BPM31510-IV is an ubidecarenone containing intravenous nanodispersion targeting the metabolic machinery in cancer, shifting bioenergetics from lactate dependency towards mitochondrial OxPhos and reversing the Warburg effect. BPM31510-IV enables delivery of ubidecarenone preferentially into cancer cell mitochondria, generating reactive oxygen species and activation of apoptosis. In a phase I study, BPM31510-IV was well-tolerated as a monotherapy, and in combination with gemcitabine, with an established maximum tolerated dose (MTD) of 110mg/kg and thus, formed the basis for the Phase 2 investigation.
In this study eligible patients (aged ≥ 18 y) relapsed/refractory to standard treatment for advanced metastatic pancreatic cancer and meeting inclusion/exclusion criteria receives either two 72 hr infusions of 110mg/kg BPM31510-IV or in combination with gemcitabine (1000 mg/m3 over 30 min, weekly x 3 weeks every 28 days). Tumor response is evaluated at wk 10, and then every 8 wks. This study occurs in two parts: Part 1 is designed to enroll 10 patients in the BPM31510-IV (monotherapy arm) and 10 patients in the BPM31510-IV plus gemcitabine (combination therapy arm) with intent to enroll an additional 15 patients (Part 2) into the applicable treatment arm(s). Endpoints will include overall response rate in both groups along with overall survival, progression-free survival, and time to progression. Additionally, patient reported Quality of Life using the validated FACT-HEP as well as tumor response measured by CA 19-9 levels will be combined with multi-omic molecular profiling to assess adaptive molecular responses. An exploratory objective of this study is to comprehensively perform multi-omic profiling for identification of biomarker panels for patient stratification for later stages of clinical development.
Clinical trial identification
Legal entity responsible for the study
Khampaseuth Thapa. BERG, LLC.
A.A. Niewiarowska, R. Ramanathan, P. Ritch: Research sponsorship: BERG, LLC. D.M. Lucius: Employee, stock owner: BERG, LLC. R. Sarangarajan: Co-founder, employee, stock options owner: BERG, LLC. N.R. Narain: Co-founder, president & CEO, stock options owner: BERG, LLC. J. Strauss: Research sponsorship: BERG, LLC; stock & other ownership: Abbott, Abbvie, Bristol-Myers Squibb, Intuitive surgical, Merck. M. Kundranda: Research sponsorship: BERG, LLC; Honoraria: Bayer. D. Propper: Research sponsorship: BERG, LLC; Consulting, advisory role: BMS; Speaker\'s bureau: Merck; Travel, accommodations, expenses: Celgene.