NEW HAVEN, Conn., Jan. 9, 2018 /PRNewswire/ — Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) ("Biohaven" or the "Company") announced positive results today from its bioequivalence study with BHV-0223, an innovative sublingual formulation of riluzole. The study was designed to demonstrate pharmacokinetic equivalence of 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) achieves bioequivalent exposures relative to Rilutek (50 mg). In the study, 138 healthy volunteers were administered BHV-0223 and Rilutek under fasted conditions. In the pre-specified primary analysis, BHV-0223 achieved area-under-the-curve and peak exposures of approximately 90% and 113%, respectively, compared to those generated by generic riluzole. The 90% confidence intervals were within the 80% to 125% range that is used to define bioequivalence.
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 designed to meet the needs of patients with ALS. BHV-0223 is an innovative, 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. That is, sublingual administration of 40 mg BHV-0223 results in bioequivalent blood exposures to orally ingested 50 mg tablets of Rilutek. Based on this observation and reduced drug exposure to the liver, BHV-0223 may have a lessened risk for causing liver test elevations.
Vlad Coric, M.D., Chief Executive Officer of Biohaven, commented, "We are very excited about favorable results from the current study establishing the bioequivalence of our innovative sublingually administered BHV-0223. 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."
Beyond the current advance with BHV-0223, Biohaven is making progress across its oral, small molecule calcitonin gene receptor peptide (CGRP) antagonist for the treatment of migraine and glutamate modulation technology platforms for neurodegenerative diseases with high unmet need.
This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements involve substantial risks and uncertainties, including statements that are based on the current expectations and assumptions of the Company's management. All statements, other than statements of historical facts, included in this press release, including the Company's timing of the expected NDA submission for BHV-0223, the potential regulatory approval of BHV-0223, the ability of BHV-0223 to a lessen risk of liver test elevations relative to Rilutek and the ability of BHV-0223 to achieve therapeutic blood levels at a lower milligram dose than Rilutek, are forward-looking statements. The use of certain words, including the "believe", "could", "expect" and "will" and similar expressions are intended to identify forward-looking statements. The Company may not actually achieve the plans and objectives disclosed in the forward-looking statements and you should not place undue reliance on the Company's forward-looking statements. Various important factors could cause actual results or events to differ materially from those that may be expressed or implied by our forward-looking statements, including uncertainties relating to the preparation and submission of an NDA with respect to BHV-0223 and the regulatory review process. Additional important factors to be considered in connection with forward-looking statements are described in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 14, 2017. The forward-looking statements are made as of this date and the Company does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
Dr. Vlad Coric
Chief Executive Officer
SOURCE Biohaven Pharmaceuticals, Inc.