Biohaven picks up AstraZeneca CNS drug, plans phase 3

Biohaven Pharmaceutical has licensed an experimental treatment of the neurodegenerative disorder multiple system atrophy (MSA) from AstraZeneca. The deal follows a midphase trial that linked oral myeloperoxidase (MPO) inhibitor AZD3241 to numerical improvements on a scale of MSA symptoms.

AstraZeneca completed the 32-patient trial in 2016 but held off on advancing the drug to the next stage of development. The involvement of Biohaven means AZD3241, now known as BHV3241, will now start progressing down the pipeline again. Biohaven plans to run a phase 3 trial in MSA to see if the initial signs of efficacy are evident in a larger study.

For now, the clinical data in support of BHV3241 are very preliminary. AstraZeneca included an exploratory efficacy endpoint in the phase 2a that linked BHV3241 to dose-dependent improvements in MSA symptoms. No statistical analysis was performed on the efficacy data but Biohaven thinks it is promising enough to justify further development.

"We believe the early signal observed at the 12-week timepoint in AstraZeneca's phase 2a study provides a strong rationale to initiate a longer-term, definitive trial to establish its efficacy and safety in patients with MSA," Biohaven CEO Vlad Coric, M.D., said in a statement.

Biohaven is paying an upfront, cash-and-stock fee of undisclosed size and committing to milestones and double-digit royalties to land the rights to develop the candidate in all indications outside of cardiovascular disease. AstraZeneca remains interested in the cardiovascular uses of MPOs and has a pipeline of early-stage candidates but has agreed not to develop these drugs in neurological diseases for five years.

That gives Biohaven a window in which to get BHV3241 to market without having to worry about the prospect of AstraZeneca advancing rival assets. If successful, Biohaven will address an unmet need. Levodopa improves motor function in some patients with MSA but overall there is a lack of effective drugs.

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