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Avidity Biosciences Announces FDA Eases Partial Clinical Hold on AOC 1001 Providing a Clear Path Forward to Finalize Pivotal Dose and Phase 3 Design in Adults with Myotonic Dystrophy Type 1
Avidity plans to double the number of participants receiving 4 mg/kg of AOC 1001 in MARINA Open-Label Extension (MARINA-OLE™) study by dose escalating
About this update from Atrium Therapeutics, Inc.
[{"type":"text","content":"Avidity plans to double the number of participants receiving 4 mg/kg of AOC 1001 in MARINA Open-Label Extension (MARINA-OLE™) study by dose escalating participants currently on 2 mg/kg of AOC 1001\nFDA allows new participant enrollment for 2 mg/kg of AOC 1001\nFirst look at data from the MARINA-OLE study planned for the end of 2023\nSAN DIEGO, May 17, 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 announced that the U.S. Food and Drug Administration (FDA) has eased the partial clinical hold on AOC 1001, allowing Avidity to double the number of participants in the MARINA Open-Label Extension (MARINA-OLE™) study receiving 4 mg/kg of AOC 1001. The FDA is also allowing new participant enrollment for AOC 1001 at 2 mg/kg. Data from the MARINA-OLE study will be used to finalize the AOC 1001 pivotal dose and Phase 3 study design for adults with myotonic dystrophy type 1 (DM1), an underrecognized, progressive and often fatal neuromuscular disease with no approved treatment options.\n\n \n \n \n \n \n \n\n \n\"This positive step forward in our discussions with FDA provides the opportunity to gather additional data on the 2-4 mg/kg dose range of AOC 1001 while, in parallel, finalizing our Phase 3 study design and aligning with health authorities on a global regulatory path for AOC 1001,\" said Sarah Boyce, president and chief executive officer at Avidity. \"We recently announced positive topline data for AOC 1001 demonstrating functional improvement across multiple clinical outcome measures and look forward to sharing a first look at data from the MARINA-OLE study at the end of this year. We are focused on advancing AOC 1001 into a pivotal trial as quickly as possible as we know that the patient community is desperate for a treatment. We are extremely grateful to the DM1 community for their trust, support and ongoing partnership.\"\nThe easing of the partial clinical hold allows a number of current participants to be dose escalated to 4 mg/kg of AOC 1001 in the MARINA-OLE study and allows new participant enrollment at 2 mg/kg of AOC 1001. In September 2022, FDA placed a partial clinical hold on all new participant enrollment of AOC 1001 due to a rare serious adverse event reported in a...