A drug candidate aimed at helping patients suffering from amyotrophic lateral sclerosis has proven to be ineffective against the disease in a clinical trial.
New Haven-based pharmaceutical company Biohaven announced the results in late September.
ALS is a deadly, progressive neurodegenerative condition commonly referred to as Lou Gehrig’s Disease. The median age of onset is 55, with average survival of between three to five years after the onset of symptoms. There is no cure, and treatment options are limited.
The phase 2/3 trial tested the safety and effectiveness of verdiperstat in treating ALS.
According to the company, there was no statistical difference between verdiperstat and a placebo in evaluating the drug’s effectiveness during the 24-week study period.
Dr. Irfan Qureshi, who was recently promoted to chief medical officer, said in an announcement, “While we are disappointed that verdiperstat did not demonstrate efficacy for ALS, Biohaven remains committed to developing treatments for people who suffer from neurodegenerative diseases.”
Qureshi said the company is grateful to its collaborators at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, HEALEY ALS Platform Trial study investigators, and the ALS community, especially people with ALS and their families, who made the trial possible.
Additional analyses are ongoing, with complete study results to be presented to the scientific community, according to the company.
Earlier this month, Pfizer Inc. completed its $11.6 billion acquisition of Biohaven Pharmaceutical Holding Company Ltd., a deal which included its blockbuster migraine drug Nurtec ODT and a portfolio of calcitonin gene-related peptide (CGRP) receptor antagonists.
The ALS candidate was not part of the Pfizer acquisition.
Officials have said that the spinoff company, Biohaven Ltd., retains Biohaven’s non-migraine pipeline, such as its Kv7 ion channel activators, glutamate modulation and myostatin inhibition platforms.
Biohaven Ltd. launched with $258 million in cash, no debt, and 13 clinical and preclinical programs. These are focused on neuroscience and rare disorders, including ailments such as obsessive compulsive disorder, spinocerebellar ataxia, epilepsy, pain and mood disorders, and spinal muscular atrophy.
Contact Michelle Tuccitto Sullo at msullo@newhavenbiz.com.
