LB: SAT-3247: An Oral Small Molecule Inhibitor Targeting AAK1, a Critical Effector Of Skeletal Muscle Regeneration


Translational Research

Poster Number: T432


Phil Lambert, PhD, Satellos Bioscience

Satellos is a biotechnology company founded on game changing science in skeletal muscle regeneration that shows muscle stem cells in Duchenne are unable to adequately facilitate the repair of existing, or generate new, muscle fibers throughout life. We believe this is a more significant factor in the progressive muscle damage experienced by people living with Duchenne than the absence of dystrophin in existing muscle fibers. Dystrophin expression within muscle stem cells facilitates their asymmetric division, leading to the production of the progenitor cell population ultimately responsible for the repair and regeneration of damaged muscle fibers. Through the use an in-situ muscle stem cell screening platform, the company has identified a highly druggable protein kinase target called adapter associated kinase 1 (AAK1), that when inhibited, promotes functional rescue of asymmetric stem cell division, resulting in the robust production of progenitors in vitro and in vivo. We recently announced the nomination of a lead drug candidate SAT-3247, which is a potent, orally available, muscle penetrant, small molecule inhibitor of AAK1. SAT-3247 is efficacious in the gold standard Mdx model and is currently progressing through IND enabling studies to support clinical entry in 2024. Here we present a summary of key preclinical findings and draft clinical plans.