FDA Slaps Clinical Hold On Avidity's Early-Stage Myotonic Dystrophy Trial

FDA Slaps Clinical Hold On Avidity's Early-Stage Myotonic Dystrophy Trial
  • The FDA has placed a partial clinical hold on new participant enrollment in Avidity Biosciences Inc's RNA Phase 1/2 MARINA trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1). 
  • DM1 is a type of muscular dystrophy, a group of genetic disorders that cause progressive muscle loss and weakness.
  • Close to 40 participants have currently enrolled in the MARINA and MARINA open-label extension (MARINA-OLE) trials.
  • The partial clinical hold is in response to a serious adverse event reported in a single participant in the MARINA study's 4mg/kg cohort. 
  • Related: Chardan Initiates Coverage On These 'Intriguing Small-Cap Stories".
  • Avidity is working closely with the FDA and the trial investigator to assess the cause of this event.
  • Avidity remains on track to conduct a preliminary assessment of safety, tolerability, and key biomarkers in approximately half of the study participants in the MARINA trial in Q4 of 2022. 
  • Avidity received IND clearance from the FDA to proceed with the clinical trial of AOC 1020 for facioscapulohumeral muscular dystrophy and AOC 1044 for Duchenne muscular dystrophy with mutations amenable to exon 44 skipping. 
  • These programs are now advancing into the clinic. 
  • Price Action: RNA shares are down 26.9% at $13.71 on the last check Tuesday.

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