Upacicalcet

Summary

Upacicalcet (CHEMBL6068421) is a phase-3 clinical-stage small molecule targeting CASR; indicated across 3 conditions including secondary hyperparathyroidism and chronic kidney disease.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 1 (CASR)
• Indications: 3 conditions
• Clinical trials: 5
• Chemistry: 351.76 Da · C11H14ClN3O6S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL6068421
Name	Upacicalcet
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	53374467
Molecular formula	C11H14ClN3O6S
Molecular weight	351.76
InChIKey	LHEYGVSDVBEYQF-QMMMGPOBSA-N

SMILES: CC1=C(C=C(C=C1Cl)S(=O)(=O)O)NC(=O)NC[C@@H](C(=O)O)N

IUPAC name: (2S)-2-amino-3-[(3-chloro-2-methyl-5-sulfophenyl)carbamoylamino]propanoic acid

Also known as: AJT-240, AJT240, PLS-240, Pls240, PLS240, Sk-1403, SK-1403, Upacicalcet, Upacicalcet sodium hydrate, Upasita, UPACICALCET

Parent form; salt/anhydrous children: CHEMBL6068422

Patent coverage: 11 distinct patent families (34 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 28 (82%) 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
CASR	CaS receptor	Positive	8.09	0.1%	P41180

Bioactivity

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

Target pathways

Aggregated over 1 target gene(s): CASR.

Top Reactome pathways

7 total, by targets touching each:

Pathway	Targets	Genes
Signal Transduction	1	CASR
Signaling by GPCR	1	CASR
GPCR downstream signalling	1	CASR
G alpha (q) signalling events	1	CASR
G alpha (i) signalling events	1	CASR
Class C/3 (Metabotropic glutamate/pheromone receptors)	1	CASR
GPCR ligand binding	1	CASR

Dominant GO biological processes

GO term	Targets
ossification	1
response to ischemia	1
detection of calcium ion	1
intracellular calcium ion homeostasis	1
G protein-coupled receptor signaling pathway	1
adenylate cyclase-inhibiting G protein-coupled receptor signaling pathway	1
phospholipase C-activating G protein-coupled receptor signaling pathway	1
JNK cascade	1
chemosensory behavior	1
positive regulation of cell population proliferation	1
anatomical structure morphogenesis	1
positive regulation of gene expression	1
positive regulation of insulin secretion	1
bile acid secretion	1
cellular response to hepatocyte growth factor stimulus	1

Indications & clinical

Indications

3 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
secondary hyperparathyroidism	3	MONDO:0006964	EFO:1001173
chronic kidney disease	1	MONDO:0005300	EFO:0003884

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 5.

Phase distribution

Phase	Trials
PHASE3	4
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03626948	PHASE3	COMPLETED	SK-1403 Long-term Treatment Study; Long-term Study in Patients With Secondary Hyperparathyroidism Receiving Hemodialysis
NCT03801980	PHASE3	COMPLETED	Phase 3 Study of SK-1403
NCT05832931	PHASE3	COMPLETED	Parathyroid Hormone (PTH) Attenuation Trial in Hemodialysis-1
NCT05836220	PHASE3	COMPLETED	Parathyroid Hormone (PTH) Attenuation Trial in Hemodialysis-2
NCT03226171	PHASE2	COMPLETED	Dose Adjustment Trial of SK-1403 in Hemodialysis Patients With Secondary Hyperparathyroidism

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

8 molecules share ≥1 primary target. Top 8 by shared-target count:

Molecule	Source	Status	Shared targets
CINACALCET	ChEMBL + PubChem	Phase 4 (approved)	CASR
ENCALERET	ChEMBL	Phase 3	CASR
EVOCALCET	ChEMBL	Phase 3	CASR
FENDILINE	ChEMBL	Phase 2	CASR
RONACALERET	ChEMBL	Phase 2	CASR
SB-423562	ChEMBL	Phase 2	CASR
TECALCET	ChEMBL	Phase 2	CASR
tryptophan	PubChem	Approved	CASR