Under an exclusive worldwide license agreement, Fulcrum will develop and commercialize losmapimod, a potential disease-modifying therapy for facioscapulohumeral muscular dystrophy (FSHD).
Fulcrum is planning to advance losmapimod into a phase 2b trial in FSHD, a rare and devastating genetic disease with no approved treatments.
Under the deal, Fulcrum has received existing drug substance and drug product materials from GSK for use in its clinical trials.
The product engine of Fulcrum has identified inhibitors of p38α/β mitogen activated protein kinase (MAPK) as advanced inhibitors of DUX4 expression.
DUX4 is the gene responsible for FSHD, a progressive muscle wasting disorder. Losmapimod is a selective p38α/β MAPK inhibitor that GSK has assessed extensively in clinical trials, but not in muscular dystrophies.
GSK assessed losmapimod in over 3,500 healthy volunteers and patients in 24 clinical trials across multiple indications, including several phase 2 trials and a phase 3 trial in acute coronary syndrome.
Fulcrum has carried out preclinical testing of losmapimod in patient-derived cell models and noticed precise and potent downregulation of DUX4 expression and restoration of a healthy muscle phenotype without an effect on myogenesis.
As per terms of the deal, GSK has secured shares of Fulcrum preferred stock, which serves as a high single-digit ownership percentage of the company on a fully diluted basis.
GSK has also provided with an option to secure future milestone payments and royalties.
Under the deal, Fulcrum has received existing drug substance and drug product materials for use in its clinical trials.
The agreement also allows Fulcrum to have a right of reference to INDs filed with the FDA relating to losmapimod and an exclusive license to all related patents and data, which are based on Fulcrum-generated intellectual property.
Fulcrum president and CEO Robert Gould said: “Losmapimod is a foundational clinical asset for Fulcrum that has the potential to become the first approved therapy that targets the root cause of FSHD.
“Fulcrum believes losmapimod has the potential to slow or halt the progressive muscle weakness that characterizes the condition, which would significantly improve patients’ quality of life.”