Avidity Reports Effective Delivery Of SiRNA Into Muscle; Shows Meaningful DMPK Reduction

(RTTNews) - Avidity Biosciences, Inc. (RNA) reported positive AOC 1001 data from the preliminary assessment of the Phase 1/2 MARINA trial showing the first-ever successful targeted delivery of RNA into muscle. AOC 1001 is the company's lead clinical program utilizing its AOC platform, and is designed to address the root cause of myotonic dystrophy type 1. In the trial, 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. Early signs of clinical activity was reported with improvement in myotonia in some participants.

"Utilizing our AOC platform technology, we have demonstrated for the first time ever the successful targeted delivery of siRNA to muscle in humans, a major breakthrough for the field of RNA therapeutics," said Art Levin, chief scientific officer at Avidity.

In September 2022, the FDA placed a partial clinical hold on new participant enrollment in the Phase 1/2 MARINA trial of AOC 1001 in adults with myotonic dystrophy type 1 due to a serious adverse event reported in a single participant in the 4 mg/kg dose cohort. The company continues to work to resolve the partial hold on new participant enrollment.

Shares of Avidity Biosciences are up 31% in pre-market trade on Wednesday.

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