- Presentations highlight clinical outcomes in pediatric PCQD subjects and translational evidence supporting tissue delivery and mitochondrial metabolic correction
- Data support planned Phase 3 pivotal trial in PCQD following FDA Type C meeting
BOSTON, June 17, 2026 (GLOBE NEWSWIRE) — BPGbio, a clinical-stage biopharmaceutical company advancing mitochondrial-targeted therapeutics for patients, today presents new clinical and translational findings supporting the potential of BPM31510, BPGbio’s investigational mitochondrial therapy, in primary CoQ10 deficiency (PCQD) at the 2026 United Mitochondrial Disease Foundation (UMDF) Mitochondrial Medicine Conference in Orlando, Florida.
The company’s oral presentation and two poster presentations feature findings from pediatric compassionate use subjects with genetically confirmed PCQD, alongside translational research in preclinical mouse models of encephalopathy and nephropathy, providing further evidence supporting tissue delivery, mitochondrial metabolic correction, and functional improvement across multiple manifestations of disease.
PCQD is a rare, progressive mitochondrial disease caused by mutations in genes involved in CoQ10 biosynthesis and is associated with neurological, muscular, and renal dysfunction. Currently, there is no FDA approved therapeutics for PCQD and treatment approaches rely largely on oral CoQ10 supplementation, which may have limited and variable tissue bioavailability.
Building on the growing body of translational and clinical evidence supporting BPM31510 in PCQD, BPGbio is planning a Phase 3 pivotal trial of BPM31510 for primary CoQ10 deficiency following a Type C meeting with the U.S. Food and Drug Administration (FDA) earlier this spring, in which the Company aligned with the Agency on the path forward for late-stage clinical development in this ultra-rare mitochondrial disease.
“The totality of evidence generated to date — spanning compassionate use experience, translational science, and regulatory engagement — provides an encouraging foundation for continued development of BPM31510 in PCQD,” said Frank J. Sasinowski, M.S., M.P.H., J.D., Director at Hyman, Phelps & McNamara, P.C. and regulatory counsel to BPGbio. “The emerging body of evidence supporting BPM31510, coupled with constructive FDA engagement, reflects a thoughtful and rigorous approach toward advancing a potential therapy for patients facing a serious unmet medical need.”
In the compassionate use setting, BPM31510 was generally well tolerated, with no drug-related clinically significant adverse events reported across the four pediatric subjects, all who had previously received oral CoQ10 supplementation with minimal or short-lived benefit prior to treatment with intravenous BPM31510.
Treatment with BPM31510 was associated with improvements in ataxia and mobility in all four subjects, measured through objective clinical assessments and evaluations. Improvements were observed as early as four weeks following treatment initiation, with reported gains in balance, muscle strength, fatigue, mobility, and day-to-day functioning.
In translational studies, BPGbio also presents findings demonstrating that BPM31510 increased the CoQ pool in affected tissues, including the brain in a Coq9 encephalopathy model and the kidneys in a Pdss2 nephropathy model, supporting delivery to highly metabolic organs commonly impacted in PCQD.
“Primary CoQ10 deficiency is a devastating mitochondrial disorder with profound unmet need, particularly for pediatric patients facing progressive neurological decline,” said Niven R. Narain, Ph.D., President and CEO of BPGbio. “As we advance preparations for a planned Phase 3 pivotal trial following constructive discussions with the FDA this spring, we remain focused on bringing a potential new therapeutic option to patients and families affected by this severe mitochondrial disease.”
BPGbio will be hosting an investigator’s meeting at UMDF in Orlando, FL during UMDF with leading scientists and physicians in the field to gain insight, feedback, and clinically relevant insights for an enriched and data-driven approach to proposing the most appropriate primary and secondary endpoints.
About BPGbio
BPGbio is a clinical-stage biopharmaceutical company advancing mitochondrial-targeted therapeutics for patients. The company’s lead investigational therapeutic, BPM31510, is designed to modulate mitochondrial function and address diseases associated with mitochondrial dysfunction and altered cellular metabolism. BPGbio is advancing a pipeline of mitochondrial therapies aimed at improving outcomes for patients with significant unmet medical needs. Learn more www.bpgbio.com
Contact: [email protected]
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