Ilaris (canakinumab) / Novartis - LARVOL DELTA

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

Canakinumab in combination with first-line (1L) pembrolizumab plus chemotherapy for advanced non‑small cell lung cancer (aNSCLC): Results from the CANOPY-1 phase 3 trial (AACR 2022) - "Patients with previously untreated stage IIIB/C or IV NSCLC, of any histology and no known EGFR or ALK alterations, were randomized 1:1 and stratified based on PD-L1 status, geographic region, and histology to canakinumab 200 mg or PBO every 3 weeks, plus pembrolizumab and histology-guided PDC for 4 cycles, followed by maintenance canakinumab or PBO, with pembrolizumab ± pemetrexed. The addition of canakinumab to pembrolizumab plus PDC did not statistically improve PFS nor OS for 1L treatment of patients with aNSCLC. No unexpected safety findings were observed with the addition of canakinumab to pembrolizumab plus PDC."

Clinical • Combination therapy • IO biomarker • P3 data • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • ALK • EGFR • IL1B • PD-L1

CANOPY-1: Study of Efficacy and Safety of Pembrolizumab Plus Platinum-based Doublet Chemotherapy With or Without Canakinumab in Previously Untreated Locally Advanced or Metastatic Non-squamous and Squamous NSCLC Subjects (clinicaltrials.gov) - P3 | N=673 | Active, not recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: Jun 2027 ➔ May 2026

Trial completion date • Lung Cancer • Lung Non-Small Cell Squamous Cancer • Lung Non-Squamous Non-Small Cell Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • PD-L1

Targeting Interleukin-1beta to Overcome Adaptive Immune Resistance in Renal Cell Carcinoma (KCRS 2023) - P1 | "We therefore initiated a clinical trial of neoadjuvant anti-IL1ß (canakinumab) plus anti-PD-1 (spartalizumab) in localized ccRCC. We will also identify novel immunotherapy targets in ccRCC, which will be tested in murine models and a high-throughput organoid T cell co-culture system to rapidly accelerate the development of novel combination regimens for ccRCC. This application addresses the KCRP focus on novel therapeutic targets and strategies for kidney cancer."

Clear Cell Renal Cell Carcinoma • Genito-urinary Cancer • Immune Modulation • Kidney Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor • CD8 • IL1B

Canakinumab Versus Placebo in Combination With First-Line Pembrolizumab Plus Chemotherapy for Advanced Non-Small-Cell Lung Cancer: Results From the CANOPY-1 Trial. (PubMed, J Clin Oncol) - "The addition of canakinumab to first-line pembrolizumab and CT did not prolong PFS or OS in patients with NSCLC."

Clinical • Combination therapy • IO biomarker • Journal • Metastases • Hematological Disorders • Infectious Disease • Lung Cancer • Neutropenia • Non Small Cell Lung Cancer • Oncology • Pain • Pulmonary Disease • Solid Tumor • ALK • CRP • EGFR • IL6

Long-term outcomes of vedolizumab targeted therapy in patients with very early onset Inflammatory Bowel Disease (ECCO-IBD 2026) - "63% of patients were biologic-naive, and VEDO was used as dual therapy in 16 patients, including canakinumab (n=3), tacrolimus (n=2), ruxolitinib (n=1), methotrexate (n=4), adalimumab (n=1), sirolimus (n=3), ustekinumab (n=1)...Three patients discontinued VEDO after 6 months due to worsening symptoms, transitioning to ustekinumab (n=1) and infliximab (n=2)...Conclusion In our experience, vedolizumab showed 12 months therapy durability in 95% of the patients with VEO-IBD in this cohort. Quiescent disease and clinical remission were achieved in > 70% of patients with few adverse events."

Clinical • Crohn's disease • Immunology • Inflammation • Inflammatory Bowel Disease • Ulcerative Colitis

Immunometabolic disorders in type 2 diabetes mellitus mediated by NLRP3 inflammasome activation and methods of pharmacological correction thereof (PubMed, Probl Endokrinol (Mosk)) - "Given the role of interleukin-1β (IL-1β) in the inflammation associated with type 2 DM, anti-IL-1 therapies such as anakinra, canakinumab and gevokizumab are being investigated in both experimental models of DM and clinical trials. Among the inhibitors of NLRP3 inflammasome activation, MCC950, OLT1177, CY-09 are the most studied, but none of the compounds in this group are currently used in clinical practice. The aim of this review is to assess the role of the NLRP3 inflammasome in the pathogenesis of type 2 diabetes, as well as the potential of inflammasome pathway inhibitors as promising therapeutic agents."

Journal • Review • Diabetes • Infectious Disease • Inflammation • Metabolic Disorders • Type 2 Diabetes Mellitus • IL1B • NLRP3

Targeting cytokine pathways: the role of biologics in autoinflammatory disorders. (PubMed, Expert Rev Clin Immunol) - "This review summarizes molecular mechanisms and therapeutic strategies, focusing on IL-1 blockade with anakinra, canakinumab, and rilonacept, as well as IL-6 and TNF inhibitors. Recognition of novel syndromes such as HA20, OTULIN deficiency, and PRAAS has expanded the therapeutic landscape. Next-generation biologics targeting multiple cytokine pathways may further personalize therapy."

Journal • Review • Amyloidosis • Genetic Disorders • Inflammation • Interferonopathies

A Diagnostic Challenge: Recurrent Uveitis Leading to the Diagnosis of Hyper-IgD Syndrome in a 28-Year-Old Patient. (PubMed, Ocul Immunol Inflamm) - "Following the diagnosis, the patient was started on targeted anti-IL-1 therapy (anakinra or canakinumab). Recurrent uveitis, even in adult patients, should prompt the investigation of rare autoinflammatory syndromes. A timely genetic diagnosis and a multidisciplinary approach are crucial for initiating targeted biological treatments, which offer a superior visual and systemic prognosis compared to conventional therapies."

Journal • Conjunctivitis • Inflammation • Macular Edema • Ocular Infections • Ocular Inflammation • Ophthalmology • Pain • Uveitis

Canakinumab as effective therapy in anakinra-refractory Schnitzler syndrome: a case-based review. (PubMed, Intern Med J) - "Interleukin-1 (IL-1) targeted therapy constitutes the basis for treatment and can lead to complete resolution of symptoms. We report a case of Schnitzler syndrome in a 49-year-old man refractory to anakinra and subsequently treated successfully with canakinumab."

Journal • Dermatology • Immunology • Monoclonal Gammopathy • Musculoskeletal Pain • Pain • Urticaria • IL1B

Macrophage Activation Syndrome Associated with Adult-onset Still's Disease Successfully Treated with Plasma Exchange and Canakinumab: A Case Report. (PubMed, Intern Med) - "Despite the administration of prednisolone and tocilizumab, MAS developed with hyperferritinemia, thrombocytopenia, hypertriglyceridemia, and liver dysfunction. Steroid pulse therapy, cyclosporine A, and plasma exchange (PE) improved the patient's condition...Subcutaneous canakinumab (300 mg/month) was initiated, thus enabling rapid prednisolone tapering to 10 mg within one month without relapse. This case highlights that PE followed by canakinumab is a potentially effective strategy for treating severe AOSD with MAS, particularly when minimizing steroid exposure is critical."

Journal • Diabetes • Dyslipidemia • Hematological Disorders • Hepatology • Hypertriglyceridemia • Immunology • Inflammation • Liver Failure • Metabolic Disorders • Thrombocytopenia

Safety and Effectiveness of Canakinumab in Patients with Systemic Juvenile Idiopathic Arthritis in Japan: A Post-Marketing Surveillance Study. (PubMed, Rheumatol Ther) - "Canakinumab showed a favourable long-term safety profile and sustained effectiveness in Japanese patients with sJIA inadequately controlled by existing therapies. No new safety concerns emerged, and the safety profile was comparable to that observed in the earlier phase III study."

Journal • P4 data • Hemophagocytic lymphohistiocytosis • Idiopathic Arthritis • Immunology • Inflammation • Rare Diseases • Rheumatology

CANOPY-A: Phase III study of canakinumab (CAN) as adjuvant therapy in patients (pts) with completely resected non-small cell lung cancer (NSCLC) (ESMO 2022) - P3 | "Methods CANOPY-A (NCT03447769) is a Phase III study of adult pts with Stage II–IIIB (AJCC/UICC v8), completely resected NSCLC who had received adjuvant, cisplatin-based chemotherapy (CTx; if applicable) and thoracic radiotherapy (if applicable). Conclusions CANOPY-A did not show a DFS benefit with CAN vs PBO after surgery and adjuvant CTx in pts with resected, Stage II–IIIB NSCLC. No new safety signals were identified with CAN treatment."

Clinical • Late-breaking abstract • P3 data • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • IL1B

Canakinumab as Adjuvant Therapy in Patients With Completely Resected Non-Small-Cell Lung Cancer: Results From the CANOPY-A Double-Blind, Randomized Clinical Trial. (PubMed, J Clin Oncol) - P3 | "CANOPY-A did not show a DFS benefit of adding canakinumab after surgery and adjuvant cisplatin-based chemotherapy in patients with resected, stage II-III NSCLC. No new safety signals were identified with canakinumab."

Clinical • Journal • Cardiovascular • Hematological Disorders • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • Thrombosis • CRP • IL1B • IL6

Effect of Clonal Hematopoiesis Mutations and Canakinumab Treatment on Incidence of Solid Tumors in the CANTOS Randomized Clinical Trial. (PubMed, Cancer Prev Res (Phila)) - P3 | "The cumulative incidence of at least one reported malignancy was lower for patients with TET2 mutations treated with canakinumab vs those treated with placebo. These findings support a potential role for canakinumab in cancer prevention and provide evidence of IL-1β blockade cooperating with CH mutations to modify the disease course."

Clinical • Journal • Cardiovascular • Myocardial Infarction • Solid Tumor • Thrombosis • DNMT3A • IL1B • TET2

Advancing Precision Medicine in Adult-Onset Still's Disease: Insights into Biomarkers, Therapies, and COVID-19 Impacts. (PubMed, Mediterr J Rheumatol) - "Biologic therapies (anakinra, canakinumab, tocilizumab) achieve remission in 80-90% of systemic cases. This review synthesises diagnostic challenges, novel biomarkers (e.g., gasdermin D), and emerging therapies (e.g., IL-18 binding protein), emphasising precision medicine and future research needs."

Biomarker • Journal • Review • Idiopathic Arthritis • Immunology • Infectious Disease • Inflammation • Musculoskeletal Pain • Novel Coronavirus Disease • Pulmonary Disease • Respiratory Diseases • Rheumatology • IL18 • IL6

The IL-1β inhibitor canakinumab in previously treated lower-risk myelodysplastic syndromes: a phase 2 clinical trial. (PubMed, Nat Commun) - P2 | "Sequential post-hoc prospective single-cell RNA sequencing analyses of HSPCs and bone marrow mononuclear cells at different time points during therapy showed that canakinumab's on-target effects in hematopoietic populations expressing the IL-1β receptor decreased the TNF-mediated inflammatory signaling pathway but rescued ineffective erythropoiesis only in the context of lower genetic complexity. This study demonstrates that better stratification strategies could target lower-risk MDS patients more effectively."

Journal • P2 data • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • IL1B

Canakinumab for the Prevention of Progression to Cancer in Patients With Clonal Cytopenias of Unknown Significance, IMPACT Study (clinicaltrials.gov) - P2 | N=110 | Not yet recruiting | Sponsor: Uma Borate

New P2 trial • Hematological Disorders • Immunology • Inflammation • Oncology • ASXL1 • SF3B1 • SRSF2 • TET2 • TP53 • U2AF1 • ZRSR2

Autoinflammatory syndrome associated with a variant of the NLRP1 gene (NAIAD): description of a pediatric case treated with canakinumab. (PubMed, Med Clin (Barc)) - No abstract available

Journal • Pediatrics • NLRP1

How Physicians Manage Colchicine-Resistant FMF: Insights from a Multinational Survey in High-Prevalence Countries. (PubMed, J Rheumatol) - "Paediatric rheumatologists tended to favor discontinuation of biologic therapy rather than lifelong use. On-demand use of biologics also emerged as a treatment approach among treating physicians. Issues such as colchicine compliance, disease-specific triggers, and the potential to maintain efficacy upon reintroduction of IL-1 inhibitors after discontinuation should be prioritized in future research."

Journal • Genetic Disorders • Pediatrics • Rheumatology

Evaluation of the Effect of IL-1 Antagonists on Pituitary Function. (PubMed, Int J Endocrinol) - "IL-1 inhibitors, such as anakinra, rilonacept, and canakinumab, have been introduced as adjunctive treatments...The control group consisted of patients receiving colchicine treatment only...However, differences were noted in FSH, total testosterone, and GH levels, with higher FSH and GH in the control group and higher testosterone in the experimental group. Although IL-1 plays a role in hormone secretion pathways, further studies are needed to better understand the effect of IL-1 antagonists on pituitary function, as no significant adrenal or pituitary insufficiencies were observed."

Journal • Endocrine Disorders • Genetic Disorders • Inflammatory Arthritis • Musculoskeletal Pain • Oncology • Pain • IGF1 • IL1B • PRL

Inflammatory Mechanisms in Acute Coronary Syndromes: From Pathophysiology to Therapeutic Targets. (PubMed, Cells) - "Anti-inflammatory therapies including colchicine, canakinumab (IL-1β inhibition), and methotrexate have shown cardiovascular benefits in clinical trials. While colchicine and IL-1β inhibition have demonstrated clinical efficacy, future strategies should focus on precision medicine approaches targeting specific inflammatory pathways based on individual patient profiles. Integration of anti-inflammatory therapy with lipid management and antithrombotic strategies offers promise for improving ACS outcomes through comprehensive targeting of the multifactorial pathophysiology underlying coronary artery disease."

Journal • Review • Acute Coronary Syndrome • Cardiovascular • Coronary Artery Disease • Hematological Disorders • Inflammation • Thrombosis • CRP • IL1B • IL6

A Hidden Pathway to a Major Concern: The Role of Pyroptosis in Inducing Myocardial Infarction Reperfusion Injury and Emerging Therapeutic Targets. (PubMed, Cureus) - "Trials of agents like cyclosporine A and remote ischemic conditioning have yielded mixed results...MCC950, OLT1177, and VX-765 are NLRP3, caspase-1, and GSDMD inhibitors that decrease the infarct size in mice prior to use in humans. The clinical IL-1β antagonist canakinumab was effective in reducing recurrent events in the CANTOS (Cardiovascular Risk Reduction Study (Reduction in Recurrent Major CV Disease Events)) trial...Focusing on this can help expand our aim to look for more potential therapeutics. The re-purposing of existing drugs, such as colchicine and disulfiram, and the development of new, specific NLRP3 inhibitors represent a promising pipeline toward finally achieving clinically relevant cardioprotection that enhances the prognosis in patients suffering from myocardial infarction."

Journal • Review • Cardiovascular • Inflammation • Myocardial Infarction • Reperfusion Injury • IL18 • IL1B • NLRP3

Neonatal-onset multisystem inflammatory disease caused by a de novo NLRP3 gene mutation: a case report and literature review. (PubMed, Front Pediatr) - "This case highlights the importance of early recognition of NOMID in neonates with antibiotic-unresponsive systemic inflammation. Early genetic confirmation and targeted IL-1 blockade with canakinumab are crucial to preventing devastating complications."

Journal • CNS Disorders • Dermatology • Immunology • Infectious Disease • Inflammation • Urticaria • IL1B • NLRP3

Disproportionality Analysis of Adverse Events Associated with IL-1 Inhibitors in the FDA Adverse Event Reporting System (FAERS). (PubMed, Pharmaceuticals (Basel)) - "Importantly, we also identified less common or previously unreported AEs, including cardiac disorders (e.g., postural orthostatic tachycardia syndrome with anakinra; pulmonary valve incompetence with rilonacept) and endocrine disorders (e.g., secondary adrenocortical insufficiency with canakinumab). Furthermore, 36.33% of cases emerged after more than 360 days of treatment with IL-1 inhibitors. This study revealed real-world safety data on IL-1 inhibitors, providing important insights to enhance the clinical use of IL-1 inhibitors and minimize potential AEs."

Adverse events • Journal • Cardiovascular • Dermatology • Endocrine Disorders • Genetic Disorders • Infectious Disease • Inflammation