Cemiplimab-RWLC

在全球生物科技产业加速重构的当下，中国创新药正以锐不可当之势崛起为全球研发版图的关键力量。2025年一季度，国内药企共完成30余起创新药BD出海交易，中国创新药已然成为全球生物科技产业发展的源动力之一。在一众药企中，和铂医药无疑是拥有尖端研发实力的典型代表，截至目前其已完成17次BD出海交易，成为中国BD出海交易最多的药企，堪称“BD之王”。6月23日，和铂医药BD交易数量再度“喜加一”，宣布与大塚制药达成全球战略合作，共同推进BCMAxCD3双特异性T细胞衔接器（TCE）开发。根据协议条款，大塚制药将获得在全球范围内（不包括大中华区，即中国大陆、中国香港、中国澳门和中国台湾）开发、生产和商业化HBM7020（一款BCMAxCD3双特异性T细胞衔接器）的独家许可。和铂医药将获得总计4700万美元的首付款和近期里程碑付款。此外，在达成特定研发和商业里程碑后，和铂医药还有权获得高达6.23亿美元的额外付款，以及基于未来产品净销售额的分级特许权使用费。在此次战略合作的基础上，双方将探讨未来在T细胞衔接器领域进一步合作的机会。然而，重磅BD交易并非和铂医药唯一的好消息。今年3月底公布的2024年财报中，和铂医药连续第二年实现连续盈利，商业模式已经得到实际验证；在5月中旬国际权威指数编制公司MSCI公布的最新季度调整结果中，和铂医药正式被纳入MSCI全球小盘股指数，将吸引全球被动资金配置，提升股票流动性，该调整于2025年5月30日收盘后生效。投资圣经《穷查理宝典》中，查理・芒格曾提出了著名的“Lollapalooza效应”（多重因素叠加的爆发式增长）思维模型。所谓“Lollapalooza效应”指的是任何经济体的爆发都不是由单一因素驱动的，而是几大长期核心要素叠加后所产生的效应，也就是“好上加好”。在和铂医药身上，“Lollapalooza效应”的轮廓已经若隐若现。若以查理・芒格的“Lollapalooza效应”审视，和铂医药的“三重复合优势”（技术实力、战略聚焦、生态体系）正构筑起难以复制的竞争壁垒。01技术壁垒：稀缺性全人源抗体平台HBM7020的成功BD交易，再次向市场验证了和铂医药强大的研发实力。以技术角度切入，HBM7020是一款利用全人源HBICE®双抗技术及Harbour Mice®平台开发的BCMAxCD3双特异性抗体，将目标细胞与T细胞交联，从而有效激活T细胞并杀伤目标细胞。HBM7020最独特设计在于非对称“2+1”形式，即通过设计两个抗BCMA结合位点，实现了对目标细胞的精准靶向，而单价优化的CD3活性则有效降低了细胞因子释放综合征（CRS）的发生风险。凭借这些创新机制，HBM7020展现出强大的细胞杀伤能力，在免疫学和肿瘤领域均具有广阔的应用前景。纵观全球创新药BD趋势，时代的脚步已然走到TCE双抗爆发的前夜。自2022年4月，和铂医药以3.5亿美元的价格将HBM7022（CLDN18.2/CD3双靶点TCE双抗）授权给阿斯利康，便开启了中国TCE药物出海的先河。随后几年中，海外药企对于TCE平台的关注度不断加码，这一赛道正在复制当年ADC的发展趋势，HBM7020的授出无疑再次将TCE双抗的热度推向高潮。通过本次BD交易，市场再次见证了和铂医药Harbour Mice®的稀缺平台价值。Harbour Mice®是全球首个能够产生全人源重链抗体（HCAb）的转基因小鼠技术平台，能够开发仅有传统IgG抗体一半大小的新一代全人源重链抗体，由于HCAb的结构中没有常规抗体的轻链结构，大大降低了轻链错配或异源二聚化等常见问题。由Harbour Mice®所研发的抗体具有与IgG抗体类似的代谢特征和效应功能，而且不需要额外的人源化和其他抗体工程改造。正是基于如此独特的技术优势，才造就了和铂医药“BD之王”的身份。图：Harbour Mice®平台，来源：公司公告“Lollapalooza效应”可谓知易行难。它追求的是关键因素的简洁性，但就像爱因斯坦说的：事情应该力求简单，但不能过于简单。就医药行业而言，投资者皆知研发能力是企业核心竞争力所在，但究竟应该如何判断药企的研发竞争力呢？这对于非医药出身的投资者而言，又是一道难题。但这个问题在和铂医药身上却并不存在，多年持续的BD交易已经验证了公司强大的研发实力，毕竟大型海外药企是不会轻易将白花花的美金扔到水中的。基于此，长期持续兑现的BD交易，验证了和铂医药强劲的研发实力。02战略壁垒：创新药产业链的“价值截取者”曾几何时，成为大型制药企业是中国创新药企们的夙愿，为了能够快速撑起营收，因此出现很多企业想办法拼凑商业化管线的情况。然而，这条借鉴了美国MNC发展轨迹的道路，最终却成功者寥寥。其中的原因很简单，那就是对于资源有限的中国创新药企业而言，无论是人才积累，生产链条，还是商业化团队，实则都存在比较大的缺口。如此情况下，药企想要面面俱到，那么最终很可能只会换来一场徒劳。《晏子春秋》有云：不吃全鱼。这个典故用在中国创新药身上同样再合适不过，与其想着掌控整个药品链条，倒不如集中资源到最核心的环节。对于企业长期发展方向，和铂医药不走寻常路，虽然其所开发的早期管线潜力十足，基本都是First in class，但公司管理层却并没有独自开发，而是选择与海外大型药企BD合作的方式兑现预期。和铂医药以 Harbour Mice® 平台为支点，将临床开发、生产及商业化环节让渡给全球合作伙伴，实现“低投入、高回报”的价值分配。正是基于聚焦优势环节的专注，才是和铂医药实现连续BD合作的底层逻辑。这条发展路径还曾经孕育出大名鼎鼎的美股明星股再生元制药。再生元制药专注于早期研发，依托于VelociSuite®技术平台，持续孵化高质量的潜力分子，如Dupixent、Praluent、Libtayo等。虽然管线竞争力十足，但再生元制药并没有选择独自开发这些潜力分子，而是与赛诺菲合作的方式进行开发。随着双方合作的不断迈进，再生元制药预期不断兑现，股价持续攀升，成为过去十年全球现象级药企。图：再生元制药股价走势，来源：雪球以企业发展路径而论，和铂医药走得正是再生元制药曾经的路。Harbour Mice®平台有着与VelociSuite®平台相似的稀缺价值，通过不断向海外巨头企业输出稳定的前沿分子，从而逐渐提升自身的长期竞争力。拥有前沿研发实力的企业可能很多，但能够长期坚守护城河的企业却凤毛麟角，这更凸显出和铂医药的可贵之处。03生态壁垒：全球创新生态共建者和铂医药 BD 的底层逻辑是“筑生态”—— 通过持续输出高质量管线，实现与全球顶级药企之间的深度绑定。研发实力与战略聚焦，这是投资者能够直接捕获的价值。但要想成为一家伟大的企业，光靠这两点是不够的，管理层还必须拥有长期的定力，这亦是影响企业发展的隐藏线索。“不吃全鱼”的做法，注定了和铂医药走的是一条“长期主义”的道路。依托于Harbour Mice®平台，和铂医药有能力产生一系列颇具潜力的优质管线，可BD交易的价值并不会立马全部兑现，首付款仅是其中的一部分，更多的款项依然要取决于后续管线临床进度所兑现的里程碑款项。因此，唯有专注长期价值，把管线研发当成终生事业去做，将海外药企当成长期合作伙伴，才有可能最终取得好的结果。此外，BD交易还有另一个好处，那就是能够与海外药企之间建立紧密的联系。即使BD额度前期可能并不高，但却能让企业感知到和铂医药超强的研发能力，从而促成后续更深入的合作。如今年3月与和铂医药达成46亿美元惊天交易的阿斯利康，正是通过前两次的“试水”合作而体验到了和铂医药的研发实力，然后才选择以“分子授权+股权投资”形式深度绑定的。图：和铂医药与阿斯利康合作一览，来源：公司公告这其实亦是一种品牌效应的体现。除阿斯利康外，和铂医药这些年还与辉瑞、艾伯维、莫德纳等全球大药企展开合作。随着授权管线进度的不断推进，这些合作药企中极有可能诞生第二个“阿斯利康”。能够做好BD生意的药企，注定是需要一颗“长期主义”之心的，只有与生态伙伴实现彼此共赢，才能让这个模式长期延续。04Lollapalooza时刻：技术势能转化为产业动能BD合作不断落地，创新药政策窗口全面开启，投资者认可度持续提升，和铂医药在2025年迎来了属于它的“Lollapalooza时刻”。年初至今，和铂医药年内涨幅超过380%，是中国股价涨幅最高的药企之一，这正是“Lollapalooza效应”的具象化体现。如今的和铂医药，正站在“三重爆发因子”的交汇点。技术壁垒、战略壁垒、生态壁垒，三个简单要素相叠加，恰是芒格所言的“Lollapalooza 效应”—— 不是单一优势的线性增长，而是多重变量在临界点后的指数级爆发，这是对于管理层长期主义的最佳褒奖。当多数药企还在纠结“做自研还是做 BD”时，和铂医药早已用 17 次出海交易证明：在全球化分工深度细化的今天，成为“全球创新药研发体系的关键节点”，或许远比“成为全产业链巨头”更具现实可能性。 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：医曜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。

Approval based on pivotal results showing improvements in sustained disease remission and reductions in itch and oral corticosteroid use compared to placebo in adults with BP BP is a chronic, debilitating and relapsing rare skin disease affecting approximately 27,000 adults in the U.S. whose disease is uncontrolled by systemic corticosteroids Dupixent is now approved in the U.S. to treat eight distinct diseases with underlying type 2 inflammation, including diseases of the skin, gut, and respiratory system that affect a broad range of patients, from infants to elderly people TARRYTOWN, NY, USA and PARIS, France I June 20, 2025 I Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the U.S. Food and Drug Administration (FDA) has approved Dupixent ® (dupilumab) for the treatment of adult patients with bullous pemphigoid (BP). BP primarily affects elderly patients, and is characterized by intense itch, painful blisters and lesions, as well as reddening of the skin. It can be chronic and relapsing with underlying type 2 inflammation. The blisters and rash can form over much of the body and cause the skin to bleed and break down, resulting in patients being more prone to infection and affecting their daily functioning. Available treatment options are limited and can add to overall disease burden by suppressing a patient’s immune system. “People affected by bullous pemphigoid endure unrelenting itch and painful blisters that can damage the skin. Until now, these primarily elderly patients have had limited therapeutic options available, with potential side effects that have often added to their burden,” Patrick Dunn, Executive Director at the International Pemphigus and Pemphigoid Foundation. “The approval of Dupixent for bullous pemphigoid brings a novel treatment approach to patients and their caregivers, and we are grateful for the tireless efforts of the scientific community who helped us reach this critical milestone.” “Today’s approval extends the remarkable ability of Dupixent to transform treatment paradigms for people living with a variety of diseases with underlying type 2 inflammation, from common conditions like asthma and atopic dermatitis, to rarer ones such as eosinophilic esophagitis and prurigo nodularis, and now including bullous pemphigoid,” said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer at Regeneron, and a principal inventor of Dupixent. “Dupixent has shown the potential to improve the most challenging effects of bullous pemphigoid, while helping some patients achieve sustained disease remission and decreased oral corticosteroid use. Additionally, this approval further reinforces the demonstrated safety profile of Dupixent in a broad age range of patients, from infants to elderly people, and across dermatological, respiratory and gastrointestinal diseases.” The FDA approval is based on data from the pivotal ADEPT Phase 2/3 trial that evaluated the efficacy and safety of Dupixent compared to placebo in adults with moderate-to-severe BP. Patients were randomized to receive Dupixent 300 mg (n=53) or placebo (n=53) added to standard-of-care oral corticosteroids (OCS). During treatment, all patients underwent a protocol-defined OCS tapering regimen if control of disease activity was maintained. During the FDA review, the analyses were updated; the FDA-approved results at 36 weeks in the label for Dupixent compared to placebo are: In this elderly population, the most common adverse events (≥2%) more frequently observed in patients on Dupixent compared to placebo were arthralgia, conjunctivitis, blurred vision, herpes viral infections and keratitis. Additionally, 1 case of acute generalized exanthematous pustulosis was reported in 1 patient treated with Dupixent and 0 patients treated with placebo. “Until now, treating bullous pemphigoid was very challenging for elderly patients struggling with the debilitating impact of blisters and lesions, and potentially co-morbid conditions,” said Alyssa Johnsen, M.D., Ph.D., Global Therapeutic Area Head, Immunology and Oncology Development at Sanofi. “By addressing two central drivers of the underlying type 2 inflammation that contributes to bullous pemphigoid, Dupixent is the first targeted medicine to allow patients the potential to achieve sustained remission and reduce itch. This approval in the U.S. is important for the thousands of patients living with bullous pemphigoid, and we look forward to working with regulators around the world to bring this innovative medicine to more patients in need.” The FDA evaluated Dupixent under Priority Review, which is reserved for medicines that represent potentially significant improvements in efficacy or safety in treating serious conditions. Dupixent was previously granted Orphan Drug Designation by the FDA for BP, which applies to investigational medicines intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Additional regulatory applications are also under review around the world, including in the EU, Japan and China. About the Dupixent BP Pivotal Trial ADEPT was a randomized, Phase 2/3, double-blind, placebo-controlled trial evaluating the efficacy and safety of Dupixent in 106 adults with moderate-to-severe BP for a 52-week treatment period. After randomization, patients received Dupixent or placebo every two weeks after an initial loading dose, along with OCS treatment. During treatment, OCS taper was initiated after patients experienced two weeks of sustained control of disease activity. OCS tapering could start between four to six weeks after randomization and was continued if disease control was maintained, with the intent of completion by 16 weeks. After OCS tapering, patients were only treated with Dupixent or placebo for at least 20 weeks (rescue treatment could be used if required). The primary endpoint evaluated the proportion of patients achieving sustained disease remission at 36 weeks. Sustained disease remission was defined as complete clinical remission with completion of OCS taper by 16 weeks without relapse after completion of the OCS taper and no rescue therapy use during the 36-week treatment period. Relapse was defined as appearance of ≥3 new lesions a month or ≥1 large lesion or urticarial plaque (>10 cm in diameter) that did not heal within a week. Rescue therapy could include treatment with high-potency topical corticosteroids, OCS (including increase of OCS dose during the taper or re-initiation of OCS after completion of the OCS taper), or systemic non-steroidal immunosuppressive medications or immunomodulating biologics. Select secondary endpoints evaluated at 36 weeks included: About Dupixent Dupixent is an injection administered under the skin (subcutaneous injection) at different injection sites. In adults with BP, Dupixent 300 mg is administered every other week after an initial loading dose, and in combination with a tapering course of oral corticosteroids. Dupixent is intended for use under the guidance of a healthcare professional and can be given in a clinic or at home after training by a healthcare professional. Dupixent, which was invented using Regeneron’s proprietary VelocImmune ® technology, is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant. The Dupixent development program has shown significant clinical benefit and a decrease in type 2 inflammation in Phase 3 trials, establishing that IL-4 and IL-13 are two of the key and central drivers of the type 2 inflammation that plays a major role in multiple related and often co-morbid diseases. Regeneron and Sanofi are committed to helping patients in the U.S. who are prescribed Dupixent gain access to the medicine and receive the support they may need with the DUPIXENT MyWay ® program. For more information, please call 1-844-DUPIXENT (1-844-387-4936) or visit www.DUPIXENT.com . Dupixent has received regulatory approvals in more than 60 countries in one or more indications including certain patients with atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps (CRSwNP), eosinophilic esophagitis (EoE), prurigo nodularis, chronic spontaneous urticaria (CSU), chronic obstructive pulmonary disease (COPD) and BP in different age populations. More than 1,000,000 patients are being treated with Dupixent globally. 1 About Regeneron’s VelocImmune Technology Regeneron’s VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron’s co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite ® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create a substantial proportion of all original, FDA-approved fully human monoclonal antibodies. This includes Dupixent ® (dupilumab), Libtayo ® (cemiplimab-rwlc), Praluent ® (alirocumab), Kevzara ® (sarilumab), Evkeeza ® (evinacumab-dgnb), Inmazeb ® (atoltivimab, maftivimab and odesivimab-ebgn) and Veopoz ® (pozelimab-bbfg). In addition, REGEN-COV ® (casirivimab and imdevimab) had been authorized by the FDA during the COVID-19 pandemic until 2024. Dupilumab Development Program Dupilumab is being jointly developed by Regeneron and Sanofi under a global collaboration agreement. To date, dupilumab has been studied across more than 60 clinical trials involving more than 10,000 patients with various chronic diseases driven in part by type 2 inflammation. In addition to the currently approved indications, Regeneron and Sanofi are studying dupilumab in a broad range of diseases driven by type 2 inflammation or other allergic processes in Phase 3 trials, including chronic pruritus of unknown origin and lichen simplex chronicus. These potential uses of dupilumab are currently under clinical investigation, and the safety and efficacy in these conditions have not been fully evaluated by any regulatory authority. U.S. INDICATIONS DUPIXENT is a prescription medicine used: DUPIXENT is not used to relieve sudden breathing problems and will not replace an inhaled rescue medicine or to treat any other forms of hives (urticaria). Please see accompanying full Prescribing Information including Patient Information. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases. Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite, which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center ® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases. For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn , Instagram , Facebook or X . About Sanofi Sanofi is an R&D driven, AI-powered biopharma company committed to improving people’s lives and delivering compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people’s lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time. 1 Data on File SOURCE: Regeneron Pharmaceuticals