472TiP - Phase 2 clinical investigation of BPM31510 (ubidecarenone) alone and in combination with gemcitabine in patients with advanced pancreatic cancer

Abstract

Background: BPM31510 is an ubidecarenone lipid nanodispersion that switches cancer energy generation from glycolysis to mitochondrial oxidative phosphorylation to elicit anticancer effect. BPM31510 is well tolerated as a monotherapy and at an established MTD of 110mg/kg in combination with Gemcitabine in a Phase I clinical trial. Preclinical in vivo pancreatic models demonstrate that BPM31510 alone and in combination with gemcitabine significantly improves duration of survival; supporting the Phase 2 evaluation of BPM31510 in patients with advanced metastatic adenocarcinoma. Trial design: Eligible patients (aged ≥ 18 y) relapsed/refractory to standard treatment (ST) and met inclusion/exclusion criteria. Each patient receives 110mg/kg IV BPM31510 in a 144-hour infusion alone or in combination with gemcitabine. Tumor response is evaluated at wk10 and then every 8 wks. Investigator observations and reports by treated patients provide clinical assessments not specifically defined in the protocol. This study initially will enroll ten (10) patients in the BPM31510 (monotherapy arm) and ten (10) patients in the BPM31510 plus gemcitabine (combination therapy arm) with intent to enroll the additional 15 patients into the applicable treatment arm(s) into the expansion stage based on RECIST v1.1 clinical response. The goal is to evaluate the Overall Response Rate (ORR) in patients treated with BPM31510 alone or in combination with gemcitabine along with Overall Survival (OS); Progression-Free Survival (PFS); Time to Progression (TTP); Tumor Response using Adaptive Molecular Responses (multi-omic molecular profiling); Evaluate Change in CA 19-9 levels and patient reported Quality of Life using the validated FACT-HEP patient-reported outcomes instrument. Clinical trial identification: BPM31510IV-05 Legal entity responsible for the study: BERG, LLC Funding: BERG, LLC Disclosure: A. Niewiarowska: Clinical investigator paid by BERG, LLC to conduct studies. R. Sarangarajan: Employee, co-founder and has stock options of BERG, LLC. D. Lucius: Employee and has stock options of BERG, LLC. N. Narain: Employee, co-founder and has stock options of BERG, LLC R.K. Raminathan: Clinical investigator paid by BERG, LLC to conduct studies. M. Hidalgo: Clinical investigator paid by BERG, LLC to conduct studies.