Therapy Areas: Cardiovascular
Biohaven Completes Enrollment Ahead of Timelines in International Phase 3 Clinical Trial of Verdiperstat in Multiple System Atrophy
23 July 2020 - - US-based biopharmaceutical company Biohaven Pharmaceutical Holding company Ltd. (NYSE: BHVN) has successfully completed enrollment in the M-STAR study, an international Phase 3 clinical trial evaluating the safety and efficacy of verdiperstat in multiple system atrophy, the company said.

Verdiperstat is an investigational drug and potential first-in-class myeloperoxidase inhibitor designed to target sources of neuroinflammation that contribute to brain cell death in neurodegenerative diseases including MSA and amyotrophic lateral sclerosis.

Verdiperstat has received Orphan Drug and Fast Track designations for MSA from the US Food and Drug Administration, as well as Orphan Drug designation from the European Medicines Agency.

M-STAR is a Phase 3 randomized, double-blind, placebo-controlled clinical trial designed to evaluate the safety and efficacy of verdiperstat in approximately 300 patients with a clinical diagnosis of MSA between 40-80 years of age.

Patients across approximately 50 sites in the United States and Europe are randomized to 48 weeks of treatment with verdiperstat 600 mg oral tablet taken twice daily or placebo.

The study's primary efficacy endpoint will assess disease progression measured by the change from baseline to week 48 on a modified version of the Unified MSA Rating Scale (UMSARS) in patients receiving verdiperstat versus placebo.

Topline data are expected by the end of 2021.

MSA is a rare, rapidly progressive, and fatal neurodegenerative disease that leads to death within a median of 6-10 years after receiving a diagnosis, often due to cardiac or respiratory complications. MSA affects approximately 50,000 people in the United States and Europe alone.

While it is often not diagnosed until the fifth or sixth decade of life, symptoms can first appear earlier in a person's 40s and go undiagnosed for years.

In MSA, portions of the brain that regulate internal body functions and motor control progressively deteriorate, severely compromising the body's involuntary (autonomic) functions.

Symptoms of autonomic failure that may be seen in MSA include fainting spells and problems with heart rate (due to orthostatic hypotension), erectile dysfunction, and bladder control issues.

Motor impairments (loss of or limited muscle control or movement, or limited mobility) may include tremor, rigidity, and/or loss of muscle coordination as well as difficulties with speech and gait (the way a person walks).

Some of these features are similar to those seen in Parkinson's disease, and early in the disease course it often may be difficult to distinguish these disorders.

Currently, patients receive only symptomatic and palliative therapies as there are no disease-modifying treatments and no cure for MSA.

Verdiperstat (BHV-3241) is an investigational first-in-class, potent, selective, brain-penentrant, and irreversible myeloperoxidase enzyme inhibitor that Biohaven is developing for the treatment of neurodegenerative diseases.

Verdiperstat may help preserve neurons through inhibition of MPO-induced pathological oxidative stress and further inflammation that contribute to cellular injury in neurodegenerative diseases such as MSA and ALS. Biohaven licensed verdiperstat (BHV-3241) from AstraZeneca in September 2018.

The M-STAR study is a currently ongoing Phase 3 clinical trial designed to evaluate the efficacy of verdiperstat in MSA.

Verdiperstat has received Orphan Drug and Fast Track designations for MSA from the US FDA, as well as Orphan Drug designation from the European Medicines Agency.

Verdiperstat also has the potential to be developed in a number of other diseases associated with oxidative stress, inflammation, and neurodegeneration.

A clinical trial designed to evaluate the efficacy of verdiperstat in amyotrophic lateral sclerosis is being planned in collaboration with the Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital.

Biohaven is a biopharmaceutical company focused on the development and commercialization of innovative best-in-class therapies to improve the lives of patients with debilitating neurological and neuropsychiatric diseases.

Biohaven's neuroinnovation portfolio includes FDA-approved NURTEC ODT (rimegepant) for the acute treatment of migraine and a broad pipeline of late-stage product candidates across three distinct mechanistic platforms: CGRP receptor antagonism for the acute and preventive treatment of migraine; glutamate modulation for obsessive-compulsive disorder, Alzheimer's disease, and spinocerebellar ataxia; and myeloperoxidase inhibition for multiple system atrophy and amyotrophic lateral sclerosis.
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