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Satellos Bioscience Inc. reported dosing the first participant in BASECAMP, a three-month, randomized, double-blind, placebo-controlled, proof-of-concept Phase 2 pediatric study of its oral drug SAT-3247 for Duchenne muscular dystrophy.
The trial plans to enroll 51 ambulatory children with Duchenne aged 7 to 9 years. Primary endpoints focus on safety, tolerability and effects on muscle force, while secondary endpoints assess muscle quality, function and regeneration.
SAT-3247 is described as a first-of-its-kind small molecule that targets AAK1 to help restore muscle stem cell signaling disrupted in Duchenne, and is being developed as a potential disease-modifying, dystrophin-independent treatment initially for Duchenne muscular dystrophy.