Velzatinib

Approval based on AZUR-1 data showing clinically significant proportion of participants with no detectable signs of cancer one year or more after treatment No disease recurrences reported among responders after median follow up of more than two years Jemperli monotherapy could help eligible patients avoid chemotherapy, radiation and surgery PHILADELPHIA--(BUSINESS WIRE)-- #AZUR --GSK plc (LSE/NYSE: GSK) today announced the US Food and Drug Administration (FDA) has approved Jemperli (dostarlimab-gxly) as the first and only immunotherapy for adults with stage II and III microsatellite instability-high (MSI-H)/mismatch repair deficient (dMMR) locally advanced rectal cancer under the accelerated approval pathway. Current standard of treatment for locally advanced rectal cancer includes chemotherapy, radiation and surgery 1 , but these can have lasting consequences, including bowel, urinary and sexual dysfunction, secondary cancers and infertility 2,3 . For patients with MSI-H/dMMR tumors, a biomarker found in around 5-10% of rectal cancers 4 , Jemperli monotherapy could help avoid these interventions. Tony Wood, Chief Scientific Officer, GSK, said: “Today, Jemperli becomes the first and only approved immunotherapy for adults in the US with MSI-H/dMMR locally advanced rectal cancer. For decades, treatment has relied on chemotherapy, radiation and surgery. This approval offers a new treatment approach and represents an important step towards more personalized care for these patients.” Dr. Andrea Cercek, MD, Principal Investigator of AZUR-1, Section Head of Colorectal Cancer and Co-Director of the Center for Young Onset Colorectal and Gastrointestinal Cancer, Memorial Sloan Kettering Cancer Center, said: “The AZUR-1 results demonstrate the potential for a fundamentally different treatment approach in this biomarker-defined patient population, and today’s approval means MSI-H/dMMR patients can now access a licensed treatment tailored to the biology of their disease.” Michael Sapienza, CEO, Colorectal Cancer Alliance, said: “People facing rectal cancer are often thinking not only about treating their disease, but also worrying about what life will look like afterwards. The possibility of avoiding chemotherapy, radiation and surgery represents a meaningful advance for patients and their families. Biomarker testing at diagnosis will be essential to identifying patients who may be eligible for this treatment and helping them understand their options.” Approval was based on data from the phase II AZUR-1 trial. The study met its primary objective, demonstrating a clinically significant proportion of participants achieved sustained clinical complete responses at 12 months (cCR12), with data continuing to mature. Investigator-assessed data, a clinically relevant endpoint, showed 68% of evaluable patients (98/144; 95% CI: 60%, 76%) achieved cCR12. In an exploratory analysis, 76% (110/144; 95% CI: 69%, 83%) of evaluable patients had no evidence of cancer one year or more after treatment. This includes 68% (98/144) (95% CI: 60%, 76%) of patients who reached cCR12 without surgery, plus other participants (n=12) who had their complete response confirmed through tissue analysis after surgery (pathological complete response, or pCR). No disease recurrences have been reported among responders to date, after a median follow up of more than two years. Data on the primary objective, cCR12 assessed by Independent Central Review, continue to mature. 47% (54/116) (95% CI: 37%, 56%) of evaluable patients have achieved cCR12. An additional 38 participants who are still progressing through to 12 month follow up have already achieved a clinical complete response. The safety pro Jemperli was generally consistent with its established pro solid tumors. Serious adverse reactions occurred in 20% of patients receiving Jemperli, and the most common adverse reactions were fatigue (32%), rash (22%), diarrhea (18%) and pruritus (17%). Jemperli was permanently discontinued due to adverse reactions in 3% of patients. Full data from the AZUR-1 interim analysis will be presented at the European Society for Medical Oncology (ESMO) Congress in October 2026. Jemperli has received FDA Fast Track and Breakthrough Therapy designations for this indication. The application was also granted Priority Review and received a National Priority Voucher, resulting in an accelerated approval ahead of the PDUFA date. The supplemental Biologics License Application (sBLA) is being reviewed under Project Orbis, an FDA Oncology Center of Excellence initiative that enables concurrent review by international health authorities. Regulatory reviews are ongoing in participating countries. About stage II/III MSI-H/dMMR locally advanced rectal cancer Around 5–10% of rectal cancers are microsatellite instability-high (MSI-H) or mismatch repair-deficient (dMMR) 4 . These tumors have a specific genetic characteristic where they are unable to properly repair DNA damage, leading to an accumulation of mutations. This unique biological feature often makes them highly responsive to immunotherapies like dostarlimab-gxly 5,6 . These biomarkers are commonly found in colorectal, endometrial and other gastrointestinal cancers, but can also be present in other solid tumors 7 . About AZUR-1 AZUR-1 is a global, open-label, single-arm, registrational phase II trial evaluating dostarlimab-gxly monotherapy in patients (n=154) with previously untreated stage II and III MSI-H/dMMR locally advanced rectal cancer. The trial was designed to assess clinical complete responses sustained for 12 months (cCR12) as determined by Independent Central Review, and to determine whether dostarlimab-gxly alone could enable patients to avoid chemotherapy, radiation and surgery. Additional efficacy outcomes include cCR12 based on investigator assessment and objective response rate (ORR), defined as patients who achieved cCR, clinical near-complete response, or clinical partial response as their best overall response. Patients received nine cycles of dostarlimab-gxly over six months, administered as a 500mg intravenous infusion every three weeks. The safety and tolerability pro dostarlimab-gxly was generally consistent with its well-characterized safety pro across solid tumors. Serious adverse reactions occurred in 20% of patients receiving Jemperli and treatment was permanently discontinued due to adverse reactions in 3% of patients. The most common adverse reactions (≥15%) were fatigue, rash, diarrhea and pruritus. About Jemperli (dostarlimab-gxly) Jemperli , a programmed death receptor-1 (PD-1)-blocking antibody, plays an important role in GSK’s immune-oncology research and development program. A robust clinical trial program includes studies of Jemperli alone and in combination with other therapies in gynecologic, colorectal, and head and neck cancers, as well as where there are opportunities for transformational outcomes. Jemperli was discovered by AnaptysBio, Inc. and licensed to TESARO, Inc., under a collaboration and exclusive license agreement signed in March 2014. Under this agreement, GSK is responsible for the ongoing research, development, commercialization and manufacturing of Jemperli . More information about the product and its currently approved indications is available at EU product information 8 and US product information 9 . Indications and Important Safety Information for JEMPERLI (dostarlimab-gxly) JEMPERLI, in combination with carboplatin and paclitaxel, followed by JEMPERLI as a single agent, is indicated for the treatment of adult patients with primary advanced or recurrent endometrial cancer (EC). JEMPERLI, as a single agent, is indicated for the treatment of adult patients with mismatch repair deficient (dMMR) recurrent or advanced: EC, as determined by an FDA-authorized test, that has progressed on or following prior treatment with a platinum-containing regimen in any setting and are not candidates for curative surgery or radiation, or solid tumors, as determined by an FDA-authorized test, that have progressed on or following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). JEMPERLI, as a single agent, is indicated for the treatment of adult patients with Stage II/III, locally advanced rectal cancer (LARC) that is microsatellite instability-high (MSI-H) or dMMR, as determined by an FDA-authorized test for solid tumors. This indication is approved under accelerated approval based on sustained clinical complete response for 12 months. The long-term risk of relapse is unknown, with additional follow-up expected. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Important Safety Information Severe and Fatal Immune-Mediated Adverse Reactions Immune-mediated adverse reactions, which can be severe or fatal, can occur in any organ system or tissue and can occur at any time during or after treatment with a PD-1/PD-L1–blocking antibody, including JEMPERLI. Monitor closely for signs and symptoms of immune-mediated adverse reactions. Evaluate liver enzymes, creatinine, and thyroid function tests at baseline and periodically during treatment. For suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate. Based on the severity of the adverse reaction, withhold or permanently discontinue JEMPERLI. In general, if JEMPERLI requires interruption or discontinuation, administer systemic corticosteroids (1 to 2 mg/kg/day prednisone or equivalent) until improvement to ≤Grade 1. Upon improvement to ≤Grade 1, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose immune-mediated adverse reaction is not controlled with corticosteroids. Immune-Mediated Pneumonitis JEMPERLI can cause immune-mediated pneumonitis, which can be fatal. In patients treated with other PD-1/PD-L1–blocking antibodies, the incidence of pneumonitis is higher in patients who have received prior thoracic radiation. Pneumonitis occurred in 2% (15/759) of patients receiving JEMPERLI as a single agent, including Grade 2 (1.2%), Grade 3 (0.7%), and Grade 4 (0.1%) pneumonitis. Immune-Mediated Colitis Colitis occurred in 1.2% (9/759) of patients receiving JEMPERLI as a single agent, including Grade 2 (0.7%) and Grade 3 (0.5%) adverse reactions. Cytomegalovirus infection/reactivation have occurred in patients with corticosteroid-refractory immune-mediated colitis. In such cases, consider repeating infectious workup to exclude alternative etiologies. Immune-Mediated Hepatitis JEMPERLI can cause immune-mediated hepatitis, which can be fatal. Grade 3 hepatitis occurred in 0.5% (4/759) of patients receiving JEMPERLI as a single agent. Immune-Mediated Endocrinopathies Adrenal Insufficiency Adrenal insufficiency occurred in 1.1% (8/759) of patients receiving JEMPERLI as a single agent, including Grade 2 (0.4%) and Grade 3 (0.7%). For Grade 2 or higher adrenal insufficiency, initiate symptomatic treatment per institutional guidelines, including hormone replacement as clinically indicated. Withhold or permanently discontinue JEMPERLI depending on severity. Hypophysitis JEMPERLI can cause immune-mediated hypophysitis. Grade 3 hypophysitis occurred in 0.4% (1/241) of patients receiving JEMPERLI in combination with carboplatin and paclitaxel. Grade 2 hypophysitis occurred in 0.3% (2/759) of patients receiving JEMPERLI as a single agent. Initiate hormone replacement as clinically indicated. Withhold or permanently discontinue JEMPERLI depending on severity. Thyroid Disorders Grade 2 thyroiditis occurred in 0.4% (3/759) of patients receiving JEMPERLI as a single agent. Grade 2 hypothyroidism occurred in 12% (30/241) of patients receiving JEMPERLI in combination with carboplatin and paclitaxel. Grade 2 hypothyroidism occurred in 8% (58/759) of patients receiving JEMPERLI as a single agent. Hyperthyroidism occurred in 3.3% (8/241) of patients receiving JEMPERLI in combination with carboplatin and paclitaxel, including Grade 2 (2.9%) and Grade 3 (0.4%). Hyperthyroidism occurred in 2.2% (17/759) of patients receiving JEMPERLI as a single agent, including Grade 2 (2.1%) and Grade 3 (0.1%). Initiate thyroid hormone replacement or medical management of hyperthyroidism as clinically indicated. Withhold or permanently discontinue JEMPERLI depending on severity. Type 1 Diabetes Mellitus, Which Can Present with Diabetic Ketoacidosis JEMPERLI can cause type 1 diabetes mellitus, which can present with diabetic ketoacidosis. Grade 3 type 1 diabetes mellitus occurred in 0.4% (1/241) of patients receiving JEMPERLI in combination with carboplatin and paclitaxel. Grade 3 type 1 diabetes mellitus occurred in 0.1% (1/759) of patients receiving JEMPERLI as a single agent. Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Initiate treatment with insulin as clinically indicated. Withhold or permanently discontinue JEMPERLI depending on severity. Immune-Mediated Nephritis with Renal Dysfunction JEMPERLI can cause immune-mediated nephritis, which can be fatal. Grade 2 nephritis, including tubulointerstitial nephritis, occurred in 0.4% (3/759) of patients receiving JEMPERLI as a single agent. Immune-Mediated Dermatologic Adverse Reactions JEMPERLI can cause immune-mediated rash or dermatitis. Bullous and exfoliative dermatitis, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN), and drug rash with eosinophilia and systemic symptoms (DRESS), have occurred with PD-1/PD-L1–blocking antibodies. One patient receiving JEMPERLI experienced Grade 3 SJS, which resolved. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate non-bullous/exfoliative rashes. Withhold or permanently discontinue JEMPERLI depending on severity. Other Immune-Mediated Adverse Reactions The following clinically significant immune-mediated adverse reactions occurred in 2%) were decreased lymphocytes, decreased sodium, increased alanine aminotransferase, increased creatinine, decreased neutrophils, decreased albumin, and increased alkaline phosphatase. The most common adverse reactions (≥20%) in patients with dMMR solid tumors who received JEMPERLI as a single agent were fatigue/asthenia, anemia, diarrhea, and nausea. The most common Grade 3 or 4 laboratory abnormalities (≥2%) were decreased lymphocytes, decreased sodium, increased alkaline phosphatase, and decreased albumin. The most common adverse reactions (≥15%) in patients with MSI-H/dMMR LARC who received JEMPERLI as a single agent were fatigue, rash, diarrhea, and pruritus. The most common Grade 3 or 4 laboratory abnormalities (≥2%) were decreased lymphocytes, decreased hemoglobin, and increased AST. Please see full Prescribing Information, including Medication Guide. GSK in oncology Our ambition in oncology is to help increase overall quality of life, maximize survival and change the course of disease, expanding from our current focus on blood and women’s cancers into lung and gastrointestinal cancers, as well as other solid tumors. This includes accelerating priority programs such as antibody-drug conjugates Ris-Rez targeting B7-H3 and Mo-Rez targeting B7-H4, and velzatinib, a highly selective KIT tyrosine kinase inhibitor. About GSK GSK is a global biopharma company with a purpose to unite science, technology, and talent to get ahead of disease together. Find out more at Cautionary statement regarding forward-looking statements GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described in the “Risk Factors” section in GSK’s Annual Report on Form 20-F for 2025, and GSK’s Q2 Results for 2026. Registered in England & Wales: No. 3888792 Registered Office: 79 New Oxford Street London WC1A 1DG References: Cercek A, Lumish M, Sinopoli J, et al. PD-1 blockade in mismatch repair–deficient, locally advanced rectal cancer. N Engl J Med 2022; 386: 2363-76. Negro, S. et al. (2025) ‘Quality of life in rectal cancer treatments: a systematic review’, Cancers, 17(14), 2310. Available at: Neibart, S.S. et al. (2020) ‘Quality of life after radiotherapy for rectal cancer’, Current Colorectal Cancer Reports, 16(1), pp. 1–10. Available at: Cercek A, et al. Mismatch Repair-Deficient Rectal Cancer and Resistance to Neoadjuvant Chemotherapy. Clin Cancer Res. 2020 Jul 1;26(13):3271-3279. doi: 1158/1078-0432.CCR-19-3728. Epub 2020 Mar 6. PMID: 32144135; PMCID: PMC7348681. Andre T, Berton D, Curigliano G, et al. Antitumor Activity and Safety of Dostarlimab Monotherapy in Patients With Mismatch Repair Deficient Solid Tumors: A Nonrandomized Controlled Trial. JAMA Netw Open. 2023;6(11):e2341165. doi:10.1001/jamanetworkopen.2023.41165. Le DT, et al. PD-1 blockade in tumors with mismatch repair deficiency. N Engl J Med. 2015;372(26):2509-2520. National Cancer Institute at the National Institutes of Health. Definition of mismatch repair deficiency. Accessed September 2026. Available at: JEMPERLI (dostarlimab) Summary of Product Characteristics. GlaxoSmithKline (Ireland) Limited. Available at: Accessed October 2026. JEMPERLI (dostarlimab-gxly) prescribing information. GlaxoSmithKline. Available at: Accessed October 2026. Memorial Sloan Kettering Cancer Center Disclosure: Dr Cercek has financial interests related to GSK. Contacts GSK inquiries Media: Tim Foley +44 (0) 20 8047 5502 (London) Sarah Clements +44 (0) 20 8047 5502 (London) Kathleen Quinn +1 202 603 5003 (Washington DC) Alison Hunt +1 540 742 3391 (Washington DC) Stephanie Caccomo +1 202 893 2840 (Washington DC) Investor Relations: James Dodwell +44 (0) 7881 269066 (London) Mick Readey +44 (0) 7990 339653 (London) Joanna Tuplin +44 (0) 7788 351650 (London) Sam Piper +44 (0) 7824 525779 (London) Dan Smith +44 (0) 7823 523885 (London) Jeff McLaughlin +1 215 751 7002 (Philadelphia) Frannie DeFranco +1 215 751 3126 (Philadelphia)

2027年5月9日，CPIC焕新升级，以更大规模、更强阵容，在上海“四叶草”金色大厅盛大启幕，向世界发出中国医药创新的最强音。 9月，GSK接连落子中国创新药资产：月初先拿下和黄医药KRAS-EGFR ATTC的海外权益，月中又从恩沐生物引进一款三抗TCE，增厚血液瘤管线。 同期，在2026年世界肺癌大会（WCLC）上，GSK与许可方翰森制药宣布，其在中国开展的关键性III期试验ARTEMIS-008取得OS获益。该试验评估B7-H3 ADC瑞康立伏妥塔单抗（Ris-Rez）对比托泊替康，用于一线铂类治疗后疾病进展的复发性小细胞肺癌（SCLC）患者。 这些交易和数据的背后，是GSK一个更宏大的目标。2025年2月，GSK将2031年销售目标上调至突破400亿英镑（约3700亿人民币）。 然而，宏大的目标面前横亘着即将到来的专利悬崖：其HIV核心成分dolutegravir专利最早2027年到期，相关4款药物2025年合计营收76.87亿英镑，占公司总营收超过23%。 为此，GSK急需找到新的增长引擎，而中国创新药，正是这套逻辑中最具确定性的增量之一。 从2023年引进翰森两款ADC开始，GSK持续系统性下注中国资产，累计交易达到15笔，首付款与里程碑总规模在200亿美元左右。 合作逻辑也在迭代：从成熟管线商业授权，到并购海外关联研发主体，再到前置卡位早期源头资产——中国创新药已不只是“可拥抱的补充”，而是决定跨国药企未来布局甚至增长命运的关键变量。 TONACEA 01 ADC狂飙 先看看瑞康立伏妥塔单抗交出的“成绩单”。 截至2026年6月6日，中位随访12.2个月时，瑞康立伏妥塔单抗组的中位OS长达18.5个月，托泊替康组为10.3个月，两组相差8.2个月，死亡风险相对降低54%（HR=0.46）。 18.5个月——这是目前二线SCLC临床研究中报告过的最长OS，复发SCLC的疗效天花板被抬到了一个新的高度。 其它终点从不同侧面补充了这份生存答卷。瑞康立伏妥塔单抗组的中位PFS达到7.2个月，较托泊替康组延长4.2个月，疾病进展或死亡风险降低67%（HR=0.33）。 两组客观缓解率（ORR）分别为58.3%和12.6%，疾病控制率（DCR）分别为90.4%和60.2%，中位缓解持续时间（DOR）分别为6.9个月和5.6个月。其中，瑞康立伏妥塔单抗组有4例患者实现了完全缓解（CR）。 疗效之外，安全性是复发SCLC治疗取舍绕不开的另一面。 瑞康立伏妥塔单抗组3级或以上治疗相关不良事件（TRAEs）发生率为60.9%，低于托泊替康组的78.2%，血液学毒性也更低，尤其是≥3级血小板计数下降——两组分别为13.9%和59.7%，差距显著。 不过需要注意的是，瑞康立伏妥塔单抗的间质性肺病（ILD）发生率高于对照组（11.7% vs 1.9%），但大部分为1-2级事件，没有4-5级事件。ILD是ADC药物的已知类效应，在真实世界中仍需监测和管理。 GSK研发高级副总裁、全球肿瘤负责人Hesham Abdullah表示：“这些结果进一步充实了Ris-Rez的证据体系，并标志着我们肺癌产品组合向前迈出重要一步。本研究中观察到的生存显著改善，以及令人鼓舞的安全性特征，为GSK在全球范围内推进Ris-Rez用于小细胞肺癌后线和更早期治疗设置提供了更多动力。” 2023年12月，GSK以1.85亿美元首付款、最多15.25亿美元的里程碑付款，拿下翰森制药这款在研ADC的海外权益——随着ARTEMIS-008的阳性结果公布，GSK对中国ADC资产的判断得到了初步验证。 目前，GSK正在全球范围内推进其在肺癌、前列腺癌及其他实体瘤中的开发，包括针对复发性广泛期SCLC的III期EMBOLD SCLC-301试验，关键数据预计明年公布。 除了这款靶向B7-H3的ADC，在这之前，GSK还从翰森制药获得另一款B7-H4 ADC（Mo-Rez）大中华区以外的全球开发及商业化权益，首付款8500万美元，后续里程碑付款高达14.85亿美元。 2026年妇科肿瘤学会年会上，GSK展示了该产品BEHOLD-1研究结果：针对铂耐药性卵巢癌，最高剂量5.8mg/kg组的确认ORR达到62%，21位患者出现肿瘤缩小；针对复发或晚期子宫内膜癌，4.8mg/kg组的确认ORR更高，为67%。 安全性同样可圈可点：因副作用停药的患者比例极低，卵巢癌组为0，子宫内膜癌组为4%；间质性肺炎总发生率仅3%，且全部为轻中度，这在ADC治疗中是一个相当重要的安全指标。 I期数据让GSK信心大增。这家巨头已宣布，将在2026年内启动五项全球性III期临床试验，覆盖卵巢癌和子宫内膜癌的多个治疗线别，包括一线维持治疗、二线治疗等更早期的应用场景——纵观GSK过往，这是它首次在妇科肿瘤领域将一款ADC从I期直接推入大规模III期开发。 目前，还没有一款B7-H4 ADC获批上市，进入临床的也屈指可数。Mo-Rez之外，无非阿斯利康的ADZ8205、Seagen的SGN-B7H4V以及Mersana的XMT-1660，基本在中早期开发阶段。 近期，在伦敦Sidley医疗健康投资会议上，谈及与翰森制药的交易，GSK肿瘤业务拓展总监Hoyin Lam表示，“那是我们第一次从战略上真正体验中国近年来实际能够交付的速度、规模和质量。” TONACEA 02 “中国解药” GSK对中国创新药的兴趣，并非只有翰森制药。翰森制药创始人钟慧娟的丈夫，也就是孙飘扬担任董事长的恒瑞医药，也在两年后进入GSK的朋友圈。 “我们还常说，我们是最早在中国建立真正大型合作伙伴关系的（制药公司）之一。”Hoyin Lam说道。 2025年7月，GSK与恒瑞医药达成一项总金额高达120亿美元的战略合作，GSK将向恒瑞医药支付5亿美元首付款，获得HRS-9821的全球权益，以及最多11个项目的全球独家选择权。 HRS-9821是一款潜在的同类最佳PDE3/4抑制剂，正处于临床开发阶段，用于治疗COPD。而PDE3/4抑制剂领域，进入市场的仅有2024年6月获批的Ohtuvayre。SeekingAlpha预测，Ohtuvayre的年峰值销售额将在21亿至36亿美元。 2025年7月，为获得这款重磅产品，默沙东以约100亿美元的对价把开发公司Verona Pharma买下，足可见这个其中的增长空间。 跟恒瑞医药120亿美元的大单，不仅让GSK闯进PDE3/4抑制剂赛场，还为其带来一系列其他候选管线权益。公告称，这些项目经严格筛选，旨在扩充GSK在呼吸、自免和炎症、肿瘤等治疗领域已建立的研发管线，且所有项目经评估均具有成为同类最佳或同类首创的资质。 如果再往前看看，就会发现，GSK与恒瑞医药的羁绊，其实更早发生。 2024年1月，GSK以10亿美元首付款、4亿美元里程碑付款作为条件收购了Aiolos Bio（前身为One Bio），而这家Biotech的唯一管线，就是2023年8月从恒瑞医药引进的TSLP单抗SHR-1905（后更名为AIO-001）。 彼时，One Bio为这笔交易支付的首付款和近期里程碑付款不过2500万美元，不到半年，溢价达到40倍。 在Aiolos Bio之后，今年1月，GSK再一次“被中间商赚差价”，GSK以22亿美元收购RAPT Therapeutics，获得一款IgE抗体。而这一管线是RAPT在2024年从上海济煜医药引进的。 两次类似的交易说明：中国资产一旦被海外团队接手、产生海外临床数据，GSK并不介意支付更高溢价。海外开发能力和数据背书，本身就是加价项。 聚焦肿瘤领域，TCE也是GSK关注的方向。 2025年11月，GSK与泽安生物达成合作，推进新型髓系细胞衔接器（MCE）开发，涉及4种潜在同类首创MCE疗法，首付款5000万美元；更早的2024年10月，GSK还以8.5亿美元总交易价格引进恩沐生物的CD3/CD19/CD20三抗（CMG1A46）。 还有这两年火出天际的小核酸领域，GSK同样没有错过。 2026年2月，GSK从前沿生物引进两款早期siRNA管线，首付款4000万美元、近期里程碑1300万美元，后续最高9.5亿美元里程碑。 紧接着5月，时安生物也宣布，将ALK7 siRNA新药SA030的大中华区外全球权益授权给GSK，总作价高达10.05亿美元，外加分级销售分成，该候选管线已进入I期临床。 综合来看，GSK对中国资产的兴趣是全方位的：从小分子、抗体等较为成熟的药物模态，到ADC、MCE、siRNA这些风头正盛的新兴技术，治疗领域涉及其肿瘤、呼吸、感染等多个核心方向。 TONACEA 03 利用中国创新与速度 比“买了什么”更值得关注的，是“怎么在买”——交易结构本身正在发生变化。 GSK与中国药企的交易呈现出两个清晰趋势：一是复购，对中国资产的态度，已从审慎尝试转向系统性下注；二是早期化，越来越偏向临床早期资产，这既意味着对平台能力的信任，也对应着低首付、高里程碑的交易结构。 最近的这两笔交易，把这两个特征体现得尤为明显。 先说跟和黄医药这笔合作。HMPL-A830是一款由高选择性、高活性的KRAS小分子抑制剂作为有效载荷，与抗EGFR抗体偶联而成的ATTC药物，这也是和黄医药ATTC平台首个对外授权的分子。 ATTC平台是和黄医药依托自身在靶向小分子领域二十余年的积淀，将小分子抑制剂作为有效载荷与抗体偶联，开创了与传统ADC截然不同的技术路线。 该平台通过“靶向递送+通路抑制” 的双重机制，既利用了抗体的精准靶向性，又通过小分子抑制剂的通路阻断作用实现协同抗肿瘤效果。 在临床前模型中，ATTC展现出比单独使用抗体或小分子抑制剂更优的疗效和安全性。此外，ATTC有望克服全身给药小分子抑制剂在非肿瘤靶向毒性方面的限制，确保长期用药的安全性，并支持在更早的治疗线中与化疗和免疫疗法进行联合用药。 肿瘤是GSK四大核心治疗领域之一，此前通过与翰森制药、映恩生物的合作，已经引进3条ADC管线，此次合作，将帮助GSK从ADC进一步拓展到ATTC，丰富其肿瘤管线布局。 此外，值得关注的是，针对这次合作，GSK还获得了一款更早期ATTC候选药物的优先谈判权。首付款将在交易完成时支付，但须满足惯常的交割条件，包括完成反垄断监管审查。 与恩沐生物的这笔交易，则是一次精准的“二次复购”，背后是GSK在肿瘤领域的两层考量：短期看，是补强多发性骨髓瘤管线；长期看，是锁定恩沐在多特异性TCE上的平台能力。 这款三特异性TCE被GSK定位为“潜在同类最佳”，同时靶向T细胞和两个肿瘤抗原，用于治疗多发性骨髓瘤，计划2027年推进到I期临床。 其设计的突破性在于，它试图解决当前TCE疗法在临床上面临的核心矛盾——疗效与耐受性难以兼得。三特异性TCE在结合T细胞的同时，靶向两个经过战略筛选和验证的肿瘤相关抗原，旨在实现更深、更持久的缓解，同时改善耐受性。 如果这一设计在临床上得到验证，将意味着该疗法有望从后线推向更早的治疗线，覆盖更广泛的多发性骨髓瘤患者群体。 更值得关注的是，GSK看中的显然不只是这一条管线本身。 之前引进的CMG1A46本身也是一个三特异设计：CD19×CD20×CD3。它利用CD19和CD20同时覆盖B细胞，并通过低亲和力CD3设计，希望实现更深、更广泛的B细胞清除，同时控制传统TCE容易出现的系统性T细胞激活毒性。GSK后来将这一项目的重要开发方向，放到了系统性红斑狼疮等B细胞驱动的自身免疫疾病上。 两年后，GSK再次从同一家公司购买“双抗原×CD3”三特异TCE，只不过这一次，战场从B细胞自身免疫疾病转向了多发性骨髓瘤，这说明GSK看中的，是恩沐围绕多特异TCE形成的分子设计能力——一种可复用的平台价值，而非单条管线。 两个案例放在一起看，GSK的交易逻辑已经清晰：复购，是因为验证过的平台值得持续下注；早期化，则是源于对中国临床推进速度和平台能力的信心。 而最终，这套打法能否持续，取决于一个更根本的问题——中国创新的质量与速度，是否真的在系统性提升。 Hoyin Lam认为，中国的研究质量近年来“确实一直在提高”，“因为此前在大型药企工作的那些人……要么在中国创办自己的公司，要么为几家较大的中国药企工作。” 他说：“你不能忽视近年来中国创新提速有多快。GSK乐于与中国伙伴合作，真正利用这一点。” TONACEA 04 “错失恐惧症” GSK之所以连续押注中国早期资产，更深层的因素是一种错失恐惧症（FOMO）——在抗肿瘤领域，GSK失去过数年。 2014年4月，GSK与诺华达成资产互换协议：诺华以145亿美元买下GSK的肿瘤治疗业务，GSK则整合诺华除流感疫苗之外的疫苗业务。 时任CEO Andrew Witty认为，GSK在抗肿瘤领域不具备竞争规模，将该业务转到“全球领导者手中”是明智的，交易也强化了疫苗和消费者保健两大核心业务。 随着2017年4月Walmsley掌舵，GSK制药版图再次洗牌，肿瘤被提到与呼吸系统、HIV、免疫炎症并列的四大核心领域之一。到2017年末，GSK借着BCMA CAR-T进入II期临床的消息，高调重返抗肿瘤赛道。 但船大难掉头：PD-1抗体Jemperli、PARP抑制剂Zejula销售一度不及预期；2020年FDA加速批准的全球首款BCMA ADC Blenrep，也在2022年11月宣布未能达到确证性临床主要终点，15天后启动撤市，直到2025年10月才二度进入美国市场。 不过，坚持也开始显露成效。 2025年，GSK营收326.67亿英镑，特药板块收入135亿英镑，成为增长的绝对引擎。其中，抗肿瘤业务表现最突出，全年销售额为20亿英镑，同比增长43%，增速位居四大板块之首。 但抗肿瘤要成为顶梁柱仍有距离，相对之下，HIV药品销售额77亿英镑，呼吸/免疫炎症38亿英镑，疫苗板块92亿英镑。 更大的挑战还在前方。HIV核心成分dolutegravir分子专利最早2027年到期，相关4款药物Tivicay、Triumeq、Juluca、Dovato 2025年合计营收76.87亿英镑，占总营收超过23%。 严峻形势之下，GSK必须找到新的增长引擎，而交易是最直接的方法。 因而，过去两年，GSK的“买买买”并不令人意外：收购IDRx，获得胃肠道间质瘤药物IDRX-42；从Boston Pharmaceuticals买下已进入III期临床的efimosfermin alfa，用于治疗代谢相关脂肪性肝炎；收购35Pharma，拿下肺动脉高压药物HS235…… 今年6月，GSK还打破自己“补强型收购”的交易逻辑，以106亿美元收购美国生物技术公司Nuvalent，成为2026年业内最大的交易之一。 “这并不排除中国以外还存在其他创新，”Hoyin Lam继续说，“我们最近在欧洲也看到了值得注意的交易。在美国，我们与Nuvalent完成了一笔即便不是最大、也是我们最大之一的并购交易。” “因此，我们确实在平衡西方与东方，但真正取各自在不同方面所擅长之处。”Hoyin Lam总结道。 — 写在最后 — 摩根大通亚太区医疗健康投资银行副主席刘伯伟曾在采访中表示，“成本与效果的极致平衡，正成为跨国药企将中国视为核心研发管线来源地的关键逻辑”。 回到GSK上，中国市场既是补足管线的“武器库”，也是验证其转型战略成败的“试验场”。 近期B7-H3 ADC管线的成功，证明了GSK的判断是对的。而在恒瑞、和黄、恩沐等更多中国药企身上的下注结果，将很大程度决定着，这家制药巨头在2031年及其之后，能否完成年度营收400亿英镑的超越。 参考资料： 1.Ris-Rez reduced risk of death by 54% versus topotecan in patients with relapsed small cell lung cancer in China 2.FierceBiotech，GSK aiming to ‘balance West and East’ after Hansoh deal success showed China’s potential 3.医药魔方，改变全球小细胞肺癌治疗方向！国产B7-H3 ADC冲刺FIC/BIC 4.深蓝观，GSK的肿瘤翻身仗，底牌在中国吗 5.同写意，GSK上强度 6.同写意，转型中的GSK：太好了，是中国创新药 7.药创新，GSK任命CEO候选人，预计2031年营收超400亿英镑