Preclinical study demonstrates potent anticancer activity, blood-brain barrier penetration, and improved survival in glioma models
Findings highlight a novel therapeutic approach targeting microtubule dynamics, with evidence suggesting potential immune memory

BOSTON, Oct. 28, 2026 (GLOBE NEWSWIRE) — BPGbio, Inc., a clinical-stage biopharmaceutical company advancing innovative therapeutics for patients, today announced the publication of new preclinical research in the peer-reviewed Journal of Medicinal Chemistry, describing a novel brain-penetrant small-molecule therapeutic candidate for the treatment of glioma, including glioblastoma (GBM).

The paper, titled “Discovery and Optimization of a Novel Brain-Penetrant Colchicine-Binding Site Ligand for Treatment of Glioma,” details the development of Compound 40, an investigational therapeutic candidate designed to disrupt microtubule formation by targeting the colchicine-binding site on tubulin, a protein essential for cell division. The compound demonstrated potent anticancer activity, the ability to penetrate the blood-brain barrier, and significant antitumor activity in preclinical models of glioma.

“Compound 40 (BRG399) is a clinical candidate ready for Phase 1 studies, with encouraging preclinical evidence of brain penetration, antitumor activity, and potential immune memory,” said Vivek K. Vishnudas, Ph.D., Chief Technology Officer of BPGbio and corresponding author of the publication. “We are particularly excited by its potential to induce immunogenic cell death (ICD), which could open new opportunities for combination therapies with immune checkpoint inhibitors (ICI). These findings support further clinical development of BRG399 as a novel therapeutic approach for hard to treat and treatment resistant cancers.”

Key Preclinical Findings

Potent anticancer activity: Compound 40 demonstrated strong antiproliferative activity across human glioma cell lines
Blood-brain barrier penetration: The compound demonstrated good distribution to the brain, supporting its potential for treating brain tumors.
Significant antitumor activity: In a rat model of glioma, Compound 40 reduced tumor burden and improved survival, in a highly aggressive Glioma Tumor Model
Potential immune-memory response: Long-term surviving animals showed no detectable tumor growth following rechallenge with glioma cells, suggesting a possible tumor immune response.

“This publication is a testament to BPGbio’s biology-first approach that goes beyond any single pathway or disease, enabling us to pursue diverse therapeutic strategies,” said Niven R. Narain, Ph.D., President and CEO of BPGbio. “Glioblastoma remains one of the most difficult cancers to treat. Alongside this discovery program, we are also advancing BPM31510, our investigational mitochondrial therapeutic that recently completed a Phase 2 clinical trial for newly diagnosed glioblastoma. Together, these programs reflect our commitment to pursuing innovative treatment strategies for diseases with significant unmet needs.”

The research was conducted by BPGbio scientists and academic collaborators, including investigators from Stanford University, Oakridge National Labs (University of Tennessee) and Paul Scherer Institute (University of Basel). The findings support further investigation of Compound 40 as a potential therapeutic candidate for glioma and potentially for combination with ICI.

About BPGbio, Inc.

BPGbio, Inc. is a clinical-stage biopharmaceutical company advancing novel therapeutics for patients with cancer, rare diseases, and other conditions associated with mitochondrial dysfunction. The company combines a biology-first approach with proprietary AI-powered discovery technologies, advanced multi-omics capabilities, and an extensive biobank to accelerate therapeutic discovery and development. Its clinical pipeline includes BPM31510, an investigational mitochondrial therapeutic being developed across oncology and rare diseases.

For more information, visit www.bpgbio.com.

Media Contact

media@bpgbio.com

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