Posaconazole

Summary

Posaconazole (CHEMBL1397) is an approved small-molecule trypanocidal drug (ATC J02AC04); indicated across 15 conditions including aspergillosis and coccidioidomycosis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: J02AC04
• Indications: 15 conditions
• Clinical trials: 65
• Chemistry: 700.8 Da · C37H42F2N8O4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1397
Name	Posaconazole
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	468595
ChEBI	CHEBI:64355
ATC	J02AC04
Molecular formula	C37H42F2N8O4
Molecular weight	700.8
InChIKey	RAGOYPUPXAKGKH-XAKZXMRKSA-N

SMILES: CC[C@@H]([C@H](C)O)N1C(=O)N(C=N1)C2=CC=C(C=C2)N3CCN(CC3)C4=CC=C(C=C4)OC[C@H]5C[C@](OC5)(CN6C=NC=N6)C7=C(C=C(C=C7)F)F

IUPAC name: 4-[4-[4-[4-[[(3R,5R)-5-(2,4-difluorophenyl)-5-(1,2,4-triazol-1-ylmethyl)oxolan-3-yl]methoxy]phenyl]piperazin-1-yl]phenyl]-2-[(2S,3S)-2-hydroxypentan-3-yl]-1,2,4-triazol-3-one

ChEBI definition: An N-arylpiperazine that consists of piperazine carrying two 4-substituted phenyl groups at positions 1 and 4. A triazole antifungal drug.

Pharmacological roles (ChEBI): trypanocidal drug.

Also known as: (-)-posaconazole, Noxafil, Noxafil powdermix kit, Posaconazol, Posaconazole, Posaconazole accord, Posaconazole ahcl, Posaconazole sp, SCH 56592, SCH-56592, posaconazole, pasaconazole

Parent form; salt/anhydrous children: CHEMBL1908108, CHEMBL4303216

Patent coverage: 229 distinct patent families (541 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 322 (60%) 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

Broader ChEMBL bioactivity targets: 14 (assay-derived). Sample: Sterol 14-alpha demethylase, Sterol 14-alpha demethylase cyp51A, Lanosterol 14-alpha demethylase, Cytochrome P450 11B1, mitochondrial, Aromatase, Prostaglandin G/H synthase 2, Cytochrome P450 11B2, mitochondrial, Lanosterol 14-alpha demethylase, Cytochrome P450 2C9, Cytochrome P450 3A4.

Bioactivity

ChEMBL activities: 23 potent at pChembl ≥ 5 of 23 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
Q7Z1V1	7.75	Kd	18	nM	CHEMBL_ACT_19003260
CYP11B2	7.68	IC50	21	nM	CHEMBL_ACT_18261046
CYP17A1	7.38	IC50	42	nM	CHEMBL_ACT_18261048
Q7Z1V1	7.32	IC50	48	nM	CHEMBL_ACT_18544865
CYP3A4	7.3	IC50	50	nM	CHEMBL_ACT_15010517
Q7Z1V1	7.22	Kd	60	nM	CHEMBL_ACT_12662252
CYP3A4	7.12	IC50	75	nM	CHEMBL_ACT_18261016
P10613	7.09	Kd	81	nM	CHEMBL_ACT_18303828
CYP3A4	7.09	IC50	82	nM	CHEMBL_ACT_3299980
B8NUK6	6.88	Kd	131	nM	CHEMBL_ACT_18303836
CYP3A4	6.82	IC50	150	nM	CHEMBL_ACT_18951847
CYP11B1	6.51	IC50	310	nM	CHEMBL_ACT_18261044
CYP3A4	6.46	IC50	350	nM	CHEMBL_ACT_2610839
CYP3A4	6.17	IC50	672.1	nM	CHEMBL_ACT_19050864
Q7Z1V1	6.06	IC50	880	nM	CHEMBL_ACT_24815548
CYP17A1	5.75	IC50	1800	nM	CHEMBL_ACT_18261050
Q4WNT5	5.57	Kd	2690	nM	CHEMBL_ACT_5229870
PTGS2	5.48	AC50	3300	nM	CHEMBL_ACT_25166757
CYP8B1	5.42	IC50	3800	nM	CHEMBL_ACT_19326110
ABCG2	5.22	IC50	6000	nM	CHEMBL_ACT_24777413
CYP2C9	5.14	IC50	7200	nM	CHEMBL_ACT_18261042
ABCB11	5.09	IC50	8100	nM	CHEMBL_ACT_18129072
CYP19A1	5.08	IC50	8400	nM	CHEMBL_ACT_18261052

Target pathways

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

Indications & clinical

Indications

15 indications (5 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
aspergillosis	4	MONDO:0005657	EFO:0007157
coccidioidomycosis	4	MONDO:0005706	EFO:0007211
neutropenia	4	MONDO:0001475	MONDO:0001475
candidiasis	4	MONDO:0002026	MONDO:0002026
fungal infectious disease	4	MONDO:0002041	MONDO:0002041
leukopenia	3	MONDO:0003785	EFO:0004233
oral candidiasis	3	MONDO:0005886	EFO:0007406
Chagas disease	2	MONDO:0001444	MONDO:0001444
Zygomycosis	2	MONDO:0019136	EFO:0007380
chronic granulomatous disease	2	MONDO:0018305	MONDO:0018305
tinea unguium	2	MONDO:0001628	MONDO:0001628
neoplasm	1	MONDO:0005070	EFO:0000616
acute myeloid leukemia	1	MONDO:0018874	EFO:0000222
leukemia	1	MONDO:0005059	EFO:0000565
glioblastoma	0	MONDO:0018177	EFO:0000519

Clinical trials

Total trials: 65.

Phase distribution

Phase	Trials
PHASE3	14
PHASE2	13
PHASE1	11
Not specified	10
PHASE4	9
PHASE1/PHASE2	4
EARLY_PHASE1	3
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03717623	PHASE4	RECRUITING	Posaconazole Pharmacokinetics in Patients Receiving Chemotherapy or Stem Cell Transplants
NCT00177749	PHASE4	TERMINATED	Limited Access Protocol of Posaconazole in Invasive Fungal Infections Study PO2095
NCT00686543	PHASE4	COMPLETED	Oral Posaconazole in High Risk Patients With Gastrointestinal Dysfunction (Study P05115)
NCT01200355	PHASE4	COMPLETED	Posaconazole Versus Micafungin for Prophylaxis Against Invasive Fungal Infections During Neutropenia in Patients Undergoing Chemotherapy for Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia or Myelodysplastic Syndrome
NCT01667107	PHASE4	TERMINATED	A Pharmacokinetic Analysis of Posaconazole in Lung Transplant Recipients (MK-5592-105)
NCT02020213	PHASE4	WITHDRAWN	Posaconazole as Salvage Therapy for Aspergillus Pulmonary Infection.
NCT02805946	PHASE4	COMPLETED	Pharmacokinetics of Posaconazole (Noxafil®) as Prophylaxis for Invasive Fungal Infections
NCT02875743	PHASE4	COMPLETED	King’s Invasive Aspergillosis Study II
NCT02968134	PHASE4	COMPLETED	Single Dose Population Pharmacokinetics of Intravenous Posaconazole in Critically Ill Patients
NCT07261150	PHASE3	NOT_YET_RECRUITING	Histoplasmosis Induction and Consolidation Therapy Factorial Randomized Clinical Trial (Histo-FACT)
NCT00002446	PHASE3	COMPLETED	Safety and Effectiveness of Fluconazole Versus SCH 56592 to Treat Thrush in HIV-Positive Patients
NCT00033982	PHASE3	COMPLETED	Posaconazole to Treat Invasive Fungal Infections
NCT00034632	PHASE3	COMPLETED	Study of Posaconazole in the Treatment of Invasive Fungal Infections (Study P02095)
NCT00034645	PHASE3	COMPLETED	Prophylaxis Trial in High Risk Allogenic Stem Cell Transplant Recipients With Graft vs. Host Disease
NCT00034658	PHASE3	COMPLETED	Open Label Study of Posaconazole in the Treatment of Invasive Fungal Infections (Study P00041)
NCT00044486	PHASE3	COMPLETED	Prophylaxis Trial of Posaconazole Versus Standard Azole Therapy for Neutropenic Patients (Study P01899)
NCT00423267	PHASE3	COMPLETED	POS vs FLU for First Line Treatment of Coccidioidomycosis (Study P04558Am1)(COMPLETED)
NCT00750737	PHASE3	COMPLETED	Oral Posaconazole Three Times Per Day vs Weekly High Dose Amphotericin B Lipid Complex (ABLC)
NCT00811642	PHASE3	COMPLETED	Posaconazole Treatment of Invasive Fungal Infection (IFI) (P05551)
NCT00811928	PHASE3	COMPLETED	Safety and Efficacy Study of Posaconazole vs. Fluconazole for Prevention of Invasive Fungal Infection (P05387 AM1)(COMPLETED)
NCT01782131	PHASE3	COMPLETED	A Study of the Safety and Efficacy of Posaconazole Versus Voriconazole for the Treatment of Invasive Aspergillosis (MK-5592-069)
NCT02180165	PHASE3	COMPLETED	Assessing the Safety and Efficacy of MK-5592 (Posaconazole) in Japanese Participants With Fungal Infection (MK-5592-101)
NCT04368559	PHASE3	COMPLETED	Study of Rezafungin Compared to Standard Antimicrobial Regimen for Prevention of Invasive Fungal Diseases in Adults Undergoing Allogeneic Blood and Marrow Transplantation
NCT04966234	PHASE2/PHASE3	UNKNOWN	A New Posaconazole Dosing Regimen for Paediatric Patients With Cystic Fibrosis and Aspergillus Infection
NCT04771130	PHASE1/PHASE2	RECRUITING	A Study of BGB-11417 in Participants With Myeloid Malignancies
NCT06541067	PHASE2	RECRUITING	Study of Posaconazole Prophylaxis in Patients Receiving Hematopoietic Stem Cell Allograft (Allo-HSC) at High Risk of Invasive Fungal Infection (IFI)
NCT06802757	PHASE2	RECRUITING	Posaconazole Plus PD-1 Inhibitors and Chemotherapy vs PD-1 Inhibitors and Chemotherapy in Neoadjuvant Therapy for Triple Negative Breast Cancer
NCT07155226	PHASE1/PHASE2	RECRUITING	Study of AZD3632 Monotherapy or in Combination With Anticancer Agents in Participants With Advanced Haematologic Malignancies With KMT2Ar, NPM1m, or Other Genotypes Associated With HOX Overexpression
NCT00002399	PHASE2	COMPLETED	A Comparison of SCH 56592 and Fluconazole in the Treatment of Oropharyngeal Candidiasis (OPC) in HIV-Positive Patients
NCT00034671	PHASE2	COMPLETED	PK/PD Study of Posaconazole for Empiric Treatment of Invasive Fungal Infections in Neutropenic Patients or Treatment of Refractory Invasive Fungal Infections (Study P01893)
NCT00491764	PHASE2	COMPLETED	A Study to Evaluate Efficacy and Safety of Four Posaconazole Regimens With Placebo and Terbinafine in the Treatment of Toenail Onychomycosis (Study P05082AM2)(COMPLETED)
NCT00550732	PHASE2	COMPLETED	A Study to Examine the Efficacy and Safety of Posaconazole When Introduced Early in the Treatment of Refractory Fungal Infections (P05090 AM2)
NCT00799071	PHASE2	COMPLETED	Pharmacokinetics of Posaconazole in Children With Chronic Granulomatous Disease (CGD)
NCT00936117	PHASE2	COMPLETED	Pharmacokinetics of Posaconazole Prophylaxis in Acute Leukemia
NCT01162967	PHASE2	COMPLETED	Clinical Trial For The Treatment Of Chronic Chagas Disease With Posaconazole And Benznidazole
NCT01377480	PHASE2	COMPLETED	A Study of the Use of Oral Posaconazole (POS) in the Treatment of Asymptomatic Chronic Chagas Disease (P05267)
NCT03318159	PHASE2	COMPLETED	Posaconazole Prophylaxis During ATG Treatment for hMDS/AA Patients
NCT03760445	PHASE1/PHASE2	WITHDRAWN	HDM201 Added to CT in R/R or Newly Diagnosed AML
NCT04194086	PHASE1/PHASE2	COMPLETED	Population Pharmacokinetics and Safety of Oral Posaconazole in Children With Leukemia
NCT04218851	PHASE2	COMPLETED	Posaconazole (MK-5592) Intravenous and Oral in Children With Invasive Aspergillosis (IA) (MK-5592-104)

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

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