Pharmaceutical company, Catabasis, and American charity Jain Foundation have announced a preclinical research collaboration to study edasalonexent in dysferlinopathy (LGMD 2B). Edasalonexent works by turning off an enzyme called NF-kB, which is known to be overactive in many neuromuscular conditions, including dysferlinopathy and Duchenne muscular dystrophy. The drug has been shown to slow the progression of Duchenne and is currently being evaluated in a phase 3 trial.
The new study between Catabasis and Jain Foundation will evaluate the potential for edasalonexent as a treatment for dysferlinopathy by testing it in a mouse model. Results from this preclinical study are expected in the first half of 2020.
For more information, read this press release.