- Taldefgrobep alfa is the only myostatin inhibitor in
clinical development that targets both myostatin and activin A
signaling, two key regulators of muscle mass and adipose
tissue
- RESILIENT, the pivotal clinical trial in spinal
muscular atrophy, was designed to test the efficacy and safety of
taldefgrobep alfa as adjunctive therapy to increase muscle in SMA
patients treated with standard of care nusinersen, risdiplam or
onasemnogene abeparvovec-xioi
NEW
HAVEN, Conn., Sept. 14,
2023 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN)
("Biohaven"), a global clinical-stage biopharmaceutical company
focused on the discovery, development and commercialization of
life-changing therapies to treat a broad range of rare and common
diseases, today announced completing enrollment in RESILIENT, the
Phase 3 pivotal study of taldefgrobep alfa in spinal muscular
atrophy (SMA).
Lindsey Lair, MD, Biohaven Vice
President, Clinical Development Lead for the SMA program stated,
"We are thrilled to complete enrollment in this pivotal trial for
SMA as it brings us one step closer to advancing a novel muscle
targeting therapy for patients with SMA. Despite recent advances in
SMA genetic treatments, patients still experience weakness and
impairments in quality of life that can be alleviated by enhancing
muscle mass and function, on top of what is delivered by current
standard of care treatments." Dr. Lair added, "Our team has been
inspired by the entire global SMA community of clinicians, patients
and family members – we are particularly grateful to the patients
and investigators who helped us complete enrollment ahead of our
timelines."
Taldefgrobep is an investigational, muscle-targeted recombinant
protein with the potential to enhance muscle mass and strength in
people living with SMA when used in combination with other approved
treatments. Taldefgrobep targets myostatin, a natural protein that
limits skeletal muscle growth. 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
functional milestones.
RESILIENT is a Phase 3 placebo-controlled, double-blind trial
designed to evaluate the efficacy and safety of taldefgrobep at 48
weeks as an adjunctive therapy for participants who are already
taking a stable dose of nusinersen, risdiplam and/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.
Taldefgrobep was granted EU Orphan Drug Designation, along with
both Fast Track and Orphan Drug Designation by the US FDA.
"While we have had good progress with 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. We appreciate the partnership of
investigators, patients, and researchers to expedite the
development of new efficacious therapies that will work in
combination with current options to help restore muscle strength
and function," said Kenneth Hobby,
President, Cure SMA.
Biohaven targeted randomizing approximately 180 patients in this
global trial conducted in 9 countries. The primary objective is to
determine the safety and efficacy of taldefgrobep alfa compared to
placebo after 48 weeks of subcutaneous administration as determined
by change from baseline in the 32 item Motor Function Measure
(MFM-32) total score. Additional details about the trial can be
found at SMATrials.com or
https://clinicaltrials.gov/NCT05337553.
About Taldefgrobep alfa
Taldefgrobep alfa
(BHV-2000) is a fully human recombinant protein specifically
designed to inhibit the signaling of myostatin and activin A; two
key regulators of muscle and adipose tissue. Taldefgrobep binds
myostatin and acts as an Activin 2b
receptor antagonist. Taldefgrobep's novel mechanism of action
offers the potential for meaningful reductions in fat mass,
increased lean mass, and improvements in multiple metabolic
parameters.
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. Globally, SMA affects approximately 1 in 11,000
births, and about 1 in every 50 individuals is a genetic
carrier.
About Biohaven
Biohaven is a global clinical-stage
biopharmaceutical company focused on the discovery, development and
commercialization of life-changing therapies for people with
debilitating neurological and neuropsychiatric diseases, including
rare disorders. Biohaven is advancing a pipeline of therapies for
diseases with little or no treatment options, leveraging its proven
drug development capabilities and proprietary platforms, including
Kv7 ion channel modulation for epilepsy and
neuronal hyperexcitability; glutamate modulation for
obsessive-compulsive disorder and spinocerebellar ataxia and
myostatin inhibition for neuromuscular diseases. Biohaven's
portfolio of early- and late-stage product candidates also includes
discovery research programs focused on TRPM3 channel activation for
neuropathic pain and CD-38 antibody recruiting, bispecific
molecules for multiple myeloma. More information about Biohaven is
available at www.biohaven.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
taldefgrobep alfa as treatment for patients with neuromuscular
disease. Investors are cautioned that any forward-looking
statements, including statements regarding the future development,
timing and potential marketing approval and commercialization of
development candidates are not guarantees of future performance or
results and involve substantial risks and uncertainties. Additional
important factors to be considered in connection with
forward-looking statements are described in Biohaven's filings with
the Securities and Exchange Commission, including within the
sections titled "Risk Factors" and "Management's Discussion and
Analysis of Financial Condition and Results of Operations". The
forward-looking statements are made as of the date of this new
release, 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:
Jennifer
Porcelli
Vice President, Investor Relations
jennifer.porcelli@biohavenpharma.com
201-248-0741
Mike Beyer
Sam Brown Inc.
mikebeyer@sambrown.com
312-961-2502
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