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ciprocopan   Click here for help

GtoPdb Ligand ID: 14472

Synonyms: Compound A [WO2025119266] | HSK-39297 | HSK39297
Immunopharmacology Ligand
Compound class: Synthetic organic
Comment: Ciprocopan is the INN for a complement factor B (CFB) inhibitor. CFB inhibitors are intended to reduce activation of complement-mediated inflammation by inhibiting formation of C3bBb alternative pathway C3 convertase [3]. Ciprocopan is a deuterated compound, proposed for clinical application in the treatment of pathologies that are driven by hyperactivation of the alternative complement pathway. The structure is claimed in patents held by Tibet Haisco Pharmaceutical [2], and reported by IUPAC name in [1] as the clinical candidate HSK39297.
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Ligand Activity Visualisation Charts

These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.

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2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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Physico-chemical Properties
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Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/). All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

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Hydrogen bond acceptors 5
Hydrogen bond donors 2
Rotatable bonds 6
Topological polar surface area 61.8
Molecular weight 418.53
XLogP 3.54
No. Lipinski's rules broken 0

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)

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Canonical SMILES CC1=CC(=C(CN2CC[C@H](C[C@H]2C3=CC=C(C=C3)C(=O)O)C4CC4)C5=C1NC=C5)OC([2H])([2H])[2H]
Isomeric SMILES [2H]C([2H])([2H])OC1=C(C2=C(C(=C1)C)NC=C2)CN3CC[C@H](C[C@H]3C4=CC=C(C=C4)C(=O)O)C5CC5
InChI InChI=1S/C26H30N2O3/c1-16-13-24(31-2)22(21-9-11-27-25(16)21)15-28-12-10-20(17-3-4-17)14-23(28)18-5-7-19(8-6-18)26(29)30/h5-9,11,13,17,20,23,27H,3-4,10,12,14-15H2,1-2H3,(H,29,30)/t20-,23+/m1/s1/i2D3
InChI Key VHBASIXWLLDPFC-HRFXIYCZSA-N

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
No information available.
Summary of Clinical Use Click here for help
HSK39297 has progressed through clinical trials for various inflammatory primary glomerular diseases, the most advanced being in paroxysmal nocturnal hemoglobinuria (PNH) and IgA nephropathy.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT07390123 Phase Ⅲ Study of Efficacy and Safety of HSK39297 Tablet in Treatment of Patients With Primary IgAN Phase 3 Interventional Haisco Pharmaceutical Group Co., Ltd.
NCT07052838 Efficacy and Safety of HSK39297 in Anti-C5 Treated PNH Patients With Anemia Phase 3 Interventional Haisco Pharmaceutical Group Co., Ltd.
NCT06799546 A Phase III Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy Phase 3 Interventional Haisco Pharmaceutical Group Co., Ltd.
NCT06350279 A Phase I Study to Assess the Safety,Tolerability, PK, PD, and Food Effect of HSK39297 in Healthy Subjects Phase 1 Interventional Haisco Pharmaceutical Group Co., Ltd. 1