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Investing.com -- Avidity Biosciences (NASDAQ:RNA) stock rose 2% after the company announced positive new data showing its experimental drug del-zota demonstrated reversal of disease progression in patients with Duchenne muscular dystrophy (DMD44).
The San Diego-based biopharmaceutical company reported that patients treated continuously with del-zota for approximately one year in the EXPLORE44 and EXPLORE44-OLE trials showed "unprecedented improvement" across multiple functional measures compared to baseline and natural history data.
Key functional improvements included Time to Rise from Floor (improving by 3.2 seconds), 4-Stair Climb (improving by 2.1 seconds), Performance of Upper Limb (improving by 1.5 points), and 10-Meter Walk/Run Test (improving by 0.7 seconds). In contrast, natural history comparison groups showed decline in all these measures.
The company also reported a rapid reduction in creatine kinase (CK) levels to near normal levels, maintained over 16 months of follow-up, with 50% of participants having CK levels within normal range at one year. Dystrophin production increased by approximately 25% of normal.
"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 the press release.
Del-zota is designed to deliver phosphorodiamidate morpholino oligomers to skeletal muscle and heart tissue to enable production of functional, near-full length dystrophin in patients with DMD44, a rare genetic condition characterized by progressive muscle damage.
The drug demonstrated a favorable long-term safety profile, with most adverse events being mild or moderate. Avidity remains on track to submit a Biologics License Application to the FDA for accelerated approval by the end of 2025.
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