Nipocalimab-aahu

The ITC compared all published Phase 3 data of these treatments, leveraging longitudinal results, and findings reinforce the importance of consistent, sustained disease control in managing a chronic autoantibody disease like gMG IMAAVY, an FcRn blocker, received U.S. FDA approval earlier this year for the broadest population of individuals living with gMG, including anti-AChR and anti-MuSK antibody positive adults and pediatric gMG patients aged 12 and older SPRING HOUSE, Pa., June 23, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced new data from an indirect treatment comparison (ITC) that showed consistent and sustained disease control with IMAAVY™ (nipocalimab-aahu) versus other approved FcRn blockers in adults with generalized myasthenia gravis (gMG). These data, featured at the European Academy of Neurology (EAN) 2025 Congress in Helsinki, Finland, are among the 11 abstracts Johnson & Johnson is presenting. Based on the ITC, which included the pivotal Phase 3 Vivacity-MG3 study data, IMAAVY showed comparable onset of symptom relief at Week 1 and showed consistent and sustained disease control with greater or statistically significant improvement of MG-ADLa scores versus the published Phase 3 data of other marketed FcRn blockers at several timepoints up to 24 weeksb of treatment.1 Results were consistent across multiple ITC methodsc. Population-adjusted ITCs without a common control exhibited significantly greater mean improvements in MG-ADL scores favoring IMAAVY over other FcRn blockers, at Weeks 8-24d versus one comparator and at Weeks 10-14d versus another comparator.1 In placebo-adjusted ITCs, IMAAVY was associated with numerically greater efficacy versus one treatment comparator at Weeks 8e and 18-24e and versus another at Weeks 10-14e, with statistical significance at Weeks 10f and 12f.1 "These analyses provide useful population-adjusted comparative data and add to the body of evidence supporting the use of IMAAVY for the treatment of gMG for certain patients," said Saiju Jacob, M.D., Professor, Department of Immunology and Immunotherapy at University of Birmingham, UK f, "The significantly greater mean improvements on MG-ADL scores with IMAAVY reflect important new evidence of the ongoing need for sustained disease control in a chronic condition like gMG." Unlike cyclic therapies that require clinical evaluation and symptom relapse prior to initiating subsequent treatment cycles, IMAAVY has a biweekly dosing regimen that may allow for a schedule that patients and healthcare providers can plan around.2 These data provide insights about the predictability that IMAAVY may offer and potentially help clinical decision making when treating patients with gMG. "At Johnson & Johnson, we recognize that for people living with gMG, the goal isn't just temporary relief, but rather sustained disease control. This analysis provides additional insights into the profile of IMAAVY and highlights its potential as a reliable treatment option for appropriate patients aged 12 and older living with gMG," said Katie Abouzahr, M.D., Vice President, Autoantibody Portfolio and Maternal Fetal Immunology Disease Area Leader, Johnson & Johnson Innovative Medicine. "As we continue to research the potential impact of IMAAVY and work with regulators worldwide, we are committed to helping patients with chronic, debilitating autoantibody conditions, like gMG." ITCs are utilized by regulatory agencies, health technology assessment agencies and medical guideline committees to comparatively evaluate treatment options when there is no or limited availability of evidence from head-to-head clinical trials.3 ITCs, however, cannot replace and should not be considered the same as head-to-head clinical trials.3 Unanchored population-adjusted and placebo-anchored Bucher ITC methods were used in this analysis.3 Unanchored population-adjusted indirect comparisons allow for adjustment of population differences using individual patient-level data from IMAAVY and aggregate data from other approved FcRn blockers.3 Placebo-anchored Bucher ITCs evaluate a small number of treatments through a common comparator such as the trial placebo and use aggregate data from different trials.3 IMAAVY is approved in the U.S. for adult and pediatric patients (12 years of age and older) with anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive gMG. Johnson & Johnson also submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of nipocalimab in gMG in September 2024. Editor's notes: a. MG-ADL (Myasthenia Gravis-Activities of Daily Living) provides a rapid clinical assessment of the patient's recall of symptoms impacting activities of daily living, with a total score range of 0 to 24; a higher score indicates greater symptom severity.4 b. There are no head-to-head data available for any FcRn blockers, and no claim of superiority can be made about any FcRn blockers in the absence of such head-to-head trial data. c. The ITC did not evaluate safety among the FcRn blocker agents. d. In unanchored population-adjusted indirect comparison, IMAAVY versus one treatment at Week 8 had a mean difference of -2.36 [(95% confidence interval [CI]: -3.56, -1.16); P=0.001]; this trend continued up to Week 24 (P<0.05). For another comparator, at Week 10 IMAAVY had a mean difference of –2.38 at one comparator dose [(95% CI: -3.57, -1.18); P<0.001] and a mean difference of -3.14 with a different comparator dose [(95% CI: -4.15, -2.14); P<0.001]; this trend continued up to Week 14 (P<0.001). e. In placebo-anchored indirect comparison, MG-ADL change from baseline (CFB) was greater at Week 8 by -1.24 versus one treatment comparator (95% CI: -2.78, 0.30), at Week 18 by -1.13 (95% CI: -2.77, 0.50), at Week 20 by -1.44 (95% CI: -3.21, -0.33), at Week 22 by -1.79 (95% CI: -4.16, 0.59), and at Week 24 by -2.89 (95% CI: -5.67, -0.12). f. In placebo-anchored indirect comparison, treatment with IMAAVY demonstrated a greater MG-ADL CFB versus a comparator dose at Week 10 (mean CFB=-1.19, 95% CI: -2.75, 0.37), Week 12 (mean CFB= -1.41, 95% CI: -2.94, 0.12) and Week 14 (mean CFB= -1.01, 95% CI: -2.51, 0.49). • Similar trends were observed for IMAAVY versus a different comparator dose at Week 10 (mean CFB= -2.16, 95% CI: -3.58, -0.73), Week 12 (mean CFB= -1.99, 95% CI: -3.53, -0.45) and Week 14 (mean CFB= -1.12, 95% CI: -2.55, 0.31). g. Saiju Jacob, M.D., has provided consulting, advisory, and speaking services to Johnson & Johnson. He has not been paid for any media work. ABOUT GENERALIZED MYASTHENIA GRAVIS (gMG) Myasthenia gravis (MG) is an autoantibody disease in which the immune system mistakenly makes antibodies (e.g., anti-acetylcholine receptor [AChR], anti-muscle-specific tyrosine kinase [MuSK]), which target proteins at the neuromuscular junction and can block or disrupt normal signaling from nerves to muscles, thus impairing or preventing muscle contraction.5,6,7 The disease impacts an estimated 700,000 people worldwide.5 The disease affects both men and women and occurs across all ages, racial and ethnic groups, but most frequently starts in young women and older men.8 Roughly 50 percent of individuals diagnosed with MG are women, and about one in five of those women are of child-bearing potential.9,10,11 Approximately 10 to 15% of new cases of MG are diagnosed in pediatric patients 12-17 years of age.12,13,14 Among juvenile MG patients, girls are affected more often than boys with over 65% of pediatric MG cases in the U.S. diagnosed in girls.15,16,17 Initial disease manifestations are usually eye-related but approximately 85% of MG patients experience additional advancements to the disease manifestations, referred to as generalized myasthenia gravis (gMG). This is characterized by severe muscle weakness and difficulties in speech and swallowing.18,19,20,21,22 Approximately 100,000 individuals in the U.S. are living with gMG.23 Vulnerable gMG populations, such as pediatric patients, have more limited therapeutic options.24 ABOUT THE INDIRECT TREATMENT COMPARISON Indirect treatment comparisons (ITCs) were conducted to compare efficacy onset using 1-week timepoint, and for consistency and sustained disease control, comparisons were conducted for multiple timepoints up to 24 weeks for one treatment comparator (up to 3-cycle duration) and up to 14 weeks for another treatment comparator (final visit data reported).1 The data used in the analysis came from published registrational trials of IMAAVY and comparator FcRn blockers approved to treat gMG (efgartigimod and rozanolixizumab).1 The differences in clinical trial design across FcRn blockers coupled with differences in background standard of care (SOC) required multiple indirect treatment comparison methods to be utilized.1 Therefore, ITCs were conducted using unanchored population-adjusted indirect comparisons (with active treatment arm only and adjusting for cross-trial patient differences) and placebo-anchored (includes both active treatment and placebo arms) Bucher method. Differences <0 favored nipocalimab for all comparisons.1 This ITC adheres to all governing standards and requirements as demanded by global health technology assessment agencies, journal review committees and regulatory authorities. The ITC was funded by Janssen Research & Development, LLC.1 THE PHASE 3 VIVACITY-MG3 STUDY The Phase 3 Vivacity-MG3 study (NCT04951622) was specifically designed to measure sustained efficacy and safety with consistent dosing in this unpredictable chronic condition where unmet need remains high. Antibody positive or negative adult gMG patients with insufficient response (MG-ADL ≥6) to ongoing SOC therapy were identified and 199 patients, 153 of whom were antibody positive, enrolled in the 24-week double-blind placebo-controlled trial.25,26 Randomization was 1:1, nipocalimab plus current SOC (30 mg/kg IV loading dose followed by 15 mg/kg every two weeks) or placebo plus current SOC.25 Baseline demographics were balanced across arms (77 nipocalimab, 76 placebo).25 The primary efficacy endpoint was the comparison of the mean change from baseline to Weeks 22, 23, and 24 between treatment groups in the MG-ADL total score.25 A key secondary endpoint included change in Quantitative Myasthenia Gravis (QMG) score. Long-term safety and efficacy were further assessed in an ongoing open-label extension (OLE) phase.26 ABOUT IMAAVY™ (nipocalimab-aahu) IMAAVY is a monoclonal antibody, designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies that underlie generalized myasthenia gravis (gMG) without additional detectable effects on other adaptive and innate immune functions. IMAAVY is currently approved in the U.S. for the treatment of gMG in adults and pediatric patients 12 years of age and older who are AChR or MuSK antibody positive.2 Nipocalimab is continuing to be investigated across three key segments in the autoantibody space including Rare Autoantibody diseases, Maternal Fetal diseases mediated by maternal alloantibodies and Rheumatic diseases.28,29,30,31,32,32,33,34,35,36 The investigational monoclonal antibody is designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) auto and alloantibodies potentially without additional detectable effects on other adaptive and innate immune functions. The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted several key designations to nipocalimab including: U.S. FDA Fast Track designation in hemolytic disease of the fetus and newborn (HDFN) and warm autoimmune hemolytic anemia (wAIHA) in July 2019, gMG in December 2021, fetal and neonatal alloimmune thrombocytopenia) FNAIT in March 2024 and Sjögren's disease (SjD) in March 2025 U.S. FDA Orphan drug status for wAIHA in December 2019, HDFN in June 2020, gMG in February 2021, chronic inflammatory demyelinating polyneuropathy (CIDP) in October 2021 and FNAIT in December 2023 U.S. FDA Breakthrough Therapy designation for HDFN in February 2024 and for Sjögren's disease in November 2024 U.S. FDA granted Priority Review in gMG in Q4 2024 EU EMA Orphan medicinal product designation for HDFN in October 2019 and FNAIT in April 2025 WHAT IS IMAAVY™ (nipocalimab-aahu)? IMAAVY™ is a prescription medicine used to treat adults and children 12 years of age and older with a disease called generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. It is not known if IMAAVY™ is safe and effective in children under 12 years of age. IMPORTANT SAFETY INFORMATION What is the most important information I should know about IMAAVY™? IMAAVY™ is a prescription medicine that may cause serious side effects, including: Infections are a common side effect of IMAAVY™ that can be serious. Receiving IMAAVY™ may increase your risk of infection. Tell your healthcare provider right away if you have any of the following infection symptoms: o fever o chills o shivering o cough o sore throat o fever blisters o burning when you urinate Allergic (hypersensitivity) reactions may happen during or up to a few weeks after your IMAAVY™ infusion. Get emergency medical help right away if you get any of these symptoms during or after your IMAAVY™ infusion: o a swollen face, lips, mouth, tongue, or throat o difficulty swallowing or breathing o itchy rash (hives) o chest pain or tightness Infusion-related reactions are possible. Tell your healthcare provider right away if you get any of these symptoms during or a few days after your IMAAVY™ infusion: o headache o rash o nausea o fatigue o dizziness o chills o flu-like symptoms o redness of skin Do not receive IMAAVY™ if you have a severe allergic reaction to nipocalimab-aahu or any of the ingredients in IMAAVY™. Reactions have included angioedema and anaphylaxis. Before using IMAAVY™, tell your healthcare provider about all of your medical conditions, including if you: ever had an allergic reaction to IMAAVY™. have or had any recent infections or symptoms of infection. have recently received or are scheduled to receive an immunization (vaccine). People who take IMAAVY™ should not receive live vaccines. are pregnant, plan to become pregnant, or are breastfeeding. It is not known whether IMAAVY™ will harm your baby. Pregnancy Safety Study. There is a pregnancy safety study for IMAAVY™ if IMAAVY™ is given during pregnancy or you become pregnant while receiving IMAAVY™. Your healthcare provider should report IMAAVY™ exposure by contacting Janssen at 1-800-526-7736 or www.IMAAVY.com. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. What are the possible side effects of IMAAVY™? IMAAVY™ may cause serious side effects. See "What is the most important information I should know about IMAAVY™?" The most common side effects of IMAAVY™ include: respiratory tract infection, peripheral edema (swelling in your hands, ankles, or feet), and muscle spasms. These are not all the possible side effects of IMAAVY™. Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. Please see the full Prescribing Information and Medication Guide for IMAAVY™ and discuss any questions you have with your doctor. Dosage Form and Strengths: IMAAVY™ is supplied as a 300 mg/1.62 mL and a 1,200 mg/6.5 mL (185 mg/mL) single-dose vial per carton for intravenous injection. ABOUT JOHNSON & JOHNSON At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow and profoundly impact health for humanity. Learn more at https://www.jnj.com/ or at https://innovativemedicine.jnj.com/ Follow us at @JNJInnovMed. Janssen Research & Development, LLC and Janssen Biotech, Inc. are Johnson & Johnson companies. CAUTIONS CONCERNING FORWARD-LOOKING STATEMENTS This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of IMAAVY™. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's most recent Annual Report on Form 10-K, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. Johnson & Johnson does not undertake to update any forward-looking statement as a result of new information or future events or developments. REFERENCES 1 Saiju Jacob, Mahmoud Hashim, Brian Hutton, Kavita Gandhi, Rafal Slowik, Chris Drudge, Antoine C El Khoury, Maria Ait-Tihyaty, Mi Jun Keng, Xiwu Lin, Sumeet Singh, Nils Erik Gilhus. Comparative efficacy of nipocalimab with other FcRN-blocker therapies in generalized myasthenia gravis. Presented at the 2025 European Academy of Neurology (EAN) Congress. Available at: https://www.eanvirtualcongress.org/ean/ean2025/en-GB/presentation/5308352 IMAAVY™ U.S. Prescribing Information3 Nils Erik Gilhus, Saiju Jacob, Mahmoud Hashim, Suzy Van Sanden, Christopher Drudge, Anna Nero, Sumeet Singh, Kavita Gandhi, and Brian Hutton Network connectivity, between-study heterogeneity and timepoint challenges in generalized myasthenia gravis: a feasibility assessment of indirect treatment comparisons. (2025) Journal of Comparative Effectiveness Research. DOI: 10.57264/cer-2025-00094 Wolfe GI Myasthenia gravis activities of daily living profile. Neurology. 1999;22;52(7):1487-9. doi: 10.1212/wnl.52.7.1487.5 Chen J, Tian D-C, Zhang C, et al. Incidence, mortality, and economic burden of myasthenia gravis in China: A nationwide population-based study. The Lancet Regional Health - Western Pacific. https://www.thelancet.com/action/showPdf?pii=S2666-6065%2820%2930063-8 6 Bacci ED et al. 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Available at: https://clinicaltrials.gov/study/NCT04882878. Last accessed: June 2025.35 ClinicalTrials.gov Identifier: NCT06449651. Available at: https://clinicaltrials.gov/study/NCT06449651. Last accessed: June 2025.36 ClinicalTrials.gov Identifier: NCT06533098. Available at: https://clinicaltrials.gov/study/NCT06533098. Last accessed: June 2025. Media contact: Bridget Kimmel bkimmel@its.jnj.com Investor contact: Lauren Johnson investor-relations@its.jnj.com View original content to download multimedia:https://www.prnewswire.com/news-releases/imaavy-nipocalimab-aahu-showed-greater-sustained-disease-control-versus-approved-fcrn-blockers-for-generalized-myasthenia-gravis-gmg-at-multiple-timepoints-over-24-weeks-in-newly-published-indirect-treatment-comparison-itc-302487317.html SOURCE Johnson & Johnson

编者按：刚刚过去的“全球重症肌无力关爱日”再次提醒大家关注这一罕见疾病。重症肌无力（MG）是一种罕见的神经肌肉-自身免疫性疾病，会导致患者虚弱和可能危及生命的肌无力。目前该疾病尚无治愈方法。随着研究进展和产业持续投入，近十年来多款针对重症肌无力的创新药获监管机构批准上市，极大改善了患者的生活。与此同时，全球更多MG新药管线已进入临床阶段，涵盖抗体疗法、siRNA药物、小分子、CAR-T疗法等多种类型，有望在不远的未来迎来新突破。作为全球医药及生命科学行业创新的赋能者，药明康德也持续通过一体化、端到端的CRDMO平台，为全球合作伙伴提供赋能，助力重症肌无力等罕见肌肉疾病新药的开发，为患者带来更多突破性的治疗方案。回顾2025年上半年，重症肌无力疾病领域新药再次迎来多项新进展。5月，强生公司（Johnson & Johnson）宣布，美国FDA已批准其抗体疗法Imaavy（nipocalimab）上市，用于治疗抗AChR、抗MuSK抗体阳性的全身性重症肌无力（gMG）成人与12岁以上儿童患者。这是FDA批准的首个用于治疗该类患者群体的新生儿Fc受体（FcRn）阻断剂。这款疗法还被Evaluate列为今年有望上市的10大潜在重磅疗法之一。同月，翰森制药宣布其伊奈利珠单抗（inebilizumab）的新适应症上市申请获得中国NMPA受理，用于治疗全身性重症肌无力成人患者。这是一款靶向CD19 B细胞消耗性抗体，由翰森制药和安进（Amgen）共同开发。该药物的全球关键性3期试验MINT的结果于今年4月发表于《新英格兰医学杂志》。同样是5月，荣昌生物的BLyS/APRIL双靶点融合蛋白创新药物泰它西普在中国获批新适应症，用于治疗全身性重症肌无力成人患者。该产品可同时靶向B细胞激活因子（又称BLyS）和增殖诱导配体（APRIL），针对致病性抗体产生的源头——B细胞及浆细胞。此外，在CAR-T疗法领域，Cartesian Therapeutics于今年4月公布其在研mRNA细胞疗法Descartes-08用于治疗gMG的2b期研究的12个月数据。该研究早前已经取得积极结果，80%的可评估患者维持了临床显著应答达1年。Descartes-08是一款靶向BCMA的在研CAR-T细胞疗法，目前已启动3期临床研究，是MG领域目前研发进度较快的CAR-T细胞疗法，有望成为MG治疗领域首个细胞疗法。从这些进展可以看到，在重症肌无力治疗药物研发领域，2025年正在迎来多元化的新突破。图片来源：123RF过去十年间，至少7款重症肌无力新药获批上市重症肌无力是一种慢性的罕见自身免疫疾病，目前尚无治愈方法。该病的致病机制涉及患者体内的免疫球蛋白G（IgG）抗体，这些抗体会破坏神经与肌肉之间的突触传递，导致肌肉疲劳无力，进而导致极度疲劳和面部表情、言语、吞咽和活动困难，严重时甚至危及生命。大约85%的重症肌无力患者属于全身性重症肌无力（gMG）。根据2021年文献数据，gMG的患病率和发病率在全球范围内正在增加，全球患病率估计为每10万人中有2~36例。过去十年间，全球至少有7款重症肌无力新药获批上市，包括FcRn拮抗剂、补体抑制剂和B细胞耗竭剂等，为MG患者带来了更多治疗选择。C5补体抑制剂包括抗体和大环肽类药物，比如阿斯利康的ravulizumab（瑞利珠单抗）、eculizumab（依库珠单抗），以及优时比公司的大环肽类C5补体抑制剂zilucoplan（泽勒普肽）等，这类药物能够减少补体激活介导的神经肌肉接头损伤；FcRn抑制剂类药物以抗体为主，比如开头提到的强生公司的nipocalimab（尼卡利单抗），以及优时比公司的rozanolixizumab（罗泽利昔珠单抗）、Argenx和再鼎医药共同开发的efgartigimod（艾加莫德）等，它们可降低循环IgG抗体水平，从而减少致病抗体的循环，改善MG症状；B细胞耗竭剂类的代表药物为荣昌生物研发的BLyS/APRIL双靶点融合蛋白泰它西普，针对致病性抗体产生的源头——B细胞及浆细胞，减少致病性自身抗体的产生，发挥治疗作用。寻找“治愈”曙光，下一代重症肌无力疗法有望迎来新突破梳理公开资料可知，除了已获批的新药，全球范围内还有数十款新药正在临床阶段探索用于重症肌无力治疗的潜力，未来有望在多个方向取得新突破。在重症肌无力临床管线中，抗体类药物数量较多，包含单抗、双抗，以及一些抗体融合蛋白等，靶点涉及C5、FcRn、CD19/CD20等。例如阿斯利康在研的第三代C5补体抑制剂抗体gefurulimab已经进入3期临床阶段，其分子量小，具更好的渗透性，有望开发成为一款每周一次、由患者自行给药的皮下注射疗法。再如开头提到的抗CD19单抗伊奈利珠单抗，其翰森制药提交的针对重症肌无力的上市申请已获得中国NMPA受理，安进在两个月前也宣布了该产品治疗gMG成年患者的疗效与安全性的最新临床数据：每年两次给药的情况下，其在乙酰胆碱受体自身抗体阳性（AChR+）gMG患者中具有持久的疗效。CAR-T疗法也在临床研究中展现出“治愈”重症肌无力的潜力。CAR-T疗法近年来在自身免疫性疾病领域进展迅速，其可以使患者实现长期无药物缓解，被认为有望对重症肌无力的治疗产生潜在变革作用。在重症肌无力临床管线中，包括了十多款CAR-T疗法。这些在研管线以靶向B细胞或浆细胞为主，主要包括BCMA和CD19。在MG临床研发管线中，也有多款小分子药物。例如诺华在研的补体B因子口服抑制剂iptacopan（伊普可泮）和BTK抑制剂remibrutinib（瑞米布替尼），目前均已进入重症肌无力治疗研究3期临床阶段。再如NMD Pharma在研的氯离子通道（ClC-1）抑制剂NMD670正处于重症肌无力2b期临床阶段，该产品作用机制是针对骨骼肌的非免疫调节。多肽和核酸药物研发管线也有新突破。泽勒普肽（zilucoplan）作为首个获FDA批准用于治疗重症肌无力的每日一次经皮下给药的新型大环肽类C5补体抑制剂，是肽类药物在这一治疗领域的重要突破。除此之外，siRNA疗法cemdisiran联合抗体疗法pozelimab治疗重症肌无力的临床研究已处于3期阶段。前者由Alnylam公司和再生元（Regeneron）联合开发，作为一种靶向C5补体的RNAi疗法，cemdisiran在进入细胞后通过与靶标mRNA结合，从而使mRNA降解，避免功能性蛋白产生。Pozelimab是再生元开发的新一代C5补体抑制剂，此前已获FDA批准用于CD55缺陷型蛋白丢失性肠病。再生元正在开发这一C5抑制剂“抗体+siRNA”组合疗法以治疗包括重症肌无力在内的补体介导的疾病。其中，cemdisiran可以减少C5的产生，pozelimab可以阻断剩余C5的功能，二者共同抑制终端补体活性，有望在较低剂量下达到完全、持续的C5抑制，减少给药频率，并且皮下给药方便临床应用。值得一提的是，cemdisiran使用的GalNAc递送技术，是当下核酸疗法尤其是siRNA递送到肝细胞的主要策略。迄今FDA批准的7款siRNA疗法中，6款均使用了这项技术。也得益于此类递送技术的持续革新，纵览全球，siRNA疗法研发蓬勃发展，在研管线已超过350款。在这一产业新兴的趋势下，围绕GalNAc等复杂分子，药明康德旗下专注于寡核苷酸和多肽及相关化学偶联药物的WuXi TIDES平台已打造了涵盖定制合成、工艺开发及生产的一站式服务平台，更好地满足全球合作伙伴的寡核苷酸及各类化学偶联物的研发需求。公开信息显示，在定制化GalNAc方面，WuXi TIDES团队已经生产过涵盖不同类型、总数超过100款的GalNAc化合物，覆盖不同类型的GalNAc分子；通过优化GalNAc树脂负载工艺，将固相合成的收率提高50%。同时，借助流动化学平台，加速高纯度GalNAc的规模化生产。在疾病的征程上，科学与技术探索的步伐从未停歇。作为创新的赋能者、客户信赖的合作伙伴以及全球健康产业的贡献者，药明康德也将持续以独特的CRDMO业务模式，助力全球合作伙伴开发包括重症肌无力在内的更多疾病领域的创新疗法，持续造福病患！ 参考资料：[1]Binks, S.N.M., Morse, I.M., Ashraghi, M. et al. Myasthenia gravis in 2025: five new things and four hopes for the future. J Neurol 272, 226 (2025). https://doi.org/10.1007/s00415-025-12922-7[2]Dresser L., Wlodarski, R., Rezania, K., & Soliven, B. (2021). Myasthenia Gravis: Epidemiology, Pathophysiology and Clinical Manifestations. J Clin Med, 10(11):2235.[3]Bubuioc, A. M., Kudebayeva, A., Turuspekova, S., Lisnic, V., & Leone, M. A. (2021). The epidemiology of myasthenia gravis. Journal of Medicine and Life, 14(1):7-16.版权说明：本文欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权或其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。