医药周报 20251221 In Vivo CAR-T 国内外进展大 医药行情回顾&分析&近期判断 > 1) 行情回顾：当周（12.15-12.19）医药生物指数环比-0.14%，跑赢创业板指数和沪深 300 指数, 在所有行业中，当周（12.15-12.19）医药涨跌幅排在第 18 位, V 型走势。周度结构上看, 蚂蚁阿 福、"搞赏经济"主题等相关标的表现较好，周五表现更为强势，CXO、小核酸、部分创新药和 AI 创 新药相对较好。医药成交总额 3898.24 亿元，沪深总成交额为 86901.56 亿元，医药成交额占比沪深 总成交额比例为 4.49%（2013 年以来成交额均值为 7.12%）。 2）原因分析：医药近期暂未有较大的产业催化，板块贝塔效应不明显，但经过一段时间消化，很多 标的进入价值区间，或潜在具备一定的反攻可能，这期间结构性热度会逐步提升。本周蚂蚁阿福带 动相关个股演绎：一类是阿里的持股相关医药公司，一类阿福可能引流的公司（渠道如部分药店、 部分消费端器械、检测服务、药品等 ) ，一类是有数据沉淀未来潜在可能对标公司。全市场 "椅赏经 济"也影射了医药中部分公司，如部分药店。周五医药表现 ...

编者按： 长期以来，我国创新药商业化面临着"进院难、支付难"的结构性困境，尤其是高值创新药，常常受限于 基本医保"保基本"的定位而难以纳入报销范围。此次政策组合拳的核心逻辑，在于通过明确的分工，厘 清了政府与市场的支付边界。 对外经济贸易大学创新与风险管理研究中心副主任龙格在接受采访时指出，《商保创新药目录》聚焦超 出基本医保保障范围，但临床价值显著的创新药，作为基本医保目录的补充。两者形成了"医保保基 本、商保接高端"的分工：基本医保覆盖基础药品，商保目录则满足多元化、高层次需求，并为创新药 提供"先商保后医保"的梯度准入路径。 具体而言，新版基本医保目录继续发挥"压舱石"作用。2025年医保目录新增114种药品，谈判/竞价成功 率高达88.19%，创下近七年新高。新增药品中，50种为1类创新药，重点覆盖肿瘤、慢性病、罕见病等 领域。调整后的目录内药品总数增至3253种，其中西药1857种、中成药1396种，极大地提升了重点领域 的用药保障水平。 与此同时，首版《商保创新药目录》则精准承接了基本医保"溢出"的高端需求。首批商保目录共纳入19 种药品，涵盖了CAR-T疗法、神经母细胞瘤和戈谢病等罕见病特效药以 ...

南方财经全媒体记者 孙诗卉 实习生 徐若萱 2025年12月7日，国家医保局与人力资源社会保障部联合发布了《国家基本医疗保险、生育保险和工伤保险药品目录（2025年）》（以下简称 2025年医保目录）及首版《商业健康保险创新药品目录》（以下简称《商保创新药目录》），两份目录均将于2026年1月1日起正式实施。 作为构建多层次医疗保障体系的里程碑事件，业内人士表示，此次"双目录"的同步发布，意味着我国医药支付体系正式进入"基本医保+商业健 康险"双轮驱动的新阶段。 长期以来，商业健康险在特药责任设计上存在"专业性不足"的痛点。资深从业者迈伦向21世纪经济报道记者指出，保险公司缺乏药物经济学专 家，自行制定目录难度极大。此次《商保创新药目录》的制定相当于为保险行业提供了一份官方背书的、高质量的"药品池"，极大地降低了产 品开发的准入门槛和信息不对称风险。 对于消费者而言，这一政策不仅夯实了基础用药的保障底线，更使得此前因价格高昂而难以企及的百万级CAR-T疗法、前沿阿尔茨海默病药物 等高值创新药，有了实质性的支付解决方案。 承接医保"溢出"创新药支付需求 长期以来，我国创新药商业化面临着"进院难、支付难"的结构性 ...

Core Insights - The 2025 National Medical Insurance Drug List has been officially released, adding 114 new drugs, including 50 innovative Class 1 drugs, with a negotiation success rate of 88%, the highest in seven years [1][3] - The first version of the commercial insurance innovative drug list has been introduced, featuring 5 CAR-T cell therapies and 19 high-priced innovative drugs, marking a significant expansion in coverage [1][5] - The adjustment reflects a strategic upgrade in China's medical insurance from "basic coverage" to "basic + high-end coverage," addressing the demand for accessible and affordable innovative drugs [1][9] Drug Additions and Market Impact - The new drug list includes 114 drugs, with 44% being Class 1 innovative drugs, and a total of 3,253 drugs now covered under the insurance [3] - Notable winners include Heng Rui Pharma, which secured 20 new drug approvals, and Eli Lilly, whose drug Tirzepatide has entered the Chinese market, benefiting 140 million diabetes patients [3][4] - The inclusion of CAR-T therapies in the commercial insurance list allows for reimbursement of high-cost treatments, which were previously inaccessible to many patients [5][7] Innovations in Treatment - The new list includes significant advancements in treatments for Alzheimer's disease, with two new drugs targeting amyloid beta accumulation, providing options for over 10 million patients in China [7] - The addition of 13 rare disease drugs fills coverage gaps in four rare disease areas, expanding the total number of rare disease drugs covered to approximately 100 [8] - The adjustment also emphasizes pediatric medications, with a separate review channel for children's formulations, indicating a positive shift in addressing children's healthcare needs [9] Strategic Implications - The 2025 adjustment signifies a shift towards a dual-track medical payment system in China, combining basic medical insurance with commercial insurance to meet both fundamental and advanced healthcare needs [9] - The rapid inclusion of innovative drugs into the insurance system reflects a decade of progress in China's pharmaceutical landscape, transitioning from rare domestic innovations to a robust pipeline of new therapies [9]

Core Viewpoint - The first version of the commercial insurance innovative drug directory in China has been released, including 19 drugs, and will be implemented starting January 1, 2026 [1][3]. Group 1: Overview of the Innovative Drug Directory - The directory aims to fill gaps in clinical medication coverage by including innovative drugs that are not reimbursed by basic medical insurance [3][4]. - The drugs included are primarily those with significant clinical value and patient benefits, exceeding the coverage of basic medical insurance [3][4]. - Notable drugs in the directory include CAR-T cell therapies and treatments for rare diseases prevalent in children [3][4]. Group 2: Market Impact and Growth - The commercial health insurance market in China has been rapidly growing, with premium income reaching 977.3 billion yuan in 2024 [4]. - The innovative drug directory is expected to enhance the efficiency of insurance coverage by reducing overlaps with basic medical insurance [4][5]. - Experts believe that the directory will create a win-win situation for pharmaceutical companies, patients, and insurance providers, encouraging innovation and market expansion [5][6]. Group 3: Policy Support and Implementation - The National Medical Insurance Administration has provided "three exclusions" to support the implementation of the directory, allowing for easier access to innovative drugs in hospitals [5][7]. - Although the drugs in the directory are not reimbursed by basic medical insurance, patients can still benefit from commercial insurance products that cover these medications [7][8]. - The directory is expected to facilitate the use of high-value drugs and expand the coverage of commercial health insurance products [7][8]. Group 4: Challenges and Recommendations - There are concerns about the sustainability and accessibility of the innovative drug directory, with suggestions for establishing a comprehensive payment control and pricing mechanism [9][10]. - Insurance companies face challenges in managing the introduction of innovative drugs due to the lack of historical claims data, which complicates cost predictions [10]. - Recommendations include enhancing risk management capabilities and integrating data to improve the overall service and coverage of innovative drugs [10].

Core Viewpoint - The first version of the commercial insurance innovative drug directory in China has been released, including 19 drugs, and will be implemented on January 1, 2026, aiming to fill gaps in clinical medication coverage and enhance patient access to innovative treatments [1][2]. Group 1: Innovative Drug Directory - The directory includes drugs that are clinically valuable and significantly benefit patients, exceeding the coverage of basic medical insurance [2]. - Notable inclusions are CAR-T cell therapies and treatments for rare diseases prevalent in children, such as neuroblastoma and Gaucher disease [2]. - The directory is designed to complement the basic medical insurance system, addressing the limitations of existing coverage [2]. Group 2: Market Impact - The commercial health insurance market in China is rapidly growing, with premiums reaching 977.3 billion yuan in 2024, nearing the total funding level of resident medical insurance [3]. - The innovative drug directory is expected to enhance the efficiency of insurance coverage by reducing overlaps with basic medical insurance [3]. - Experts believe that the directory will create a win-win situation for pharmaceutical companies, patients, and insurance providers, encouraging investment in innovative drug development [4]. Group 3: Policy Support - The National Healthcare Security Administration has introduced a "three exclusions" policy to support the implementation of the innovative drug directory, allowing for more flexible use of innovative drugs in hospitals [5][6]. - This policy aims to alleviate the burden on hospitals and improve patient access to necessary medications [6]. Group 4: Future Considerations - The directory's introduction may lead to a slight increase in insurance premiums in the short term, but long-term stability is expected [7]. - The estimated market size for innovative drug sales in China is projected to reach 162 billion yuan in 2024, with commercial health insurance covering approximately 12.4 billion yuan [8]. - There are concerns regarding the effective implementation of the directory, as it is not mandatory and lacks a clear pricing mechanism [9]. Group 5: Challenges for Insurance Companies - The introduction of innovative drugs poses challenges for insurance companies in terms of precise cost prediction due to limited historical claims data [10]. - Recommendations for insurance companies include adjusting product strategies, enhancing risk management capabilities, and integrating into the broader health ecosystem [10].

Core Viewpoint - CAR-T therapy, a cutting-edge cancer immunotherapy, offers significant potential for treating blood cancers but faces challenges due to high costs and limited accessibility [1][2][8] Group 1: CAR-T Therapy Overview - CAR-T therapy utilizes genetically modified T-cells to target and destroy cancer cells, showing promising results in treating certain blood cancers like B-cell acute lymphoblastic leukemia and lymphoma [2][5] - The therapy is highly personalized, requiring individual production for each patient, which contributes to its high cost, typically ranging from 999,000 to 1,290,000 RMB [6][9] Group 2: Market Dynamics - The global CAR-T market is projected to reach $21.8 billion by 2030, with a compound annual growth rate of 22.1% from 2024 to 2030 [6][7] - China has emerged as a significant player in the CAR-T market, with eight out of fifteen globally approved CAR-T products originating from Chinese companies [6][7] Group 3: Challenges and Innovations - High production costs and limited insurance coverage are major barriers to the widespread adoption of CAR-T therapy [9][10] - Companies are exploring innovative payment models and cost control strategies, such as partnerships with commercial health insurance and sourcing materials from domestic suppliers [10][11] Group 4: Regulatory Environment - The upcoming regulatory framework in China aims to enhance the oversight of CAR-T therapies, which may lead to improved quality and safety standards in the industry [13][15] - The introduction of new CAR-T therapies targeting solid tumors and autoimmune diseases is expected to expand the market significantly [8][15] Group 5: Future Outlook - With advancements in technology and payment systems, CAR-T therapy may transition from a last-resort option to a standard treatment for various cancers [11][16] - The potential for cost reduction through innovations like in vivo CAR-T therapy could make these treatments more accessible to a broader patient population [11][16]

Market Performance - The pharmaceutical and biotechnology index declined by 2.62% from November 3 to November 7, underperforming the Shanghai Composite Index by 2.37 percentage points, marking seven consecutive weeks of underperformance [1] - The innovative drug sector (BK1106) fell by 3.51% during the week, while the Hang Seng Healthcare Index dropped by 2.39% and the Hong Kong innovative drug ETF (513120) decreased by 3.92% [1] Industry Commentary - After a strong rally, the innovative drug industry has entered a bubble-popping phase, with a return to rational investment not necessarily being negative for the sector. However, the current market adjustment appears excessive, with leading companies like Kangfang Biotech, Zai Lab, and Kelun-Biotech experiencing declines exceeding 30% [2] - Zai Lab's recent performance has been impacted by two public announcements: disappointing clinical data for its gastric cancer drug Bemarituzumab and a Q3 report showing total revenue of $116 million, a 14% year-on-year increase, but a net loss of $35.96 million, which is a narrowing of losses compared to the previous year. Revenue growth was primarily driven by sales of "Nusinersan" and "Dingyoule," offset by a slowdown in "Zele" sales [2] Short-term Outlook - In the short term, the innovative drug industry is undergoing emotional recovery and valuation reconstruction, with stock volatility heavily influenced by clinical data and earnings guidance. Zai Lab faces short-term emotional pressure due to the termination of a key clinical trial and downward adjustments in performance expectations. However, from a medium to long-term perspective, the innovative logic of the industry remains unchanged, with globally competitive pipeline assets being the core support for company valuations [3] IPO Developments - Nanjing Haina Pharmaceutical Technology Co., Ltd. has submitted an IPO application to the Hong Kong Stock Exchange, with CICC as the sole sponsor. This follows the termination of a major asset restructuring plan in June 2023, where Haina was to be acquired by Chengdu Xian Dao [4] - Haina Pharmaceutical, established in 2001, integrates drug research and manufacturing, providing CXO services and proprietary product pipelines. The company's revenue primarily comes from CXO services, with 398 ongoing CXO projects as of mid-2025 [4] Financial Performance - For 2024, Haina Pharmaceutical projects revenue of 425 million yuan, a year-on-year increase of 3.65%, but a net profit of 53.295 million yuan, reflecting a 27% decline, indicating a situation of "increased revenue but decreased profit." In the first half of the year, both revenue and net profit saw declines of 16.97% and 25.82%, respectively, attributed to a decrease in CRO service income and a 45.8% drop in sales of proprietary drugs [5] Clinical Trial Updates - From November 3 to November 7, the National Medical Products Administration disclosed 110 new clinical trial registrations, with 33 of these being innovative drugs in Phase II or above, primarily covering oncology, immunology, cardiovascular, and psychiatric fields [6] - Four innovative drugs were approved during the week [7] Notable Approvals - The first CAR-T therapy in China, Pucalunase injection (pCAR-19B), was approved for treating pediatric acute B lymphoblastic leukemia patients aged 3 to 21. This drug was previously included in breakthrough therapy and priority review categories [8] - Pucalunase is the fifth CD19 CAR-T product approved in China, following four others from various companies [9]

Core Viewpoint - WuXi AppTec's subsidiary WuXi Biologics has submitted data for the Phase I study of Relma-cel in adult patients with active systemic lupus erythematosus (SLE) to the National Medical Products Administration (NMPA) in China, marking a significant step in the development of CAR-T therapies for autoimmune diseases [1][4] Group 1: Study Overview - The Phase I study is a single-arm, open-label, multi-center, dose-exploration trial aimed at assessing the safety of Relma-cel in SLE patients, with an initial dose set at 50×10^6 CAR+T cells [1] - The study has enrolled 12 participants as of July 2025, focusing on safety, efficacy, pharmacokinetics, and pharmacodynamics across three dose levels: 50×10^6, 75×10^6, and 100×10^6 CAR+T cells [1][4] Group 2: Patient Demographics and Baseline Characteristics - All 12 enrolled patients were female, with a median age of 27 years and a median disease history of 9.5 years [2] - The patients exhibited moderate to severe active SLE, with 100% having renal involvement and other common organ systems affected including skin (50%) and hematologic systems (50%) [2] Group 3: Efficacy Results - Among the 12 patients evaluated for 6-month efficacy, 100% achieved SRI-4 response, 50% met LLDAS criteria, and 100% were drug-free [2] - Various disease activity metrics, including SLEDAI-2K and SELENA-SLEDAI scores, showed a downward trend, indicating significant therapeutic effects [2] Group 4: Safety Results - Initial safety results indicated that 11 out of 12 patients experienced grade 1 cytokine release syndrome (CRS), while one patient experienced grade 2 immune effector cell-associated neurotoxicity syndrome (ICANS) [3] - No dose-limiting toxicities (DLT) were reported, suggesting a favorable safety profile for Relma-cel in treating moderate to severe active SLE [3][4] Group 5: Future Prospects - The ongoing study aims to gather longer follow-up data, with the potential for Relma-cel to advance to the biologics license application (BLA) stage [4] - As the first commercial CAR-T therapy approved for clinical trials in the SLE treatment space, Relma-cel demonstrates significant potential for providing breakthrough treatment options for SLE patients [4]

Core Viewpoint - WuXi AppTec's CAR-T therapy, Relma-cel, has submitted Phase I study data for treating adult patients with active systemic lupus erythematosus (SLE) to the National Medical Products Administration (NMPA) in China, indicating significant potential for this innovative treatment in a challenging therapeutic area [1][4]. Group 1: Study Overview - The Phase I study is a multicenter, open-label, dose-exploration trial designed to assess the safety and efficacy of Relma-cel in SLE patients, with doses set at 50×10^6, 75×10^6, and 100×10^6 CAR+T cells [1]. - As of July 2025, a total of 12 female patients have been enrolled, with a median age of 27 years and a median disease history of 9.5 years [2]. Group 2: Patient Characteristics - All enrolled patients exhibited moderate to severe active SLE, with a median SELENA-SLEDAI score of 10 [2]. - 100% of patients had renal involvement, and other affected systems included skin (50%), hematologic (50%), and joints (16.7%) [2]. Group 3: Efficacy Results - Among the 12 patients evaluated for 6-month efficacy, 100% achieved SRI-4 response, and 50% met LLDAS criteria, indicating promising therapeutic effects [2]. - Various disease activity scales showed a downward trend, suggesting significant efficacy of Relma-cel in managing SLE [2]. Group 4: Safety Results - Initial safety results indicated that 11 out of 12 patients experienced grade 1 cytokine release syndrome (CRS), with one patient experiencing grade 2 immune effector cell-associated neurotoxicity syndrome (ICANS) [3]. - No dose-limiting toxicities (DLT) were reported, highlighting the favorable safety profile of Relma-cel in this patient population [3]. Group 5: Future Prospects - The ongoing study aims to gather longer follow-up data, with the potential for Relma-cel to advance to the biologics license application (BLA) stage [4]. - The company anticipates further discussions with regulatory authorities to expedite the Phase II pivotal study, aiming to provide a breakthrough treatment option for SLE patients [4].