Biohaven announced it has enrolled the first patient in a Phase 3 clinical trial to evaluate the efficacy and safety of verdiperstat in subjects with Multiple System Atrophy (MSA).

Verdiperstat is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of myeloperoxidase, an enzyme that acts as a key driver of pathological oxidative stress and inflammation in the brain. Results from a previous Phase 2 trial in MSA completed by AstraZeneca AB showed evidence of target engagement and favorable trends over 12 weeks on the Unified MSA Rating Scale, an exploratory clinical outcome measure. Verdiperstat has received orphan drug designation for the treatment of MSA from the FDA Office of Orphan Products Development as well as the European Commission upon recommendation from the European Medicines Agency's Committee for Orphan Medicinal Products.

"We are so pleased to partner with Biohaven in building hope for the patients and families affected by this devastating disease. This clinical trial presents an exciting opportunity to empower people living with MSA by playing an active role in research," said Pamela Bower of the MSA Coalition.

MSA is a rare, rapidly progressive, and fatal neurodegenerative disease. Only symptomatic and palliative therapies are currently available. MSA causes Parkinson's disease-like movement problems (slow movement, rigid muscles, tremor, and poor balance), cerebellar ataxia, as well as problems with involuntary (autonomic) functions, including blood pressure control, bladder function, and digestion. MSA usually leads to death after an average of 6 to 10 years from the onset of symptoms.

"Verdiperstat is the first product candidate from Biohaven's neuroinflammation platform and highlights our commitment to developing innovative medicines for neurological diseases with high unmet need," Irfan Qureshi, MD, Biohaven Vice President and Development Lead for Verdiperstat, said.

Biohaven expects to recruit approximately 250 patients in the study, across approximately 50 sites in the United States and Europe. The study will enroll subjects with MSA diagnosed using consensus clinical criteria and include both subtypes of MSA, MSA-Parkinsonism (MSA-P) and MSA-Cerebellar (MSA-C). Researchers will evaluate the efficacy of verdiperstat, compared to placebo, as measured by a change from baseline in a modified version of the Unified MSA Rating Scale (UMSARS) at Week 48.

"We are excited to have enrolled the first patient in this study. Working together with our patients and the Biohaven team, we are optimistic that this is the beginning of a new era of treatment and eventually cure for this challenging neurodegenerative disorder," Jeremy D. Schmahmann, MD, Professor of Neurology at Harvard Medical School and Founding Director of the Massachusetts General Hospital Ataxia Unit, said.

Biohaven licensed verdiperstat (BHV-3241) from AstraZeneca AB in September 2018, where it was known as AZD3241. Approximately 250 healthy volunteers and patients were treated with verdiperstat in Phase 1 and Phase 2 clinical trials. Results from a Phase 2 trial in MSA showed evidence of target engagement and favorable trends over 12 weeks on the UMSARS, an exploratory clinical outcome measure. Verdiperstat received Orphan Drug designation from the U.S Food and Drug Association as well as the European Commission due to the unmet medical need in MSA. Verdiperstat also has the potential to be developed in a number of other diseases associated with oxidative stress, inflammation, and neurodegeneration.

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