NEW HAVEN, Conn., Nov. 15, 2021 /PRNewswire/ -- Biohaven
Pharmaceutical Holding Company Ltd. (NYSE: BHVN) ("Biohaven")
announced today that enrollment is now complete in the verdiperstat
arm of the HEALEY ALS Platform Trial. This first-ever platform
trial in ALS, sponsored by the Sean M. Healey & AMG Center for
ALS at Massachusetts General Hospital (MGH), is designed to
evaluate the safety and efficacy of multiple drug candidates in
people with Amyotrophic Lateral Sclerosis ("ALS"). Verdiperstat was
selected as one of the inaugural investigational treatments for the
HEALEY ALS Platform Trial which began enrolling patients in
July 2020 across 52 sites from the
Northeast ALS Consortium (NEALS). Topline results for verdiperstat
in the treatment of ALS are anticipated in mid-2022.
"We are enthusiastic about achieving this enrollment goal as it
translates to being one step closer to finding a life-changing
treatment for ALS. Our team is grateful to the ALS community and
people living with ALS who have trusted us in this
first-of-its-kind trial", said Merit Cudkowicz, MD, MSc, Principal
Investigator and Sponsor of the HEALEY ALS Platform Trial, Director
of the Sean M. Healey & AMG Center for ALS and chief of the
Department of Neurology at Mass General, and the Julieanne Dorn
Professor of Neurology at Harvard Medical
School.
Irfan Qureshi, MD, Biohaven Vice
President and Verdiperstat Development Lead, stated, "People with
ALS deserve novel and more effective therapeutic options. Biohaven
is very pleased that the verdiperstat arm of the trial is fully
enrolled. This important milestone brings us closer to knowing
whether verdiperstat can benefit people with ALS. We are deeply
grateful to the ALS community, especially the study participants
and their families, for their unwavering commitment to the
trial."
The HEALEY ALS Platform Trial, the first-ever platform trial in
ALS, is designed to evaluate multiple investigational treatments
simultaneously, thus accelerating the development of effective and
breakthrough treatments for people living with ALS. The
trial has enrolled approximately 160 adults with ALS into the
verdiperstat regimen. Participants are 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 is
measurement of 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
include change in respiratory function, muscle strength, and
survival. The study is designed to support a potential regulatory
filing and approval if the top line results are positive. More
information about the HEALEY ALS Platform Trial can be found at
www.massgeneral.org/neurology/als/research/platform-trial.
Calaneet Balas, President and CEO of The ALS Association, added,
"We celebrate the successful enrollment of the HEALEY ALS Platform
Trial as it moves us closer to potential new treatment options for
ALS. The ALS Association is proud to support the trial and grateful
to the HEALEY Center for championing this effort." More information
about the ALS Association can be found at www.als.org.
About ALS
ALS is a progressive, life-threatening, and rare neuromuscular
disease that affects approximately 30,000 people in the
United States. The median age of
onset is 55 years and average survival is 3-5 years after onset of
first symptoms. 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 in development by Biohaven 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 amyotrophic lateral sclerosis.
Biohaven licensed verdiperstat (BHV-3241) from AstraZeneca in
September 2018, where it was known as
AZD3241. More information about verdiperstat can also be found at
the Company's website: .
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 and preventive 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 and CGRP-mediated
neuroimmune/neuroinflammatory diseases; glutamate modulation for
obsessive-compulsive disorder, Alzheimer's disease, and
spinocerebellar ataxia; and myeloperoxidase (MPO) inhibition for
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", "continue", "may",
"will" and similar expressions, are intended to identify
forward-looking statements. 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. Various
important factors could cause actual results or events to differ
materially from those that may be expressed or implied by
forward-looking statements. 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 for the year ended December 31,
2020, filed with the Securities and Exchange Commission on
March 1, 2021, and in Biohaven's
subsequent filings with the Securities and Exchange Commission. 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.