NEW HAVEN, Conn.—Biohaven Pharmaceuticals reported today that the company 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 (MSA).
Verdiperstat is an investigational drug and potential first-in-class, irreversible myeloperoxidase (MPO) enzyme inhibitor designed to target sources of neuroinflammation which contribute to brain cell death in neurodegenerative diseases — including MSA and amyotrophic lateral sclerosis (ALS). Verdiperstat may help preserve neurons through inhibition of MPO-induced pathological oxidative stress and further inflammation that contributes to cellular injury.
“The M-STAR trial has proceeded with great success despite the unique challenges created by COVID-19,” said Dr. Phillip A. Low, M.D., the Robert D. and Patricia E. Kern Professor of Neurology at the Mayo Clinic. “This demonstrates the determination of the MSA community to work towards a potential new treatment for this devastating disease. I applaud the true champions of the M-STAR trial — the MSA patients — whose hope has propelled us all.”
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 between 40-80 years of age with a clinical diagnosis of MSA. Patients across approximately 50 sites in the U.S. 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.
“Verdiperstat has the potential to be the first disease-modifying treatment for people suffering from MSA, and we are deeply indebted to the international community of MSA investigators, our trial sites across the globe, and patients who helped complete trial enrollment in less than a year. The M-STAR trial participants and families are our inspiration,” added Irfan Qureshi, M.D., vice president of Neurology at Biohaven. “Thank you for your ongoing commitment as together we advance this first large pivotal trial in MSA, and we all look forward to the results by the end of next year.”
MSA is a rare, rapidly progressive, fatal neurodegenerative disease. It can lead 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 U. S. and Europe alone. While it often isn’t 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 which regulate internal body functions and motor control progressively deteriorate, severely compromising the body’s autonomic functions. Symptoms of autonomic failure include fainting spells and problems with heart rate (due to orthostatic hypotension), erectile dysfunction and bladder control issues.
Motor impairments may include tremor, rigidity and/or loss of muscle coordination, as well as difficulties with speech and gait. Some of these features are similar to those seen in Parkinson’s disease, and early in the disease course it may be difficult to distinguish between these disorders. Patients currently receive symptomatic and palliative therapies, as there are no disease-modifying treatments and no cure for MSA.
“The MSA patient community is optimistic that the M-STAR study will figure prominently in the history of our collective efforts to defeat this debilitating disease. On behalf of MSA patients worldwide, we are thankful to Biohaven for their effort to stand with us against MSA,” stated Philip M. Fortier, MA, executive director of the Defeat MSA Alliance.
“The MSA Coalition is so pleased to hear about the outstanding momentum of enrollment in the M-STAR study,” commented Cyndi Roemer, chair of the board of directors at the MSA Coalition, Inc. “This news represents new medical possibilities and much needed hope for the patients and families who are living with MSA.”
Verdiperstat has received Orphan Drug and Fast Track designations for MSA from the U.S. Food and Drug Administration, as well as Orphan Drug designation from the European Medicines Agency. A clinical trial designed to evaluate the efficacy of verdiperstat in ALS is being planned in collaboration with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital.