Biohaven's neurological disorder drug flops in late-stage trials

Sept. 27, 2021

Biohaven Pharmaceutical Holding announced that its drug candidate, verdiperstat, failed to show effectiveness in treating a rare neurological disorder called multiple system atrophy (MSA).

According to the company, data from a phase 3 trial indicate that verdiperstat did not statistically differentiate from placebo on the pre-specified primary efficacy measure, nor on the key secondary efficacy measures.

Verdiperstat is an investigational first-in-class, potent, selective, brain-penetrant, and irreversible myeloperoxidase (MPO) enzyme inhibitor that the New Haven, Connecticut-based biopharma is developing for the treatment of neurodegenerative diseases.

MSA is a degenerative disorder that affects the body's involuntary functions such as blood pressure and breathing. MSA shares many Parkinson's disease symptoms, such as slow movement and poor balance, and can eventually be fatal. Currently, there are no approved therapies that target the underlying disease.

Biohaven says additional analyses are still pending and full-study results will be presented at an upcoming scientific meeting. The company is also testing verdiperstat in amyotrophic lateral sclerosis (Lou Gehrig's disease) and expects to complete patient enrollment in the ongoing clinical trial by the end of 2021.

Read the press release