Rapcabtagene autoleucel

最近一段时间，大型制药公司围绕体内CAR-T（in vivo CAR-T）的并购交易可谓风起云涌。礼来一年内连吞两家公司，吉利德、艾伯维、百时美施贵宝、阿斯利康纷纷下场，动辄几亿乃至数十亿美元的收购案接连不断。在这场被行业称为“体内CAR-T frenzy”的军备竞赛中，诺华却显得格外安静。 “我们仍在持续评估体内CAR-T领域，但目前没有正在推进中的交易。”诺华CEO瓦斯·纳拉西姆汉向行业媒体Fierce Biotech明确表示。这句话，既是对外界猜测的回应，也是诺华在当前生物技术寒冬与专利悬崖夹击下，所展现出的独特战略定力。 压力并非不存在 诺华并不缺乏补充管线的紧迫感。2026年第一季度，公司净销售额同比下滑5%，直接原因是三款重磅药物——心衰药Entresto（诺欣妥）、白血病药物Tasigna（达希纳）和血小板减少症疗法Promacta（艾曲泊帕）, 同时遭遇仿制药冲击，专利悬崖的“三重打击”正在侵蚀现金流。 按理说，这种压力足以驱动任何一家药企投身并购热潮。诺华也确实在第一季度完成两笔交易：以20亿美元整体收购过敏生物技术公司Excellergy，获得潜在接替Xolair（茁乐）的下一代产品；另支付20亿美元首付款引进Synnovation Therapeutics的泛突变选择性PI3Kα抑制剂，用以捍卫其乳腺癌专营权，抵御来自礼来等对手的竞争。 但这两笔交易恰恰暴露了诺华的真实偏好——20亿美元以下首付款、早期至中期分子、核心治疗领域——而不是冲着体内CAR-T的风口而去。 一次“试水”，不是“押注” 诺华并非对体内CAR-T无动于衷。2024年，该公司与Vyriad达成合作，利用后者的活性靶向慢病毒载体平台进行技术探索。但这种“轻触式”合作，与礼来、艾伯维们的全资收购形成鲜明对比。 在纳拉西姆汉看来，诺华的并购策略“过去几年一直保持一致”：优先收购首付款20亿美元以下的公司，以此在核心治疗领域构建多个“射门得分”的机会；偶尔才会像收购Avidity（约120亿美元）那样做一笔大交易，但前提是“极具吸引力的机会”。 体内CAR-T目前显然没有进入这个“极具吸引力”的名单。理由也很直接：多数体内CAR-T资产仍处于临床前或研究者发起的早期试验阶段，递送效率、脱靶编辑风险、工艺可重复性等关键技术瓶颈尚未突破。对于一家强调“早期至中期分子”但依然希望看到一定临床验证的药企而言，现在全资收购一家纯平台型公司，风险与估值并不匹配。 自己的“船”更重要 诺华之所以能够“不跟风”，也因为它手里已经有一艘属于自己的细胞治疗大船。CEO重点提及了rapcabtagene autoleucel，是一款靶向CD19的自体型CAR-T疗法，目前正处于2期临床阶段，适应症不是肿瘤，而是狼疮性肾炎和系统性硬化症。该药物计划于2028年提交上市申请。如果成功，它将把CAR-T从肿瘤治疗扩展到自体免疫疾病这一更广阔的市场，其战略价值不亚于任何一项体内递送技术。 换句话说，诺华并不是在放弃细胞治疗，而是在用自己的节奏、自己的资产结构，去对冲下一代技术的不确定性。体内CAR-T如果真能成熟，诺华可以通过Vyriad合作快速跟进；如果技术路径遇阻，它也不会背上高估值收购的包袱。 窗口期还有多长？ 分析师们正在计算：礼来、吉利德、艾伯维等已经把市面上较成熟的体内CAR-T标的扫了一遍，剩下的独立公司如Umoja Biopharma等，要么已有大药企合作羁绊，要么估值被推得过高。这意味着，诺华“持续评估”的窗口期可能不会太长。 纳拉西姆汉的表态留下了足够的弹性空间：“我们希望继续保持健康的收购策略，确保产品组合有足够储备以实现长期增长。”翻译成白话就是：如果体内CAR-T领域出现像Avidity那样“非常有吸引力”的机会，诺华不会犹豫；但在那之前，它更愿意把子弹留给那些已经在自己核心治疗领域内、处于早期到中期、且首付款不高于20亿美元的“稳健型”目标。 结语 在这场数十亿美元的体内CAR-T狂欢中，诺华选择坐在观众席的第一排，而不是冲上舞台。它合作、观察、评估，但不急于出价。这种“纪律性”可能让它错过一个爆发式风口，也可能帮它避开一个尚不确定的技术泡沫。真正的赢家，往往不是跑得最快的，而是知道什么时候该跑、什么时候该停的那一个。至少目前，诺华认为自己知道。

\"If we see something that\'s compelling, we\'ll certainly take a look at it,” Novartis CEO Vas Narasimhan told Fierce.\n Novartis is “continuing to evaluate” the in vivo CAR-T space, although the pharma’s CEO has told Fierce Biotech that no deals are currently in the works.In vivo CAR-Ts—which use gene editing to generate CAR-T cells within a patient’s body—have been a key component in the frenzy of Big Pharma dealmaking this spring. Eli Lilly bought two in vivo companies so far this year, leading industry analysts to question how many potential acquisition targets are still out there.Novartis dipped a toe into the space in 2024 when the company leveraged Vyriad\'s active targeting lentiviral vector platform.When asked by Fierce Biotech on an earnings call this morning whether the Swiss pharma is considering buying its own in vivo biotech, Novartis CEO Vas Narasimhan said the company is “very excited about our own immune reset portfolio.”Narasimhan pointed to rapcabtagene autoleucel, a CD19-targeted autologous CAR-T therapy, which is in phase 2 studies for blood cancers such as B-cell lymphoma, as well as for autoimmune indications such as lupus nephritis and systemic sclerosis. The company is aiming to submit the CAR-T to regulators for lupus nephritis in 2028.“Very much our focus is hopefully bringing that medicine to market in the coming years and having a strong launch,” Narasimhan said.Beyond rapcabtagene autoleucel, the company is “following that on with a portfolio of bispecifics and then potentially future CAR-T technologies like in vivo CAR-T,” the CEO told Fierce.“We\'ll continue to evaluate that space,” he continued. While Novartis has “no specific plans for any acquisitions” in this area, Narasimhan said his team is “very aware of what\'s going on.” “And, of course, if we see something that\'s compelling, we\'ll certainly take a look at it,” he added. It makes sense for Novartis to seek further sources to restock its pipeline—the company blamed the arrival of generic competition to its heart drug Entresto, leukemia med Tasigna and thrombocytopenia therapy Promacta for a 5% decline in net sales in the first quarter of the year. It explains why Novartis was happy to join in the flurry of Big Pharma dealmaking in the first quarter, penning a $2 billion deal for allergy biotech Excellergy and its potential successor to Xolair. Novartis also paid $2 billion upfront for a pan-mutant‑selective PI3Kα inhibitor from Synnovation Therapeutics that could help defend the pharma’s breast cancer franchise from challengers like Lilly.When asked by Fierce whether Novartis would be buying more biotechs this year to help shore up its pipeline from generic erosion, Narasimhan said Novartis is “going to stay consistent with our M&A strategy.”“The last few years have focused on acquiring companies typically in the sub-$2 billion upfront range, which build out our portfolio and then allow us to have multiple shots on goal in our key therapeutic areas,” he told Fierce.“I think Excellergy and Synnovation both fit that profile,” he said. “These are early- to mid-stage molecules in our core therapeutic areas, which we think can bolster our long-term growth profile.”“We do, on occasion, do larger deals like Avidity when there\'s a very compelling opportunity,” Narasimhan continued. “But I think overall, our strategies remain the same. And I think over the last few years, we\'ve been one of the more active dealmakers in the space.” “We want to continue to have a healthy acquisition approach to make sure we have enough in our portfolio for long-term growth,” the CEO said.