First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort

SAT-3247 is an oral tablet that is a potent, muscle penetrant, small molecule inhibitor of AAK1; inhibition of AAK1 rescues perturbed asymmetric division of satellite stem cells, resulting in increased muscle regeneration in animal models of DMD. In vitro and in vivo animal pharmacology studies have demonstrated the efficacy of SAT-3247 in improving muscle strength and the necessary target coverage to maximize functional muscle improvement.

matched placebo