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
NCT00609544 ↗	Study of the Safety and Effectiveness of Rilonacept (IL-1 Trap) Administered Subcutaneously in Subjects With Non Dialysis-Dependent Chronic Kidney Disease (NDD-CKD) and Anemia	Withdrawn	INC Research	Phase 2	1969-12-31	Anemia of inflammation is a common complication in patients with chronic kidney disease (CKD). Patients with CKD and anemia of inflammation also exhibit decreased response to erythropoietic agents, even in the presence of adequate iron stores. This decreased responsiveness is associated with increased levels of proinflammatory cytokines. Rilonacept is being developed for the treatment of inflammatory disorders. This is a clinical research study to determine the safety and effectiveness of rilonacept for the treatment of anemia in inflamed patients with CKD who are not yet on dialysis.
NCT00609544 ↗	Study of the Safety and Effectiveness of Rilonacept (IL-1 Trap) Administered Subcutaneously in Subjects With Non Dialysis-Dependent Chronic Kidney Disease (NDD-CKD) and Anemia	Withdrawn	Syneos Health	Phase 2	1969-12-31	Anemia of inflammation is a common complication in patients with chronic kidney disease (CKD). Patients with CKD and anemia of inflammation also exhibit decreased response to erythropoietic agents, even in the presence of adequate iron stores. This decreased responsiveness is associated with increased levels of proinflammatory cytokines. Rilonacept is being developed for the treatment of inflammatory disorders. This is a clinical research study to determine the safety and effectiveness of rilonacept for the treatment of anemia in inflamed patients with CKD who are not yet on dialysis.
NCT00609544 ↗	Study of the Safety and Effectiveness of Rilonacept (IL-1 Trap) Administered Subcutaneously in Subjects With Non Dialysis-Dependent Chronic Kidney Disease (NDD-CKD) and Anemia	Withdrawn	Regeneron Pharmaceuticals	Phase 2	1969-12-31	Anemia of inflammation is a common complication in patients with chronic kidney disease (CKD). Patients with CKD and anemia of inflammation also exhibit decreased response to erythropoietic agents, even in the presence of adequate iron stores. This decreased responsiveness is associated with increased levels of proinflammatory cytokines. Rilonacept is being developed for the treatment of inflammatory disorders. This is a clinical research study to determine the safety and effectiveness of rilonacept for the treatment of anemia in inflamed patients with CKD who are not yet on dialysis.
NCT00645840 ↗	Anti-inflammatory Therapy With Anakinra in Newly Diagnosed Type 1 Diabetes	Completed	Children's Medical Center Dallas	Phase 1/Phase 2	2008-03-01	The purpose of this study is to determine whether control of inflammatory pathways mediated by IL-1 beta using the IL-1 receptor antagonist anakinra will yield measurable decreases in expression of genes that are otherwise overexpressed as a consequence of IL-1 beta effects in children with newly diagnosed type 1 diabetes. Ultimately, we believe that control of IL-1 beta pathways will be associated with preserved insulin secretory capacity.

Clinical Trial Conditions for rilonacept

Condition Name

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

Condition MeSH

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

Clinical Trial Locations for rilonacept

Trials by Country

Trials by Country for rilonacept
Location	Trials
United States	153
Canada	7
Germany	4
Italy	3
Taiwan	2

Trials by US State

Trials by US State for rilonacept
Location	Trials
Texas	9
California	9
Ohio	8
New York	8
Maryland	7

Clinical Trial Progress for rilonacept

Clinical Trial Phase

Clinical Trial Phase for rilonacept
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	6
Phase 2	11

Clinical Trial Status

Clinical Trial Status for rilonacept
Clinical Trial Phase	Trials
Completed	21
Withdrawn	2
Terminated	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	21
Industry	15
NIH	5

Rilonacept Market Analysis and Financial Projection

Rilonacept: Clinical Trials, Market Analysis, and Projections

Introduction to Rilonacept

Rilonacept, marketed as Arcalyst, is a biologic medication that functions as an interleukin-1 (IL-1) blocker. It is designed to treat conditions associated with inflammation, particularly in patients with cryopyrin-associated periodic syndromes (CAPS) and other autoinflammatory diseases. Here, we will delve into the recent clinical trials, market analysis, and future projections for rilonacept.

Clinical Trials Update

RHAPSODY Trial

One of the pivotal clinical trials for rilonacept is the RHAPSODY trial, which focused on its efficacy and safety in patients with recurrent pericarditis. Here are the key findings:

• Efficacy: The trial demonstrated that rilonacept significantly improved health-related quality of life, sleep, pain, and global symptom severity in patients with recurrent pericarditis. During the 12-week run-in period, all patient-reported outcome (PRO) scores improved significantly (P < 0.001)[1][4][5].
• Recurrence Prevention: The randomized withdrawal period showed that rilonacept recipients experienced a 96% reduction in the risk of recurrent pericarditis events. The median time to pericarditis recurrence for placebo recipients was 8.6 weeks, while it could not be estimated for rilonacept recipients due to the low number of recurrences[4].
• Symptom Management: Rilonacept maintained clinical response and minimized pericarditis symptoms in a significant proportion of patients. For instance, 81% of rilonacept recipients had absent or minimal pericarditis symptoms at Week 16 of the randomized withdrawal period, compared to 25% of placebo recipients[4].

Transition to Monotherapy

The RHAPSODY trial also explored the feasibility of transitioning patients from oral therapies to rilonacept monotherapy. The study found that rapid discontinuation of oral pericarditis therapies while transitioning to rilonacept monotherapy was feasible without triggering pericarditis recurrence. Investigators successfully transitioned patients receiving multidrug regimens to rilonacept monotherapy using either a sequential or concurrent approach[5].

Market Analysis

Current Market Trends

The Arcalyst (rilonacept) market is experiencing significant growth driven by several factors:

• Increasing Prevalence: The rising awareness and diagnosed prevalence of rare inflammatory diseases like CAPS are contributing to the market growth[2].
• Innovations in Biologic Therapies: Advances in biologic therapies have expanded treatment options, further driving market demand. The unique mechanism of rilonacept as an IL-1 blocker offers a competitive edge over traditional treatments[2].
• Personalized Medicine: The increasing adoption of personalized medicine approaches and the use of biologics, which are often favored for their targeted therapeutic effects, are also boosting the market[2].

Market Players

Key players in the Arcalyst market include:

• Kiniksa Pharmaceuticals: The developer of Arcalyst, Kiniksa has seen substantial market growth driven by the increasing prevalence of CAPS and growing awareness among healthcare professionals. Strategic partnerships and investments in research and development are expected to enhance its market position[2].
• Novartis: With a diverse portfolio in immunology and inflammation management, Novartis provides competition to Kiniksa. Their significant financial resources enable robust investment in innovative therapies[2].
• Regeneron Pharmaceuticals: Known for its wealth of monoclonal antibody therapies, Regeneron is also involved in the inflammatory disease space, positioning it favorably in the competitive landscape[2].

Market Projections

Growth Rate

The Arcalyst market is projected to grow at a compounded annual growth rate (CAGR) of approximately 4.2% during the forecast period from 2024 to 2031. This growth is driven by rising awareness of rare inflammatory diseases, expanded indication approvals, and ongoing research exploring additional therapeutic areas[2].

Market Size

The global market for autoinflammatory therapies, which includes Arcalyst, is expected to reach $5 billion by 2026. The market for rilonacept specifically is anticipated to capture significant share, particularly as patient access programs expand and healthcare systems prioritize precision medicine[2].

Emerging Trends

• Expanded Indications: Ongoing research focused on expanding Arcalyst's indications to include other inflammatory disorders is expected to enhance its market potential[2].
• Patient-Centric Care: A growing emphasis on patient-centric care is leading to improved adherence to treatment regimes, further driving market growth[2].

Key Takeaways

• Clinical Efficacy: Rilonacept has demonstrated significant efficacy in reducing pericarditis recurrences and improving patient-reported outcomes.
• Market Growth: The Arcalyst market is expected to grow at a CAGR of 4.2% from 2024 to 2031, driven by increasing awareness and prevalence of rare inflammatory diseases.
• Competitive Landscape: Key players like Kiniksa Pharmaceuticals, Novartis, and Regeneron Pharmaceuticals are driving innovation and competition in the market.
• Future Outlook: The market is poised for further growth as research expands the drug's indications and patient access programs improve.

FAQs

What is rilonacept used for?

Rilonacept is used to treat conditions associated with inflammation, particularly cryopyrin-associated periodic syndromes (CAPS) and recurrent pericarditis.

How does rilonacept work?

Rilonacept functions as an interleukin-1 (IL-1) blocker, providing relief from symptoms associated with autoinflammatory conditions.

What were the key findings of the RHAPSODY trial?

The RHAPSODY trial showed that rilonacept significantly improved health-related quality of life, sleep, pain, and global symptom severity in patients with recurrent pericarditis, and reduced the risk of pericarditis recurrences by 96%.

Who are the major players in the Arcalyst market?

The major players include Kiniksa Pharmaceuticals, Novartis, and Regeneron Pharmaceuticals.

What is the projected growth rate of the Arcalyst market?

The Arcalyst market is expected to grow at a CAGR of approximately 4.2% from 2024 to 2031.

What emerging trends are driving the Arcalyst market?

Emerging trends include the increased adoption of personalized medicine approaches, the use of biologics, and a growing emphasis on patient-centric care.

Sources

1. PubMed: Results From the Phase 3 Clinical Trial RHAPSODY - PubMed
2. GitHub: Market-Research-Report-List-1/arcalyst-market.md at main - GitHub
3. Market Research Reports: Rilonacept Drugs - Global Market Share and Ranking, Overall Sales and Demand Forecast 2024-2030
4. Kiniksa Pharmaceuticals: RHAPSODY Phase 3 Results Every Second Counts™
5. BMJ Heart: Transition to rilonacept monotherapy from oral therapies in patients with recurrent pericarditis[5].