WMS 2021: fairly encouraging results of losmapimod in facio-scapulo-humeral myopathy

A selective inhibitor of p38 α and β MAP kinases, losmapimod has been investigated in a phase IIAu cours d’un essai clinique de phase II, un médicament, dont il a été montré au préalable qu’il était bien toléré (au cours d’un essai de phase I) est administré à un groupe de malades dans le but de déterminer l’efficacité thérapeutique, les doses optimales et la sécurité du traitement (Quel est le mode d’administration et la dose maximale tolérée ?). La phase II peut être divisée en deux étapes : la phase IIa étudie le dosage et la phase IIb l’efficacité du traitement. trial called ReDUX4, randomized, double-blind, placebo-controlled, lasting 48 weeks in 80 adults with type 1 Facioscapulohumeral muscular dystrophy (FDH, or FSHD)/

Already mentioned at the FSHD society congress in June 2021, the results of ReDUX4 were presented in a new oral communication during the WMS 2021. It reported that the main endpoint of the trial had not been met (expression of DUX4 target genes), but positive effects of losmapimod on fatty infiltration of certain muscles on MRI, muscle strength (shoulders, ankle), muscle function and self-assessment by patients their progress under treatment, compared to placeboLe placebo est un produit dont la présentation est identique à celle d’un médicament, mais qui ne contient pas de principe actif. Dans un essai clinique, un placebo est utilisé pour mesurer l’action réelle du médicament testé, en comparant les effets du médicament testé contenant le principe actif et ceux du placebo.. 

An open-label extension of ReDUX4 is underway, scheduled to last until February 2025. Without waiting for its results, the Fulcrum therapeutics laboratory, which is developing losmapimod in FSH, plans to start discussions with the health authorities, starting with the FDA, at the end of the year. 

A phase 2, randomized, double-blind, placebo-controlled, 48-Week study of the efficacy and safety of losmapimod in subjects with FSHD. Tawil R, Wagner K on behalf of the ReDUX4 Study Group. Neuromuscular Disorders 31 (2021) S48-S49

 

ReDUX4 Data. Events and Presentations. Fulcrum Therapeutics, juin 2021

 

Fulcrum therapeutics announces results from ReDUX4 trial with losmapimod in Facioscapulohumeral Muscular Dystrophy (FSHD) demonstrating slowed disease progression and improved function. Fulcrum Therapeutics. Communiqué de presse du 24 juin 2021.