Fulcrum Therapeutics Acquires Global Rights to Losmapimod, a Potential Disease-Modifying Therapyfor Facioscapulohumeral Muscular Dystrophy


Fulcrum plans to launch a Phase 2b clinical trial in FSHD in mid-2019
CAMBRIDGE, Mass., April 23, 2019 – Fulcrum Therapeutics, a biotechnology company focused on
discovering and developing therapies to rebalance gene expression, today announced an exclusive
worldwide license agreement with GlaxoSmithKline (GSK) for development and commercialization of the
investigational drug losmapimod. Fulcrum intends to advance losmapimod into a Phase 2b trial in the
rare and devastating genetic disease facioscapulohumeral muscular dystrophy (FSHD), for which there
are currently no approved treatments.
Under the terms of the agreement, as payment for the license, GSK received shares of Fulcrum
preferred stock representing a high single-digit ownership percentage of the company on a fully diluted
basis, and will be eligible to receive future milestone payments and royalties. Fulcrum obtained all
worldwide development and commercialization rights for losmapimod, as well as existing drug
substance and drug product materials for use in its clinical trials. Fulcrum also received a right of
reference to INDs filed with the FDA relating to losmapimod and an exclusive license to all related
patents and data, which build on Fulcrum-generated intellectual property.
“Losmapimod is a foundational clinical asset for Fulcrum that has the potential to become the first
approved therapy that targets the root cause of FSHD. Fulcrum believes losmapimod has the potential
to slow or halt the progressive muscle weakness that characterizes the condition, which would
significantly improve patients’ quality of life,” said Robert J. Gould, Ph.D., Fulcrum’s president and chief
executive officer. “The agreement shows confidence in our unique approach to rebalancing gene
expression in severe genetically defined disorders. We will work urgently to advance the compound
through the clinic.”
Fulcrum’s proprietary product engine identified inhibitors of p38/ mitogen activated protein kinase
(MAPK) as powerful inhibitors of DUX4 expression. DUX4 is the gene that is the root cause of FSHD, a
progressive muscle wasting disorder. Losmapimod is a selective p38/ MAPK inhibitor that GSK has
tested extensively in clinical trials, but never in muscular dystrophies. Fulcrum’s novel insight into the
DUX4 regulatory pathway led the team to review existing p38/ MAPK inhibitors, and Fulcrum
identified losmapimod as a compound with the potential to address the root cause of FSHD by
decreasing DUX4 expression.
GSK evaluated losmapimod in more than 3,500 healthy volunteers and patients in 24 clinical trials across
multiple indications, including several Phase 2 trials and a Phase 3 trial in acute coronary syndrome. The
data provide evidence that losmapimod is a well-tolerated agent. Fulcrum has conducted preclinical
testing of losmapimod in patient-derived cell models and observed precise and potent downregulation
of DUX4 expression and restoration of a healthy muscle phenotype without an effect on myogenesis.
Fulcrum has developed an extensive clinical trial network of physicians working on FSHD. An ongoing
natural history study of the disease is informing the clinical development plan. Fulcrum expects to
initiate a Phase 2b clinical trial of losmapimod in patients with FSHD at multiple clinical sites in the U.S.
and Europe in mid-2019.
About FSHD
FSHD, one of the most common muscular dystrophies, is a progressive, degenerative and profoundly
disabling disorder estimated to affect about 1 in 8,333 to 1 in 20,000 people globally. There are no
approved treatments. Symptoms typically arise in adulthood, often beginning with muscle weakness in
the face, leading to an inability to smile. The weakness progresses to the upper body and advances to
the lower limbs, leaving many patients unable to lift their arms above shoulder level or to rise from a
sitting position. People with FSHD often have difficulty performing daily tasks on their own and may
experience severe fatigue and pain. FSHD is caused by a single gene, DUX4, which is normally switched
off at the earliest stages of embryonic development. Patients with FSHD have a mutation that causes the
gene to remain “on” and to continue producing a protein toxic to muscle tissue.
About Losmapimod
Losmapimod is a selective p38/ mitogen activated protein kinase (MAPK) inhibitor initially developed
by GlaxoSmithKline and in-licensed by Fulcrum Therapeutics. Fulcrum identified the compound as a
potent regulator of the expression of the DUX4 gene, which causes FSHD. Losmapimod has been
evaluated in more than 3,500 healthy volunteers and patients in 24 clinical trials across multiple
indications, including in several Phase 2 trials and a Phase 3 trial. It has been shown to be generally welltolerated.
About Fulcrum Therapeutics
Fulcrum Therapeutics is discovering and developing small molecule therapies to treat genetically
defined diseases at their root cause by modulating the expression of the genes known to drive or
ameliorate disease. Fulcrum’s proprietary approach to studying disease biology in patient-derived and
other relevant human cell lines, coupled with a computational biology engine, generates valuable
insights into a wide array of genetically defined diseases. Please visit www.fulcrumtx.com.

Tuba Khan

Tuba Khan is an Editor and Digital Marketing expert at PharmaShots. She is curious, creative, and passionate about recent updates and innovation in Lifesciences industry. She covers Pharma, Medical devices, Diagnostics and Digital health segment. Tuba also has an experience of digital and social media marketing and can run the campaign independently. She is also certified as a Digital marketer and social media marketer. She can be contacted on tuba@pharmashots.com

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