NEW HAVEN, Conn., Aug. 3, 2020 /PRNewswire/ -- Biohaven
Pharmaceutical Holding Company Ltd. (NYSE: BHVN) ("Biohaven" or the
"Company") announced today that the first patients were enrolled in
the HEALEY ALS Platform Trial, which is evaluating the efficacy of
three 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, "Biohaven
continues to advance its novel pipeline across a variety of CNS
diseases, and we are so pleased to enroll the first patient in a
trial of verdiperstat in ALS. Given the tremendous burden of the
disease on people with ALS and their families, more effective
treatments are urgently needed. It is imperative that we rapidly
and efficiently study promising new treatments such as
verdiperstat, which targets brain inflammation. The HEALEY ALS
Platform Trial will allow us to efficiently enroll this trial and
determine if verdiperstat has the potential to help people with
ALS."
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, MSc, Director of the Healey Center and
Chief of Neurology at Mass General, commented, "I am
proud of our collaborators and grateful for the support of this
courageous community. We are excited to be at this point and
thankful to all teams involved for their invaluable input and
support."
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.
"By testing multiple drugs at the same time, this platform trial
will dramatically speed our search for effective ALS treatments for
everyone living with ALS. The ALS Association is proud to be
supporting this effort, and we are grateful to the Healey Center
for their leadership," said Calaneet Balas, President and CEO of
The ALS Association. More information about the ALS Association can
be found at www.als.org.
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. Biohaven recently announced completion of enrollment in
the M-STAR study, a Phase 3 clinical trial to assess the safety and
efficacy of verdiperstat in MSA. Additional details can be found at
clinicaltrials.gov/ct2/show/NCT03952806. Verdiperstat has
received Orphan Drug and Fast Track designations for MSA from the
US FDA, as well as Orphan Drug designation from the European
Medicines Agency. 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 can
be found at the Company's website:
www.biohavenpharma.com/science-pipeline/mpo/verdiperstat.
About Biohaven
Biohaven is a biopharmaceutical company focused on the
development and commercialization of innovative best-in-class
therapies to improve the lives of patients with debilitating
neurological and neuropsychiatric diseases. 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 myeloperoxidase inhibition for multiple
system atrophy and amyotrophic lateral sclerosis. For more
information, visit 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.
These forward-looking statements involve substantial risks and
uncertainties, including statements that are based on the current
expectations and assumptions of Biohaven's management. All
statements, other than statements of historical facts, included in
this press release, including the expected enrollment for
Biohaven's pivotal trial of verdiperstat, the potential results of
Biohaven's pivotal trial of verdiperstat in ALS, the potential role
of verdiperstat in ALS, the possible benefits of verdiperstat as a
disease-modifying therapy for ALS 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. Biohaven may not actually
achieve the plans and objectives disclosed in the forward-looking
statements and you should not place undue reliance on Biohaven'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 Biohaven's Annual Report on Form 10-K
filed with the Securities and Exchange Commission on February 26, 2020 and Biohaven's Quarterly Report
on Form 10-Q for the quarter ended March 31,
2020, filed with the Securities and Exchange Commission on
May 7, 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.
Biohaven Contact
Dr. Vlad Coric
Chief Executive Officer
Vlad.Coric@biohavenpharma.com
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SOURCE Biohaven Pharmaceutical Holding Company Ltd.