SAN DIEGO - Avidity Biosciences, Inc. (NASDAQ:RNA) has announced the acceleration of its global Phase 3 HARBOR study to the second quarter of 2024, following regulatory agreement on the study design for Delpacibart etedesiran (AOC 1001), a potential treatment for myotonic dystrophy type 1 (DM1).
The study will focus on the primary endpoint of video hand opening time (vHOT) and secondary endpoints including muscle strength and activities of daily living.
The company, which specializes in RNA therapeutics, reported that AOC 1001 demonstrated consistent and durable improvements in myotonia, muscle strength, and activities of daily living in people with DM1 in long-term data from the MARINA open-label extension (MARINA-OLE) trial. Compared to END-DM1 natural history data, results showed a reversal of disease progression in multiple functional measures.
John W. Day, MD, PhD, from Stanford University School of Medicine and an investigator in the MARINA and MARINA-OLE trials, noted the long-term data from MARINA-OLE indicating that the investigational treatment improved measures of disease progression in DM1 patients compared to natural history data.
Avidity's President and CEO, Sarah Boyce, expressed gratitude to the DM1 community for their support and highlighted the significance of initiating the global pivotal study for del-desiran, which is the approved international nonproprietary name for AOC 1001.
The MARINA-OLE study, which continues to assess the safety, tolerability, and efficacy of del-desiran, has shown favorable safety data with no serious adverse events or discontinuations reported. The study involves participants who previously took part in the Phase 1/2 MARINA clinical trial and are now receiving quarterly doses of del-desiran.
Avidity also hosted an investor and analyst event series on Monday to discuss the new long-term data from the MARINA-OLE trial. The company's mission is to improve lives by delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs). Avidity is leading clinical development programs for rare muscle diseases, with del-desiran being its lead product candidate.
The information in this article is based on a press release statement from Avidity Biosciences, Inc.
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