BRG399 / BPGbio - LARVOL DELTA

Application of a deep learning based drug sensitivity prediction model on a novel anticancer drug (AACR 2024) - "However, on BRG399 tested CLs (n=101) model had RMSE 1.84 and R2 < 0, which indicated that predictions were on average worse compared to a mean-predictor model for BRG399. These results suggest that DL models that can generalize predictions to a held-out set for compounds that were present in the training set may not accurately predict IC50 of novel compounds not in the training set."

Oncology

Exploring BRG399, a novel microtubule-binding agent, as an inducer of immunogenic cell death in cancer therapy (AACR 2025) - "Our findings suggest that BRG399 acts as an ICD agent, effectively modulating both tumor and immune cells within the TME and highlighting its potential for future combination therapies with immunotherapeutics. Further in vivo studies are currently being conducted to comprehensively elucidate the immune-mediated effects of BRG399 aiming to clarify its role in enhancing anti-tumor responses ultimately contributing to the development of more effective cancer therapies."

Immunogenic cell death • IO biomarker • Oncology • CALR

BRG399: A novel colchicine-binding microtubule agent inducing early immunogenic cell death independent of mitotic arrest (SITC 2025) - "This distinctive early induction profile suggests that low doses of BRG399 may effectively stimulate antitumor immune surveillance while minimizing systemic toxicity, potentially enhancing immunotherapy efficacy. These characteristics position BRG399, for further investigation, as a candidate for rational combination strategies aimed at improving treatment outcomes across multiple cancer types."

Immunogenic cell death • IO biomarker • Colon Cancer • Colorectal Cancer • Oncology • Solid Tumor • CALR • HMGB1

BPGbio Presents Advances in Spatial Omics Technology and Targeted Protein Degradation Insights at ASMS 2025 (GlobeNewswire) - "Presentations highlight cutting-edge research in mitochondrial and neurodegeneration medicine, neurodegeneration, immuno-oncology, and E2-targeted protein degradation using next-generation mass spectrometry."

Preclinical • Oncology

BPGbio Presents New Research on Tumor Metabolism, Immunotherapy, and Diagnostics at AACR Annual Meeting 2025 (GlobeNewswire) - "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."

Clinical data • Diagnostic • Glioblastoma • Glioma • Oncology

Improved anti-glioblastoma efficacy by BRG399, a novel oral microtubule binding agent. (SNO 2024) - "These results demonstrate a therapeutic effect of BRG399 on intracranial glioma-bearing rats with survival of the BRG399 treatment groups of strong statistical significance over the control group. Analysis of drug levels in plasma and brain will serve as guidance for dose and scheduling decisions for further therapeutic development."

Clinical • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor

BPGbio to Present New Data on Novel Glioblastoma Therapies at SNO 2024 (Businesswire) - "BPGbio, Inc...today announced that it will co-present two pivotal studies with Stanford Medicine’s Department of Neurology at the Society for Neuro-Oncology (SNO) 2024 Annual Meeting...The presentations will highlight BPM31510 and BRG399—potential glioblastoma multiforme (GBM) therapies that were identified by the company’s proprietary NAi Interrogative Biology Platform, a causal AI-powered system designed to reveal hidden cause-and-effect relationships within patient biology."

P2 data • Preclinical • Glioblastoma • Oncology • Solid Tumor

BRG399, a novel oral microtubule-binding agent, exhibits immune-modulatory properties enhancing anti-tumor responses (SITC 2024) - "Conclusions This data demonstrates that BRG399 is an anti-cancer small molecule with the potential to ablate tumor cells through immune modulation. This data provides useful guidance to further investigations using BRG399 in combination with immunotherapeutic approaches."

IO biomarker • Oncology • CD8 • PD-1

BRG399, a novel oral microtubule binding agent, induces tumor regression and immune memory in an orthotopic glioblastoma rat model (SITC 2024) - "Conclusions These results demonstrate that BRG399 sufficiently distributes in the brain to have a therapeutic effect on orthotopic glioma rat model. The constrain of tumor growth and regression of tumor after the introduction of a drug holiday suggests an involvement of immune components in the TME, possibly due to induction of immunogenic cell death and/or regulation of the immunosuppressive TME."

Preclinical • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor

BPGbio to Present Transformative Immuno-Oncology Research on BPM31510 and BRG399 at SITC 2024 (Businesswire) - "BPM31510 enhances anti-tumor responses through immune cell regulation in the tumor microenvironment and is being evaluated for efficacy in glioblastoma in a phase 2b study; BRG399, a novel oral microtubule binding agent, enhances tumor cell ablation through immune modulation; BRG399 achieves 100% tumor regression in a glioblastoma model, highlighting immune system involvement...BPGbio, Inc...today announced that it will present three scientific posters at the Society for Immunotherapy of Cancer (SITC) 2024 Annual Meeting, taking place November 6-10, 2024, in Houston, Texas. The posters will highlight the latest research on the company’s drug candidates BPM31510 and BRG399 in the immuno-oncology space, providing further validation and paving the way for new potential therapeutic strategies in cancer treatment."

Preclinical • Oncology

BPGbio Presents Key Advances in its Oncology Pipeline related to the NAi Interrogative Biology Platform (Businesswire) - "Highlights from posters include: New data further validating the mitochondrial-centric effect of BPM31510 on the immune system. In addition to its direct cell-killing effect on tumor cells, data supports the potential use of BPM31510 in the intersection of tumor immunology and tumor metabolism; BPGbio’s NAi Platform coupled with a subsequent deep learning algorithm implemented on BRG399, a novel pan-cancer drug candidate, can improve efficiency in drug development by rapidly predicting anti-cancer efficacy in silico before conducting resource intensive preclinical experiments."

Preclinical • Oncology • Solid Tumor