TIP-14. Trial in Progress (TiP): Interim analysis of a Phase 2 study of BPM31510 (a lipid nanodispersion of oxidized CoQ10) with Vitamin K in combination with Standard of Care (SOC) RT and TMZ in Glioblastoma Multiforme (GBM) patients without prior therapy

BACKGROUND: BPM31510 is an investigational, drug-lipid conjugate nanodispersion. Preclinical studies demonstrate that intravenous infusion results in plasma concentrations of oxidized CoQ10 approximately 10,000 times higher than baseline physiological levels. In Phase 1, the main adverse events were related to hepatotoxicity and coagulopathy. All coagulation related issues were alleviated by coadministration of Vitamin K. Preclinical studies show that supraphysiological concentrations of CoQ10 induce metabolic shifts in cancer cells and generate Reactive Oxygen Species (ROS) leading to apoptosis. The Phase 2 trial in newly diagnosed patients with glioblastoma test the hypothesis of whether reprogramming of mitochondrial metabolism with BPM31510 results in improved efficacy of TMZ and radiotherapy. Trial Design: (BPM31510IV-11; NCT04752813) is a single-arm, non-randomized, open-label, phase 2 study of BPM 31510 + Vitamin K1 with standard chemoradiation and TMZ in newly diagnosed GBM patients. The primary endpoint is Progression free Survival at 6 months (PFS6) and 12 months (PFS12) while the secondary endpoints are overall survival and safety. Patients with recent hemorrhage, coagulopathy or requiring anticoagulants are excluded. Intravenous BPM31510 is initiated 2-4 weeks after biopsy or tumor resection. After two weeks, standard chemoradiation and TMZ are started in combination with weekly BPM31510. After 8 weeks, BPM31510 treatment ends with the end of standard RT schedule. Subjects continue to receive maintenance TMZ treatment for up to 12 cycles as per standard of care. Of the approximately 50 patients that will be enrolled, 25 will be available for PFS6 landmark analysis on June 15, 2025. A 90% power in rejecting the null hypothesis of PFS6 of ≤ 30% at the interim analysis will drive futility. This data will be available and presented.