Catabasis Pharmaceuticals (CATB) Investor Presentation - Slideshow

Edasalonexent's Potential in DMD - Edasalonexent, an oral NF-κB inhibitor, shows promise in slowing disease progression and preserving muscle function in Duchenne Muscular Dystrophy (DMD)[5] - The Phase 3 PolarisDMD trial, designed for global registration, is fully enrolled, with top-line results expected in Q4 2020[5, 16] - The PolarisDMD trial enrolled 131 patients across 8 countries[19] - The Phase 3 PolarisDMD trial and Phase 2 MoveDMD trial have similar baseline characteristics, supporting the assumptions on which the Phase 3 trial was powered[36, 37] Clinical Trial Results - Phase 2 MoveDMD trial and open-label extension demonstrated clinically meaningful slowing of disease progression with edasalonexent[24] - Edasalonexent significantly improved the rate of change of MRI T2 compared to the off-treatment control period in the Phase 2 MoveDMD trial and open-label extension[28] - Muscle enzymes significantly decreased on edasalonexent, supporting a positive impact on muscle health in the Phase 2 MoveDMD trial and open-label extension[27] - Edasalonexent showed potential for positive cardiac effects in DMD, with a significant decrease in mean resting heart rate in the Phase 2 MoveDMD trial and open-label extension[31, 33] Market and Commercial Opportunity - DMD affects approximately 1 in 3,500-5,000 males worldwide[38] - Approximately 15,000 males in the US and 19,000 males in the EU are affected by DMD[39]