Avidity Biosciences Shares Gain On Encouraging Data From Early-Stage Myotonic Dystrophy Trial

Avidity Biosciences Inc RNA announced AOC 1001 data from the preliminary assessment of the Phase 1/2 MARINA trial demonstrating the first-ever successful targeted delivery of RNA into muscle.
AOC 1001, Avidity's lead clinical program utilizing its AOC platform, is designed to address the root cause of myotonic dystrophy type 1 (DM1).
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.
AOC 1001 produced a splicing improvement of 31% in a key set of muscle-specific genes in people with DM1, with a splicing improvement of 16% across a broad 22-gene panel.
Early signs of clinical activity, with improvement in myotonia, were demonstrated in adults with DM1 treated with AOC 1001.
In September, The FDA has placed a partial clinical hold on new participant enrollment in Avidity Biosciences Inc's RNA+ Free Alerts
Phase 1/2 MARINA trial of AOC 1001 in adults with 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.
The company expects to share topline data from the MARINA trial in 2023.
Price Action: RNA shares are up 60.10% at $17.52 on the last check Wednesday.