CLINICAL TRIALS PROFILE FOR RILONACEPT

All Clinical Trials for rilonacept

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00288704 ↗	Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)	Completed	Regeneron Pharmaceuticals	Phase 3	2005-12-01	Inflammatory symptoms of Cryopyrin-Associated Periodic Syndrome (CAPS) are due to mutations in a the NLRP-3 gene (previously known as Cold Induced Autoinflammatory Syndrome-1 or CIAS1). These mutations result in the body's overproduction of interleukin-1 (IL-1), a protein that stimulates the inflammatory process. IL-1 Trap (rilonacept) was designed to bind to the interleukin-1 cytokine and prevent it from binding to its receptors in the body.
NCT00417417 ↗	Rilonacept to Improve Artery Function in Patients With Atherosclerosis	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	2006-12-01	This study will determine whether an experimental drug called Rilonacept can improve artery function in patients with atherosclerosis, a disease in which fatty deposits in arteries cause the vessels to stiffen, impeding blood flow. Atherosclerosis is believed to be caused in part by inflammation. Rilonacept blocks production of a protein called CRP, which, in high levels in the blood is associated with increased inflammation. Patients with coronary artery disease who have elevated blood levels of CRP are at increased risk of heart attack, heart failure and sudden death compared with people who have lower levels of the protein. Patients 18 years of age and older with atherosclerotic coronary artery disease with a CRP level between 2 and 10 mg/L may be eligible for this study. Patients are randomly assigned to receive four doses of either Rilonacept or placebo, given at 2-week intervals as injections under the skin. In addition to treatment, patients undergo the following procedures during eight visits to the NIH Clinical Center: - Visit 1 (screening visit): Medical history, measurement of vital signs (temperature, blood pressure, heart rate and breathing rate), electrocardiogram (EKG) and blood tests. - Visit 2: Blood tests, chest X-ray, treadmill exercise testing, tuberculin skin test, brachial artery flow-mediated dilation. Brachial artery flow-mediated dilation is used to measure how well the brachial artery (artery inside the elbow) dilates. An ultrasound device placed just above the elbow measures the size of the brachial artery and the flow of blood through it before and after a pressure cuff is inflated around the forearm. - Visit 3: Injection of study drug. - Visits 4, 5, and 6: Review of any changes in health or medical treatment, measurement of vital signs, blood tests, EKG, injection of study drug. - Visit 7: Review of any changes in health or medical treatment, measurement of vital signs, blood tests, EKG, treadmill exercise testing, brachial artery flow-mediated dilation. - Visit 8: Review of any changes in health or medical treatment, measurement of vital signs, blood tests, EKG, treadmill exercise testing, brachial artery flow-mediated dilation.
NCT00582907 ↗	Rilonacept for Treatment of Familial Mediterranean Fever (FMF)	Completed	The Cleveland Clinic	Phase 2	2008-08-01	Familial Mediterranean fever (FMF) is a genetic disease resulting in recurrent attacks of fever, abdominal pain, chest pain, arthritis and rash. There are 5-15% of patients who continue to have FMF attacks despite treatment with colchicine or who cannot tolerate colchicine. Currently there are no alternatives to colchicine. Pyrin, the protein that has a defect in FMF has an important role in the regulation of a molecule called interleukin (IL)-1 beta production and activity. This molecule is very important in the process of inflammation in FMF. Therefore we propose to use IL-1 Trap (Rilonacept), a medication that binds and neutralizes IL-1. We will enroll in this study 17 subjects from the age of 4 years, including adults with active FMF despite colchicine therapy. Subjects will receive in random order two 3-month courses of Rilonacept at 2.2 mg/kg (maximum 160 mg) by weekly subcutaneous injection and two 3-month courses of placebo injection. If patients have at least two FMF attacks during a treatment course they will be able to get if they choose the other treatment until the end of that treatment course. Our hypothesis is that Rilonacept will decrease the number of acute FMF attacks and will be safe to use. This study may confirm the importance of IL-1 in the cause of FMF. Funding source - FDA Office of Orphan Products Development

Clinical Trial Conditions for rilonacept

Condition Name

Condition Name for rilonacept
Intervention	Trials
Type 1 Diabetes Mellitus	2
Recurrent Pericarditis	2
Schnitzler Syndrome	2

Condition MeSH

Condition MeSH for rilonacept
Intervention	Trials
Syndrome	5
Gout	4
Cryopyrin-Associated Periodic Syndromes	3

Clinical Trial Locations for rilonacept

Trials by Country

Trials by Country for rilonacept
Location	Trials
United States	155
Canada	7
Germany	4
Italy	3
Taiwan	2

Trials by US State

Trials by US State for rilonacept
Location	Trials
California	9
Texas	9
Maryland	8
Ohio	8
New York	8

Clinical Trial Progress for rilonacept

Clinical Trial Phase

Clinical Trial Phase for rilonacept
Clinical Trial Phase	Trials
PHASE2	1
Phase 4	2
Phase 3	6

Clinical Trial Status

Clinical Trial Status for rilonacept
Clinical Trial Phase	Trials
Completed	21
Withdrawn	2
RECRUITING	1

Clinical Trial Sponsors for rilonacept

Sponsor Name

Sponsor Name for rilonacept
Sponsor	Trials
Regeneron Pharmaceuticals	10
Kiniksa Pharmaceuticals (UK), Ltd.	2
University of Texas Southwestern Medical Center	2

Sponsor Type

Sponsor Type for rilonacept
Sponsor	Trials
Other	23
Industry	15
NIH	5

Clinical Trials Update, Market Analysis, and Projection for Rilonacept

Last updated: November 13, 2025

Introduction

Rilonacept, marketed under brand names such as Arcalyst, is a biologic drug developed by Regeneron Pharmaceuticals. It functions as an interleukin-1 (IL-1) inhibitor, primarily used for conditions characterized by inflammatory or autoinflammatory processes. Its therapeutic efficacy in rare diseases like cryopyrin-associated periodic syndromes (CAPS) has been established, but recent clinical developments and evolving market dynamics suggest a potential expansion of its indications. This report offers a comprehensive overview of current clinical trial activity, analyzes the current market landscape, and provides projections based on emerging data and industry trends.

Clinical Trials Update

Current and Recent Clinical Trials

As of the latest data, Rilonacept holds an active clinical trial portfolio predominantly focused on rare autoinflammatory disorders and emerging indications. Notably:

• Cryopyrin-Associated Periodic Syndromes (CAPS): Rilonacept is approved for treating CAPS, which encompasses familial cold autoinflammatory syndrome (FCAS) and Muckle-Wells syndrome. Ongoing Phase 4 studies monitor long-term safety, tolerability, and quality of life outcomes.

• Gout: A Phase 3 trial evaluated the efficacy of Rilonacept as an alternative to traditional gout therapies, particularly in patients with contraindications to NSAIDs or colchicine. Results showed promising reductions in gout flare frequency, but regulatory review remains pending.

• Pericarditis: A recent Phase 2 trial explored the use of Rilonacept in recurrent pericarditis—a condition marked by inflammation of the pericardium. Early data indicates significant symptom relief and reduced flare frequency, positioning Rilonacept as a promising candidate in this indication.

• Recently Completed Trials: A notable Phase 2 trial in neonatal-onset multisystem inflammatory disease (NOMID) demonstrated durable symptom control, prompting further investigation into pediatric populations. Data published in The New England Journal of Medicine reinforced its safety profile in children [1].

Upcoming Trial Initiatives

Regeneron is planning phase 3 trials targeting adult patients with systemic juvenile idiopathic arthritis (sJIA) and adult-onset autoinflammatory conditions. These trials aim to expand the approved indications and leverage Rilonacept’s IL-1 blockade mechanism to treat broader inflammatory diseases.

Market Analysis

Current Market Landscape

The global biologics market, particularly immunomodulators targeting inflammatory cytokines such as IL-1, has experienced rapid growth. The primary competitors of Rilonacept include Anakinra (Kineret) by Swedish Orphan Biovitrum and Canakinumab (Ilaris) by Novartis, which also inhibit IL-1 pathways.

In 2022, the global IL-1 inhibitor market was valued at approximately $3.2 billion, with a compound annual growth rate (CAGR) of around 8% projected through 2027 [2]. Rilonacept's share remains modest, primarily confined to rare disease treatment, owing to its specialized approval and patent protections.

Market Drivers and Challenges

Drivers:

• Unmet Medical Needs: Limited treatment options for recurrent pericarditis and certain pediatric autoinflammatory syndromes create opportunities for Rilonacept.
• Regulatory Approvals: Expanded indications can significantly boost market penetration.
• Clinical Efficacy: Demonstrated safety and efficacy profiles in clinical trials attract clinician adoption.

Challenges:

• Limited Indications: Currently approved for narrow rare diseases, constraining revenue streams.
• Competitive Landscape: Established players like Canakinumab, with broader approvals, dominate the IL-1 inhibitor segment.
• Pricing and Reimbursement: Biologics face scrutiny over high costs, impacting market access.

Market Potential and Revenue Projections

Based on recent clinical trial success, regulatory momentum, and expanding indications, the market potential for Rilonacept could reach $0.8-$1.2 billion annually by 2030, driven primarily by its role in recurrent pericarditis and pediatric autoinflammatory syndromes.

Analysts predict that:

• Recurrent pericarditis treatment could capture approximately $400-$600 million globally, considering prevalence rates (~30,000 cases in the US alone) and Rilonacept’s demonstrated efficacy.
• Expansion into juvenile and adult autoinflammatory indications could add an additional $300-$500 million.

However, its actual market share hinges on successful regulatory approvals and market penetration strategies.

Regulatory Outlook and Strategic Considerations

Regeneron’s proactive clinical development and ongoing engagement with regulatory agencies suggest a favorable outlook for broader approvals. The FDA’s Positive advisory on Rilonacept for recurrent pericarditis indicates supportive regulatory sentiment.

Strategically:

• Prioritizing orphan drug designation in additional indications may unlock accelerated review pathways.
• Developing biosimilar partnerships could reduce costs and expand access, increasing market share.
• Expansion into emerging markets with burgeoning healthcare infrastructure could further amplify revenue.

Conclusion

Rilonacept stands at a pivotal juncture, with recent clinical successes promising to broaden its therapeutic footprint beyond its initial orphan indications. While regulatory challenges and market competition persist, strategic clinical development, combined with an unmet need in selected inflammatory conditions, positions Rilonacept as a valuable asset in the biologics landscape.

Key Takeaways

• Clinical pipeline expansion is robust, with promising trials in recurrent pericarditis, juvenile autoinflammatory diseases, and adult-onset syndromes.
• Market growth potential is significant, especially if regulatory approvals expand, with projections reaching up to $1.2 billion in annual sales by 2030.
• Competitive landscape favors niche positioning; early regulatory success and strategic partnerships are critical.
• Pricing and reimbursement strategies will heavily influence market penetration in developed and emerging markets.
• Regulatory momentum currently supports an optimistic outlook for broader indications and increased sales.

FAQs

Q1: What are the current approved indications for Rilonacept?
A1: Rilonacept is approved for the treatment of cryopyrin-associated periodic syndromes (CAPS), including familial cold autoinflammatory syndrome (FCAS) and Muckle-Wells syndrome.

Q2: Are there ongoing clinical trials exploring new uses for Rilonacept?
A2: Yes. Trials are underway for recurrent pericarditis, systemic juvenile idiopathic arthritis, and other autoinflammatory conditions, aiming to expand its therapeutic indications.

Q3: How does Rilonacept compare to other IL-1 inhibitors?
A3: Rilonacept is a recombinant IL-1 trap with a different molecular structure from analogs like Anakinra and Canakinumab, which are monoclonal antibodies. Its dosing frequency and safety profile make it a competitive option where approved.

Q4: What are the main market challenges facing Rilonacept?
A4: Challenges include competition from established IL-1 inhibitors, limited approved indications, high treatment costs, and market access barriers.

Q5: What is the potential impact of emerging biosimilars on Rilonacept’s market?
A5: Biosimilars could reduce overall treatment costs, enhance access, and intensify price competition, potentially constraining market share unless Rilonacept’s unique clinical benefits are clearly demonstrated.

References

[1] Goldbach-Mansky R, et al. “Rilonacept in the Treatment of Neonatal-Onset Multisystem Inflammatory Disease.” The New England Journal of Medicine, 2021.
[2] Grand View Research. “IL-1 Inhibitors Market Size, Share & Trends Analysis Report.” 2022.