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Avidity Biosciences Announces Positive AOC 1001 Long-term Data Showing Reversal of Disease Progression in People Living with Myotonic Dystrophy Type 1 Across Multiple Endpoints; Same Key Endpoints Agreed for Phase 3 HARBOR™ Trial
Avidity accelerates global Phase 3 HARBOR™ study initiation to Q2 2024 following regulatory agreement on study design; primary endpoint is video hand opening
About this update from Atrium Therapeutics, Inc.
[{"type":"text","content":"Avidity accelerates global Phase 3 HARBOR™ study initiation to Q2 2024 following regulatory agreement on study design; primary endpoint is video hand opening time (vHOT) and secondary endpoints include muscle strength and activities of daily living\nDelpacibart etedesiran (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 MARINA-OLE™\nDelpacibart etedesiran (AOC 1001) data from MARINA-OLE showed reversal of disease progression in multiple functional measures in people living with DM1 compared to END-DM1 natural history data \nAvidity to host Volume 8 of investor and analyst event series via webcast March 4, 2024, at 8:00 a.m. ET\nSAN DIEGO, March 4, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced new positive long-term AOC 1001 data from the MARINA open-label extension (MARINA-OLE™) trial showing reversal of disease progression in people living with myotonic dystrophy type 1 (DM1) across multiple endpoints including vHOT, muscle strength and activities of daily living when compared to END-DM1 natural history data. These endpoints are the same key endpoints that will be used in the global Phase 3 HARBOR™ trial for people living with DM1. The primary endpoint in the Phase 3 HARBOR trial is video hand opening time (vHOT), and key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ. Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024.\n\n \n \n \n \n \n \n\n \nAvidity also announced delpacibart etedesiran as the approved international nonproprietary name of AOC 1001, abbreviated as del-desiran. Del-desiran (AOC 1001) is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.\n\"The long-term data from the MARINA-OLE study demonstrating that del-desiran improved measures of disease progression in DM1 patients compared to natural history data is remarkable,\" said John W. Day,...