Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
Phosphoenolpyruvate carboxykinase 1 (soluble) (PCK1)
- Target
- PCK1
- Molecular classification
- Enzyme, Lyase (EC 4.1.1.32)
01
Overview
Phosphoenolpyruvate carboxykinase 1 (PCK1) is a cytosolic enzyme that catalyzes the conversion of oxaloacetate to phosphoenolpyruvate, the rate-limiting step in gluconeogenesis, which is the metabolic pathway producing glucose from non-carbohydrate precursors. PCK1 is highly expressed in the liver, kidney, adipose tissue, and small intestine. Its activity is tightly regulated by hormones such as insulin, glucagon, and glucocorticoids, as well as by various post-translational modifications. Dysregulation of PCK1 has been implicated in diabetes, cancer (especially hepatocellular carcinoma), and metabolic disorders. Direct inhibitors of PCK1 are of research interest for potential therapeutic modulation of glucose metabolism and cancer cell metabolic reprogramming but are not in clinical use.
- Other names
- PEPCK-CPEPCK1PEPCK
02
Mechanism of action
Inhibition of PCK1 blocks gluconeogenesis, reducing hepatic glucose output.\nIn cancer models, inhibition may disrupt tumor cell metabolism and impair anabolic processes needed for growth
03
Biological functions
Gluconeogenesis (rate-limiting step)Regulation of glucose metabolismLipid metabolismCell proliferationApoptosis (indirectly, via metabolic and oxidative stress pathways)Immune response modulation
04
Disease associations
Diabetes (implicated in impaired glucose regulation)Cancer (suppressed or dysregulated in certain tumor types, e.g. hepatocellular carcinoma)Obesity/metabolic syndromeNonalcoholic fatty liver disease (NAFLD)Aging (linked to lifespan regulation in model organisms)
05
Safety considerations
Risk of hypoglycemia due to impaired gluconeogenesisPotential disruption of hepatic or systemic energy homeostasisUnknown long-term effects on lipid metabolism, immune response, and tumor biology
06
Interacting drugs
There are no FDA-approved PCK1 inhibitors as drugs, but experimental small-molecule inhibitors and siRNA approaches have been reported in publications for research use. Glucocorticoids, insulin, and glucagon affect PCK1 expression but are not direct PCK1 inhibitors
07
Biomarkers
PCK1 expression (mRNA/protein) in liver, cancer tissue, or bloodDownstream metabolites (e.g., phosphoenolpyruvate, oxaloacetate, glucose)Functional glucose production assays
Beyond the preview
Go deeper on Phosphoenolpyruvate carboxykinase 1 (soluble) (PCK1).
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Clinical trials
Full profile accessFollow the clinical studies evaluating therapies directed at this target.
- Trial design
- Status
- Readouts
Competitive landscape
Full profile accessCompare approaches across drug candidates, modalities, and indications.
- Programs
- Modalities
- Indications
Literature & evidence
Full profile accessInvestigate the research and source evidence behind target biology and development.
- Publications
- Sources
- Analysis
Patents
Full profile accessExplore patent activity around therapies and technologies addressing this target.
- Patents
- Assignees
- Technologies
Research & analysis
Full profile accessConnect target biology, drug development, and emerging evidence in your research.
- Biology
- Development news
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on Phosphoenolpyruvate carboxykinase 1 (soluble) (PCK1).