RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Overseas New Drugs Urgently Needed in Clinical Settings (China), Breakthrough Therapy (United States)

Structure/Sequence

Sequence Code 319372596

Source: *****

Clinical Trials associated with Rilonacept

NCT06660732

/ RecruitingPhase 2IIT

A RandomizEd PhAse II TrIal of Rilonacept in Subjects with Cardiac Sarcoidosis (REPAIR-CS)

The primary objective of this study is to evaluate the effect of rilonacept, added to standard therapy and compared with standard therapy alone, on improvement in myocardial inflammation in subjects with cardiac sarcoidosis after 24 weeks of therapy.

Start Date01 Mar 2025

Sponsor / Collaborator

Mayo Clinic

[+1]

NCT03737110

/ CompletedPhase 3

Phase 3, Double-Blind, Placebo-Controlled, Randomized Withdrawal Study With Open-label Extension, to Assess the Efficacy and Safety of Rilonacept Treatment in Subjects With Recurrent Pericarditis

The primary objective of this study was to assess the efficacy of rilonacept treatment in participants with recurrent pericarditis.

Start Date07 Jan 2019

Sponsor / Collaborator

Kiniksa Pharmaceuticals (UK) Ltd.

NCT03980522

/ CompletedPhase 2

An Open-Label Pilot Study of KPL-914 in Recurrent Pericarditis

The purpose of this study is to assess the preliminary efficacy and safety of KPL-914 treatment in participants with recurrent pericarditis.

Start Date24 Jan 2018

Sponsor / Collaborator

Kiniksa Pharmaceuticals (UK) Ltd.

100 Clinical Results associated with Rilonacept

100 Translational Medicine associated with Rilonacept

100 Patents (Medical) associated with Rilonacept

263

Literatures (Medical) associated with Rilonacept

01 Jan 2025·RHEUMATOLOGY

Efficacy and safety of biologic drugs in Still's disease: a systematic review and network meta-analysis of randomized controlled trials

Article

Author: Kilic, Berkay ; Hacioglu, Ervanur ; Ugurlu, Serdal ; Ozturk, Admir ; Karup, Sejla

Abstract:

Objectives:

Still’s disease is a rare autoinflammatory disorder characterized by systemic inflammation, fever, rash and arthritis. The term ‘Still’s disease’ covers the paediatric subtype systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still’s disease (AOSD), which affects adults. Biologic drugs, including the anti-IL-1 agents anakinra, canakinumab and rilonacept and the IL-6 antagonist tocilizumab, are used in the management of Still’s disease.

Methods:

We conducted a systematic review and network meta-analysis of randomized controlled trials, and the study protocol was registered in PROSPERO (CRD42023450442). MEDLINE, EMBASE and CENTRAL were screened from inception until 17 September 2023. We included patients with Still’s disease who received placebo or biologic drugs: anakinra, canakinumab, rilonacept or tocilizumab. The primary efficacy and safety outcomes were achievement of ACR50 response and occurrence of serious adverse events, respectively. The interventions were ranked using rankograms and SUCRA values.

Results:

Nine trials with 430 patients were included. All biologic drugs were associated with greater odds of ACR50 response compared with placebo. There was no statistically significant association between biologic drugs and serious adverse events. The multivariate meta-analysis found no difference between biologic drugs. As per SUCRA rankings, anakinra was the most effective and safe option with respect to ACR50 response and occurrence of serious adverse events.

Conclusion:

This is the first systematic review and meta-analysis to assess the efficacy and safety of biologic drugs in paediatric and adult patients with Still’s disease. Biologic drugs were effective in achieving ACR response and demonstrated a low adverse event profile in the management of Still’s disease.

01 Jan 2025·JACC. Advances

Assessing the Impact of Rilonacept on Lipid Profiles in Patients With Recurrent Pericarditis

Article

Author: Klein, Allan L ; Yesilyaprak, Abdullah ; Kumar, Ashwin ; Bayat, Aqieda ; Agrawal, Ankit ; Majid, Muhammad ; Verma, Beni

31 Dec 2024·European Heart Journal-Cardiovascular Imaging

Longitudinal cardiac magnetic resonance imaging following clinical response to rilonacept and prior to recurrence upon treatment suspension: a RHAPSODY subgroup analysis

Article

Author: Paolini, John F ; Jellis, Christine L ; Kwon, Deborah H ; Clair, JoAnn ; Luis, Sushil A ; Imazio, Massimo ; Wang, Sheldon ; Curtis, Allison ; Cremer, Paul C ; Insalaco, Antonella ; Lin, David ; Brucato, Antonio ; Klein, Allan L

Abstract:

Aims:

In the Phase 3 trial, RHAPSODY, rilonacept effectively resolved active pericarditis recurrences, and long-term treatment led to sustained pericarditis recurrence risk reduction. Prior analysis suggested association between higher late gadolinium enhancement (LGE) at baseline and more rapid recurrence upon rilonacept suspension after 12 weeks of treatment. This subgroup analysis assessed the utility of longitudinal serial cardiac magnetic resonance (CMR) imaging for tracking clinical improvement and predicting post-treatment cessation outcomes to help guide clinical decision-making.

Methods and results:

At an 18-month decision milestone (18MDM) in the RHAPSODY long-term extension, investigators decided if patients would continue rilonacept, suspend rilonacept for off-treatment observation, or discontinue the study. Pericardial thickness, pericardial oedema (T2-short tau inversion recovery, T2-STIR), and LGE were determined at baseline and 18MDM by an imaging core lab blinded to clinical data, and pericarditis recurrence was investigator-assessed. CMR results in patients with data at both baseline and 18MDM (n = 13) showed that pericardial thickness, T2-STIR, and LGE were reduced during rilonacept treatment. Among patients with CMR data who suspended rilonacept at the 18MDM (n = 7), five (71%) had a pericarditis recurrence within 1–4 months of rilonacept suspension, despite all having had none/trace LGE (n = 7) and negative T2-STIR (n = 7) at the 18MDM and two having received prophylactic colchicine.

Conclusion:

Continued clinical improvement during prolonged rilonacept treatment corresponded with improvement on CMR, including reduced pericardial thickness, resolution of pericardial oedema, and resolution of LGE. However, none/trace LGE at 18MDM while on treatment did not predict absence of pericarditis recurrence upon subsequent rilonacept suspension in this size-limited subgroup.

News (Medical) associated with Rilonacept

25 Feb 2025

·www.fiercebiotech.com

Kiniksa adds to Sjögren\'s exodus with trial termination, axes AstraZeneca asset

In the wake of its recent pipeline decisions, Kiniksa is evaluating strategic alternatives for abiprubart.\n Kiniksa Pharmaceuticals is stopping development of a midphase autoimmune asset and returning a drug candidate to AstraZeneca as it doubles down on its cardiovascular disease pipeline.The asset licensed from AstraZeneca, GM-CSF antagonist mavrilimumab, was already on the back-burner, but the termination of the midphase autoimmune program comes as more of a shock. Six weeks ago, Kiniksa chief medical officer John Paolini, M.D., told investors the company was “certainly very excited” about a phase 2b trial of abiprubart in Sjögren’s syndrome. Today, Kiniksa said it is stopping the study.Kiniksa, which is evaluating strategic alternatives for abiprubart, framed the decision to end the trial as a result of a “strategic reprioritization of its portfolio and certain capital allocation considerations.” The action will cost $33 million to $37 million, Kiniksa said, but will free the biotech from the cost of funding a study that was scheduled to run into 2027.Abiprubart, an antibody that inhibits the signaling between CD40 and CD154, was in development in a competitive niche but Kiniksa had previously presented that as a positive. Last month, Kiniksa CEO Sanj Patel told investors “there has been some external derisking of the mechanism through other assets,” but he said abiprubart was differentiated through its monthly subcutaneous dosing.Amgen’s CD40L antagonist fusion protein, dazodalibep, is ahead of abiprubart in the race to market, with the big biotech starting phase 3 trials in Sjögren’s last year. But that drug is given intravenously.The high-concentration liquid formulation of abiprubart opened up the option of subcutaneous dosing. The list of rivals that Kiniksa believed may be able to offer subcutaneous dosing has thinned in recent years. Novartis axed its rival subcutaneous anti-CD40 antibody in Sjögren’s last month after assessing the risks and benefits of the molecule. Sanofi reached a similar decision in April, when it crossed Sjögren’s off the list of potential indications for its anti-CD40L prospect. Bristol Myers Squibb terminated a phase 1 trial of its CD40 candidate in Sjögren’s in 2023 because of “insufficient enrollment.” Kiniksa disclosed the winddown of its Sjögren’s trial, which it expects to complete by the end of the year, alongside the news that it is terminating development of mavrilimumab. The biotech licensed the asset from AstraZeneca’s MedImmune in 2017, paying $23 million in upfront and subsequent payments. But by early last year, the company was evaluating potential partnership opportunities to advance the molecule.The hunt for a partner ended last week, when Kiniksa told MedImmune it was stopping development of the asset and terminating the license agreement. Kiniksa’s actions leave it focused on a cardiovascular disease pipeline led by the IL-1 antagonist KPL-387. The program is targeting recurrent pericarditis, the same indication as Kiniksa’s approved drug Arcalyst, and a phase 2/3 trial is set to start this year.

License out/inPhase 2Phase 3Clinical Trial Termination

25 Feb 2025

·investors.kiniksa.com

Kiniksa Pharmaceuticals Announces Development of KPL-387 in Recurrent Pericarditis and Updates Corporate Strategy

– Kiniksa plans to initiate a Phase 2/3 clinical trial of KPL-387 in recurrent pericarditis in mid-2025 – – KPL-387 Phase 1 single ascending dose data support potential monthly dosing – – Kiniksa continues to focus development on diseases with unmet need, prioritizing cardiovascular indications – – Kiniksa to discontinue abiprubart development in Sjögren’s Disease – LONDON, Feb. 25, 2025 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals International, plc (Nasdaq: KNSA) (Kiniksa), a biopharmaceutical company developing and commercializing novel therapies for diseases with unmet need, with a focus on cardiovascular indications, today announced the development program for KPL-387 in recurrent pericarditis and provided an update on its corporate strategy. KPL-387 is an independently developed monoclonal antibody that binds human interleukin-1 receptor 1 (IL-1R1), inhibiting the signaling activity of the cytokines interleukin-1α (IL-1α) and interleukin-1β (IL-1β). “Through the successful development and commercialization of ARCALYST, Kiniksa has helped thousands of patients suffering from recurrent pericarditis. Since the launch in 2021, we have generated over $800 million in product revenue and have become cash flow positive on an annual basis. We plan to further increase our penetration into the recurrent pericarditis population with ARCALYST,” said Sanj K. Patel, Chairman and Chief Executive Officer of Kiniksa. “We also continue to focus development on novel therapies for diseases with unmet need, prioritizing cardiovascular indications. Today, we are excited to extend our leadership in the recurrent pericarditis market through the development of KPL-387. We believe KPL-387 could expand the treatment options for recurrent pericarditis patients by providing a single monthly subcutaneous injection in a liquid formulation.” “KPL-387 could provide a meaningful addition to the therapeutic options available to patients suffering from recurrent pericarditis. Data generated from the single ascending dose portion of the ongoing Phase 1 study support our belief that KPL-387 could enable dosing with a single monthly subcutaneous injection in a liquid formulation,” said John F. Paolini, M.D., Ph.D., FACC, Chief Medical Officer of Kiniksa. “We have interacted with the FDA, and we plan to initiate a Phase 2/3 clinical trial of KPL-387 in recurrent pericarditis in mid-2025, with Phase 2 data expected in the second half of 2026.” Corporate Update Kiniksa continues to focus development on diseases with unmet need, prioritizing cardiovascular indications. Kiniksa is developing KPL-387, a fully human immunoglobulin G2 (IgG2) monoclonal antibody that binds IL-1R1, inhibiting the signaling of the cytokines IL-1α and IL-1β, in recurrent pericarditis with a target profile of monthly subcutaneous (SC) dosing. Kiniksa is conducting a single ascending dose (SAD) and multiple ascending dose Phase 1 clinical trial of KPL-387 in healthy volunteers. Topline data from the SAD portion of the Phase 1 trial of KPL-387 support potential monthly SC dosing in recurrent pericarditis. Kiniksa has interacted with the U.S. Food and Drug Administration (FDA) and expects to initiate a Phase 2/3 clinical trial of KPL-387 in recurrent pericarditis in mid-2025, with Phase 2 data expected in the second half of 2026. Kiniksa is advancing KPL-1161, an Fc-modified IgG2 monoclonal antibody that binds IL-1R1, inhibiting the signaling of the cytokines IL-1α and IL-1β, towards clinical development with a target profile of quarterly SC dosing. Kiniksa plans to discontinue the development of abiprubart in Sjögren’s Disease. The company will explore strategic alternatives for the asset. Kiniksa has exercised its right to terminate its exclusive license agreement for mavrilimumab with MedImmune. Kiniksa is conducting a single ascending dose (SAD) and multiple ascending dose Phase 1 clinical trial of KPL-387 in healthy volunteers. Topline data from the SAD portion of the Phase 1 trial of KPL-387 support potential monthly SC dosing in recurrent pericarditis. Kiniksa has interacted with the U.S. Food and Drug Administration (FDA) and expects to initiate a Phase 2/3 clinical trial of KPL-387 in recurrent pericarditis in mid-2025, with Phase 2 data expected in the second half of 2026. Topline data from the SAD portion of the Phase 1 trial of KPL-387 support potential monthly SC dosing in recurrent pericarditis. About KiniksaKiniksa is a biopharmaceutical company dedicated to improving the lives of patients suffering from debilitating diseases by discovering, acquiring, developing, and commercializing novel therapies for diseases with unmet need, with a focus on cardiovascular indications. Kiniksa’s portfolio of assets is based on strong biologic rationale or validated mechanisms and offers the potential for differentiation. For more information, please visit www.kiniksa.com. About ARCALYSTARCALYST® (rilonacept) is a weekly, subcutaneously injected recombinant dimeric fusion protein that blocks interleukin-1 alpha (IL-1α) and interleukin-1 beta (IL-1β) signaling. ARCALYST was discovered by Regeneron Pharmaceuticals, Inc. (Regeneron) and is approved by the U.S. Food and Drug Administration (FDA) for the treatment of recurrent pericarditis (RP) and reduction in risk of recurrence in adults and children 12 years and older. ARCALYST is also approved by the FDA for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) in adults and children 12 years and older, and the maintenance of remission of Deficiency of Interleukin-1 Receptor Antagonist (DIRA) in adults and pediatric patients weighing 10 kg or more. The FDA granted Orphan Drug Exclusivity to ARCALYST upon its approval for recurrent pericarditis in 2021. The European Commission granted Orphan Drug Designation to ARCALYST for the treatment of idiopathic pericarditis in 2021. IMPORTANT SAFETY INFORMATION ABOUT ARCALYST ARCALYST may affect your immune system and can lower the ability of your immune system to fight infections. Serious infections, including life-threatening infections and death, have happened in patients taking ARCALYST. If you have any signs of an infection, call your doctor right away. Treatment with ARCALYST should be stopped if you get a serious infection. You should not begin treatment with ARCALYST if you have an infection or have infections that keep coming back (chronic infection). While taking ARCALYST, do not take other medicines that block interleukin-1, such as Kineret® (anakinra), or medicines that block tumor necrosis factor, such as Enbrel® (etanercept), Humira® (adalimumab), or Remicade® (infliximab), as this may increase your risk of getting a serious infection. Talk with your doctor about your vaccine history. Ask your doctor whether you should receive any vaccines before you begin treatment with ARCALYST. Medicines that affect the immune system may increase the risk of getting cancer. Stop taking ARCALYST and call your doctor or get emergency care right away if you have any symptoms of an allergic reaction. Your doctor will do blood tests to check for changes in your blood cholesterol and triglycerides. Common side effects include injection-site reactions (which may include pain, redness, swelling, itching, bruising, lumps, inflammation, skin rash, blisters, warmth, and bleeding at the injection site), upper respiratory tract infections, joint and muscle aches, rash, ear infection, sore throat, and runny nose. For more information about ARCALYST, talk to your doctor and see the Product Information. About KPL-387KPL-387 is an independently developed, investigational, fully human IgG2 monoclonal antibody that binds IL-1R1, inhibiting the signaling of the cytokines IL-1α and IL-1β. Kiniksa believes KPL-387 could expand the treatment options for recurrent pericarditis patients by enabling dosing with a single monthly SC injection in a liquid formulation. About KPL-1161KPL-1161 is an independently developed, investigational, Fc-modified IgG2 monoclonal antibody that binds IL-1R1, inhibiting the signaling of the cytokines IL-1α and IL-1β, with a target profile of quarterly SC dosing. Kiniksa is currently engaging in IND-enabling development activities for KPL-1161. About AbiprubartAbiprubart is an investigational humanized monoclonal antibody that binds to CD40 and is designed to inhibit the CD40-CD154 (CD40 ligand) interaction, a key T-cell co-stimulatory signal critical for B-cell maturation and immunoglobulin class switching and Type 1 immune responses. Kiniksa believes disrupting the CD40-CD154 co-stimulatory interaction is an attractive approach to addressing multiple autoimmune disease pathologies. Forward-Looking StatementsThis press release contains forward-looking statements. In some cases, you can identify forward- looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these identifying words. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation, statements regarding: our plan to initiate a Phase 2/3 clinical trial of KPL-387 in recurrent pericarditis in mid-2025, with Phase 2 data expected in the second half of 2026; our plan to discontinue the development of abiprubart in Sjögren’s Disease and explore strategic alternatives for the asset; our plan to prioritize development in cardiovascular indications; our plan to further increase our penetration into the recurrent pericarditis market with ARCALYST; our belief that KPL-387 could expand the treatment options for recurrent pericarditis patients by providing a single monthly SC injection in a liquid formulation; our target profile of monthly and quarterly SC dosing for KPL-387 and KPL-1161, respectively; our beliefs about the mechanisms of our assets and potential impact of their approach; and our belief that our portfolio of assets offers the potential for differentiation. These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including without limitation, the following: delays or difficulty in enrollment of patients in, and activation or continuation of sites for, our clinical trials; delays or difficulty in completing our clinical trials as originally designed; potential for changes between final data and any preliminary, interim, top-line or other data from clinical trials; our inability to replicate results from our earlier clinical trials or studies; impact of additional data from us or other companies, including the potential for our data to produce negative, inconclusive or commercially uncompetitive results; potential undesirable side effects caused by our products and product candidates; our inability to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities; potential for applicable regulatory authorities to not accept our filings, delay or deny approval of any of our product candidates or require additional data or trials to support approval; our reliance on third parties as the sole source of supply of the drug substance and drug product used in our products and product candidates; raw material, important ancillary product and drug substance and/or drug product shortages; our reliance on third parties to conduct research, clinical trials, and/or certain regulatory activities for our product candidates; complications in coordinating requirements, regulations and guidelines of regulatory authorities across jurisdictions for our clinical trials; business development activities and their impact on our financial performance and strategy; changes in our operating plan, business development strategy or funding requirements; and existing or new competition. These and other important factors discussed in our filings with the U.S. Securities and Exchange Commission, including under the caption “Risk Factors” contained therein, could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. Except as required by law, we disclaim any intention or obligation to update or revise any forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release. ARCALYST® is a registered trademark of Regeneron Pharmaceuticals, Inc. Every Second Counts!® Kiniksa Investor ContactJonathan Kirshenbaum(781) 829-3949jkirshenbaum@kiniksa.com Kiniksa Media ContactTyler Gagnon (781) 431-9100tgagnon@kiniksa.com

Phase 1Orphan DrugDrug ApprovalLicense out/in

25 Feb 2025

·investors.kiniksa.com

Kiniksa Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial Results and Recent Portfolio Execution

– ARCALYST® (rilonacept) Q4 2024 and full year 2024 net product revenue of $122.5 million and $417.0 million, respectively –– ARCALYST 2025 net product revenue expected to be $560 - $580 million –– KPL-387 Phase 2/3 clinical trial in recurrent pericarditis expected to initiate in mid-2025; Phase 2 data expected in 2H 2026 –– Abiprubart development in Sjögren’s Disease to be discontinued –– Current operating plan expected to remain cash flow positive on an annual basis –– Conference call and webcast scheduled for 8:30 am ET today – LONDON, Feb. 25, 2025 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals International, plc (Nasdaq: KNSA) (Kiniksa), a biopharmaceutical company developing and commercializing novel therapies for diseases with unmet need, with a focus on cardiovascular indications, today reported fourth quarter and full year 2024 financial results and recent portfolio execution. “Strong commercial execution in 2024 resulted in 79% year-over-year ARCALYST sales growth to $417.0 million. We believe substantial opportunity remains for ARCALYST, and expect 2025 sales of between $560 and $580 million,” said Sanj K. Patel, Chairman and Chief Executive Officer of Kiniksa. “Today, we are excited to announce the development program for KPL-387, which we believe could expand the treatment options for recurrent pericarditis patients by enabling a single monthly subcutaneous injection in a liquid formulation. We have interacted with the FDA and plan to initiate a Phase 2/3 clinical trial of KPL-387 in recurrent pericarditis in mid-2025. In line with our prioritization of cardiovascular indications, we plan to discontinue the development of abiprubart in Sjögren’s Disease. On behalf of our entire organization, I would like to thank the patients, caregivers, and investigators who contributed to our study.” Corporate Update Kiniksa continues to focus development on diseases with unmet need, prioritizing cardiovascular indications. Kiniksa announced today the development of KPL-387 in recurrent pericarditis, with a target profile of monthly subcutaneous (SC) dosing. KPL-387 is a fully human immunoglobulin G2 (IgG2) monoclonal antibody that binds human interleukin-1 receptor 1 (IL-1R1), inhibiting the signaling of the cytokines interleukin-1α (IL-1α) and interleukin-1β (IL-1β). Kiniksa announced today that it is advancing KPL-1161 towards clinical development with a target profile of quarterly SC dosing. KPL-1161 is an Fc-modified IgG2 monoclonal antibody that binds IL-1R1, inhibiting the signaling of the cytokines IL-1α and IL-1β. Kiniksa announced today that it plans to discontinue abiprubart development in Sjögren’s Disease. The company will explore strategic alternatives for the asset. Kiniksa announced today that it has exercised its right to terminate its exclusive license agreement for mavrilimumab with MedImmune. Kiniksa announced today the development of KPL-387 in recurrent pericarditis, with a target profile of monthly subcutaneous (SC) dosing. KPL-387 is a fully human immunoglobulin G2 (IgG2) monoclonal antibody that binds human interleukin-1 receptor 1 (IL-1R1), inhibiting the signaling of the cytokines interleukin-1α (IL-1α) and interleukin-1β (IL-1β). Kiniksa announced today that it is advancing KPL-1161 towards clinical development with a target profile of quarterly SC dosing. KPL-1161 is an Fc-modified IgG2 monoclonal antibody that binds IL-1R1, inhibiting the signaling of the cytokines IL-1α and IL-1β. Kiniksa announced today that it plans to discontinue abiprubart development in Sjögren’s Disease. The company will explore strategic alternatives for the asset. Kiniksa announced today that it has exercised its right to terminate its exclusive license agreement for mavrilimumab with MedImmune. Portfolio Execution ARCALYST (IL-1α and IL-1β cytokine trap) ARCALYST net product revenue was $122.5 million and $417.0 million for the fourth quarter and full year 2024, respectively. Since launch, more than 2,850 prescribers have written ARCALYST prescriptions for recurrent pericarditis. As of the end of the fourth quarter of 2024, average total duration of ARCALYST therapy in recurrent pericarditis was approximately 27 months. As of the end of the fourth quarter of 2024, approximately 13% of the target 14,000 multiple-recurrence patients were actively on ARCALYST treatment. KPL-387 (monoclonal antibody IL-1 receptor antagonist) Kiniksa is conducting a single ascending dose (SAD) and multiple ascending dose Phase 1 clinical trial of KPL-387 in healthy volunteers. Topline data from the SAD portion of the Phase 1 trial support potential monthly SC dosing in recurrent pericarditis. Kiniksa has interacted with the U.S. Food and Drug Administration (FDA) and expects to initiate a Phase 2/3 clinical trial of KPL-387 in recurrent pericarditis in mid-2025, with Phase 2 data expected in the second half of 2026. Topline data from the SAD portion of the Phase 1 trial support potential monthly SC dosing in recurrent pericarditis. KPL-1161 (Fc-modified monoclonal antibody IL-1 receptor antagonist) Kiniksa is conducting Investigational New Drug (IND)-enabling development activities with a target profile of quarterly SC dosing. Financial Results Total revenue for the fourth quarter of 2024 was $122.5 million, compared to $83.4 million for the fourth quarter of 2023. Total revenue for the full year 2024 was $423.2 million, compared to $270.3 million for the full year 2023. Total revenue for the fourth quarter of 2024 did not include any license and collaboration revenue, compared to $12.2 million for the fourth quarter of 2023. Total revenue for the full year 2024 included $6.2 million in license and collaboration revenue, compared to $37.1 million for the full year 2023. Total operating expenses for the fourth quarter of 2024 were $141.8 million, compared to $83.3 million for the fourth quarter of 2023. Total operating expenses for the full year 2024 were $468.9 million, compared to $295.5 million for the full year 2023. Total operating expenses for the fourth quarter of 2024 included $48.2 million in collaboration expenses, which are driven by ARCALYST collaboration profitability, compared to $16.9 million for the fourth quarter of 2023. Total operating expenses for the full year 2024 included $128.3 million in collaboration expenses, compared to $56.5 million for the full year 2023. Total operating expenses for the fourth quarter of 2024 included $8.3 million in non-cash, share-based compensation expense, compared to $7.8 million for the fourth quarter of 2023. Total operating expenses for the full year 2024 included $30.7 million in non-cash, share-based compensation expense, compared to $27.1 million for the full year 2023. Net loss for the fourth quarter of 2024 was $8.9 million, compared to a net income of $25.2 million for the fourth quarter of 2023. Net loss for the full year 2024 was $43.2 million, compared to net income of $14.1 million for the full year 2023. Net loss for the fourth quarter of 2024 included a tax benefit of $8.1 million, compared to a tax benefit of $22.8 million for the fourth quarter of 2023, both primarily due to the treatment of non-cash deferred tax assets. Net loss for the full year 2024 included a tax expense of $7.0 million, compared to a tax benefit of $30.7 million for the full year 2023, both primarily due to the treatment of non-cash deferred tax assets. As of December 31, 2024, Kiniksa had $243.6 million of cash, cash equivalents, and short-term investments and no debt, compared to $206.4 million as of December 31, 2023. Total revenue for the fourth quarter of 2024 did not include any license and collaboration revenue, compared to $12.2 million for the fourth quarter of 2023. Total revenue for the full year 2024 included $6.2 million in license and collaboration revenue, compared to $37.1 million for the full year 2023. Total operating expenses for the fourth quarter of 2024 included $48.2 million in collaboration expenses, which are driven by ARCALYST collaboration profitability, compared to $16.9 million for the fourth quarter of 2023. Total operating expenses for the full year 2024 included $128.3 million in collaboration expenses, compared to $56.5 million for the full year 2023. Total operating expenses for the fourth quarter of 2024 included $8.3 million in non-cash, share-based compensation expense, compared to $7.8 million for the fourth quarter of 2023. Total operating expenses for the full year 2024 included $30.7 million in non-cash, share-based compensation expense, compared to $27.1 million for the full year 2023. Net loss for the fourth quarter of 2024 included a tax benefit of $8.1 million, compared to a tax benefit of $22.8 million for the fourth quarter of 2023, both primarily due to the treatment of non-cash deferred tax assets. Net loss for the full year 2024 included a tax expense of $7.0 million, compared to a tax benefit of $30.7 million for the full year 2023, both primarily due to the treatment of non-cash deferred tax assets. Financial Guidance Kiniksa expects 2025 ARCALYST net product revenue of between $560 million and $580 million. Kiniksa expects its current operating plan to remain cash flow positive on an annual basis. Conference Call Information Kiniksa will host a conference call and webcast at 8:30 a.m. Eastern Time on Tuesday, February 25, 2025, to discuss fourth quarter and full year 2024 financial results and to provide a corporate update. Individuals interested in participating in the call via telephone may register here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. To access the webcast, please visit the Investors and Media section of Kiniksa’s website. A replay of the event will also be available on Kiniksa’s website within approximately 48 hours after the event. About KiniksaKiniksa is a biopharmaceutical company dedicated to improving the lives of patients suffering from debilitating diseases by discovering, acquiring, developing, and commercializing novel therapies for diseases with unmet need, with a focus on cardiovascular indications. Kiniksa’s portfolio of assets is based on strong biologic rationale or validated mechanisms and offers the potential for differentiation. For more information, please visit www.kiniksa.com. About ARCALYSTARCALYST is a weekly, subcutaneously injected recombinant dimeric fusion protein that blocks interleukin-1 alpha (IL-1α) and interleukin-1 beta (IL-1β) signaling. ARCALYST was discovered by Regeneron Pharmaceuticals, Inc. (Regeneron) and is approved by the U.S. Food and Drug Administration (FDA) for the treatment of recurrent pericarditis (RP) and reduction in risk of recurrence in adults and children 12 years and older. ARCALYST is also approved by the FDA for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) in adults and children 12 years and older, and the maintenance of remission of Deficiency of Interleukin-1 Receptor Antagonist (DIRA) in adults and pediatric patients weighing 10 kg or more. The FDA granted Orphan Drug Exclusivity to ARCALYST upon its approval for recurrent pericarditis in 2021. The European Commission granted Orphan Drug Designation to ARCALYST for the treatment of idiopathic pericarditis in 2021. IMPORTANT SAFETY INFORMATION ABOUT ARCALYST ARCALYST may affect your immune system and can lower the ability of your immune system to fight infections. Serious infections, including life-threatening infections and death, have happened in patients taking ARCALYST. If you have any signs of an infection, call your doctor right away. Treatment with ARCALYST should be stopped if you get a serious infection. You should not begin treatment with ARCALYST if you have an infection or have infections that keep coming back (chronic infection). While taking ARCALYST, do not take other medicines that block interleukin-1, such as Kineret® (anakinra), or medicines that block tumor necrosis factor, such as Enbrel® (etanercept), Humira® (adalimumab), or Remicade® (infliximab), as this may increase your risk of getting a serious infection. Talk with your doctor about your vaccine history. Ask your doctor whether you should receive any vaccines before you begin treatment with ARCALYST. Medicines that affect the immune system may increase the risk of getting cancer. Stop taking ARCALYST and call your doctor or get emergency care right away if you have any symptoms of an allergic reaction. Your doctor will do blood tests to check for changes in your blood cholesterol and triglycerides. Common side effects include injection-site reactions (which may include pain, redness, swelling, itching, bruising, lumps, inflammation, skin rash, blisters, warmth, and bleeding at the injection site), upper respiratory tract infections, joint and muscle aches, rash, ear infection, sore throat, and runny nose. For more information about ARCALYST, talk to your doctor and see the Product Information. About KPL-387KPL-387 is an independently developed, investigational, fully human IgG2 monoclonal antibody that binds IL-1R1, inhibiting the signaling of the cytokines IL-1α and IL-1β. Kiniksa believes KPL-387 could expand the treatment options for recurrent pericarditis patients by enabling dosing with a single monthly SC injection in a liquid formulation. About KPL-1161KPL-1161 is an independently developed, investigational, Fc-modified IgG2 monoclonal antibody that binds IL-1R1, inhibiting the signaling of the cytokines IL-1α and IL-1β, with a target profile of quarterly SC dosing. Kiniksa is currently engaging in IND-enabling development activities for KPL-1161. About AbiprubartAbiprubart is an investigational humanized monoclonal antibody that binds to CD40 and is designed to inhibit the CD40-CD154 (CD40 ligand) interaction, a key T-cell co-stimulatory signal critical for B-cell maturation and immunoglobulin class switching and Type 1 immune responses. Kiniksa believes disrupting the CD40-CD154 co-stimulatory interaction is an attractive approach to addressing multiple autoimmune disease pathologies. Forward-Looking Statements This press release contains forward-looking statements. In some cases, you can identify forward looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these identifying words. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation, statements regarding: our belief that substantial opportunity remains for ARCALYST and our expectation that ARCALYST 2025 net product revenue will be between $560 million and $580 million; our plan to initiate a Phase 2/3 clinical trial of KPL-387 in recurrent pericarditis in mid-2025, with Phase 2 data expected in the second half of 2026; our plan to discontinue abiprubart development in Sjögren’s Disease and explore strategic alternatives for the asset; our expectation that our current operating plan will remain cash flow positive on an annual basis; our plan to focus development on diseases with unmet need, prioritizing cardiovascular indications; our belief that KPL-387 could expand the treatment options for recurrent pericarditis patients by enabling a single monthly SC injection in a liquid formulation; our target profile of monthly and quarterly dosing for KPL-387 and KPL-1161, respectively; our beliefs about the mechanisms of our assets and potential impact of their approach; and our belief that our portfolio of assets offers the potential for differentiation. These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including without limitation, the following: delays or difficulty in enrollment of patients in, and activation or continuation of sites for, our clinical trials; delays or difficulty in completing our clinical trials as originally designed; potential for changes between final data and any preliminary, interim, top-line or other data from clinical trials; our inability to replicate results from our earlier clinical trials or studies; impact of additional data from us or other companies, including the potential for our data to produce negative, inconclusive or commercially uncompetitive results; potential undesirable side effects caused by our products and product candidates; our inability to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities; potential for applicable regulatory authorities to not accept our filings, delay or deny approval of any of our product candidates or require additional data or trials to support approval; our reliance on third parties as the sole source of supply of the drug substance and drug product used in our products and product candidates; raw material, important ancillary product and drug substance and/or drug product shortages; our reliance on third parties to conduct research, clinical trials, and/or certain regulatory activities for our product candidates; complications in coordinating requirements, regulations and guidelines of regulatory authorities across jurisdictions for our clinical trials; business development activities and their impact on our financial performance and strategy; changes in our operating plan, business development strategy or funding requirements; and existing or new competition. These and other important factors discussed in our filings with the U.S. Securities and Exchange Commission, including under the caption “Risk Factors” contained therein, could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. Except as required by law, we disclaim any intention or obligation to update or revise any forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release. ARCALYST® is a registered trademark of Regeneron Pharmaceuticals, Inc. Every Second Counts! ® Kiniksa Investor ContactJonathan Kirshenbaum(781) 829-3949jkirshenbaum@kiniksa.com Kiniksa Media ContactTyler Gagnon (781) 431-9100tgagnon@kiniksa.com

Phase 1License out/inDrug ApprovalOrphan DrugFinancial Statement

100 Deals associated with Rilonacept

External Link

KEGG	Wiki	ATC	Drug Bank
D06635	Rilonacept	L04AC04	DB06372

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Muckle-Wells Syndrome	China	Hangzhou Zhongmeihuadong Pharmaceutical Co., Ltd.	22 Nov 2024
Recurrent pericarditis	United States	Kiniksa Pharmaceuticals (UK) Ltd.	18 Mar 2021
Deficiency of Interleukin-1 Receptor Antagonist	United States	Kiniksa Pharmaceuticals (UK) Ltd.	18 Dec 2020
Familial cold urticaria	United States	Kiniksa Pharmaceuticals (UK) Ltd.	18 Dec 2020
Cryopyrin-Associated Periodic Syndromes	United States	Kiniksa Pharmaceuticals (UK) Ltd.	27 Feb 2008
Familial Cold Autoinflammatory Syndrome 2	United States	Kiniksa Pharmaceuticals (UK) Ltd.	27 Feb 2008

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Pericarditis	NDA/BLA	European Union	FGK Representative Service GmbH	-
Primary gout	Phase 3	United States	Regeneron Pharmaceuticals, Inc.	01 Mar 2009
Gout	Phase 3	United States	Regeneron Pharmaceuticals, Inc.	01 Feb 2009
Gout	Phase 3	Canada	Regeneron Pharmaceuticals, Inc.	01 Feb 2009
Scleroderma, Diffuse	Phase 2	United States	Regeneron Pharmaceuticals, Inc.	01 Dec 2011
Anemia	Phase 2	United States	Regeneron Pharmaceuticals, Inc.	07 Feb 2008
Chronic Kidney Diseases	Phase 2	United States	Regeneron Pharmaceuticals, Inc.	07 Feb 2008
Hearing Loss	Phase 1	United States	Regeneron Pharmaceuticals, Inc.	01 Feb 2017
Systemic onset juvenile chronic arthritis	Phase 1	United States	Regeneron Pharmaceuticals, Inc.	01 Nov 2005

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03737110 (RHAPSODY Long-term Extension, Pubmed)	Phase 3	COVID-19 \| Vaccination interleukin-1 pathway	-	Rilonacept	lviardzowp(egdbtocugq) = mild in 13 patients, moderate in 2, and severe in 1 sevhefgfoe (tuuhkjskpg )	Positive	01 Jun 2024
				Placebo
NCT03737110 (RHAPSODY, Pubmed \| J Am Heart Assoc) Manual	Phase 3	Recurrent pericarditis	74	Rilonacept	ngakvjmlej(yzqsrueysh) = nhvstlrqyc xnfbdwlgnl (tdfrkeeckl ) View more	Positive	19 Mar 2024
				oral therapies	ngakvjmlej(qebqxzwxeh) = njpkbjcqak lsbyppviod (tdveagihzu )
NCT03737110 (RHAPSODY, ACR2021)	Phase 3	Recurrent pericarditis C-reactive protein (CRP)	86	Rilonacept 320 mg SC load, 160 mg SC weekly	ynehuwpsat(bewejaocga) = uhfbehvlwf elucrxizgm (xvvijohtzu )	Positive	08 Nov 2021
				Placebo	ynehuwpsat(bewejaocga) = bsrxhvgimz elucrxizgm (xvvijohtzu )
NCT03980522 (CTgov)	Phase 2	Recurrent pericarditis	26	KPL-914 (KPL-914: Part 1 Participants)	rieajvpspp(cshgipyvmr) = ffmmakojcc doervtaqhj (poqumrpjlz, 5.3423) View more	-	27 May 2021
				KPL-914 (KPL-914: Part 2 Participants)	rieajvpspp(cshgipyvmr) = brbxynrrbj doervtaqhj (poqumrpjlz, 0.4313) View more
NCT03980522 (Pubmed \| Heart) Manual	Phase 2	Idiopathic Recurrent Pericarditis	25	rilonacept	dehisxfrfc(imgixiokil) = from 4.62 mg/dL at baseline to 0.38 mg/dL klbopuzzjp (xyyfsuqcjm ) View more	Positive	23 Nov 2020
NCT01538719 (CTgov)	Phase 1/2	Scleroderma, Diffuse	24	Placebo (Placebo)	sqptvbdygp(cowemgfwuc) = emlmfltpat rhaasymbxh (piglbwquvw, yxugbcmqkp - waingyhpaf) View more	-	01 May 2018
				Rilonacept (Rilonacept)	sqptvbdygp(cowemgfwuc) = awwvhxlhmv rhaasymbxh (piglbwquvw, amemsexrwx - edibpqstmn) View more
NCT01801449 (CTgov)	Phase 2	Deficiency of Interleukin-1 Receptor Antagonist	6	Rilonacept	ishhxekygr = bsrrhdjzaw ybnkyshykj (orzonsqdjg, oyyelkxvje - gyhqzizpwv) View more	-	16 Oct 2017
NCT01801449 (Pubmed \| JCI Insight) Manual	Phase 3	Deficiency of Interleukin-1 Receptor Antagonist	6	rilonacept	bzdnajlnxn(anowkryrju) = No serious adverse events were reported. kvvvnjcyhe (ksnszcbwmq )	Positive	17 Aug 2017
				Placebo
NCT01459796 (UPSURGE, CTgov)	Phase 3	Gout	220	Placebo+Allopurinol (Placebo)	eubhhedxiz = eiwojiqrus zrqxoglsrj (oncpbnvefx, ehdvhcalul - aqtiwzyjym) View more	-	18 Jul 2017
				Allopurinol+Rilonacept (Rilonacept 80 mg)	eubhhedxiz = hsfpfzmzen zrqxoglsrj (oncpbnvefx, ybsucpvaha - yjvcltygmj) View more
NCT00610363 (CTgov)	Phase 2	Gout	83	Placebo (for Rilonacept) (Placebo)	muhqrzvpfu(yekcddluzv) = tkvhogulwq dtvuszgbnk (jjqzcfkmqk, 1.071) View more	-	02 May 2017
				Rilonacept (Rilonacept 160 mg)	muhqrzvpfu(yekcddluzv) = cckrqjfvei dtvuszgbnk (jjqzcfkmqk, 0.358) View more

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Biosimilar

Competitive landscape of biosimilars in different countries/locations. Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis