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The Importance of CoQ10 and Mitochondria

Known as the “powerhouses of the cell,” mitochondria produce the energy necessary for the cell’s survival and functioning.

A key driver of mitochondrial function is Coenzyme Q10 (CoQ10), also known as ubiquinone, which is a vitamin-like fat-soluble substance found in the inner mitochondrial membrane of cells. CoQ10 is one of the key molecules involved in the electron transport system, which helps generate energy through the formation of adenosine triphosphate (ATP), which drives cellular function and life.

BPGbio has developed a therapy that increases the levels of CoQ10. In many mitochondrial diseases, restoring or increasing CoQ10 levels can overcome the effect of mutations in genes that lead to mitochondrial dysfunction. This restoration of energy generation can minimize tissue damage from toxic metabolites and preserve function in organs like the brain, heart, and kidney.

What is Primary CoQ10 Deficiency (PCQD)?

 

Primary CoQ10 Deficiency (PCQD) is a rare but often severe multisystem disease caused by mutations in one of at least ten genes required for CoQ10 biosynthesis, leading to significantly reduced CoQ10 levels.

This deficiency impairs mitochondrial energy production and can result in dysfunction across multiple organ systems, including the central nervous system, heart, skeletal muscle, kidneys, and liver. Clinical manifestations may appear from infancy through adulthood, depending on the severity of the genetic defect.

 

 

It is estimated that less than one in 100,000 people are affected with PCQD in the United States. These are likely underestimates due in part to its rarity resulting from misdiagnosis or lack of adequate genetic testing. One study estimated ~200 people affected in the United States; however, it noted the number could be higher if we expand to “predicted” generic variation (the study can be found here).

 

Read Julia’s full My Mito Story: Living with CoQ10 Deficiency.

Oral Supplementation Efficacy Inconsistent

Historically, PQCD has been treated with oral supplementation of CoQ10. However, oral supplementation has not shown consistent efficacy due to varying formulations and low bioavailability (see below).

 

BPM31510 has high potential for
Primary CoQ10 Deficiency

BPGbio’s BPM31510(IV) has high therapeutic potential for Primary CoQ10 Deficiency

Derived from our NAi Interrogative Biology® Platform using Bayesian methodologies, BPM31510(IV) is a novel, highly bioavailable, CoQ10-based therapy that uses a proprietary nanoparticle formulation to overcome the bioavailability limitations of traditional oral CoQ10 supplementation.

Administered via IV, BPM31510 is:

  • a stable proprietary formulation
  • contains oxidized CoQ10
  • a bio-membrane like capsule
  • offers high bioavailability
  • achieves high levels in mitochondria

 

BPM31510 is formulated to optimize tissue uptake and impact mitochondrial function. Critically, BPM31510 achieves high circulating levels after infusion and is known to be delivered to the brain and other organs.

In vivo CoQ10 levels in blood plasma and muscle tissue show tremendous differences when given in equal doses via oral OTC versus BPM31510-IV in experimental models.

BPM31510-IV increased CoQ10 levels >500x in plasma, and >50X in muscle tissue versus oral OTC CoQ10 which showed a negligible effect in both in experimental models.

 

BPM31510 addresses CoQ10 issues in a highly underserved population

Rare diseases often have many hurdles in therapeutic development. Thanks to our NAi Interrogative Biology® Platform we can overcome many of them and offer new novel therapeutics, such as BPM31510, to these underserved populations.

BPM31510’s mechanism of action is promising as a treatment for PCQD:

  • It addresses lackluster success rates of oral CoQ10 replacement due to poor blood and tissue penetration
  • It is a highly bioavailable form of CoQ10 administered parenterally
  • CoQ10 therapy has shown limited benefit in treating PCQD, as it has historically achieved only 2–4× higher CoQ10 levels in plasma above baseline, while BPM31510 has demonstrated levels more than 900-fold above baseline with a favorable safety profile.

Presentation Posters

MitoCon 2026 Poster In-vivo CoQ4 PDSS2

MitoCon 2026 Poster CoQ10 Deficiency Clinical

MMTD 2025 – Poster – BPM31510 CoQ10 Deficiency

MMTD 2025 – Poster – BPM31510 Increases CoQ Pool

ASHG 2025 – CoQ10 Deficiency

UMDF Mito 2025 Poster – BPM31510 and PCQD

ASMS 2025 Poster – CoQ10 Quinomics

Mito Trans 2025 Poster_COQ10 Quinomics

MitoCon 2024 Poster – Employment of Spatial Omics and Quinomics Technologies for the Advancement of Mitochondrial Targeted Therapeutics

Trial Status

We are currently planning a potential pivotal trial for PCQD.

BPGbio has received FDA Orphan and Rare Pediatric Disease Designation for its Potential Treatment for Primary Coenzyme Q10 Deficiency.

Additionally, BPM31510 demonstrated a tolerable safety profile in clinical trials and is currently being studied as an oncology drug candidate for two disease indications:

Ongoing phase 2 trial for GBM here.

Ongoing phase 2 trial for Pancreatic Cancer here.

 

1DiMauro S, Schon EA, Carelli V, Hirano M. The clinical maze of mitochondrial neurology. Nat Rev Neurol. 2013;9:429-44. [PMC free article] [PubMed] [Reference list]