Catabasis has announced its Phase 3 PolarisDMD trial discontinuation with edasalonexent, an anti-inflammatory medication under investigation for Duchenne muscular dystrophy.
The stop to the PolarisDMD trial was due to a lack of efficacy (no benefit compared to placebo) and not any safety issues. Catabasis is extremely disappointed by this outcome and thanks to the Duchenne community and all those who participated in the trials. This news is a reminder of the uncertainty we face when participating in clinical trials; however, the company is committed to sharing its data, which will provide an essential contribution to our overall knowledge of Duchenne and its natural history.
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 tested the potential to decrease inflammation and fibrosis, promote muscle regeneration, and slow disease progression. Edasalonexent was being developed as both a monotherapy and for use with other therapies, including exon-skipping treatments 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 might not address the multiple underlying defects causing 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, allowing bioactives to reach their targets more efficiently and have greater efficacy.