RPT-E-50

RPT-E-50 is a novel, orally bioavailable, and blood-brain barrier permeable small molecule inhibitor of nicotinamide phosphoribosyltransferase (NAMPT). Developed by Remedy Plan Therapeutics, it is characterized as a "hyperbolic inhibitor" designed to selectively impair tumor cell viability while preserving NAMPT function in normal cells, thereby minimizing potential toxicity. NAMPT is the rate-limiting enzyme in the NAD+ salvage pathway, which is essential for energy metabolism, DNA repair, and redox balance in cancer cells. In preclinical evaluations for gliomas, RPT-E-50 demonstrated superior potency in inhibiting proliferation and inducing programmed cell death in molecularly heterogeneous glioma stem-like cells (GSCs) compared to other candidates in the series (RPT-E-36 and RPT-E-51). Preliminary in vivo data in intracranial xenograft models indicate a survival advantage, supporting its potential for clinical development in glioblastoma.