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DRUG CLASS:

PDGFR α inhibitor

13d
Benign Keratoses With Inverted Follicular Keratosis-Like Features in a Patient Receiving Ripretinib: A Case Report. (PubMed, J Cutan Pathol)
Inverted follicular keratosis (IFK) is a rare benign neoplasm of the follicular infundibulum and typically presents as a solitary, nonpigmented verrucous papule on the head and neck of older males and can mimic viral warts, basal cell carcinoma (BCC), or squamous cell carcinoma (SCC). To the best of our knowledge, this represents the first reported association between repretinib treatment and benign keratoses with IFK-like features. Further studies are needed to clarify the clinical significance and biological implications of this observation.
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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Qinlock (ripretinib)
29d
EGFR/KIT-linked proliferative bias in normal breast lobules from matched non-Hispanic Black and White women is rapidly reversible by RTK inhibition. (PubMed, Am J Pathol)
Functional plausibility was assessed in pre-stasis human mammary epithelial cells (HMECs) exposed for 24 h to clinically relevant EGFR (lapatinib) or KIT (ripretinib) inhibitors. Together, matched normal tissue and primary-cell data indicate a coordinated EGFR/KIT-linked proliferative bias in NHBW epithelium that is rapidly reversible in vitro. These findings motivate composition-aware validation in larger normal-tissue cohorts and pharmacodynamic window studies to test whether short-term RTK modulation can reset proliferation-biased states in at-risk breast epithelium.
Journal
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EGFR (Epidermal growth factor receptor) • CDK4 (Cyclin-dependent kinase 4)
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lapatinib • Qinlock (ripretinib)
2ms
INTRIGUE: A Study of Ripretinib vs Sunitinib in Advanced GIST Patients After Treatment With Imatinib (clinicaltrials.gov)
P3, N=453, Active, not recruiting, Deciphera Pharmaceuticals, LLC | Trial completion date: Dec 2025 --> Dec 2026
Trial completion date
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imatinib • sunitinib • Qinlock (ripretinib)
2ms
New P2/3 trial
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KIT (KIT proto-oncogene, receptor tyrosine kinase)
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KIT exon 9 mutation
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Stivarga (regorafenib) • NB003
3ms
Disruption of gastrointestinal pdgfrα+ cells leads to loss of post-junctional inhibitory motor responses. (PubMed, Am J Physiol Gastrointest Liver Physiol)
Purinergic inhibitory post-junctional motor responses were greatly attenuated in the GI tracts of crenolanib treated animals compared to vehicle treated controls in response to electric field evoked nerve stimulation. These data provide evidence for a functional role of PDGFRα+ cells in inhibitory neuroeffector motor responses throughout the gastrointestinal tract.
Journal
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KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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FOLR1 expression • KIT expression
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crenolanib (ARO-002)
3ms
A Study of NB003 in Patients With Advanced Malignancies (clinicaltrials.gov)
P1, N=258, Completed, Ningbo Newbay Technology Development Co., Ltd | Recruiting --> Completed | Trial completion date: Dec 2025 --> Aug 2025
Trial completion • Trial completion date
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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NB003
3ms
INSIGHT: A Study of Ripretinib vs Sunitinib in Patients With Advanced GIST With Specific KIT Exon Mutations Who Were Previously Treated With Imatinib (clinicaltrials.gov)
P3, N=54, Active, not recruiting, Deciphera Pharmaceuticals, LLC | Recruiting --> Active, not recruiting | Trial completion date: Dec 2027 --> Dec 2028 | Trial primary completion date: Feb 2026 --> Dec 2027
Enrollment closed • Trial completion date • Trial primary completion date
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imatinib • sunitinib • Qinlock (ripretinib)
4ms
INTEREST: Ripretinib (QINLOCK®) According to Current SmPC (clinicaltrials.gov)
P=N/A, N=10, Completed, iOMEDICO AG | Recruiting --> Completed | N=100 --> 10 | Trial completion date: Nov 2027 --> Sep 2025 | Trial primary completion date: Sep 2027 --> Aug 2025
Trial completion • Enrollment change • Trial completion date • Trial primary completion date
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Qinlock (ripretinib)
4ms
Host angiogenic reprogramming by Echinococcus multilocularis protoscoleces protein via PDGFR/PI3K/AKT cascade. (PubMed, Front Microbiol)
Interventions utilizing a range of inhibitors at the in vitro level, including the PDGFR-β inhibitor AG1296, the PI3K inhibitor LY294002, the AKT inhibitor MK2206, and the FAK inhibitor Y15, demonstrated that E. multilocularis protoscoleces protein (EmP) induces angiogenesis through PDGFR/PI3K/AKT/FAK signaling pathway. Our findings provide new perspectives on how E. multilocularis infection triggers pathological angiogenesis in the host liver, and may provide a novel anti-angiogenic therapeutic strategy against E. multilocularis infection.
Journal
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PDGFRB (Platelet Derived Growth Factor Receptor Beta)
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MK-2206 • LY294002 • AG-1296
6ms
Metabolite identification of ripretinib by UPLC-ESI-MS/MS: in silico prediction, in vitro metabolism, and stability assessment. (PubMed, Food Chem Toxicol)
Results highlighted neurotoxicity as a significant concern for RTB and its metabolites, while metabolite 1 exhibited hepatotoxicity and nephrotoxicity. The current study unveils metabolic profiles of RTB and helps to understand the biotransformation products-related toxicity.
Preclinical • Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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Qinlock (ripretinib)
6ms
Selective EV Protein Sorting and Pathway Perturbation in AML Upon Synergistic FLT3 and Hedgehog Pathway Inhibition. (PubMed, J Extracell Vesicles)
These findings were corroborated by comparative proteomics of EVs derived from AML patients and healthy donors. Ribosomal and ErbB signalling pathway proteins may play an important role in microenvironmental modulation by EVs, and Crenolanib treatment potentially acts by interfering with leukaemia niche formation.
Journal
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FLT3 (Fms-related tyrosine kinase 3) • RPS26 (Ribosomal Protein S26) • GAB1 (GRB2 Associated Binding Protein 1) • RPL27A (Ribosomal Protein L27a)
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FLT3 mutation
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crenolanib (ARO-002)
8ms
Design, synthesis and biological evaluation of novel platelet-derived growth factor receptor-α (PDGFR-α) inhibitors based on 4-anilinoquinoline and 4-anilinoquinazoline scaffolds. (PubMed, Eur J Med Chem)
Novel 4-anilinoquinoline and 4-anilinoquinazoline hybrids were designed and synthesized as PDGFR-α inhibitors based on lenvatinib, ripretinib and sorafenib. Furthermore, molecular docking results showed that 6o could stably bind to the ATP site of PDGFR-α rationalizing their potent anti-PDGFR-α activity. Based on the above findings, compound 6o could be considered an effective anti-angiogenesis candidate.
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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sorafenib • Lenvima (lenvatinib) • Qinlock (ripretinib)