MFC0101

MFC0101 is a first-in-class small molecule degrader targeting sterol regulatory element-binding protein 1 (SREBP1), developed by Medific. It acts by specifically engaging the SREBP cleavage-activating protein (SCAP), blocking the interaction between SCAP and SREBP1, which leads to degradation of SREBP1. This mechanism disrupts lipid metabolism pathways critical for glioblastoma stem cells, reducing their tumor-initiating capacity. In preclinical studies, MFC0101 demonstrated high oral bioavailability and blood-brain barrier permeability, with no significant toxicity observed in animal models. When combined with temozolomide (TMZ), it showed synergistic efficacy resulting in complete tumor remission in glioblastoma multiforme (GBM) mouse models[2][3][4][5].