Abstract CT242: 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
Authors: Brian Stocksdale, Rebecca Brown, Kevin Elmore, Chirag Patel, Adam Cohen, Rupa Juthani, Brian Vaillant, Alexander Spira, Jedrzej Wykretowicz, Bing Nie, Michael A. Kiebish, Stephane Gesta, Niven R. Narain, Vijay Modur, Seema Nagpal
Journal: Cancer Research (2025)

Abstract Background: GBM is characterized by altered metabolic activity driven by several factors including lower CoQ10 levels resulting in reduced oxidative phosphorylation and higher glycolytic dependence compared to normal neural tissue. BPM31510 is a novel drug-lipid conjugate nanodispersion that achieves high levels of oxidized CoQ10 following IV infusion that in preclinical studies shows metabolic rewiring in GBM cells and generation of reactive oxygen species (ROS) resulting in selective cancer cell apoptosis. Treatment with BPM31510 is associated with tumor response and long-term survival of C6 glioma rat model. In Phase 1 GBM trial (NCT0302060), BPM31510 reduced plasma circulating markers associated with glioma progression, including KITLG (the ligand for the receptor-type protein tyrosine kinase KIT), the oncogenic driver SRC (SRC proto-oncogene, non-receptor tyrosine kinase), and carboxypeptidase Q. Given that the expression of KITLG and SRC is known to be upregulated by cancer cells under hypoxic conditions, the observed reduction in these markers suggests that BPM31510 may mitigate tumor hypoxia in patients as well. The ongoing Phase 2 trial in newly diagnosed patients with glioblastoma investigates the hypothesis of whether reprogramming of mitochondrial metabolism with BPM31510 results in improved efficacy of TMZ and radiotherapy in the frontline adjuvant setting. Trial Design: (BPM31510IV-11; NCT04752813) is a single-arm, 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) while the secondary endpoints are overall survival, PFS12 and safety. Exploratory endpoints include effects of BPM31510 on metabolism and change in markers associated with glioma progression. 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 of BPM31510 treatment, standard chemoradiation and TMZ is started in combination with weekly BPM31510 treatment. 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. Approximately 50 patients will be enrolled. A 90% power in rejecting the null hypothesis of PFS6 of ≤ 30%. The study is currently open at 8 US sites and has enrolled 20 patients. Citation Format: Brian Stocksdale, Rebecca Brown, Kevin Elmore, Chirag Patel, Adam Cohen, Rupa Juthani, Brian Vaillant, Alexander Spira, Jedrzej Wykretowicz, Bing Nie, Michael A. Kiebish, Stephane Gesta, Niven R. Narain, Vijay Modur, Seema Nagpal. 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 [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2025; Part 2 (Late-Breaking, Clinical Trial, and Invited Abstracts); 2025 Apr 25-30; Chicago, IL. Philadelphia (PA): AACR; Cancer Res 2025;85(8_Suppl_2):Abstract nr CT242.