nilotinib / Generic mfg. - LARVOL DELTA

CMLXI: Frontline Asciminib Combination in Chronic Phase CML (clinicaltrials.gov) - P2 | N=125 | Active, not recruiting | Sponsor: University of Jena | Not yet recruiting ➔ Active, not recruiting

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

ASIM-POST Ph+: Asciminib & Standard-of-Care Integration in Maintenance Therapy for POST Allogeneic Stem Cell Transplant (Allo-HSCT) of Patient With Ph+ B-ALL or Blastic Transformed CML (clinicaltrials.gov) - P2 | N=45 | Not yet recruiting | Sponsor: The University of Hong Kong

New P2 trial • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology • Transplantation

Tyrosine kinase inhibitors, chronic myeloid leukemia, and pregnancy: pharmacotherapeutic challenges and recommendations. (PubMed, Expert Opin Pharmacother) - "All TKIs are not recommended during the first trimester due to their risk of teratogenesis, but imatinib and nilotinib may be cautiously used from Weeks 16-18 onward. Non-TKI therapies, such as hydroxyurea and interferon-α, are considered safe throughout pregnancy. Data on ponatinib and asciminib remain insufficient to allow the safe use of these agents during pregnancy. Future research should aim to improve treatment-free remission rates through novel agents and combination strategies to allow a higher proportion of younger patients to discontinue therapy. Clinicians should always counsel women on pregnancy risks during therapy while reassuring male patients of TKI safety when fathering children."

Journal • Review • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1

Computational Repurposing of Nilotinib and Radotinib as SIRT2 Inhibitors for Neurodegenerative Diseases. (PubMed, J Mol Neurosci) - No abstract available

Journal • CNS Disorders

From Molecular Silence to Lymphoid Blast Phase: Diagnostic and Therapeutic Challenges in a Young Female Patient With Chronic Myeloid Leukemia. (PubMed, Cureus) - "Notably, during treatment with tyrosine kinase inhibitors (TKIs), she became intolerant to first- and second-generation TKIs, including the branded and generic imatinib, nilotinib, and dasatinib, followed by progression into lymphoid blast phase. This case highlights the diagnostic challenges and therapeutic complexity of managing CML in the setting of multi-TKI intolerance. Importantly, it underscores the persistent molecular silence despite repeated RT-qPCR testing and the successful introduction of asciminib as a novel therapeutic alternative."

Journal • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Myeloproliferative Neoplasm • Oncology • ABL1 • BCR

Treatment-free remission after two nilotinib consolidation durations in chronic myeloid leukemia treated with imatinib: Phase 3 ENESTPath results. (PubMed, Leukemia) - "In the TFR phase, MR4.0 rates at 12 months (Arm 1: 31.9%, Arm 2: 37.5%; p = 0.383) and 24 months (Arm 1: 29.4%, Arm 2: 30.8%) revealed no differences in TFR success between 2 and 3 years of nilotinib. Irrespective of the consolidation duration, switching to nilotinib 300 mg BID provided the opportunity to achieve TFR if patients were unable to reach stable DMR with first-line imatinib."

Journal • P3 data • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1

Efficacy of Bidirectional Paclitaxel plus Capecitabine or Nilotinib for Peritoneal Carcinomatosis: A Single Institution Analysis of Two Phase II Clinical Trials. (PubMed, Ann Surg Oncol) - "Bidirectional paclitaxel-based chemotherapy plus capecitabine may delay progression of gastric adenocarcinoma with peritoneal-only metastasis. Bidirectional paclitaxel-based chemotherapy plus nilotinib was associated with mostly stable peritoneal disease in this small, heterogenous cohort. Bidirectional paclitaxel combinations are feasible and may have a role in therapy for disease stabilization in individuals with peritoneal carcinomatosis."

Journal • P2 data • Appendix Cancer • Colorectal Cancer • Gastric Adenocarcinoma • Gastric Cancer • Oncology • Peritoneal Cancer • Small Intestinal Carcinoma • Solid Tumor

A novel case of nilotinib-induced porphyria cutanea tarda (AAD 2026) - "He began treatment with hydroxychloroquine 200mg twice weekly at last follow-up. Though imatinib– a similar tyrosine kinase inhibitor– has previously been linked to porphyria cutanea tarda as well as pseudoporphyria, to our knowledge this is the first case of nilotinib-induced PCT in the literature."

Clinical • Chronic Myeloid Leukemia • Genetic Disorders • Hematological Disorders • Hematological Malignancies • Hepatology • Human Immunodeficiency Virus • Infectious Disease • Leukemia • Metabolic Disorders • Pruritus • Rare Diseases

ASCIMINIB (ASC) SHOWS SUPERIOR TOLERABILITY VS NILOTINIB (NIL) IN NEWLY DIAGNOSED CHRONIC MYELOID LEUKEMIA IN CHRONIC PHASE (CML-CP): PRIMARY ENDPOINT RESULTS OF THE PHASE (PH) 3B ASC4START TRIAL (EHA 2025) - P3 | "ASC4START met the primary endpoint in this interim analysis with ASC showing superior tolerability vs NIL based on TTDAE. The study is ongoing, with analyses planned for longer-term tolerability, quality of life, and efficacy. Combined with ASC4FIRST data, results suggest ASC may be a preferred therapy for newly diagnosed CML-CP, enabling more pts to reach tx goals without requiring a tx switch"

Cardiovascular • Chronic Myeloid Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Musculoskeletal Pain • Oncology • Pain • Pancreatitis • Thrombocytopenia

An Expedition of FDA-Approved Anticancer Drugs in 2024: Synthetic Routes, Mechanisms of Action, and Clinical Indications. (PubMed, Curr Top Med Chem) - "Among the small molecules, kinase inhibitors such as Ribociclib, Imatinib Mesylate, and Nilotinib Hydrochloride Dihydrate modulate critical signaling cascades, while antimetabolites like Fluorouracil and Pemetrexed Disodium disrupt nucleotide biosynthesis. Other classes include DNA-damaging agents (Mitomycin, Gemcitabine Hydrochloride), immune modulators (Pomalidomide, Lenalidomide), and hormonal antagonists (Abiraterone Acetate, Erleada). The macromolecular therapeutics primarily consist of monoclonal antibody-based agents, such as Keytruda, Tecentriq, and Imfinzi, which enhance immune checkpoint inhibition, and antibody-drug conjugates like Enhertu and Elahere, which deliver cytotoxic payloads with high specificity. This review systematically examines the chemical synthesis, mechanisms of action, and therapeutic indications of these FDAapproved agents, emphasizing their role in improving patient outcomes. By analyzing their structural frameworks and biological targets,..."

FDA event • Journal • Oncology

Novel inhibitors of BCRP and P-gp found among drugs used in the treatment of cancer (AACR 2026) - "Of the investigated compounds, cabozantinib (IC50 of 0.65 µM), midostaurin (0.69 µM), and entrectinib (5.8 µM) showed the strongest inhibition of BCRP. Nilotinib (1.0 µM), osimertinib (2.0 µM), and abemaciclib (2.4 µM) showed the strongest inhibition of the P-gp. The highest I2/IC50 ratios for BCRP were observed for mitotane (6190), cabozantinib (1730), and abiraterone (831). For P-gp, the highest I2/IC50 ratios were observed for nilotinib (2880), pazopanib (1580), and mitotane (1480)...The highest I1/IC50 ratios for BCRP were observed for doxorubicin (8.2), etoposide (2.8), and fosaprepitant (0.84). For P-gp, the highest I1/IC50 ratios were observed for amscarine (1.6), vinorelbine (0.55), and fosaprepitant (0.50)...Mechanistic static model for BCRP inhibitors suggested that cabozantinib, midostaurin, and apalutamide could almost fully inhibit intestinal BCRP, increasing the exposure to concomitantly administered rosuvastatin by 94%, 89%,..."

Breast Cancer • Oncology • Solid Tumor

Targeting regulation of F-actin in cutaneous T-cell lymphoma: Synergistic inhibition of p38β with p38γ inhibitors (AACR 2026) - "To extend these findings in vivo, ongoing studies using Hut78 xenograft mice are evaluating tumor reduction, apoptosis, survival benefit, and systemic safety following triple-drug combination (Nilotinib + F7 + CSH71). Collectively, these results support dual p38β/p38γ inhibition as a promising therapeutic approach for CTCL and other hematologic malignancies driven by cytoskeletal and stress-kinase dysregulation, while providing mechanistic insight into how targeted therapies remodel the F-actin network in vivo."

Cutaneous T-cell Lymphoma • Hematological Malignancies • Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma

Synergistic effects of antineoplastic drugs and gap junction enhancer in heterogenous colorectal cancer (AACR 2026) - "Doxorubicin, erlotinib, nilotinib, and cediranib were tested across multiple concentrations, alone and in combination with the gap junction enhancer, PQ, and drug interactions were quantified using the ZIP model, analytic tool and visualized using gigamap approach. Similarly, the BCR-ABL targeted therapy nilotinib displayed a biphasic response in combination with PQ, characterized by two synergistic peaks at low and high concentrations separated by an antagonistic valley at intermediate doses. In conclusion, these results highlight both the synergistic and antagonistic properties of PQ with commonly used antineoplastic agents and support the approach of combinational treatment for colorectal cancer."

Colorectal Cancer • Oncology • Solid Tumor • ABL1 • BCR

Imatinib and nilotinib are equally successful in achieving therapy-free remission. (PubMed, Blood Cancer J) - No abstract available

Journal

HEARTBREAKING THERAPY: PONATINIB-ASSOCIATED NONISCHEMIC CARDIOMYOPATHY (ACC 2026) - "Case: A 75-year-old female with chronic-phase chronic myeloid leukemia (CML), previously treated with dasatinib, imatinib, and nilotinib, was started on ponatinib in June 2024. This case demonstrates the risk of anchoring on common causes of heart failure, emphasizing the need to consider drug-induced cardiomyopathy for timely diagnosis and management. Multiple studies (including PACE, OPTIC, PhALLCON) have reported heart failure with ponatinib, but the overall incidence remains low. This case additionally underscores the importance of cardiac surveillance in patients on TKIs, especially in those with prior exposure."

Cardiomyopathy • Cardiovascular • Chronic Kidney Disease • Chronic Myeloid Leukemia • Cognitive Disorders • Congestive Heart Failure • Heart Failure • Hematological Malignancies • Leukemia • Nephrology • Pulmonary Disease • Renal Disease

BRUGADA PATTERN UNMASKED BY TYROSINE KINASE INHIBITOR THERAPY IN A PATIENT WITH A JPH2 MUTATION (ACC 2026) - "Decision-Making: The patient had failed two prior TKIs - imatinib for a treatment-limiting skin rash, and nilotinib for refractory pain, leaving limited oncologic options. FDA review indicates that avapritinib can inhibit cardiac sodium-channel activity (reported IC₅₀ ≈ 0.3 µM), providing a biologically plausible mechanism by which the drug, particularly when combined with high-grade fever, could transiently reduce INa and unmask a Type I Brugada EKG pattern. In our patient the Brugada pattern normalized with defervescence and brief interruption of avapritinib, and continuous monitoring revealed no ventricular arrhythmias. This case highlights TKI-related sodium-channel effect warranting vigilance."

Clinical • Cardiovascular • Genetic Disorders • Sarcoma • Solid Tumor

ALLOGENEIC TRANSPLANTATION OUTCOMES OF PHILADELPHIA-POSITIVE ACUTE LYMPHOBLASTIC LEUKEMIA WITH OR WITHOUT ADDITIONAL CYTOGENETIC ABNORMALITIES AND COMPLEX KARYOTYPE: A STUDY FROM THE ALWP/EBMT (EBMT 2026) - " 514 Ph+ pts that received TKI (Imatinib-62%, Dasatinib-17.7%, Nilotinib-13.3%, other-7%) followed by HSCT while CR1 between 2010-2022 were included. Harboring ACA and CK did not affect outcomes of Ph+ ALL pts receiving a TKI and subsequent HSCT. Notably, achieving MRD-negativity pre HSCT was significantly higher in pts with ACA. These data may indicate that the administration of a TKI upfront, followed by HSCT, may overcome the poor prognosis influence of the ACA, including CK in Ph+ ALL."

Acute Lymphocytic Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Transplantation • IKZF1

OPTIMIZING TRANSPLANT OUTCOMES IN PHILADELPHIA CHROMOSOME–POSITIVE ACUTE LYMPHOBLASTIC LEUKEMIA WITH PRE-TRANSPLANT SECOND- AND THIRD-GENERATION TKIS AND POST-TRANSPLANT PROPHYLACTIC MAINTENANCE: A STUDY FROM THE EBMT ALWP (EBMT 2026) - "Peripheral blood stem cells were used in 84% of cases, with 79% receiving myeloablative conditioning, and 51% receiving total body irradiation.TKI use prior to transplant included imatinib (N=612, 64%), dasatinib (N=210, 22%), nilotinib (N= 96, 10%), and ponatinib (N=40, 4%). In this large, multicenter cohort of patients with Ph+ ALL undergoing allo-HCT in CR1, the use of second- and third-generation TKIs during induction was associated with superior survival outcomes compared to imatinib. Post-transplant TKI prophylaxis significantly improved survival regardless of pre-transplant MRD status. These findings support the incorporation of newer TKIs and prophylactic strategies in the transplant setting to optimize long-term outcomes in Ph+ ALL."

Clinical • Post-transplantation • Pre-transplantation • Acute Lymphocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Transplantation

OLVEREMBATINIB-MEDIATED DEEP REMISSION IMPROVES ALLOGENEIC STEM CELL TRANSPLANTATION OUTCOME IN PATIENTS WITH BLAST CRISIS CHRONIC MYELOID LEUKEMIA: THE FIRST REAL-WORLD PRACTICE (EBMT 2026) - "All patients received TKIs+chemotherapy before transplantation and were stratified by pre-transplant TKIs exposure: 1/2G-TKI cohort (n=43, imatinib, nilotinib, flumatinib or dasatinib) versus olverembatinib (n=26). This real-world analysis provides the first clinical evidence supporting the efficacy and safety of olverembatinib in transplant-eligible BC-CML. Olverembatinib significantly enhances pre-transplant molecular responses, compared to 1/2G-TKIs, associating with improved survival after transplantation. With a manageable safety profile, olverembatinib represents a promising bridging strategy prior to allo-HSCT, potentially redefining frontline management of BC-CML."

Clinical • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Myeloid Leukemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Transplantation • ABL1 • ASXL1 • IKZF1 • RUNX1

THIRD-LINE TREATMENT OF CHRONIC MYELOID LEUKEMIA WITH TKI IN THE POST-BMT SETTING IN PATIENT WITH POSITIVE MRD (EBMT 2026) - "Starts on Dasatinib 100 mg per day, after one year only achieves complete hematologic response (CHR), remaining qPCR BCR-ABL: 72%IS; T315I mutation by PCR: undetected. Switch of therapy with Nilotinib 800 mg per day with no molecular response in 6 months (qPCR BCR-ABL: 44%IS)...Ponatinib was again discontinued due to hematologic toxicity.Starts oh Asciminib by day 124, achieving MR 4.0 by day 195 which is maintained by day 395... This clinical case shows that there still cases of chronic myeloid leukemia resistant to second generation TKI, even with early initiation at diagnosis. Toxicity is often associated with resistance. In our case, starting Ascminib as maintenance treatment post BMT, instead of Ponatinib due to hematologic and cardiologic toxicity, had a major impact on response achieving MR 4.0 sustained with no toxicity associated."

Clinical • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • ABL1 • BCR

ALLOGENEIC TRANSPLANTATION OUTCOMES OF PHILADELPHIA-POSITIVE ACUTE LYMPHOBLASTIC LEUKEMIA WITH OR WITHOUT ADDITIONAL CYTOGENETIC ABNORMALITIES AND COMPLEX KARYOTYPE: A STUDY FROM THE ALWP/EBMT (EBMT 2026) - " 514 Ph+ pts that received TKI (Imatinib-62%, Dasatinib-17.7%, Nilotinib-13.3%, other-7%) followed by HSCT while CR1 between 2010-2022 were included. Harboring ACA and CK did not affect outcomes of Ph+ ALL pts receiving a TKI and subsequent HSCT. Notably, achieving MRD-negativity pre HSCT was significantly higher in pts with ACA. These data may indicate that the administration of a TKI upfront, followed by HSCT, may overcome the poor prognosis influence of the ACA, including CK in Ph+ ALL."

Acute Lymphocytic Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Transplantation • IKZF1

Asciminib (ASC) Demonstrates Favorable Safety and Tolerability Compared with Each Investigator-Selected Tyrosine Kinase Inhibitor (IS TKI) in Newly Diagnosed Chronic Myeloid Leukemia in Chronic Phase (CML-CP) in the Pivotal Phase 3 ASC4FIRST Study (ASH 2024) - "Introduction : About 1/3rd of patients (pts) with newly diagnosed CML discontinue/switch treatments (Tx) regardless of TKI (imatinib [IMA], nilotinib [NIL], dasatinib [DAS], or bosutinib [BOS]). ASC's superior efficacy vs all IS TKIs and more favorable safety/tolerability compared with each IS TKI (IMA, NIL, DAS, and BOS) suggests that ASC may transform the CML Tx paradigm. Key secondary endpoints, including MMR rate at wk 96, and other long-term secondary efficacy and safety/tolerability results, will be presented at ASH 2024."

Clinical • P3 data • Atherosclerosis • Cardiovascular • Chronic Myeloid Leukemia • Congestive Heart Failure • Coronary Artery Disease • Gastroenterology • Gastrointestinal Disorder • Heart Failure • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Myocardial Infarction • Oncology • Pancreatitis • Respiratory Diseases • Thrombocytopenia

MOST plus: Adapting Treatment to the Tumor Molecular Alterations for Patients With Advanced Solid Tumors: MyOwnSpecificTreatment (clinicaltrials.gov) - P2 | N=560 | Recruiting | Sponsor: Centre Leon Berard | Not yet recruiting ➔ Recruiting

Enrollment open • Tumor mutational burden • Neutropenia • Oncology • Solid Tumor

MULTISARC: Molecular Profiling of Advanced Soft-tissue Sarcomas (clinicaltrials.gov) - P3 | N=960 | Recruiting | Sponsor: Institut National de la Santé Et de la Recherche Médicale, France | Not yet recruiting ➔ Recruiting | Trial completion date: Mar 2024 ➔ Oct 2024 | Initiation date: Mar 2019 ➔ Oct 2019 | Trial primary completion date: Mar 2022 ➔ Oct 2022

Enrollment open • IO biomarker • Trial completion date • Trial initiation date • Trial primary completion date • Oncology • Sarcoma • Soft Tissue Sarcoma

MOST plus: Adapting Treatment to the Tumor Molecular Alterations for Patients With Advanced Solid Tumors: MyOwnSpecificTreatment (clinicaltrials.gov) - P2 | N=560 | Recruiting | Sponsor: Centre Leon Berard | Trial completion date: Nov 2019 ➔ Oct 2022 | Trial primary completion date: Jul 2018 ➔ Jan 2020

Trial completion date • Trial primary completion date • Tumor mutational burden • Hematological Disorders • Neutropenia • Oncology • Solid Tumor