Skip navigation

Primary CoQ10 Deficiency

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. Through a series of chemical reactions, mitochondria break down glucose into ATP, which is used to fuel cellular processes.

Coenzyme Q10 (CoQ10), also known as ubiquinone, is a vitamin-like fat-soluble substance found in the mitochondria of human cells. CoQ10 is one of the key molecules involved in the electron transport chain and generation of energy molecule known as adenosine triphosphate (ATP).

BPGbio has developed a therapy that increases the levels of CoQ10. Increasing levels of CoQ10 in cancer cells restores the normal process of energy generation in the mitochondria leading to tumor cell death. In many mitochondrial diseases, restoring 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 and heart.

 

What is Primary CoQ10 Deficiency?

Primary CoQ10 Deficiency is a rare disease with severe manifestations.

CoQ10 is a lipid component of the mitochondrial respiratory chain, leading to the classification of Primary CoQ10 Deficiency as a mitochondrial respiratory chain disorder1. It is characterized by a reduction in CoQ10 levels brought on by mutation in one of at least ten genes required for its synthesis.

Ultimately, lack of CoQ10 leads to poor functioning mitochondria which can  manifest as a severe disease affecting different organ systems including the CNS, cardiovascular, muscle, kidney, and liver. These manifestations can appear anytime from birth to late in life depending on the severity of genetic defect.

 

 

It is estimated that between 1000 and 1500 people are affected in the United States alone or about one in 100,000 people or less. These are likely underestimates due in part to its rarity resulting from misdiagnosis or lack of adequate genetic testing.

Historically, Primary CoQ10 Deficiency 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 potential for Primary CoQ10 Deficiency

Our NAi Interrogative Platform derived BPM31510(IV) is a novel, highly bioavailable, CoQ10 metabolic approach to therapy. This revolutionary lipid conjugate molecule addresses CoQ10 bioavailability issues that limit oral 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 circulation lifetime and tissue uptake. Critically, BPM31510 achieves high circulating levels after infusion and is known to penetrate the brain and other organs.

Administered via IV, BPM31510 has more than 1000x greater drug exposure than oral options.

 

BPM31510 addresses CoQ10 issues in a highly underserved population

Rare diseases often have many hurdles in therapeutic development. Thanks to our NAi Interrogative 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 primary CQ10 deficiency:

  • It addresses lackluster success rates due to poor blood and tissue penetration associated with oral CoQ10 replacement
  • It is a highly bioavailable form of CoQ10 administered parenterally
  • It has achieved >200x CoQ10 plasma levels in clinical trials and has a favorable safety profile
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.

 

Trial Status

BPGbio is currently exploring clinical trials for Primary CoQ10 Deficiency.

 

 

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]