NEW HAVEN: Biohaven Pharmaceutical Holding Company Ltd. [NYSE:BHVN] announced positive results of its bioequivalence study with BHV-0223, what it calls an “innovative sublingual [can be taken without swallowing] formulation of riluzole.”
The study was designed to demonstrate “pharmacokinetic equivalence” of the company’s drug sublingual BHV-0223 compared to the reference listed drug Rilutek (riluzole), which is currently the standard of care treatment for patients with Amyotrophic Lateral Sclerosis (ALS).
Topline results confirmed that sublingual BHV-0223 (40 mg) achieved bioequivalent exposures relative to Rilutek (50 mg). In the study, 138 healthy volunteers were administered BHV-0223 and Rilutek under fasted conditions.
Biohaven is developing BHV-0223 as a potential treatment for patients with ALS. Biohaven previously received regulatory feedback from the FDA that the Section 505(b)(2) pathway is acceptable for BHV-0223 in ALS, and that no additional efficacy or toxicology studies are necessary for submission of a new drug application (NDA) for this indication. With these positive results, Biohaven will advance towards completing an NDA with the goal of submitting in the first half of 2018.
BHV-0223 is sublingually administered orally dissolving tablet (ODT) that makes use of the unique Zydis® ODT fast-dissolve technology. It is being developed under an exclusive, worldwide agreement with Catalent. While riluzole is FDA-approved for ALS, conventional tablets may be difficult to administer to ALS patients, who often have dysphagia or trouble swallowing.
By contrast, when BHV-0223 is placed under the tongue, it dissolves in seconds and does not require swallowing. In addition, riluzole is associated with dose-dependent effects on liver tests (transaminases). BHV-0223 offers bioequivalent exposures compared to Rilutek with a 20% lower dose.
Vlad Coric, M.D., Chief Executive Officer of Biohaven, said, "we are very excited about favorable results from the current study. Sublingual BHV-0223 is unique in that it is a Zydis® orally dissolving tablet (ODT) formulation, that is optimized to allow administration to patients with dysphagia and to provide therapeutic blood levels with a lower milligram dose for all patients suffering from ALS."
Rob Berman M.D., Chief Medical Officer added, "this is a meaningful advancement for patients with ALS, especially those who would be able to continue taking riluzole despite difficulties swallowing. In addition, the possibility to achieve a therapeutic exposure with a lower drug dose would make BHV-0223 more amenable to those patients with concerns about liver toxicity."