Pembrolizumab

First patient safely dosed at Calvary Mater Newcastle Hospital in Australia, marking a significant milestone for ImmutepGlobal Phase III with efti will enrol approximately 756 patients at more than 150 clinical sitesTrial results will inform potential marketing approval application in non-small cell lung cancer, one of the largest indications in oncology SYDNEY, AUSTRALIA, March 25, 2025 (GLOBE NEWSWIRE) -- Immutep Limited (ASX: IMM; NASDAQ: IMMP) (“Immutep” or “the Company”), a late-stage immunotherapy company targeting cancer and autoimmune diseases, today announces the first patient has been successfully dosed in the Company’s pivotal TACTI-004 Phase III trial. TACTI-004 will evaluate Immutep’s eftilagimod alfa, a first-in-class MHC Class II agonist, in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy KEYTRUDA® (pembrolizumab) and chemotherapy as first line treatment for patients with advanced or metastatic non-small cell lung cancer (1L NSCLC). Dr. Ina Nordman, who treated the first patient at Calvary Mater Newcastle Hospital in Australia, stated, “We are very excited to participate in this important Phase III trial. Despite advancements in the treatment landscape for non-small cell lung cancer, there remains a high unmet need for new approaches that can safely extend patients’ lives. The anti-cancer immune response driven by efti’s unique mechanism of action as an MHC Class II agonist in combination with KEYTRUDA has led to strong efficacy across all PD-L1 levels with favourable safety in multiple lung cancer trials. We hope to see this study confirm the promise of this novel combination to provide patients with a powerful new treatment option.” Immutep CEO, Marc Voigt, said, “Dosing the first patient in our pivotal Phase III trial ranks among the most significant milestones in the Company’s history. We are excited about the potential of the TACTI-004 study to deliver a new standard-of-care therapy for patients with metastatic or advanced non-small cell lung cancer that includes efti in combination with KEYTRUDA. If successful, the study will result in a clinically meaningful and statistically improved survival benefit and thus could potentially be practice changing.” Immutep CSO, Frédéric Triebel, M.D., Ph.D, commented, “As a result of all global regulatory interactions to date including previous discussion with US FDA under Project Optimus and tolerability issues at 90 mg1, we are moving forward with 30 mg subcutaneous efti dosing used in previous studies. The ability of 30 mg efti in combination with KEYTRUDA to activate the immune system and fight non-small cell lung cancer regardless of PD-L1 expression has been demonstrated across multiple clinical trials.2 Importantly, this novel approach has an excellent safety profile while delivering strong efficacy that compares favourably to standard-of-care therapies, including high rates of durable responses and compelling progression-free survival and overall survival.” Recruitment in TACTI-004 is underway at a growing number of activated clinical sites and countries with approvals from all regulatory authorities continues to expand including Australia, Austria, Belgium, Bulgaria, Canada, Germany, Greece, Hungary, India, Ireland, Italy, Latvia, Lithuania, Portugal, Spain, and the United Kingdom. Further regulatory clearances in three additional countries are expected shortly, with the remaining countries anticipated in the weeks and months ahead. Lung cancer is the leading cause of death among all cancer types and the incidence is set to increase to approximately 3 million cases worldwide by 2030.3 NSCLC is the most common type of lung cancer representing ~80-85% of all diagnoses.4 The condition is often diagnosed at a late stage, and less than 30% of patients are alive five years after diagnosis.5,6 There remains a high unmet need for additional treatment options for people living with NSCLC.KEYTRUDA® is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA. About TACTI-004TACTI-004 (Two ACTive Immunotherapies) is a randomised, double-blind, controlled Phase III study evaluating eftilagimod alfa (efti), a first-in-class MHC Class II agonist, in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy KEYTRUDA® (pembrolizumab) and chemotherapy as first line therapy for patients with advanced or metastatic non-small cell lung cancer with no EGFR, ALK or ROS1 genomic tumour aberrations. The global trial will enrol approximately 756 patients regardless of PD-L1 expression and with non-squamous or squamous tumours at over 150 clinical sites in over 25 countries. Patients will be randomised 1:1 to receive either efti in combination with pembrolizumab and chemotherapy in the treatment arm or pembrolizumab in combination with chemotherapy and placebo in the control arm. The study’s dual primary endpoints are progression-free survival and overall survival. About ImmutepImmutep is a late-stage biotechnology company developing novel immunotherapies for cancer and autoimmune disease. The Company is a pioneer in the understanding and advancement of therapeutics related to Lymphocyte Activation Gene-3 (LAG-3), and its diversified product portfolio harnesses LAG-3’s ability to stimulate or suppress the immune response. Immutep is dedicated to leveraging its expertise to bring innovative treatment options to patients in need and to maximise value for shareholders. For more information, please visit www.immutep.com. 1. Patients randomised 1:1 in AIPAC-003 Phase II to receive 30mg or 90mg dosing of eft in combination with paclitaxel to determine the optimal biological dose consistent with the FDA’s Project Optimus initiative.2. Immutep’s Efti in Combination with KEYTRUDA® Generates Excellent Overall Survival Benefit in Patients with Metastatic Non-Small Cell Lung Cancer,October 2023, and Immutep’s Efti Shows Excellent Survival Data from INSIGHT-003 Trial in Non-Small Cell Lung Cancer, November 20243. International Agency for Research on Cancer – World Health Organization. Rates of trachea, bronchus and lung cancer.4. Zappa C & Mousa Non-small cell lung cancer: current treatment and future advances, Transl Lung Cancer Res. 2016 Jun; 5(3): 288–300.5. Polanco D et al. Prognostic value of symptoms at lung cancer diagnosis: a three-year observational study. J Thorac Dis 2021;13:1485–1494. 6. National Cancer Institute Surveillance, Epidemiology, and End Results (SEER) - https://seer.cancer.gov/statfacts/html/lungb.html Australian Investors/Media:Catherine Strong, Sodali & Co.+61 (0)406 759 268; catherine.strong@sodali.com U.S. Media:Chris Basta, VP, Investor Relations and Corporate Communications+1 (631) 318 4000; chris.basta@immutep.com

得mRNA者得天下。 今日，“AI+创新药”第一股云顶新耀在自研AI驱动mRNA肿瘤领域，又迎来了更重磅的里程碑事件，其通用型的现货肿瘤治疗性疫苗EVM14注射液的新药临床试验申请(IND)获美国食品药品监督监督管理局(FDA)的批准。 EVM14不仅是云顶新耀首个获得FDA IND批准的自主研发新药和首款进入国际临床阶段的mRNA肿瘤治疗性疫苗，也是国产首款获得FDA IND批准的通用型的现货mRNA肿瘤疫苗。 此次事件不仅标志着中国首款现货型mRNA肿瘤疫苗迈入国际临床阶段，更在全球mRNA技术竞赛中投下一枚深水炸弹，掀起了新一轮关于癌症治疗未来的想象。 01 通用型现货mRNA肿瘤疫苗获批临床 或实现“长期无癌生存” 时间回溯至2021年，当全球仍深陷新冠阴霾时，云顶新耀悄然启动mRNA技术布局。彼时，Moderna与BioNTech凭借新冠疫苗一战成名，而中国药企在这一领域尚处起步阶段。 2022年，云顶新药mRNA新冠疫苗PTX-COVID19-B的II期临床数据首次展现媲美国际巨头的实力，成为技术验证的关键转折点。正是这一突破，为云顶新耀转向更复杂的肿瘤疫苗研发埋下伏笔。 如今，全球mRNA战场已悄然转移。Moderna的V940黑色素瘤疫苗预计2027年上市，BioNTech的个体化新抗原疫苗BNT122在胰腺癌中初见曙光。 而中国药企的突围，则以云顶新耀的EVM14为标志——这款靶向多种肿瘤相关抗原的“现货型”疫苗，不仅在小鼠实验中展现出抑制肿瘤复发的能力，更通过与PD-1抑制剂的联用实现疗效倍增，直击当前癌症治疗中免疫记忆缺失的痛点。 其临床前数据显示，治疗后的小鼠即便再次接触肿瘤细胞亦无生长迹象，有望实现“长期无癌生存”。 国际舞台上，mRNA肿瘤疫苗的竞争格局尚未固化。Moderna、BioNTech虽领跑，但其产品多聚焦于个体化疫苗，需针对患者肿瘤突变特征定制，周期长。 此外，个体化疫苗的高成本与医保控费存在冲突。Moderna的V940若定价过高，可能重蹈CAR-T疗法“叫好不叫座”的覆辙，而云顶新耀的降本策略或将打开下沉市场。  具体来看云顶新耀选择的“通用型”路径，其瞄准广泛存在的肿瘤相关抗原，可批量生产、快速覆盖多癌种，理论上能将成本压低至单抗药物水平。这一差异化策略，恰与全球肿瘤疫苗研发的“降本普惠”趋势不谋而合。 而国内外市场的特殊性也在为这场竞赛增添变数。美国FDA的“突破性疗法”认定加速审评；中国医保目录对创新药的纳入比例提升至70%。依托政策红利，云顶新耀的IgA肾病药物耐赋康纳入医保后，单月销售额即达1.67亿元，为其肿瘤疫苗管线提供了现金流支撑。 如今，云顶新耀正在研发多个肿瘤及自免相关的mRNA治疗药物，其中三款核心管线产品，即通用型的现货肿瘤治疗性疫苗 EVM14、已进入临床阶段的个性化肿瘤治疗性疫苗EVM16、自体生成CAR-T产品，已成为其在肿瘤及自身免疫疾病领域创新战略的三大核心支点。 02 港股18A黑马的研发布局 据Precedence Research发布的报告，2024 年全球mRNA治疗市场规模超过196.8亿美元，预计到2034年将达到426.4亿美元。尽管曙光初现，mRNA肿瘤疫苗的商业化之路仍布满荆棘。 技术层面，如何平衡通用型疫苗的广谱性与特异性？临床应用中，免疫记忆的持久性仍需人体数据验证。市场端，患者教育、监管合规与成本控制的三重考验接踵而至。云顶新耀的EVM14虽跻身全球第一梯队，但距离真正惠及患者，还需跨越III期临床与上市审批的漫漫长路。 从云顶新耀的国际化布局来看，可以看到这家港股18A黑马的“技术-生产-商业”全链条能力的系统化构建。 尤其是在浙江嘉善投资7000万元建设的GMP生产基地，目前已具备临床级mRNA疫苗产能，其模块化设计可快速切换个性化与通用型疫苗生产，破解了行业长期面临的规模化难题。 这种“本土研发+全球申报”的模式，使得EVM14在中美双报中抢占先机，而伊曲莫德等引进产品的本地化生产，则为mRNA管线提供了商业化反哺的现金流。 此外，其更深层的战略意图，在于通过技术输出重构全球合作生态。 当罗永庆宣布“双轮驱动战略进入新阶段”时，云顶新耀已悄然完成从license-in（授权引进）到license-out（技术输出）的身份转换。其拥有的全部知识产权和全球权益，不仅可能催生跨国药企的BD合作，更将中国创新药企推向了技术标准制定的前沿。 03 新药研发的“第二引擎” 云顶新耀的崛起，与AI密不可分。传统药物研发依赖经验与试错，而该公司通过AI算法重构了mRNA序列设计、递送系统优化的流程。 前段时间（3月6日），云顶新耀宣布，其通过AI辅助研发的新型mRNA个性化肿瘤治疗性疫苗EVM16已在北大肿瘤医院顺利完成首例患者给药。当日该公司股价顺势涨至超过60港元，创下近三年半新高，并成功突破2020年IPO发行价，被业内称为“AI+创新药”第一股。 据了解，EVM16是AI算法驱动识别肿瘤新抗原的新型mRNA疫苗，根据每位患者特有的肿瘤细胞突变，使用自研的“妙算”肿瘤新抗原AI算法，识别出具有较高免疫原性的肿瘤新抗原，并设计出编码数十种肿瘤新抗原的mRNA治疗性疫苗。 这一专有的“妙算”系统在某些新抗原预测性能上已超越国际顶尖机构，将18个月的研发周期压缩至数月。这种“数据+AI驱动”的模式，不仅加速了EVM14的诞生，更可能重塑整个行业的竞争规则。 如今AI赋能新药研发已成为重要趋势，尤其在mRNA疫苗领域，AI正逐渐成为推动提升研发效率和精准度的关键力量，受到生物制药行业的高度重视。 国际巨头同样嗅到危机。就在云顶新耀宣布EVM16顺利完成首例患者给药的同一时期，远在大洋彼岸的美国疫苗厂商Moderna在投资者会议上也强调了AI对肿瘤疫苗研发的赋能，而云顶新耀的实践则证明，中国药企已从技术跟随者跃升为并行者。 将镜头拉向全球，mRNA肿瘤疫苗的竞争已形成“三极格局”。Moderna的V940联合Keytruda将黑色素瘤复发风险降低49%，计划2027年上市；BioNTech的个体化疫苗BNT122在胰腺癌治疗中实现86%的复发风险降幅。 而云顶新耀的EVM14则以通用型设计填补了“现货疫苗”赛道的空白，其与PD-1联用在小鼠模型中展现的协同效应，或将改写晚期实体瘤治疗范式。 值得一提的是，这三家企业不约而同地将AI深度融入研发：Moderna利用深度学习优化抗原设计，BioNTech搭建自动化生产平台，而云顶新耀的“妙算”系统甚至在新抗原预测准确率上超越了纪念斯隆凯特琳癌症中心的算法。 总结： 获批FDA临床试验，于云顶新耀而言既是里程碑，更是新起点。EVM14的Ⅰ期临床需要验证其安全性，Ⅱ期需在复杂人体环境中重现动物实验的数据，而商业化落地更需平衡成本与疗效。但无论如何，这家曾以引进策略立足的药企，已用EVM14证明：中国创新药企不仅能参与全球技术竞赛，更有能力定义赛道的方向。  喜欢我们文章的朋友点个“在看”和“赞”吧，不然微信推送规则改变，有可能每天都会错过我们哦~ 免责声明 “汇聚南药”公众号所转载文章来源于其他公众号平台，主要目的在于分享行业相关知识，传递当前最新资讯。图片、文章版权均属于原作者所有，如有侵权，请在留言栏及时告知，我们会在24小时内删除相关信息。 信息来源：新康界 往期推荐 本平台不对转载文章的观点负责，文章所包含内容的准确性、可靠性或完整性提供任何明示暗示的保证。