Zotarolimus

Summary

Zotarolimus (CHEMBL219410) is a phase-3 clinical-stage small molecule targeting FKBP1A.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 1 (FKBP1A)
• Chemistry: 966.2 Da · C52H79N5O12

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL219410
Name	Zotarolimus
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	9876378
Molecular formula	C52H79N5O12
Molecular weight	966.2
InChIKey	CGTADGCBEXYWNE-JUKNQOCSSA-N

SMILES: C[C@@H]1CC[C@H]2C[C@@H](/C(=C/C=C/C=C/[C@H](C[C@H](C(=O)[C@@H]([C@@H](/C(=C/[C@H](C(=O)C[C@H](OC(=O)[C@@H]3CCCCN3C(=O)C(=O)[C@@]1(O2)O)[C@H](C)C[C@@H]4CC[C@@H]([C@@H](C4)OC)N5C=NN=N5)C)/C)O)OC)C)C)/C)OC

IUPAC name: (1R,9S,12S,15R,16E,18R,19R,21R,23S,24E,26E,28E,30S,32S,35R)-1,18-dihydroxy-19,30-dimethoxy-12-[(2R)-1-[(1S,3R,4S)-3-methoxy-4-(tetrazol-1-yl)cyclohexyl]propan-2-yl]-15,17,21,23,29,35-hexamethyl-11,36-dioxa-4-azatricyclo[30.3.1.04,9]hexatriaconta-16,24,26,28-tetraene-2,3,10,14,20-pentone

Also known as: ABT-578, Mdt-4107, Zotarolimus, ZOTAROLIMUS, zotarolimus

Patent coverage: 2,040 distinct patent families (5,042 SureChEMBL compound mentions), from 1 matched compound structure(s). 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
FKBP1A	FKBP prolyl isomerase 1A	Binding	8.55	21.5%	P62942

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Serine/threonine-protein kinase mTOR.

Bioactivity

ChEMBL activities: 1 potent at pChembl ≥ 5 of 1 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
MTOR	8.48	IC50	3.3	nM	CHEMBL_ACT_1660108

Target pathways

Aggregated over 1 target gene(s): FKBP1A.

Top Reactome pathways

7 total, by targets touching each:

Pathway	Targets	Genes
mTORC1-mediated signalling	1	FKBP1A
Calcineurin activates NFAT	1	FKBP1A
TGF-beta receptor signaling activates SMADs	1	FKBP1A
TGF-beta receptor signaling in EMT (epithelial to mesenchymal transition)	1	FKBP1A
TGFBR1 LBD Mutants in Cancer	1	FKBP1A
Potential therapeutics for SARS	1	FKBP1A
SARS-CoV-1 activates/modulates innate immune responses	1	FKBP1A

Dominant GO biological processes

GO term	Targets
heart morphogenesis	1
protein folding	1
‘de novo’ protein folding	1
regulation of cardiac muscle contraction by regulation of the release of sequestered calcium ion	1
regulation of skeletal muscle contraction by regulation of release of sequestered calcium ion	1
negative regulation of transforming growth factor beta receptor signaling pathway	1
regulation of protein localization	1
negative regulation of activin receptor signaling pathway	1
protein refolding	1
T cell activation	1
positive regulation of canonical NF-kappaB signal transduction	1
regulation of immune response	1
protein maturation	1
ventricular cardiac muscle tissue morphogenesis	1
heart trabecula formation	1

Indications & clinical

Indications

0 indications (0 at ChEMBL trial phase 4).

Clinical trials

Total trials: 0.

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.

6 molecules share ≥1 primary target. Top 6 by shared-target count:

Molecule	Source	Status	Shared targets
CYCLOSPORINE	ChEMBL	Phase 4 (approved)	FKBP1A
SIROLIMUS	ChEMBL	Phase 4 (approved)	FKBP1A
TACROLIMUS	ChEMBL	Phase 4 (approved)	FKBP1A
THIABENDAZOLE	ChEMBL	Phase 4 (approved)	FKBP1A
BIRICODAR	ChEMBL	Phase 2	FKBP1A
CYCLOHEXIMIDE	ChEMBL	Phase 2	FKBP1A