NEW HAVEN, Conn., Feb. 10, 2020 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) today reported negative topline results from its Phase 3 clinical trial evaluating troriluzole compared to placebo for the treatment of patients with Generalized Anxiety Disorder (GAD). This eight-week trial randomized 402 adult patients equally at more than 45 centers in the United States. In this trial, troriluzole monotherapy at 100mg twice daily did not differentiate from placebo on the primary endpoint of the mean change from baseline on the Hamilton Anxiety Rating Scale (HAM-A) after eight weeks of treatment.
Vlad Coric, M.D., Chief Executive Officer of Biohaven, "While these efficacy results were disappointing and do not support continued development of troriliuzole as a monotherapy in GAD, we have multiple ongoing studies evaluating troriluzole in other disease indications and with different dosing paradigms. Since it is often difficult to predict clinical outcomes from preclinical and early clinical proof of concept studies, our strategy has been to test troriluzole in four distinct disorders where glutamate has been implicated in the underlying pathophysiology of the illnesses. We eagerly await topline data from our adjunctive therapy trial in OCD, and our symptomatic treatment trials in Alzheimer's disease and Spinocerebellar Ataxia. We thank the patients and clinical investigators who participated in the GAD program and sincerely appreciate all that they have done to see this study through completion."
After eight weeks of treatment, troriluzole treated subjects had a mean improvement change from baseline of -9.28 points [95% CI: -10.23 to -8.32] on the HAM-A total score versus -9.35 points [95% CI: -10.34 to -8.36] on placebo, p-value = 0.917. Troriluzole was well tolerated with a low discontinuation rate due to adverse events (troriluzole 4% versus placebo 4.5%).
Loren Aguiar, M.D., Vice President of Research and Development stated, "Although the efficacy results are disappointing, the team conducted a well-designed study that provided us with definitive data. We are grateful to the community of patients and researchers who participated in this trial and allowed us to efficiently enroll the study in under 10 months."
Biohaven has a deep and broad portfolio of assets in development with five ongoing Phase 3 trials across it its calcitonin gene-related peptide (CGRP) antagonists, glutamate modulators and myeloperoxidase inhibitor platforms. Biohaven continues to prepare for potential launch with its upcoming 1Q2020 PDUFA date for rimegepant and expects topline data from the rimegepant prevention trial in 1Q2020. Earlier this year, troriluzole dosed at 280mg successfully advanced past an interim futility analysis in its pivotal Phase 2/3 trial in Alzheimer's disease study performed after 100 subjects completed 6 months of treatment. Completion of the troriluzole in Alzheimer's Disease trial is expected in 4Q2020, and topline data is anticipated from the troriluzole adjunctive therapy obsessive compulsive disorder (OCD) trial in 2Q2020 and from the troriluzole in SCA trial in 2021. Additionally, Biohaven's verdiperstat trial in Multiple System Atrophy (MSA) is enrolling ahead of schedule with topline results expected in 2021.
Troriluzole is a third-generation prodrug and new chemical entity that modulates glutamate, the most abundant excitatory neurotransmitter in the human body. The primary mode of action of troriluzole is normalizing synaptic levels of glutamate. Troriluzole increases glutamate uptake from the synapse, by augmenting the expression and function of excitatory amino acid transporters (i.e., EAAT2) located on glial cells that play a key role in clearing glutamate from the synapse. More information about troriluzole can be found at the Company's website https://www.biohavenpharma.com/science-pipeline/glutamate/troriluzole
Biohaven is a clinical-stage biopharmaceutical company with a portfolio of innovative, late-stage product candidates targeting neurological diseases, including rare disorders. Biohaven has combined internal development and research with intellectual property licensed from companies and institutions including Bristol-Myers Squibb Company, AstraZeneca, Yale University, Catalent, Rutgers, and ALS Biopharma LLC. Currently, Biohaven's lead development programs include multiple compounds across its CGRP receptor antagonist, glutamate modulation, and myeloperoxidase inhibitor platforms. Biohaven's common shares are listed on the New York Stock Exchange and traded under the ticker symbol BHVN. More information about Biohaven is available at www.biohavenpharma.com.
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 expected enrollment for the Company's Phase 3 trial of troriluzole, the potential results of the Company's Phase 3 trial of troriluzole in GAD, the potential for the Phase 3 trial to be a pivotal trial, the role of glutamate in GAD, the possible benefits of troriluzole compared to current standard of care for GAD patients, as well as the timetable for the topline data and completion of trials, are forward-looking statements. The use of certain words, including the "believe" 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 future clinical success of troriluzole. Additional important factors to be considered in connection with forward-looking statements are described in the "Risk Factors" section of the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 28, 2019 and the Company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, filed with the Securities and Exchange Commission on November 1, 2019. 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.
For further information, contact: Dr. Vlad Coric Chief Executive Officer [email protected]
SOURCE Biohaven Pharmaceutical Holding Company Ltd.