― Losmapimod failed to show an improvement in relative surface area (RSA), a measure of reachable workspace (RWS), versus placebo at week 48
― Fulcrum to suspend future losmapimod development
―
Robust cash position enables Fulcrum to continue its mission to develop
therapies addressing diseases of high unmet need including pociredir
for the treatment of sickle cell disease (SCD)
Fulcrum
Therapeutics is a biopharmaceutical company that focuses on developing
small molecule therapies to treat rare diseases. The company was founded
in Cambridge, Massachusetts in July 2016 by Third Rock Ventures.
It went public on July 17, 2019.
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 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.
