Fulcrum Therapeutics to Host Key Opinion Leader Breakfast Symposium on Facioscapulohumeral Dystrophy (FSHD), on November 7, 2...
October 31 2019 - 8:00AM
Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage
biopharmaceutical company focused on improving the lives of
patients with genetically defined rare diseases, today announced
that it will host a key opinion leader (KOL) breakfast symposium
focused on understanding Facioscapulohumeral Dystrophy (FSHD)
genetics, biology, and clinical implications, on Thursday, November
7, 2019 from 8:30 a.m. to 10:30 a.m. ET in New York, NY. Fulcrum is
currently conducting Phase 2 trials investigating the safety,
tolerability, and efficacy of losmapimod to treat the root cause of
FSHD.
Guest speakers scheduled to present at the event include:
- Kathryn Wagner, M.D., Ph.D., Professor of
Neurology and Neuroscience at Johns Hopkins School of Medicine and
Director of the Center for Genetic Muscle Disorders Kennedy Krieger
Institute
- Peter Jones, Ph.D., Mick Hitchcock, Ph.D.
Endowed Chair in Medical Biochemistry and Associate Professor of
Pharmacology at University of Nevada, Reno School of Medicine
- Fran Sverdrup, Ph.D., Associate Professor of
Biochemistry and Molecular Biology at Saint Louis University,
School of Medicine
A live webcast of the presentation will be available through the
investor relations section of the Company's website at
https://ir.fulcrumtx.com/events-and-presentations. Following the
live webcast, an archived replay will also be available.
About FSHDFSHD is characterized by progressive
skeletal muscle loss that initially causes weakness in muscles in
the face, shoulders, arms and trunk, and progresses to weakness
throughout the lower body. Skeletal muscle weakness results in
significant physical limitations, including an inability to smile
and difficulty using arms for activities, with many patients
ultimately becoming dependent upon the use of a wheelchair for
daily mobility.
FSHD is caused by mis-expression of DUX4 in skeletal muscle,
resulting in the presence of DUX4 proteins that are toxic to muscle
tissue. Normally, DUX4-driven gene expression is limited to early
embryonic development, after which time the DUX4 gene is silenced.
In people with FSHD, the DUX4 gene is turned “on” as a result of a
genetic mutation. The result is death of muscle and its replacement
by fat, leading to skeletal muscle weakness and progressive
disability. There are no approved therapies for FSHD, one of the
most common forms of muscular dystrophy, with an estimated patient
population of 16,000 to 38,000 in the United States alone.
About LosmapimodLosmapimod is a selective
p38α/β mitogen activated protein kinase (MAPK) inhibitor that was
exclusively in-licensed by Fulcrum Therapeutics following Fulcrum’s
discovery of the role of p38α/β inhibitors in the reduction of DUX4
expression and an extensive review of known compounds. Utilizing
its internal product engine, Fulcrum discovered that inhibition of
p38α/β reduced expression of the DUX4 gene in muscle cells derived
from patients with FSHD. Although losmapimod has never previously
been explored in muscular dystrophies, it has been evaluated in
more than 3,500 subjects in clinical trials across multiple other
indications, including in several Phase 2 trials and a Phase 3
trial. No safety signals were attributed to losmapimod in any of
these trials. Fulcrum is currently conducting Phase 2 trials
investigating the safety, tolerability, and efficacy of losmapimod
to treat the root cause of FSHD.
About Fulcrum Therapeutics Fulcrum Therapeutics
is a clinical-stage biopharmaceutical company focused on improving
the lives of patients with genetically defined diseases in areas of
high unmet medical need, with an initial focus on rare diseases.
Fulcrum’s proprietary product engine identifies drug targets which
can modulate gene expression to treat the known root cause of gene
mis-expression. Please visit www.fulcrumtx.com.
Investor Contact:Christina Tartaglia Stern
Investor Relations, Inc.
212.362.1200christina@sternir.com
Media Contact: Lynn GranitoBerry & Company
Public Relationslgranito@berrypr.com212-253-8881
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