The Role of Coproporphyrins As Endogenous Biomarkers for Organic Anion Transporting Polypeptide 1B Inhibition-Progress from 2016 to 2023. 2023

Yurong Lai
Drug Metabolism, Gilead Sciences Inc., Foster City, California yurong.lai@gilead.com.

Since the initial clinical study investigating coproporphyrins I and III (CP-I and CP-III) as endogenous biomarkers for organic anion transporting polypeptide (OATP) inhibition drug-drug interactions (DDIs) published in 2016, significant progress has been made in confirming the usefulness of the CPs, particularly CP-I, as biomarkers in assessing OATP functions. CP-I exhibits selectivity toward OATP1B activity in human subjects with genetic variants of OATP1B1. Its sensitivity to a broad spectrum of clinical OATP1B inhibitors has been established from weak to vigorous. Dose-dependent CP-I changes in healthy human subjects show agreement with DDI magnitudes of probe substrates by rifampin treatment. Physiologically based pharmacokinetic models have been established for concentration changes of plasma CP-I with OATP inhibitors, demonstrating the usefulness of supporting the quantitative translation of the effect of CP-I levels into the DDI risk assessment of potential OATP inhibitors. As plasma CP-I's sensitivity, specificity, and selectivity have been validated in humans, monitoring CP-I levels in single and multiple clinical phase I dose escalation studies is recommended for early assessment of DDI risks and understanding the full dose-response of an investigational drug to OATP inhibitions. A decision tree is proposed to preclude the need to conduct a dedicated DDI study by administering a probe substrate drug to human subjects. SIGNIFICANCE STATEMENT: The minireview summarized the validation paths of coproporphyrins I and III (CP-I and CP-III) as biomarkers of organic anion transporting polypeptide 1B (OATP1B) inhibition in humans for their selectivity, specificity, and sensitivity. The utility of monitoring CP-I to assess drug-drug interactions of OATP1B inhibition in early drug development is proposed. Changes in plasma CP-I in phase I dose range studies can be used to frame plans for late-stage development and facilitate the mechanistic understanding of complex drug-drug interactions.

UI MeSH Term Description Entries
D003306 Coproporphyrins Porphyrins with four methyl and four propionic acid side chains attached to the pyrrole rings. Elevated levels of Coproporphyrin III in the urine and feces are major findings in patients with HEREDITARY COPROPORPHYRIA.
D004347 Drug Interactions The action of a drug that may affect the activity, metabolism, or toxicity of another drug. Drug Interaction,Interaction, Drug,Interactions, Drug
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D012293 Rifampin A semisynthetic antibiotic produced from Streptomyces mediterranei. It has a broad antibacterial spectrum, including activity against several forms of Mycobacterium. In susceptible organisms it inhibits DNA-dependent RNA polymerase activity by forming a stable complex with the enzyme. It thus suppresses the initiation of RNA synthesis. Rifampin is bactericidal, and acts on both intracellular and extracellular organisms. (From Gilman et al., Goodman and Gilman's The Pharmacological Basis of Therapeutics, 9th ed, p1160) Rifampicin,Benemycin,Rifadin,Rimactan,Rimactane,Tubocin
D015415 Biomarkers Measurable and quantifiable biological parameters (e.g., specific enzyme concentration, specific hormone concentration, specific gene phenotype distribution in a population, presence of biological substances) which serve as indices for health- and physiology-related assessments, such as disease risk, psychiatric disorders, ENVIRONMENTAL EXPOSURE and its effects, disease diagnosis; METABOLIC PROCESSES; SUBSTANCE ABUSE; PREGNANCY; cell line development; EPIDEMIOLOGIC STUDIES; etc. Biochemical Markers,Biological Markers,Biomarker,Clinical Markers,Immunologic Markers,Laboratory Markers,Markers, Biochemical,Markers, Biological,Markers, Clinical,Markers, Immunologic,Markers, Laboratory,Markers, Serum,Markers, Surrogate,Markers, Viral,Serum Markers,Surrogate Markers,Viral Markers,Biochemical Marker,Biologic Marker,Biologic Markers,Clinical Marker,Immune Marker,Immune Markers,Immunologic Marker,Laboratory Marker,Marker, Biochemical,Marker, Biological,Marker, Clinical,Marker, Immunologic,Marker, Laboratory,Marker, Serum,Marker, Surrogate,Serum Marker,Surrogate End Point,Surrogate End Points,Surrogate Endpoint,Surrogate Endpoints,Surrogate Marker,Viral Marker,Biological Marker,End Point, Surrogate,End Points, Surrogate,Endpoint, Surrogate,Endpoints, Surrogate,Marker, Biologic,Marker, Immune,Marker, Viral,Markers, Biologic,Markers, Immune
D027361 Organic Anion Transporters Proteins involved in the transport of organic anions. They play an important role in the elimination of a variety of endogenous substances, xenobiotics and their metabolites from the body. Membrane Transport Proteins, Organic Anion,OAT Transport Protein,OATP Transporter,Organic Anion Transport Polypeptide,Organic Anion Transport Polypeptides,Organic Anion Transport Protein,Organic Anion Transporter,OAT Transport Proteins,OATP Proteins,OATP Transporters,Organic Anion Transport Proteins,Anion Transporter, Organic,Anion Transporters, Organic,Protein, OAT Transport,Transport Protein, OAT,Transport Proteins, OAT,Transporter, OATP,Transporter, Organic Anion,Transporters, OATP,Transporters, Organic Anion

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