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FDA Grants Fast Track Designation to AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
LA JOLLA, Calif., Oct. 18, 2021 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of
About this update from Atrium Therapeutics, Inc.
[{"type":"text","content":"LA JOLLA, Calif., Oct. 18, 2021 /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 that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its lead program, AOC 1001, for the treatment of myotonic dystrophy type 1 (DM1).\n\n \n \n \n \n \n \n\n \nFast Track Designation enables more frequent interactions with the FDA to expedite the development and review process for drugs intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.\n\"DM1 is an underrecognized, progressive and often fatal disease with no therapeutic options. Fast Track designation for AOC 1001 underscores this unmet need and allows us to expeditiously work with FDA to potentially deliver this first-in-class therapy to people living with DM1 as quickly as possible,\" said Sarah Boyce, president and CEO of Avidity. \nAbout AOC 1001 and the Phase 1/2 MARINA™ Trial\nAOC 1001, Avidity's lead program utilizing its AOC platform, is designed to address the root cause of DM1 by reducing levels of a disease-related mRNA called DMPK. AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA. In preclinical studies, AOC 1001 successfully delivered siRNAs to muscle cells, resulting in durable, dose-dependent reductions of DMPK RNA across a broad range of muscles including skeletal, cardiac, and smooth muscles. In preclinical studies, AOC 1001 had a favorable safety profile that supports advancement into the clinic. The FDA has cleared Avidity to proceed with the Phase 1/2 MARINATM study of AOC 1001 in adults with DM1. FDA and EMA have granted Orphan Designation for AOC 1001 and the FDA has granted AOC 1001 Fast Track Designation.\nThe MARINA trial is a randomized, double-blind, placebo-controlled, Phase 1/2 clinical trial expected to enroll approximately 44 adults with DM1. The primary objective of this study is to evaluate the safety and tolerability of single and multiple ascending doses of AOC 1001 administered intravenously. The MARINA trial will assess the activity of AOC 1001 across key biomarkers, including spliceopathy, a key biomar...