BPGbio has unveiled groundbreaking data demonstrating that its investigational drug BPM31510 successfully crosses the blood-brain barrier and delivers CoQ10 to critical brain regions, marking a significant advancement in mitochondrial disease treatment. The findings were presented at the inaugural Mitochondrial Transplantation and Next Generation Therapeutics Conference in Long Island, New York.
Novel Delivery Mechanism Overcomes Treatment Barriers
Using cutting-edge spatial quinomics techniques, BPGbio researchers demonstrated that BPM31510 significantly increased CoQ10 levels across multiple tissues—including the brain, kidney, muscle, and heart—in Coq4 KI/KI mice, a genetic model of primary CoQ10 deficiency. The study was conducted in collaboration with Dr. Catarina Quinzii of Columbia University.
"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."
Addressing Critical Unmet Medical Need
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.
The therapeutic potential of BPM31510 was further validated in preclinical studies using human cells with severe metabolic impairment. In these studies, BPM31510 preserved cell viability, whereas cells treated with CoQ10 alone died within a week, highlighting the superior efficacy of the novel formulation.
Targeted Cerebellar Delivery Shows Promise for Ataxia
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. This breakthrough in blood-brain barrier penetration represents a major advancement in addressing neurological symptoms of mitochondrial diseases.
"It also significantly boosts CoQ pools in the heart, kidney, and muscle which makes it a potential therapy for other mitochondrial diseases as well," Kiebish noted, suggesting broader therapeutic applications beyond primary CoQ10 deficiency.
Regulatory Recognition and Clinical Development
BPM31510 has been granted a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for the treatment of primary CoQ10 deficiency. The compound has also received Orphan Drug Designation by the FDA for glioblastoma multiforme (GBM), pancreatic cancer, and epidermolysis bullosa (EB).
"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."
Advanced Technology Platform Drives Discovery
The research utilized quinomics, which 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.
BPGbio's 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 powerful supercomputing resources.
The company continues to advance its clinical data on Primary CoQ10 Deficiency while seeking optimal commercial and community partnerships to maximize the therapeutic potential for patients and their families affected by these rare, life-altering diseases.
