BPGbio Highlights Groundbreaking Data on BPM31510 for Mitochondrial Diseases at the Inaugural Mitochondrial Transplantation and Next Generation Therapeutics Conference

Advanced Spatial Quinomics Study Reveals Novel Insights into the Therapeutic Impact of BPM31510, Being Studied as a Potential Treatment for Primary CoQ10 Deficiency and Other Mitochondrial Diseases

/EIN News/ -- BOSTON, April 25, 2025 (GLOBE NEWSWIRE) -- BPGbio, Inc., a leading biology-first, AI-powered clinical-stage biopharma company focused on mitochondrial biology and protein homeostasis, today announced a poster presentation at the inaugural Mitochondrial Transplantation and Next Generation Therapeutics Conference, taking place April 27–29, 2025, in Long Island, N.Y. The presentation will feature novel insights into the pharmacodynamics and tissue distribution of BPM31510, providing valuable biological evidence that it effectively delivers CoQ10 to critically important tissues and tissue regions, including the cerebellum, and restores CoQ10 levels in a genetic model of primary CoQ10 deficiency.

Using cutting-edge spatial quinomics techniques, BPGbio researchers show that BPM31510 significantly increased CoQ10 levels across multiple tissues—including the brain, kidney, muscle, and heart—in Coq4 KI/KI mice (a mouse model of CoQ deficiency) in collaboration with Dr. Catarina Quinzii of Columbia University. Notably, BPM31510 achieved targeted delivery to the cerebellum, a region of the brain whose dysfunction is associated with ataxia, a hallmark symptom of mitochondrial disorders.

Primary CoQ10 deficiency and other mitochondrial diseases are often treated with oral CoQ10 supplements, which have poor bioavailability and limited clinical benefits. BPM31510, a novel formulation of CoQ10, was developed to overcome these delivery challenges, helping improve mitochondrial functioning. In preclinical studies using human cells with severe metabolic impairment, BPM31510 preserved cell viability, whereas cells treated with CoQ10 alone died within a week. BPM31510 has been granted a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for the treatment of primary CoQ10 deficiency.

“Advanced omics technology allowed us to pinpoint the exact cells in which CoQ10 is being delivered following treatment with BPM31510,” said Michael Kiebish, Ph.D., Vice President, Platform and Translational Sciences of BPGbio. “These data shows that BPM31510 crosses the blood-brain barrier—a major achievement—and delivers CoQ10 to the cerebellum, which may help address the ataxic symptoms common in primary CoQ10 deficiency. It also significantly boosts CoQ pools in the heart, kidney, and muscle which makes it a potential therapy for other mitochondrial diseases as well.”

“At BPGbio, we are redefining what’s possible in mitochondrial medicine,” said Niven R. Narain, Ph.D., President and CEO of BPGbio. “These compelling new data on BPM31510 reinforce our commitment to advancing the science of mitochondrial biology and delivering transformative therapies to patients with rare, life-altering diseases like primary CoQ10 deficiency. As we advance our clinical data on Primary CoQ10 Deficiency, we look forward to the best commercial and community partnerships to unleash the full potential of this therapy for patients and their families.”

Poster Presentation Details:
Title: Spatial and LC MS/MS Based Quinomics Reveals Novel Therapeutic Impact of BPM31510 For the Treatment of CoQ10 Deficiency Using the Coq4^KI/KI Mouse Model
Date and Time: Monday, April 28 and Tuesday, April 29

About Quinomics
Quinomics is the comprehensive measurement of quinone metabolites predominantly characterized by the classical phenolic ring structure and can represent synthesis or metabolites of CoQ10. Quinones have critical roles in antioxidant status, mitochondrial function, redox balance, and coagulation status.

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 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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