Friday, September 27, 2019
Catabasis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, and the Jain Foundation, a non-profit foundation whose mission is to cure muscular dystrophies caused by dysferlin protein deficiency, announced a preclinical research collaboration to study edasalonexent (CAT-1004) in Dysferlinopathy. Dysferlinopathy (Limb-girdle muscular dystrophy type 2B / Miyoshi myopathy) is a serious rare disease that causes progressive muscle weakness for which there are currently no approved treatment options. Edasalonexent is in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) and has the potential to benefit patients with other diseases, such as Dysferlinopathy.
In Dysferlinopathy, muscles lack dysferlin and as a result NF-kB is chronically activated. Edasalonexent, an oral small molecule designed to inhibit NF-kB, has the potential to slow disease progression in dysferlin-deficient populations. Under this collaboration, Catabasis and the Jain Foundation are conducting a preclinical study to evaluate the potential of edasalonexent as a therapeutic intervention for Dysferlinopathy by measuring disease progression in dysferlin-deficient mice treated with edasalonexent. The study will utilize magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) to measure muscle volume, fat accumulation, and other changes in the dysferlin-deficient mice. Initial results are expected in the first half of 2020.
“We look forward to working with Catabasis to advance research for Dysferlinopathy,” said Laura Rufibach, Ph.D., and Doug Albrecht, Ph.D., Co-Presidents of the Jain Foundation. “Patients with Dysferlinopathy (LGMD2B / Miyoshi myopathy) experience a progressive and debilitating decline in muscle function which significantly impacts their lives. As there are currently no treatment options, we are excited to explore the potential of edasalonexent to benefit those living with this disease.”
“The chronic activation of NF-kB is a key driver in many neuromuscular disorders, including Duchenne muscular dystrophy. Evidence of NF-kB activation in Dysferlinopathy suggests a similar disease mechanism and opportunity for intervention. In the MoveDMD trial and open-label extension, treatment with edasalonexent slowed disease progression compared to the off-treatment control period. Through this collaboration, we look forward to learning more about the potential of edasalonexent in Dysferlinopathy, where, similar to Duchenne, there is also a significant unmet need,” said Andrew Nichols, Ph.D., Chief Scientific Officer at Catabasis Pharmaceuticals.
