PALETTE- Adaptive Platform Trial for Personnalisation of Sepsis Treatment in Children and Adults: a Multi-national, Treatable Traits-guided, Adaptive, Bayesian Basket Trial"

PALETTE is a perpetual adaptive platform to efficiently study sepsis interventions within 'treatable traits' in all-ages patients enabling prompt evaluation of pandemic treatments. Treatable traits, therapeutic targets identified by phenotypes or endotypes (defined by biological mechanism or by treatment response) through validated biomarkers (measurable characteristic reflecting normal or pathogenic processes, or treatment responses), may include multi-omics, cellular, immune, metabolic, endocrine features, or intelligent algorithms. PALETTE Bayesian adaptive design enables parallel investigations of multiple interventions for sepsis, and quick inclusion of pandemic pathogens. PALETTE's new conceptual model will respond to the challenges of standard approaches, i.e. series of sepsis trials, each investigating one or two interventions, expensive, time consuming, and inappropriate in pandemic context.

Start Date01 Apr 2026

Sponsor / Collaborator

Assistance Publique des Hôpitaux de Paris SA

NCT06666335

/ Not yet recruitingPhase 4

A Prospective, Open-label, Single Arm, Multicenter, Post-authorization Efficacy and Safety Study of Subcutaneous Anakinra in Chinese Patients With Colchicine-resistant Familial Mediterranean Fever (FMF)

The purpose of this study is to evaluate the efficacy and safety of anakinra in Chinese patients with colchicine-resistand Familial Mediterranian Fever (FMF). The study consists of up to one month screening, to see if a patient is suitable to the study, 6 months of treatment with anakinra and one month safety follow up after last dose of anakinra. In total 3 patients, male and female from 2 years of age (minimum 10kg weight), will be enrolled to the study.

Start Date01 Apr 2025

Sponsor / Collaborator

Swedish Orphan Biovitrum AB

[+1]

NCT06697431

/ Not yet recruitingPhase 4IIT

A Randomized, Controlled, Open-label, Non Inferiority KawasakI Trial With Anakinra

This is a multicenter, open-label, randomized, controlled, interventional trial followed by a long-term observational extension period in patients with Kawasaki Disease (KD) to be treated eitherwith endovenous Immunoglobulins (IVIG-standard treatment) versus anakinra
Aim of the study: to demonstrate that anakinra is non-inferior to IVIG in KD, in terms of fever control in the acute phase and development of coronary artery dilation/aneurisms (CAA) within one year from the onset.

Start Date01 Apr 2025

Sponsor / Collaborator

ASST degli Spedali Civili di Brescia

[+1]

100 Clinical Results associated with Anakinra (Amgen)

100 Translational Medicine associated with Anakinra (Amgen)

100 Patents (Medical) associated with Anakinra (Amgen)

2,699

Literatures (Medical) associated with Anakinra (Amgen)

01 Dec 2025·Current Pain and Headache Reports

Familial Mediterranean Fever (FMF): Emerging Concepts in Diagnosis, Pain Management, and Novel Treatment Options: A Narrative Review

Review

Author: Islam, Kazi N ; Baptiste, Carlo Jean ; Norwood, Haley A ; Nguyen, Ivan D ; Islam, Rahib K ; Ahmadzadeh, Shahab ; Kaye, Alan D ; Barrie, Sonnah ; McGregor, David W ; Robin, Connor J ; Parker-Actlis, Tomasina Q ; Shekoohi, Sahar

PURPOSE OF REVIEW:

Familial Mediterranean Fever (FMF) is a hereditary autoinflammatory disorder predominantly affecting individuals of Mediterranean and Middle Eastern descent, including those with certain heritages including Sephardic Jewish, Armenian, Turkish, and Arab. The disorder affects up to 1 in 200 people making it a very common etiology for pain states worldwide, including serositis mediated painful states of the chest, joint, and abdomen.

RECENT FINDINGS:

Defined by recurrent episodes of fever and inflammation, FMF can lead to not only severe pain, but complications such as renal amyloidosis, if untreated. This narrative review explores the genetic basis, clinical manifestations, diagnostic criteria, and current management strategies for FMF. Mutations in the MEFV gene result in the dysregulation of the pyrin inflammasome, leading to excessive production of inflammatory cytokines. Diagnosis primarily relies on clinical criteria supported by genetic testing. Colchicine remains the cornerstone of treatment, effectively preventing inflammatory attacks and complications. For colchicine-resistant patients, IL-1 antagonists like anakinra and canakinumab show promise, although their long-term benefits require further investigation. The present investigation underscores the importance of early diagnosis and integrated treatment approaches to improve patient outcomes, pain management, and quality of life.

01 Apr 2025·Current Research in Translational Medicine

Trends in drug repurposing: Advancing cardiovascular disease management in geriatric populations

Review

Author: Moka, Murali Krishna ; D K, Sriram ; Rathakrishnan, Deepalaxmi ; Jagadeeshwaran, V ; George, Melvin

Drug repurposing is a promising strategy for managing cardiovascular disease (CVD) in geriatric populations, offering efficient and cost-effective solutions. CVDs are prevalent across all age groups, with a significant increase in prevalence among geriatric populations. The middle-age period (40-65 years) is critical due to factors like obesity, sedentary lifestyle, and psychosocial stress. In individuals aged 65 and older, the incidence of CVDs is highest due to age-related physiological changes and prolonged exposure to risk factors. In this review we find that certain drugs, such as non-cardiovascular drugs like anakinra, probenecid, N-acetyl cysteine, quercetin, resveratrol, rapamycin, colchicine, bisphosphonates, hydroxychloroquine, SGLT-2i drugs, GLP-1Ras drugs and sildenafil are recommended for drug repurposing to achieve cardiovascular benefits in geriatric patients. However, agents such as canakinumab, methotrexate, ivermectin, erythromycin, capecitabine, carglumic acid, chloroquine, and furosemide are constrained in their therapeutic use and warrant meticulous consideration, rendering them less favorable for this specific application. This review emphasizes the importance of exploring alternative therapeutic strategies to improve outcomes in geriatric populations and suggests drug repurposing as a promising avenue to enhance treatment efficacy.

01 Apr 2025·HEPATOLOGY

Acute kidney injury in severe alcohol-associated hepatitis treated with anakinra plus zinc or prednisone

Article

Author: Dasarathy, Srinivasan ; Sanyal, Arun J. ; Cotter, Thomas G ; Patidar, Kavish R ; Asgharpour, Amon ; Thevkar Nagesh, Prashanth ; Mehal, Wajahat Z ; Mehal, Wajahat Z. ; Simonetto, Douglas A. ; Patidar, Kavish R. ; Yu, Yunpeng ; Tu, Wanzhu ; Szabo, Gyongyi ; Jan, Muhammad Y ; McClain, Craig J ; Tang, Qing ; Shah, Vijay H. ; Nagy, Laura E ; Nagy, Laura E. ; Li, Yang ; Kamath, Patrick S ; Jan, Muhammad Y. ; Shah, Vijay H ; Mitchell, Mack ; Cotter, Thomas G. ; Kamath, Patrick S. ; Sanyal, Arun J ; Bataller, Ramon ; Taiwo, Moyinoluwa ; Gawrieh, Samer ; McClain, Craig J. ; Chalasani, Naga ; Simonetto, Douglas A

Background and Aims::

In a recent trial, patients with severe alcohol-associated hepatitis treated with anakinra plus zinc (A+Z) had lower survival and higher acute kidney injury (AKI) rates versus prednisone (PRED). We characterize the clinical factors and potential mechanisms associated with AKI development in that trial.

Approach and Results::

Data from 147 participants in a multicenter randomized clinical trial (74 A+Z, 73 PRED) were analyzed. AKI, AKI phenotypes, and kidney injury biomarkers were compared between participants who did/did not develop AKI in the 2 treatment arms. Multivariable competing risk analyses were performed to identify baseline risk factors for incident AKI, with death treated as a competing event. Risk factors considered were age, sex, mean arterial pressure, white blood cell count, albumin, MELD, ascites, HE, and treatment arm. At baseline, no participants had AKI; 33% (n=49) developed AKI during follow-up. AKI incidence was higher in A+Z than in PRED (45% [n=33] versus 22% [n=16], p=0.001). AKI phenotypes were similar between the 2 treatment arms (p=0.361), but peak AKI severity was greater in A+Z than PRED (stage 3 n=21 [63.6%] vs. n=8 [50.0%], p=0.035). At baseline, urine-neutrophil-gelatinase–associated lipocalin levels were similar between participants who developed AKI in both treatment arms (p=0.319). However, day 7 and 14 urine-neutrophil-gelatinase–associated lipocalin levels were significantly elevated in participants treated with A+Z who developed AKI versus participants treated with PRED who developed AKI (p=0.002 and 0.032, respectively). On multivariable competing risk analysis, only A+Z was independently associated with incident AKI (subdistribution hazard ratio 2.35, p=0.005).

Conclusions::

AKI occurred more frequently and was more severe in participants treated with A+Z. A+Z–treated participants with AKI had higher urine-neutrophil-gelatinase–associated lipocalin, suggesting that A+Z maybe nephrotoxic in patients with severe alcohol-associated hepatitis.

News (Medical) associated with Anakinra (Amgen)

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

10 Jan 2025

·www.prnewswire.com

Sobi's full year 2024 revenue higher than previous estimate

STOCKHOLM, Jan. 10, 2025 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi®) (STO:SOBI) announces today that revenue for the full year 2024 was higher than previous estimate. Full-year revenue was approximately SEK 26,000 M, representing approximately 19% growth at constant exchange rate (CER) (1). Adjusted EBITA margin (1,2) was in the mid-30s per cent of revenues. The main reasons for the increased revenue are higher sales than expected in Q4 across the Haemophilia portfolio and for Kineret. Altuvoct: Higher than expected rate of new patients switching to Altuvoct in markets where the product has been launched, mainly Germany and Switzerland. Elocta: Benefited from higher patient numbers across markets and in markets where Altuvoct is launched there were less switches than expected from Elocta. Favorable gross-to-net effects were also observed. Alprolix: Higher than expected number of new patients as well as increase in on-demand treatments across Europe. Kineret: Higher than expected sales driven mainly by positive gross-to-net adjustments and favorable order phasing but also supported by increased demand. The adjusted EBITA margin remained in the expected range as the stronger revenue performance was offset by negative mix effects on the gross margin as well as investments into our launch and pipeline products in the fourth quarter. At the publication of the Q3 2024 report on 24 October 2024 Sobi stated the outlook for the full year 2024 to be: Revenue was anticipated to grow by a mid-teens percentage at CER and adjusted EBITA margin was anticipated to be in the mid-30s per cent of revenue. Sobi will announce its fourth quarter and full year 2024 report on Wednesday 5 February 2025 at 8:00 am CET. About Sobi Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information is information that Sobi is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out below, at 18:00 CET on 10 January 2025. Gerard Tobin Head of Investor Relations [1] Alternative Performance Measures (APMs). [2] Excluding items affecting comparability (IAC). This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Financial StatementBiosimilar

100 Deals associated with Anakinra (Amgen)

External Link

KEGG	Wiki	ATC	Drug Bank
D02934	Anakinra (Amgen)	L04AC03	DB00026

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Deficiency of Interleukin-1 Receptor Antagonist	United States	Swedish Orphan Biovitrum AB	18 Dec 2020
COVID-19	European Union	Swedish Orphan Biovitrum AB	08 Mar 2002
COVID-19	Iceland	Swedish Orphan Biovitrum AB	08 Mar 2002
COVID-19	Liechtenstein	Swedish Orphan Biovitrum AB	08 Mar 2002
COVID-19	Norway	Swedish Orphan Biovitrum AB	08 Mar 2002
Cryopyrin-Associated Periodic Syndromes	European Union	Swedish Orphan Biovitrum AB	08 Mar 2002
Cryopyrin-Associated Periodic Syndromes	Iceland	Swedish Orphan Biovitrum AB	08 Mar 2002
Cryopyrin-Associated Periodic Syndromes	Liechtenstein	Swedish Orphan Biovitrum AB	08 Mar 2002
Cryopyrin-Associated Periodic Syndromes	Norway	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial cold urticaria	European Union	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial cold urticaria	Iceland	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial cold urticaria	Liechtenstein	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial cold urticaria	Norway	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial Mediterranean Fever	European Union	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial Mediterranean Fever	Iceland	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial Mediterranean Fever	Liechtenstein	Swedish Orphan Biovitrum AB	08 Mar 2002
Familial Mediterranean Fever	Norway	Swedish Orphan Biovitrum AB	08 Mar 2002
Muckle-Wells Syndrome	European Union	Swedish Orphan Biovitrum AB	08 Mar 2002
Muckle-Wells Syndrome	Iceland	Swedish Orphan Biovitrum AB	08 Mar 2002
Muckle-Wells Syndrome	Liechtenstein	Swedish Orphan Biovitrum AB	08 Mar 2002

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Mucocutaneous Lymph Node Syndrome	Phase 3	France	Swedish Orphan Biovitrum AB	20 Oct 2023
COVID-19 respiratory infection	Phase 3	France	Swedish Orphan Biovitrum AB	27 Apr 2020
Inflammation	Phase 3	United States	Swedish Orphan Biovitrum AB	02 Apr 2020
Inflammation	Phase 3	Italy	Swedish Orphan Biovitrum AB	02 Apr 2020
Respiratory Distress Syndrome	Phase 3	United States	Swedish Orphan Biovitrum AB	02 Apr 2020
Respiratory Distress Syndrome	Phase 3	Italy	Swedish Orphan Biovitrum AB	02 Apr 2020
B-Cell Lymphoma	Phase 2	United States	Swedish Orphan Biovitrum AB	27 Dec 2021
Carney Complex	Phase 2	United States	Swedish Orphan Biovitrum AB	27 Dec 2021
Neurotoxicity Syndromes	Phase 2	United States	Swedish Orphan Biovitrum AB	27 Dec 2021
Cytokine Release Syndrome	Phase 2	United States	Swedish Orphan Biovitrum AB	01 Dec 2021

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EULAR2024 Manual	Not Applicable	Still's Disease, Adult-Onset	29	Anakinra	rlnbrxvork(cneoemqhoh) = trqihpzopb udnsnxeyeg (fvioerbunw ) View more	Positive	05 Jun 2024
EULAR2024	Not Applicable	COVID-19	-	High dose intravenous anakinra	nuwtfimjgr(ojakixnzix) = fcelhftdpv uqvzykpqnp (ryzcxymxsn ) View more	Positive	05 Jun 2024
				Anakinra (Standard of care (SoC))	meaykoodzq(vonhingbgy) = eimxhuduwh utggfcfeog (saoixnglac ) View more
EULAR2024	Not Applicable	Chondrocalcinosis IL-1β	5	Anakinra 100mg	humsrxwsgx(ycleyqkvtn) = lrqiupjvha queepctacc (ngqkeqrawi )	Positive	05 Jun 2024
EULAR2024	Not Applicable	COVID-19	-	High-dose intravenous anakinra + baricitinib	xjjfiauiop(hoehzusbfc) = wgvpaifkfr llkfodjisq (tfkelzrsqu )	Positive	05 Jun 2024
				high-dose intravenous anakinra (Control group)	xjjfiauiop(hoehzusbfc) = avjvskzyru llkfodjisq (tfkelzrsqu )
NCT04099901 (AFFECT-2, EHA 12024 \| EHA 22024) Manual	Phase 2	Neutropenia	88	Anakinra	gnnszvfkyh(alhhnmnqkp) = pvkosoalgj kecimpnqda (jneeqimdeb ) View more	Negative	14 May 2024
				placebo	gnnszvfkyh(alhhnmnqkp) = kqxekvfduc kecimpnqda (jneeqimdeb ) View more
NCT04680949 (SAVE-MORE, Pubmed \| Crit Care) Manual	Phase 3	COVID-19 IL1RL1 Positive	393	Anakinra	mhgxlfwxiq(fmkcinguwy) = bbvhrfhshf diujumoqvu (qhyewgqjay ) View more	Positive	12 Mar 2024
				Placebo	mhgxlfwxiq(fmkcinguwy) = lwvkanjubc diujumoqvu (qhyewgqjay ) View more
NCT02179853 (CTgov)	Phase 1/2	Coronary Artery Disease \| Mucocutaneous Lymph Node Syndrome	22	Anakinra (Dose Level 1 (Anakinra 2-4 mg/kg/Day))	mycmwgewef = jnhlemzxhn idbxqzsonm (wcowqlseha, lzldpsemhl - aecusnangt) View more	-	04 Mar 2024
				Anakinra (Dose Level 1 (Anakinra 5-7 mg/kg/Day))	mycmwgewef = teqxjzejdg idbxqzsonm (wcowqlseha, wmdvsapwuc - uatqppqrcf)
NCT04359784 (Tandem Meetings ASTCT and CIBMTR2024) Manual	Phase 2	B-Cell Lymphoma CD19	15	Anakinra 20Anakinra SC	grjoqbyznp(evuvdreutb) = ihxcyrlbky qeumhstnaf (hgevretgdn ) View more	Negative	01 Feb 2024
				Liso-cel alone	grjoqbyznp(evuvdreutb) = eanrxknuhr qeumhstnaf (hgevretgdn ) View more
NCT06071156 (Pubmed)	Not Applicable	Recurrent pericarditis C-reactive protein (CRP)	18	anakinra (CMR-guided patients)	wbfdsioomo(jxuribluxs) = xbiwimuais vspctpbcec (djxtfrxfpx ) View more	Positive	16 Jan 2024
				anakinra (CRP-guided patients)	wbfdsioomo(jxuribluxs) = wmonrvigah vspctpbcec (djxtfrxfpx ) View more
NCT04412291 (IMMCoVA, Pubmed \| PLoS One)	Phase 2	COVID-19 CRP \| ferritin \| lymphocytes ... View more	77	Tocilizumab	yfqrdztvrg(ystpouxuka) = iuuhtodjqq klhmtkcawo (qlibyvcsuw )	Negative	29 Dec 2023
				Anakinra	yfqrdztvrg(ystpouxuka) = ihdjwfojke klhmtkcawo (qlibyvcsuw )

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