Biohaven Pharmaceutical Holding Company Ltd. announced that the U.S. Food and Drug Administration (FDA) has notified its collaborators at the Sean M. Healey & AMG Center for Amyotrophic Lateral Sclerosis (ALS) at Massachusetts General Hospital (MGH) that they may proceed with clinical investigation of verdiperstat, Biohaven's novel myeloperoxidase (MPO) inhibitor, in ALS. This will be the second potentially registrational trial of verdiperstat in neurodegenerative disorders. Biohaven has already initiated a separate trial of verdiperstat in multiple system atrophy. The HEALEY ALS Platform Trial is a major innovation in the field of ALS drug development that is aimed at accelerating the identification of effective new treatments. The HEALEY ALS Platform Trial is being conducted by leading ALS experts from the Healey Center in collaboration with the Northeast ALS Consortium (NEALS) clinical trial network. In contrast with traditional trials that typically evaluate only one drug at a time, platform trials create an infrastructure for the ongoing study of multiple investigational drugs in parallel using specialized statistical methods. This approach helps to maximize resources and efficiency. The FDA is now encouraging the use of innovative clinical trial designs, such as platform trials, which can be used to provide substantial evidence of clinical efficacy to support a drug approval. Verdiperstat is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of the MPO enzyme. MPO is believed to play an important role in multiple neurodegenerative diseases, because it increases oxidative stress and inflammation levels in the nervous system. Inhibiting MPO is anticipated to ameliorate these pathological mechanisms, which are strongly implicated in the onset and progression of ALS. Biohaven previously announced that verdiperstat was selected for inclusion in the first HEALEY ALS Platform Trial by an independent scientific advisory committee.