New Haven, Connecticut Dec 8, 2016 -Biohaven Pharmaceutical Holding Company Ltd. (the “Company” or “Biohaven”) announced today that the U.S. Food and Drug Administration (“FDA”) has granted the Company’s orphan drug designation request covering its drug candidate BHV-0223, an orally dissolving tablet being developed for the treatment of Amyotrophic Lateral Sclerosis (“ALS”), also referred to as Lou Gehrig’s disease. This is the Company’s third orphan drug designation request granted by the FDA.
ALS is a progressive neurodegenerative motor neuron disease that affects nerve cells in the brain and the spinal cord. The disease belongs to a group of disorders known as motor neuron diseases,which are characterized by the gradual degeneration and death of motor neurons. ALS affects up to 20,000 individuals in the United States and typically presents in patients with painless muscle weakness, trouble swallowing and muscle atrophy that ultimately progresses to paralysis and death. Since the FDA’s approval of riluzole in 1995, there have not been further clinical improvements or advances in ALS drug therapeutics over the last two decades.Several therapies are currently in clinical trials.
BHV-0223 is a sublingually absorbed and oral dissolving tablet (ODT) formulation of riluzole.BHV-0223’s novel formulation is designed to address some of the shortcomings associated with the solid oral dosage form of riluzole that ALS patients have difficulty swallowing. Because BHV-0223 is designed to be systemically absorbed through the oral mucosa, rather than through the gastrointestinal system, the Company believes that it can eliminate the negative food effect associated with riluzole, bypass first-pass metabolism and deliver effective doses of the drug at lower concentrations, while also allowing sublingual absorption in patients who experience
difficulty swallowing and eliminating the need for three hour fasting twice daily.
Robert Berman, M.D., CMO of Biohaven commented, “Patients with ALS develop a wide range of disabilities with the vast majority developing significant difficulty swallowing. Eventually, most muscles under voluntary control are affected, and individuals lose their strength and the ability to move their arms, legs, and body. As a sublingually administered and orally dissolving tablet form of riluzole, we believe that BHV-0233 may offer important advantages to ALS patients.”
Vlad Coric, M.D., CEO of Biohaven, added, “Receiving the orphan drug designation request approval for BHV-0223 in the treatment of ALS advances our global development strategy and one of our primary goals of providing therapies for patients suffering from neurologic disorders with high unmet need. If approved, this unique formulation will provide another therapeutic option to patients living with this devastating disease.”
BHV-0223 is a novel formulation of a glutamate-modulating agent that utilizes the Zydis® ODT fast-dissolve technology under an exclusive worldwide agreement with Catalent. Agents that modulate glutamate neurotransmission may have therapeutic potential in multiple disease states involving glutamate dysfunction, including ALS, ataxia, Alzheimer’s disease, Rett syndrome, dementia, dystonia, tinnitus, anxiety disorders, and affective disorders like major depressive disorder. Biohaven is pursuing the use of glutamate-modulating agents across several therapeutic indications. The Company intends to pursue regulatory approval of BHV-0223 for ALS in the United States under Section 505(b)(2) of the U.S. Federal Food, Drug and Cosmetic Act. The FDA cleared the Company’s investigational new drug application (IND) for BHV-0223 in August 2015, and Biohaven has completed a pharmacokinetic study with this drug candidate in humans and is planning to launch a pivotal bioequivalence study in 2017.
About Orphan Drug Designation
The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. Orphan drug designation provides a drug developer with several benefits and incentives, including a period of orphan product exclusivity, which means that the FDA may not approve any other applications to market the same drug or biological product for the same indication for seven years, except in limited circumstances. Further information regarding Orphan Drug
Designation can be found at:
Biohaven is a privately-held biopharmaceutical company with particular expertise in late-stage clinical development, with a portfolio of multiple late-stage drug candidates. Biohaven has licensed intellectual property from Yale University, Catalent, ALS Biopharma LLC, Massachusetts General Hospital and two global pharmaceutical companies. The Company has advanced multiple drug candidates into the clinic and plans to commence pivotal trials of its lead drug candidate in early 2017.
This news release includes forward-looking statements within the meaning of the U.S. federal and Canadian securities laws. 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 regarding the Company’s plans and objectives, expectations and assumptions of management are forward-looking statements. The use of certain words, including the words “estimate,” “project,” “intend,” “expect,” “believe,” “anticipate,” “will,” “plan,” “could,” “may” and similar expressions are intended to identify forward-looking statements. Examples of forward-looking statement in this news release include, among others, statements regarding the future development, performance and regulatory approval of BHV-0223 and the expected timing of future trials. The Company may not actually achieve the plans, intentions or expectations disclosed in the forward-looking statements and the Company’s actual results could differ materially from those indicated in the forward-looking statements. 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 receipt of regulatory approvals, the performance of the Company’s candidates in future trials and market conditions. 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.
Dr. Vlad Coric
CEO, Biohaven Pharmaceutical Holding Company Ltd.