Edasalonexent Clinical Development - Edasalonexent is a late-stage therapeutic candidate for Duchenne Muscular Dystrophy (DMD), designed to limit muscle degeneration, promote muscle regeneration, and reduce inflammation and fibrosis[7, 72] - The Phase 3 PolarisDMD trial enrolled 131 boys ages 4 to 7, with top-line results expected in Q4 2020[26, 30] - In the Phase 2 MoveDMD trial and open-label extension, all assessments of function stabilized on Edasalonexent compared to the off-treatment control period[48] - Edasalonexent has been well-tolerated in more than 150 years of cumulative patient exposure[25, 72] - A trial in non-ambulatory DMD patients is expected to initiate in 2021 with support from Duchenne UK, who granted over $600,000 in funding[71] Market Opportunity and Commercial Strategy - DMD affects 1 in 3,500-5,000 males worldwide, with a median life expectancy of approximately 30 years[8, 82, 83] - Approximately 40% of patients are diagnosed by age 4[82, 85] - The company has developed strong relationships with global Duchenne patient advocacy organizations[86] - Most US Duchenne patients have access to expert care and treatment through concentrated treatment centers[92, 94] - Edasalonexent aims to be a foundational therapy for all patients affected by Duchenne, regardless of dystrophin mutation, and can be used as mono- or potential combo-therapy[20, 99]
Catabasis Pharmaceuticals Inc (CATB) Investor Presentation - Slideshow