Satellos asks FDA to clear Phase 2 trial of DMD treatment
Study aims to test SAT-3247 in DMD kids who are able to walk
Satellos Bioscience asked the U.S. Food and Drug Administration (FDA) for clearance to conduct a Phase 2 clinical trial testing SAT-3247 in children with Duchenne muscular dystrophy (DMD) who are able to walk.
The three-month study will randomly assign participants to SAT-324, taken as a pill, or a placebo. Its goal is to assess the therapy’s safety and tolerability, as well as its pharmacokinetics (how the drug moves into, through, and out of the body) and the most effective doses. Muscle biopsies and functional measures will also be used. Satellos said it expects the first patient to be enrolled by the end of the year.
The company’s investigational new drug application to the FDA follows promising data from a Phase 1 study (NCT06565208), which tested SAT-3247 in healthy volunteers and in adults with DMD.
Similar applications have been submitted in Australia, Serbia, U.K., and other countries in Europe. An open-label extension study lasting nine months is also planned.
“Filing our Phase 2 clinical trial submissions in the US and globally marks a major milestone for Satellos in advancing SAT-3247’s potential to treat Duchenne in a novel way,” Frank Gleeson, Satellos‘ CEO, said in a company press release.
Treatment addresses DMD’s ‘fundamental challenge’
Muscular dystrophy encompasses a group of genetic disorders that gradually weaken and deteriorate muscles. The most common type, DMD, is caused by mutations that result in virtually no dystrophin (a crucial protein that helps shield muscle fibers from damage during physical activity).
Under normal conditions, muscle stem cells are activated to repair damage. In DMD, this repair mechanism may not function properly. SAT-3247 is designed to enhance the activity of muscle stem cells, supporting regeneration and potentially slowing disease progression. Because it is independent of dystrophin, the treatment may be used on its own or in combination with other therapies. SAT-3247 targets AAK1, a protein that, according to Satellos, may replace the signal normally provided by dystrophin in muscle stem cells.
“Current therapies do not address the fundamental challenge in Duchenne, which we have identified — the body’s impaired muscle-repair process,” Gleeson said. “With SAT-3247, our goal is to re-boot that regenerative cycle with the potential to restore muscle, improve functional outcomes and truly change lives.”
The recently completed Phase 1 study consisted of two parts: a first part in healthy volunteers, followed by a second in five adults with DMD on steroids.
Healthy volunteers were assigned to one of five single ascending dose groups, followed by four multiple ascending dose groups. The five adults with DMD were all on their prescribed steroid regimens. They took SAT-3247 for 28 days.
Results showed that the therapy was safe and well tolerated. Its pharmacokinetics profile in DMD patients was consistent with results seen in healthy participants. The treatment showed early signs of effectiveness: DMD patients experienced roughly a twofold increase in grip strength and a 5% improvement in lung function, as tested with a measure called forced vital capacity.
Satellos plans to present new analyses from the Phase 1b clinical trial of SAT-3247 at the 30th Annual Congress of the World Muscle Society, scheduled for Oct. 7–11, in Vienna.
“With our focus on targeting the underlying defect in muscle repair and regeneration, Satellos brings a unique lens to the future of Duchenne treatment,” Gleeson said in a separate company press release. “We’re looking forward to sharing our data from the Phase 1b study of SAT-3247 along with our clinical progress and plans for this novel investigational medicine.”
An 11-month extension study is underway in Australia to assess the long-term safety and potential benefits of SAT-3247 in Phase 1b trial participants. Additional trial expansions are planned.
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