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Company’s presentations highlight the power of mitochondrial medicine and protein homeostasis technologies to address aggressive cancers including glioblastoma and pancreatic cancerBOSTON, April 23, 2025 (GLOBE NEWSWIRE) -- BPGbio, Inc., a leading biology-first, AI-powered clinical-stage biopharma company focused on mitochondrial biology and protein homeostasis, today announced the presentation of seven presentations at the American Association for Cancer Research (AACR) Annual Meeting, taking place April 25–30, 2025, in Chicago. The presentations highlight BPGbio’s advances in mitochondrial medicine, immuno-oncology, and precision diagnostics, powered by its proprietary NAi Interrogative Biology® Platform. The featured research spans BPGbio’s oncology pipeline and precision medicine efforts, including mechanistic and clinical insights into BPM31510, a first-in-class oxidized CoQ10 nanodispersion that has demonstrated encouraging results in multiple clinical trials in oncology and other mitochondrial disease areas, and BRG399, a novel microtubule-binding agent that induces immunogenic cell death (ICD). These presentations reinforce BPGbio’s leadership in reprogramming cancer metabolism and targeting mitochondrial dysfunction to drive therapeutic advancement. “We are proud of the tremendous progress across our oncology pipeline at BPGbio both in the BPM31510 and BRG399 franchises. Our NAi platform has revealed hallmark mechanistic insights into mitochondrial control mechanisms in cancer,” said Niven R. Narain, Ph.D., President and CEO at BPGbio. “Our AACR presentations reinforce the importance of stabilizing mitochondrial function and capacity in oncology drug development. BPGbio is now keenly focused on advancing our clinical data and assessing the best commercialization partners to serve patients and families.” Highlights from BPGbio’s AACR 2025 presentations include: Clinical Progress in GBM: A Phase 2 trial of BPM31510 in newly diagnosed glioblastoma multiforme (GBM) demonstrates favorable tolerability and early signals of disease control, reinforcing its potential to address one of oncology’s toughest indications.Mitochondrial Reprogramming in the Tumor Microenvironment: BPM31510 remodels immune landscapes and rewires cancer metabolism, showing immune-enhancing and anti-tumor effects in an immunogenic oncology model.Ferroptosis-Independent Apoptosis: Data show BPM31510 induces ROS-mediated apoptosis in cancer cells without triggering ferroptosis, underscoring a novel mechanism of selective cytotoxicity.C6 Glioma Warburg Reversal: BPM31510 reverses the Warburg effect in quinone-deficient glioma models, restoring oxidative phosphorylation leading to tumor resolution in vivo.BRG399 as a Next-Generation ICD Agent: Preclinical studies show BRG399 drives potent immunogenic cell death and immune infiltration, especially at lower doses, positioning it as a strong candidate for combination immunotherapy.Targeting CoQ Deficiency in Cancer: Multi-omic analysis of TCGA and GTEx datasets identifies cancers with disrupted ubiquinone biosynthesis pathways, spotlighting new indications for mitochondrial-targeted therapies.Project Survival – Early Detection of PDAC: A 7-year, multi-site biomarker study identified a four-protein diagnostic panel that distinguishes pancreatic cancer patients from at risk populations with high sensitivity and specificity. “The breadth of our AACR data—spanning diagnostics, drug mechanism of action, and immuno-oncology— showcases the power of BPGbio’s NAi Interrogative Biology Platform to link biology to therapeutic outcomes,” said Michael Kiebish, Ph.D., Vice President, Platform and Translational Sciences at BPGbio. “This reinforces the critical importance of mitochondrial medicine and protein homeostasis in oncology and immunology to impact preclinical and clinical development leading to improved outcomes for patients.” Presentation Details: Abstract 1591 / 7: Mechanisms of BPM31510-induced cell death in cancer: lipid peroxidation and apoptosis pathwaysPresentation: Monday, April 28, 2025, 9:00 a.m. – 12:00 p.m. in Poster Section 16 Abstract 1527 / 9: Reversal of the Warburg Effect with BPM31510 treatment in a quinone deficient C6 Glioma resulting in efficacyPresentation: Monday, April 28, 2025, 9:00 a.m. – 12:00 p.m. in Poster Section 14 Abstract 3277 / 9: Investigating the link between metabolic reprogramming and immune modulation in tumors: exploring the therapeutic potential of BPM31510Presentation: Monday, April 28, 2025, 2:00 p.m. – 5:00 p.m. in Poster Section 30 Abstract 4177 / 27: Integrated assessment of alterations in coenzyme Q biosynthesis genes impacting cancer metabolism leading to critical vulnerabilities in tumorsPresentation: Tuesday, April 29, 2025, 9:00 a.m. – 12:00 p.m. in Poster Section 13 Abstract 5937 / 29: Development of a protein and clinical multiparametric biomarker panel for assessing pancreatic adenocarcinoma riskPresentation: Tuesday, April 29, 2025, 2:00 p.m. – 5:00 p.m. in Poster Section 32 Abstract CT242 / 11: 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 therapyPresentation: Tuesday, April 29, 2025, 2:00 p.m. – 5:00 p.m. in Poster Section 51 Abstract 6161 / 13: Exploring BRG399, a novel microtubule-binding agent, as an inducer of immunogenic cell death in cancer therapyPresentation: Tuesday, April 29, 2025, 2:00 p.m. – 5:00 p.m. in Poster Section 40 About BPM31510BPM31510IV is BPGbio’s lead candidate in late-stage development for aggressive solid tumors such as glioblastoma multiforme (GBM) and pancreatic cancer. Other topical and oral formulations of the investigational agent are also being developed as a potential treatment for several rare diseases. The compound has demonstrated a tolerable safety profile and shown potential clinical benefits across multiple disease indications. Validated by BPGbio’s NAi Interrogative Biology platform, BPM31510 induces a hallmark shift in the tumor microenvironment (TME) by modulating mitochondrial oxidative phosphorylation in aggressive tumors, leading to cancer cell death. In many mitochondrial diseases, restoring CoQ10 levels can overcome the effect of mutations in genes that lead to mitochondrial dysfunction. BPM31510 has been granted Orphan Drug Designation by the FDA for GBM, pancreatic cancer, and epidermolysis bullosa (EB), as well as Rare Pediatric Disease Designation for primary CoQ10 deficiency and EB. About BRG399BRG399 is a BPGbio-developed candidate being studied for its therapeutic potential as a treatment for solid and liquid tumor cancers as well as diseases associated with inflammation. This experimental drug, a first-in-class, anti-mitotic agent with broad-spectrum anti-cancer activity and favorable pharmacological properties for clinical testing, is being designed for oral delivery. BRG399 is leading the new oncology drug pipeline for BPGbio which includes drug candidates uniquely targeting E2 enzymes. About BPGbioBPGbio 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 400 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.Media Contact: media@bpgbio.com

BOSTON--( BUSINESS WIRE )-- BPGbio, Inc. , a leading biology-first, AI-powered clinical-stage biopharma company focused on mitochondrial biology and protein homeostasis, today announced the presentation of new findings in pancreatic cancer subtyping from Project Survival ® ( NCT2781012 ), the company’s seven-year longitudinal pancreatic cancer precision medicine clinical trial. The results will be presented at the ASCO Gastrointestinal Cancers Symposium , being held January 23–25, 2025, in San Francisco, Calif. Aimed at improving pancreatic cancer survival rate, the multi-center clinical trial employed comprehensive biomarker discovery to understand patient survival, enabling the development of new diagnostic, therapeutic, and clinical support solutions. Leveraging the biology-first approach and causal AI capabilities of BPGbio’s proprietary NAi Interrogative Biology® platform , researchers conducted a comprehensive multiomics analysis of plasma samples from 280 pancreatic ductal adenocarcinoma (PDAC) patients over seven years and found distinct molecular patterns correlated with both long-term and short-term survival outcomes. “Pancreatic cancer is not a one-size-fits-all disease. One of the biggest challenges we face in treating it is the rapid progression of the disease, which often leaves us with little time to make critical decisions,” said Madappa Kundranda, M.D., Ph.D. , Chief of Division of Cancer Medicine, Banner MD Anderson Cancer Center, who was the principal investigator of the study. “Our research aims to address this by identifying pancreatic cancer subtypes quicker, allowing us to better select patients for clinical trials and, ultimately, develop more tailored treatments that can significantly improve patient outcomes." Notably, elevated cholesterol metabolism was observed in long-term survivors, while reduced levels of specific lipids characterized short-term survivors, among other biomarkers. These findings offer the potential of non-invasive blood-based biomarker panels for precise patient stratification and more tailored treatment. “This study identifies how critical it is to go beyond genomics in biomarker discovery in order to capture the biological diversity of patients which impacts therapeutic decisions,” said Michael A. Kiebish, Ph.D., Vice President of Platform and Translational Sciences, BPGbio. "To effectively treat the patient, not just the disease, we must identify the right molecular signature, which can be revealed through advanced multi-omics technologies that provide a holistic view of the body's biology." Pancreatic cancer remains one of the deadliest cancers due to its typically late diagnosis and rapid progression. The current method of identifying tumor tissue subtypes such as ‘Cholesterogenic’ and ‘Classical’ is associated with improved survival, but tissue-based profiling poses challenges due to the invasive nature of sample collection and the time it takes to analyze tissue subtypes, relative to the quick advancement of the disease. “At BPGbio, we believe that understanding differences in patient biology, such as between diseased and non-diseased populations, requires a systems medicine approach. These findings from Project Survival ® are a testament to the power of that perspective,” said Niven R. Narain, Ph.D ., President and CEO of BPGbio. “By expanding our focus beyond cancer tissues and by performing comprehensive molecular profiling on patient plasma, we unlock critical insights into pancreatic cancer biology. This approach enables us to move toward more tailored treatments for various categories of patients, bringing us closer to the ultimate goal of this meaningful project - survival.” ASCO-GI Presentation Details Abstract #: 775 Abstract Title: Lipogenic and Metabolic Subtypes Define Survival Outcome in Pancreatic Adenocarcinoma Patients In-Person Session Name: Poster Session B: Cancers of the Pancreas, Small Bowel, and Hepatobiliary Tract In-Person Poster Session Date: January 24, 2025, 11:30 a.m. - 1 p.m. PST Presenter: Madappa Kundranda, M.D., Ph.D. About BPM31510 BPM31510IV is BPGbio’s lead candidate in late-stage development for aggressive solid tumors such as glioblastoma multiforme (GBM) and pancreatic cancer . Other topical and oral formulations of the investigational agent are also being developed as a potential treatment for several rare diseases. The compound has demonstrated a tolerable safety profile and shown potential clinical benefits across multiple disease indications. Validated by BPGbio’s NAi Interrogative Biology platform , BPM31510 induces a hallmark shift in the tumor microenvironment (TME) by modulating mitochondrial oxidative phosphorylation in aggressive tumors, leading to cancer cell death. In many mitochondrial diseases, restoring CoQ 10 levels can overcome the effect of mutations in genes that lead to mitochondrial dysfunction. BPM31510 has been granted Orphan Drug Designation by the FDA for GBM, pancreatic cancer, and epidermolysis bullosa (EB), as well as Rare Pediatric Disease Designation for primary CoQ10 deficiency and EB. About BPGbio 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 400 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 .