Drug intelligence / Profile preview

kartogenin

Development stage
Phase 1
Lead developer
Scripps Research
Modality
Small Molecules
Administration
Intraarticular, Oral
01

Overview

Kartogenin is a **small-molecule heterocyclic compound** developed to promote the chondrogenic differentiation of mesenchymal stem cells (MSCs), thereby **stimulating cartilage regeneration**. Its primary mechanism of action involves binding to the actin-binding protein filamin A and disrupting its interaction with core-binding factor β (CBFβ), which enables CBFβ to enter the nucleus and form a transcriptional complex with runt-related transcription factor 1 (RUNX1), initiating chondrogenesis-related protein transcription and cartilage extracellular matrix synthesis[3][5][7]. Additional reported mechanisms include activation of the BMP-7/Smad5 and IL-6/Stat3 signaling pathways, attenuation of RUNX1 degradation, modulation of JNK phosphorylation and β-catenin, and influence on PI3K–Akt and Hippo pathways[5]. Kartogenin demonstrates chondroprotective and chondrogenic effects in vitro and in vivo, effectively reducing cartilage degeneration and enhancing cartilage repair, notably in models of osteoarthritis and temporomandibular joint osteoarthritis[1][3][5]. In preclinical and early clinical studies, kartogenin (as KA34) has been delivered via intra-articular injection and explored in combination with biomaterials and platelet-rich plasma for tissue engineering applications[2][4][5][6].

Other names
2-([1,1'-Biphenyl]-4-ylcarbamoyl)benzoic acidN-Biphenyl-4-yl-phthalamic acidN-Biphenyl4-yl-phthalamic acidN-Biphenyl 4-yl-phthalamic acid2-[(4-phenylphenyl)carbamoyl]benzoic acid
02

Targets

RPS6KA2 (Ribosomal protein S6 kinase alpha-2)Filamin A – Core-binding factor subunit beta protein–protein interaction interfaceJNK (Mitogen-activated protein kinase kinase kinase family)

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