Benzinga | Nov 15, 2021 11:07AM EST

Biohaven Completes Enrollment Of Verdiperstat Arm Of Pivotal Healey ALS Platform Trial Conducted By The Healey Center For ALS At Massachussetts General Hospital

Biohaven Pharmaceutical Holding Company Ltd. (NYSE:BHVN) ("Biohaven") announced today that enrollment is now complete in the verdiperstat arm of the HEALEY ALS Platform Trial. This first-ever platform trial in ALS, sponsored by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH), is designed to evaluate the safety and efficacy of multiple drug candidates in people with Amyotrophic Lateral Sclerosis ("ALS"). Verdiperstat was selected as one of the inaugural investigational treatments for the HEALEY ALS Platform Trial which began enrolling patients in July 2020 across 52 sites from the Northeast ALS Consortium (NEALS). Topline results for verdiperstat in the treatment of ALS are anticipated in mid-2022.

"We are enthusiastic about achieving this enrollment goal as it translates to being one step closer to finding a life-changing treatment for ALS. Our team is grateful to the ALS community and people living with ALS who have trusted us in this first-of-its-kind trial", said Merit Cudkowicz, MD, MSc, Principal Investigator and Sponsor of the HEALEY ALS Platform Trial, Director of the Sean M. Healey & AMG Center for ALS and chief of the Department of Neurology at Mass General, and the Julieanne Dorn Professor of Neurology at Harvard Medical School.

Irfan Qureshi, MD, Biohaven Vice President and Verdiperstat Development Lead, stated, "People with ALS deserve novel and more effective therapeutic options. Biohaven is very pleased that the verdiperstat arm of the trial is fully enrolled. This important milestone brings us closer to knowing whether verdiperstat can benefit people with ALS. We are deeply grateful to the ALS community, especially the study participants and their families, for their unwavering commitment to the trial."

The HEALEY ALS Platform Trial, the first-ever platform trial in ALS, is designed to evaluate multiple investigational treatments simultaneously, thus accelerating the development of effective and breakthrough treatments for people living with ALS. The trial has enrolled approximately 160 adults with ALS into the verdiperstat regimen. Participants are randomized in a 3-to-1 ratio to be treated with verdiperstat 600 mg oral tablet twice daily or placebo for 24 weeks. The study's primary efficacy endpoint is measurement of the change in disease severity from baseline to week 24 on the ALS Functional Rating Scale-Revised (ALSFRS-R) in patients receiving treatment versus placebo. Secondary endpoints include change in respiratory function, muscle strength, and survival. The study is designed to support a potential regulatory filing and approval if the top line results are positive. More information about the HEALEY ALS Platform Trial can be found at www.massgeneral.org/neurology/als/research/platform-trial.

Calaneet Balas, President and CEO of The ALS Association, added, "We celebrate the successful enrollment of the HEALEY ALS Platform Trial as it moves us closer to potential new treatment options for ALS. The ALS Association is proud to support the trial and grateful to the HEALEY Center for championing this effort." More information about the ALS Association can be found at www.als.org.