HAVEN, Conn., July 7, 2022
/PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE:
BHVN) a commercial-stage biopharmaceutical company with a portfolio
of innovative, late-stage product candidates targeting neurological
and neuropsychiatric diseases, today announced that it has
commenced enrollment in a Phase 3 clinical trial assessing the
efficacy and safety of taldefgrobep alfa in Spinal Muscle Atrophy
Taldefgrobep is an investigational, muscle-targeted recombinant
protein with the potential to enhance muscle mass and strength in
people living SMA when used in combination with other approved
treatments. Taldefgrobep targets myostatin, a
natural protein that limits skeletal muscle growth, through two
mechanisms: lowering myostatin directly and blocking key
downstream signaling mechanisms. Myostatin inhibition is a
potential therapeutic strategy for children and adults with a range
of neuromuscular conditions for whom active myostatin can limit the
skeletal muscle growth needed to achieve developmental and
Irfan Qureshi, M.D., Senior Vice
President, Neurology at Biohaven, stated, "This is an important
milestone for the taldefgrobep program and for people living with
SMA. There have been great strides in advancing therapeutics in
this challenging disease. Yet, there remains a significant unmet
need to address the residual weakness and functional impairments,
such as difficulty walking, that are caused by the disease. We are
thankful for the SMA community that has welcomed and worked with us
to develop this patient-focused study."
RESILIENT, is a Phase 3 placebo-controlled, double-blind
trial designed to evaluate the efficacy and safety of taldefgrobep
as an adjunctive therapy for participants who are already taking a
stable dose of nusinersen or risdiplam or have a history of
treatment with onasemnogene abeparvovec-xioi, compared to
placebo. The study is not restricted nor limited to patients
based on ambulatory status or classification of SMA.
Han Phan, MD, Director of
Clinical Research, Rare Disease Research, said, "We are happy to
see progress being made with additional novel therapies focused on
neuromuscular health. Despite current therapies, a high unmet
need for safe and effective supportive treatments for SMA remains,
as many patients still experience significant weakness and reduced
levels of functioning. Additional research is needed to help
improve the quality of life for people living with SMA".
Biohaven expects to enroll approximately 180 patients in this
randomized, double-blind, placebo-controlled global trial.
Additional details about the trial can be found at SMATrials.com or
About Taldefgrobep alfa
Taldefgrobep alfa (also known as BMS-986089) is a modified
adnectin designed to specifically bind to myostatin (GDF-8).
Taldefgrobep is a fully human anti-myostatin recombinant protein
that lowers free myostatin and acts as an Activin 2b receptor antagonist with the
myostatin-taldefgrobep complex. Adnectins are an established
proprietary protein therapeutic class based on human fibronectin,
an extracellular protein that is naturally abundant in human serum.
The intrinsic properties of an adnectin align with the properties
needed to make a successful drug, including high potency,
specificity, stability, and favorable half-life.
About Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a rare genetic
neurodegenerative disorder characterized by the loss of motor
neurons, atrophy of the voluntary muscles of the limbs and trunk
and progressive muscle weakness that is often fatal and typically
diagnosed in young children. The underlying pathology of SMA is
caused by insufficient production of the SMN (survival of motor
neuron) protein, essential for the survival of motor neurons, and
is encoded by two genes, SMN1 and SMN2. In the U.S., SMA affects
approximately 1 in 11,000 births, and about 1 in every 50 Americans
is a genetic carrier.
Biohaven is a global 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
(EMA-approved as Vydura® for the acute treatment
of migraine with or without aura, and prophylaxis of episodic
migraine in adults who have at least four migraine attacks per
month) and a broad pipeline of late-stage product candidates across
five distinct mechanistic platforms: CGRP receptor antagonism for
the acute and preventive treatment of migraine and other
CGRP-mediated diseases; glutamate modulation for
obsessive-compulsive disorder and spinocerebellar ataxia;
myeloperoxidase (MPO) inhibition for amyotrophic lateral sclerosis;
Kv7 ion channel activators for focal epilepsy and neuronal
hyperexcitability, and myostatin inhibition for neuromuscular
diseases. More information about Biohaven is available at
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
taldefgrobep as treatment for patients with neuromuscular disease.
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 Biohaven's Annual Report
on Form 10-K filed with the Securities and Exchange Commission on
February 25, 2022, 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. Neuroinnovation is a trademark of
Biohaven Pharmaceutical Holding Company Ltd.
Vice President, Investor Relations
Sam Brown Inc.
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SOURCE Biohaven Pharmaceutical Holding Company Ltd.