pipeline.

But, almost five months later, the company has gone 0 for 2 in trial readouts, the second of which came Thursday when Biohaven announced verdiperstat failed to improve disease progression in patients with amyotrophic lateral sclerosis (ALS) compared to placebo. The myeloperoxidase enzyme inhibitor also didn’t statistically improve secondary endpoints, which included respiratory function, muscle strength and survival.

The company said it would provide a more detailed analysis of the data at an upcoming conference. 

Given that verdiperstat was only indicated to treat ALS, the failure would naturally throw into question the future of the oral tablet. But in an email to Fierce Biotech, Biohaven CEO Vlad Coric, M.D., said the company wasn’t dumping the asset yet.

“We will assess verdiperstat for other indications but have no immediate plans to announce at this time,” Coric wrote. The company said in a release that it “remains committed to developing treatments for people who suffer from neurodegenerative diseases” but did not specify whether verdiperstat will continue to be included. 

Biohaven licensed verdiperstat from AstraZeneca back in 2018, but the therapy has struggled in the clinic since. Almost a year ago to the day, Biohaven reported that the drug failed to improve symptoms in patients with multiple system atrophy compared to placebo. 

The latest flop follows lukewarm results released by Biohaven in May showing that troriluzole failed to treat all types of spinocerebellar ataxia. But the company chalked up the loss in part to lower-than-expected disease progression across the patient population and also suggested there was a treatment effect in a particular genotype of the disease. As a result, Coric said at the time that Biohaven would work with regulators to discuss the data and potentially advance the program forward. 

Regardless of whether those conversations with regulators pan out, the reality for Biohaven is that it has yet to gain traction since selling off its migraine wing, which most notably included FDA-approved Nurtec ODT. The $11.6 billion deal with Pfizer also included Biohaven’s faster-acting nasal formulation, zavegepant. 

The two disappointing readouts now leave just one remaining near-term, phase 3 data drop to come: troriluzole as a treatment for obsessive-compulsive disorder. The company has projected those data will come in the second half of 2022.