Avidity stock rises after del-zota shows disease reversal in DMD44 trial

Published 10/09/2025, 12:30
© Reuters.

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.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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