Canakinumab

关注并星标我们 1月24日，金赛药业宣布收到国家药品监督管理局核准签发的《受理通知书》，旗下产品伏欣奇拜单抗注射液（曾用名：金纳单抗注射液）上市申请获国家药品监督管理局受理，拟用于成年痛风性关节炎急性发作患者。 痛风性关节炎（GA）是一种单钠尿酸盐沉积在关节所致的晶体相关性关节病，可分为发作期、发作间歇期和慢性痛风性关节炎期，其中发作期的典型表现是关节剧烈疼痛，起病急骤，且疼痛进行性加重。 IL-1β是急性痛风性关节炎发作时的关键促炎因子。研究发现，急性痛风的发作需要两个信号共同激活，使未活化的pro-IL-1β成为活化的IL-1β。一是启动信号：由巨噬细胞感受到内源性危险信号PAMPs触发巨噬细胞膜上的TLR-2和TLR-4所识别，激活NF-κB，从而引起前体IL-1β的转录，储备大量痛风发作最重要的前炎性因子Pro-IL-1β。二是激活信号：巨噬细胞吞噬msu后激活NLRP3，ASC和前半胱氨酸天冬氨酸蛋白酶1（caspase-1）募集，激活的caspase-1会切割未活化的pro-IL-1β，使其成为活化的IL-1β。活化的IL-1β与IL-1R结合，导致血管扩张和趋化中性粒细胞募集在MSU析出部位，靶细胞持续激活，导致更多下游炎症因子的释放或蛋白的表达，如TNF，IL-6等，从而驱动扩大炎症发作。 近年来，GA发病率逐渐上升并趋向于年轻化。据《2021年中国高尿酸及痛风趋势白皮书》，中国高尿酸血症患者约有1.77亿，成为仅次于糖尿病的第二大代谢类疾病，其中GA患者超过1466万人。 目前，GA发作期一线治疗药物推荐秋水仙碱和非甾体类抗炎药（NSAIDs），当存在治疗禁忌或治疗效果不佳时，可考虑短期应用糖皮质激素抗炎治疗。但对于发作频繁，或对NSAIDs和/或秋水仙碱禁忌、不耐受及缺乏疗效，以及不适合反复使用类固醇激素的GA患者，目前国内没有合适的治疗药物供临床选择。 伏欣奇拜单抗是金赛药业研发的一款抗白介素 1β（IL-1β）单抗，通过阻断IL-1β与IL-1受体I（IL-1RI）和IL-1受体II（IL-1RII）结合起到调节炎症的作用。 据药物临床试验登记与信息公示平台，伏欣奇拜单抗在国内登记多项临床试验，适应症涉及痛风性关节炎、结缔组织病相关的间质性肺病、全身型幼年特发性关节炎等。 金赛药业针对伏欣奇拜单抗开发了冻干粉针和注射液两种剂型。2024年4月，注射用伏欣奇拜单抗（曾用名：注射用金纳单抗）针对痛风性关节炎的新药上市申请获CDE受理。 对比冻干粉针，注射液免去注射前需要复溶与配置的步骤，更方便临床使用。已完成的一项伏欣奇拜单抗在中国健康成年男性受试者中的随机、双盲、单次给药的生物等效性临床试验结果显示：伏欣奇拜单抗注射液和注射用伏欣奇拜单抗具有生物等效性，且安全性良好。 目前，国内NMPA尚未批准抗IL-1β单抗用于治疗GA急性发作，伏欣奇拜单抗有望成为国内首款获批治疗GA急性发作的抗IL-1β单抗。而且，伏欣奇拜单抗或可成为IL-1β单克隆抗体中的“Best-in-class”。 2024年美国风湿病学会（ACR）上公布的最新3期临床研究GUARD-1结果显示：对NSAIDs和/或秋水仙碱禁忌、不耐受或缺乏疗效的，以及不适合反复使用类固醇激素的成年GA急性发作的患者，伏欣奇拜单抗在痛风发作急性期更安全，缓解急性GA发作时的疼痛方面非劣于复方倍他米松；在缓解期能更有效的延缓痛风的急性复发，在用药12周及24周内可显著降低患者痛风复发的风险（分别降低90%和87%），显著延长患者痛风急性复发的时间。 在国外，诺华抗IL-1β单抗卡那奴单抗于2013年3月被批准用于治疗GA急性发作。在国内，卡那奴单抗治疗急性痛风的临床试验处于3期阶段，但已主动终止。值得一提的是，卡那单抗在国外获批治疗急性GA基于的两项为期12周的双盲主动对照试验（β-RELIEVED研究和β-RELIEVED-II研究）选取的阳性对照药物是曲安奈德，该药是一款中效激素，而伏欣奇拜单抗选取的阳性对照药物复方倍他米松是一款速效及长效的激素（含长效二丙酸倍他米松及速效的倍他米松磷酸钠）。 此外，目前国内还有几款抗IL-1β单抗被开发用于治疗GA的急性发作。其中三生国健的SSGJ-613是其研发的具有全新可变区序列的抗IL-1β抗体，已公布的2期临床试验结果显示其在急性GA疼痛缓解和预防复发方面疗效确切，且安全性、耐受性良好。目前，SSGJ-613治疗急性GA的3期临床研究（SSGJ-613-AG-III-01）已经启动，而该试验选取二丙酸倍他米松+倍他米松磷酸酯为阳性对照药。 【智药研习社近期直播】 扫码领取CPHI & PMEC China 2025展会门票 来源：CPHI制药在线 声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。 投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~

Leiden, the Netherlands, January 21, 2025: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces today that the Board of Directors has nominated Fabrice Chouraqui to become Pharming’s new Executive Director and Chief Executive Officer, succeeding Sijmen de Vries. Pharming will nominate Mr. Chouraqui for the appointment as Executive Director and Chief Executive Officer for a term of four years at an upcoming Extraordinary General Meeting of Shareholders (EGM). Information regarding the EGM, including the notice to convene, will be shared shortly in a separate press release. Upon the appointment of Mr. Chouraqui, Sijmen de Vries will resign from the Board of Directors. To ensure a smooth hand-over of tasks and responsibilities, Mr. de Vries will remain a strategic advisor to the new CEO until December 31, 2025. Dr. Richard Peters, Chairman of the Board of Directors, commented:“After an extensive search, the Board and I are confident that Fabrice Chouraqui is the right candidate to lead Pharming and continue to implement our growth strategy, shaping Pharming into the rare disease company of choice. Fabrice is a deeply experienced global pharmaceutical and biotechnology executive, who brings a wealth of profound global expertise and experience, across the entire biopharmaceutical value chain, to Pharming. On behalf of the entire Board of Directors, I would like to thank Sijmen de Vries for his great commitment to Pharming over the past 16 years and for creating the company that it is today, serving patients and paving the way for delivery on the company’s strategy for growth. We are grateful that Sijmen will continue to be available through the end of the year to ensure a smooth hand-over with Fabrice.” Sijmen de Vries, Chief Executive Officer, commented:“I am very pleased with the nomination of Fabrice Chouraqui as my successor. Fabrice brings strong leadership experience with global pharmaceutical companies and highly innovative biotechnology companies to Pharming. To make way for Fabrice to take over the executive leadership of the Company, I will resign from the Board of Directors upon the appointment of Fabrice by our shareholders. I look forward to supporting Fabrice, working closely together through the remainder of the year, and ensuring a smooth hand-over.” Fabrice Chouraqui commented:“I am honored to have the opportunity to take over the leadership of Pharming, building on the strong foundation and joining at a time when I can help propel the company through its next stage of growth in the rare disease space. I am impressed by Pharming’s growing portfolio of products and commercial opportunities which will pave the way for us to become the rare disease company of choice.” ProfileMr. Fabrice Chouraqui (date of birth: August 1, 1970, French national, U.S. citizen) is a global pharmaceutical executive with a record of value creation at Flagship Pioneering, Novartis and Bristol-Myers Squibb. A purpose-driven leader with a passion for bringing innovative treatments to patients, Mr. Chouraqui has a reputation for building strong followership in organizations. He is scientifically minded, with a deep understanding across the business spectrum, from research and development to access and commercialization, including strong expertise in investor management and business development. Across his career, Mr. Chouraqui has led the launch of treatments in many therapeutic areas, including highly targeted specialty care drugs and large primary care medicines. He consistently demonstrates strategic and innovative thinking to achieve long-lasting results and has a strong record of developing next generation talents and highly diverse teams. Until recently, Mr. Chouraqui was a CEO-Partner at Flagship Pioneering, one of the largest Biotech Venture Capital firms in the US. Mr. Chouraqui also served as CEO of Cellarity, Inc., one of the companies created by Flagship Pioneering. In his CEO capacity, Mr. Chouraqui was leading the development of Cellarity, a company which is driving a radically new approach to drug discovery by working at the intersection of AI and biology. Mr. Chouraqui raised over $250M and attracted world-renowned investors. He initiated a collaboration with Novo Nordisk and had Cellarity recognized as a top biotech to watch by several sources. Today, Mr. Chouraqui holds the position of Executive Chairman at Cellarity. Prior to joining Flagship Pioneering, Mr. Chouraqui spent 10 years at Novartis. He was President of Novartis Pharmaceuticals USA from 2016 to 2019, during which he delivered strong growth despite negative price impact and significantly improved profitability. Promoted to turn around performance after the business suffered issues with recent launches, Mr. Chouraqui developed sophisticated commercial and access capabilities to meet the new market environment. He achieved strong market positions for two significant growth drivers (Cosentyx and Entresto), drove the uptake of the rare disease drug Ilaris, initiated the landmark PIONEER clinical trial in heart failure, and championed several business development initiatives. Mr. Chouraqui moved the organization’s culture from “consensus building, conservative and siloed” to “questioning, experimenting and collaborative.” He also served as Novartis representative on the board of BIO. Before leading the US organization, Mr. Chouraqui was President, Latin America & Canada (2014-2016) for Novartis Pharmaceuticals, responsible for leading a portfolio shift from established primary care medicines to innovative specialty care drugs. He also served as Global Head, Business Franchise Neuroscience (2012-2014). In this role, he relaunched Gilenya (Multiple Sclerosis) following a post-launch regulatory review and made Gilenya the largest growth contributor in the Novartis five-year strategic plan. Mr. Chouraqui also addressed late-stage pipeline gaps with the start of the siponimod program in Secondary Progressive Multiple Sclerosis (approved in 2019) and the identification of business development opportunities, which led to the in-licensing of ofatumumab in MS from GSK (launched as Kesimpta) and a partnership with Amgen on erenumab for migraine (launched as Aymovig). From 2000 to 2010, Mr. Chouraqui held multiple international roles at Bristol-Myers Squibb, ultimately serving as Vice President, Commercial Operations, Asia-Pacific and General Manager, Southeast Asia (2008-2010). He began his career in R&D roles at Roussel Uclaf and Hoechst Marion Roussel, pharmaceutical companies that were predecessors to today’s Sanofi. Mr. Chouraqui earned a MBA from INSEAD and a Doctorate in Pharmacy, a Post-Graduate Degree in Quality Assurance of Medicines, and a MSc in Biological and Medicinal Sciences from University of Paris V. Mr. Chouraqui shall have stepped down as Executive Chairman of Cellarity, Inc., a non-listed company, no later than upon his appointment as the new Executive Director and Chief Executive Officer of Pharming. Thereafter, Mr. Chouraqui intends to serve as a non-executive Board member of Cellarity. Mr. Chouraqui also holds the position of independent Board member of OranoMed, a non-listed (and therefore private) subsidiary of Orano Group. OranoMed is a company specialized in the research and development of Pb-212 based radioligand alpha therapies for the treatment of various types of cancers. The resume of Mr. Chouraqui is available on our website. For further public information, contact:Pharming Group, Leiden, the NetherlandsMichael Levitan, VP Investor Relations & Corporate CommunicationsT: +1 (908) 705 1696E: investor@pharming.com FTI Consulting, London, UKVictoria Foster Mitchell/Alex Shaw/Amy ByrneT: +44 203 727 1000 LifeSpring Life Sciences Communication, Amsterdam, the NetherlandsLeon MelensT: +31 6 53 81 64 27E: pharming@lifespring.nl About Pharming Group N.V. Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific. For more information, visit www.pharming.com and find us on LinkedIn. Forward-Looking Statements   This press release may contain forward-looking statements. Forward-looking statements are statements of future expectations that are based on management’s current expectations and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance, or events to differ materially from those expressed or implied in these statements. These forward-looking statements are identified by their use of terms and phrases such as “aim”, “ambition”, ‘‘anticipate’’, ‘‘believe’’, ‘‘could’’, ‘‘estimate’’, ‘‘expect’’, ‘‘goals’’, ‘‘intend’’, ‘‘may’’, “milestones”, ‘‘objectives’’, ‘‘outlook’’, ‘‘plan’’, ‘‘probably’’, ‘‘project’’, ‘‘risks’’, “schedule”, ‘‘seek’’, ‘‘should’’, ‘‘target’’, ‘‘will’’ and similar terms and phrases. Examples of forward-looking statements may include statements with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory, commercial, competitive and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2023 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. All forward-looking statements contained in this press release are expressly qualified in their entirety by the cautionary statements contained or referred to in this section. Readers should not place undue reliance on forward-looking statements. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release. Pharming does not undertake any obligation to publicly update or revise any forward-looking statement as a result of new information, future events or other information. Inside InformationThis press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation. Attachments Fabrice Chouraqui Pharming to nominate new Executive Director and Chief Executive Officer_EN_21JAN25