Biohaven plans to conduct a Phase 3 clinical trial of this product candidate for the treatment of multiple system atrophy a rare, rapidly progressive and fatal neurogenerative disease with no cure or effective treatments.
AZD3241 will now be referred to as BHV3241.
MPO is a key driver of oxidative and inflammatory processes and is significantly increased in a range of brain disorders. It is thought that inhibiting MPO activity may be a promising therapeutic strategy for neuroinflammatory and neurodegenerative conditions, including MSA.
Studies have also linked increased MPO levels with multiple sclerosis and Alzheimer's disease.
BHV3241 is a clinical stage MPO inhibitor that has completed Phase 1 studies at doses up to 900mg twice a day. Preliminary results from a Phase 2a trial completed by AstraZeneca in patients with MSA showed numerical improvements on the primary outcome measure of efficacy, change from baseline as measured by the Unified MSA Rating Scale.
After 12 weeks of treatment, placebo-treated patients worsened by 4.6 points (SE=1.1, n=17), while BHV3241- treated patients showed declines of 3.7 points (SE= 1.2, n=17) at the 300mg twice-daily dose and 2.6 points (SE=1.4, n=18) at the 600mg twice-daily dose.
Corresponding benefits were also observed in other outcome measures, such as the Composite Autonomic Symptom Score and MSA-Quality of Life scale. These clinical findings were consistent with neuroprotective effects of BHV3241 observed in animal models.
In the Phase 2a trial, BHV3241 significantly decreased MPO activity in human blood, a biomarker of the drug engaging its target. BHV3241 has been generally safe and well tolerated in approximately 250 patients.
The in-licensing of BHV3241 expands Biohaven's portfolio of innovative, late-stage product candidates for the treatment of neurologic and psychiatric disease indications.
Under the terms of the agreement, AstraZeneca will receive upfront cash payment and shares in Biohaven, and AstraZeneca is eligible for further development and commercial milestone payments, and up to double-digit sales based royalties.
Biohaven may pursue all therapeutic indications of BHV3241 with the exception of cardiovascular disease, and is obligated to use commercially reasonable efforts to develop and, if approved, commercialize BHV3241.
AstraZeneca has agreed not to pursue additional MPO inhibitors it has in early stage cardiovascular development for neurologic diseases for a period of five years.
Biohaven's pipeline now includes investigational agents across three novel mechanisms of action to target central nervous system disorders.
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