Drug Database
NI

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

PubMedCancer cell international2026-07-17

A multi-omics prognostic model integrating disulfidptosis-related molecular features in colorectal cancer.

Zhou Huabin H, Xiang Songrui S, Wu Yongjun Y, Li Shipeng S et al.

Disulfidptosis is a recently described cell-death-related process associated with intracellular disulfide stress and may be relevant to tumor progression and immune regulation. However, its prognostic significance and broader biological relevance in colorectal cancer (CRC) remain unclear. Transcriptomic and clinical data from TCGA and GEO cohorts were systematically integrated to characterize disulfidptosis-related molecular patterns in CRC. A four-gene prognostic signature was developed in a randomly assigned training cohort and evaluated in the corresponding internal test cohort. Public datasets were further used for gene-level expression analysis, and survival associations were assessed in the GSE39582 cohort. Immune infiltration, tumor mutation burden, microsatellite instability, and estimated drug-sensitivity analyses were performed to explore the biological correlates and therapeutic-response-related patterns associated with the model. The four model genes were further examined using single-cell RNA-seq data and public immunohistochemical images from the Human Protein Atlas, and were preliminarily assessed in 11 paired CRC tissues and matched adjacent normal tissues by RT-qPCR and western blotting. A four-gene signature consisting of SPINK1, GPRASP1, TIMP1, and KRT6A stratified patients into prognostically distinct risk groups. The high-risk group showed poorer survival, higher tumor mutation burden, higher TIDE scores, and elevated stromal, immune, and ESTIMATE scores, suggesting a more immune-evasive tumor microenvironment. Enrichment analyses indicated that high-risk tumors were associated with extracellular matrix remodeling and PI3K-Akt-related pathways, whereas low-risk tumors were enriched in metabolic processes. Estimated drug-sensitivity analyses indicated agent-specific differences between the two risk groups. The high-risk group showed lower estimated IC50 values for several cytotoxic and targeted agents, including cisplatin, docetaxel, doxorubicin, etoposide, vinorelbine, nilotinib, and gefitinib, whereas the low-risk group showed lower estimated IC50 values for AKT inhibitor VIII, sorafenib, lapatinib, erlotinib, and BIBW2992. This study identified a four-gene prognostic signature associated with disulfidptosis-related molecular heterogeneity in CRC. The signature was associated with survival differences, distinct tumor microenvironmental features, and divergent biological programs across risk groups, while KRT6A showed the most consistent pattern across public-dataset analyses and tissue-level analyses. These findings support the potential relevance of this subtype-guided signature to prognostic stratification in CRC and identify related genes for further biological investigation and independent validation.

PMID 42464330
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PubMedCancer2026-07-15

Longitudinal analysis of tyrosine kinase inhibitor therapy outcomes and BCR-ABL1 transcript decline velocity in chronic myeloid leukemia: A 16-year real-world study.

Mustafa Ali Moaath K MK, Zabor Emily C EC, Abdurakhmanov Kamilla K, Zureigat Hadil H et al.

No large, randomized trials have compared three or more tyrosine kinase inhibitors (TKIs) in a single chronic myeloid leukemia (CML) cohort. Most studies are two-arm comparisons versus imatinib, or rely on indirect methods. A retrospective cohort study was conducted of 349 patients with chronic- and accelerated-phase CML treated between 2007 and 2023 at Cleveland Clinic centers in Northeast Ohio. Overall survival (OS), event-free survival (EFS), 12-month therapeutic milestone achievement, and BCR-ABL1 transcript decline velocity across first-, second-, and third-line TKIs were compared. Baseline demographics, comorbidities, cytogenetics, and hematologic parameters were collected. Multivariable regression and time-dependent Cox models were adjusted for confounders and varying therapy initiation times. First-line TKIs included imatinib (53%), dasatinib (30%), nilotinib (15%), and bosutinib (2%). Median age ranged from 52 to 60 years, most patients were White, and high-risk cytogenetics were rare. Five-year OS ranged from 78% for dasatinib to 90% for nilotinib, with nilotinib associated with improved OS (hazard ratio [HR], 0.43) and EFS (HR, 0.48) and the fastest BCR-ABL1 decline (β = -8.3%) versus imatinib. In second- (n = 181) and third-line therapy (n = 91), no significant differences in outcomes were observed. Across all lines, time-dependent modeling showed improved EFS only for nilotinib (HR, 0.61). Unadjusted OS was higher in patients starting treatment in 2007-2010 versus later periods but differences disappeared after adjustment. Real-world data indicate that imatinib achieves comparable response rates to newer TKIs, with durable survival. Nilotinib consistently shows faster BCR-ABL1 decline and improved EFS overall, consistent with prior network meta-analyses. Lack of improvement in OS over time suggests the need to investigate factors influencing long-term outcomes in CML.

PMID 42455913
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PubMedInternational journal of hematology2026-07-15

Therapeutic drug monitoring guides personalized ponatinib dose reduction for chronic myeloid leukemia.

Abumiya Maiko M, Takahashi Naoto N

The arterial occlusive events (AOEs) associated with ponatinib are dose-dependent. Although the phase II OPTIC trial showed that reducing the dose to 15 mg daily (QD) after achieving a response provided an optimal benefit-risk profile, this has not been adequately verified using plasma drug concentrations data. A 66-year-old woman with chronic-phase chronic myeloid leukemia (CP-CML) was switched to ponatinib because of nilotinib-induced grade 3 thrombocytopenia. For safety, ponatinib was initiated at 15 mg every other day (Q2D) and gradually increased to 45 mg QD. A major molecular response (MMR) was achieved 30.6 months after starting ponatinib. The dose was then reduced to 15 mg QD to mitigate cardiovascular risks, and the MMR was successfully sustained for approximately 2 years (21.2 months) at this reduced dose. The total overall observation period was more than 5 years (65.2 months). A strong correlation was observed between the ponatinib dose and plasma trough concentrations (R2 = 0.8132). Although 30 mg QD was required to reach the target concentration for suppressing resistant clones (23 ng/mL), MMR was sustained at 15 mg QD, with a trough level of 14.1 ng/mL. Once the tumor burden is significantly reduced, lower concentrations of ponatinib may be sufficient to maintain the molecular response.

PMID 42449088
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PubMedJournal of clinical oncology : official journal of the American Society of Clinical Oncology2026-07-15

Development and External Validation of a Transcriptome-Based Multivariable Prediction Model for Treatment-Free Remission in Chronic Myeloid Leukemia.

Alcazer Vincent V, Dulucq Stéphanie S, Mosnier Isabelle I, Chabane Kaddour K et al.

Treatment-free remission (TFR) is a major therapeutic objective in chronic myeloid leukemia (CML). However, nearly 50% of patients relapse after tyrosine kinase inhibitor (TKI) discontinuation, and no robust predictive biomarker is currently available. We profiled peripheral blood cell transcriptomes at imatinib (IMA) discontinuation in patients from the multicenter STIM2 trial (n = 96) to develop a transcriptome-based model predicting TFR by 2 years. A DESEQ2-based machine learning approach was compared with classical machine learning algorithms. The signature was then externally validated in an independent real-world cohort of patients attempting IMA or nilotinib cessation (n = 70). The biologic processes associated with the signature were further explored. We identified a 50-gene signature discriminating patients with sustained 2-year TFR from those experiencing molecular relapse (area under the receiver operating characteristic curve [AUROC], 0.83 [95% CI, 0.73 to 0.93] and 0.75 [95% CI, 0.55 to 1.00] in the training and internal validation cohorts, respectively). The discriminative performance was confirmed in the external test cohort, both as a binary predictor of 2-year TFR (AUROC, 0.71 [95% CI, 0.58 to 0.83] overall; 0.77 [95% CI, 0.61 to 0.92] in IMA-treated patients) and as a time-to-event predictor (log-rank P = .0042). The high TFR-signature group showed a higher proportion of myeloid immune cells and natural killer T cells, with an enrichment in Hedgehog signaling, whereas the low TFR-signature group demonstrated a higher proportion of lymphoid cells with an enrichment in mTOR signaling and a trend for oxidative phosphorylation activation. T-cell receptor and immunoglobulin heavy-chain repertoire analyses showed significantly greater polyclonality in the high TFR-signature group. These findings demonstrate that transcriptomic profiling at TKI discontinuation can predict TFR outcomes in patients with CML and provide biologic insights into the mechanisms underlying sustained TFR.

PMID 42454992
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PubMedClinicoEconomics and outcomes research : CEOR2026-07-14

Financial Impact of Treatment Choice in Chronic Myeloid Leukemia: A Comparison of Later Generation TKIs versus Imatinib from Patient and Payer Perspectives.

Vaughn Jennifer E JE, Zawadzki Nadine N, Zhang Shurui S, Pinkston Paul P et al.

To evaluate the financial impact of treating CML with later-generation TKIs relative to imatinib on US patients and payers. The financial impact of later-generation TKIs (asciminib, bosutinib, dasatinib, nilotinib, and ponatinib) and imatinib was measured from patients' and payers' perspectives before (2024) and after the implementation of Inflation Reduction Act (2025), respectively. Changes in patient's annual out-of-pocket (OOP) costs of CML therapies by payer type (Medicare, Medicaid, and commercial) were used to indicate the financial impact on patients. Per patient per year (PPPY) treatment costs were measured as the weighted average cost across indicated adult CML populations derived using FDA label-based dosing and wholesale acquisition costs. Annual OOP limits by payer types were determined through literature review. The financial impact from payers' perspective was estimated as difference in monthly premium based on per member per month (PMPM) budget impact. In 2025, the estimated PPPY cost for imatinib ($22,430 across generic and branded products, weighted by market share) and for later-generation TKIs (all >$100,000 except for dasatinib and nilotinib, which were $72,693 and $89,138, respectively) exceeded annual OOP limits under Medicare Part D, the average commercial plan, and the commercial plan with highest possible annual OOP limit, resulting in no change in patient financial impact when switching from imatinib to a later-generation TKI. An all-payer blended US health plan with 1 million members could expect to have 200 patients with Philadelphia chromosome-positive CML eligible for TKI therapy. Switching from imatinib to a later-generation TKI would increase the PMPM budget impact between $0.76 and $4.23. Both imatinib and later-generation TKI costs were projected to exceed annual OOP caps in 2025, likely resulting in minimal change in insured CML patients' OOP costs. The impact on insurance premiums from the adoption of TKI is likely to be relatively modest.

PMID 42445105
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PubMedInternational review of neurobiology2026-07-14

Crosstalk in Alzheimer's-delirium nexus: Molecular mechanisms and therapeutic repurposing.

Iqbal Saleem S, Shen Bairong B

Alzheimer's disease (AD) and delirium, though distinct in clinical tempo, converge mechanistically at the intersection of neurovascular dysfunction, glial activation, and metabolic collapse. This chapter explores the integrative framework of neurovascular-glia crosstalk, emphasizing how endothelial injury, astrocytic reactivity, and microglial hyperactivation collectively undermine brain energy metabolism. We highlight evidence that blood-brain barrierc (BBB) breakdown, mitochondrial insufficiency, and oxidative stress establish a "metabolic vulnerability state" predisposing the AD brain to delirium. Single-cell and transcriptomic analyses delineate shared molecular circuits involving MAPK, TP53, APOE, and δ-secretase (LGMN)-the latter regulated by disease-relevant miRNAs such as miR-124 and miR-146a. These networks couple neuroinflammation with impaired energy dynamics, bridging chronic neurodegeneration and acute encephalopathic stress. We further discuss how tyrosine-kinase signaling, serotonergic dysregulation, and glial-vascular miscommunication coalesce into a unified pathophysiological axis. Therapeutic repurposing strategies-ranging from tyrosine kinase inhibitors (nilotinib, imatinib) to metabolic modulators (metformin, pioglitazone)-offer promising cross-disease interventions. Finally, we underscore the transformative role of artificial intelligence (AI) and large language models (LLMs) in accelerating drug repurposing through integrative omics and pathway-based reasoning. Together, these advances redefine the AD-delirium nexus as a systems-level disorder of energy and communication, opening translational avenues for precision therapeutics that restore neurovascular balance and cognitive resilience.

PMID 42442909
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