Biohaven's Verdiperstat Receives FDA May Proceed Letter to Initiate Trial in Amyotrophic Lateral Sclerosis Conducted by the Healey Center for ALS At Mass General
January 23, 2020 at 02:45 am IST
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Biohaven Pharmaceutical Holding Company Ltd. announced that the U.S. Food and Drug Administration (FDA) has notified its collaborators at the Sean M. Healey & AMG Center for Amyotrophic Lateral Sclerosis (ALS) at Massachusetts General Hospital (MGH) that they may proceed with clinical investigation of verdiperstat, Biohaven's novel myeloperoxidase (MPO) inhibitor, in ALS. This will be the second potentially registrational trial of verdiperstat in neurodegenerative disorders. Biohaven has already initiated a separate trial of verdiperstat in multiple system atrophy. The HEALEY ALS Platform Trial is a major innovation in the field of ALS drug development that is aimed at accelerating the identification of effective new treatments. The HEALEY ALS Platform Trial is being conducted by leading ALS experts from the Healey Center in collaboration with the Northeast ALS Consortium (NEALS) clinical trial network. In contrast with traditional trials that typically evaluate only one drug at a time, platform trials create an infrastructure for the ongoing study of multiple investigational drugs in parallel using specialized statistical methods. This approach helps to maximize resources and efficiency. The FDA is now encouraging the use of innovative clinical trial designs, such as platform trials, which can be used to provide substantial evidence of clinical efficacy to support a drug approval. Verdiperstat is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of the MPO enzyme. MPO is believed to play an important role in multiple neurodegenerative diseases, because it increases oxidative stress and inflammation levels in the nervous system. Inhibiting MPO is anticipated to ameliorate these pathological mechanisms, which are strongly implicated in the onset and progression of ALS. Biohaven previously announced that verdiperstat was selected for inclusion in the first HEALEY ALS Platform Trial by an independent scientific advisory committee.
Biohaven Pharmaceutical Holding Company Ltd. is a United States-based commercial-stage biopharmaceutical company. The Company has a portfolio of therapies to improve the lives of patients with debilitating neurological and neuropsychiatric diseases, including rare disorders. The Company's Neuroinnovation portfolio includes NURTEC ODT (rimegepant) for the acute and preventive treatment of migraine and a pipeline of product candidates across five platforms: calcitonin gene related peptide (CGRP) receptor antagonism for the acute and preventive treatment of migraine as well as other pain-related disorders and non-migraine indications; glutamate modulation for obsessive-compulsive disorder (OCD); myeloperoxidase (MPO) inhibition for multiple system atrophy (MSA) and amyotrophic lateral sclerosis (ALS); Kv7 Ion Channel Activators (Kv7) to target indications including epilepsy, pain disorders and effective disorders; and Myostatin offers a natural protein that limits skeletal muscle growth.
Biohaven's Verdiperstat Receives FDA May Proceed Letter to Initiate Trial in Amyotrophic Lateral Sclerosis Conducted by the Healey Center for ALS At Mass General