Avidity announces results from EXPLORE44 and EXPLORE44-OLE trials

Avidity Biosciences (RNA) announced positive new data from participants treated continuously with del-zota for one year in the EXPLORE44 and EXPLORE44-OLE trials. These data demonstrated reversal of disease progression and unprecedented improvement compared to baseline and natural history across multiple functional measures. Additional data from the EXPLORE44 program will be presented at upcoming scientific congresses. DMD is a rare genetic condition characterized by progressive muscle damage and weakness beginning at a very young age due to the absence of dystrophin protein from birth. Del-zota is designed to deliver phosphorodiamidate morpholino oligomers, or PMOs, to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin mRNA and enable production of functional, near-full length dystrophin. Near-full length dystrophin retains key functional domains and may offer improved muscle protection for people living with DMD44. Data from EXPLORE44 Clinical Development Program: Trial participants treated with del-zota demonstrated statistically significant increases of approximately 25% of normal in dystrophin production and restored total dystrophin up to 58 percent of normal. Creatine kinase, or CK, levels rapidly reduced by greater than 80% compared to baseline and were sustained at near normal levels throughout the duration of evaluation with participants followed for up to 16 months. Additionally, 50% of participants had CK levels within the normal range at one year of treatment. A total of 17 participants who began on the del-zota treated arm of EXPLORE44 and continued into the EXPLORE44-OLE have been followed for approximately one year. Avidity remains on track to submit a BLA to the FDA at year end 2025. This will be the Company’s first of three planned BLA submissions over a 12-month period. Avidity continues to prepare for a confirmatory study to support full global approval.