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Also known as Losmapimod oral tablet
Losmapimod · 1 trial · 1 indication
Skeletal muscle biopsies were collected at Baseline and post-Baseline. DUX4 activity in skeletal muscle biopsies was assessed by measuring expression levels of a panel of 6 genes known to be regulated by DUX4. Expression levels of genes were measured using a validated quantitative RT-PCR assay and expressed as Ct (PCR cycles). Raw Ct for each of the 6 genes was normalized to the specified reference genes to generate a normalized Ct. The DUX4 activity is the average of the normalized Cts of each of the identified 6 genes, where the Ct for each of the 6 genes is first normalized to reference genes before the average is generated. Change in Baseline is calculated as \[average delta Ct across the 6 genes post-baseline\] minus \[average delta Ct across the 6 genes at baseline\].
| Arm | Type | Description |
|---|---|---|
| Treatment | EXPERIMENTAL | FSHD1 patients with genetic confirmation will receive Losmapimod 15 mg twice daily given as two 7.5 mg tablets per dose by mouth; for a total of 4 pills or 30 mg daily for 48 weeks. |
| Placebo | PLACEBO_COMPARATOR | FSHD1 patients with genetic confirmation will receive a Placebo twice daily given as two 7.5 mg tablets per dose by mouth; for a total of 4 pills or 30 mg daily for 48 weeks. |
| Name | Type | Description |
|---|---|---|
| Losmapimod oral tablet | DRUG | Losmapimod will be administered with food when possible. |
| Placebo oral tablet | DRUG | Placebo will be administered with food when possible |
Inclusion Criteria: * The patient must have consented to participate and must have provided signed, dated and witnessed IRB-approved informed consent form that conforms to federal and institutional guidelines. * Male or female subjects * Confirmed diagnosis of FSHD1 with 1 to 9 repeats via assessme...
Losmapimod is being developed for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder. It is an investigational oral small molecule and is not approved for any indication. Fulcrum Therapeutics is evaluating it in a Phase 2 clinical program.
Losmapimod targets MAPK11 and MAPK14, also known as p38 alpha and p38 beta mitogen-activated protein kinases. It acts as an inhibitor of these kinases. This mechanism is being studied for its potential effect in facioscapulohumeral muscular dystrophy.
Losmapimod is being developed by Fulcrum Therapeutics, Inc., which trades on the Nasdaq under the ticker FULC. The company is responsible for its clinical development in facioscapulohumeral muscular dystrophy.
Losmapimod is in Phase 2 clinical development for facioscapulohumeral muscular dystrophy. It is an investigational therapy and has not been approved by the FDA for any indication. The Phase 2 trial has been completed.
Losmapimod has been studied in a Phase 2 trial, NCT04003974, titled Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD). The trial was completed and enrolled 80 participants across the United States, Canada, France, and Spain.