DSP-0390

DSP-0390 is an investigational small molecule inhibitor of emopamil-binding protein (EBP), a key enzyme involved in one of the final steps of cholesterol biosynthesis. By inhibiting EBP, DSP-0390 disrupts de novo cholesterol synthesis, which is critical for the growth and survival of glioma cells that cannot import cholesterol from circulation due to the blood-brain barrier. Preclinical studies have shown antitumor activity in glioblastoma models. The drug is being developed primarily for high-grade gliomas, including glioblastoma multiforme (GBM), and has received orphan drug designation from the FDA for brain cancer. It is currently being evaluated in phase 1 clinical trials as an oral monotherapy for recurrent high-grade gliomas[1][2][3][6][8].