Biohaven Receives FDA May Proceed Letter for BHV-3241 Trial

Biohaven announced the U.S. Food and Drug Administration (FDA) has notified the company that it may proceed with its clinical investigation of BHV-3241, a novel myeloperoxidase (MPO) inhibitor, which Biohaven is developing as a potential treatment for multiple system atrophy (MSA). The FDA May Proceed Letter was received following Biohaven's reactivation of the investigational new drug (IND) application initially filed by AstraZeneca prior to licensing the compound to Biohaven.

BHV-3241 is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of MPO, an enzyme that acts as a key driver of pathological oxidative stress and inflammation in the brain. MPO inhibition is a promising therapeutic strategy for treating a number of neurodegenerative diseases, including MSA.

"We are very pleased to report the FDA's acceptance of the IND for BHV-3241 for the treatment of MSA. We believe BHV-3241 has the potential to be the first effective treatment for people living with MSA and expect to start our Phase 3 trial in the third quarter of 2019," Vlad Coric, M.D., CEO of Biohaven, said.

MSA is a rare, rapidly progressive, and fatal neurodegenerative disease that leads to death after an average of 6 to 10 years from disease onset. MSA causes Parkinson's disease-like movement problems (e.g., 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. There is no cure for MSA. Only symptomatic and palliative therapies are available.

"People living with MSA urgently need new therapies. BHV-3241 offers the potential to treat MSA by inhibiting myeloperoxidase enzyme activity, thereby alleviating oxidative stress and neuroinflammation," Irfan Qureshi, M.D., Executive Director of Neurology at Biohaven, said. "AstraZeneca's Phase 2 clinical trial suggested that BHV-3241 may have reduced MSA disease progression in a dose-dependent manner, measured by the Unified MSA Rating Scale. We are excited to advance into a Phase 3 trial to assess the efficacy of BHV-3241 for the treatment of MSA."

BHV-3241 also has the potential to be developed in a number of other disease indications associated with oxidative stress, inflammation, and neurodegeneration. Approximately 250 healthy volunteers and patients have been treated with BHV-3241 in Phase 1 and Phase 2 clinical trials performed by AstraZeneca. Results from a Phase 2 trial in MSA showed evidence of target engagement and favorable trends over 12 weeks on the Unified MSA Rating Scale, an exploratory clinical outcome measure. Biohaven licensed BHV-3241 from AstraZeneca in September 2018, where it was known as AZD3241.

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