Catabasis Pharma's DMD candidate successful in Phase 1

• Part A of a Phase 1/2 clinical, MoveDMD, assessing Catabasis Pharmaceuticals' (CATB +15%) Orphan Drug- and Fast Track-tagged CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD) successfully demonstrated an acceptable safety profile 17 ambulatory boys with DMD. Three doses of CAT-1004 (33 mg/kg/day, 67 mg/kg/day and 100 mg/kg/day) were generally well tolerated with no safety signals or serious adverse events observed and there were no drug discontinuations. The majority of adverse events were mild, with the most common being diarrhea. Plasma exposure levels associated with NF-kB inhibition were consistent with those previously observed in adults.
• The company intends to initiate Part B of MoveDMD in H1, a randomized, double-blind, placebo-controlled study that will assess CAT-1004's safety and efficacy over a 12-week period. The boys in Part A will be asked to participate in Part B.
• CAT-1004 is a small molecule that inhibits a protein called NF-kB, that plays a key role in muscle health.
• DMD-related tickers: (BMRN +1.9%)(SRPT +2.9%)(PFE -0.3%)(MRNA -5.5%)(PTCT -2.2%)(SMMT)