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."
About Biohaven
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.
Forward-Looking Statements
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
Vlad.Coric@biohavenpharma.com
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SOURCE Biohaven Pharmaceutical Holding Company Ltd.