Fulcrum Therapeutics Announces Phase 1 Clinical Trial Results

Fulcrum Therapeutics (NASDAQ:FULC) has announced preliminary results of its Phase 1 clinical trial of lsomapimod to treat facioscapulohumeral dystrophy (FSHD).

As quoted in the press release:

Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor. Fulcrum exclusively in-licensed losmapimod following Fulcrum’s discovery that the inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD, a disease which is caused by the mis-expression of DUX4 in skeletal muscle. Results were presented today by Michelle Mellion, MD, medical director at Fulcrum Therapeutics, in an oral presentation during the 24^th International Annual Congress of the World Muscle Society in Copenhagen, Denmark.

“Losmapimod has previously been shown to have adequate safety and tolerability in over 3,500 patients and healthy volunteers across multiple indications, with no safety signals attributed to the drug in those trials. Until now, losmapimod had not been tested in patients with FSHD, nor was it known if it was muscle-penetrant in humans,” said Dr. Mellion. “The preliminary results from our Phase 1 clinical trial of losmapimod in patients with FSHD indicate that losmapimod was generally well tolerated and achieved dose-dependent concentrations in plasma and muscle believed to be adequate for efficacy based on preclinical pharmacology studies.”

The primary objective of the trial was to investigate the safety and tolerability of losmapimod in healthy volunteers and in FSHD patients. The secondary objective was to evaluate repeated dose pharmacokinetics (PK) and target engagement (TE) in FSHD patients. In the first cohort, 10 healthy volunteers were randomized to a single oral dose of losmapimod (n=8) 7.5 mg followed by a single oral dose of 15 mg after a wash out period or to single oral dose placebo (n=2) in both dosing periods. In the second cohort, 15 FSHD patients were randomized and treated with placebo (n=3) or losmapimod 7.5 mg (n=6) or 15 mg (n=6) taken orally twice daily for 14 days.

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