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
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
By AP News
Published - Mar 30, 2023, 07:31 AM ET
Last Updated - Apr 12, 2024, 09:47 AM EDT
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 provides more information on the rare serious adverse event in a single participant that led to the partial clinical hold
AOC 1001 top-line safety and functional data to be presented at the American Academy of Neurology (AAN) Annual Meeting on April 27, 2023
Avidity to hold a webcast/conference call today at 8:00 a.m. ET/5:00 a.m. PT
