1st boys dosed in Phase 2 trial of oral DMD treatment SAT-3247
Therapy aims to restore cells' ability to produce healthy muscle tissue
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Dosing has begun in a Phase 2 clinical trial testing the oral therapy SAT-3247 in boys with Duchenne muscular dystrophy (DMD).
The study, dubbed BASECAMP (NCT07287189), aims to enroll 51 boys with DMD aged 7-9 who can walk. Patients who have received treatments such as exon skippers, corticosteroids, Duvyzat (givinostat), and Elevidys (delandistrogene moxeparvovec-rokl) may be eligible. The study will include sites across several countries; some sites in the U.S. have begun recruitment.
Participants in the BASECAMP study will be randomly assigned to take SAT-3247 at one of two doses (60 or 120 mg), or a placebo, daily for about three months. The study’s main goals are to evaluate the safety and tolerability of SAT-3247 and to assess the therapy’s effect on muscle strength, as measured by dynamometry (objective tests that measure exactly how much force specific muscles exert).
The Phase 2 trial is sponsored by SAT-3247’s developer Satellos Bioscience. The company said the trial is designed to be pivotal, meaning that positive results could support regulatory approval applications for SAT-3247.
“BASECAMP marks a significant step for Satellos in evaluating the therapeutic potential of SAT-3247 in children living with Duchenne,” Frank Gleeson, co-founder and CEO of Satellos, said in a company press release. “Data generated from BASECAMP could play a meaningful role in accelerating the development of SAT-3247 as a novel treatment for this disease.”
DMD is caused by mutations that lead to a deficiency of dystrophin, a protein that’s vital for maintaining muscle health through several different mechanisms. When muscles are damaged, muscle stem cells become activated, growing to produce new muscle tissue that can replace the damaged tissue. Recent research has shown that dystrophin deficiency in DMD leads to problems with the regenerative ability of these stem cells. SAT-3247 aims to restore these cells’ ability to produce new healthy muscle tissue.
Satellos conducted a Phase 1 trial (NCT06565208) that tested SAT-3247 in five adults with DMD aged 20 or older, all of whom had relatively advanced disease at the trial’s start. Results indicated the therapy led to significant improvements in muscle function, most notably a roughly doubling in patients’ grip strength. A long-term study, LT-001 (NCT06867107), is now underway to collect additional data on the therapy’s use in these adults. Testing in new participants was also part of the plan.
The newly launched Phase 2 study aims to expand on these early findings by examining the impact of SAT-3247 at an earlier stage of DMD disease development.
“BASECAMP will focus on an important period in Duchenne when muscle health and function begin to decline more rapidly,” said Wildon Farwell, MD, chief medical officer of Satellos. “Treatment options remain limited for this devastating disease, and this study will evaluate a novel approach to potentially increase muscle regeneration and thereby improve function among children with Duchenne.”
