DMD treatment SAT-3247 moves toward 1st trial
Satellos seeks approval to start safety, efficacy trial in Australia
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Satellos Bioscience is gearing up to launch a Phase 1 clinical trial of its Duchenne muscular dystrophy (DMD) treatment SAT-3247.
The company said it has submitted an application to authorities in Australia seeking permission to start the trial, which is expected to test the safety and pharmacological properties of the oral treatment in healthy volunteers. Assuming authorities give the go-ahead, Satellos hopes to start the trial in the next few months, and if all goes well, to start trials of SAT-3247 in DMD patients by early 2025.
“Our team is proud to have reached this important milestone on the timeline we have been working towards for the past year and see this achievement as a catalyst towards a steady cadence of milestones to come,” Frank Gleeson, co-founder and CEO of Satellos, said in a company press release.
Satellos’ application to start the first trial is based on a package of preclinical data testing the therapy’s efficacy and safety in laboratory and animal models.
“We are pleased with the results from our preclinical studies and look forward to initiating clinical development of SAT-3247,” said Phil Lambert, PhD, chief scientific officer at Satellos. “Prior to submitting our regulatory documentation, we conducted our preclinical and toxicology studies to the standards of relevant global regulatory bodies. Thus, we expect to be able to leverage these results for additional Phase 1 and subsequent clinical trials in Australia and further jurisdictions including the United States and Canada, where we plan to advance into trials with DMD patients.”
DMD treatment repaired muscle in dogs
DMD is caused by mutations in the gene that encodes dystrophin, a protein that’s vital for maintaining muscle health. Lacking functional dystrophin, DMD patients’ muscles accumulate damage over time, leading to symptoms like weakness and wasting.
When muscles are damaged, muscle stem cells are normally able to activate to help regenerate and repair muscle tissue. But data suggest this process is defective in people with DMD. SAT-3247 is a small molecule designed to boost the activity of these stem cells to help promote muscle regeneration. The investigational therapy has shown promise in mouse models of DMD as well as another form of muscular dystrophy.
Satellos recently announced new data from preclinical tests of SAT-3247 in a dog model of DMD. Findings showed that, after two weeks of treatment, muscle regeneration more than quadrupled, while muscle force doubled.
“As Satellos’ first results in a large animal study, we are pleased that these findings continue to validate the improvement in muscle repair and regeneration that we have consistently seen in the [DMD] mouse model,” Gleeson said in a separate press release. Gleeson said the results “further support that SAT-3247 treatment may be capable of restoring muscle repair and regeneration that is impaired in people living with Duchenne.”
Lambert said the findings, while preliminary, “further build and support our understanding of the unique mechanism of action of SAT-3247.”
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