Arcerix (goflikicept) / R-Pharm - LARVOL DELTA

Efficacy and safety of goflikicept, an IL-1β and IL-1α inhibitor, in familial Mediterranean fever: an international randomized clinical trial (ACR Convergence 2026) - "Background/Purpose: To address the need for targeted therapies in familial Mediterranean fever (FMF), this trial examined the safety and efficacy of goflikicept (GFC; RPH-104), a novel interleukin (IL)-1-inhibiting fusion protein designed to neutralize IL-1-driven inflammation. In this international, multicenter, double-blind phase 2 trial, adults with colchicine-resistant or intolerant FMF and active attacks were randomized to receive either subcutaneous GFC or placebo. Treatment with GFC significantly decreased disease activity versus placebo in colchicine-resistant/intolerant FMF patients, with a safety profile aligning with established IL-1 inhibitors."

Clinical • Genetic Disorders • Inflammation • CRP • IL1B

Single-cell RNA profiling suggests goflikicept-mediated immune modulation in idiopathic recurrent pericarditis. (PubMed, Inflamm Res) - "Goflikicept effectively normalized dysregulated immune responses in IRP, supporting the broader therapeutic potential of IL-1 blockade in NLRP3-mediated inflammatory diseases. This study provides the first single-cell resolution insights into the molecular mechanisms of IL-1 blockade, informing the development of targeted therapies for autoinflammatory conditions."

Journal • Cardiovascular • Immune Modulation • Immunology • Inflammation • IL1B • NLRP3

Study of the Efficacy and Safety of Goflikicept and Olokizumab as the Second-line Therapy in Patients With Still's Disease (clinicaltrials.gov) - P3 | N=52 | Not yet recruiting | Sponsor: R-Pharm International, LLC

New P3 trial • Immunology

Updates on Pathophysiology of Pericarditis to Guide Development of Therapeutics. (PubMed, Curr Cardiol Rep) - "This evidence supports the central role of the NLRP3 inflammasome/IL-1β axis in driving acute inflammation of the pericardium and subsequent flares, as indirectly highlighted in seminal clinical trials of anakinra (AIRTRIP trial), rilonacept (RHAPSODY trial), and goflikicept. Accordingly, a shift moving from the prescription of glucocorticoids as second-line therapy in favor of IL-1 blockers, particularly in those patients presenting with an auto-inflammatory phenotype, is ongoing. To this end, an imaging-guided approach may help choosing the best treatment and monitoring its effects across time, thus allowing a personalized approach to patients with recurrent pericarditis."

Journal • Review • Cardiovascular • Inflammation • IL1B • NLRP3

Efficacy of interleukin-1 blockade for prevention of heart failure outcomes in patients with prior myocardial Infarction: a systematic review and meta-analysis of 10301 patients (HEART FAILURE 2026) - "Three IL-1 inhibitors were evaluated: canakinumab, anakinra, and goflikicept. Conclusions Among patients with prior myocardial infarction, interleukin-1 blockade did not significantly reduce heart failure hospitalization, the composite of heart failure hospitalization or all-cause mortality, or all-cause mortality compared with placebo. While point estimates suggest a possible benefit, existing trial data are insufficient, and larger trials targeting post-MI patients are required to clarify the therapeutic role of IL-1 inhibition in heart failure prevention."

Retrospective data • Review • Cardiovascular • Congestive Heart Failure • Heart Failure • Myocardial Infarction

INTERLEUKIN-1 BLOCKERS IN ST-SEGMENT ELEVATION MYOCARDIAL INFARCTION: INDIRECT COMPARISON OF ANAKINRA AND GOFLIKICEPT (ACC 2026) - "We present an indirect comparison between two IL-1 blockers tested in phase II trials: anakinra, an IL-1 receptor antagonist, and goflikicept, a recombinant IL-1 trap. In STEMI, anakinra and goflikicept achieve a similar reduction in systemic inflammation and HF-related events, supporting IL-1 blockade as a class effect and promising strategy to improve outcomes."

Cardiovascular • Congestive Heart Failure • Heart Failure • Inflammation • Myocardial Infarction • CRP

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

Efficacy of Anti-inflammatory Therapies for Pericarditis: A Systematic Review and Network Meta-analysis (AHA 2025) - "Interestingly, there was not enough data to make a meaningful comparison between steroids and control.ConclusionIn our analysis, goflikicept, rilonacept, and anakinra were most effective at reducing recurrent pericarditis rates. Our analysis also corroborated evidence regarding the efficacy of colchicine in reducing pericarditis recurrence. For a comprehensive analysis of the benefits and harms of anti-inflammatory therapies, larger comparative studies or network meta-analyses of patient-level data are required."

Retrospective data • Review • Cardiovascular

Interleukin-1 Blockade in Patients With ST-Segment-Elevation Myocardial Infarction: a Pooled Analysis of Randomized Clinical Trials of Anakinra and Goflikicept (AHA 2025) - "Phase II trials of IL-1 inhibition with anakinra and goflikicept show inhibition of systemic inflammation, prevention of LV systolic dysfunction, and reduction of HF-related events at 1 year. Adequately powered phase III clinical trials are needed to validate and expand these findings."

Retrospective data • Cardiovascular • Congestive Heart Failure • Heart Failure • Inflammation • Myocardial Infarction • CRP

Interim Results of a Randomized Placebo Controlled Study of IL-1 Inhibitor Goflikicept in Patients With Familial Mediterranean Fever (ACR Convergence 2025) - "Background/Purpose: Goflikicept (GFC; RPH-104) is a novel fusion protein inhibiting interleukin-1 (IL-1). GFC provided a significant improvement in the disease activity of colchicine-refractory/intolerant FMF patients compared to placebo, and its safety profile was similar to other IL-1 inhibitors."

Clinical • Genetic Disorders • Inflammation • IL1B

Pharmacokinetics, Safety and Immunogenicity of RPH-104 at a New Dosage and Different Doses Via Single Subcutaneous and Intravenous Administration in Healthy Volunteers (clinicaltrials.gov) - P1 | N=130 | Completed | Sponsor: R-Pharm International, LLC

New P1 trial

Heart failure with preserved ejection fraction: how far we've come - a brief report from two outstanding educational events. (PubMed, J Cardiovasc Pharmacol) - No abstract available

Educational event • Journal • Cardiovascular • Congestive Heart Failure • Heart Failure • Inflammation • Myocardial Infarction

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