MAS825 / Novartis - LARVOL DELTA

MAS NLRC4-GOF: Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations (clinicaltrials.gov) - P2 | N=17 | Active, not recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: Jan 2031 ➔ Feb 2029

Trial completion date • Gastrointestinal Disorder • Inflammation • CDC42 • IL18 • XIAP

A Study to Investigate the Efficacy, Safety, and Tolerability of DFV890 and MAS825 for Inflammatory Marker Reduction in Adult Participants With Coronary Heart Disease and Clonal Hematopoiesis of Indeterminate Potential (CHIP) (clinicaltrials.gov) - P2 | N=31 | Completed | Sponsor: Novartis Pharmaceuticals | Active, not recruiting ➔ Completed

Trial completion • Cardiovascular • Coronary Artery Disease • Heart Failure • Hematological Disorders • DNMT3A • IL18 • IL6 • TET2

MAS NLRC4-GOF: Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations (clinicaltrials.gov) - P2 | N=17 | Active, not recruiting | Sponsor: Novartis Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed • Gastrointestinal Disorder • Inflammation • CDC42 • IL18 • XIAP

A Study to Investigate the Efficacy, Safety, and Tolerability of DFV890 and MAS825 for Inflammatory Marker Reduction in Adult Participants With Coronary Heart Disease and Clonal Hematopoiesis of Indeterminate Potential (CHIP) (clinicaltrials.gov) - P2 | N=31 | Active, not recruiting | Sponsor: Novartis Pharmaceuticals | Recruiting ➔ Active, not recruiting | Trial completion date: Feb 2026 ➔ Nov 2024 | Trial primary completion date: Feb 2026 ➔ Oct 2024

Enrollment closed • Trial completion date • Trial primary completion date • Cardiovascular • Coronary Artery Disease • Heart Failure • Hematological Disorders • DNMT3A • IL18 • IL6 • TET2

MAS NLRC4-GOF: Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations (clinicaltrials.gov) - P2 | N=18 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: Oct 2028 ➔ Jan 2031

Trial completion date • Gastrointestinal Disorder • Inflammation • CDC42 • IL18 • XIAP

Study of Efficacy and Safety of Investigational Treatments in Patients With Moderate to Severe Hidradenitis Suppurativa (clinicaltrials.gov) - P2 | N=248 | Active, not recruiting | Sponsor: Novartis Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed • Dermatology • Hidradenitis Suppurativa • Immunology

Long-term efficacy of MAS825, a bispecific anti-IL1β and IL-18 monoclonal antibody, in two patients with sJIA and recurrent episodes of MAS. (PubMed, Rheumatology (Oxford)) - "In light of the pivotal role of IL-1β and IL-18 in sJIA, MAS and ILD, MAS825 might represent a possible valid and safe option in the treatment of drug-resistant sJIA, especially in the presence of high serum IL-18 levels."

Journal • CNS Disorders • Idiopathic Arthritis • Immunology • Infectious Disease • Inflammation • Interstitial Lung Disease • Novel Coronavirus Disease • Pulmonary Disease • Respiratory Diseases • Rheumatoid Arthritis • Rheumatology • IL18 • IL1B

Macrophage Activation Syndrome (EULAR 2024) - "She was treated with high dose of glucocorticoid, ciclosporin, anakinra and emapalumab in compassionate use (CU) regimen with improvement of clinical condition and laboratory features.In May 2021 emapalumab was discontinued without disease flare.In June 2022 for the recurrence of hypertransaminasemia and cytopenia after any viral infection and considering the persistently high levels of IL18, treatment with MAS825,a bispecific monoclonal antibody targeting IL-1b and IL-18, was started with. progressive improvement of clinical condition and normalization of laboratory parameters. Glucocorticoids were completely discontinued after five infusions of MAS825."

Anemia • Ankylosing Spondylitis • Bone Marrow Transplantation • Celiac Disease • Dermatology • Diabetes • Dyslipidemia • Hematological Disorders • Hepatology • Hidradenitis Suppurativa • Hypertriglyceridemia • Idiopathic Arthritis • Immunology • Infectious Disease • Inflammation • Metabolic Disorders • Musculoskeletal Pain • Orthopedics • Pain • Pediatrics • Pulmonary Disease • Respiratory Diseases • Rheumatology • Seronegative Spondyloarthropathies • Spondylarthritis • Thrombocytopenia • Transplantation • IL18 • IL1B • PRF1 • XIAP

Study of Efficacy and Safety of Investigational Treatments in Patients With Moderate to Severe Hidradenitis Suppurativa (clinicaltrials.gov) - P2 | N=245 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial primary completion date: Oct 2026 ➔ Nov 2024

Trial primary completion date • Dermatology • Hidradenitis Suppurativa • Immunology

A Single-patient Clinical Trial of MAS825 in a Patient With XIAP Deficiency (clinicaltrials.gov) - P3 | N=1 | Completed | Sponsor: The Hospital for Sick Children

New P3 trial

A Study to Investigate the Efficacy, Safety, and Tolerability of DFV890 and MAS825 for Inflammatory Marker Reduction in Adult Participants With Coronary Heart Disease and Clonal Hematopoiesis of Indeterminate Potential (CHIP) (clinicaltrials.gov) - P2 | N=28 | Recruiting | Sponsor: Novartis Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open • Cardiovascular • Coronary Artery Disease • Heart Failure • Hematological Disorders • DNMT3A • IL18 • IL6 • TET2

A Study to Investigate the Efficacy, Safety, and Tolerability of DFV890 and MAS825 for Inflammatory Marker Reduction in Adult Participants With Coronary Heart Disease and Clonal Hematopoiesis of Indeterminate Potential (CHIP) (clinicaltrials.gov) - P2 | N=28 | Not yet recruiting | Sponsor: Novartis Pharmaceuticals | Phase classification: P2a ➔ P2

Phase classification • Cardiovascular • Coronary Artery Disease • Heart Failure • Hematological Disorders • DNMT3A • IL18 • IL6 • TET2

Experience of Anti IL-1β and Anti IL-18 combined therapy (MAS825) in recurrent and recalcitrant macrophage activation syndrome. (PubMed, Rheumatology (Oxford)) - No abstract available

Journal • IL18 • IL1B

A Study to Investigate the Efficacy, Safety, and Tolerability of DFV890 and MAS825 for Inflammatory Marker Reduction in Adult Participants With Coronary Heart Disease and Clonal Hematopoiesis of Indeterminate Potential (CHIP) (clinicaltrials.gov) - P2a | N=28 | Not yet recruiting | Sponsor: Novartis Pharmaceuticals

New P2a trial • Cardiovascular • Coronary Artery Disease • Heart Failure • Hematological Disorders • DNMT3A • IL18 • IL6 • TET2

Efficacy and safety of MAS825 (anti-IL-1ꞵ/IL-18) in COVID-19 patients with pneumonia and impaired respiratory function. (PubMed, Clin Exp Immunol) - "MAS825 used in conjunction with SoC was well tolerated. None of the adverse events (AEs) or serious AEs were treatment-related."

Journal • Critical care • Infectious Disease • Inflammation • Novel Coronavirus Disease • Pneumonia • Respiratory Diseases • CRP • IFNG • IL18 • IL1B • IL6

Study of Efficacy and Safety of Investigational Treatments in Patients With Moderate to Severe Hidradenitis Suppurativa (clinicaltrials.gov) - P2 | N=200 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: May 2023 ➔ Jul 2026 | Trial primary completion date: May 2023 ➔ Jul 2026

Trial completion date • Trial primary completion date • Dermatology • Hidradenitis Suppurativa

MAS NLRC4-GOF: MASter-1 Study to Evaluate the Efficacy and Safety of MAS825 in NLRC4-GOF Patients (clinicaltrials.gov) - P2 | N=8 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: Dec 2027 ➔ Sep 2028 | Trial primary completion date: Oct 2024 ➔ Jun 2025

Trial completion date • Trial primary completion date • Gastrointestinal Disorder • Inflammation

Study of Efficacy and Safety of Investigational Treatments in Patients With Moderate to Severe Hidradenitis Suppurativa (clinicaltrials.gov) - P2 | N=200 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: Aug 2023 ➔ May 2023 | Trial primary completion date: Aug 2023 ➔ May 2023

Trial completion date • Trial primary completion date • Dermatology • Hidradenitis Suppurativa

MAS-FIH: A First-in-human Study to Investigate the Safety, Tolerability and Pharmacokinetics of MAS825 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=80 | Completed | Sponsor: Novartis Pharmaceuticals | Active, not recruiting ➔ Completed

Trial completion

Study of Efficacy and Safety of Investigational Treatments in Patients With Moderate to Severe Hidradenitis Suppurativa (clinicaltrials.gov) - P2 | N=200 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial primary completion date: Aug 2022 ➔ Jun 2023

Trial primary completion date • Dermatology • Hidradenitis Suppurativa

MAS NLRC4-GOF: MASter-1 Study to Evaluate the Efficacy and Safety of MAS825 in NLRC4-GOF Patients (clinicaltrials.gov) - P2 | N=8 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: Apr 2027 ➔ Dec 2027 | Trial primary completion date: Feb 2024 ➔ Oct 2024

Trial completion date • Trial primary completion date • Gastrointestinal Disorder • Immunology • Inflammation

Improvement of Refractory Systemic Juvenile Idiopathic Arthritis-Associated Lung Disease with Single-Agent Blockade of IL-1β and IL-18. (PubMed, J Clin Immunol) - "The patient was able to wean entirely off systemic corticosteroids and other biologics after 10 months of treatment with MAS-825 and experienced no side effects of the drug. This case demonstrates improvement in pulmonary symptoms, lung inflammation, and burden of immunomodulatory therapy after treatment with MAS-825 and suggests that simultaneous targeting of both IL-1β and IL-18 may be a safe and effective treatment strategy in SJIA-LD."

Journal • Idiopathic Arthritis • Immune Modulation • Immunology • Inflammation • Interstitial Lung Disease • Pneumonia • Pulmonary Disease • Respiratory Diseases • Rheumatoid Arthritis • Rheumatology • CD4 • CD8 • IL18 • IL1B

Inhibition of Relevant Clinical and Pathway Biomarkers in Patients with COVID-19 Pneumonia and Inflammation in a Phase 2 Multicenter Randomized Study with MAS825, an Anti-IL-1β/IL-18 Bispecific Monoclonal Antibody (FOCIS 2022) - P2 | "MAS825 used in conjunction with SoC was safe and well tolerated, inhibited relevant clinical and inflammatory pathway biomarkers and resulted in faster virus clearance in COVID-19 pneumonia patients when compared with placebo."

Biomarker • Clinical • P2 data • Immunology • Inflammation • CRP • IFNG • IL18 • IL1B • IL6

MAS-FIH: A First-in-human Study to Investigate the Safety, Tolerability and Pharmacokinetics of MAS825 in Healthy Volunteers (clinicaltrials.gov) - P1; N=80; Active, not recruiting; Sponsor: Novartis Pharmaceuticals; Recruiting ➔ Active, not recruiting; Trial completion date: Apr 2022 ➔ Aug 2022; Trial primary completion date: Apr 2022 ➔ Aug 2022

Clinical • Enrollment closed • Trial completion date • Trial primary completion date

Study of Efficacy and Safety of Investigational Treatments in Patients With Moderate to Severe Hidradenitis Suppurativa (clinicaltrials.gov) - P2; N=200; Recruiting; Sponsor: Novartis Pharmaceuticals; N=130 ➔ 200

Clinical • Enrollment change • Dermatology • Hidradenitis Suppurativa