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Atlas / Drug / Perifosine

Perifosine

drug
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Also known as AEZS-104KRX-0401NSC-639966PerifosinaAlkylphospholipid analogueSID499931SID144206916SID174006514SID225144339

Summary

Perifosine (CHEMBL372764) is a phase-3 clinical-stage small-molecule EC 2.7.1.137 (phosphatidylinositol 3-kinase) inhibitor; indicated across 39 conditions including plasma cell myeloma and colorectal neoplasm; with CIViC clinical evidence for 3 variant-indication associations (e.g. PIK3CA E542K in thyroid cancer).

At a glance

  • Status: Max clinical phase 3 (not approved)
  • Modality: Small molecule
  • Indications: 39 conditions
  • Clinical trials: 42
  • Precision-oncology evidence (CIViC): 3 variant–indication associations
  • Chemistry: 461.7 Da · C25H52NO4P

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL372764
NamePerifosine
TypeSmall molecule
Max phase3
FDA approvedno
PubChem CID148177
ChEBICHEBI:67272
Molecular formulaC25H52NO4P
Molecular weight461.7
InChIKeySZFPYBIJACMNJV-UHFFFAOYSA-N

SMILES: CCCCCCCCCCCCCCCCCCOP(=O)([O-])OC1CC[N+](CC1)(C)C

IUPAC name: (1,1-dimethylpiperidin-1-ium-4-yl) octadecyl phosphate

ChEBI definition: A phospholipid consisting of 1,1-dimethylpiperidinium-4-yl hydrogen phosphate in which the hydrogen is replaced by a stearyl (octadecyl) group.

Pharmacological roles (ChEBI): EC 2.7.1.137 (phosphatidylinositol 3-kinase) inhibitor.

Also known as: AEZS-104, KRX-0401, NSC-639966, Perifosina, Perifosine, Alkylphospholipid analogue, SID499931, SID144206916, perifosine, SID174006514, SID225144339, PERIFOSINE

Patent coverage: 3,620 distinct patent families (15,127 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

Broader ChEMBL bioactivity targets: 4 (assay-derived). Sample: Mitogen-activated protein kinase 14, Telomerase reverse transcriptase, RAC-alpha serine/threonine-protein kinase, Ubiquitin carboxyl-terminal hydrolase isozyme L3.

Bioactivity

ChEMBL activities: 13 potent at pChembl ≥ 5 of 14 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
UCHL37.3IC5050nMCHEMBL_ACT_24772988
TERT6.74IC50184nMCHEMBL_ACT_25506791
TERT6.74IC50184nMCHEMBL_ACT_25506792
TERT6.74IC50184nMCHEMBL_ACT_25506793
TERT6.74IC50184nMCHEMBL_ACT_25506794
TERT6.74IC50184nMCHEMBL_ACT_25506795
TERT6.74IC50184nMCHEMBL_ACT_25506796
TERT6.74IC50184nMCHEMBL_ACT_25506797
TERT6.74IC50184nMCHEMBL_ACT_25506798
TERT6.74IC50184nMCHEMBL_ACT_25506799
TERT6.74IC50184nMCHEMBL_ACT_25506800
MAPK145.92IC501200nMCHEMBL_ACT_19256339
AKT15.28IC505300nMCHEMBL_ACT_12713907

Target pathways

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

Indications & clinical

Indications

36 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)PhaseMONDOEFO
plasma cell myeloma3MONDO:0009693EFO:0001378
colorectal neoplasm3MONDO:0005335MONDO:0005575
B-cell chronic lymphocytic leukemia2MONDO:0004948EFO:0000095
lymphoma2MONDO:0005062EFO:0000574
leukemia2MONDO:0005059EFO:0000565
prostate adenocarcinoma2MONDO:0005082EFO:0000673
renal cell carcinoma2MONDO:0005086EFO:0000681
cutaneous melanoma2MONDO:0005012EFO:0000389
exocrine pancreatic carcinoma2MONDO:0005192EFO:0002618
head and neck cancer2MONDO:0005627EFO:0006859
Waldenstrom macroglobulinemia2MONDO:0100280EFO:0009441
soft tissue sarcoma2MONDO:0018078EFO:1001968
kidney cancer2MONDO:0002367MONDO:0002367
breast neoplasm2MONDO:0021100MONDO:0007254
colonic neoplasm2MONDO:0005401MONDO:0021063
gastrointestinal stromal tumor2MONDO:0011719MONDO:0011719
neoplasm1MONDO:0005070EFO:0000616
non-small cell lung carcinoma1MONDO:0005233EFO:0003060
myelodysplastic syndrome1MONDO:0018881EFO:0000198
T-cell acute lymphoblastic leukemia1MONDO:0004963EFO:0000209
acute monocytic leukemia1MONDO:0007896EFO:0000221
acute myelomonocytic leukemia M41MONDO:0018871EFO:0000223
acute promyelocytic leukemia1MONDO:0012883EFO:0000224
chronic myeloid leukemia1MONDO:0011996EFO:0000339
glioblastoma1MONDO:0018177EFO:0000519
anaplastic astrocytoma1MONDO:0016684EFO:0002499
anaplastic oligodendroglioma1MONDO:0016696EFO:0002501
acute megakaryoblastic leukemia1MONDO:0018872EFO:0003025
acute myeloblastic leukemia without maturation1MONDO:0005224EFO:0003027
acute myeloblastic leukemia with maturation1MONDO:0020320EFO:0003028
gliosarcoma1MONDO:0016681EFO:1001465
acute erythroid leukemia1MONDO:0017858EFO:0000218
glioma1MONDO:0021042MONDO:0003268
ovarian cancer1MONDO:0008170MONDO:0008170
brain neoplasm1MONDO:0021211EFO:0003833
oligodendroglioma1MONDO:0016695MONDO:0002543

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

Clinical trials

Total trials: 42.

Phase distribution

PhaseTrials
PHASE221
PHASE115
PHASE1/PHASE23
PHASE32
Not specified1

Top trials by phase / activity

NCTPhaseStatusTitle
NCT01002248PHASE3TERMINATEDAssessment of Efficacy and Safety of Perifosine, Bortezomib and Dexamethasone in Multiple Myeloma Patients
NCT01097018PHASE3COMPLETEDPerifosine Plus Capecitabine Versus Placebo Plus Capecitabine in Patients With Refractory Advanced Colorectal Cancer
NCT00053781PHASE2COMPLETEDPerifosine in Treating Patients With Metastatic or Recurrent Malignant Melanoma
NCT00053794PHASE2COMPLETEDPerifosine in Treating Patients With Metastatic or Locally Advanced Soft Tissue Sarcoma
NCT00053924PHASE2COMPLETEDPerifosine in Treating Patients With Advanced Pancreatic Cancer
NCT00054145PHASE2COMPLETEDPerifosine in Treating Patients With Recurrent, Refractory, Locally Advanced, or Metastatic Breast Cancer
NCT00058214PHASE2TERMINATEDPerifosine in Treating Patients With Recurrent Prostate Cancer
NCT00059982PHASE2COMPLETEDPerifosine in Treating Patients With Locally Advanced, Unresectable, or Metastatic Pancreatic Cancer
NCT00060437PHASE2COMPLETEDPerifosine in Treating Patients With Metastatic, Androgen-Independent Prostate Cancer
NCT00062387PHASE2TERMINATEDPerifosine in Treating Patients With Recurrent or Metastatic Head and Neck Cancer
NCT00064324PHASE2COMPLETEDPerifosine in Treating Patients With Advanced Soft Tissue Sarcoma
NCT00375791PHASE2COMPLETEDEfficacy of Perifosine Alone and in Combination With Dexamethasone for Patients With Multiple Myeloma
NCT00389077PHASE2COMPLETEDTrial of Two Schedules of Perifosine for Patients With Solid Tumors or Lymphomas
NCT00391560PHASE2COMPLETEDPhase II Study of Perifosine in Patients With Refractory and Relapsed Leukemia
NCT00398710PHASE2COMPLETEDA Phase II Study of Perifosine in Patients With Relapsed/Refractory Waldenström’s Macroglobulinemia
NCT00398879PHASE2COMPLETEDPlacebo-Controlled Study of Perifosine + Single Agent Chemotherapy for Metastatic Cancer Patients
NCT00399789PHASE1/PHASE2COMPLETEDA Phase 1/2 Trial of Perifosine in the Treatment of Non-Small Cell Lung Cancer
NCT00401011PHASE1/PHASE2COMPLETEDSafety & Efficacy Study of Perifosine + Bortezomib +/- Dexamethasone for Multiple Myeloma Patients
NCT00401388PHASE2COMPLETEDA Trial of Perifosine in Patients With Chemo-Insensitive Sarcomas
NCT00422656PHASE2COMPLETEDPerifosine in Patients With Relapsed/Refractory Waldenstrom’s Macroglobulinemia
NCT00448721PHASE2COMPLETEDA Phase II Trial of Perifosine Following Tyrosine Kinase Inhibitor (TKI) - Failure in Patients With Renal Cancer
NCT00455559PHASE2COMPLETEDPh II Study of Perifosine Plus Gleevec for Patients With GIST
NCT00498966PHASE2COMPLETEDPh II Study of Perifosine for Patients With Carcinoma of the Kidney
NCT00590954PHASE2COMPLETEDClinical and Molecular-Metabolic Phase II Trial of Perifosine for Recurrent/Progressive Malignant Gliomas
NCT00873457PHASE2COMPLETEDPerifosine in Relapsed or Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
NCT01051557PHASE1/PHASE2COMPLETEDTemsirolimus and Perifosine in Treating Patients With Recurrent or Progressive Malignant Glioma
NCT00005794PHASE1COMPLETEDPerifosine in Treating Patients With Advanced Solid Tumors
NCT00019656PHASE1COMPLETEDPerifosine in Treating Patients With Refractory Solid Tumors or Hematologic Cancer
NCT00301938PHASE1COMPLETED7-Hydroxystaurosporine and Perifosine in Treating Patients With Relapsed or Refractory Acute Leukemia, Chronic Myelogenous Leukemia or High Risk Myelodysplastic Syndromes
NCT00398697PHASE1COMPLETEDPhase I Perifosine and Gemcitabine Study
NCT00398814PHASE1COMPLETEDPhase I Study of Perifosine + Sorafenib for Patients With Advanced Cancers
NCT00399087PHASE1COMPLETEDPhase I Trial of Docetaxel With Perifosine
NCT00399126PHASE1COMPLETEDPhase I Trial of Paclitaxel With Perifosine
NCT00399152PHASE1COMPLETEDPerifosine + Sunitinib Malate for Patients With Advanced Cancers
NCT00415064PHASE1COMPLETEDPhase I Study of Perifosine + Lenalidomide and Dexamethasone for Patients With Multiple Myeloma
NCT00431054PHASE1COMPLETEDPerifosine and Docetaxel in Patients With Relapsed Epithelial Ovarian Cancer
NCT00776867PHASE1COMPLETEDStudy of Single Agent Perifosine for Recurrent Pediatric Solid Tumors
NCT01048580PHASE1COMPLETEDStudy of Perifosine + Capecitabine for Colon Cancer Patients
NCT01049841PHASE1COMPLETEDPerifosine With Temsirolimus for Recurrent Pediatric Solid Tumors
NCT01224730PHASE1COMPLETEDA Food Effect and QTc Study of Perifosine in Patients With Advanced Malignancies
NCT02238496PHASE1COMPLETEDPerifosine and Torisel (Temsirolimus) for Recurrent/Progressive Malignant Gliomas
NCT00847366Not specifiedCOMPLETEDOpen Label Trial of Perifosine in Patients Currently Being Treated on Perifosine Trials in Solid Tumors or Multiple Myeloma

Clinical evidence (CIViC)

Variant × indication × effect (3 predictive associations from 3 curated evidence items):

VariantIndicationEffectTherapyLevelCIViC
PIK3CA E542KThyroid CancerSensitivity/ResponsePerifosine + TemsirolimusCIViC DEID1626
PIK3CA H1047RThyroid CancerSensitivity/ResponsePerifosine + TemsirolimusCIViC DEID1625
PTEN R130*Thyroid CancerSensitivity/ResponsePerifosine + TemsirolimusCIViC DEID1627

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

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

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.4
BioBTree
v2.0.2

Sources 17

This page pulls from 17 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclinical_trialsefogtopdb_interactiongtopdb_ligandhgncmeshmondopatent_compoundpharmgkbpharmgkb_guidelinepubchemuniprot