Ravulizumab-CWVZ

-- XmAb819, novel first-in-class ENPP3 x CD3 T-cell engaging bispecific antibody, Phase 1 dose expansion in CRC, NSCLC and pRCC open to enrollment; on track to present clinical data to support a recommended Phase 3 dose in ccRCC in 2H26 -- -- Final results from healthy-volunteer study of Xmb942, Xtend™ TL1A antibody, and preclinical characterization of XmAb412, TL1A x IL23p19 XmAb® bispecific antibody, to be presented at DDW 2026 -- PASADENA, Calif.--(BUSINESS WIRE)--Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered antibodies for the treatment of cancer and autoimmune diseases, today reported financial results for the fourth quarter and full year ended December 31, 2025. “Xencor’s lead oncology drug candidate is XmAb819, a novel first-in-class T-cell engager that could offer a much-needed new therapeutic modality for patients with advanced clear cell renal cell carcinoma (ccRCC). Excitement from the clinical community on our initial data from the dose-escalation study presented during ENA 2025 supports achieving our goal of presenting dose-expansion data during a medical meeting in the second half of 2026 and our plans to initiate a pivotal study during 2027,” said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. “Building on the early success in ccRCC, we recently opened additional dose expansion cohorts in other tumor types that have high ENPP3 expression: colorectal cancer, non-small cell lung cancer and papillary renal cell carcinoma.” “In 2026, we plan to present additional key clinical data and progress updates as we also advance XmAb541 and our ongoing B-cell depleting autoimmune programs, respectively. Our TL1A pipeline in inflammatory bowel disease continues to advance, as well. We are making great progress with enrollment in our Phase 2b XENITH-UC study of XmAb942 and are excited to begin the first-in-human study of XmAb412, our novel TL1A x IL23p19 bispecific antibody in the second half of 2026. We are proud of our execution across an evolved clinical pipeline and of the Xencor team’s focus on designing proteins to deliver novel medicines for patients.” Wholly Owned Pipeline Overview XmAb819 (ENPP3 x CD3), a first-in-class, tumor-targeted T-cell engaging XmAb® 2+1 bispecific antibody in development for patients with advanced clear cell renal cell carcinoma (ccRCC). The dose-expansion portion of the ongoing Phase 1 study is enrolling patients and dose-optimization continues. Tumor expansion cohorts in colorectal cancer (CRC), non-small cell lung cancer (NSCLC) and papillary renal cell carcinoma (pRCC) are now open to enrollment. Xencor plans to present new clinical data to support a recommended Phase 3 dose in 2H26 and initiate a pivotal study of XmAb819 in ccRCC during 2027. XmAb541 (CLDN6 x CD3), a first-in-class, tumor-targeted T-cell engaging XmAb 2+1 bispecific antibody in Phase 1 clinical development for patients with advanced gynecologic and germ cell tumors. Xencor plans to present new clinical data to support a recommended Phase 3 dose in 2H26 and evaluate plans for a pivotal study of XmAb541 during 2027. XmAb942 (Xtend™ anti-TL1A), a potential best-in-class, high-potency, extended half-life antibody in development for patients with inflammatory bowel disease. Xencor is conducting the global XENITH-UC Study, a Phase 2b study of XmAb942 in ulcerative colitis (UC). XENITH-UC is a randomized, double-blind, placebo-controlled trial in patients with moderate-to-severe UC, whose disease has progressed after at least one conventional or advanced therapy. Xencor is on-track to present final results from the Phase 1 study of XmAb942 in healthy volunteers in a poster titled “XmAb942, a Potential Best-in-class, Long-acting Anti-TL1A Antibody for the Treatment of Inflammatory Bowel Disease: Phase 1 Final Results” at Digestive Disease Week® (DDW), being held May 2-5, 2026 in Chicago and online, and to provide an update on progress achieved in the XENITH-UC study around year-end 2026. XmAb412 (TL1A x IL23p19), a bispecific antibody for dual targeting of important inflammatory pathways in autoimmune and inflammatory disease, while avoiding the complexities of dosing and formulary access for two separate TL1A and IL23 targeted drugs. Xencor is on track to present the preclinical characterization of XmAb412 in a poster titled “Discovery and Characterization of XmAb412: A Novel, High-Affinity, Anti-TL1A x Anti-IL23 Native-like Bispecific Antibody With Extended Half-life for the Treatment of Inflammatory Bowel Disease” at DDW in May 2026 and to initiate a first-in-human study of XmAb412 in 2H26. Plamotamab (CD20 x CD3), a clinical-stage, B-cell depleting bispecific T-cell engager in Phase 1 development for patients with rheumatoid arthritis (RA), who have progressed through prior standard-of-care treatment. Xencor plans to provide an update on progress achieved in the Phase 1b study of plamotamab in RA in 2H26. XmAb657 (CD19 x CD3), a clinical-stage, potent, extended half-life B-cell depleting bispecific T-cell engager in Phase 1 development for patients with idiopathic inflammatory myopathies (IIM). Xencor plans to provide an update on progress achieved in the Phase 1 study of XmAb657 in 2H26. XmAb808 (B7-H3 x CD28), a bispecific antibody designed to provide conditional co-stimulation of T cells when also bound to tumor cells. Xencor plans to present a poster characterizing preclinical combinations of XmAb808 with multiple CD3 T cell engaging bispecific antibodies at a medical meeting in 1H26. Xtend U.S. Patent Term Extension: In December 2025, Xencor announced the issuance of U.S. Patent 12,492,253 , which covers Xencor’s Xtend Fc domain for extending the half-life of antibodies targeting C5, with a term that extends into December 2028. Xencor anticipates receiving low-single digit royalties on net sales of Ultomiris® (ravulizumab-cwvz), an anti-C5 antibody engineered with a licensed Xtend Fc domain, into December 2028 in the United States. Ultomiris is a drug being developed and commercialized by Alexion Pharmaceuticals, Inc., and is a registered trademark of Alexion. Xencor previously secured regulatory extensions of exclusivity in several EU countries, Japan and Australia. Based upon consensus sales forecasts, Xencor estimates recognizing potential royalty revenue in excess of the caps under its royalty agreement with OMERS in the range of $100 million to $120 million in aggregate for the extended patent term through 2028. Financial Guidance: Based on current operating plans, Xencor expects to end 2026 with between $400 million and $430 million in cash, cash equivalents and marketable debt securities, and to have sufficient cash resources to fund research and development programs and operations through 2028. Financial Results for the Fourth Quarter and Full Year Ended December 31, 2025 Cash, cash equivalents, and marketable debt securities totaled $610.8 million as of December 31, 2025 compared to $706.7 million as of December 31, 2024. Total revenue for the fourth quarter ended December 31, 2025 was $28.2 million compared to $52.8 million for the same period in 2024. Revenue for the full year ended December 31, 2025 was $125.6 million compared to $110.5 million for the same period in 2024. The increase in revenue in 2025 compared to 2024 was primarily driven by revenue recognition associated with Alexion and Incyte license agreements. Research and development (R&D) expenses for the fourth quarter ended December 31, 2025 were $64.8 million compared to $51.1 million for the same period in 2024. R&D expenses for the full year ended December 31, 2025 were $239.4 million compared to $227.7 million for the same period in 2024. Increased R&D expenses in 2025 compared to 2024 were primarily driven by higher external and internal costs associated with pipeline programs, partially offset by lower stock-based compensation expense. General and administrative (G&A) expenses for the fourth quarter ended December 31, 2025 were $17.0 million compared to $14.9 million for the same period in 2024. G&A expenses for the full year ended December 31, 2025 were $63.6 million compared to $61.2 million for the same period in 2024. G&A expenses in 2025 compared to 2024 remained relatively consistent. Other income, net for the fourth quarter ended December 31, 2025 was $49.4 million compared to other expense, net of $31.4 million for the same period in 2024. Other income, net for the full year ended December 31, 2025 was $87.9 million compared to other expense, net of $56.5 million in the same period in 2024. Other income, net, in 2025 compared to other expense, net, in 2024 was primarily driven by a combination of realized and unrealized gains from marketable equity securities. Net loss attributable to Xencor for the fourth quarter ended December 31, 2025 was $6.7 million or $(0.09) on a fully diluted per share basis compared to net loss of $45.6 million or $(0.62) on a fully diluted per share basis, for the same period in 2024. For the full year ended December 31, 2025 net loss attributable to Xencor was $91.9 million or $(1.24) on a fully diluted per share basis compared to net loss of $232.6 million or $(3.58) on a fully diluted per share basis, for the same period in 2024. About Digestive Disease Week® Digestive Disease Week® (DDW) is the largest international gathering of physicians, researchers, and academics in the fields of gastroenterology, hepatology, endoscopy, and gastrointestinal surgery. Jointly sponsored by the American Association for the Study of Liver Diseases (AASLD), the American Gastroenterological Association (AGA), the American Society for Gastrointestinal Endoscopy (ASGE), and the Society for Surgery of the Alimentary Tract (SSAT), DDW is an in-person and online meeting. Digestive Disease Week® is a registered trademark of DDW, LLC. About Xencor Xencor is a clinical-stage biopharmaceutical company developing engineered antibodies for the treatment of patients with cancer and autoimmune diseases. More than 20 candidates engineered with Xencor's XmAb® technology are in clinical development, and multiple XmAb medicines are marketed by partners. Xencor's XmAb engineering technology enables small changes to a protein’s structure that result in new mechanisms of therapeutic action. For more information, please visit . Forward-Looking Statements Certain statements contained in this press release may constitute forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements that are not purely statements of historical fact, and can generally be identified by the use of words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “seek,” “look forward,” “believe,” “committed,” “investigational,” “indicates,” “supports,” and similar terms, or by express or implied discussions relating to Xencor’s business, including, but not limited to, statements regarding our expectations regarding regulatory and partnership milestone achievements, clinical pipeline advancements , planned receipt and presentations of clinical data, including the expected timing thereof, and planned and ongoing clinical trials, including the expected timing thereof, projected financial resources and financial guidance, including estimated cash, cash equivalents and marketable debt securities at year end and cash runway for research and development programs and operations, expectations for and estimates of future royalty revenues, the quotations from Xencor's president and chief executive officer, and other statements that are not purely statements of historical fact. Such statements are made on the basis of the current beliefs, expectations, and assumptions of the management of Xencor and are subject to significant known and unknown risks, uncertainties and other factors that may cause actual results, performance or achievements and the timing of events to be materially different from those implied by such statements, and therefore these statements should not be read as guarantees of future performance or results. Such risks include, without limitation, the risks associated with the process of discovering, developing, manufacturing and commercializing drugs that are safe and effective for use as human therapeutics, the ability of publicly disclosed preliminary clinical trial data to support continued clinical development and regulatory approval for specific treatments, the risk of loss of key members of management, the risk that the fair value of our marketable equity securities will decline and the risks, uncertainties and other factors described under the heading “Risk Factors” in Xencor's Annual Report on Form 10-K for the year ended December 31, 2025 as well as Xencor's subsequent filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Xencor undertakes no obligation to revise or update these forward-looking statements to reflect events or circumstances after the date hereof, except as required by law. Xencor, Inc. Selected Consolidated Balance Sheet Data (in thousands) December 31, December 31, 2025 2024 Cash, cash equivalents and marketable debt securities - current $ 435,231 $ 449,846 Other current assets 164,590 127,755 Marketable debt securities - long term 175,602 256,833 Other long-term assets 100,072 117,511 Total assets $ 875,495 $ 951,945 Total current liabilities $ 95,907 87,432 Liabilities related to the sales of future royalties - long term 76,482 115,159 Other long-term liabilities 67,519 75,328 Total liabilities 239,908 277,919 Total stockholders' equity 635,587 674,026 Total liabilities and stockholders’ equity $ 875,495 $ 951,945 Xencor, Inc. Consolidated Statements of Operations and Comprehensive Loss (in thousands, except per share amounts) Three Months Ended December 31, Year Ended December 31, 2025 2024 2025 2024 Revenue Collaborations, milestones, and royalties $ 28,237 $ 52,794 $ 125,576 $ 110,493 Operating expenses: Research and development 64,824 51,056 239,434 227,686 General and administrative 17,041 14,916 63,644 61,215 Total operating expenses 81,865 65,972 303,078 288,901 Operating loss (53,628 ) (13,178 ) (177,502 ) (178,408 ) Total other income (expense) 49,362 (31,404 ) 87,869 (56,515 ) Loss before income tax expense and noncontrolling interest (4,266 ) (44,582 ) (89,633 ) (234,923 ) Income tax expense 2,387 1,617 2,504 1,617 Net loss including noncontrolling interest (6,653 ) (46,199 ) (92,137 ) (236,540 ) Net loss attributable to noncontrolling interest — (647 ) (214 ) (3,922 ) Net loss attributable to Xencor, Inc. $ (6,653 ) $ (45,552 ) $ (91,923 ) $ (232,618 ) Net loss per share attributable to Xencor, Inc. (basic and diluted) $ (0.09 ) $ (0.62 ) $ (1.24 ) $ (3.58 ) Weighted-average shares used in calculating net loss per share (basic and diluted) 74,586 73,176 74,239 65,041 Other comprehensive income (loss), net of tax: Net unrealized gain (loss) on marketable debt securities 418 (2,464 ) 2,239 (1,954 ) Comprehensive loss (6,235 ) (48,663 ) (89,898 ) (238,494 ) Less: comprehensive loss attributable to the noncontrolling interest — (647 ) (214 ) (3,922 ) Comprehensive loss attributable to Xencor, Inc. $ (6,235 ) $ (48,016 ) $ (89,684 ) $ (234,572 ) Contacts For Investors: Charles Liles cliles@xencor.com (626) 737-8118 For Media: Cassidy McClain Inizio Evoke cassidy.mcclain@inizioevoke.com (619) 694-6291

导读：重症肌无力（MG）——这一让患者"眼睑下垂、四肢无力、甚至呼吸困难"的自身免疫性疾病，长期以来是神经免疫领域的治疗难题。约20%的患者对传统免疫抑制剂无效，沦为"难治性MG"，反复发作严重影响生存质量。近年来，以B细胞靶向药、补体C5抑制剂、FcRn拮抗剂和IL-6R抑制剂为代表的生物靶向药物，正在深刻重塑MG的治疗格局。然而，高昂的治疗费用、有限的医保覆盖、基层医生认知不足以及自主创新能力薄弱，仍是横亘在中国MG精准治疗道路上的重重壁垒。近期发表于Intractable & Rare Diseases Research的一篇综述，由复旦大学华山医院教授团队撰写，系统梳理了中国MG生物靶向治疗的最新进展与核心挑战。一、沉重的疾病负担：被低估的"神经免疫孤儿病" 重症肌无力是一种由自身抗体攻击神经肌肉接头（NMJ）导致的获得性自身免疫病，核心表现为波动性肌无力和易疲劳。约85%的患者体内存在抗乙酰胆碱受体（AChR）抗体，其余患者则以抗肌肉特异性酪氨酸激酶（MuSK）抗体或抗低密度脂蛋白受体相关蛋白4（LRP4）抗体为主，少部分患者抗体检测阴性，被归类为血清阴性MG。 中国MG的疾病负担不容忽视。 流行病学数据显示，中国MG的年发病率约为0.68/10万，患病率为7.3/10万，全国患者总数估计约65万人。患者中位住院时长为8天，中位住院费用约1037美元；而最新的全国注册登记研究显示，年均直接医疗费用中位数高达2219美元。 更令人忧虑的是死亡率数据。过去三十年间，中国MG的年龄标准化死亡率为1.86/百万，男性显著高于女性；MG患者的中位死亡年龄仅为59.45岁，比一般人群（75.47岁）低了整整16年。这些数字无声地揭示了一个事实：MG并非一种"温和"的慢性病，它实实在在地缩短着患者的生命。 疾病的影响远不止于患者本身。研究显示，约三分之一的中国MG患者存在临床显著的疾病进展恐惧（FoP），比例高于许多其他慢性病群体；而在中国西北地区的一项调查中，MG患者照护者普遍承受着沉重的家庭负担，尤其体现在经济压力和日常活动干扰方面。 此外，MG的不典型表现——如远端肢体无力、单纯球部起病、单侧或不对称起病、以呼吸衰竭为首发症状等——容易被误诊为卒中、肌病、周围神经病或肌萎缩侧索硬化症，进一步延误了患者的及时诊治。二、免疫病理机制：靶向治疗的理论基石 生物靶向治疗的兴起，根植于对MG免疫病理机制的深入理解。综述详细阐述了这一复杂的免疫学级联反应。 在疾病的启动阶段，胸腺异常（如胸腺增生或胸腺瘤）导致AChR或相关抗原在胸腺内异常表达。自身反应性CD4+ T细胞逃逸中枢免疫耐受机制，被抗原提呈细胞（如树突状细胞和巨噬细胞）激活。活化的CD4+ T细胞（主要为Th1和Th17亚群）通过细胞表面分子相互作用和促炎细胞因子分泌，为B细胞的活化提供关键的协同刺激信号。同时，Th17细胞数量增加、功能增强，加上Treg细胞功能受损，共同导致免疫调节网络失衡。 在效应阶段，自身反应性B细胞在生发中心内增殖分化为记忆B细胞和长寿浆细胞，持续产生高亲和力的抗AChR或抗MuSK自身抗体。这些抗体到达神经肌肉接头后，通过三种机制破坏突触传递：①激活补体级联反应（尤其是C5b-9膜攻击复合物），直接损伤突触后膜；②诱导AChR交联、加速内化和降解，减少功能性受体数量；③直接阻断乙酰胆碱结合位点，干扰神经递质-受体相互作用。 值得一提的是，MuSK抗体阳性MG的发病机制有所不同。抗MuSK抗体主要为不激活补体的IgG4亚类，通过阻断agrin-LRP4-MuSK信号通路，破坏突触后膜的组织结构，影响NMJ的形成与稳定。 正是基于对上述免疫病理环节的精准认知，针对B细胞、FcRn、IL-6通路和补体系统的靶向治疗策略才得以应运而生，并在临床实践中展现出令人瞩目的疗效。三、靶向治疗最新进展：六大获批药物、多线布局 综述系统回顾了各类生物靶向药物在中国的研发与临床应用进展，截至2025年9月底，中国已有6种靶向药物获批上市用于全身型MG（gMG）治疗，另有3种药物正在国家药监局审评中。3.1 B细胞靶向药物 利妥昔单抗（Rituximab） 作为抗CD20单克隆抗体，在中国临床实践中已被广泛超适应证用于难治性gMG。多项中国前瞻性和回顾性研究表明，低剂量利妥昔单抗可有效缓解患者症状、减少糖皮质激素用量，在MuSK抗体阳性患者中疗效尤为突出。虽然缺乏大型RCT支持，但真实世界数据和欧洲指南已将其推荐为gMG的替代一线治疗选择。 伊奈利珠单抗（Inebilizumab） 是一种创新的抗CD19单克隆抗体，其关键性全球III期临床试验（MINT研究）已获得阳性结果。CD19靶点较CD20覆盖范围更广，还包括部分CD19+浆细胞，可能为高滴度抗体患者提供更强效的治疗。该药已在中国获批用于NMOSD，其gMG适应症正在NMPA审评中。 泰它西普（Telitacicept） 是一种双靶点重组融合蛋白，同时阻断BAFF（BLyS）和APRIL通路，广泛抑制B细胞和抗体产生。基于积极的II期和III期关键临床试验结果，泰它西普于2025年在中国获批用于gMG治疗。这是中国首个自主研发并获批上市的MG生物靶向治疗药物，具有里程碑意义。3.2 补体C5抑制剂 依库珠单抗（Eculizumab） 于2023年在中国获批，用于难治性AChR抗体阳性gMG。多项中国前瞻性观察性研究显示，其在肌无力危象和胸腺瘤相关MG中表现出良好的疗效和耐受性。 拉伐利珠单抗（Ravulizumab） 于2025年在中国获批，作为长效C5抑制剂，负荷剂量后仅需每8周维持给药一次（依库珠单抗为每2周一次），显著减少了输注频率和相关医疗负担。 齐鲁考普兰（Zilucoplan） 于2025年10月在中国获批，是一种皮下注射的大环肽类C5抑制剂，其结合位点不同于单克隆抗体，对某些影响单克隆抗体结合的C5变异体（如PNH中报道的p.Arg885His突变）可能具有潜在抑制优势。3.3 FcRn拮抗剂 FcRn拮抗剂通过竞争性抑制IgG与FcRn的结合和循环再利用，降低循环中总IgG水平和致病性自身抗体滴度，已成为gMG治疗的新里程碑。 艾加莫德（Efgartigimod） 于2023年在中国获批，是首个获批的FcRn拮抗剂。多项中国真实世界研究已探索了其在肌无力危象、濒临危象、胸腺瘤相关MG、新发MG、眼肌型MG和血清阴性MG等不同亚型中的应用，积累了丰富的中国证据。 罗泽利昔珠单抗（Rozanolixizumab） 于2025年在中国获批，适用于AChR或MuSK抗体阳性gMG成人患者，但目前中国的使用经验仍非常有限。 巴托利单抗（Batoclimab） 和尼泊利单抗（Nipocalimab） 均为FcRn靶向单克隆抗体，其关键II/III期临床试验均取得阳性结果，目前已向NMPA提交上市前审评申请。值得特别指出的是，巴托利单抗的注册临床试验是在中国独立完成的，是中国首个发表的、在RCT中证实对gMG具有显著疗效的靶向药物。3.4 IL-6R抑制剂与细胞治疗前沿 萨特利珠单抗（Satralizumab） 在一项包含中国中心的III期研究中显示出良好的耐受性，虽对AChR抗体阳性gMG有一定改善，但疗效并不突出，罗氏公司已终止其开放标签延伸研究。 托珠单抗（Tocilizumab） 则以超适应证形式在部分中国中心使用，初步数据显示可改善gMG症状并减少糖皮质激素用量，但尚需更多证据支持。 在细胞治疗前沿，中国的探索同样令人瞩目。已有研究报道了双特异性BCMA/CD19靶向CAR-T细胞疗法成功治疗难治性AChR-MG和难治性MuSK-MG的案例，且安全性良好。这些珍贵的案例提示，CAR-T疗法有望为MG患者提供"功能性治愈"的可能。四、核心挑战：五重壁垒待破 综述坦诚而深刻地剖析了中国MG生物靶向治疗面临的五大核心挑战。4.1 传统治疗与靶向治疗的优化整合 传统免疫抑制剂仍是MG治疗的基础，但长期使用（尤其是感染风险）不容忽视。FcRn拮抗剂和C5抑制剂可快速改善AChR阳性gMG症状，但其靶点在免疫级联反应的下游，并不直接清除产生自身抗体的上游记忆B细胞和浆细胞。当前的主要挑战在于：如何在经济考量与临床疗效之间取得平衡，优化传统药物与靶向药物的联合策略。 综述提出，治疗决策应以抗体亚型（AChR、MuSK、LRP4）、临床分型（眼肌型vs全身型）和胸腺瘤状态为导向，实施分层化、个体化、动态评估的治疗方案。4.2 可及性与可负担性困境 生物靶向药物疗效显著，但高昂的治疗费用和有限的医保报销严重制约了患者获益。以艾加莫德为例，即便纳入医保报销，患者仍需自付约20%-30%的费用，而经济欠发达的农村或偏远地区患者则可能完全无法获得。医保目录更新的滞后性进一步加剧了准入差距。 例如，罗泽利昔珠单抗虽已在中国获批上市并适用于MuSK-MG患者，但由于尚未纳入医保目录，符合条件的患者无法获得报销。作者呼吁加速新型生物制剂的医保准入进程，并探索基本医保与商业保险相结合的多元化支付模式。4.3 高质量循证证据匮乏 中国现有的MG治疗证据基础仍然薄弱，大多数研究为小样本回顾性分析，缺乏高质量RCT。此外，中国人群遗传多样性对靶向治疗疗效影响的系统评估尚未开展，限制了现有指南的本土化适配。真实世界登记研究的缺失也阻碍了疗效预测模型和个体化治疗策略的发展。迫切需要建立全国性MG数据库，启动高质量、多学科RCT，以填补证据空白。4.4 基层医生实践与学术共识的滞后 中国MG管理存在显著的区域差异。大城市三甲医院已广泛采用生物靶向治疗，而基层医疗机构仍主要依赖传统方案，对FcRn拮抗剂等新型制剂认知有限。尽管2025年版中国MG诊疗指南已发布，但许多临床医生尚未将最新进展融入日常诊疗。综述建议通过全国继续医学教育项目加强学术培训，建立区域性多学科平台促进经验共享，并推动针对MG各亚型靶向治疗优先序和监测指标的专家共识。4.5 自主创新研发能力不足 在已获批或在审的MG靶向药物中，仅有泰它西普代表了中国原创创新。在已完成的众多RCT中，仅有泰它西普和巴托利单抗是在中国独立完成的。这一令人尴尬的现状折射出中国在生物靶向治疗领域的研发能力短板。 长期以来依赖临床需求驱动和仿制药路径，导致自主创新能力相对有限，基础研究投入不足，真正意义上的"first-in-class"或"best-in-class"创新药仍然凤毛麟角。此外，不断变化的政策环境、市场准入难度以及国际化的复杂性，也对中国创新生物技术企业构成了严峻挑战。 五、结语：从"追随者"到"引领者"的转型之路 生物靶向治疗已深刻重塑了中国MG的治疗格局，为难治性患者带来了全新的希望。 然而，优化临床应用、改善药物可及性和可负担性、加大自主创新投入、以及产出高质量的本土循证证据，仍然是实现MG精准治疗全国普惠的关键任务。 值得欣慰的是，变革的晨光已经显现——中国正积极参与全球多中心临床试验，泰它西普的获批标志着中国MG靶向治疗"从0到1"的突破，CAR-T细胞治疗的前沿探索更昭示着"功能性治愈"的巨大潜力。未来，建立以生物标志物驱动、基于亚型分层的治疗框架，实施适应性策略调整，将是实现MG精准医学的核心方向。 中国在MG靶向治疗领域正迈向从"追随者"到"引领者"的关键转型期。这条道路虽然挑战重重，但每一步前行，都在为全国65万MG患者拓展生命的宽度与深度。 参考文献：Gu S, Zhao C. Current status and challenges of biologic targeted therapy for myasthenia gravis in China. Intractable & Rare Diseases Research. 2026; 15(1):45-53. DOI: 10.5582/irdr.2025.01061