Avidity Biosciences (NASDAQ:RNA) gained 2% on Wednesday after releasing fresh clinical data showing that its experimental therapy del-zota reversed disease progression in patients with Duchenne muscular dystrophy (DMD44).
The San Diego-based biotech said that patients who received del-zota continuously for about one year in the EXPLORE44 and EXPLORE44-OLE studies experienced “unprecedented improvement” across several functional measures compared with both baseline performance and natural history trends.
Reported gains included a 3.2-second improvement in Time to Rise from Floor, a 2.1-second gain in 4-Stair Climb, a 1.5-point increase in Performance of Upper Limb, and a 0.7-second boost in the 10-Meter Walk/Run Test. In contrast, natural history groups typically showed deterioration in these same metrics.
The drug also produced a rapid decline in creatine kinase (CK) levels, which moved close to normal and were sustained for 16 months of follow-up. After one year, half of the trial participants had CK levels within the normal range. Meanwhile, dystrophin production rose to roughly 25% of typical levels.
“These unprecedented data underscore the impact of our revolutionary targeted approach to deliver RNA directly to muscle,” said Sarah Boyce, President and CEO of Avidity, in a statement.
Del-zota is designed to transport phosphorodiamidate morpholino oligomers into skeletal and cardiac muscle, helping patients with DMD44 generate functional, near-full length dystrophin—a protein crucial for maintaining healthy muscle.
The therapy showed a strong safety profile, with most adverse events classified as mild to moderate. Avidity said it remains on track to file a Biologics License Application with the FDA for accelerated approval by the end of 2025.
Avidity Biosciences stock price
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