Arcerix (goflikicept) / R-Pharm - LARVOL DELTA

Current Drug Treatment for Acute and Recurrent Pericarditis. (PubMed, Drugs) - "Low- to moderate-dose glucocorticoids are reserved for patients with a first recurrence for which NSAIDs and colchicine failed and/or who have an autoimmune disorder, with a slow tapering. Interleukin-1 blockers-anakinra, rilonacept, and goflikicept-are used as a third-line option in patients who cannot come off glucocorticoids or as second-line therapy after NSAIDs and colchicine in patients with contraindications to glucocorticoids or in those with high-risk features (i.e., multiple episodes, markedly elevated inflammatory markers, or extensive abnormalities at pericardial imaging) in whom treatment with glucocorticoids is unlikely to succeed."

Journal • Review • Cardiovascular • Immunology

Comparative efficacy and safety of colchicine and interleukin-1 antagonists in recurrent pericarditis: a network meta-analysis. (PubMed, Panminerva Med) - "IL-1 antagonists significantly reduce recurrent pericarditis episodes compared to colchicine, with anakinra, rilonacept, and goflikicept demonstrating high efficacy and acceptable safety profiles. These findings support their consideration as alternative therapies in colchicine-refractory cases of recurrent pericarditis. Further studies are warranted to refine treatment guidelines and optimize patient outcomes."

Journal • Retrospective data • Cardiovascular

Safety and Efficacy of RPH-104 Used to Prevent Recurrent Fever Attacks in Adult Patients With Colchicine Resistant or Colchicine Intolerant Familial Mediterranean Fever (clinicaltrials.gov) - P2 | N=60 | Recruiting | Sponsor: R-Pharm International, LLC | Trial completion date: Aug 2026 ➔ Jan 2029 | Trial primary completion date: Aug 2026 ➔ Dec 2028

Trial completion date • Trial primary completion date • Genetic Disorders

Interleukin-1 Blockade With RPH-104 (Goflikicept) in Patients With ST-Segment Elevation Myocardial Infarction: Secondary End Points From an International, Double-Blind, Randomized, Placebo-Controlled, Phase 2a Study. (PubMed, J Cardiovasc Pharmacol) - "In conclusion, in patients with STEMI, interleukin-1 blockade with goflikicept 80 mg or 160 mg was well tolerated and associated with significant reduction of systemic inflammation. Further adequately powered studies are warranted to determine whether the reduction in systemic inflammation with goflikicept translates into a clinical benefit in patients with STEMI."

Biomarker • Clinical • Journal • P2a data • Cardiovascular • Congestive Heart Failure • Heart Failure • Inflammation • Myocardial Infarction

Efficacy and Safety of RPH-104 for Resolution and Prevention of Recurring Attacks in Adult Subjects With Familial Mediterranean Fever With Resistance to or Intolerance of Colchicine (clinicaltrials.gov) - P2 | N=84 | Active, not recruiting | Sponsor: R-Pharm International, LLC | Recruiting ➔ Active, not recruiting | N=60 ➔ 84 | Trial completion date: Oct 2024 ➔ Oct 2025 | Trial primary completion date: Aug 2024 ➔ Jan 2025

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Genetic Disorders • CRP

Efficacy and Safety of RPH-104 Treatment in Patients With Recurrent Pericarditis (clinicaltrials.gov) - P2/3 | N=80 | Suspended | Sponsor: R-Pharm Overseas, Inc. | Trial completion date: Oct 2025 ➔ Oct 2027 | Not yet recruiting ➔ Suspended | Trial primary completion date: May 2025 ➔ May 2027

Trial completion date • Trial primary completion date • Trial suspension • Cardiovascular • CRP

Study of the Efficacy and Safety of RPH-104 in Adult Subjects With Schnitzler Syndrome (clinicaltrials.gov) - P2 | N=0 | Withdrawn | Sponsor: R-Pharm Overseas, Inc. | N=14 ➔ 0 | Not yet recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal • Dermatology • Immunology • Urticaria • Vasculitis • CRP • IL1B • IL1R1 • IL6

Long-term goflkicept therapy for patients with idiopathic recurrent pericarditis: results of the interim analysis of an ongoing open-label extension study (PubMed, Ter Arkh) - P2/3, P3 | "Long-term goflkicept therapy resulted in a significant reduction in the risk of recurrence and prolonged remission without an increase in adverse events."

Journal • Cardiovascular • Infectious Disease • Pain • CRP

Interleukin-1 blockade with RPH-104 (goflikicept) in patients with ST-segment elevation myocardial infarction (STEMI): secondary endpoints from an international, double blind, randomized, placebo-controlled, phase IIa study. (PubMed, J Cardiovasc Pharmacol) - "In conclusion, in patients with STEMI, IL-1 blockade with goflikicept 80 mg or 160 mg was well tolerated and associated with significant reduction of systemic inflammation. Further adequately powered studies are warranted to determine whether the reduction in systemic inflammation with goflikicept translates into a clinical benefit in patients with STEMI."

Journal • P2a data • Cardiovascular • Congestive Heart Failure • Heart Failure • Inflammation • Myocardial Infarction • CRP

Results of International, Double-Blind, Randomized, Placebo-Controlled, Phase IIa Study of Interleukin-1 Blockade With RPH-104 (Goflikicept) in Patients With ST-Segment-Elevation Myocardial Infarction (STEMI). (PubMed, Circulation) - No abstract available

Clinical • Journal • P2a data • Cardiovascular • Inflammation • Myocardial Infarction

Pericardial Diseases: International Position Statement on New Concepts and Advances in Multimodality Cardiac Imaging. (PubMed, JACC Cardiovasc Imaging) - "Recent clinical trials involving anti-IL-1 agents in recurrent pericarditis, including anakinra (AIRTRIP), rilonacept (RHAPSODY), and goflikicept have demonstrated their efficacy. The present document represents an international position statement from world leaders in the pericardial field, focusing on novel concepts and emphasizing the role of multimodality cardiac imaging as well as new therapeutics in pericardial diseases."

Clinical guideline • Journal • Review • Cardiovascular

Goflikicept and Related IL-1 Inhibitors in the Treatment of Recurrent Pericarditis. (PubMed, Cardiol Rev) - "Several studies have proved the efficacy and safety of interleukin-1 (IL-1) inhibitors, such as anakinra and rilonacept in patients with recurrent pericarditis. In patients with recurrences, the mean time to flare was 28.4 days in the placebo group versus 76.5 days in the anakinra group. IL-1 inhibitors require further research and have the potential to decrease the use of first-line drug regimens for recurrent pericarditis that are not tolerated in specific patient groups."

Journal • Cardiovascular • IL1A

GENE EXPRESSION ANALYSIS AND BIOMARKERS OF INFLAMMATION DURING GOFLIKICEPT THERAPY FOR IDIOPATHIC RECURRENT PERICARDITIS: PHASE II/III STUDY RESULTS (ACC 2024) - P2/3 | "IL-1 pathway plays important role in pathogenesis of IRP, IL-1 blocker affects the production of the IL-1, IL-6, IL-10, IL-12, IL-8 cytokines."

Biomarker • P2/3 data • Cardiovascular • Inflammation • CXCL8 • IL10 • IL12A • IL1B • IL6

Interleukin-1 Blockade With RPH-104 (Goflikicept) in Patients With ST-Segment Elevation Myocardial Infarction (STEMI) (AHA 2023) - "Conclusions In patients with STEMI, IL-1 blockade with RPH-104 80 mg or 160 mg significantly reduced CRP compared with placebo, and treatment with RPH-104 appears safe. Additional studies are needed to determine whether reduction in systemic inflammation with RPH-104 translates into a clinical benefit in patients with STEMI."

Clinical • Cardiovascular • Congestive Heart Failure • Heart Failure • Immunology • Inflammation • Myocardial Infarction • Pain • Systemic Inflammatory Response Syndrome • IL1A

Study of the Safety and Efficacy of RPH-104 in Preventing Recurrences in Patients With Idiopathic Recurrent Pericarditis (clinicaltrials.gov) - P3 | N=17 | Active, not recruiting | Sponsor: R-Pharm International, LLC | Trial completion date: Aug 2024 ➔ Jan 2026 | Trial primary completion date: Aug 2024 ➔ Jan 2026

Trial completion date • Trial primary completion date • Cardiovascular • CRP

Efficacy and Safety Clinical Study of RPH-104 in Adult Onset Still's Disease (AOSD) (clinicaltrials.gov) - P3 | N=0 | Withdrawn | Sponsor: R-Pharm International, LLC | N=56 ➔ 0 | Trial completion date: Dec 2024 ➔ Aug 2025 | Not yet recruiting ➔ Withdrawn | Trial primary completion date: Dec 2024 ➔ Aug 2025

Enrollment change • Trial completion date • Trial primary completion date • Trial withdrawal • Immunology • CRP

Paradigm Shift in Diagnosis and Targeted Therapy in Recurrent Pericarditis. (PubMed, Curr Cardiol Rep) - "With increased understanding of the autoinflammatory pathophysiology of recurrent pericarditis, IL-1 inhibitors including anakinra, canakinumab, and rilonacept have been applied to this condition with great promise...The next IL-1 inhibitor on the block for pericarditis, goflikicept, is also discussed. Understanding the role of the inflammasome via the autoinflammatory pathway in pericarditis has led to incorporation of IL-1 inhibitors in the treatment of recurrent pericarditis, with proven efficacy and safety and randomized trials. This will lead to increase uptake of this agent which demonstrated lower rates of recurrence and faster time to resolution."

Journal • Review • Cardiovascular • Inflammation • Pain

Treatment of Idiopathic Recurrent Pericarditis With Goflikicept: Phase II/III Study Results. (PubMed, J Am Coll Cardiol) - P2/3 | "Treatment with goflikicept prevented recurrences and maintained IRP remission with a favorable risk-benefit ratio. Goflikicept reduced the risk of recurrence compared with placebo. (Study to Evaluate the Efficacy and Safety of RPH-104 Treatment in Patients With Idiopathic Recurrent Pericarditis; NCT04692766)."

Journal • P2/3 data • P3 data • Cardiovascular • Inflammation • Pain • CRP • IL1B

Safety and Efficacy of RPH-104 Used to Prevent Recurrent Fever Attacks in Adult Patients With Colchicine Resistant or Colchicine Intolerant Familial Mediterranean Fever (clinicaltrials.gov) - P2 | N=60 | Recruiting | Sponsor: R-Pharm International, LLC | Trial completion date: Jan 2023 ➔ Aug 2026 | Trial primary completion date: Jan 2023 ➔ Aug 2026

Trial completion date • Trial primary completion date • Genetic Disorders • CRP

Efficacy and Safety of RPH-104 for Resolution and Prevention of Recurring Attacks in Adult Subjects With Familial Mediterranean Fever With Resistance to or Intolerance of Colchicine (clinicaltrials.gov) - P2 | N=60 | Recruiting | Sponsor: R-Pharm International, LLC | Trial completion date: Mar 2023 ➔ Oct 2024 | Trial primary completion date: Jan 2023 ➔ Aug 2024

Trial completion date • Trial primary completion date • Genetic Disorders • CRP

Study to Evaluate the Effect on Parameters of Systemic Inflammation and Disease Outcomes and Safety of RPH-104 in Subjects With Acute ST-elevation Myocardial Infarction (clinicaltrials.gov) - P2 | N=102 | Completed | Sponsor: R-Pharm Overseas, Inc. | Active, not recruiting ➔ Completed

Trial completion • Cardiovascular • Immunology • Inflammation • Myocardial Infarction • CRP

Study of the Safety and Efficacy of RPH-104 in Preventing Recurrences in Patients With Idiopathic Recurrent Pericarditis (clinicaltrials.gov) - P3 | N=17 | Active, not recruiting | Sponsor: R-Pharm International, LLC

New P3 trial • Cardiovascular • CRP

Study of the Efficacy and Safety of RPH-104 in Adult Subjects With Schnitzler Syndrome (clinicaltrials.gov) - P2 | N=14 | Not yet recruiting | Sponsor: R-Pharm Overseas, Inc. | Trial completion date: Jan 2021 ➔ Jan 2024 | Trial primary completion date: Aug 2020 ➔ Aug 2023

Trial completion date • Trial primary completion date • Dermatology • Immunology • Urticaria • Vasculitis • CRP

Affinity Capture Elution (ACE) ELISA Method Development and Validation for Novel RPH-104 Drug Immunogenicity Evaluation. (PubMed, Biomedicines) - "Method specificity was confirmed in the presence of the interfering compounds, namely IL-1α, IL-1β, and IL1-Ra. The developed and validated ELISA method was successfully applied to subject samples."

Journal • IL1B • IL1R1

Study to Evaluate the Efficacy and Safety of RPH-104 Treatment in Patients With Idiopathic Recurrent Pericarditis (clinicaltrials.gov) - P2/3 | N=20 | Completed | Sponsor: R-Pharm International, LLC | Recruiting ➔ Completed | Trial completion date: Jun 2022 ➔ Mar 2022

Trial completion • Trial completion date • Cardiovascular • CRP