Drug intelligence / Profile preview

BQ-123, MitoQ, tetrahydrobiopterin

Development stage
Unknown
Lead developer
BioMarin
Modality
Peptides, Small Molecules
Administration
Oral
01

Overview

This is a hypothetical combination of three distinct compounds: BQ-123, MitoQ, and tetrahydrobiopterin. BQ-123 is a cyclic pentapeptide that acts as a potent and selective antagonist of the endothelin A (ETA) receptor, inhibiting endothelin-1-mediated cellular proliferation and demonstrating anti-inflammatory effects through polymorphonuclear myeloid-derived suppressor cell activation. MitoQ is a mitochondria-targeted antioxidant, a modified Coenzyme Q10 derivative designed to accumulate within mitochondria. It neutralizes free radicals, reduces oxidative stress, enhances endogenous antioxidant enzymes, and supports mitochondrial function, potentially inhibiting the NLRP3 inflammasome and activating the Nrf2-ARE pathway. Tetrahydrobiopterin (BH4), also known as sapropterin, is an essential endogenous cofactor for aromatic amino acid hydroxylase enzymes (phenylalanine hydroxylase, tyrosine hydroxylase, tryptophan hydroxylase) and nitric oxide synthases. It is crucial for phenylalanine metabolism, neurotransmitter biosynthesis (serotonin, dopamine, norepinephrine, epinephrine), and nitric oxide production. BH4 also contributes to energy generation, antioxidant defense, and inflammation modulation. While tetrahydrobiopterin is an approved drug for conditions like phenylketonuria and BH4 deficiency, BQ-123 and MitoQ are primarily in research and development stages for various indications.

Other names
BQ-123 + MitoQ + tetrahydrobiopterinBQ-123 + MitoQ + BH4
02

Targets

EDNRA (Endothelin receptor type A)

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