DOCSLIB.ORG

Annual Report 2

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
Annual Report 2 KINIKSA PHARMACEUTICALS KINIKSA ANNUAL REPORT 2019 RELENTLESS. PASSIONATE. FOCUSED.™ 2019 ANNUAL REPORT ANNUAL 2019 Every Second Counts! ™ Dear Fellow Shareholders, MANAGEMENT TEAM WEBSITE Sanj K. Patel* Kiniksa.com I hope this letter finds you well as we adjust our routines and • We are evaluating KPL-404 in a single-ascending-dose Chief Executive Officer & Chairman of the Board navigate the impact of the coronavirus. At Kiniksa, the health trial in healthy volunteers to measure safety data and LEGAL COUNSEL and safety of our employees as well as the patients and people pharmacokinetics as well as receptor occupancy and John F. Paolini, MD, PhD* Senior Vice President, Chief Medical Officer Latham & Watkins LLP participating in and operating our clinical trials are of paramount T-cell Dependent Antibody Response (TDAR). The CD40- Boston, Massachusetts importance. We entered 2020 with our clinical-stage assets having CD40L pathway has been established in a broad range Thomas Beetham* the potential to generate data-driven value; these milestones of autoimmune diseases, such as rheumatoid arthritis, Executive Vice President, Corporate Development & Operations, Conyers Dill & Pearman Limited Chief Legal Officer Hamilton, HM CX, Bermuda remain on track thanks to the utmost dedication of our team. We will Sjogren’s syndrome, Graves’ disease, systemic lupus continue to monitor the pandemic and plan to adapt as necessary. erythematosus and solid organ transplant. Qasim Rizvi* Senior Vice President, Operations & Chief Commercial Officer INDEPENDENT REGISTERED ACCOUNTING FIRM Kiniksa is a clinical-stage biopharmaceutical company focused In 2019, each of our clinical-stage assets posted key accomplishments. PricewaterhouseCoopers LLP on developing immune-modulating medicines for the benefit Eben Tessari Boston, Massachusetts Senior Vice President, Chief Business Officer of patients suffering from devastating diseases. Through highly- • Rilonacept: The FDA granted Breakthrough Therapy dedicated clinical and corporate execution, 2019 represented a designation for rilonacept for the treatment of recurrent Dave Nichols TRANSFER AGENT AND REGISTRAR significant step toward this vision. pericarditis. Additionally, we reported final data from an open- Senior Vice President, Technical Operations American Stock Transfer & Trust Company, LLC label Phase 2 trial of rilonacept in recurrent pericarditis. The Brooklyn, New York Dana Martin Our clinical-stage assets are based on strong biologic rationale data provided first evidence that rilonacept treatment in the Senior Vice President, Global Medical Affairs and/or validated mechanisms. study improved clinically meaningful outcomes associated STOCK INFORMATION Martina Struck, PhD with the unmet medical need in recurrent pericarditis. We also Nasdaq Global Select Market: KNSA • Rilonacept is an inhibitor of interleukin-1 alpha (IL-1 т) and achieved target enrollment on time for our pivotal Phase 3 trial Vice President, Regulatory Affairs interleukin-1 beta (IL-1у), cytokines that have been shown in recurrent pericarditis (RHAPSODY) and continued to prepare Mike Megna INVESTOR RELATIONS to play a role in inflammatory diseases. for commercialization by generating evidence on disease Vice President, Finance & Chief Accounting Officer Mark Ragosa burden, building disease awareness with payers, • Mavrilimumab is a monoclonal antibody inhibitor of Melissa Manno Vice President, Investor Relations granulocyte macrophage colony stimulating factor (GM- physicians and advocacy groups and establishing core Vice President, Human Resources [email protected] CSF) receptor signaling. GM-CSF is a key growth factor capabilities such as distribution and patient services. and cytokine in autoinflammation and autoimmunity. * Executive officers as defined under Rule 3b-7 under the This Annual Report contains forward-looking statements that involve risks, • Mavrilimumab: We achieved target enrollment on time for Securities Exchange Act of 1934, as amended. uncertainties and other important factors that could cause results to differ • Vixarelimab (KPL-716) is a monoclonal antibody inhibitor our Phase 2 trial in GCA and initiated a clinical collaboration materially from those projected. In some cases, you can identify these statements with Kite, a Gilead company, to evaluate the investigational by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” of signaling through oncostatin M receptor beta (OSMRу), BOARD OF DIRECTORS “intend,” “goal,” “design,” “target,” “project,” “contemplate,” “believe,” “estimate,” combination of Yescarta® (axicabtagene ciloleucel) and “predict,” “potential” or “continue” or their negative or other similar expressions. the shared receptor subunit for interleukin-31 (IL-31) and Chairman mavrilimumab in relapsed or refractory large B-cell lymphoma. These important factors include those discussed in our Annual Report on Form oncostatin M (OSM) signaling. IL-31 and OSM are key Sanj K. Patel 10-K for the year ended December 31, 2019 (which forms a part of this Annual cytokines implicated in inflammation, pruritus, and fibrosis. Chief Executive Officer Report) under the caption “Risk Factors.” Accordingly, you are cautioned not to • Vixarelimab: Interim repeated-single-dose Phase 1b data place undue reliance on such statements. We undertake no obligation to update • KPL-404 is a monoclonal antibody inhibitor of the for vixarelimab in subjects with moderate-to-severe atopic Lead Independent Director any forward-looking statements. CD40-CD40 ligand (CD40L) interaction, a key T-cell dermatitis showed a rapid and sustained anti-pruritic Felix J. Baker, PhD co-stimulatory signal critical for B-cell maturation and response as measured by weekly average Worst-Itch Co-Managing Member, Baker Bros. Advisors LP Numeric Rating Scale (WI-NRS). These results supported immunoglobulin class switching. Directors further development of vixarelimab in a Phase 2a trial Additionally, our clinical-stage assets target diseases with significant in prurigo nodularis and an exploratory Phase 2 trial in Stephen R. Biggar, MD, PhD Partner, Baker Bros. Advisors LP unmet medical need and offer the potential for differentiation. diseases characterized by chronic pruritus. Richard S. Levy, MD • We are evaluating rilonacept for the potential treatment • KPL-404: The FDA accepted our investigational new drug Biopharmaceutical Consultant of recurrent pericarditis, a painful autoinflammatory application for the clinical study of KPL-404, and we initiated Thomas R. Malley cardiovascular disease, in a pivotal Phase 3 trial. There a Phase 1 trial. President, Mossrock Capital, LLC are no U.S. Food and Drug Administration (FDA)-approved therapies for recurrent pericarditis. In 2020, our clinical-stage assets are expected to generate clinical Tracey L. McCain data, including pivotal Phase 3 data from rilonacept, Phase 2 data Executive Vice President, Chief Legal and • We are evaluating mavrilimumab for the potential treatment from mavrilimumab, Phase 2 data from vixarelimab, and Phase 1 Compliance Officer, Blueprint Medicine Corporation of giant cell arteritis (GCA), a chronic inflammatory disease data from KPL-404. We believe these data have the potential to Kimberly J. Popovits of medium-to-large arteries, in a Phase 2 trial. While help inform our portfolio strategy and capital allocation decisions. Former Chief Executive Officer & Chairman of the Board, there is one FDA-approved therapy for GCA, we believe Genomic Health, Inc. mavrilimumab’s mechanism of action acts upstream and that As we continue to execute toward our goal of becoming a generational biopharmaceutical company, we will keep science Barry D. Quart, PharmD an unmet medical need remains. We are also exploring the President, Chief Executive Officer & Director, and patients at the center of our decisions. Thank you for your potential for mavrilimumab to treat a host of vasculitides and Heron Therapeutics, Inc. oncologic indications. ongoing support. ADDRESS • We are evaluating vixarelimab for the potential treatment of Every Second Counts!™ Kiniksa Pharmaceuticals, Ltd. prurigo nodularis, a chronic inflammatory skin condition, in Unless otherwise expressly stated, we obtained the industry, business, market and Clarendon House a Phase 2a trial. There are no FDA-approved therapies for other data contained in this Annual Report from reports, research surveys, clinical 2 Church Street trials, studies and similar data prepared by market research firms and other third prurigo nodularis. We are also investigating the potential Hamilton HM 11 parties, from industry, medical and general publications, and from government Bermuda data and similar sources. for vixarelimab to treat diseases characterized by chronic Sanj K. Patel pruritus in an exploratory Phase 2 trial. CEO and Chairman of the Board UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2019 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-38492 Kiniksa Pharmaceuticals, Ltd. (Exact name of registrant as specified in its charter) Bermuda 98-1327726 (State or other jurisdiction of (I.R.S. Employer incorporation or organization) Identification Number) Kiniksa Pharmaceuticals, Ltd. Clarendon House 2 Church Street Hamilton HM11, Bermuda + (44) 808-189-6257
Load more

Recommended publications
  • Fig. L COMPOSITIONS and METHODS to INHIBIT STEM CELL and PROGENITOR CELL BINDING to LYMPHOID TISSUE and for REGENERATING GERMINAL CENTERS in LYMPHATIC TISSUES
    (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (10) International Publication Number (43) International Publication Date Χ 23 February 2012 (23.02.2012) WO 2U12/U24519ft ft A2 (51) International Patent Classification: AO, AT, AU, AZ, BA, BB, BG, BH, BR, BW, BY, BZ, A61K 31/00 (2006.01) CA, CH, CL, CN, CO, CR, CU, CZ, DE, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, (21) International Application Number: HN, HR, HU, ID, IL, IN, IS, JP, KE, KG, KM, KN, KP, PCT/US201 1/048297 KR, KZ, LA, LC, LK, LR, LS, LT, LU, LY, MA, MD, (22) International Filing Date: ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, 18 August 201 1 (18.08.201 1) NO, NZ, OM, PE, PG, PH, PL, PT, QA, RO, RS, RU, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, (25) Filing Language: English TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, (26) Publication Language: English ZW. (30) Priority Data: (84) Designated States (unless otherwise indicated, for every 61/374,943 18 August 2010 (18.08.2010) US kind of regional protection available): ARIPO (BW, GH, 61/441,485 10 February 201 1 (10.02.201 1) US GM, KE, LR, LS, MW, MZ, NA, SD, SL, SZ, TZ, UG, 61/449,372 4 March 201 1 (04.03.201 1) US ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, MD, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, (72) Inventor; and EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, ΓΓ, LT, LU, (71) Applicant : DEISHER, Theresa [US/US]; 1420 Fifth LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, Avenue, Seattle, WA 98101 (US).
    [Show full text]
  • Targeted Review List Kaiser HMO, Multi-Choice, PPO, Senior Advantage Effective 1/01/2020
    Kaiser Permanente Georgia Region Provider Targeted Review List Kaiser HMO, Multi-Choice, PPO, Senior Advantage Effective 1/01/2020 The Affiliated Community Physician is responsible for verification of member eligibility and benefit coverage by logging on to KP Online-Affiliate or calling Member Services at 404-261- 2590. Failure to obtain authorization prior to providing the services listed below will result in a denial of payment. A medical necessity review decision will be issued to your office and to the member upon completion of the review process. Receipt of complete clinical information with the initial request will expedite the process. Authorization for approval of services based on medical necessity is never a guarantee of payment which must also meet the criteria of member eligibility and benefit availability. The Kaiser Permanente Quality Resource Management Department requires authorization of all post stabilization care including observation and inpatient admissions. Please call our Emergency Care Management hub at 404-365-4254 for authorization. For emergency authorization of home health or durable medical services after regular business hours, contact 404-365-0966 or 800-611-1811 to speak with the QRM case manager on call. Hours of Operation: Quality Resource Management Department Monday through Friday 8:00 am – 5:00 pm 404-364-7320/800-221-2412 Member Services Department Monday through Friday 7:00 am – 7:00 pm 404-261-2590 Emergency Care Management Hub Available 24/7 404-365-4254 Medical necessity review is required for the following services/conditions: HMO members require QRM review and approval for reimbursement of any non- contracted, out of network (office based, outpatient, inpatient) services.
    [Show full text]
  • Challenges and Approaches for the Development of Safer Immunomodulatory Biologics
    REVIEWS Challenges and approaches for the development of safer immunomodulatory biologics Jean G. Sathish1*, Swaminathan Sethu1*, Marie-Christine Bielsky2, Lolke de Haan3, Neil S. French1, Karthik Govindappa1, James Green4, Christopher E. M. Griffiths5, Stephen Holgate6, David Jones2, Ian Kimber7, Jonathan Moggs8, Dean J. Naisbitt1, Munir Pirmohamed1, Gabriele Reichmann9, Jennifer Sims10, Meena Subramanyam11, Marque D. Todd12, Jan Willem Van Der Laan13, Richard J. Weaver14 and B. Kevin Park1 Abstract | Immunomodulatory biologics, which render their therapeutic effects by modulating or harnessing immune responses, have proven their therapeutic utility in several complex conditions including cancer and autoimmune diseases. However, unwanted adverse reactions — including serious infections, malignancy, cytokine release syndrome, anaphylaxis and hypersensitivity as well as immunogenicity — pose a challenge to the development of new (and safer) immunomodulatory biologics. In this article, we assess the safety issues associated with immunomodulatory biologics and discuss the current approaches for predicting and mitigating adverse reactions associated with their use. We also outline how these approaches can inform the development of safer immunomodulatory biologics. Immunomodulatory Biologics currently represent more than 30% of licensed The high specificity of the interactions of immu- biologics pharmaceutical products and have expanded the thera- nomodulatory biologics with their relevant immune Biotechnology-derived peutic options available
    [Show full text]
  • Evaluation of Antibody Properties and Clinically Relevant Immunogenicity
    Drug Safety https://doi.org/10.1007/s40264-018-00788-w ORIGINAL RESEARCH ARTICLE Evaluation of Antibody Properties and Clinically Relevant Immunogenicity, Anaphylaxis, and Hypersensitivity Reactions in Two Phase III Trials of Tralokinumab in Severe, Uncontrolled Asthma Mats Carlsson1 · Martin Braddock2 · Yuling Li3 · Jihong Wang3 · Weichen Xu3 · Nicholas White4 · Ayman Megally5 · Gillian Hunter6 · Gene Colice5 © The Author(s) 2019 Abstract Introduction Tralokinumab is a monoclonal antibody (mAb) that neutralizes interleukin (IL)-13, a cytokine involved in the pathogenesis of asthma. Objective The objectives of this study were to characterize the potential immunogenic properties of tralokinumab and report data for anti-drug antibodies (ADAs) and hypersensitivity reactions from two phase III clinical trials. Methods The oligosaccharide structure of tralokinumab, Fab-arm exchange, and ADAs were characterized by standard techniques. Hypersensitivity adverse events (AEs) were evaluated in two pivotal clinical trials of tralokinumab in severe, uncontrolled asthma: STRATOS 1 and 2 (NCT02161757 and NCT02194699). Results No galactose-α-1,3-galactose (α-Gal) epitopes were found in the Fab region of tralokinumab and only 4.5% of glycoforms contained α-Gal in the Fc region. Under non-reducing conditions, Fab-arm exchange did not take place with another immunoglobulin (Ig) G­ 4 mAb (mavrilimumab). However, following glutathione reduction, a hybrid antibody with monovalent bioactivity was detected. ADA incidences (titers) were as follows: STRATOS 1—every 2 weeks (Q2 W) 0.8% (26.0), every 4 weeks (Q4 W) 0.5% (26.0), placebo 0.8% (52.0); STRATOS 2—Q2 W 1.2% (39.0), placebo 0.8% (13.0). Participant-reported hypersensitivity AE rates were as follows: STRATOS 1—Q2 W 25.9%, Q4 W 25.0%, placebo 25.5%; STRATOS 2—Q2 W 13.2%, placebo 9.0%.
    [Show full text]
  • WHO EML Application Tocilizumab
    WHO EML Application Tocilizumab Condition Juvenile Idiopathic Arthritis 1 Summary statement of the proposal for inclusion. The application proposes the inclusion of Tocilizumab on the complementary list of the EML/EMLc/both for the treatment of Systemic Onset Juvenile Idiopathic Arthritis (SOJIA). The rationale for the complementary list is that the use of this drug requires specialised care. The proposed listing on both the EML and EMLc reflects the fact that JIA affects children through adolescence and into adulthood. This rationale is consistent with the listing for the anti-TNF biologics currently listed for JIA. Juvenile Idiopathic Arthritis (JIA) is the most common chronic rheumatic disease of childhood, affecting approximately one per 1000 children (1, 2). JIA is characterised by joint inflammation of more than 6 weeks’ duration, with onset before age sixteen years and where no other cause is found (2). JIA is an autoimmune, non-infective, inflammatory joint disease, the cause of which remains poorly understood with both genetic and environmental contributions (3). It is a distinct entity from rheumatoid arthritis, differing in clinical presentations, prognosis, disease outcomes and treatment approaches. The age of onset in JIA is typically young, with a peak incidence between 1-3 years of age, although the disease persists into adulthood in approximately 50% of cases (4). Even in patients in whom the inflammatory disease resolves, joint or extra-articular damage – with associated disability – are common and if not treated then can result in irreversible sequelae and impact on quality of life (5). Current treatment approaches for children with JIA aim for normal physical and psychosocial functioning, and with access to modern treatments, good outcomes are a realistic and achievable goal for many children with this condition (6) .
    [Show full text]
  • Collaborations on Imaging – the Medimmune’S Innovative Way
    Collaborations on Imaging – The Medimmune’s Innovative Way Jerry Wu, PhD, Medimmune Developments in Healthcare Imaging – Connecting with Industry 18th October 2017 Contents 1 A brief overview of Medimmune 2 Scientific collaborations 3 Scientific Interest Group for Imaging 2 A brief overview of Medimmune Global Biologics Research and Development Arm of ~2,200 Employees in the US and UK Robust pipeline of 120+ Biologics in Research & Development with 40+ projects in Clinical Stage Development California Gaithersburg Cambridge 3 Medimmune – Biologics arm of AstraZeneca Late-stage Discovery and Early Development Development Innovative Medicines and Early Development Unit (Small Molecules) Global Internal and Collaboration and Medicines external combinations Development Market opportunities MedImmune (Biologics) 4 Current therapeutic areas Respiratory, Inflammation and Cardiovascular and Infectious Disease Oncology Autoimmunity Metabolic Disease Neuroscience Main Therapeutic Areas Opportunity-driven Protein Biologics Small Molecules Immuno-therapies Devices Engineering 5 RESPIRATORY, INFLAMMATION AND AUTOIMMUNITY ONCOLOGY (RIA) Medimmune R&D pipeline INFECTIOUS DISEASE (ID), NEUROSIENCE AND CARDIOVASCULAR AND METABOLIC DISEASE (CVMD) GASTROINTESINAL DISEASE PHASE 1 PHASE 2 PHASE 2 PIVOTAL/PHASE 3 Durvalumab + MEDI-573 Durvalumab MEDI-565 MEDI0562 MEDI4276 Durvalumab MEDI0680 Metastatic ≥2nd Line Advanced Solid Tumors Solid Tumors Solid Tumors Stage III NSCLC Solid Tumors Breast Cancer Bladder Cancer Durvalumab/AZD5069/ Durvalmab + MEDI0680
    [Show full text]
  • Specialty Guideline Management
    Reference number 1800-A SPECIALTY GUIDELINE MANAGEMENT ARCALYST (rilonacept) POLICY I. INDICATIONS The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy. FDA-Approved Indications Treatment of Cryopyrin Associated Periodic Syndromes (CAPS), including Familial Cold Auto-inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) in adults and children 12 years of age and older. All other indications are considered experimental/investigational and not medically necessary. II. CRITERIA FOR INITIAL APPROVAL Cryopyrin-associated periodic syndrome (CAPS) Authorization of 12 months may be granted for treatment of CAPS when all of the following criteria are met: A. Member has a diagnosis of familial cold auto-inflammatory syndrome (FCAS) with classic signs and symptoms (i.e., recurrent, intermittent fever and rash that were often exacerbated by exposure to generalized cool ambient temperature) or Muckle-Wells syndrome (MWS) with classic signs and symptoms (i.e., chronic fever and rash of waxing and waning intensity, sometimes exacerbated by exposure to generalized cool ambient temperature). B. Member has functional impairment limiting the activities of daily living. III. CONTINUATION OF THERAPY Authorization of 12 months may be granted for all members (including new members) who are using the requested medication for an indication outlined in Section II and who achieve or maintain positive
    [Show full text]
  • Study Protocol
    2016N278580_02 CONFIDENTIAL GlaxoSmithKline group of companies 201789 TITLE PAGE Division: Worldwide Development Information Type: Protocol Amendment Title: A Phase 1/2, Double-Blind, Placebo-Controlled Study of the Pharmacokinetics, Safety and Tolerability of GSK3196165 in Combination with Methotrexate Therapy, in Japanese Subjects with Active Moderate-Severe Rheumatoid Arthritis Despite Treatment with Methotrexate. Compound Number: GSK3196165 Development Phase I/II Effective Date: 19- MAY-2017 Protocol Amendment Number: 02 Author(s): PPD Revision Chronology: GlaxoSmithKline Date Version Document Number 2016N278580_00 17-AUG-2016 Original 2016N278580_01 26-SEP-2016 Amendment Number 01 Revision contents: This amendment addresses PMDA modifications requested during the clinical trial notification process. It includes an additional Inclusion Criterion for FVC in Section 5.1; additional Exclusion Criterion and Stopping Criterion for HBV-DNA for subjects with positive anti-HBs antibody in Section 5.2 and Section 5.4; addition of a preventive dose of co-trimoxazole in Section 4.6.1 and Section 6.10.2.2; addition of HBV-DNA test at Screening and addition of footnote for clarification in Section 7.1; addition of “past week’s pain” in Section 12.6.2.1; correction of analysis populations in Section 9.3.1; and deletion of unapproved contraception methods in Japan in Section 12.2. 2016N278580_02 19-MAY-2017 Amendment Number 02 Revision contents: Change of Inclusion Criterion for CRP in Section 5.1. Copyright 2017 the GlaxoSmithKline group of companies. All rights reserved. Unauthorised copying or use of this information is prohibited. 1 2016N278580_02 CONFIDENTIAL 201789 SPONSOR SIGNATORY: Kihito Takahashi Date Vice President, Head of Development and Medical Affairs Division, GlaxoSmithKline K.K.
    [Show full text]
  • Prior Authorization Policy
    PRIOR AUTHORIZATION POLICY POLICY: Inflammatory Conditions – Arcalyst® (rilonacept for subcutaneous injection Regeneron Pharmaceuticals) DATE REVIEWED: 11/06/2019; selected revision 04/01/2020 OVERVIEW Arcalyst is an interleukin-1 (IL-1) blocker indicated for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) in adults and children aged 12 years and older.1 Arcalyst, also known is a recombinant dimeric fusion protein that blocks IL-1β signaling and to a lesser extent also binds IL-1α and IL-1 receptor antagonist (IL-1ra). In adults ≥ 18 years of age, Arcalyst is initiated with a loading dose of 320 mg delivered as two subcutaneous (SC) injections of 160 mg on the same day at two separate sites. Dosing is continued with 160 mg once weekly as a single injection. In adolescents aged 12 to 17 years, therapy is initiated with a loading dose of 4.4 mg/kg, up to a maximum of 320 mg, delivered as one or two SC injections with a maximum single-injection volume of 2 mL. If the initial dose is two injections, then patients should be given Arcalyst on the same day at two separate sites. In adolescents, dosing is continued with 2.2 mg/kg, up to a maximum of 160 mg, once weekly as a single injection. Disease Overview CAPS is a rare inherited inflammatory disease associated with overproduction of IL-1. CAPS encompasses three rare genetic syndromes. FCAS, MWS, and neonatal onset multisystem inflammatory disorder (NOMID) or chronic infantile neurological cutaneous and articular syndrome (CINCA) are thought to be one condition along a spectrum of disease severity.2-3 FCAS is the mildest phenotype and NOMID is the most severe.
    [Show full text]
  • Promising Therapeutic Targets for Treatment of Rheumatoid Arthritis
    REVIEW published: 09 July 2021 doi: 10.3389/fimmu.2021.686155 Promising Therapeutic Targets for Treatment of Rheumatoid Arthritis † † Jie Huang 1 , Xuekun Fu 1 , Xinxin Chen 1, Zheng Li 1, Yuhong Huang 1 and Chao Liang 1,2* 1 Department of Biology, Southern University of Science and Technology, Shenzhen, China, 2 Institute of Integrated Bioinfomedicine and Translational Science (IBTS), School of Chinese Medicine, Hong Kong Baptist University, Hong Kong, China Rheumatoid arthritis (RA) is a systemic poly-articular chronic autoimmune joint disease that mainly damages the hands and feet, which affects 0.5% to 1.0% of the population worldwide. With the sustained development of disease-modifying antirheumatic drugs (DMARDs), significant success has been achieved for preventing and relieving disease activity in RA patients. Unfortunately, some patients still show limited response to DMARDs, which puts forward new requirements for special targets and novel therapies. Understanding the pathogenetic roles of the various molecules in RA could facilitate discovery of potential therapeutic targets and approaches. In this review, both Edited by: existing and emerging targets, including the proteins, small molecular metabolites, and Trine N. Jorgensen, epigenetic regulators related to RA, are discussed, with a focus on the mechanisms that Case Western Reserve University, result in inflammation and the development of new drugs for blocking the various United States modulators in RA. Reviewed by: Åsa Andersson, Keywords: rheumatoid arthritis, targets, proteins, small molecular metabolites, epigenetic regulators Halmstad University, Sweden Abdurrahman Tufan, Gazi University, Turkey *Correspondence: INTRODUCTION Chao Liang [email protected] Rheumatoid arthritis (RA) is classified as a systemic poly-articular chronic autoimmune joint † disease that primarily affects hands and feet.
    [Show full text]
  • Rheumatoid Arthritis) – Forecast and Market Analysis to 2023
    REFERENCE CODE GDHC509DFR | PUBLICAT ION DATE DECEMBER 2014 MAVRILIMUMAB (RHEUMATOID ARTHRITIS) – FORECAST AND MARKET ANALYSIS TO 2023 MAVRILIMUMAB (RHEUMATOID ARTHRITIS) – FORECAST AND MARKET ANALYSIS TO 2023 Executive Summary The table below presents the key metrics for The major driver for the growth of Mavrilimumab in Mavrilimumab in the 10MM Rheumatoid Arthritis the RA market over the forecast period is: (RA) pharmaceutical markets (US, France, Potential to be a first-in-class therapy. Germany, Italy, Spain, UK, Japan, Australia, China, India) in 2023. Major barrier to the growth of Mavrilimumab in the RA market over the forecast period is: Mavrilimumab: Key Metrics in the 10 Major Pharmaceutical Markets Crowded market, with multiple new entrants Level of Key Events (2013–2023) targeting the same patient population of TNF- Impact Launch of AstraZeneca’s mavrilimumab in inadequate responders. ↑↑ 2020 across the 6MM 2023 Market Sales The figure below illustrates the global US $306.0m Mavrilimumab sales by region during the forecast 5EU $60.4m period. Japan N/A Australia N/A Sales for Mavrilimumab by Region, 2023 China N/A 2023 India N/A Total: $366.3m Total $366.3m 16% Source: GlobalData 10MM = US, France, Germany, Italy, Spain, UK, Japan, Australia, China, and India 6MM = US, France, Germany, Italy, Spain, and UK US 5EU = France, Germany, Italy, Spain, and UK N/A = Not Available 5EU Sales for Mavrilimumab in the Rheumatoid Arthritis Market 84% GlobalData estimates sales of Mavrillimumab at Source: GlobalData the end of the forecast period in 2023, in the US & 5EU at $366.3 million increasing from $145.3 million in 2020.
    [Show full text]
  • Targeting GM-CSF in Rheumatoid Arthritis A.B
    Targeting GM-CSF in rheumatoid arthritis A.B. Avci1, E. Feist2, G.R. Burmester2 1Department of Internal Medicine, ABSTRACT well-known as a haemopoietic growth Rheumatology, Akdeniz University, Granulocyte-macrophage colony-stim- factor used to treat neutropenia follow- Faculty of Medicine, Antalya, Turkey; ulating factor (GM-CSF) is well-known ing chemotherapy. It was a long-time 2Department of Rheumatology and as a haemopoietic growth factor. How- concern that targeted therapies against Clinical Immunology, Charite-University Medicine Berlin, Berlin, Germany. ever, it is also essential in regulating this cytokine could cause severe side functions of mature myeloid cells such effects such as neutropenia or pulmo- Ali Berkant Avci, MD, Assoc. Prof. Eugen Feist, PD Dr as macrophages. Preclinical studies nary alveolar proteinosis. Therefore, Gerd-Rüdiger Burmester, Prof. Dr. and observations of flares of arthritis in during the early development phase Please address correspondence to: patients following GM-CSF treatment of compounds targeting GM-CSF or Ali Berkant Avci, MD, supported its important contribution to its receptor special attention was paid Akdeniz Üniversitesi Hastanesi, the pathogenesis of rheumatoid arthritis to this potential adverse event reveal- İç Hastalıkları AD Romatoloji BD, (RA). As the most advanced compound, ing no evidence for such an associated Kampüs 07059 Konyaaltı/Antalya, mavrilimumab, a monoclonal antibody risk profile. On the other hand the so Turkey. against GM-CSF receptor, has already far available results clearly showed E-mail:[email protected] completed phase II trials with a long rapid and sustained effects on disease Received and accepted on June 29, 2016. term of follow-up period of 74 weeks.
    [Show full text]