• BPM31510 targets cancer cells in the brain while allowing growth of healthy cells
• BRG399 shows promising results in disrupting tumor growth and extending survival in glioma models

BPGbio, Inc., a leading biology-first, AI-powered clinical-stage biopharma company focused on mitochondrial biology and protein homeostasis, 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, taking place November 21-24, 2024, in Houston, Texas. 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.

The first study, titled “Optimizing brain cancer therapy: balancing tumor eradication and normal tissue preservation with BPM31510,” explores BPM31510’s superior ability to preserve healthy cells compared to standard cancer treatments in mouse, rat, and human glioblastoma cell lines. The study demonstrates BPM31510’s ability to effectively target and diminish cancer cells while selectively allowing the growth of healthy brain cells, an effect that standard treatments fail to achieve. Researchers also showed that the BPM31510 significantly improved survival in glioma implanted mice and rats compared to the control group, mirroring the results from the ongoing phase 2b trial on BPM31510 for GBM and highlighting BPM31510's potential in brain cancer therapy.

The second study, titled “Improved anti-glioblastoma efficacy by BRG399, a novel oral microtubule binding agent,” presents findings on BRG399’s anti-cancer activity. BRG399, a novel pan-cancer therapy, works by disrupting the cell division process in both solid and liquid tumors. BRG399 was also found to induce an immune memory response, protecting against tumor recurrence. The study confirmed that BRG399 can cross into the brain, target tumor cells, and improve survival in rat glioma models.

“Standard cancer treatments that focus solely on destroying tumors often harm healthy tissue in the process,” said Seema Nagpal, M.D., principal investigator of the BPM31510 GBM phase 2b trial and Clinical Professor of Neurology and Neurological Sciences at Stanford Medicine. “These new treatments are taking novel targeted approaches on glioma cancer cells and we are excited to share the encouraging results from our studies with our peers and industry researchers.”

“Our ongoing clinical and preclinical trials for BPM31510 and BRG399 are continuously deepening our understanding of these promising therapies,” said Niven R. Narain, Ph.D., President and CEO of BPGbio. “By putting biology first, our NAi Platform has enabled us to optimize every aspect of these therapies, from dosing and timing to patient selection and potential indication expansion opportunities.”

Poster Presentation Details:

1. Optimizing brain cancer therapy: balancing tumor eradication and normal tissue preservation with BPM31510
   
   Date and Time: November 22, 2024, 7:30 p.m. - 9:30 p.m. CST
   
   Location: Hall B3, George R. Brown Convention Center, Houston, Texas
   
   Presenter: Abbas Khojasteh, Ph.D.
   
   Abstract Number: DDDR-13
   
   
   
   
2. Improved anti-glioblastoma efficacy by BRG399, a novel oral microtubule binding agent
   
   Date and Time: November 22, 2024, 7:30 p.m. - 9:30 p.m. CST
   
   Location: Hall B3, George R. Brown Convention Center, Houston, Texas
   
   Presenter: Stephane Gesta, Ph.D.
   
   Abstract Number: DDDR-14
   
   

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 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 BRG399

BRG399 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 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.

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