Biohaven Pharmaceutical has started patient enrolment in a Phase III clinical trial being conducted to assess verdiperstat for the treatment of multiple system atrophy (MSA).
MSA is a rare, progressive neurodegenerative disease that causes movement problems, cerebellar ataxia, and issues with involuntary functions such as blood pressure and digestion.
Verdiperstat is an oral, brain-penetrant designed to irreversibly block myeloperoxidase enzyme, which plays an important role in pathological oxidative stress and brain inflammation.
Biohaven licensed the therapeutic from AstraZeneca in September last year.
Biohaven Pharmaceutical vice-president and Verdiperstat development lead Irfan Qureshi said: “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.”
The Phase III trial will investigate the safety and efficacy of the drug candidate in around 250 participants at nearly 50 sites across the US and Europe. It will enrol both MSA-Parkinsonism (MSA-P) and MSA-Cerebellar (MSA-C) patients.
Verdiperstat’s efficacy will be measured as a change in the modified version of the Unified MSA Rating Scale (UMSARS) from baseline to week 48. This will be considered as the primary outcome of the study.
The secondary outcomes are efficacy measured using the Clinical Global Impression of Change (CGI-C) score and the therapeutic’s effect on quality of life measured by the MSA-Quality of Life (MSA-QoL) scale at week 48.
Previously, AstraZeneca reported favourable trends on the Unified MSA Rating Scale in a Phase II study.
The drug candidate holds orphan drug designation from the US Food and Drug Administration (FDA) and the European Commission (EC) for the treatment of MSA.