About the REACH Trial
REACH (NCT05397470) is a Phase 3 multi-center, randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of losmapimod
for the treatment of FSHD. The trial enrolled 260 patients who were randomized 1:1 to receive either losmapimod, administered orally as a 15 mg tablet twice a day, or placebo over a 48-week treatment
period. The primary endpoint was the absolute change from baseline in Reachable Workspace (RWS). Secondary endpoint measurements included Muscle Fat Infiltration (MFI) as measured by MRI, shoulder abductor strength as measured by hand-held
dynamometry, Patient Global Impression of Change (PGIC), and the Neuro QoL Upper Extremity.
About Losmapimod
Losmapimod is a selective p38α/ß mitogen activated protein kinase (MAPK) inhibitor. Fulcrum exclusively
in-licensed losmapimod from GSK following Fulcrum’s discovery of the role of p38α/ß inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Although
losmapimod had never previously been explored in muscular dystrophies, it had been evaluated in more than 3,600 subjects in clinical trials across multiple other indications with no safety signals attributed to losmapimod. Losmapimod has been
granted U.S. Food and Drug Administration (FDA) Fast Track designation and Orphan Drug Designation for the treatment of FSHD.
About FSHD
FSHD is a serious, rare, progressive and debilitating disease for which there are no approved treatments. It is characterized by fat
infiltration of skeletal muscle leading to muscular atrophy involving primarily the face, scapula and shoulders, upper arms, and abdomen. Impact on patients includes relentless and accumulating muscle and functional loss impacting their ability
to perform activities of daily living, loss of upper limb function, loss of mobility and independence, and chronic pain. FSHD is one of the most common forms of muscular dystrophy and has an estimated patient population of 30,000 in the
United States alone.
About Fulcrum Therapeutics
Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically
defined rare diseases in areas of high unmet medical need. Fulcrum’s two lead programs in clinical development are losmapimod, a small molecule in development for the treatment of facioscapulohumeral muscular dystrophy (FSHD), and pociredir, a
small molecule designed to increase expression of fetal hemoglobin and in development for the treatment of sickle cell disease (SCD). Fulcrum uses proprietary technology to identify drug targets that can modulate gene expression to treat the known
root cause of gene mis-expression. For more information, visit www.fulcrumtx.com and follow us on Twitter/X (@FulcrumTx) and LinkedIn.
Forward-Looking
Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of
1995 that involve substantial risks and uncertainties. All statements, other than statements of historical fact, contained in this press release are forward-looking statements, including express or implied statements regarding suspending losmapimod
development and the future of the program; completing a full evaluation of the REACH data and sharing the same; intended use of cash and cash equivalents and marketable securities; further advancement of pociredir and Fulcrum’s earlier stage
program for DBA and other discovery programs; among others. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,”
“plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not
all forward-looking statements contain these identifying words. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual
results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with continued development of losmapimod; resuming
development of losmapimod following an unsuccessful Phase 3 trial; sufficiency of Fulcrum’s cash resources; Fulcrum’s ability to advance any earlier stage product candidates in or into clinical trials; initiating and enrolling clinical
trials on the timeline expected or at all; replicating in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials; obtaining, maintaining or protecting intellectual property rights related to any product
candidates; obtaining and maintaining necessary approvals from the FDA and other regulatory authorities; managing expenses; managing executive and employee turnover; and raising the substantial additional capital needed to achieve its business
objectives, among others. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Fulcrum’s actual results to differ from those contained in the forward-looking statements, see the “Risk
Factors” section, as well as discussions of potential risks, uncertainties, and other important factors, in Fulcrum’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in
this press release represent Fulcrum’s views as of the date hereof and should not be relied upon as representing Fulcrum’s views as of any date subsequent to the date hereof. Fulcrum anticipates that subsequent events and developments will
cause Fulcrum’s views to change. However, while Fulcrum may elect to update these forward-looking statements at some point in the future, Fulcrum specifically disclaims any obligation to do so.
Contact:
Chris Calabrese
LifeSci Advisors, LLC
ccalabrese@lifesciadvisors.com
917-680-5608
