In a news release, biopharmaceutical company Fulcrum Therapeutics, Inc. (“Fulcrum”) shared that the first patient was dosed in the Phase 3 REACH study. Within the study, researchers are evaluating the safety, efficacy, and tolerability of losmapimod for patients with facioscapulohumeral muscular dystrophy (FSHD). As of July 5, 2022, the researchers have now dosed the first person in the study.
About Losmapimod
An article in Muscular Dystrophy News explains that losmapimod is:
an oral medication designed to block the activity of the proteins p38 alpha and p38 beta. Over 90% of FSHD patients carry mutations in the DUX4 gene, causing its abnormally high activity and, as a consequence, muscle degeneration and fat infiltration.
Therefore, losmapimod is designed to block p38 alpha and p38 beta, stopping overactivity and thus halting disease progression. This orally-administered therapy has received both Orphan Drug and Fast Track designations within the United States.
Currently, the Phase 3 REACH trial is working to better understand the impact of losmapimod on FSHD. Altogether, 230 patients will enroll. Researchers hope to understand how losmapimod reduces symptoms, improves muscle strength and function, and impacts shoulder and proximal arm mobility. Prior studies have showed that losmapimod treatment could help maintain muscle function.
If eventually approved, losmapimod would be the first approved treatment of its kind.
About Facioscapulohumeral Muscular Dystrophy (FSHD)
DUX4 gene mutations cause facioscapulohumeral muscular dystrophy (FSHD), one of nine forms of muscular dystrophy. Altogether, FSHD causes progressive muscle weakness and atrophy. It most often affects muscles in the shoulder blades, upper arms, and face. In rare cases, FSHD symptoms appear during infancy. However, in many cases, individuals begin experiencing symptoms prior to 20 years old. At first, some people may experience eye, abdominal, leg, upper arm, shoulder, and mouth weakness which often disproportionately affects one side of the body. Symptoms and characteristics associated with FSHD can include:
- Difficulty drinking from a straw or whistling
- Eye dryness, often caused from inability to fully close the eyes during sleep
- Scapular winging
- Foot drop
- Difficulty walking or climbing stairs
- Lordosis
- Mild hearing loss
- Trendelenburg gait
