IL-17RA

Application in moderate-to-severe hidradenitis suppurativa based on results from two Phase 3 studies where BIMZELX® (bimekizumab-bkzx) showed clinically meaningful improvements vs. placebo at Week 16 which were sustained to Week 48 Application for the additional BIMZELX 2mL device presentations aims to provide more options to optimize the individual patient experience ATLANTA, April 4, 2024 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental Biologics License Application (sBLA) for BIMZELX® (bimekizumab-bkzx), an IL-17A and IL-17F inhibitor, for the treatment of adults with moderate-to-severe hidradenitis suppurativa (HS). In addition, a second sBLA for the BIMZELX 2mL device presentations has also been accepted. "We are excited to share progress on our FDA applications. The most recent sBLA seeks approval for BIMZELX in moderate-to-severe hidradenitis suppurativa, and is aligned to our goal of expanding the reach of BIMZELX to more patients living with IL-17–mediated diseases," said Emmanuel Caeymaex, Executive Vice President, Immunology Solutions, and Head of U.S., UCB. "In addition, the sBLA for the 2mL device presentations aims to offer increased convenience for patients. Today, one dose of BIMZELX in moderate-to-severe plaque psoriasis is administered as two 1mL injections. Approval of the 2mL device presentations would mean that patients would have an alternative one-injection regimen option." These new regulatory milestones represent two of five sBLAs accepted by the FDA for BIMZELX this year, following the previously announced applications in psoriatic arthritis (PsA), non-radiographic axial spondyloarthritis (nr-axSpA) and ankylosing spondylitis (AS). BIMZELX was approved in the U.S. in October 2023 for the treatment of moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy.1 BIMZELX is not approved in the U.S. for the treatment of moderate-to-severe HS, PsA, nr-axSpA, and AS, or for the 2mL device presentation. In the U.S., the efficacy and safety of BIMZELX in the treatment of moderate-to-severe HS, PsA, nr-axSpA, and AS have not been established and these are investigational indications. The sBLA in moderate-to-severe HS is supported by data from the Phase 3 BE HEARD I and BE HEARD II studies where BIMZELX demonstrated clinically meaningful improvements in HiSCR50 vs. placebo at Week 16, the primary endpoint.2 A greater proportion of patients treated with BIMZELX vs. placebo also achieved HiSCR75 at Week 16, a key secondary endpoint.2 In addition, over 48 weeks, improvements increased for patients in these studies.2 The safety profile of BIMZELX was consistent with previous studies with no new safety signals observed.2 The sBLA for the additional device presentations seeks approval of BIMZELX 2mL safety syringe and 2mL autoinjector with the aim of providing a second option to the currently approved 1mL presentations. Notes to editors: About Hidradenitis Suppurativa (HS) Hidradenitis suppurativa (HS) is a chronic, recurring, painful, and debilitating inflammatory skin disease.3,4 The main symptoms are nodules, abscesses, and pus-discharging tunnels (channels leading out of the skin), which typically occur in the armpits, groin, and buttocks.3,4 People with HS experience flare-ups of the disease as well as severe pain, which can have a major impact on quality of life.3,4 HS most commonly develops in early adulthood and affects approximately one percent of the population in most studied countries.3,4 Approximately one-third of people with HS have a family history of HS, and lifestyle factors such as smoking and obesity can also play a crucial role in the clinical course of HS.3,4 The symptoms of pain, discharge, and scarring are not only a physical burden. People with HS also experience stigma: worrying about or directly experiencing negative attitudes and reactions from society in response to their symptoms.5 These feelings can lead to embarrassment, social isolation, low self-esteem, and sexual life impairment, and impact all areas of life, including interpersonal relationships, education, and work.6 About BE HEARD I and BE HEARD II BE HEARD I and BE HEARD II are randomized, double-blind, placebo-controlled, parallel group, multicenter, Phase 3 studies designed to evaluate the efficacy and safety of bimekizumab in adults with moderate-to-severe hidradenitis suppurativa (HS).7,8 The two studies had a combined enrollment of 1,014 participants with a diagnosis of moderate-to-severe HS. 7,8 The primary endpoint in both trials was HiSCR50 at Week 16. 7,8 A key secondary endpoint was HiSCR75 at Week 16. HiSCR50 and HiSCR75 are defined as at least either a 50 or 75 percent reduction from baseline in the total abscess and inflammatory nodule count, with no increase from baseline in abscess or draining tunnel count. 7,8 A bout BIMZELX (bimekizumab-bkzx) Bimekizumab is a humanized IgG1 monoclonal antibody that selectively binds to IL-17A, IL-17F and IL-17AF cytokines, blocking their interaction with the IL-17RA/IL-17RC receptor complex.1 In the U.S., BIMZELX is approved for the treatment of moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy.1 BIMZELX is not approved in the U.S. for the treatment of moderate-to-severe hidradenitis suppurativa (HS), PsA, nr-axSpA and AS, or for the 2mL device presentation. In the U.S., the efficacy and safety of BIMZELX in the treatment of moderate-to-severe HS, PsA, nr-axSpA, and AS have not been established, and these are investigational indications. BIMZELX is not approved in HS by any regulatory authority worldwide. Please see Important Safety Information below and full U.S. prescribing information at . BIMZELX U.S. IMPORTANT SAFETY INFORMATION 1 Suicidal Ideation and Behavior BIMZELX® (bimekizumab-bkzx) may increase the risk of suicidal ideation and behavior (SI/B). A causal association between treatment with BIMZELX and increased risk of SI/B has not been established. Prescribers should weigh the potential risks and benefits before using BIMZELX in patients with a history of severe depression or SI/B. Advise monitoring for the emergence or worsening of depression, suicidal ideation, or other mood changes. If such changes occur, advise to promptly seek medical attention, refer to a mental health professional as appropriate, and re-evaluate the risks and benefits of continuing treatment. Infections BIMZELX may increase the risk of infections. Do not initiate treatment with BIMZELX in patients with any clinically important active infection until the infection resolves or is adequately treated. In patients with a chronic infection or a history of recurrent infection, consider the risks and benefits prior to prescribing BIMZELX. Instruct patients to seek medical advice if signs or symptoms suggestive of clinically important infection occur. If a patient develops such an infection or is not responding to standard therapy, monitor the patient closely and do not administer BIMZELX until the infection resolves. Tuberculosis Evaluate patients for tuberculosis (TB) infection prior to initiating treatment with BIMZELX. Avoid the use of BIMZELX in patients with active TB infection. Initiate treatment of latent TB prior to administering BIMZELX. Consider anti-TB therapy prior to initiation of BIMZELX in patients with a past history of latent or active TB in whom an adequate course of treatment cannot be confirmed. Closely monitor patients for signs and symptoms of active TB during and after treatment. Liver Biochemical Abnormalities Elevated serum transaminases were reported in clinical trials with BIMZELX. Test liver enzymes, alkaline phosphatase and bilirubin at baseline, periodically during treatment with BIMZELX and according to routine patient management. If treatment-related increases in liver enzymes occur and drug-induced liver injury is suspected, interrupt BIMZELX until a diagnosis of liver injury is excluded. Permanently discontinue use of BIMZELX in patients with causally associated combined elevations of transaminases and bilirubin. Avoid use of BIMZELX in patients with acute liver disease or cirrhosis. Inflammatory Bowel Disease Cases of inflammatory bowel disease (IBD) have been reported in patients treated with IL-17 inhibitors, including BIMZELX. Avoid use of BIMZELX in patients with active IBD. During BIMZELX treatment, monitor patients for signs and symptoms of IBD and discontinue treatment if new onset or worsening of signs and symptoms occurs. Immunizations Prior to initiating therapy with BIMZELX, complete all age-appropriate vaccinations according to current immunization guidelines. Avoid the use of live vaccines in patients treated with BIMZELX. Most Common Adverse Reactions Most common adverse reactions (≥1 percent) are upper respiratory infections, oral candidiasis, headache, injection site reactions, tinea infections, gastroenteritis, Herpes Simplex Infections, acne, folliculitis, other Candida infections, and fatigue. For further information, contact UCB: Investor Relations Antje Witte T +32.2.559.94.14 email [email protected] U.S. Communications Erica Puntel T +1.770.970.8465 email [email protected] About UCB UCB, Brussels, Belgium () is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 9,000 people in approximately 40 countries, the company generated revenue of €5.3 billion in 2023. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCBUSA. Forward looking statements This press release may contain forward-looking statements including, without limitation, statements containing the words "believes", "anticipates", "expects", "intends", "plans", "seeks", "estimates", "may", "will", "continue" and similar expressions. These forward-looking statements are based on current plans, estimates and beliefs of management. All statements, other than statements of historical facts, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, arbitration, political, regulatory or clinical results or practices and other such estimates and results. By their nature, such forward-looking statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties and assumptions which might cause the actual results, financial condition, performance or achievements of UCB, or industry results, to differ materially from those that may be expressed or implied by such forward-looking statements contained in this press release. Important factors that could result in such differences include: changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms or within expected timing, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, product liability claims, challenges to patent protection for products or product candidates, competition from other products including biosimilars, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws, and hiring and retention of its employees. There is no guarantee that new product candidates will be discovered or identified in the pipeline, will progress to product approval or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans. So far, the complexity of the human body cannot be reproduced in computer models, cell culture systems or animal models. The length of the timing to complete clinical trials and to get regulatory approval for product marketing has varied in the past and UCB expects similar unpredictability going forward. Products or potential products, which are the subject of partnerships, joint ventures or licensing collaborations may be subject to differences disputes between the partners or may prove to be not as safe, effective or commercially successful as UCB may have believed at the start of such partnership. UCB's efforts to acquire other products or companies and to integrate the operations of such acquired companies may not be as successful as UCB may have believed at the moment of acquisition. Also, UCB or others could discover safety, side effects or manufacturing problems with its products and/or devices after they are marketed. The discovery of significant problems with a product similar to one of UCB's products that implicate an entire class of products may have a material adverse effect on sales of the entire class of affected products. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment, including pricing pressure, political and public scrutiny, customer and prescriber patterns or practices, and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement activities and outcomes. Finally, a breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of UCB's data and systems. Given these uncertainties, you should not place undue reliance on any of such forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labelling in any market, or at any particular time, nor can there be any guarantee that such products will be or will continue to be commercially successful in the future. UCB is providing this information, including forward-looking statements, only as of the date of this press release. UCB expressly disclaims any duty to update any information contained in this press release, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations. Additionally, information contained in this document shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any offer, solicitation or sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such jurisdiction. References 1 BIMZELX® (bimekizumab-bkzx) U.S. PI. Available at: . Last accessed: April 2024. 2. Zouboulis C. 2023, EADV 2023, Oral Presentation # 3262. 3. Jemec GBE. Clinical practice. Hidradenitis suppurativa. N Engl J Med. 2012;366(2):158-64. 4 Sabat R, Jemec GBE, Matusiak L, et al. Hidradenitis suppurativa. Nat Rev Dis Primers. 2020;6(18):1-20. 5. Koumaki D, Efthymiou O, Bozi E, et al. Perspectives on Perceived Stigma And Self-Stigma In Patients With Hidradenitis Suppurativa. Clin Cosmet Investig Dermatol. 2019;12:785–90. 6. Kokolakis G, Wolk K, Schneider-Burrus S, et al. Delayed Diagnosis of Hidradenitis Suppurativa and Its Effect on Patients and Healthcare System. Dermatology. 2020;236:421–430. 7. ClinicalTrials.gov. A Study to Evaluate the Efficacy and Safety of Bimekizumab in Study Participants With Moderate to Severe Hidradenitis Suppurativa (BE HEARD I). Accessed date: April 2024. 8. ClinicalTrials.gov. A Study to Evaluate the Efficacy and Safety of Bimekizumab in Study Participants With Moderate to Severe Hidradenitis Suppurativa (BE HEARD II). . Accessed date: April 2024. US-BK-2400301 BIMZELX® is a registered trademark of the UCB Group of Companies. ©2024 UCB, Inc. Smyrna, GA 30080. All rights reserved. SOURCE UCB

自免药物市场重新变得充满不确定性。自从修美乐全球销量出现拐点，K药取代修美乐，坐上“药王”宝座，自免药物的开发者们又有了新奔头。在一个非共识的市场里，建立新的秩序，无疑将带来巨大的商业价值。可以看到，全球自免药物市场加速变得拥挤。原本的自免药物巨头没有功成离场，新的跨国药企手持重金加入战局。2023年6月，诺华35亿美元收购IgA肾病药物领先公司Chinook Therapeutics，礼来24亿美元收购DICE Therapeutics，获得其口服IL-17抑制剂等自免用药。在国内，自免新药的开发，相对肿瘤新药，并不算十分热门的领域，但随着越来越多的新靶点被验证，不少企业开始加码布局自免管线。数据显示，国内有数十家新药研发企业，布局了上百条自免新药管线。其中，TNFα和IL-12/IL-23两个自免领域老靶点引发的国产生物类似药竞逐，依然激烈，而JAK、IL-17A等新靶点，则吸引大量创新药企深耕，在研产品数量最多。国产自免药无战事在国内，自免药物市场一直充满不确定性。在生物制剂取代传统抗炎药物，成为自免疾病主流治疗方案的很长一段时间里，这个领域的进口药是干不过国产的。2002年，三生国健的前身中信国健成立。3年后，三生国健推出了靶向TNF-α的重磅产品益赛普，用于治疗类风湿关节炎，2年后新增强直性脊柱炎、银屑病适应症。后来，这款药主导国内自免药市场近15年之久。根据三生国健招股书，2018年至2020年间，益赛普的销售收入是公司营收的主要来源，占比超过99%。2017年9月，益赛普纳入国家医保，进一步推高了销售额。财报显示，2018年，益赛普的销售额达到 11.1 亿元，同比增长 9.7%，占国内TNF-α药物市场份额达到64%。在这个过程中，依那西普的原研药恩利，和在海外卖翻了的TNF-α重磅药物修美乐，一直在国内被益赛普压着打。2010年，恩利和修美乐在国内上市。失去了先发优势的原研明星药，加上高出至少一倍的用药费用，在国内销售十分低迷。2018年，修美乐在样本医院的销售额才刚刚接近2000万元，是益赛普同期销量的1/20。早期国内销售的自免生物制剂  数据来源：动脉网根据公开信息整理不过，建立在外部因素之上的市场独占地位，很容易被撼动。转折发生在2019年。2019年，修美乐和类克进入医保，百奥泰的格乐立、海正药业的安健宁获批上市。当年，修美乐、格乐立、安健宁的年治疗费用分别降低到3.35万元、3.01万元和2.99万元，远低于益赛普彼时的6.69万元。2020年，修美乐在样本医院销售额达7008万元，较2019年增长270.76%。2022年，益赛普在TNF-α类药品的市场份额为27%，益赛普的市场份额较去年同期略有下降。2018年至2022年间，国内TNF-α药物市场变化  数据来源：三生国健财报实际上，益赛普独占市场背后的另一重原因，在于国内自免药物市场的整体规模一直没有起量。据Frost & Sullivan统计，尽管一直保持着上升趋势，到2022年，国内的自免药物市场规模仍没有突破50亿美元，而同期的全球自免药物市场已经接近1500亿美元。与之形成对比的是，2022年，全球肿瘤药物市场规模约1500亿美元，而国内的这一市场规模已经达到354亿美元。当然，自免药物自有其独特性。自免疾病的患者基数大，治疗周期长，加上药物在适应症之间的拓展性强，本身就很容易出现重磅药物。近年来，随着人们对自免疾病机理研究走向深入，新靶点自免药物逐步改变国内的临床实践和患者习惯，拓宽了自免药物的市场边界。益赛普被迫走下神坛之初，三生国健曾经尝试走出舒适区，去拓展新的疾病领域。但多番尝试后，三生国健又再聚焦自免新药。2023年末，三生国健在财报中表示，将集中全力在自免领域深耕。动脉网注意到，自2023年开始，三生国健陆续减少在其他领域的研发费用，甚至交易其他领域的在研管线。2023年4月，三生国健将公司在研抗肿瘤项目602、609、705、707以及眼科在研药物601A项目独家授予沈阳三生，允许其在中国大陆以及美国开发以上药物。此外，三生国健还将304R项目所产生的所有化合物及其成为药物后的其他在全球范围内的一切权利转让给沈阳三生。白介素闯入者充分挖掘过TNF-α靶向药的潜力之后，扩大自免药物市场规模的希望，落到这个行业的闯入者之上。在国内，IL-17A无疑是最热门的自免新药靶点。除了已经上市的司库奇尤单抗、依奇珠单抗等进口药，从研发进度看，公开信息可以查询的在研IL-17A新药，形成了三个梯队，即以赛立奇单抗和夫那奇珠单抗为代表的NDA药物梯队，由国内的创新药企开发，以QX002N、古莫奇单抗等为代表的已经进入III期临床阶段的管线，和尚处于研发中早期阶段的大量管线。现阶段，全球共上市5款靶向IL-17A/IL-17RA药物，即司库奇尤单抗、依奇珠单抗、尼塔奇单抗、比吉利珠单抗和布罗利尤单抗。其中，司库奇尤单抗、依奇珠单抗和布罗利尤单抗已在国内获批，比吉利珠单抗也已在国内申报上市。作为自免新药，IL-17A/IL-17RA药物极大减少了自免患者的治疗支出，但花费仍然不低。以诺华制药的司库奇尤单抗为例，初始定价每支单价2998元，年治疗费用达4.04万元，而礼来的依奇珠单抗，单支定价4318元，年治疗费用为2.59万元。当然，纳入医保后的治疗价格大幅下降，这是后话。属于国产第一梯队的赛立奇单抗和夫那奇珠单抗，分别由智翔金泰和恒瑞开发，2023年3月和4月，赛立奇单抗和夫那奇珠单抗相继申请在国内上市，成为替代进口IL-17A/IL-17RA药物最有力的竞争者。目前，赛立奇单抗和夫那奇珠单抗都在大人群的临床研究中，表现出了优秀的安全性和有效性。数月前，恒瑞曾公布了夫那奇珠单抗的一项研究结果，评估其在中至重度斑块状银屑病患者中的多中心双盲2期研究（NCT03463187）取得积极结果。数据显示，在第12周，与安慰剂组相比，所有夫那奇珠单抗组的银屑病PASI 75都有所改善，40、80、160和240mg组PASI 75分别为56.8%、65.8%、81.6%和86.5%，而安慰剂组为5.4%；使用夫那奇珠单抗时，达到0或1的医师整体评估反应的患者也更高，40、80、160和240mg组分别为45.9%、47.4%、60.5%和73.0%，而安慰剂组为8.1%。未观察到意想不到的不良反应。本月初，智翔金泰也公布了赛立奇单抗的最新研究数据。数据显示，第12周，200mg赛立奇单抗试验组达到PASI 75的受试者比例为90.7%（安慰剂对照组为8.6%），达到皮损清除/几乎清除（PGA 0/1）的受试者比例为74.4%（安慰剂对照组为3.6%），达到主要临床终点。与此同时，PASI90应答率为74.4%，第52周PASI75应答率96.5%，PASI90应答率为84.1%，PGA0/1应答率为83.7%，保持了高水平的皮损改善，获得持续性的治疗应答。智翔金泰的强势入局，让国产自免一哥之争充满变数。相比之下，国产IL-17A/IL-17RA药物第二梯队的企业颇为庞大，既有国产自免的老大哥三生国健，也有跨界而来的肿瘤新药明星企业君实生物、康方生物。主要的国产IL-17A药物研发进展  数据来源：动脉网根据公开信息整理至少7家企业挤在IL-17A药物的III期临床之上，适应症选择高度重叠，破局之争不可谓不激烈。更重要的是，目前冲在研发最前线的创新药企，医学临床和商业渠道能力都不容置疑，也不相上下。作为曾经国内自免生物制剂市场的霸主，三生国健没能跑进IL-17A药物第一梯队，已是遗憾，但基于益赛普建立的，对国内自免疾病治疗特点的理解，和品牌认知，无疑会增加一些胜算。而君实生物和康方生物，作为肿瘤新药领域拓荒者，荃信生物的创始团队也并非新药新兵，他们对新药研发到上市的认知，复制到IL-17A的开发之上，也能减少这个过程的不确定性。从某种意义上讲，或许IL-17A/IL-17RA药物的上市之争，才是国内创新药企业之间，开发能力的第一次真正拼刺刀。弯道超车的JAK抑制剂？以往谈及JAK抑制剂，人们第一反应可能是做肿瘤新药。但从在研项目数量看，JAK是吸引药企数量仅次于TNFα的靶点，热度甚至比IL-17A更高。JAK-STAT信号通路由酪氨酸激酶相关受体、酪氨酸激酶JAK和转录因子STAT组成，由细胞因子刺激的信号转导，参与细胞的增殖、分化、凋亡以及免疫调节等许多重要的生物学过程，是人体内重要的信号通路之一。作为细胞质酪氨酸激酶家族的一员，JAKs共有JAK1、JAK2、JAK3、TYK2四种亚型，亚型之间有重叠的结合对象。其中JAK1、JAK3更多负责免疫调节，而JAK2主要与红细胞和血小板的生成相关。JAK－STAT信号通路与多种自身免疫性疾病的发病机制相关，包括类风湿关节炎、银屑病和炎症性肠病等。从上个世纪90年代全球首个JAK抑制剂被发现以来，全球范围内已经有11款JAK抑制剂原研药获批上市。不同代际的JAK抑制剂  数据来源：动脉网根据公开信息整理不同代际 JAK 抑制剂之间的差异，主要体现在对体内信号通路的选择性强弱。第一代JAK抑制剂为非选择性JAK抑制剂，同时阻断多条相关信号通路，缺乏选择性。第二代JAK抑制剂减少了第一代的严重不良反应，选择性地抑制JAK家族成员。到了第三代JAK抑制剂，可高度选择性地结合细胞上的相应结构域，特异性地抑制剂细胞活性。2017年，首款JAK抑制剂登陆国内市场，快速得到临床端认可，JAK抑制剂的市场规模也迅速扩容。数据显示，2022年，国内JAK抑制剂市场规模达到9.43亿元，同比增长222.95%。据弗若斯特沙利文预测，国内JAK抑制剂的销售还将持续增长，到2024年和2030年，市场规模将分别达到100亿、481亿元人民币，2019年至2024年间的复合增长率可以达到92.2%。不过，总体来看，国产JAK抑制剂的自免适应症开发，进展稍逊于IL-17A。动脉网查询发现，现阶段，国内注册申报JAK抑制剂涉及60个品种，313个受理号。其中，在国内JAK抑制剂研发数量TOP10企业中，恒瑞、凌科药业、泽璟生物等，开发进度最靠前。恒瑞开发的SHR0302是一种高选择性的JAK1抑制剂，已在中国获批开展类风湿关节炎、白癜风、溃疡性结肠炎等多种自免疾病的临床试验。其中，SHR0302的斑秃、特应性皮炎、溃疡性结肠炎、类风湿性关节炎、强直性脊柱炎等适应症进展最快，已经进入III期临床试验。泽璟制药开发的盐酸杰克替尼，也已经在进行中高危骨髓纤维化、重症斑秃、中重度特应性皮炎等自免疾病适应症的III期临床试验。除此之外，大多数国产自免JAK抑制剂还出去II期临床试验，或者更早的开发阶段。其中，凌科药业开发的LNK01003，对肠道具有限制性，将用于治疗溃疡性肠炎等疾病的治疗，颇受行业关注，目前，这款药物正在进行II期临床试验。相比IL-17A药物竞争的短兵相接，国产自免JAK抑制剂之争相对温和，但是在定价更低的JAK抑制剂市场，丢了先发优势的国产药物，能否在与进口原研药的竞争中立足，甚至凭借更强的本土化能力弯道超车，答案还不算清晰。无论是在全球还是国内，自免疾病都是第二大的临床用药市场。但与之相对应，药物供给侧的产品体系，尚不算完善。在国内，自免药物体系的缺失则更为明显。自免药物市场规模扩容之下，我们看到，越来越多的国内药企加入、加码这个并不算炙手可热的领域，而谁能笑到最后，我们拭目以待。*封面图片来源：pexels文|王世薇微信|no_th_ing添加时请注明：姓名-公司-职位网站、公众号等转载请联系授权：Rekkiiie近期推荐声明：动脉网所刊载内容之知识产权为动脉网及相关权利人专属所有或持有。未经许可，禁止进行转载、摘编、复制及建立镜像等任何使用。动脉网，未来医疗服务平台

Late-breaking platform presentation showed that BIMZELX® (bimekizumab-bkzx) rapidly achieved and maintained high rates of clinical and health-related quality-of-life responses through four years; six out of ten patients achieved complete skin clearance at Year 4 Responder-analyses demonstrated that approximately nine out of ten patients treated with BIMZELX who achieved PASI90 at Week 16, and over seven out of ten patients who achieved complete skin clearance (PASI100) maintained their response to Year 4 Four-year safety data showed that treatment-emergent adverse events were consistent or decreased with longer BIMZELX exposure, with no new safety signals ATLANTA, March 9, 2024 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced that the first presentations of BIMZELX® (bimekizumab-bkzx) four-year efficacy and safety data in the treatment of adults with moderate-to-severe plaque psoriasis are being shared this week at the 2024 American Academy of Dermatology (AAD) Annual Meeting in San Diego, California, March 8–12. "We are proud to debut the BIMZELX® four-year psoriasis data at the world's largest dermatology meeting, showing that the majority of adult patients treated with BIMZELX achieved deep and durable clinical response through four years, with a consistent tolerability profile. These results, from the largest pool of Phase 3 data, closely follow the U.S. launch, and reinforce our belief that BIMZELX has the potential to transform the lives of people with moderate-to-severe plaque psoriasis," said Emmanuel Caeymaex, Executive Vice President, Immunology Solutions and Head of U.S., UCB. "Moderate-to-severe plaque psoriasis is a chronic condition with physical skin manifestations that can place a significant burden on a patient's health-related quality-of-life. Analysis shows that through four years of bimekizumab treatment, over six out of ten patients achieved and maintained complete skin clearance, a clinically meaningful endpoint and outcome for patients. These long-term data will be highly welcomed by the dermatology community since they provide important considerations for clinical practice," said Dr. Bruce Strober, Clinical Professor of Dermatology at Yale University, and Central Connecticut Dermatology, Connecticut, U.S. The late-breaking platform presentation shared BIMZELX pooled data from treatment initiation through four years, showing that high rates of clinical and health-related quality-of-life responses were rapidly achieved and were maintained in the long-term.¹ Responder-analyses showed that approximately nine out of ten patients treated with BIMZELX who achieved ≥90 percent improvement from baseline in the Psoriasis Area Severity Index (PASI90) and over seven out of ten patients who achieved complete skin clearance (PASI100) at Week 16 maintained their responses to Year 4.² Pooled analysis from five Phase 3/3b studies showed that BIMZELX demonstrated good tolerability and a consistent safety profile with no new safety findings identified up to four years in patients with moderate-to-severe plaque psoriasis.³ Highlights from the four-year BIMZELX data in moderate-to-severe plaque psoriasis presented at the 2024 AAD: Treatment initiation through four years: Data were pooled across the 52-week BE VIVID study, the 56-week BE READY and BE SURE studies and their open-label extension (OLE Week 144) BE BRIGHT.¹ Analyzed patients were randomized to BIMZELX 320 mg every four weeks (Q4W) to Week 16, then BIMZELX Q4W or Q8W until OLE entry.¹ Clinical and health-related quality of life (PASI90 and PASI100, body surface area [BSA] ≤1 percent and Dermatology Life Quality Index [DLQI]0/1) responses were assessed through to Year 4 (OLE Week 144).¹ Data are presented below for all patients who received BIMZELX continuously from baseline and entered the OLE (n=771):¹ 90.9 percent achieved PASI90 at Week 16, and 86.1 percent through to Year 4.1¥ 65.8 percent achieved PASI100 at Week 16, and 64.7 percent through to Year 4.1¥ 78.5 percent achieved BSA≤1 percent at Week 16, and 79.8 percent through to Year 4.1¥ 71.5 percent achieved DLQI0/1 at Week 16, and 78.7 percent through to Year 4.1 ¥ Responder analysis to Year 4: Patients who completed the BE VIVID, BE SURE, and BE READY Phase 3 studies could enter the BE BRIGHT OLE.² Analyzed patients were randomized to BIMZELX 320 mg Q4W to Week 16, then BIMZELX Q4W or Q8W until OLE entry, then BIMZELX Q4W or Q8W dependent on PASI response/prior dose.² Maintenance of PASI90 and PASI100 was assessed in Week 16 responders to Year 4 (OLE Week 144) and is presented below for all patients:² 87.7 percent who achieved PASI90 at Week 16 (n=693) maintained their response to Year 4.2¥ 73.3 percent who achieved PASI100 at Week 16 (n=503) maintained their response to Year 4.2¥ Safety and tolerability through four years: Data were pooled across the 52-week BE VIVID study, the 56-week BE READY and BE SURE studies, and the OLE studies BE BRIGHT and BE RADIANT.3 The total BIMZELX exposure was 6,324.3 patient-years (PY) across the studies (n=2,186).3 Exposure-adjusted incidence rates (EAIRs) of treatment-emergent adverse events (TEAEs) remained consistent or decreased with longer BIMZELX exposure. Overall, TEAEs occurred at an EAIR of 170.5/100 PY and serious TEAEs at 5.5/100 PY.3 The most common TEAEs were nasopharyngitis (12.7/100 PY), oral candidiasis (8.9/100 PY), and upper respiratory tract infections (5.7/100 PY). Oral candidiasis decreased from 18.9/100 PY at Year 1 to 5.4/100 PY at Year 4.3 Throughout, fewer TEAEs occurred with BIMZELX every 8 weeks (Q8W) (115.4/100 PY) vs. every 4 weeks (Q4W) (224.4/100 PY).3 ¥ Modified non-responder imputation analyses Notes to Editors: About BIMZELX (bimekizumab-bkzx) Bimekizumab is a humanized IgG1 monoclonal antibody that selectively binds to IL-17A, IL-17F and IL-17AF cytokines, blocking their interaction with the IL-17RA/IL-17RC receptor complex.4 Elevated levels of IL-17A and IL-17F are found in lesional psoriatic skin.4 BIMZELX is not approved in the U.S. for the treatment of psoriatic arthritis or axial spondyloarthritis and these are investigational indications only. Please see Important Safety Information below and full U.S. prescribing information at . BIMZELX U.S. IMPORTANT SAFETY INFORMATION4 Suicidal Ideation and Behavior BIMZELX® (bimekizumab-bkzx) may increase the risk of suicidal ideation and behavior (SI/B). A causal association between treatment with BIMZELX and increased risk of SI/B has not been established. Prescribers should weigh the potential risks and benefits before using BIMZELX in patients with a history of severe depression or SI/B. Advise monitoring for the emergence or worsening of depression, suicidal ideation, or other mood changes. If such changes occur, advise to promptly seek medical attention, refer to a mental health professional as appropriate, and re-evaluate the risks and benefits of continuing treatment. Infections BIMZELX may increase the risk of infections. Do not initiate treatment with BIMZELX in patients with any clinically important active infection until the infection resolves or is adequately treated. In patients with a chronic infection or a history of recurrent infection, consider the risks and benefits prior to prescribing BIMZELX. Instruct patients to seek medical advice if signs or symptoms suggestive of clinically important infection occur. If a patient develops such an infection or is not responding to standard therapy, monitor the patient closely and do not administer BIMZELX until the infection resolves. Tuberculosis Evaluate patients for tuberculosis (TB) infection prior to initiating treatment with BIMZELX. Avoid the use of BIMZELX in patients with active TB infection. Initiate treatment of latent TB prior to administering BIMZELX. Consider anti-TB therapy prior to initiation of BIMZELX in patients with a past history of latent or active TB in whom an adequate course of treatment cannot be confirmed. Closely monitor patients for signs and symptoms of active TB during and after treatment. Liver Biochemical Abnormalities Elevated serum transaminases were reported in clinical trials with BIMZELX. Test liver enzymes, alkaline phosphatase and bilirubin at baseline, periodically during treatment with BIMZELX and according to routine patient management. If treatment-related increases in liver enzymes occur and drug-induced liver injury is suspected, interrupt BIMZELX until a diagnosis of liver injury is excluded. Permanently discontinue use of BIMZELX in patients with causally associated combined elevations of transaminases and bilirubin. Avoid use of BIMZELX in patients with acute liver disease or cirrhosis. Inflammatory Bowel Disease Cases of inflammatory bowel disease (IBD) have been reported in patients treated with IL-17 inhibitors, including BIMZELX. Avoid use of BIMZELX in patients with active IBD. During BIMZELX treatment, monitor patients for signs and symptoms of IBD and discontinue treatment if new onset or worsening of signs and symptoms occurs. Immunizations Prior to initiating therapy with BIMZELX, complete all age-appropriate vaccinations according to current immunization guidelines. Avoid the use of live vaccines in patients treated with BIMZELX. Most Common Adverse Reactions Most common adverse reactions (≥1 percent) are upper respiratory infections, oral candidiasis, headache, injection site reactions, tinea infections, gastroenteritis, Herpes Simplex Infections, acne, folliculitis, other Candida infections, and fatigue. For further information, contact UCB: Investor Relations Antje Witte T +32.2.559.94.14 email [email protected] U.S. Communications Erica Puntel T +1.770.970.8465 email [email protected] About UCB UCB, Brussels, Belgium () is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 9,000 people in approximately 40 countries, the company generated revenue of €5.3 billion in 2023. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCBUSA. Forward looking statements This press release may contain forward-looking statements including, without limitation, statements containing the words "believes", "anticipates", "expects", "intends", "plans", "seeks", "estimates", "may", "will", "continue" and similar expressions. These forward-looking statements are based on current plans, estimates and beliefs of management. All statements, other than statements of historical facts, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, arbitration, political, regulatory or clinical results or practices and other such estimates and results. By their nature, such forward-looking statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties and assumptions which might cause the actual results, financial condition, performance or achievements of UCB, or industry results, to differ materially from those that may be expressed or implied by such forward-looking statements contained in this press release. Important factors that could result in such differences include: changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms or within expected timing, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, product liability claims, challenges to patent protection for products or product candidates, competition from other products including biosimilars, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws, and hiring and retention of its employees. There is no guarantee that new product candidates will be discovered or identified in the pipeline, will progress to product approval or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans. So far, the complexity of the human body cannot be reproduced in computer models, cell culture systems or animal models. The length of the timing to complete clinical trials and to get regulatory approval for product marketing has varied in the past and UCB expects similar unpredictability going forward. Products or potential products, which are the subject of partnerships, joint ventures or licensing collaborations may be subject to differences disputes between the partners or may prove to be not as safe, effective or commercially successful as UCB may have believed at the start of such partnership. UCB's efforts to acquire other products or companies and to integrate the operations of such acquired companies may not be as successful as UCB may have believed at the moment of acquisition. Also, UCB or others could discover safety, side effects or manufacturing problems with its products and/or devices after they are marketed. The discovery of significant problems with a product similar to one of UCB's products that implicate an entire class of products may have a material adverse effect on sales of the entire class of affected products. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment, including pricing pressure, political and public scrutiny, customer and prescriber patterns or practices, and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement activities and outcomes. Finally, a breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of UCB's data and systems. Given these uncertainties, you should not place undue reliance on any of such forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labelling in any market, or at any particular time, nor can there be any guarantee that such products will be or will continue to be commercially successful in the future. UCB is providing this information, including forward-looking statements, only as of the date of this press release. UCB expressly disclaims any duty to update any information contained in this press release, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations. Additionally, information contained in this document shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any offer, solicitation or sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such jurisdiction. References 1. Strober B. 2024 AAD. Oral Presentation. 2. Blauvelt A, Foley P, Langley RG, et al. Bimekizumab 4-year maintenance of responses in Week 16 responders with moderate to severe plaque psoriasis: Results from the BE BRIGHT open label extension phase 3 trial. Abstract at the 2024 American Academy of Dermatology Annual Meeting, San Diego, CA, U.S., March 8–12, 2024. 3. Gordon KB, Thaçi D, Gooderham M, et al. Bimekizumab safety and tolerability in moderate to severe plaque psoriasis: Pooled analysis from up to 4 years of treatment in 5 phase 3/3b clinical trials. Abstract at the 2024 American Academy of Dermatology Annual Meeting, San Diego, CA, U.S., March 8–12, 2024. 4. BIMZELX [prescribing information]. Smyrna, GA: UCB, Inc. SOURCE UCB