• Phase 1a/1b study in 97 patients demonstrates mitochondrial metabolic reprogramming induced by BPM31510-IV and supports advancement in multiple aggressive solid tumor indications.
• BPM31510-IV has completed phase 2b enrollment for glioblastoma, completed phase 2a for pancreatic cancer
• Study data serves as fundamental clinical validation for NAi Interrogative Biology platform, enabling expanded pipeline development
  

BOSTON, Dec. 16, 2025 (GLOBE NEWSWIRE) -- BPGbio, Inc., a leading biology-first, AI-powered biopharmaceutical company focused on mitochondrial biology and protein homeostasis, today announced the publication of its Phase 1a/1b multi-center study evaluating BPM31510-IV, a first-in-class mitochondrial metabolic modulator, in 97 patients with advanced solid tumors in Cancer Research Communications.

The published findings—combined with computational analyses performed using BPGbio’s NAi® Interrogative Biology® platform—served as a compelling biological foundation and clinical rationale for BPM31510-IV across multiple aggressive solid tumors, including an ongoing fully-enrolled Phase 2b study in glioblastoma (GBM) and a completed Phase 2a study in pancreatic cancer.

“This study provides important clinical evidence that targeting mitochondrial metabolism is a viable therapeutic strategy for patients with advanced cancers,” said Madappa Kundranda, M.D., Banner MD Anderson Cancer Center, and co-author of the publication. “The metabolic shift we observed with BPM31510-IV—from glycolysis toward oxidative phosphorylation—opens new possibilities for treating solid tumors driven by mitochondrial dysfunction, including those that have been historically resistant to therapy.”

Patients enrolled in the Phase 1a/1b study represented a diverse set of metabolically dysregulated solid tumors, including pancreatic, breast, colorectal, ovarian, prostate, sarcomas, renal cell carcinoma, mesothelioma, gastric, and gliomas/CNS tumors, amongst others.

Evidence of partial responses, durable stable disease, and metabolic modulation across these tumor types suggests broad therapeutic potential driven by common mitochondrial dysfunction mechanism targeting the Warburg phenotype in cancer.

“The ability to safely reach therapeutic mitochondrial exposure and observe metabolic reprogramming in human tumors is a significant milestone,” said Vivek Subbiah, M.D., Chief, Early-Phase Drug Development, Sarah Cannon Research Institute and corresponding author. “These findings support the development of BPM31510 in tumor types driven by profound mitochondrial dysfunction.”

Computational Validation of BPM31510’s Mechanism and Patient Stratification

The 97-patient multi-omics dataset generated from this trial provided a real-world demonstration of NAi’s ability to uncover biologically meaningful therapeutic opportunities.

Using NAi, BPGbio:

• Integrated tumor tissue, blood-based proteomics, metabolomics, and lipidomics
• Built interpretable computational models linking mitochondrial pathway activity to clinical response
• Identified distinct metabolic phenotypes—including oxidative stress, CoQ-driven electron transport engagement, and redox imbalance—predictive of sensitivity to BPM31510
  

This publication marks one of the first examples of an AI or computational-biology platform guiding indication selection for a clinical development using clinical outcomes, demographics, patient omics, imaging, and pathology.

Key Findings from the Published Study

• Safety and Tolerability: BPM31510-IV was well tolerated up to 171 mg/kg, with reversible and manageable coagulation laboratory effects.
• Mechanistic Validation: FDG-PET imaging and multi-omics analyses confirmed a metabolic shift from glycolysis (Warburg phenotype) toward oxidative phosphorylation, consistent with the drug’s intended mechanism from pre-clinical studies
• Clinical Activity: Multiple aggressive tumor types demonstrated responses or prolonged stable disease.
  

“This study of 97 patient multi-omics datasets from NAi was groundbreaking as a use case for AI and patient biology guiding clinical decisions. The data gave us confidence to advance BPM31510-IV into clinically high-risk settings, where traditional approaches have repeatedly fallen short,” said Niven R. Narain, Ph.D., President and CEO of BPGbio. “BPM31510’s ongoing phase 2b clinical trial for GBM and encouraging results from pancreatic cancer phase 2a trial continue to demonstrate the strength of our biology-first AI strategy in drug discovery and development.”

BPGbio wishes to thank the clinical investigators, patients and their families for the participation in this clinical trial.

About BPM31510-IV

BPM31510-IV is an investigational mitochondrial metabolic modulator designed to reprogram tumor bioenergetics by restoring oxidative balance and inducing redox-mediated apoptosis. It is currently in:

• Phase 2b trial in newly diagnosed glioblastoma (NCT04752813)
• Completed Phase 2a pancreatic cancer trial
• Exploratory and compassionate-use programs in mitochondrial disorders
  

Contact: Media@bpgbio.com

About BPGbio, Inc.

BPGbio is a leading biology-first AI-powered clinical stage biopharma focused on mitochondrial biology and protein homeostasis. The company has a deep pipeline of AI-developed therapeutics spanning oncology, rare disease and neurology, including several in late-stage clinical trials. BPGbio’s novel approach is underpinned by NAi, its proprietary Interrogative Biology Platform, protected by over 500 US and international patents; one of the world’s largest clinically annotated non-governmental biobanks with longitudinal samples; and exclusive access to the most powerful supercomputer in the world. With these tools, BPGbio is redefining how patient biology can be modeled using bespoke Bayesian AI specifically designed for solving large-scale biology challenges. Headquartered in greater Boston, the company is at the forefront of a new era in medicine, combining biology, multi-modal data, and AI to transform the way we understand, diagnose, and treat disease. For more information, visit www.bpgbio.com.

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/3af446f8-a167-45fa-abd4-9ad62b3bad64