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Avidity Biosciences Provides Regulatory Update on AOC 1001 for Myotonic Dystrophy Type 1 and Plans to Present Top-line Data from Phase 1/2 MARINA™ Trial at AAN Annual Meeting
Discussions with the FDA ongoing as Avidity submits emerging AOC 1001 data from the MARINA trial AOC 1001 continues to be generally well tolerated; Avidity
About this update from Atrium Therapeutics, Inc.
[{"type":"text","content":"Discussions with the FDA ongoing as Avidity submits emerging AOC 1001 data from the MARINA trial\nAOC 1001 continues to be generally well tolerated; Avidity provides more information on the rare serious adverse event in a single participant that led to the partial clinical hold\nAOC 1001 top-line safety and functional data to be presented at the American Academy of Neurology (AAN) Annual Meeting on April 27, 2023\nAvidity to hold a webcast/conference call today at 8:00 a.m. ET/5:00 a.m. PT\nSAN DIEGO, March 30, 2023 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today provided an update on the Phase 1/2 MARINA™ trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1), an underrecognized, progressive and often fatal neuromuscular disease with no approved treatment options. Discussions are ongoing with the U.S. Food and Drug Administration (FDA) regarding the partial clinical hold on new participant enrollment as Avidity continues to provide new AOC 1001 data as it emerges from the MARINA trial. AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA.\n\n \n \n \n \n \n \n\n \nIn September 2022, the FDA placed a partial clinical hold on new participant enrollment in the Phase 1/2 MARINA clinical trial after reviewing information provided by Avidity related to a serious adverse event reported in a single participant in the 4 mg/kg cohort of the MARINA study. As previously disclosed, the event was classified by the investigator as drug related. Avidity conducted a thorough analysis with the help of multiple independent experts and concluded that the participant most likely experienced an extremely rare neurological event comprising of bilateral ischemia in the region of the lateral geniculate nuclei in the thalamus with subsequent hemorrhagic transformation. The location in the lateral geniculate nuclei and the bilateral nature of the event is what makes this event extremely rare. After this extensive investigation, Avidity cannot identify a plausible biological link to any component of AOC 1001, the AOC platform, the transferrin receptor delivery mechanism or reduction of DMPK. It is importa...