- Avidity Biosciences Inc RNA stock is down over 16% Thursday following its decision to conclude the Phase 1/2 MARINA trial with the 38 participants enrolled at 1mg/kg, 2mg/kg, and 4mg/kg of AOC 1001 and not to move forward with the 8 mg/kg dose in myotonic dystrophy type 1 (DM1).
- In September, FDA placed a partial clinical hold on new participant enrollment in the MARINA trial in response to a serious adverse event reported in a single participant in the 4mg/kg cohort.
- Avidity concluded that the participant most likely experienced an extremely rare neurological event comprising bilateral ischemia in the region of the lateral geniculate nuclei in the thalamus with subsequent hemorrhagic transformation.
- Avidity cannot identify a plausible biological link to any component of AOC 1001, the AOC platform, the transferrin receptor delivery mechanism, or the reduction of DMPK. It is important to note that AOC 1001 does not cross the blood-brain barrier.
- Avidity will continue to dose the participants at 2 mg/kg and 4 mg/kg of AOC 1001 in the MARINA open-label extension study (MARINA-OLE), with a first look at the data expected by the end of 2023.
- In December 2022, the company announced preliminary data showing that AOC 1001 delivered siRNA to skeletal muscle and produced meaningful DMPK reduction in 100% of participants, with a 45% mean reduction in DMPK after a single dose of 1 mg/kg or two doses of 2 mg/kg.
- Price Action: RNA shares are down 16.6% at $16.85 on the last check Thursday.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
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