Avidity Biosciences, Inc. (NASDAQ:RNA) released on Monday del-zota topline data from the Phase 1/2 EXPLORE44 trial in people living with Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44).
Del-zota is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal and cardiac muscle tissue to specifically skip exon 44 of the dystrophin gene and enable the production of near-full-length dystrophin.
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The data presented highlighted the consistent data across all parameters in both the 5 mg/kg and 10 mg/kg cohorts of del-zota, including:
Based on the consistent data between the 5 mg/kg every six weeks and the 10mg/kg every eight weeks groups across all parameters, Avidity has selected the dose of 5 mg/kg every six weeks of del-zota for the Biologics License Application (BLA) submission and future clinical studies.
Participants receiving the 10 mg/kg dose in the EXPLORE44-OLE trial are being transitioned to 5 mg/kg every six weeks.
The company plans to present functional data in the fourth quarter of 2025 and is on track for a BLA submission by the end of 2025 for accelerated approval of 5 mg/kg of del-zota every six weeks in DMD44.
Following alignment with the FDA on the accelerated approval path late last year, including dose selection, Avidity’s commercial preparations for a potential U.S. launch of del-zota in DMD44 are well underway.
RNA Price Action: Avidity Biosciences stock is up 6.56% at $33.15 at publication Monday.
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