Avidity Biosciences (NasdaqGM:RNA) Earnings Call Presentation

Del-zota Clinical Trial Results - Del-zota demonstrated unprecedented functional improvement on all key measures at one year across EXPLORE44® & EXPLORE44-OLE[18] - Creatine kinase (CK) levels rapidly reduced by >80% compared to baseline and sustained at near normal levels[41] - 50% of participants had CK levels within normal range at 1 year of treatment[41] - Dystrophin increase to up to 58% of normal[36] Safety and Tolerability - Del-zota continues to demonstrate favorable long-term safety and tolerability[27] - In EXPLORE44-OLE, 85% of participants experienced any adverse event (AE), 26% related to the study drug[27] - Serious adverse events occurred in 8% of participants, with 3% related to the study drug[27] - 3% of participants discontinued treatment due to adverse events[27] Regulatory and Commercialization - The company is on track for BLA submission by year end 2025 and launch in 2026[18] - The company is aligned on path for accelerated approval in the U S for DMD44, which affects approximately 900 people in the U S[14, 17]