Compound Detail
CHEMBL4594448
IPTACOPAN 💊 Approved Drug
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💊 Approved Drug
CCO[C@H]1CCN(Cc2c(OC)cc(C)c3[nH]ccc23)[C@H](c2ccc(C(=O)O)cc2)C1
Basic Information
| ChEMBL ID | CHEMBL4594448 |
| Name | IPTACOPAN |
| Phase | Approved |
| Structure Type | MOL |
| InChI Key | RENRQMCACQEWFC-UGKGYDQZSA-N |
| Therapeutic | ✓ Yes |
2
Targets
8
Activities
2
Assay Types
11
PK Parameters
5
Publications
5
Known Names
11
Indications
1
Mechanisms
Molecular Properties
422.5
MW (Da)
4.93
ALogP
4
HBA
2
HBD
74.8
PSA (Ų)
0.56
QED
0
Ro5 Violations
7
Rot. Bonds
Drug Information
| Molecule Type | Small molecule |
| First Approval | 2023 |
| Availability | Prescription |
| Chirality | Single Enantiomer |
Mechanism of Action
| Mechanism | Action Type | Target | Direct | Efficacy |
|---|---|---|---|---|
| Complement factor B inhibitor | INHIBITOR | Complement factor B | ✓ | ✓ |
Indications
Atypical Hemolytic Uremic Syndrome Glomerulonephritis, IGA Glomerulonephritis, Membranoproliferative Hemoglobinuria Glomerulonephritis Glomerulonephritis, Membranous Lupus Nephritis Macular Degeneration Hemoglobinuria, Paroxysmal Kidney Diseases Liver Diseases
ATC Classification
L04AJ08
iptacopan
Level 1: ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS
Level 2: IMMUNOSUPPRESSANTS
Activity Summary
IC50 7 meas. best 11.0
Kd 1 meas. best 8.1
Target Activity Profile
| Target | Type | Best pChEMBL | Mean pChEMBL | Best (nM) | # Data |
|---|---|---|---|---|---|
| Complement factor B CHEMBL5731 | IC50 | 11.0 | 7.634 | 0.0 | 5 |
| Complement factor B CHEMBL5731 | Kd | 8.1 | 8.1 | 7.9 | 1 |
| Complement factor B CHEMBL4630808 | IC50 | 6.96 | 6.96 | 110.0 | 1 |
Pharmacokinetic Data
| Parameter | Value | Units | Species |
|---|---|---|---|
| AUC | 3211.23 | ng.hr.mL-1 | Mus musculus |
| AUC | 32154.53 | ng.hr.mL-1 | Rattus norvegicus |
| AUC | 705.63 | ng.hr.mL-1 | Rattus norvegicus |
| AUC | 12084.36 | ng.hr.mL-1 | Rattus norvegicus |
| CL | 16.0 | mL.min-1.kg-1 | Mus musculus |
| Cmax | 2500.0 | nM | Rattus norvegicus |
| Cmax | 150.0 | nM | Rattus norvegicus |
| Cmax | 5100.0 | nM | Rattus norvegicus |
| F | 46.0 | % | Mus musculus |
| MRT | 1.6 | hr | Mus musculus |
| T1/2 | 7.2 | hr | Mus musculus |
Activity Data Samples
Top measurements by pChEMBL value
| Target | Type | Rel. | Value | Units | pChEMBL | Assay | PubMed |
|---|---|---|---|---|---|---|---|
| Complement factor B | IC50 | = | 0.01 | nM | 11.0 | Human Complement Factor B TR-FRET Assay: CVF-Bb complex prepared from purified c... | - |
| Complement factor B | Kd | = | 7.9 | nM | 8.1 | Binding affinity to human serine protease factor B by SPR assay | 32073845 |
| Complement factor B | IC50 | = | 12.0 | nM | 7.92 | Inhibition of human serine protease factor B by TR-FRET based competition bindin... | 32073845 |
| Complement factor B | IC50 | = | 110.0 | nM | 6.96 | Inhibition of serine protease factor B in 50% mouse serum assessed as reduction ... | 32073845 |
| Complement factor B | IC50 | = | 130.0 | nM | 6.89 | Inhibition of serine protease factor B in 50% human whole blood assessed as redu... | 32073845 |
| Complement factor B | IC50 | = | 400.0 | nM | 6.4 | Inhibition of human serine protease factor B in human PNH patient derived sample... | 32073845 |
| Complement factor B | IC50 | = | 1100.0 | nM | 5.96 | Human Complement Factor B TR-FRET Assay: CVF-Bb complex prepared from purified c... | - |
Literature References
| PubMed | DOI | Target Context | # Activities |
|---|---|---|---|
| 32073845 | 10.1021/acs.jmedchem.9b01870 | ADMET | 16 |
| 32073845 | 10.1021/acs.jmedchem.9b01870 | Mus musculus | 13 |
| 32073845 | 10.1021/acs.jmedchem.9b01870 | Complement factor B | 5 |
| 32073845 | 10.1021/acs.jmedchem.9b01870 | Complement factor D | 1 |
| 32073845 | 10.1021/acs.jmedchem.9b01870 | Rattus norvegicus | 1 |
Data from ChEMBL 36 via Core Engine