Avidity Biosciences has had a bumpy ride with AOC 1001 after the FDA put the trial on partial hold in 2022. Image credit: JHVEPhoto/Shutterstock.

Avidity Biosciences has announced it has reached further endpoints from its Phase I/II trial of AOC 1001 in people living with myotonic dystrophy type one (DM1).

AOC 1001 is designed to address the root cause of DM1, an under-recognised, progressive and often fatal neuromuscular disease. It is the lead candidate for the company and works by leveraging its antibody oligonucleotide conjugate (AOC) platform. It comprises a monoclonal antibody that attaches to the transferrin receptor 1 (TfR1), conjugated with an siRNA that acts on DMPK messenger RNA.

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New trial data with met endpoints

The Phase I/II Marina trial was a randomised, double-blind, placebo-controlled study designed to evaluate the safety and tolerability of single and multiple ascending doses of AOC 1001 administered intravenously in adults with DM1. The Marina open-label extension (Marina-OLE trial) study assessed the long-term safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of the pipeline drug.

The AOC 1001 follow up data from the trials shows improvement in additional functional measures including hand grip, muscle strength and patient-reported outcomes, augmenting previously reported positive data showing improvements in myotonia, muscle strength and mobility.

AOC 1001 continues to demonstrate favourable safety and tolerability with mostly mild to moderate adverse events (AEs).

Trial data reported in April 2023 showed AOC 1001 was able to achieve directional improvements in various functional endpoint assessments of myotonia, strength and mobility.

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President of Avidith, Sarah Boyce, says that the most recent data will help the company finalise the Phase III study design. She also confirmed that the first look at efficacy data from the Marina-OLE study will be published in the first half of 2024.

AOC 1001 has had some difficulties along the way

In September 2022, the US Food and Drug Administration (FDA) placed a partial hold on the Marina trial after the company reported a serious adverse event in a single subject in the high dose (4mg/kg) arm of the trial. The FDA gave the company the green light again in May 2023.

GlobalData’s Pharmaceutical Intelligence Centre reports there are five marketed drugs for DM1, two in Phase III, four in Phase II and one in Phase I. If approved, GlobalData predicts a sales forecast of $379m for the pipeline drug in 2029. GlobalData is the parent company of Clinical Trials Arena.

Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.

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