Trial Use of Edasalonexent Not Likely to Raise COVID-19 Risk, Catabasis Reports

Clinical trial data suggest that edasalonexent — a potential Duchenne muscular dystrophy (DMD) therapy — does not affect the immune system’s ability to work as it should, making it unlikely to raise a person’s COVID-19 risk, Catabasis Pharmaceuticals said.

COVID-19 is believed to pose the greatest threat to older people and those with underlying conditions. In particular, a weakened immune system and pre-existing lung conditions are thought to increase susceptibility to the viral infection.

According to recent guidelines carried by Parent Project Muscular Dystrophy (PPMD), people with DMD are at a higher risk of COVID-19 complications if they are using steroids (which weaken the immune system), and have lung and heart problems.

Edasalonexent (formerly CAT-1004) is a small molecule designed to block the activity of the NF-kappa B pathway, which is involved in the breakdown of muscle fibers. It is believed that blocking this pathway will help preserve muscle function in DMD patients.

This investigational treatment was evaluated in the Phase 2 trial MoveDMD (NCT02439216), in which boys ages 4 to 8 were given edasalonexent or a placebo. Results at 72 weeks of treatment showed preserved muscle function, slower disease progression, less inflammation, and a good safety profile. Participants who completed MoveDMD were allowed to enroll in an ongoing open-label extension study (meaning all are given edasalonexent), called GalaxyDMD (NCT03917719).

The therapy is also being evaluated in a Phase 3 clinical trial called PolarisDMD (NCT03703882), testing edasalonexent’s effectiveness and safety in about 130 boys ages 4–7. Topline results are possible later this year. Patients in PolarisDMD will also have the opportunity to join GalaxyDMD for longer-term treatment.

In a Facebook post, Catabasis said it has not found safety concerns related to COVID-19 in its clinical studies of edasalonexent. These trials, as well as long-term toxicology studies testing edasalonexent at doses that exceed those given to patients, have found no evidence of immunosuppression or a rise in infections.

The company added that no participants in either of its ongoing trials are receiving steroid therapies.

Catabasis also said it will keep monitoring the boys’ safety closely, and that health authority recommendations should be followed to reduce infection risks to boys and their families.

Duchenne UK partnered with Catabasis earlier this year, providing financial support for a one-year Phase 2 trial testing edasalonexent in non-ambulatory boys and men with DMD.