SG-02

SG-02 is a novel small molecule utrophin upregulator being developed for the treatment of Duchenne muscular dystrophy (DMD). It functions as an Aryl hydrocarbon Receptor (AhR) antagonist with high binding affinity. By upregulating utrophin, a paralogue of the deficient dystrophin protein, SG-02 aims to compensate for the lack of dystrophin in DMD patients regardless of their specific mutation type. In preclinical studies, SG-02 demonstrated a dose-dependent increase in utrophin expression (over 2.5-fold) in both C2C12 and human DMD patient-derived cell lines. Additionally, the compound promotes myogenesis, as evidenced by increased Myosin Heavy Chain (MHC) expression. Preliminary ADME assessments suggest that SG-02 is orally bioavailable.