Catabasis has provided an update on their “North Star Ambulatory Assessment in Duchenne Muscular Dystrophy” as presented at the Parent Project Muscular Dystrophy (PPMD) Conference in July 2020.
In Duchenne, the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. By inhibiting NF-kB, edasalonexent has the potential to decrease inflammation and fibrosis, promote muscle regeneration, and slow disease progression. Edasalonexent is being developed as both a monotherapy and for use with other therapies, including exon-skipping therapies and other dystrophin-targeted therapies in development such as gene therapy.
Catabasis is a community of experts from multiple disciplines who have come together with the common goal of bringing hope and life-changing therapies to patients and their families.
In 2008, they recognized that traditional approaches to drug discovery and development may not address the multiple underlying defects causing a disease, and founded Catabasis Pharmaceuticals. They set foot on a path forward leveraging a technology they now call their SMART (Safely Metabolized And Rationally Targeted) Linker drug discovery platform. With this platform, they have engineered molecules that simultaneously modulate multiple biological targets within one or more related disease pathways – thus allowing bioactives to reach their targets more efficiently and to have greater efficacy.