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nilotinib (Danziten)

✓ Approved

Azurity Pharmaceuticals, Inc. · ABL1 · Small Molecule

What is nilotinib?

nilotinib is a small molecule developed by Azurity Pharmaceuticals, Inc.. It is approved for therapeutic indications via oral (po).

Drug Profile

Brand NamesDanziten
CompanyAzurity Pharmaceuticals, Inc.
Drug ClassSmall Molecule
Molecular TargetABL1, BCR, KIT, PDGFRA
RouteOral (PO)
StatusApproved
Sources: ClinicalTrials.gov, Azurity Pharmaceuticals, Inc.

Mechanism of Action

Molecular Targets

nilotinib acts on 4 molecular targets:

ABL1ABL proto-oncogene 1, non-receptor tyrosine kinase (c-ABL, bcr/abl)
BCRBCR activator of RhoGEF and GTPase (CML, ALL)
KITKIT proto-oncogene, receptor tyrosine kinase (MASTC, CD117)
PDGFRAplatelet derived growth factor receptor alpha (PDGFR-2, CD140A)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

nilotinib is developed for 1 unique indication across 1 therapeutic area.

Therapeutic AreaConditionPhase
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Chronic myeloid leukaemia✓ Approved

Related Research Articles

PubMedFrontiers in oncology2026-11-13

Role and mechanism of ribosomal protein S3 in tyrosine kinase inhibitor resistance in chronic myeloid leukemia.

Sun Zheng Z, Liu Sijin S, Ning Yuxin Y, Dang Jinwen J et al.

Resistance to tyrosine kinase inhibitors (TKIs) remains a major cause of treatment failure in chronic myeloid leukemia (CML). This study investigated the role of ribosomal protein S3 (RPS3) in TKI resistance and its association with NF-κB signaling. Exploratory RNA sequencing and an RNA-binding protein database were used to prioritize a candidate for subsequent clinical expression and functional analyses. RPS3 expression was examined in an independent clinical cohort and resistant cell models. RPS3 knockdown and pharmacological modulation were used to evaluate drug sensitivity, apoptosis, cell cycle distribution, and NF-κB-associated signaling. RPS3 was upregulated in clinical samples from patients with TKI resistance or poor responses and in resistant cell models. Receiver operating characteristic analysis showed that RPS3 expression discriminated between resistant/poor-response patients and good responders in the validation cohort. RPS3 knockdown lowered imatinib and nilotinib IC50 values, with increased apoptosis and G0/G1-phase arrest following TKI exposure. These effects were accompanied by reduced p65 phosphorylation, a lower p65 nuclear/cytoplasmic fluorescence ratio, and decreased MCL-1 and BCL-XL mRNA levels. TNF-α treatment increased TKI tolerance and reduced TKI-induced apoptosis in RPS3-knockdown cells, whereas co-treatment with BAY-11-7082 counteracted these effects. BAY-11-7082 also showed synergistic inhibitory effects with TKIs in resistant cells under the highest single agent model. These findings identify RPS3 as a contributor to TKI resistance in CML cell models and connect its functional effects to NF-κB-associated anti-apoptotic signaling. By integrating clinical expression data with functional and pharmacological analyses, this study supports further investigation of RPS3 as a candidate target for sensitizing resistant CML cells to TKIs.

PMID 43021846
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PubMedJournal of clinical apheresis2026-11-12

Thrombocytapheresis for Extreme Thrombocytosis: A 20-Year Single-Center Experience With Acute and Chronic Use in Myeloproliferative Neoplasms.

Weli Homayemem K HK, Gorham James D JD, Balogun Rasheed A RA

Thrombocytapheresis is an established adjunctive therapy for rapid platelet cytoreduction in patients with extreme thrombocytosis, particularly in myeloproliferative neoplasms (MPNs), where it is typically used alongside cytoreductive pharmacotherapy. Real-world longitudinal data describing utilization, effectiveness, and safety remain limited. The aim of this study was to evaluate or characterize the indications, utilization patterns, hematologic responses, and safety outcomes of thrombocytapheresis over a 20-year period at a single institution and illustrate key clinical applications through two illustrative cases. We conducted a retrospective 20-year institutional review of thrombocytapheresis, highlighting two illustrative cases. Cases were identified using EPIC SlicerDicer. Patient demographics, indications, laboratory responses, adverse events, access complications, electrolyte abnormalities, and short-term outcomes were extracted and analyzed. Pre- and post-platelet count changes were assessed using paired Wilcoxon testing, with exploratory analyses of other blood cells and descriptive evaluation of safety outcomes. After applying exclusion criteria, 9 patients undergoing 38 thrombocytapheresis procedures were analyzed, with one patient accounting for nearly half of all procedures. Most procedures were performed for MPN-related thrombocytosis. In the broader cohort, platelet counts decreased significantly after the first procedure (median 1511 to 666 × 109/L; p = 0.008), corresponding to a mean reduction of 45% with no significant changes in red cell and white cell counts. Adverse events were uncommon and primarily related to citrate-associated hypocalcemia or vascular access rather than to circuit-related complications. We also report two cases describing thrombocytapheresis use in patients with extreme thrombocytosis (> 2.9 million/μL) in distinct myeloid neoplasms. The first patient was diagnosed with polycythemia vera evolving from essential thrombocythemia and demonstrated relative refractoriness and platelet count rebound despite cytoreductive therapy, followed by rapid platelet decline but poor clinical outcome. The second is a case of chronic myeloid leukemia refractory to multiple tyrosine kinase inhibitors (including dasatinib and nilotinib). The patient demonstrated sustained responsiveness to serial thrombocytapheresis over 2 years. In this cohort, thrombocytapheresis was effective for rapid platelet reduction and demonstrated an acceptable safety profile. Clinical outcomes are largely dictated by underlying disease severity and concurrent cytoreductive therapy, rather than procedural response alone. In selected patients, thrombocytapheresis may serve as an effective bridging therapy while awaiting definitive treatment.

PMID 43014826
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PubMedSupportive care in cancer : official journal of the Multinational Association of Supportive Care in Cancer2026-11-12

Development, validation, and pilot-testing of a comprehensive patient-reported EORTC custom questionnaire for monitoring real-world tyrosine kinase toxicity in chronic myeloid leukemia.

Smit Yolba Y, Timmerman Jacolien J, Currie Alexander A, Soltan Hatem H et al.

In chronic myeloid leukemia (CML), treatment choices between tyrosine kinase inhibitors (TKIs) are guided by efficacy and tolerability, yet no instrument adequately captures tolerability from the patient perspective. As a result, TKI choice may not comprehensively take into account the prevalence and severity of what patients' experience. We therefore developed, validated, and pilot-tested a new custom questionnaire to capture real-world patients' experiences, and explored pilot data. A mixed-methods study was conducted, following COREQ and STROBE guidelines. Development started with CML-specific items from existing instruments, followed by semi-structured sessions with purposefully sampled patients, and convenience-sampled professionals, focused on comprehensibility, comprehensiveness, relevance, acceptability, workflow, and graphics. Based on qualitative analyses, items were substituted or added from the EORTC Item library. Psychometric quantitative evaluation assessed filter question validity, redundancy, reliability, measurement error, responsiveness, scale construction, and symptom severity differences in pilot data and meta-analyses. A new EORTC custom questionnaire was developed, covering 50 symptoms. Seven of those symptoms are covered by scales consisting of multiple items, with a Cronbach's alpha ≥ 0.73 for each of those scales. Psychometric properties further showed fair-to-excellent reliability and positive agreement between time points ranging from 45 to 93% in test-retest, while responsiveness was indicated for half of symptoms. Twenty-four generic filter questions are available for computer adaptive testing, an option preferred by 87% of patients. For 23 issues, no existing EORTC item was available; these need to be added later. Pilot data showed that the severity of symptoms, as reported by patients, differed significantly between the three most common TKIs in a third of symptoms. These significant differences ranged from 5 to 30%. The highest difference was for swelling of the eyes or face, which was reported 30% more severe in patients on imatinib, compared to patients on nilotinib, when adjusted for age, gender, years since diagnosis, years on current treatment, and TKI-dosage. We could not adjust for comorbidity or comedication. We developed and validated an EORTC custom questionnaire and workflow to monitor 50 CML patient-reported toxicity symptoms in clinical care. Sexuality problems are excluded from pre-existing instruments, yet pilot data revealed these are prevalent and severe from patients' perspective. One-third of evaluated symptoms varied in severity by type of TKI treatment, independent from age, gender, years since diagnosis, years on current treatment, and TKI-dosage. These findings can tentatively inform shared decision-making, when switching TKIs due to intolerability.

PMID 43018785
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PubMedJournal of biochemical and molecular toxicology2026-11-11

Targeting TLR2 to Overcome Autophagy-Dependent Chemoresistance in Gastric Cancer: Integrating Molecular Docking, Mechanistic Validation, and Therapeutic Evaluation.

Zheng Kuanheng K, Lei Xinjian X, Lin Bin B, Yang Qianhong Q et al.

Chemoresistance remains a major barrier to effective treatment of gastric cancer (GC), particularly in advanced disease, where cytotoxic chemotherapy often yields limited and transient responses. The present study provides integrated computational and experimental evidence that targeting TLR2 using repurposed small-molecule inhibitors can overcome autophagy-dependent chemoresistance in gastric adenocarcinoma cells. A library comprising 1555 FDA-approved drugs was sourced from the DrugBank database, virtual screening was executed, and two compounds, Nilotinib (DB04868) and Lomitapide (DB08827), achieved affinities ≤ -10 kcal/mol. Chemoresistant GC cells were developed, and experimental assessment was performed using cell proliferation assay, apoptosis assay, real-time PCR, and western blot analysis. Nilotinib and Lomitapide significantly reduced the proliferation but increased the apoptosis of chemoresistant GC cells. Additionally, Nilotinib and lomitapide reduced the mRNA and protein levels of autophagy-related factors, LC3B, Beclin-1, and p62/SQSTM1, and activated transcription factors in chemoresistant GC cells. In conclusion, our results suggest that combining TLR2-targeted agents with standard chemotherapy could be a promising strategy to overcome refractory disease in GC.

PMID 43012433
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PubMedJournal of applied genetics2026-11-09

Differential regulation of PRSS57 by tyrosine kinase inhibitors and ATRA in myeloid leukaemia cells: an in vitro study.

Kasap Bahadır B, Gezer Sümbül S, Abacı Neslihan N, Çetin Güven G et al.

PRSS57 encodes neutrophil serine protease 4 (NSP4), a granule-associated protease involved in myeloid cell biology and innate immunity. Clinical evidence has associated high PRSS57 expression with failure to achieve early molecular response in chronic myeloid leukemia (CML) during tyrosine kinase inhibitor (TKI) therapy. However, treatment-associated regulation of PRSS57 remains poorly characterized. K562 cells were treated with imatinib, nilotinib, or dasatinib, and 48-h IC₅₀ values were determined by MTT assay. PRSS57 mRNA expression was assessed by RT-qPCR at 24, 48, and 72 h using the respective 48-h IC₅₀ concentrations. HL60, NB4, and THP-1 cells were treated with 1 µM all-trans retinoic acid (ATRA) and analyzed at the same time points. All three TKIs significantly increased PRSS57 mRNA expression in K562 cells at 24 h, with expression remaining elevated at 48 h and declining toward baseline by 72 h. Dasatinib showed the largest and most sustained increase. In acute myeloid leukemia (AML) models, ATRA produced limited and variable changes in HL60 and NB4 cells, whereas THP-1 cells showed robust and sustained PRSS57 upregulation from 24 to 72 h. PRSS57 expression is dynamically regulated in a treatment- and cell-type-dependent manner in myeloid leukemia models. These findings support further investigation of PRSS57 at the protein and functional levels.

PMID 43002371
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PubMedJournal of pharmaceutical and biomedical analysis2026-11-06

Capillary dried plasma microsampling: A novel approach for therapeutic drug monitoring of tyrosine kinase inhibitors in chronic myeloid leukemia patients by UPLC-MS/MS.

Silva Augustin Carla Miriane da CMD, Guedes Perlin Valquiria V, de Moraes Machado Maitê M, Camargo Milczarski Vitória Luiza de VL et al.

The individualized pharmacological treatment of Chronic Myeloid Leukemia (CML) with tyrosine kinase inhibitors (TKIs) has shown greater efficacy and safety when guided by plasma concentration monitoring. However, in Brazil, this strategy is not yet implemented for TKIs. In this context, alternative sampling methods, such as dried plasma spots (DPS), may improve the feasibility and accessibility of therapeutic drug monitoring. This study aimed to develop and validate a bioanalytical method for quantifying dasatinib, nilotinib, imatinib, and their metabolites in DPS using the HealthID® plasma separation device and UHPLC-MS/MS. The analytes were extracted from three 6 mm discs with 0.1% BSA and ethyl acetate:n-propanol (95:5, v/v). The Analysis was performed via UHPLC-MS/MS with positive electrospray ionization, using a C18 column and gradient elution with 0.1% formic acid in water and acetonitrile. The method was validated and applied to DPS samples from 51 CML patients. The analytical run time was 6 min, with linearity (r > 0.99), CVs between 3.33%-14.99%, and accuracy from 95.3% to 103.9%. Extraction yields ranged from 49% to 68%. The analytes were stable at room temperature in DPS for one month. Mean TKI concentrations in DPS represented 98.1-100.9% of plasma levels. These results support the potential of DPS as a viable strategy for TKI monitoring, offering logistical advantages and sample stability without refrigeration.

PMID 42994209
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