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BPGbio Announces Presentation of Preliminary Clinical Data of Investigational BPM31510 for Primary CoQ10 Deficiency

By: BPGbio via GlobeNewswire

June 18, 2025 at 07:00 AM EDT

BPM31510 provides encouraging evidence of the potential for transformative benefit for a disease with high unmet need, supporting further development.
The Company plans to seek guidance in Q3 2025 from the FDA regarding the development of BPM31510 for PCQD on a potential expedited path available for rare diseases.

BOSTON, June 18, 2025 (GLOBE NEWSWIRE) -- BPGbio, Inc., a leading biology-first, AI-powered, clinical stage biopharma focused on mitochondrial biology and protein homeostasis, today announced that Vijay Modur, MD, Ph.D., the company’s Chief Medical Officer will present on Friday, June 20, clinical update from ongoing compassionate use treatment with intravenous BPM31510 in patients with Primary CoQ10 Deficiency (PCQD) at the United Mitochondrial Disease Foundation’s Mitochondrial Medicine Conference 2025 taking place in St. Louis, MO.

Dr. Modur’s presentation will detail that BPM31510 has been well-tolerated in all three PCQD patients treated so far with no serious adverse events or drug related adverse events reported during the course of the treatment. His presentation will also highlight preliminary efficacy data for the three patients, including observations of improvements in balance, strength, coordination and activities of daily living compared to what may be expected from natural progression of disease. Patient/family caregivers also reported improvements in daily living metrics.

PCQD is an ultra-rare mitochondrial disorder that impairs the body’s ability to produce CoQ10, a critical molecule in the stabilization of biomembranes, production of cellular energy or ATP, and control of cellular redox states. Affecting fewer than 1 in 100,000 people, PCQD can cause developmental delay, muscle weakness, seizures, and life-threatening involvement in vital organs, with symptomatic onset as early as birth but more typically in early childhood. Currently, there are no approved treatments that effectively correct or ameliorate CoQ10 deficiency.

“The mitochondrial disease patient community, especially the PCQD patients and families, are very excited about this potential treatment for PCQD and we are grateful for the progress BPGbio made since BPM31510 for PCQD received Rare Pediatric Disease Designation from the FDA late last year,” said Philip Yeske, Ph.D., Science & Alliance Officer of the United Mitochondrial Disease Foundation, the largest patient advocacy organization for mitochondrial disease.

Developed and advanced with BPG’s proprietary NAi Interrogative Biology Platform, BPM31510(IV) is a novel, highly bioavailable, CoQ10 metabolic approach to PCQD therapy. In oncology-focused clinical trials, BPM31510 has been shown to target the mitochondria and repotentiate a cancer cell’s ability to undergo cell death by metabolic reprogramming of the mitochondria via increased ROS production. As a lipid conjugate molecule BPM31510 may address CoQ10 bioavailability and stability issues in many mitochondrial diseases by restoring CoQ10 levels to ameliorate the effect of mutations in genes that lead to mitochondrial dysfunction. This restoration of energy generation has the potential to minimize tissue damage from toxic metabolites and preserve function in organs, such as the brain and heart.

“We believe that BPM31510 may alleviate the immense burden on patients suffering from progressive deterioration from CoQ10 deficiency, a disease which currently has no available approved therapies,” said Niven R. Narain, Ph.D., President and CEO of BPGbio. “The partnership with UMDF in raising awareness for this unmet medical need has been crucial for BPGbio to understand the patient experience and explore further development in primary mitochondrial disease.”

The Company plans to seek guidance in Q3 2025 from the FDA regarding the development of BPM31510 for PCQD on a potential expedited path available for rare diseases.

About BPM31510

BPM31510 is an investigational compound currently under development for Primary CoQ10 Deficiency (PCQD) and other indications. BPM31510 is a CoQ10-lipid nanoparticle conjugate that is administered intravenously. The compound has been evaluated in a Phase 1 dose escalation study and was found to have a safety and tolerability profile that supports further development. BPM31510 has not been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory authority. The safety and efficacy of BPM31510 have not been established by the FDA.

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 500 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.

Media Contact: media@bpgbio.com

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/de2e6989-4507-4dfa-9bec-47c58c48f306