RARE Daily

Biohaven Therapy Fails in Phase 3 Trial in Multiple System Atrophy

September 27, 2021

Biohaven Pharmaceutical Holding reported that results from a focused analysis of a clinical trial of verdiperstat in multiple system atrophy did not statistically differentiate it from placebo on the prespecified primary efficacy measure, nor on the key secondary efficacy measures.

Photo: Irfan Qureshi, vice president of neurology at Biohaven

Multiple system atrophy (MSA) is a rare, rapidly progressive, severely debilitating, and fatal neurodegenerative disease that leads to death within a median of 6 to 10 years after the onset of symptoms. Manifestations of MSA can include urinary and sexual dysfunction, dizziness and fainting due to low blood pressure (orthostatic hypotension), and motor impairments such as tremor, rigidity, unsteady gait, and difficulty speaking and swallowing. The most common causes of death in MSA are infection and cardiopulmonary complications. Currently, patients receive only symptomatic and palliative therapies as there are no disease-modifying treatments and no cure for MSA.

Initial analysis of safety data was consistent with the overall profile of verdiperstat from prior clinical trial experience. Additional analyses are still pending, and full study results will be presented at an upcoming scientific meeting.

“While we are disappointed that verdiperstat did not demonstrate efficacy for the treatment of MSA, Biohaven remains committed to fighting on behalf of people living with neurodegenerative diseases,” said Irfan Qureshi, vice president of neurology at Biohaven. “There are currently no approved disease modifying therapies for MSA and we must continue to advance the science to improve treatment outcomes for patients suffering from this disease.”

Verdiperstat is an experimental first-in-class, potent, selective, brain-penetrant, and irreversible myeloperoxidase (MPO) enzyme inhibitor that Biohaven is developing for the treatment of neurodegenerative diseases. Verdiperstat may help preserve neurons through inhibition of MPO-induced pathological oxidative stress and further inflammation that contribute to cellular injury in neurodegenerative disease.

Although the mechanism of action for verdiperstat, myeloperoxidase inhibition, was shown not to be effective for MSA, the rationale of targeting brain inflammation remains strong in other disease states.  An ongoing clinical trial evaluating the efficacy of verdiperstat in amyotrophic lateral sclerosis is being conducted in collaboration with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and is expected to complete enrollment in the fourth quarter of 2021.

Author: Rare Daily Staff

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