The study assessed the safety and tolerability of single and multiple ascending doses of the therapy. Credit: National Cancer Institute on Unsplash.

Avidity Biosciences has reported positive topline results from the Phase I/II MARINA trial of AOC 1001 to treat myotonic dystrophy type 1 (DM1) patients.

The data were examined from the 3:1 randomised trial with 38 subjects, who received one dose of 1mg/kg AOC 1001, three doses of either 2mg/kg AOC 1001 or 4mg/kg of AOC 1001 or placebo.

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The results showed that AOC 1001 was able to achieve directional improvements in various functional endpoint assessments of myotonia, strength and mobility.

Myotonia was measured by video hand opening time (vHOT), while measures of strength covered the quantitative muscle testing (QMT) total score.

Mobility was evaluated by the 10m walk run test (10mWRT), as well as the timed up-and-go test.

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Significant reduction in the disease-related mRNA, called DMPK, and splicing changes were observed in patients treated with AOC 1001.

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The results also demonstrated favourable safety and tolerability of AOC 1001, with most adverse events being mild or moderate.

The MARINA randomised, double-blind, placebo-controlled study assessed the safety and tolerability of single and multiple ascending doses of AOC 1001 given intravenously to adults with DM1.

DM1 is an under-recognised, progressive and often fatal neuromuscular disease that currently has no approved therapies.

Avidity president and CEO Sarah Boyce said: “Our vision is to profoundly improve people’s lives by revolutionising the delivery of RNA therapeutics.

“These AOC 1001 data further demonstrate the broad and disruptive potential of our proprietary AOC platform to address targets and diseases previously unreachable with existing RNA therapies.

“We look forward to advancing all three of our clinical development programmes while we continue to expand our pipeline in skeletal muscle, cardiology, immunology and other diseases.”