Biohaven announced the U.S. Food and Drug Administration (FDA) has notified the company it may proceed with its clinical investigation of sublingual BHV-0223 as a potential treatment for patients with Amyotrophic Lateral Sclerosis (ALS). The FDA clearance to proceed was received following Biohaven's submission of an investigational new drug (IND) application for this program, and the Company expects to commence a bioequivalence study of BHV-0223 in the current quarter. Biohaven previously received regulatory feedback from the FDA that the Section 505(b)(2) pathway is acceptable for BHV-0223 in ALS, and that beyond this study, no additional efficacy or toxicology studies will be necessary for the submission of a new drug application (NDA).
BHV-0223 is an innovative sublingually administered and orally dissolving tablet (ODT) formulation of riluzole, a glutamate modulating agent, which is designed to advance beyond the current limitations of riluzole tablets. While riluzole tablets are FDA approved for ALS, they may be difficult to administer in ALS patients, who typically have difficulty in swallowing. The tablets also have certain pharmacokinetic (PK) and pharmaceutic limitations including being associated with a negative food effect that lowers drug levels. BHV-0223 is unique in that it utilizes the Zydis ODT fast-dissolve, dosing technology developed under an exclusive worldwide agreement with Catalent. In previous clinical studies, BHV-0223 was associated with less PK variability than riluzole tablets.
BHV-0223 is one of the lead drug candidates in Biohaven's glutamate modulation technology platform being developed across several therapeutic indications. Agents that modulate glutamate neurotransmission may have therapeutic potential in multiple disease states involving glutamate dysfunction, including ALS, Alzheimer's disease, Rett syndrome, dementia, dystonia, ataxias, tinnitus, anxiety disorders, and affective disorders like major depressive disorder.
"Our goal is to establish the bioequivalence of BHV-0223 to its active pharmaceutical ingredient, riluzole, with our new sublingual and lower dose formulation,” Vlad Coric, M.D., Chief Executive Officer of Biohaven, said. “If we successfully establish bioequivalence in the upcoming trial and demonstrate the advantages of this formulation to patients, we expect be in position for an NDA submission. ALS is a serious neurodegenerative disorder for which there are few treatment options, and we believe that the significant improvements gained through enhanced formulation, dosage and route of administration of BHV-0223 may benefit patients with this devastating disease."