More positive data announced in clinical trial for myotonic dystrophy type 1 therapeutic
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A Southern California biopharmaceutical company has announced positive data from a phase 1/2 clinical trial of its proprietary monoclonal antibody, AOC 1001, for those with myotonic dystrophy type 1.
According to a release from Avidity Biosciences, data from the MARINA study and its open-label extension, MARINA-OLE, which was presented at the 28th Annual Congress of the World Muscle Society in South Carolina, found treatment with the novel therapeutic led to improved functional measures including hand grip, muscle strength and patient reported outcomes. The new data bolsters previously reported positive outcomes in myotonia, muscle strength and mobility.
“With this robust data package, we are finalizing the phase 3 study design and global regulatory path for AOC 1001 and look forward to sharing a first look at efficacy data from the MARINA-OLE study in the first half of 2024,” Avidity President and CEO Sarah Boyce said in the release.
Avidity said in the release that long-term safety data from more than 200 infusions totaling 46.2 patient-years of exposure demonstrated AOC 1001 has favorable safety and tolerability profiles, with most reported adverse events labeled as “mild to moderate.”
In May, the FDA eased the partial clinical hold on AOC 1001, allowing Avidity to double the number of participants who received 4 mg/kg of AOC 1001 in the extension study from 12 to 24. Data from the 12 participants who were dose-escalated from 2 mg/kg to 4 mg/kg of AOC 1001 as part of the easement of the partial clinical hold showed no neurological events and no MRI changes following the change.
“The AOC 1001 data continues to be quite remarkable with consistent improvements across multiple functional endpoints,” Nicholas E. Johnson, MD, MSci, FAAN, lead investigator of the MARINA trial and study presenter, said in the release. “The AOC 1001 functional data coupled with the long-term favorable tolerability and safety data provide us with hope that AOC 1001 has the potential to help patients with DM1, who are in desperate need of treatments.”