Ornithine

Summary

Ornithine (CHEMBL446143) is a phase-3 clinical-stage small-molecule hepatoprotective agent targeting GPRC6A; indicated across 8 conditions including brain disorder and neuroblastoma.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 1 (GPRC6A)
• Indications: 8 conditions
• Clinical trials: 10
• Chemistry: 132.16 Da · C5H12N2O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL446143
Name	Ornithine
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	6262
ChEBI	CHEBI:15729
Molecular formula	C5H12N2O2
Molecular weight	132.16
InChIKey	AHLPHDHHMVZTML-BYPYZUCNSA-N

SMILES: C(C[C@@H](C(=O)O)N)CN

IUPAC name: (2S)-2,5-diaminopentanoic acid

ChEBI definition: An optically active form of ornithine having L-configuration.

Pharmacological roles (ChEBI): hepatoprotective agent.

Other ChEBI roles (chemical / environmental): algal metabolite, mouse metabolite.

Also known as: NSC-758894, Ornithine, Ornithine, (l)-isomer, Ornitina, L-Ornithine, ORNITHINE, Ornithine phenylacetateÊ, Ornithine phenylacetateÂ, ornithine

Parent form; salt/anhydrous children: CHEMBL3138616, CHEMBL3184113, CHEMBL4297204

Patent coverage: 47,714 distinct patent families (160,529 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 160,528 (100%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
GPRC6A	GPRC6	Full agonist	4	0%	Q5T6X5

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

Aggregated over 1 target gene(s): GPRC6A.

Top Reactome pathways

2 total, by targets touching each:

Pathway	Targets	Genes
G alpha (q) signalling events	1	GPRC6A
Class C/3 (Metabotropic glutamate/pheromone receptors)	1	GPRC6A

Dominant GO biological processes

GO term	Targets
adenylate cyclase-activating G protein-coupled receptor signaling pathway	1
calcium-mediated signaling	1
positive regulation of insulin secretion involved in cellular response to glucose stimulus	1
response to amino acid	1
regulation of testosterone biosynthetic process	1
signal transduction	1
G protein-coupled receptor signaling pathway	1

Indications & clinical

Indications

8 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
brain disorder	3	MONDO:0005560	HP:0001298
neuroblastoma	2	MONDO:0005072	EFO:0000621
skin neoplasm	2	MONDO:0002531	EFO:0004198
medulloblastoma	2	MONDO:0007959	EFO:0002939
acute liver failure	2	MONDO:0019542	MONDO:0019542
type 1 diabetes mellitus	1	MONDO:0005147	MONDO:0005147
propionic acidemia	1	MONDO:0011628	MONDO:0011628
lung neoplasm	1	MONDO:0021117	MONDO:0008903

Clinical trials

Total trials: 10.

Phase distribution

Phase	Trials
PHASE4	4
Not specified	3
PHASE2	2
PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05737030	PHASE4	ACTIVE_NOT_RECRUITING	Effect of L-ornithine-L-aspertate (LOLA) on the Gut Microbiome
NCT01722578	PHASE4	COMPLETED	L-ornithine L-aspartate in Overt Hepatic Encephalopathy
NCT02158182	PHASE4	COMPLETED	Lactulose, L-ornithine L-aspartate, or Rifaximin Versus Placebo for Preventing Hepatic Encephalopathy in Variceal Bleeding
NCT05920213	PHASE4	UNKNOWN	Efficacy of L-Ornithine L-Aspartate and Polyethylene Glycol in Cirrhotic Patients With Overt Hepatic Encephalopathy
NCT00433368	PHASE3	COMPLETED	Efficacy of L-Ornithine-L-Aspartate in Cirrhotics With Hepatic Encephalopathy
NCT07117630	PHASE2	RECRUITING	An Open-Label, Bayesian Adaptive Phase II Clinical Study in HR+/HER2- Advanced Breast Cancer After Progression on Standard Therapy
NCT00470314	PHASE2	UNKNOWN	Therapeutic Efficacy of L-Ornithine L-Aspartate Infusion in Patients With Acute Liver Failure
NCT06483737	Not specified	RECRUITING	Human Albumin Infusion in Liver Cirrhosis and Overt Hepatic Encephalopathy (HACHE)
NCT00305591	Not specified	COMPLETED	Brain Imaging in Patients With Chronic Liver Disease and Functional Impairment.
NCT05778461	Not specified	UNKNOWN	Efficacy of L-ornithine L-aspartate and Therapeutic Plasma Exchange Versus Plasma Exchange Alone in Lowering Ammonia and Improving Outcomes in Pediatric Acute Liver Failure.

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 0 clinical and 1 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).