Ilaris (canakinumab) / Novartis - LARVOL DELTA

Real world experience of patient outcomes in a dedicated clonal hematopoiesis hematology clinic (ASH 2025) - P2 | "The overall rate of progression from CH to myeloid neoplasm is typically thought to be 0.5 to1% per year. However, our experience in a dedicated CH clinic suggests that pts referred may be at ahigher risk of progression, with nearly half having prior chemotherapy or radiation exposure and up to25% of pts considered high risk by current prognostic models. All pts with high risk CCUS in HALT clinicare screened for the IMPACT study, a multi-center, randomized, placebo-controlled trial of canakinumabto prevent the progression to overt MN with 25 pts currently enrolled nationally, with 17 pts at OSU(NCT05641831)."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Breast Cancer • Hematological Malignancies • Leukemia • Multiple Myeloma • Myelodysplastic Syndrome • ASXL1 • DNMT3A • PPM1D • TET2 • TP53

A phase 2 study of canakinumab in patient with myelofibrosis: Results from part 1 (ASH 2025) - "Introduction Myelofibrosis (MF) is a myeloproliferative neoplasm (MPN) for which the only approved therapies are JAKinhibitors which improve splenomegaly and disease-related symptoms. Given the encouraging impact onMF symptoms, blood counts and reassuring safety profile, the study was amended to enroll pts on astable dose of a JAK inhibitor (ruxolitinib, fedratinib, momelotinib, or pacritinib) who have the potential tobenefit from additional therapy. Part 2 is currently enrolling at multiple sites and updated clinical andcorrelative data will be presented at the meeting."

Clinical • P2 data • Cardiovascular • Fibrosis • Immunology • Myelofibrosis • Myeloproliferative Neoplasm • Thrombocytopenia • CRP • IL1B

Comparative effectiveness and safety of systemic therapies for treatment-naïve, PD-L1 expression <1% advanced NSCLC: a systematic review and network meta-analysis. (PubMed, Transl Lung Cancer Res) - "In terms of OS, pembrolizumab + chemotherapy + canakinumab (Pembro-chemo-canakinumab) (SUCRA =0.90) showed great potential in improving outcomes, although its long-term efficacy still needed to be validated...For squamous patients, nivolumab + ipilimumab ± chemotherapy (Nivo-ipi-chemo) led in OS, while serplulimab + chemotherapy in PFS. First-line personalized treatment for PD-L1 <1%, advanced NSCLC should be histology-based, balancing efficacy and toxicity. Pembro-chemo and nivolumab + chemotherapy + bevacizumab combinations are recommended as the optimal first-line options for non-squamous patients, and Nivo-ipi-chemo for squamous patients."

HEOR • IO biomarker • Journal • Retrospective data • Lung Cancer • Lung Non-Small Cell Squamous Cancer • Lung Non-Squamous Non-Small Cell Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • ALK • EGFR • PD-L1

Model-Based Meta-Analysis With MonolixSuite: A Tutorial for Longitudinal Categorical and Continuous Data. (PubMed, CPT Pharmacometrics Syst Pharmacol) - "Two case studies are presented: the first examining naproxen in osteoarthritis and the second evaluating canakinumab compared to existing treatments in rheumatoid arthritis. Furthermore, it illustrates how to use the model in Simulx to support the decision-making process, such as by simulating clinical trials. This step-by-step guidance offers practical insights for leveraging MBMA in model-informed drug development."

Journal • Retrospective data • Immunology • Osteoarthritis • Pain • Rheumatoid Arthritis • Rheumatology

Treatment responses and relapse predictors in pediatric CNO: insights from a referral center. (PubMed, Rheumatology (Oxford)) - "Conventional DMARDs are effective in a limited subset of CNO patients, who may represent a milder disease phenotype. The presence of skin involvement and a greater number of affected bones may both suggest a more aggressive disease course. Anti-TNF therapies are the most effective agents, while other biologics offer promising results in resistant cases. Prospective studies are needed to guide individualized treatment strategies."

Journal • Fatigue • Inflammation • Musculoskeletal Pain • Pain • Pediatrics

Beyond Lipids and Platelets: A Review of Anti-Inflammatory Strategies in Secondary Prevention of Acute Coronary Syndromes. (PubMed, J Clin Med) - "Colchicine, now FDA-approved for cardiovascular risk reduction, lowered major adverse cardiovascular events in COLCOT and LoDoCo2. Canakinumab (IL-1β inhibition) reduced recurrent events in proportion to IL-6 and hsCRP suppression, while ziltivekimab (IL-6 inhibition) achieved profound biomarker reductions but remains investigational. Early-phase studies of anakinra (IL-1 receptor antagonist) and dapansutrile (oral NLRP3 inhibitor) showed anti-inflammatory effects in early trials, whereas varespladib and darapladib illustrated the challenges of targeting lipid-associated pathways...Additional emerging avenues include triptolidiol, dasatinib, and BTK or JAK/STAT inhibitors, while novel approaches, such as nanozyme delivery systems and CRISPR-based editing, extend the therapeutic horizon. This review highlights the potential of inflammation-targeted therapies to advance secondary prevention in ACS by integrating current evidence and perspectives on future therapeutic..."

Journal • Review • Acute Coronary Syndrome • Atherosclerosis • Cardiovascular • Coronary Artery Disease • Immunology • Inflammation • Myocardial Infarction • CRP • IL1B • NLRP3

Mechanistic role of the IL-1β/c-Fos/NFATc1 signaling axis in echinococcal infection-promoted osteoclast differentiation and activation in pathological osteolysis: a prospective controlled trial. (PubMed, J Orthop Surg Res) - "Echinococcal infection promotes pathological osteoclastogenesis and osteolysis through IL-1β/c-Fos/NFATc1 signaling activation."

Journal • Infectious Disease • Oncology • Osteosarcoma • Solid Tumor • CSF1 • CTSK • CXCL8 • FOS • IL1B • IL6 • MMP9 • NFATC1 • TNFA

Inflammasomes as Potential Therapeutic Targets to Prevent Chronic Active Viral Myocarditis-Translating Basic Science into Clinical Practice. (PubMed, Int J Mol Sci) - "Additionally, we discuss both novel and established therapeutic strategies targeting inflammatory and anti-fibrotic mechanisms, including IL-1 receptor blockers (anakinra, canakinumab), NOD-like receptor protein 3 (NLRP3) inhibitors (colchicine, MCC950, dapansutrile, INF200), NF-κB inhibitors, and angiotensin receptor-neprilysin inhibitors (ARNI), as well as microRNAs. Our aim is to emphasize the clinical importance of early identification of patients at risk of transitioning from acute to chronic inflammation, elucidate the role of inflammasomes, and present emerging therapies that may improve outcomes by balancing effective pathogen clearance with limitation of chronic cardiac damage."

Journal • Review • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Fibrosis • Heart Failure • Immunology • Infectious Disease • Inflammation • Respiratory Diseases • IL18 • IL1B • NLRP3

Long-term Effectiveness and Safety of Canakinumab in Patients with TRAPS: Analysis of the RELIANCE Non-Interventional Study. (PubMed, Rheumatol Ther) - "Data from this interim analysis support the long-term effectiveness and safety of canakinumab for the treatment of TRAPS."

Journal • Observational data • Fatigue • Oncology • Pediatrics • CRP • TNFA • TNFRSF1A

Pericarditis at the crossroads: Unlocking the next wave of therapies. (PubMed, Int J Cardiol Heart Vasc) - "Traditional treatments - nonsteroidal anti-inflammatory drugs (NSAIDs), colchicine, and corticosteroids - have important limitations, including steroid dependence, high recurrence rates, and side effects...Key trials of IL-1 inhibitors (anakinra, rilonacept, canakinumab, goflikicept) have demonstrated dramatic reductions in recurrence rates, validating IL-1 as a therapeutic target...Overall, the therapeutic pipeline for pericarditis is robust and poised to transform management. In the coming years, integration of targeted biologics and small molecules alongside conventional anti-inflammatories may significantly improve outcomes in recurrent pericarditis, moving towards more precise and effective treatment strategies."

Journal • Review • Cardiovascular • Inflammation • NLRP3

A systems biology approach to identify key targets in KRAS/BRAF-mutated colorectal cancer. (PubMed, Discov Oncol) - "This study sheds light on the molecular mechanisms of KRAS-mutated CRC, emphasizing IL1B as a prognostic marker. Among the identified therapeutic agents, Omeprazole demonstrated the strongest interaction with AHR, suggesting a potential for repurposing in CRC treatment, while IL1B-targeting inhibitors such as Canakinumab emerged as selective candidates for modulating tumor-promoting inflammation. Novel genes such as EPSTI1, COL20A1, CDH1, and SOX9 warrant further investigation."

Journal • Colorectal Cancer • Infectious Disease • Novel Coronavirus Disease • Oncology • Respiratory Diseases • Solid Tumor • BRAF • CDH1 • EGF • EPSTI1 • IL1B • KRAS • SOX9

Evaluation of the real-world safety of Canakinumab in the treatment of systemic juvenile idiopathic arthritis in children aged 0-16: a comprehensive analysis based on FAERS data. (PubMed, Pediatr Rheumatol Online J) - "This FAERS-based pharmacovigilance study provides preliminary safety data on the use of Canakinumab in children aged 0-16 years with sJIA, confirming several known adverse reactions and revealing additional potential risks. These findings offer clinicians valuable safety information for the use of Canakinumab in the treatment of sJIA."

Journal • Real-world evidence • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hemophagocytic lymphohistiocytosis • Idiopathic Arthritis • Immunology • Infectious Disease • Leukopenia • Novel Coronavirus Disease • Rare Diseases • Rheumatology • Thrombocytopenia • IL1B

Targeting IL-1 in Autoinflammatory Syndromes: Lessons From Canakinumab and VEXAS Syndrome. (PubMed, Int J Rheum Dis) - No abstract available

Journal

Safety and effectiveness of canakinumab in Japanese patients with cryopyrin-associated periodic syndrome: final results from a post-marketing surveillance in Japan. (PubMed, Mod Rheumatol) - "Clinical symptoms related to auditory, joint, visual, renal, and central nervous system maintained or improved in majority of patients. ConclusionsCanakinumab was well-tolerated and effective during long-term treatment for patients with CAPS in the real-world setting."

Journal • P4 data • Genetic Disorders • Infectious Disease • Inflammation • Rare Diseases • IL1B

Targeted Anti-Inflammatory Therapy in Cardiovascular Events: Challenges and Opportunities. (PubMed, J Clin Hypertens (Greenwich)) - "By directly modulating inflammatory pathways, canakinumab significantly lowered the incidence of major adverse cardiovascular events (MACE) independent of lipid levels. Similarly, colchicine, an ancient anti-inflammatory drug, has gained renewed interest due to its efficacy in reducing cardiovascular events in patients with chronic coronary disease and recent myocardial infarction...In conclusion, targeted anti-inflammatory therapy represents a promising adjunct to traditional CVD treatments, potentially revolutionizing the management of cardiovascular events. Future studies are essential to optimize these strategies and fully integrate them into clinical practice, enhancing outcomes for patients with CVD."

Journal • Review • Atherosclerosis • Cardiovascular • Inflammation • Myocardial Infarction • Oncology • IL6 • TNFA

Study of Canakinumab in Patients With Myelofibrosis (clinicaltrials.gov) - P2 | N=14 | Recruiting | Sponsor: John Mascarenhas | Trial primary completion date: Jan 2026 ➔ Jul 2026

Trial primary completion date • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Thrombocytosis

Efficacy of new immunomodulatory drugs on major adverse cardiovascular events in patients with coronary heart disease: a systematic review and meta-analysis of randomized controlled trials. (PubMed, BMC Cardiovasc Disord) - "Targeted anti-inflammatory therapies, particularly colchicine and canakinumab, significantly reduce MACE in CHD patients when used for longer than six months. Efficacy varies by mechanism of action, supporting precision use of NLRP3 and IL-1β inhibitors. Future trials should been focus on biomarker-guided, long-term anti-inflammatory interventions in cardiovascular care."

Adverse events • Journal • Retrospective data • Review • Cardiovascular • Coronary Artery Disease • Heart Failure • Inflammation • CRP • IL1B • IL6 • NLRP3

Targeting of Various Inflammatory Circuits Does Not Ameliorate Ineffective Hematopoiesis: Results of the Lucas and Canfire Studies Investigating IRAK4 or IL-1β Blockade to Target Anemia in Lower-Risk MDS (ASH 2024) - "Emavusertib treatment was associated with a reduction in neutrophil counts...Canakinumab treatment was well-tolerated with no reported SAEs...Despite the observed anti-inflammatory effects in the bone marrow, in LUCAS there was a paradoxical increase in inflammatory markers in peripheral blood, potentially due to compensatory mechanisms. These findings suggest that targeting multiple inflammatory pathways simultaneously may offer a more promising therapeutic strategy, such as the combination of IL-1β and IRAK4 inhibitors."

Anemia • Cardiovascular • Hematological Disorders • Hematological Malignancies • Infectious Disease • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • ASXL1 • CSF1 • IL10 • IL15 • IL1B • IL1R1 • IL7 • IRAK4 • SETBP1 • U2AF1

Clinical Manifestations Are Evolving and Progressive in Patients with Vexas Syndrome (ASH 2023) - P2 | "All except one patient were receiving prednisone at NIH evaluation, with median dose of 20mg (range 5mg-50mg)...Other agents included erythropoietin stimulating agents (n=2), canakinumab (n=2), adalimumab (n=2), methotrexate (n=2), and azacytidine, azathioprine, colchicine, dapsone, ustekinumab in one patient each...Most challenging are patients who have exhausted all effective steroid-sparing agents, require high dose of glucocorticoids, and are not a HSCT candidate. Development of effective therapies for VEXAS is a research priority to improve outcomes."

Clinical • Anemia • Bone Marrow Transplantation • Cardiovascular • Dermatology • Fatigue • Hematological Disorders • Hematological Malignancies • Inflammation • Multiple Myeloma • Musculoskeletal Pain • Myelodysplastic Syndrome • Nephrology • Oncology • Renal Disease • Transplantation • Venous Thromboembolism

Adult-Onset Still's Disease Complicated by Treatment Resistant Pure Red Cell Aplasia Requiring Cyclosporine: A Case Report and Review of Cases. (PubMed, Mod Rheumatol Case Rep) - "Treatment of the underlying Still's disease with glucocorticoids, IL-1 inhibitors and tocilizumab improved the clinical features of the patient's disease; however, the patient remained with severe transfusion-dependent anemia. The initiation of cyclosporine led to rapid remission of the PRCA."

Journal • Hematological Disorders • Immunology

Budget Impact of Anakinra in Turkiye: An Analysis From the Social Security Institution's Perspective (ISPOR-EU 2025) - "Other molecules reimbursed for these diseases include canakinumab (for CAPS, FMF and SJIA) and tocilizumab (for SJIA). Anakinra is a budget saving option for the SGK in treating CAPS, FMF and Still's disease in Turkiye. The SGK is expected to save 963,690,911 TRY in 2025, 1,116,436,889 TRY in 2026 and 1,252,951,238 TRY in 2027. The total savings over three years amount to 3,333,079,038 TRY."

Clinical • HEOR • Genetic Disorders • Idiopathic Arthritis • Immunology • Inflammation • Rheumatology

Phase 1b/2 Study Evaluating the Safety and Efficacy of Canakinumab with Darbepoetin Alfa in Patients with Lower-Risk Myelodysplastic Syndromes (MDS) Who Have Failed Erythropoietin Stimulating Agents (ESA) (ASH 2023) - "Additionally, 33% and 56% were refractory to lenalidomide and luspatercept therapy, respectively. The combination of canakinumab and darbepoetin was safe and well tolerated and 300mg of canakinumab is the RP2D. Canakinumab inhibited inflammasome activation as evidenced by a reduction of ASC specks although this did not translate to clinical responses. Phase 2 of the study will focus on patients with lower transfusion burden and molecular subsets with highest IL-1β expression (e.g."

Clinical • P1/2 data • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Immunology • Myelodysplastic Syndrome • Neutropenia • Oncology • Thrombocytopenia • CASP1 • DNMT3A • IL1B • TET2

Neutralization of IL-1β and P-Selectin Inhibition Attenuate Organ Injury in Mice with Sickle Cell Disease (ASH 2023) - "Continuous vaso-occlusive (VO) and inflammatory processes in the vasculature are most likely the principal cause of such lesions, and there is little evidence, to date, that long-term hydroxyurea therapy (the standard of care for SCD) reduces organ damage in SCD adults. Inhibition of P-selectin activity, using crizanlizumab, has shown some success for reducing VO crisis frequency in SCD. Furthermore, interleukin (IL)-1β blockade, in the form of canakinumab administration, was well tolerated in young patients with SCD and associated with inflammatory marker reduction...Combination treatments, however, especially in the kidney and liver, had more complex effects. We suggest that future preclinical and clinical studies should investigate the potential of anti-inflammatory/adhesion approaches (alone or in combination) for preventing damage to multiple organs, in addition to preventing VO events."

IO biomarker • Preclinical • Fibrosis • Genetic Disorders • Hematological Disorders • Hepatology • Immunology • Renal Disease • Respiratory Diseases • Sickle Cell Disease • IL1B • ITGA2B • PTPRC • SELP • TNFA

Tannin-Mediated Ischemia-Homing-Angiogenesis Nanodots (IHAND) for Synergistic Angiogenesis and Heart Failure Prevention Post-Myocardial Infarction. (PubMed, Adv Mater) - "Accordingly, IHAND can target VECs and macrophages in MI tissues, alleviate proliferation and migration disorders of VECs, induce phenotypic transformation of macrophages in early stages of MI, significantly reduce the storm of inflammatory factors, and promote the secretion of vascular endothelial growth factor A (VEGF-A) and platelet-derived growth factor-BB (PDGF-BB). IHAND demonstrates strong angiogenic ability in MI, and its therapeutic effect is remarkably superior to that of N-acetylcysteine and canakinumab."

Journal • Cardiovascular • Congestive Heart Failure • Heart Failure • Inflammation • Myocardial Infarction • CD31 • PECAM1

A Randomized Double-Blind Placebo-Controlled Phase II Multi-Center Study of Inflammation Modification of Canakinumab to Prevent Leukemic Progression of Clonal Cytopenias of Unknown Significance (CCUS): Impact Study (ASH 2023) - P2 | "A phase II clinical trial investigating the use of canakinumab in combination with azacitidine is currently studying the impact canakinumab may have on disease progression in relapsed/refractory low or intermediate risk MDS patients (NCT04239157). Accounting for 5% drop-out, this calculation assumes accrual of 89 patients over 3 years and a minimum follow-up of 5 years (or until withdrawal, death, or study closure; earliest event). To date, 5 patients have been randomized and enrolled on study (Table 1)."

Clinical • P2 data • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Inflammation • Myelodysplastic Syndrome • Oncology • ASXL1 • DNMT3A • IL1B • IL6 • TET2