Biohaven Pharmaceutical has announced the completion of enrollment of the verdiperstat arm of the Healey ALS Platform trial. Sponsored by the Sean M. Healey and AMG Center for Amyotrophic Lateral Sclerosis (ALS) at Massachusetts General Hospital, the study is intended to evaluate the safety and efficacy of multiple candidates.
According to the company, verdiperstat was identified as one of the first investigational treatments for the Healey ALS Platform Trial, which started patient enrollment in July 2020, over 52 sites that are part of the Northeast ALS Consortium (NEALS). Researchers reportedly expect topline results for verdiperstat in ALS treatment in mid-2022.
Merit Cudkowicz, principal investigator and sponsor of the Healey ALS Platform trial and director of the Healey and AMG Center, said the trial stands to bring hope to ALS patients.
"We are enthusiastic about achieving this enrollment goal as it translates to being one step closer to finding a life-changing treatment for ALS,” she said. “Our team is grateful to the ALS community and people living with ALS who have trusted us in this first-of-its-kind trial."
Irfan Qureshi, Biohaven vice president and verdiperstat development lead, commented, "People with ALS deserve novel and more effective therapeutic options. Biohaven is very pleased that the verdiperstat arm of the trial is fully enrolled.”
Qureshi added, “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 of its kind for ALS, is aimed at the evaluation of 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.
According to the study team, 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 the 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 topline results are positive.
Calaneet Balas, president and CEO of the ALS Association, remarked, "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."
ALS is a progressive, life-threatening, and rare neuromuscular disease that affects approximately 30,000 people in the US. The median age of onset is 55 years and the average survival is three to five years after the onset of first symptoms.
ALS is characterized by the loss of motor neurons in the brain, brainstem, and spinal cord that leads to progressive muscle weakness and difficulties in speaking, swallowing, and breathing. There are currently limited treatment options and no cure for ALS.
Verdiperstat (BHV-3241) is an investigational first-in-class, potent, selective, brain-penetrant, and myeloperoxidase (MPO) enzyme inhibitor in development by Biohaven for the treatment of neurodegenerative diseases. Verdiperstat may help preserve neurons through inhibition of MPO-induced pathological oxidative stress and inflammation that contribute to cellular injury in neurodegenerative diseases such as amyotrophic lateral sclerosis.