Avidity Biosciences Touts Positive Data From Early-Stage Duchenne Muscular Dystrophy Study Data, FDA Submission Expected By End Of 2025

Avidity Biosciences, Inc. 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:

• Targeted delivery of PMOs resulting in tissue concentrations of approximately 200nM in skeletal muscle;
• Statistically significant increases of approximately 40% in exon 44 skipping.
• Statistically significant increase of approximately 25% of normal in dystrophin production and restored total dystrophin up to 58% of normal.
• Reduction in creatine kinase levels to near normal with greater than 80% reductions compared to baseline:
  • Similarly, placebo participants demonstrated a reduction in creatine kinase levels to near normal upon treatment with del-zota.
  • Significant reductions in creatine kinase levels were sustained in the EXPLORE44-OLE trial with continued treatment up to one year.
• Del-zota demonstrated favorable safety and tolerability at both doses, with most treatment emergent adverse events (TEAEs) mild or moderate.

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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