NEW HAVEN, Conn., March 10, 2021 /PRNewswire/ -- Biohaven
Pharmaceutical Holding Company Ltd. (NYSE: BHVN) ("Biohaven" or the
"Company") announced today that more than 50% of participants have
been enrolled in the verdiperstat regimen of HEALEY ALS Platform
Trial, which is evaluating the efficacy of several treatments
including verdiperstat in people with amyotrophic lateral sclerosis
("ALS"). Verdiperstat is a potential first-in-class,
brain-penetrant, selective inhibitor of myeloperoxidase in
development by Biohaven for the treatment of neurodegenerative
diseases, including ALS and multiple system atrophy.
Irfan Qureshi, MD, Biohaven Vice
President and Verdiperstat Development Lead, stated, "We are
pleased that enrollment in the trial is progressing rapidly and
very grateful to our collaborators at the Healey Center and the ALS
research community for their leadership, boundless enthusiasm, and
perseverance. We are also deeply thankful to people with ALS and
their families for their commitment to the HEALEY ALS Platform
Trial."
The HEALEY ALS Platform Trial is a major innovation in the field
of ALS drug development that is aimed at accelerating the
identification of effective new treatments. The HEALEY ALS Platform
Trial is being conducted across over 50 sites by leading ALS
experts from the Healey Center in collaboration with the Northeast
ALS Consortium clinical trial network. In contrast to traditional
trials that typically evaluate only one drug at a time, platform
trials create an infrastructure for the ongoing study of multiple
investigational drugs in parallel thereby maximizing resources and
efficiency.
Merit Cudkowicz, MD, director of the Sean M. Healey & AMG
Center for ALS at Mass General and Chief of the Department of
Neurology, "We are very thankful that despite the challenges of
COVID-19, all collaborators involved in the HEALEY ALS Platform
Trial have come together to make sure people with ALS are safely
able to enroll in this groundbreaking trial."
The HEALEY ALS Platform Trial will study approximately 160
adults with ALS for each treatment. Participants will be randomized
in a 3-to-1 ratio to be treated with verdiperstat 600 mg oral
tablet twice daily or placebo for 24 weeks. The study's primary
efficacy endpoint will measure the change in disease severity from
baseline to week 24 on the ALS Functional Rating Scale-Revised
(ALSFRS-R) in patients receiving treatment versus placebo.
Secondary endpoints will include change in respiratory function,
muscle strength, and survival. More information about the HEALEY
ALS Platform Trial can be found at www.massgeneral.org/als.
ALS is a progressive, life-threatening and rare neuromuscular
condition that affects approximately 30,000 people in the United States. ALS is characterized by the
loss of motor neurons in the brain, brainstem, and spinal cord that
leads to progressive muscle weakness and difficulties in speaking,
swallowing, and breathing. There are currently limited treatment
options and no cure for ALS.
About Verdiperstat
Verdiperstat (BHV-3241) is an investigational first-in-class,
potent, selective, brain-penetrant, and myeloperoxidase (MPO)
enzyme inhibitor that Biohaven is developing for the treatment of
neurodegenerative diseases. Verdiperstat may help preserve
neurons through inhibition of MPO-induced pathological oxidative
stress and inflammation that contribute to cellular injury in
neurodegenerative diseases such as ALS and multiple system atrophy
(MSA). Biohaven licensed verdiperstat (BHV-3241) from AstraZeneca
in September 2018, where it was known
as AZD3241. More information about the HEALEY ALS Platform Trial
can be found at www.massgeneral.org/als and
clinicaltrials.gov/ct2/show/NCT04436510. More information about
verdiperstat can also be found at the Company's website:
www.biohavenpharma.com/science-pipeline/mpo/verdiperstat.
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 for
obsessive-compulsive disorder, Alzheimer's disease, and
spinocerebellar ataxia; and MPO inhibition for multiple system
atrophy and amyotrophic lateral sclerosis. More information about
Biohaven is available at www.biohavenpharma.com.
Forward-Looking Statements
This news release includes forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995.
The use of certain words, including "believe", "may" and "will" and
similar expressions, are intended to identify forward-looking
statements. These forward-looking statements involve substantial
risks and uncertainties, including statements that are based on the
current expectations and assumptions of Biohaven's management about
verdiperstat as a treatment for ALS. Forward-looking statements
include those related to: Biohaven's ability to effectively
commercialize verdiperstat, delays or problems in the supply or
manufacture of verdiperstat, complying with applicable U.S.
regulatory requirements, the expected timing, commencement and
outcomes of Biohaven's planned and ongoing clinical trials, the
timing of planned interactions and filings with the FDA, the timing
and outcome of expected regulatory filings, the potential
commercialization of Biohaven's product candidates, the potential
for Biohaven's product candidates to be first in class or best in
class therapies and the effectiveness and safety of Biohaven's
product candidates. 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. 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 March
1, 2021. 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.
NURTEC and NURTEC ODT are registered trademarks of Biohaven
Pharmaceutical Ireland DAC.
Biohaven Contact
Dr. Vlad Coric
Chief Executive Officer
Vlad.Coric@biohavenpharma.com
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SOURCE Biohaven Pharmaceutical Holding Company Ltd.