Tasigna (nilotinib) / Novartis, Inhibikase - LARVOL DELTA

Phase 2 evaluation of the nilotinib-paclitaxel combination in patients with rare solid tumors: Rapid analysis and response evaluation of combination anti-neoplastic agents in rare tumors trial 1 (RARE CANCER 1). (ASCO 2025) - P2 | "Clinical Trial Registration Number: NCT04449549 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Clinical • P2 data • Oncology • Solid Tumor

Primary endpoint results of the phase 3b ASC4START trial of asciminib (ASC) vs nilotinib (NIL) in newly diagnosed chronic phase chronic myeloid leukemia (CML-CP): Time to treatment discontinuation due to adverse events (TTDAE). (ASCO 2025) - P3 | "Clinical Trial Registration Number: NCT05456191 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Adverse events • P3 data • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology

Rapid Analysis and Response Evaluation of Combination Anti-Neoplastic Agents in Rare Tumors (RARE CANCER) Trial: RARE 1 Nilotinib and Paclitaxel (clinicaltrials.gov) - P2 | N=82 | Recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Apr 2025 ➔ Apr 2026 | Trial primary completion date: Apr 2025 ➔ Apr 2026

Trial completion date • Trial primary completion date • Adrenal Cortex Carcinoma • Breast Cancer • Cholangiocarcinoma • Endometrial Cancer • Gallbladder Cancer • Gastrointestinal Cancer • Head and Neck Cancer • Lung Cancer • Oncology • Ovarian Cancer • Testicular Cancer • NF1

Hair Re-Pigmentation After Nilotinib. (PubMed, Turk J Haematol) - No abstract available

Journal

Quantitative Analysis of Bimiralisib in Dried Blood Spots of Mouse Blood Using a Validated LC-MS/MS Method: An Application to Pharmacokinetic Study. (PubMed, Biomed Chromatogr) - "Under optimized conditions, the retention times for bimiralisib and the internal standard (IS, Nilotinib) were approximately 1.14 and 1.27 min, respectively, with a total run time of 2.00 min per injection. All validation parameters met the required acceptance criteria, and hematocrit levels had no impact on bimiralisib concentrations in DBS. The validated method was utilized to determine intravenous and oral pharmacokinetic parameters by quantifying bimiralisib in mouse blood, with results correlated to pharmacokinetic data from mice plasma."

Journal • PK/PD data • Preclinical • Oncology

Nilotinib Versus Imatinib in Philadelphia Chromosome-Positive Chronic Myeloid Leukemia (Ph+ CML): A Systematic Review and Meta-Analysis of Randomized Controlled Trials (RCTs) (Cureus) - "A total of four studies with 1,045 patients were included in the study. Patients who received nilotinib had higher odds of achieving MMR at 12 months (OR 2.83; 2.12-3.79; p < 0.0001) and maintaining the MMR, i.e., durable MMR at 24 months (OR 2.72; 2.02-3.68; p < 0.0001) compared to patients on imatinib. Similar results were noted for CCyR at 12 months (OR 1.30; 0.60-2.81; p = 0.5). Overall, nilotinib was found to be superior due to better clinical outcomes, but adverse effects, like hyperbilirubinemia and rash, were significantly higher in nilotinib as compared with imatinib."

Retrospective data • Chronic Myeloid Leukemia

CML0912: Front-line Nilotinib Treatment of BCR-ABL+ Chronic Myeloid Leukaemia in Chronic Phase (clinicaltrials.gov) - P=N/A | N=129 | Completed | Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto | Active, not recruiting ➔ Completed

Trial completion • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1 • BCR

ECOG-ACRIN EA9171: Pembrolizumab and Dasatinib, Imatinib Mesylate, or Nilotinib in Treating Patients With Chronic Myeloid Leukemia and Persistently Detectable Minimal Residual Disease (clinicaltrials.gov) - P2 | N=40 | Active, not recruiting | Sponsor: ECOG-ACRIN Cancer Research Group | Recruiting ➔ Active, not recruiting

Enrollment closed • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1 • BCR • CD4

Development and application of a mechanistic pharmacokinetic pharmacodynamic (PKPD) model to predict anti-chronic myeloid leukemia (CML) effects of tyrosine kinase inhibitors (AACR 2025) - "Although tyrosine kinase inhibitors (TKIs) for BCR::ABL1 like imatinib, dasatinib, nilotinib, bosutinib, asciminib and ponatinib are available for CML treatment, novel treatments are needed for CML that is resistant or patients who are intolerant to available therapies. This generalizable PKPD model, using available PK and in vitro potency data, was developed and accurately predicted outcomes of five approved BCR::ABL1 TKIs in patients with newly-diagnosed CML, suggesting the mechanistic PKPD model may be used to predict efficacy for novel TKIs."

PK/PD data • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1 • CRKL • STAT5 • STAT5AWqe

Identification of the clinical value and biological effects of TTN mutation in liver cancer. (PubMed, Mol Med Rep) - "The OncoPredict algorithm and CCK‑8 assays revealed that TTN mutations are associated with altered drug sensitivity, particularly to GSK1904529A, nilotinib, 5‑fluorouracil (5‑FU) and sapitinib. The findings indicated that TTN mutations increase protein stability and lower intracellular ferrous ion levels, thereby suppressing ferroptosis and contributing to resistance to 5‑FU in hepatoma cells. These results suggest that TTN mutations are associated with poor prognosis in liver cancer and could serve as a predictive biomarker for liver cancer progression, prognosis and drug resistance."

Journal • Gastrointestinal Cancer • Hepatocellular Cancer • Hepatology • Liver Cancer • Oncology • Solid Tumor • Transplantation • TTN

DASATINIB AS TARGETED THERAPY FOR POST-TRANSPLANT EXTRAMEDULLARY RELAPSE. AN OBSERVATIONAL STUDY (EBMT 2025) - "Among the 159, 70% were receiving, or had received, imatinib when EML presented...Dasatinib interruption resulted in EM relapses in 3; with nilotinib or radotinib, remission was regained... Dasatinib, alone or added to routine treatments, has produced remissions of leukemic involvement of CNS and various organs, some of unusually long duration. This is a significant advance in eradicating EML, where there is no reliably effective therapy. EML remissions were reported in most of 159 cases; including 34 of 35 with EML post-transplant."

Clinical • Observational data • Post-transplantation • CNS Lymphoma • Hematological Disorders • Hematological Malignancies • Leukemia • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • Primary Central Nervous System Lymphoma • T Acute Lymphoblastic Leukemia • Transplantation • ABL1 • LCK

DASATINIB AS TARGETED THERAPY FOR POST-TRANSPLANT EXTRAMEDULLARY RELAPSE. AN OBSERVATIONAL STUDY (EBMT 2025) - "Among the 159, 70% were receiving, or had received, imatinib when EML presented...Dasatinib interruption resulted in EM relapses in 3; with nilotinib or radotinib, remission was regained... Dasatinib, alone or added to routine treatments, has produced remissions of leukemic involvement of CNS and various organs, some of unusually long duration. This is a significant advance in eradicating EML, where there is no reliably effective therapy. EML remissions were reported in most of 159 cases; including 34 of 35 with EML post-transplant."

Clinical • Observational data • Post-transplantation • CNS Lymphoma • Hematological Disorders • Hematological Malignancies • Leukemia • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • Primary Central Nervous System Lymphoma • T Acute Lymphoblastic Leukemia • Transplantation • ABL1 • LCK

Successful desensitization to nilotinib after imatinib-induced Sweet's syndrome and nilotinib hypersensitivity reaction in a patient with chronic myeloid leukemia (EAACI 2025) - No abstract available

Clinical • Allergy • Immunology

Testing the Use of Nilotinib and Paclitaxel as a Treatment for Patients With Prior Taxane Treatment, A ComboMATCH Treatment Trial (clinicaltrials.gov) - P2 | N=40 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Jul 2025 ➔ Dec 2025 | Trial primary completion date: Jul 2025 ➔ Dec 2025

Trial completion date • Trial primary completion date • Oncology • Solid Tumor • PDGFRA

UGT1A1 POLYMORPHISM AND TOXICITY RELATED TO ALLOGENEIC STEM CELL TRANSPLANTATION WITH BUSULFAN BASED REGIMEN (EBMT 2025) - "This polymorphism has been associated with an increased risk of toxicity from irinotecan, belinostat, pazopanib and nilotinib.[1][2] However, guidelines for integrating UGT1A1 results into therapeutic decision-making are mostly absent. The UGT1A1*28 polymorphism is associated with an increased risk of liver toxicity in patients undergoing allogeneic stem cell transplantation with a busulfan-based conditioning regimen. Supporting this finding, the number of busulfan days did not impact liver toxicity (p=0.161). Further expansion of the study cohort is necessary to demonstrate the true impact of this genetic variant on transplant-related mortality and its potential effect on therapeutic management."

Bone Marrow Transplantation • Graft versus Host Disease • Hepatology • Immunology • Oncology • Transplantation • UGT1A1

Target identification and exploring drug repurposing strategies for Merkel cell carcinoma (Sarcoma-RC 2025) - "Among the 15 FDA-approved drugs docked against KIT, Ponatinib and Nilotinib—both primarily used for the treatment of chronic myeloid leukemia—achieved the highest docking scores, -13.9 and -11.1, respectively. Legal entity responsible for the study The authors. Funding Has not received any funding."

Chronic Myeloid Leukemia • Endocrine Cancer • Genetic Disorders • Hematological Malignancies • Leukemia • Merkel Cell Carcinoma • Neuroendocrine Tumor • Non-melanoma Skin Cancer • Oncology • Skin Cancer • Solid Tumor • BCL2L1 • CTNNB1 • HSPA8 • MUC1

Cardiac arrhythmias of BCR-ABL inhibitors with or without triazole antifungal agents: A real-world pharmacovigilance study based on the food and drug administration adverse event reporting system database. (PubMed, SAGE Open Med) - "The reporting odds ratios and their 95% confidence intervals for BCR-ABL inhibitor monotherapy, asciminib, nilotinib, and ponatinib were 1.31 (1.27-1.36), 2.11 (1.45-3.06), 2.66 (2.53-2.80), and 1.18 (1.05-1.33), respectively. Dasatinib plus triazole antifungal agents (reporting odds ratio: 2.98, 95% CI: 1.93-4.60) and ponatinib plus triazole antifungal agents (reporting odds ratio: 1.53, 95% CI: 1.08-2.16) were associated with a higher disproportionality of cardiac arrhythmias than BCR-ABL inhibitor monotherapy. The median time-to-onset was longer with monotherapy than with BCR-ABL inhibitors plus triazole antifungal agents (2.63 vs. 0.34 months, p < 0.001), both indicating an early failure type. BCR-ABL inhibitors plus triazole antifungal agents increase the risk of cardiac arrhythmia, particularly in the early stages of treatment, with the risk decreasing over time."

Adverse events • Journal • Real-world evidence • Cardiovascular • Chronic Myeloid Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • ABL1 • BCR

ALLOGENEIC TRANSPLANTATION IN A PATIENT WITH REFRACTORY CHRONIC MYELOID LEUKEMIA (EBMT 2025) - "The F359V mutation in the ABL1 gene was identified by Next-Generation Sequencing in November 2022, a mutation affecting the catalytic domain and conferring moderate resistance to Imatinib and Nilotinib.Second-line treatment with Ponatinib was initiated in chronic phase in November 2022 but did not result in molecular response.In April 2023, the patient was diagnosed with lymphoid blastic crisis. Induction therapy was started using the LAL Ph 2008 protocol from the PETHEMA group, with Ponatinib substituted by Dasatinib due to bradycardia associated with Ponatinib...During the second consolidation cycle, Bosutinib was introduced with good tolerance... An allo-HSCT from an HLA-matched sibling was performed in August 2023, conditioning with TBF regimen (Thiothepa, Busulfan, and Fludarabine) and Cyclosporine and Methotrexate in a short-course regimen as prophylaxis for graft-versus-host disease (GVHD)...Donor lymphocyte infusion (DLI) was initiated.After two DLIs, the patient..."

Clinical • Bone Marrow Transplantation • Cardiovascular • Chronic Myeloid Leukemia • Endocrine Disorders • Fatigue • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Leukemia • Musculoskeletal Pain • Oncology • Pain • Transplantation • ABL1

Construction and validation of a reliable disulfidptosis-related lncRNAs signature of the subtype, prognostic, and immune landscape in bladder cancer. (PubMed, Discov Oncol) - "Based on this study, it would be advisable to identify the key DRLs with potential prognostic value in BLCA to enhance the evaluation of clinical outcomes in this context."

Journal • Tumor mutational burden • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • CD4 • CD8 • TMB

Machine learning-based prognostic modeling of seven signatures associated with lysosomes for predicting prognosis and immune status in clear cell renal cell carcinoma (AUA 2025) - "The prognostic model developed in this study demonstrates a high efficacy in accurately predicting the overall survival (OS) of ccRCC patients, thereby offering a novel perspective for the advancement of ccRCC treatment."

Machine learning • Clear Cell Renal Cell Carcinoma • Genito-urinary Cancer • Oncology • Solid Tumor • CD4

Identification of Endoplasmic Reticulum Stress-related Genes for Predicting Prognosis, Immunotherapy Response, and Drug Sensitivity in Thyroid Cancer. (PubMed, J Immunother) - "Immunotherapy, as well as Palbociclib and Perifosine, were predicted to be more effective for low-risk patients. Conversely, high-risk patients were more likely to benefit from Axitinib, Imatinib, Nilotinib, and Temsirolimus. This study identified 5 signature genes as potential biomarkers and therapeutic targets for THCA. These findings provide novel insights into the prognosis and targeted therapy of THCA, offering a foundation for furture clinical applications."

IO biomarker • Journal • Endocrine Cancer • Oncology • Solid Tumor • Thyroid Gland Carcinoma • ANK1 • APOE • ERP27 • FPR2 • NOS1

Reinventing PARP1 inhibition: harnessing virtual screening and molecular dynamics simulations to identify repurposed drugs for anticancer therapeutics. (PubMed, J Biomol Struct Dyn) - "By integrating multiple computational approaches, we provide a rational framework for the selection of Nilotinib, demonstrating its PARP1 binding features and potential for therapeutic development after further experimentation. This study highlights the power of computational methods in accelerating drug repurposing efforts, offering an efficient strategy for identifying novel therapeutic options for PARP1-associated diseases."

Journal • Oncology

Treatment-free Remission After Achieving Sustained MR4.5 on Nilotinib (ENESTop) (clinicaltrials.gov) - P2 | N=163 | Completed | Sponsor: Novartis Pharmaceuticals | Active, not recruiting ➔ Completed

Trial completion • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1 • BCR

PEARL Study: PotEntial of Asciminib in the eaRly Treatment of CML (clinicaltrials.gov) - P2 | N=160 | Not yet recruiting | Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto | Trial completion date: Sep 2031 ➔ Jun 2032 | Initiation date: Sep 2024 ➔ Jun 2025 | Trial primary completion date: Sep 2028 ➔ Jun 2029

Trial completion date • Trial initiation date • Trial primary completion date • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology

Tyrosine kinase inhibitors modulate the expression of peroxiredoxins 1 and 2 in chronic myeloid leukemia cells. (PubMed, Leuk Res Rep) - "We found that TKIs, such as imatinib, nilotinib, and dasatinib, increased the gene expression of PRDX2 in K562 cells; however, only dasatinib increased the cytoplasmic protein expression of PRDX2. This discrepancy was linked to post-translational regulation through SUMOylation in cooperation with dasatinib. Our results suggest that the antioxidants PRDX1 and PRDX2 could serve as potential targets for TKIs in the treatment of CML."

Journal • Chronic Myeloid Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • ABL1 • PRDX1 • PRDX2