Deals, Licensing

Licensing AstraZeneca castoff, Biohaven preps PhIII effort in new neurodegenerative indication

Biohaven $BHVN has added another drug off of AstraZeneca’s shelf to its neurology pipeline.

Coming with a package of data from four Phase I and three Phase IIa studies, AZD3241 inhibits myeloperoxidase, or MPO, a key driver of inflammatory process that’s observed in high levels in a range of brain disorders.

The biotech plans to test the drug — now renamed BHV3241 — in a Phase III trial as a treatment of multiple system atrophy, having seen preliminary results from a small Phase IIa trial that showed a notable biomarker response and “numerical improvements” on the Unified MSA Rating Scale after 12 weeks of treatment.

Vlad Coric

That, according to Biohaven, makes the case for a potential first-in-class treatment for the rare neurodegenerative disease that often kills patients after five to seven years of tremors, slow movement, muscle rigidity and ataxia.

“These data, coupled with a favorable safety profile and evidence that BHV3241 reduced intracellular aggregates of alpha-synuclein, suppressed microglial activation and rescued neurodegeneration in MSA animal models, support our belief that BHV3241 has potential as a disease-modifying treatment in Multiple System Atrophy,” said professor Werner Poewe at Austria’s Medical University Innsbruck in a statement.

Werner Poewe

AstraZeneca is gaining some shares in Biohaven in addition to an unspecified upfront cash payment for the asset, as well as other milestones and up to double-digit royalties should the drug make it to the market. It has also promised not to pursue any of the other MPO inhibitors in its pipeline for neurologic diseases for five years as Biohaven explores other potential indications for BHV3241.

This is the second asset the UK pharma giant has handed off to the Yale spinout, following a $210 million deal that involved an NMDA antagonist.

The deal adds to Biohaven’s late-stage pipeline, which is currently dominated by rimegepant, the CGRP migraine therapy it in-licensed from Bristol-Myers Squibb. Execs are counting on rimegepant and trigriluzole — a reformulation of an old ALS drug called riluzole that’s also being studied for OCD and ataxia — for near-term FDA applications, putting their initial focus on depression and anxiety on the backburner for now.

“The in-licensing of BHV3241 expands Biohaven’s portfolio of innovative, late-stage product candidates for the treatment of neurologic and psychiatric disease indications, “ said CEO and Yale professor Vlad Coric.

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