NEW HAVEN, Conn., Jan. 18, 2021 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) today announced that it has completed a focused analysis of the topline co-primary and key secondary data from its Phase 2/3 clinical trial of troriluzole as a symptomatic treatment in mild-to-moderate Alzheimer's disease (AD). Additional secondary and exploratory efficacy analyses and biomarker data including neurofilament light chain (NfL), neurogranin, tau and amyloid are still pending and expected in the coming months.
Troriluzole did not statistically differentiate from placebo at 48 weeks on the study's prespecified co-primary endpoints on the Alzheimer's Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB) in study participants with mild-to-moderate AD. Troriluzole also did not differentiate from placebo on the key secondary measure of hippocampal volume assessed by magnetic resonance imaging (MRI) in the overall population.
A subgroup analysis consisting only of mild AD patients did, however, reveal that troriluzole exhibited a nonsignificant numerical difference of a potential benefit at week 48 on both the ADAS-cog and hippocampal volumetric MRI. Troriluzole treated participants with mild AD (n=48) had a mean deformation change from baseline hippocampal volume of -1.1% versus -1.6% for placebo treated (n=49) participants [difference -0.5%, p-value = 0.2] at week 48. Although the numerical effects on the ADAS-cog and hippocampal MRI measured in mild AD patients suggests a potential biologic effect of troriluzole in patients with early stage disease, additional analyses and biomarker data will be informative and help determine whether any further study in early AD is warranted. Full study results, including additional secondary and exploratory outcomes, biomarker, and subgroup analyses, are expected in the coming months and will be presented at an upcoming scientific meeting.
Vlad Coric, M.D., Chief Executive Officer of Biohaven commented "Alzheimer's is a devastating disease and we must continue to advance the science to improve treatment outcomes for the many patients who are in need. Our goal was to efficiently assess whether troriluzole could benefit patients relatively late in the disease process with mild-to-moderate AD. This study was well-conducted but unfortunately it is clear from this preliminary analysis that troriluzole is not efficacious as a symptomatic treatment in a mixed population of patients with mild and moderate AD. We are awaiting additional biomarker data and other secondary analyses that will help inform whether troriluzole may provide benefit in early AD as a disease modifying agent. We would like to thank the Alzheimer's Disease Cooperative Study (ADCS) at the University of California, San Diego for its leadership in AD research and for their collaboration with the Biohaven R&D team to complete this trial. The ability to efficiently assess drug candidates for AD is essential to advancing the field. Biohaven remains deeply committed to developing novel medicines for people suffering from devastating neurological and neuropsychiatric diseases."
With regard to safety and tolerability, treatment with troriluzole at a dose of 280 mg once daily was relatively well tolerated and demonstrated a safety profile consistent with previous studies of troriluzole. Biohaven is planning to amend the ongoing long-term extension study of troriluzole in AD for mild AD patients to be able to continue treatment in order to gather additional clinical and biomarker data.
Howard Feldman MD, Professor of Neurosciences at the University of California, San Diego (UCSD) and Director of the ADCS, added, "Successfully undertaking this trial during the COVID-19 pandemic has required a remarkable commitment of our participants, research site staff and Principal Investigators for which we express our gratitude. We look forward to completing our analysis of the full dataset including all the clinical measures as well as the biomarker data to understand if further investigation of troriluzole in the early stages of Alzheimer's disease is warranted."
Irfan Qureshi, M.D., Vice President of Neurology at Biohaven, stated "We are extremely grateful to the community of patients and researchers who participated in this program and sincerely appreciate all that they have done to see this study through to completion. The topline data we released today only represent the early analyses that are available and multiple analyses including important biomarker data are expected in the near term. Additional planned analysis of the clinical outcome scales and biomarkers will help to inform our long-term development plans for troriluzole in AD."
Biohaven is a commercial-stage biopharmaceutical company with a portfolio of innovative, best-in-class therapies to improve the lives of patients with debilitating neurological and neuropsychiatric diseases, including rare disorders. Biohaven's neuroinnovation portfolio includes FDA-approved NURTEC® ODT (rimegepant) for the acute treatment of migraine and a broad pipeline of late-stage product candidates across three distinct mechanistic platforms: CGRP receptor antagonism for the acute and preventive treatment of migraine; glutamate modulation with troriluzole for spinocerebellar ataxia and obsessive-compulsive disorder; and MPO inhibition for multiple system atrophy and amyotrophic lateral sclerosis. More information about Biohaven is available at www.biohavenpharma.com.
About the Alzheimer's Disease Cooperative Study at UC San Diego
The Alzheimer's Disease Cooperative Study is an academic research organization located at UC San Diego which is dedicated to developing treatments for those at risk or affected by Alzheimer's disease. For upwards of 30 years, with funding from the National Institute on Aging, it has been a field leader in progressing potential new treatments through innovation in clinical trials with design, methods, and analytics. It has a trials coordinating center in San Diego and a network of participating sites across the United States.
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. 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 for the year ended December 31, 2019, filed with the Securities and Exchange Commission on February 26, 2020 and Biohaven's Quarterly Report on Form 10-Q for the quarter ended September 30, 2020 filed with the Securities and Exchange Commission on November 9, 2020. The forward-looking statements are made as of this date and Biohaven 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
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SOURCE Biohaven Pharmaceutical Holding Company Ltd.