Retaspimycin

Summary

Retaspimycin (CHEMBL1184904) is a phase-3 clinical-stage small-molecule Hsp90 inhibitor; with CIViC clinical evidence for 2 variant-indication associations (e.g. ALK Fusion in lung non-small cell carcinoma).

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Clinical trials: 12
• Precision-oncology evidence (CIViC): 2 variant–indication associations
• Chemistry: 587.7 Da · C31H45N3O8

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1184904
Name	Retaspimycin
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	11534420
ChEBI	CHEBI:71975
Molecular formula	C31H45N3O8
Molecular weight	587.7
InChIKey	OAKGNIRUXAZDQF-TXHRRWQRSA-N

SMILES: C[C@H]1C[C@@H]([C@@H]([C@H](/C=C(/[C@@H]([C@H](/C=C\C=C(\C(=O)NC2=CC(=C(C(=C2O)C1)NCC=C)O)/C)OC)OC(=O)N)\C)C)O)OC

IUPAC name: [(4E,6Z,8S,9S,10E,12S,13R,14S,16R)-13,20,22-trihydroxy-8,14-dimethoxy-4,10,12,16-tetramethyl-3-oxo-19-(prop-2-enylamino)-2-azabicyclo[16.3.1]docosa-1(21),4,6,10,18(22),19-hexaen-9-yl] carbamate

ChEBI definition: An ansamycin that is tanespimycin in which the benzoquinone moiety has been reduced to the corresponding hydroquinone. A semi-synthetic analogue of geldanamycin, it is used (generally as the hydrochloride salt) in cancer treatment.

Pharmacological roles (ChEBI): Hsp90 inhibitor, antineoplastic agent.

Also known as: IPI-504, Retaspimicina, Retaspimycin, Retaspimycine, RETASPIMYCIN, RETASPIMYCIN (HYDROCHLORIDE), RETASPIMYCIN HYDROCHLORIDE, Retaspimycin (Hydrochloride)

Parent form; salt/anhydrous children: CHEMBL377559

Patent coverage: 608 distinct patent families (1,551 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 1,520 (98%) 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

No target linkage available.

Bioactivity

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

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

0 indications (0 at ChEMBL trial phase 4).

Clinical trials

Total trials: 12.

Phase distribution

Phase	Trials
PHASE2	5
PHASE1/PHASE2	3
PHASE1	3
PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00688766	PHASE3	TERMINATED	Study Evaluating IPI-504 in Patients With Gastrointestinal Stromal Tumors (GIST) Following Failure of at Least Imatinib and Sunitinib
NCT00431015	PHASE1/PHASE2	COMPLETED	Phase I/II Safety Study of IPI-504 in Relapsed/Refractory Stage IIIb, or Stage IV Non-small Cell Lung Cancer (NSCLC)
NCT00564928	PHASE2	COMPLETED	A Phase 2 Study to Investigate the Clinical Activity of IPI-504 in Patients With Hormone-resistant Prostate Cancer
NCT00627419	PHASE2	TERMINATED	A Study Evaluating the Safety and Antitumor Activity of IPI-504, in Patients With Metastatic Melanoma
NCT00627627	PHASE1/PHASE2	WITHDRAWN	A Study to Evaluate the Antitumor Activity and Safety of IPI-504 in Patients With Advanced Breast Cancer
NCT00817362	PHASE2	TERMINATED	Efficacy and Safety of IPI-504 With Trastuzumab Pretreated, Locally Advanced or Metastatic HER2 Positive Breast Cancer
NCT00969917	PHASE2	WITHDRAWN	Study Evaluating the Efficacy and Safety of IPI 504 in Patients With Advanced Dedifferentiated Liposarcoma
NCT01228435	PHASE2	TERMINATED	IPI-504 in NSCLC Patients With ALK Translocations
NCT01427946	PHASE1/PHASE2	COMPLETED	Phase 1b/2 Study of Retaspimycin HCl (IPI-504) in Combination With Everolimus in KRAS Mutant Non-small Cell Lung Cancer
NCT00113204	PHASE1	COMPLETED	Safety Study of IPI-504 in Patients With Relapsed and Relapsed Refractory Multiple Myeloma
NCT00276302	PHASE1	COMPLETED	Safety Study of IPI-504 in Patients With Gastrointestinal Stromal Tumors (GIST) or Soft Tissue Sarcomas (STS)
NCT00606814	PHASE1	COMPLETED	Phase I Study of IPI-504 and Docetaxel in Patients With Advanced Solid Tumors

Clinical evidence (CIViC)

Variant × indication × effect (2 predictive associations from 2 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
ALK Fusion	Lung Non-small Cell Carcinoma	Sensitivity/Response	Retaspimycin Hydrochloride	CIViC C	EID1202
EML4::ALK Fusion	Lung Non-small Cell Carcinoma	Sensitivity/Response	Crizotinib + Retaspimycin Hydrochloride	CIViC D	EID1203

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

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