CureDuchenne, a nonprofit group working to advance research and improve care for people with Duchenne muscular dystrophy (DMD), is partnering with Catabasis Pharmaceuticals to host a webinar discussing promising results from the first part of two-part MoveDMD clinical trial.
The webinar is set for Wednesday, June 22, at 1 p.m. ET (10 a.m. PT) and pre-registration is required. Anyone interested can register through this link.
- Dr. Joanne Donovan, Catabasis’ chief medical officer and an associate clinical professor of Medicine at Harvard
- Debra Miller, CureDuchenne’s founder and chief executive officer
- Dr. Michael Kelly, CureDuchenne’s chief scientific advisor,
Besides presenting results from the trial’s now completed Part A, Dr. Donovan will also review the design and inclusion criteria of the Part B, which is ongoing and recruiting patients.
MoveDMD (NCT02439216) is a Phase 1/2 clinical trial testing the safety, efficacy, pharmacokinetics and pharmacodynamics of edasalonexent (formerly known as CAT-1004) in about 30 boys between the ages 4 and 7. Catabasis is conducting this trial, taking place at five sites in the U.S., and led a Phase 1 trial of the drug in adult patients. Move information about MoveDMD is also available on the company’s website.
Edasalonexent is an oral small molecule that inhibits activated NF-ĸB, a protein complex that controls DNA transcription, cytokine production, and cell survival. In boys with DMD, the absence of dystrophin combined with mechanical stress in muscles leads to robust NF-ĸB activation, driving muscle damage and preventing regeneration.
In animal models of DMD, edasalonexent was seen to inhibit activated NF-ĸB, and reduce muscle inflammation and degeneration. Some evidence of muscle regeneration and improved muscle function was also seen.
In addition to the webinar’s web link, registration is possible by contacting the organizers at (844) 337-8088. The conference ID is 25189236.
CureDuchenne was founded in 2003 to promote research and an eventual cure for DMD, a condition that affects one in every 3,500 boys.