- Taldefgrobep alfa, a myostatin-targeting
biologic investigational agent, in Phase 3 development to increase
muscle mass for Spinal Muscular Atrophy patients now granted Fast
Track in addition to previously receiving Orphan Drug Designation
in the US
NEW
HAVEN, Conn., Feb. 21,
2023 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN;
"Biohaven") announced today that it received Fast Track designation
from the U.S. Food and Drug Administration (FDA) for taldefgrobep
alfa, a novel anti-myostatin adnectin, for the treatment of spinal
muscular atrophy (SMA). Fast Track designation enables important
new drugs to reach patients earlier by facilitating more frequent
communications with the FDA and expeditious review of a drug which
treats a serious condition and fills an unmet medical
need. Biohaven previously received orphan drug designation
from the FDA for taldefgrobep in the treatment of SMA.
SMA is a rare, progressively debilitating motor neuron disease
in which development and growth of muscle mass are compromised,
resulting in progressive weakness and muscle atrophy, reduced motor
function, impaired quality of life and often death. Inhibition of
myostatin, a naturally occurring protein that limits skeletal
muscle growth, an important process in healthy muscular
development, is a potential therapeutic target for SMA.
Taldefgrobep has the potential to be a novel therapy to be used
in combination with disease modifying therapies to enhance muscle
function by blocking myostatin activity. Taldefgrobep's novelty in
a field of myostatin inhibitors is based on its mechanism of
action. It binds to myostatin to both lower overall myostatin
levels and also function as a receptor antagonist, thereby blocking
myostatin signaling in skeletal muscles.
Lindsey Lee Lair, M.D., M.B.A.,
F.A.A.N, Vice President, Clinical Development, Biohaven commented,
"We are very pleased the FDA granted Fast Track designation for
taldefgrobep alfa for the treatment of SMA. Children and adults
living with SMA experience significant muscle weakness and
functional impairments affecting their quality of life daily, and a
substantial unmet medical need persists. We are excited about the
potential for taldefgrobep alfa to improve the lives of patients
and families affected by SMA."
Karen Chen, Ph.D., CEO,
SMA Foundation, added, "Fast Track
designation from the FDA underscores the high unmet medical need in
SMA and supports the need for additional novel combination
therapies in children and adults living with this progressive
neurologic disease. The SMA Foundation is heavily investing in
supporting the development of additional muscle and neuromuscular
regenerative medicine therapies that provide functional benefits to
patients. We are happy to see the commitment of health authorities
to rapidly advance the development of new therapies"
As a leader in innovative trials addressing neurodegenerative
diseases, Biohaven is currently enrolling a Phase 3 clinical trial
of taldefgrobep in SMA: A Study to Evaluate the Efficacy and Safety
of Taldefgrobep Alfa in Participants with Spinal Muscular Atrophy
(RESILIENT) (NCT05337553).
About Taldefgrobep alfa
Taldefgrobep alfa
(also known as BHV2000) 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.
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.
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 best-in-class
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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