A Randomized, Open-Label, 2-Arm, 2-Part, Parallel Group Study to Assess the Pharmacokinetic Comparability of Subcutaneously Administered Litifilimab (BIIB059) Delivered by 3 Devices (Pre-Filled Syringe, Autoinjector, or On-Body Injector) in Healthy Participants

In this study, researchers will learn how the body processes litifilimab when it is given under the skin in 3 different ways. Currently, ongoing studies utilize pre-filled syringes (PFS) that can deliver litifilimab subcutaneously (SC), also known as under the skin.
In this study, researchers want to learn more about new ways of delivering litifilimab SC using either an autoinjector (AI) or an on-body injector (OBI):
Both devices are designed to deliver litifilimab in an automatic way, especially helping patients who may not be able to use their hands very well, or who may be afraid of needles. While the AI is handheld, the OBI device works by being placed on the skin and can help deliver the highest amount of litifilimab through a single injection. The main objective of this study is to learn how the body processes litifilimab after using the AI device or the OBI device, as compared to using the PFS method.
The main questions researchers want to answer are:
* What is the highest amount of litifilimab found in the blood after dosing? How much total litifilimab is found in the blood throughout the study? Researchers will also learn more about: Any medical problems the participants have during the study
* Any injection site pain or reactions the participants may have. Any skin reactions to the OBI device
* Any changes in the participants' overall health after receiving litifilimab.
This study will be done as follows:
* Participants will be screened to check if they can join the study. The screening period will be up to 4 weeks, after which selected participants will check into their study research center.
* Participants will be randomly assigned to be in Part 1 or Part 2 of the study:
* Part 1: Participants will receive SC injection(s) of litifilimab through either the AI device or through PFS.
* Part 2: Participants will receive SC injection(s) of litifilimab through the OBI device or through PFS.
* Participants will remain at their study research center for the first 8 days. After that, there will be a follow-up period for 17 weeks during which participants return to the center a total of 6 times.
Each participant will be in the study for about 22 weeks.

开始日期2025-01-02

申办/合作机构

Biogen, Inc.

NCT06044337

/ Enrolling by invitation临床3期

A Multicenter, Open-Label, Single-Arm, Phase 3, Long-Term Extension Study to Evaluate Continuous Safety and Efficacy of BIIB059 (Litifilimab) in Adult Participants With Active Subacute Cutaneous Lupus Erythematosus and/or Chronic Cutaneous Lupus Erythematosus With or Without Systemic Manifestations and Refractory and/or Intolerant to Antimalarial Therapy

In this study, researchers will learn more about a study drug called BIIB059 (litifilimab) in participants with cutaneous lupus erythematosus (CLE). The study will focus on participants who have either active subacute CLE or chronic CLE, or both. They may also have systemic lupus erythematosus (SLE). The participants did not respond to antimalarial therapy or had problems with the treatment that made it hard to continue.
The study will enroll only those participants who have completed treatment with litifilimab in the parent study, 230LE301.
The main objective of the study is to learn more about the long-term safety of litifilimab.
The main question researchers want to answer is:
* How many participants have adverse events and serious adverse events after taking litifilimab? Adverse events are health problems that may or may not be caused by the study drug.
Researchers will also learn more about the effect of litifilimab on CLE. They will do this by measuring the symptoms of CLE over time using a variety of scoring tools. These include the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI), the Cutaneous Lupus Activity of Investigator's Global Assessment-Revised (CLA-IGA-R), and the SELENA-SLEDAI Flare Index (SFI).
Researchers will look at how litifilimab and CLE affect the quality of life of participants using a group of questionnaires. They will also look at how litifilimab affects laboratory tests and how participants' immune systems respond to litifilimab.
The study will be done as follows:
* The last visit of parent study 230LE301 will be the first visit of study 230LE305.
* All participants will receive litifilimab as an injection under the skin once every 4 weeks. Both researchers and participants will know the dose and identity of the study drug.
* Globally, the treatment period will last up to 208 weeks, or 4 years. Participants will have up to 53 study visits during this time.

开始日期2023-10-03

申办/合作机构

Biogen, Inc.

NCT05531565

/ Active, not recruiting临床2/3期

A 2-Part Seamless Part A (Phase 2)/Part B (Phase 3) Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BIIB059 in Participants With Active Subacute Cutaneous Lupus Erythematosus and/or Chronic Cutaneous Lupus Erythematosus With or Without Systemic Manifestations and Refractory and/or Intolerant to Antimalarial Therapy (AMETHYST)

In this study, researchers will learn more about a study drug called litifilimab (BIIB059) in participants with cutaneous lupus erythematosus (CLE). The study will focus on participants who have either active subacute CLE or chronic CLE, or both. They may also have systemic lupus erythematosus (SLE). The participants did not respond to antimalarial therapy or had problems with the treatment that made it hard to continue.
The main objective of the study is to learn about the effect litifilimab has on lowering the activity of the skin disease. Researchers will measure symptoms and signs of CLE over time using a variety of scoring tools. These include the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI), the Cutaneous Lupus Activity of Investigator's Global Assessment-Revised (CLA-IGA-R), and the SELENA-SLEDAI Flare Index (SFI).
The main questions researchers want to answer are:
* How many participants have a score of 0 or 1 on the CLA-IGA-R looking at skin redness after treatment?
* How many participants have their skin disease activity go down by at least 70% as measured by CLASI?
Researchers will also learn more about the safety of litifilimab. They will study how participants' immune systems respond to litifilimab. Additionally, they will measure the effect litifilimab and CLE have on the quality of life of participants using a group of questionnaires.
The study will be split into 2 parts - Part A and Part B. Both parts will be done as follows:
* After screening, participants will be randomized to receive either litifilimab or placebo for the 1st treatment period. A placebo looks like the study drug but contains no real medicine.
* Participants will receive either litifilimab or placebo as injections under the skin once every 4 weeks.
* The 1st treatment period will be double blinded which means neither the researchers nor the participants will know if the participants are receiving litifilimab or placebo.
* This double blinded treatment period will last 24 weeks, after which the 2nd treatment period will begin.
* During the 2nd treatment period, all participants will receive litifilimab for 28 weeks.
* After completing treatment in this study, participants that qualify will be given the choice to join the Long-Term Extension study, 230LE305. If they do not, they will move into a follow-up safety period that will last up to 24 weeks.
* The total study duration for participants will be up to 80 weeks.

开始日期2022-09-13

申办/合作机构

Biogen, Inc.

100 项与利替非利单抗相关的临床结果

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100 项与利替非利单抗相关的专利（医药）

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项与利替非利单抗相关的文献（医药）

2026-10-01·JOURNAL OF INTERNAL MEDICINE

Advances in the treatment of systemic lupus erythematosus: Biologicals, small‐molecular agents, and cell‐depleting therapies coming to the clinic

Review

作者: van Vollenhoven, Ronald

Abstract:

Systemic lupus erythematosus (SLE; lupus) remains a particular challenge for the practicing clinician on account of its chronic undulating and unpredictable course, its diverse manifestations, and—until recently—limited treatment options all associated with considerable downsides. However, a major change in this situation is currently taking place. Over the past few years, several new biological therapeutics have been approved for the treatment of SLE (belimumab and anifrolumab) or specifically for its renal organ manifestation (obinutuzumab), along with one new small molecular treatment for the latter (voclosporin). Several additional biologicals (litifilimab and dapirolizumab) and small molecules (deucravacitinib, upadacitinib, and cerenimod) are in late‐stage clinical development. Moreover, multiple case series and case reports have been published in the past years demonstrating excellent individual results following treatments aimed at fully depleting B lymphocytes using chimeric antigen receptor bearing T cells (CAR‐T cells) or bi‐specific T‐cell engagers. These reports have raised hopes that not only solid disease control, but also a more profound state of immunological reset—deep remission—can be attained in at least some patients. In this narrative review, these developments will be described, emphasizing both the promises and the challenges they bring about, leading into some predictions of where they may lead to. A reasonable expectation is that in the coming 1–2 years, several additional biologicals and small‐molecular agents will be approved for the treatment of lupus. The most important conclusion is that the future for our patients with lupus looks brighter than ever before.

2026-10-01·Dermatology and Therapy

Modern Therapies for Discoid Lupus Erythematosus: A Narrative Review

Review

作者: Jonnalagadda, Anshu ; Yildiz-Altay, Ummuguslum ; Vesely, Matthew D ; Pathak, Gaurav N ; Richmond, Rhys L ; Gehlhausen, Jeffrey R ; Desai, Amar D

BACKGROUND:

Discoid lupus erythematosus (DLE) is the most common chronic form of cutaneous lupus erythematosus and can lead to scarring, dyspigmentation, and permanent alopecia. The disease often causes visible disfigurement and has a substantial psychosocial impact. Although many patients respond to standard treatment, a subset continues to experience persistent or relapsing disease that remains difficult to control.

METHODS:

A focused literature search was performed using PubMed, Google Scholar, Web of Science, and Scopus to identify English-language studies published from January 1990 through October 2025. Search terms included "discoid lupus erythematosus," "cutaneous lupus erythematosus," "chronic cutaneous lupus," "pathogenesis," "therapy," "antimalarial," "immunosuppressant," "biologic therapy," "JAK inhibitor," "small molecule therapy," and "targeted therapy." Recent clinical trials, consensus guidelines, and review articles were prioritized. Reference lists of key publications were manually screened to capture additional relevant studies.

RESULTS:

Current first-line topical pharmacotherapeutic treatment options of DLE include photoprotection, topical corticosteroids, and calcineurin inhibitors for localized disease, and antimalarial therapy as a first-line systemic treatment. For refractory disease, systemic immunosuppressants remain common second-line options. Emerging therapies targeting interferon and cytokine signaling pathways, including anifrolumab, belimumab, and tyrosine kinase (TYK2) inhibitors such as deucravacitinib and the plasmacytoid dendritic cell modulators litifilimab and daxdilimab have shown early promise in improving cutaneous disease activity in patients with refractory DLE. Procedural modalities such as laser therapy are being studied as adjuncts for scarring and dyspigmentation.

CONCLUSIONS:

Ongoing advances in understanding the immune mechanisms underlying DLE are shaping a transition from broad immunosuppression toward more targeted treatment strategies. Integrating newer biologic and small-molecule therapies with conventional management may offer improved control and better long-term outcomes for patients with chronic disease.

2026-09-01·JOURNAL OF THE EUROPEAN ACADEMY OF DERMATOLOGY AND VENEREOLOGY

Litifilimab efficacy on skin outcomes in cutaneous lupus erythematosus in the Phase 2 LILAC study

Article

作者: Furie, Richard ; van Vollenhoven, Ronald F. ; Romero‐Diaz, Juanita ; Kalunian, Kenneth ; Merola, Joseph F. ; Li, Qianyun ; Werth, Victoria P. ; Barbey, Catherine ; Yang, Weihong

Abstract:

Background:

Cutaneous lupus erythematosus (CLE) has no approved targeted therapies. In Part B of the randomized, double‐blind, placebo‐controlled, Phase 2 LILAC study, litifilimab demonstrated a dose–response relationship for percentage change in Cutaneous Lupus Erythematosus Disease Area and Severity Index–Activity (CLASI‐A) score from baseline to Week 16.

Objectives:

These post hoc analyses of Part B of the LILAC study aimed to assess the efficacy of litifilimab on additional skin‐specific outcomes.

Methods:

Adult participants with baseline CLASI‐A score ≥8 and histologically confirmed CLE, with/without systemic manifestations, received litifilimab 50 mg, 150 mg or 450 mg, or placebo subcutaneously every 4 weeks through Week 16. Analyses included CLASI‐20/50/70/90 responses (≥20%/50%/70%/90% decrease [improvement] from baseline in CLASI‐A score), 7‐point improvement from baseline in CLASI‐A score, subgroup analyses of percentage change in CLASI‐A scores by baseline characteristics and achievement of ‘clear’ (CLASI‐A scores of 0–1) or ‘almost clear’ (0–3) skin status. Shifts from baseline in skin condition were evaluated by the Physician's Global Assessment (PGA) of CLE and the Physician's Global Impression (PGI) of change in CLE.

Results:

Greater proportions of litifilimab‐treated participants achieved CLASI‐20/50/70/90 responses (3.8%–72.1%) than placebo‐treated participants (0%–43.8%) at Week 16, with significant differences observed from Week 4 for CLASI‐20/50. From Weeks 4 to 16, more litifilimab‐treated participants had ≥7‐point CLASI‐A score decreases from baseline (13.8%–43.9%) compared with placebo (10.5%–21.1%). At Week 16, compared with placebo, more litifilimab‐treated participants achieved ‘clear’ or ‘almost clear’ skin status; CLASI‐A score improvements for subgroups were numerically higher with litifilimab, more litifilimab‐treated participants shifted from ‘severe’ or ‘moderate’ to ‘almost clear’ skin status based on PGA of CLE, and more litifilimab‐treated participants were ‘much improved’ based on PGI results.

Conclusions:

These analyses provide additional evidence to support litifilimab efficacy in clearing skin disease activity, as demonstrated in the LILAC study.

173

项与利替非利单抗相关的新闻（医药）

2026-10-15

·近岸蛋白

BDCA-2：红斑狼疮治疗"70年一遇"的新靶点，为何全球药企竞相布局？

《中国系统性红斑狼疮发展报告2020》显示，系统性红斑狼疮（SLE）困扰着我国超100万患者[1]，长期以来治疗依赖激素与免疫抑制剂，真正能改变疾病进程的创新疗法寥寥。近年来，一个几乎只在浆细胞样树突状细胞（pDC）表面表达的Ⅱ型C型凝集素——BDCA-2（CLEC4C/CD303），正从“冷门靶点”跃升为自免赛道的新星：渤健Litifilimab三项Ⅱ期研究全线告捷并已推进Ⅲ期[2]，映恩生物"自免ADC"DB-2304蓄势待发[3]，三生国健、特宝生物、科兴生物等国内企业纷纷密集入局。本文从靶点与信号通路、成药逻辑、临床格局、专利布局到上游工具供给，带你一次看懂BDCA-2。 01 靶点介绍：pDC表面的"专属名片" 树突状细胞（DC）由Ralph M. Steinman于1973年首次发现[4]，并于2011年斩获诺贝尔生理学或医学奖[5]。DC起源于骨髓，广泛分布于皮肤、气道、淋巴器官等部位，是专职的抗原递呈细胞（APC），其处于启动、调控并维持免疫应答的中心环节。图1.2011年诺贝尔生理学或医学奖根据起源和功能，DC主要分为传统DC（cDC1和cDC2）与浆细胞样DC（pDC）。其中pDC因形态酷似浆细胞而得名，通过TLR7/9识别病毒或自身核酸后产生Ⅰ型干扰素，是体内IFN-α/β最主要的产生者，堪称为专业的IFN-I工厂。 BDCA-2（血液树突状细胞抗原2），又称CLEC4C、CD303或DLEC，由Dzionek等人于2000年首次鉴定为人pDC的特异性标志物[6]。它是一段含有213个氨基酸的Ⅱ型跨膜糖蛋白，其由胞外段、跨膜区、胞内段构成：胞外段为Ca²⁺依赖性碳水化合物识别结构域（CRD），可识别半乳糖末端多糖链[7]；跨膜区为单次跨膜；胞内段较短且缺少已知信号基序，需与含ITAM基序的FcRγ链偶联传递信号。图2.BDCA-2结构（左）[PDB:4ZES]和其他含有C型CRD的2型跨膜受体的结构（右）[8] 02 信号通路：给失控的IFN-I踩"刹车" 生理状态下，pDC通过TLR7/TLR9识别病原体核酸后大量分泌IFN-α/β，是抗病毒免疫第一道防线；BDCA-2则扮演相反角色——pDC免疫反应的负调控开关。其路径如下：图3.BDCA-2(CD303)在浆细胞样树突状细胞（pDC）免疫反应中的负向调控机制 [9] 交联BDCA-2对pDC产生IFN-I的抑制是"广谱"且"上游"的——无论IFN-I由TLR7/9配体还是免疫复合物诱导，BDCA-2信号都能压制IFN-I产生[10]。抗BDCA-2抗体与靶点结合后还会诱导受体快速内化，进一步“关闭”信号门户。这种“从源头调低音量”的机制，正是BDCA-2成药的底层逻辑。 BDCA-2抑制IFN-I产生[10] 03 BDCA-2成为SLE新靶点的三大依据 1 IFN-I是SLE的核心病理引擎活动期SLE患者血清IFN-Ⅰ水平显著升高，且与疾病严重程度和进展相关；超过70%~80%的SLE患者在病程不同阶段出现皮肤病变，约25%的皮肤型红斑狼疮（CLE）可进展为SLE[11]。pDC及其过量分泌的IFN-I，正是驱动“细胞因子风暴”的源头之一。 2 IFN-I通路已被商业化验证抗IFN AR1单抗Anifrolumab在2021年获FDA和EMA批准治疗SLE，上市三年后全球销售额达到34亿元人民币[12]，为整个"pDC-IFN-I轴"的成药性打了个样，而与直接阻断下游IFN-I受体不同，BDCA-2在更上游干预，不仅影响IFN-I信号，还可能同步降低多种炎症因子和ISGs表达，理论上覆盖更广的病理环节。 3 临床数据持续兑现渤健Litifilimab的三项Ⅱ期研究（SLE、CLE、AMETHYST）均达到主要终点，相关结果两度发表于《新英格兰医学杂志》（NEJM），Ⅲ期正在推进。 04 全球临床管线全景：单抗领跑，ADC/双抗跟进根据UmabsDB全球数据库记录，目前全球已有约7款BDCA-2相关药物进入临床阶段：图4.处于临床阶段的BDCA-2相关抗体药物整体管线格局上，单抗仍是BDCA‑2赛道主流开发形式，渤健Litifilimab已经推进至Ⅲ期，是整个靶点的风向标，其 Ⅱ期临床在系统性红斑狼疮、皮肤型红斑狼疮两项适应症上均成功抵达主要终点，相关研究两度登上《新英格兰医学杂志》，也为全球后续BDCA‑2候选分子的开发确立了疗效参照标尺。国内企业一方面快速跟进经典单抗赛道，如三生国健、特宝生物、科兴生物的BDCA‑2单抗相继完成IND申报或是进入Ⅰ期人体试验；另一方面也不局限于简单me‑too路径，积极布局ADC、双抗融合蛋白等差异化创新分子。映恩生物开发全球首创BDCA‑2免疫调节型ADC DB‑2304，借助偶联GR激动剂实现靶向局部给药，发挥协同抗炎效果；维立志博LBL‑047则采用TACI/BDCA‑2双特异性融合蛋白设计，同时靶向pDC与B细胞通路，实现“抑制pDC来源Ⅰ 型干扰素分泌+B细胞活化阻断”的双重药理机制。这类创新分子跳出单纯阻断BDCA‑2信号的单一逻辑，通过多重作用通路，试图实现优于单纯单抗的治疗效果，也为红斑狼疮这类复杂自身免疫病带来更多治疗可能性。 05 国内外专利布局：MNC提前卡位，本土势力快速涌入从国家知识产权局公开数据看，抗BDCA-2抗体中国专利申请呈跨国药企早期卡位、本土创新企业近年提速双轨格局。安斯泰来、卡佩拉、森特萨等已有布局；本土普乐康、宏成、明迅、纽迈等从“新抗体分子+动物模型+应用场景”切入。对下游企业而言，高亲和力、可自由实施的参照抗体和靶点蛋白工具越来越重要。图5.国内关于抗BDCA-2抗体的专利申请一览 06 近岸蛋白BDCA-2产品布局：全流程弹药近岸蛋白针对靶向BDCA-2/CLEC4C抗体药物开发，提供高质量靶点蛋白、参照抗体和抗体一站式服务。 1 BDCA-2（CLEC4C）重组蛋白人、食蟹猴等多个种属，覆盖His、Fc、生物素化等标签形式，适用于动物免疫、抗体筛选、功能评估及质量控制等开发环节；目录号产品名称 C41U Recombinant Human CLEC4C (N-Fc) C55K Recombinant Cynomolgus CLEC4C (Low Endotoxin, N-6His) C55G Recombinant Cynomolgus CLEC4C (Low Endotoxin, N-Fc) CY215 Biotinylated Cynomolgus CLEC4C (Low Endotoxin, N-Avi-Fc) NC416 Anti-Human CLEC4C antibody (Lit_bio, Research Grade) 2 定制化技术服务近岸蛋白依托NovoiSMART®多策略抗体发现平台，可为客户提供BDCA-2相关的一站式解决方案：抗体开发：基于噬菌体展示/杂交瘤技术的抗BDCA-2抗体开发、抗体人源化、亲和力成熟；高通量抗体表达（交付即用）：一体化制备筛选含40+分析、高通量多批稳定表达、灵活交付数据与样品；活性检测：SPR/BLI分子互作平台，支持亲和力、动力学及竞争实验；参考资料 [1]《中国系统性红斑狼疮发展报告2020》 [2]https://www.chinadrugtrials.org.cn/clinicaltrials.searchlist.dhtml [3]https://www.chinadrugtrials.org.cn/clinicaltrials.searchlistdetail.dhtml [4]Steinman RM, Cohn ZA. “Identification of a novel cell type in peripheral lymphoid organs of mice. I. Morphology, quantitation, tissue distribution.” Journal of Experimental Medicine, 1973, 137(5): 1142–1162. [5]https://www.nobelprize.org/prizes/medicine/2011/summary/ [6]Dzionek A, Fuchs A, Schmidt P, Cremer S, Zysk M, Miltenyi S, Buck DW, Schmitz J. “BDCA-2, BDCA-3, and BDCA-4: three markers for distinct subsets of dendritic cells in human peripheral blood.” Journal of Immunology, 2000, 165(11): 6037–6046. [7]Jégouzo SAF, Feest ES, Taylor ME, Drickamer K. “Release of glycan ligands by endoglycosidases reveals multivalent binding of DC-SIGN to high-mannose glycans.” Journal of Biological Chemistry, 2015, 290(27): 16759–16771. [8] Jégouzo, Sabine AF, et al. "A novel mechanism for binding of galactose-terminated glycans by the C-type carbohydrate recognition domain in blood dendritic cell antigen 2." Journal of Biological Chemistry 290.27 (2015): 16759-16771. [9]Wilson, Nathaniel R., et al. "CD303 (BDCA-2)-a potential novel target for therapy in hematologic malignancies." Leukemia & lymphoma 63.1 (2022): 19-30. [10]Cho, Y. M., & Furie, R. (2024). The Development of Litifilimab (BIIB 059) for Cutaneous and Systemic Lupus Erythematosus. Immunotherapy, 16(1), 15–20. [11]《关于注射用ACT100获得药物临床试验批准通知书的公告》 [12]https://bydrug.pharmcube.com/news/detail/b5c83fdffe5efde3bd7325c418af8d25 苏州近岸蛋白质科技股份有限公司（简称近岸蛋白）深耕重组蛋白行业十余年，是一家专注于蛋白质技术与应用解决方案的高新技术企业，主营业务为生物药、体外诊断、mRNA疫苗药物、生命科学基础研究等领域的原料与技术解决方案，包括靶点及因子类蛋白、重组抗体、酶及试剂的研发、生产和销售及相关技术服务。公司在上海、苏州和菏泽建有研发、生产基地。访问www.novoprotein.com.cn或致电400-600-5970。点击“阅读原文”，了解更多产品信息

2026-10-11

·EnzyJoy悦酶

全球新药研发前沿速览（2026年10月2日—10月9日）

全球新药研发前沿周报统计窗口：2026年10月2日—10月9日（截至北京时间10月9日08:30）证据强度说明：高＝监管文件或完整随机对照数据；中高＝有明确效应量的随机 III 期公司顶线结果；中＝随机 II 期公司数据；初步＝小样本、未完整披露或临床前结果。本周最值得关注Newron 的 evenamide 因死亡病例和潜在心律失常风险继续被 FDA 暂停美国试验。epcoritamab 联合 R-CHOP 将初治 DLBCL 进展或死亡风险降低51%，可能改变一线治疗格局。三项 FDA 肿瘤批准覆盖 dMMR 结肠癌、HER2阳性乳腺癌和初治 CLL/SLL。非多巴胺机制帕金森药 solengepras III 期成功，但绝对疗效幅度不大。argenx 同日出现一成一败：乳糜泻项目 FB102 II 期阳性，Vyvgart 干燥综合征 III 期因无效性终止。交易持续活跃：Novartis–Abogen 潜在78亿美元 RNA 交易规模最大，Viatris 则以16.5亿美元收购 Pacira。重点研发与监管进展1. Evenamide：FDA维持美国临床暂停 **候选药物/靶点：**evenamide，选择性电压门控钠通道调节剂，通过调节谷氨酸释放发挥作用。 **适应症及阶段：**难治性精神分裂症，ENIGMA-TRS 2 III期。 **已证实事实：**FDA在所有 evenamide 研究中识别出治疗组4例死亡、安慰剂组1例死亡，并认为无法排除致命性心律失常机制。研究者将其中3例判定为与药物无关，1例因尸检无法确定死因而判定为“可能相关”。美国中心继续暂停入组，境外研究仍在进行。 **可信度：高。**FDA书面反馈由公司正式披露，Reuters作独立报道；但死亡与药物的因果关系尚未证实。 **潜在影响：**这是本周最重要的安全事件。项目能否恢复美国入组，取决于追加心脏筛查、监测方案及FDA对累计安全数据的评价。Newron Pharmaceuticals2. Epcoritamab联合R-CHOP：初治DLBCL III期达到PFS终点 **药物/靶点：**epcoritamab，皮下注射 CD3×CD20 双特异性抗体。 **阶段：**EPCORE DLBCL-2 III期；初治弥漫性大B细胞淋巴瘤。 **关键结果：**与R-CHOP相比，联合方案将疾病进展或死亡风险降低51%，HR 0.49（95%CI 0.36–0.67，p<0.0001）。独立数据监测委员会建议揭盲。 **可信度：中高。**随机III期且效应量明确，但仍是首次计划内期中分析；总生存、完整安全性和亚组数据尚未公布。 **潜在影响：**若监管机构认可，可能将双抗前移至DLBCL一线治疗，但仍需评估感染、细胞因子释放综合征及R-CHOP基础上的额外毒性。prnewswire.com3. FDA本周三项重要肿瘤批准 **Atezolizumab＋化疗：**获批用于已完整切除的III期dMMR结肠癌辅助治疗。ATOMIC研究共712人，DFS风险比0.50（95%CI 0.35–0.73）；两组中位DFS均未达到。**证据强度：高。**这是具有潜在治愈意义的辅助治疗扩展。FDA **Tucatinib＋trastuzumab＋pertuzumab：**获批用于HER2阳性不可切除局部晚期或转移性乳腺癌诱导治疗后的维持。654人研究中位PFS为24.9个月对16.3个月，HR 0.64；OS尚未成熟。**证据强度：高。**需继续关注肝毒性及最终生存获益。FDA **Pirtobrutinib：**获批用于无已知17p缺失的初治CLL/SLL。与苯达莫司汀＋利妥昔单抗相比，PFS HR为0.20；但对照方案在当前临床实践中的代表性需要结合地区指南判断，OS尚未成熟。证据强度：高。FDA4. Solengepras：首个GPR6抑制剂帕金森III期阳性 **机制：**口服GPR6抑制剂，不直接作用于多巴胺受体。 **阶段：**ARISE随机、双盲、安慰剂对照III期，341人。 **结果：**150 mg剂量在第12周较安慰剂额外减少每日“OFF”时间0.61小时（p=0.035）；无明显运动障碍的“ON”时间增加0.60小时。因不良事件停药率三组均为3.5%。 **可信度：中高。**达到预设主要终点，但仅为公司顶线数据，且绝对改善约37分钟/日。 **潜在影响：**机制创新明显，耐受性看起来较好；监管讨论将取决于单项III期是否足以申报以及完整功能、生活质量和长期安全数据。globenewswire.com5. Argenx：FB102阳性，但Vyvgart干燥综合征III期终止 **FB102/CD122—乳糜泻：**126人随机II期在8周麸质激发后达到绒毛高度/隐窝深度比主要终点（p=0.0176），其他组织学、炎症和症状指标方向一致；公司计划进入III期。**证据强度：中。**目前未披露绝对效应量和剂量组细节。argenx.com **Efgartigimod/Vyvgart—干燥综合征：**独立数据监测委员会判断UNITY III期无法达到第48周clinESSDAI主要终点，建议因无效性终止；未发现新安全信号。**证据强度：高。**这是明确的适应症开发失败，不代表Vyvgart已获批适应症的获益风险发生变化。argenx.com6. Firmonertinib：EGFR exon 20插入肺癌III期失败 **阶段：**FURVENT III期，398名未经治疗的晚期NSCLC患者。 **结果：**240 mg组中位PFS为11.0个月，化疗组9.5个月，HR 0.75（95%CI 0.55–1.02，p=0.0654），未达到主要终点；ORR分别为60%和33%。160 mg组PFS为8.4个月。 **可信度：中高。**随机III期结果明确，但OS仍未成熟。 **潜在影响：**高剂量有肿瘤缓解信号，却不足以支持当前一线申报路径；公司仍可能探索特定亚组、其他EGFR突变或联合方案。ArriVent Biopharma其他值得关注的临床进展 Taladegib/ENV-101—特发性肺纤维化，IIb期：213人WHISTLE-PF研究报告第24周ppFVC、FVC及定量CT纤维化指标显著优于安慰剂，73%受试者使用背景抗纤维化药。由于公司尚未公布各组数值、置信区间及停药处理，暂评为中等证据；“逆转纤维化”的表述仍属公司解释。streetinsider.com **Petrelintide—肥胖合并2型糖尿病，II期：**220人中，28周平均减重最高9.2%，安慰剂2.0%；HbA1c较安慰剂改善0.61%–0.88%，胃肠道事件导致的停药率为1.9%对1.7%。**证据强度：中。**耐受性可能是相对优势，但减重幅度低于部分多受体激动剂，需等待约7000人的III期项目。globenewswire.com **ATSN-201—X连锁视网膜劈裂症，I/II期：**18名接受治疗的成人和儿童中未见药物相关严重不良事件；关键剂量组9人中3人视力改善至20/40或更好。**证据强度：初步。**样本极小且部分比较并非随机，III期队列结果预计2028年。atsenatx.com **Litifilimab/BDCA2—皮肤型红斑狼疮，II期延长期：**连续治疗至52周者中，27.2%达到皮损清除或接近清除，28.8%达到CLASI-70；严重不良事件发生率3.4%。**证据强度：初步至中等。**第24周后缺乏同期安慰剂对照，不能据此单独确认长期比较疗效；III期结果预计2027年上半年。Biogen临床前与早期研发 RTX-001/周细胞功能—遗传性出血性毛细血管扩张症：临床前公司RougeTx完成5800万美元A轮融资，计划推进口服RTX-001，通过恢复周细胞附着来稳定异常血管并进入人体研究。现阶段未公开可独立评估的完整药效或毒理数据，属于高风险、机制驱动的临床前项目。RougeTx **Gate Bioscience–Lilly：**双方扩大“molecular gate”小分子蛋白清除合作，新增一个靶点并保留第二个靶点选择权；潜在总额超过8.7亿美元，首付款未披露。项目处于发现及临床前阶段，战略意义在于尝试用口服小分子处理传统难成药蛋白。Reuters重要交易与融资 **Novartis–Abogen：**Novartis支付5.75亿美元首付款，获得mRNA编码CD19×CD3 T细胞衔接器ABO2203的全球权利，并可选择其他RNA项目；里程碑最高约72亿美元。ABO2203已有极小规模早期人体数据，计划方向包括自身免疫疾病中的B细胞清除。**战略意义：**将T细胞衔接器由外源蛋白给药转为体内mRNA表达，但疗效可控性、细胞因子释放风险和重复给药仍需验证。Reuters **Viatris–Pacira：**Viatris拟以16.5亿美元现金收购Pacira，获得Exparel、Zilretta及包括膝骨关节炎基因治疗PCRX-201在内的管线。**战略意义：**Viatris从仿制药业务进一步转向专利保护的非阿片疼痛产品；交易仍需满足交割条件。Oct 8, 2026 **Biohaven–Ono：**Ono取得BHV-1300、BHV-1310及BHV-1320在日本、韩国、台湾和东盟的权利；Biohaven获8000万美元首付款、近期最高2000万美元里程碑及约20%销售提成。BHV-1300为III期小分子胞外IgG降解剂。**战略意义：**为新型胞外蛋白降解平台提供区域验证和非稀释性资金。Biohaven, Ltd. **Matchpoint融资：**完成1.5亿美元B轮，资金用于把口服T细胞调节剂MPT-062推进至2027年临床及支持其他共价小分子项目。项目仍处于临床前阶段，融资规模体现市场对精准共价药物平台的兴趣。Matchpoint Therapeutics建议优先跟进 **Newron/FDA：**关注FDA是否接受强化心电筛查与监测方案，以及四例死亡的独立心脏安全性判定。 **Epcoritamab：**等待完整PFS曲线、OS、亚组结果及感染和CRS数据，判断能否真正改变DLBCL一线标准。 **Taladegib：**优先获取ppFVC和定量CT的绝对效应量；在缺乏数字前，不宜采信“逆转IPF”的强结论。 **Solengepras：**评估约0.61小时/日的OFF时间改善是否达到临床意义，并关注FDA是否要求第二项确认性III期。 **FB102与petrelintide：**跟踪完整剂量反应、安全性及III期设计。 **FDA新批准药物：**重点追踪atezolizumab辅助治疗和tucatinib维持治疗的成熟OS结果。

2026-10-08

·药械合创谷

新药还没上市，AI 先说"不行"

AI + 制药 · 新闻新药还没上市，AI 先说"不行" 一款药还没进III 期，算法就说过它会失败。三个月后，股价蒸发 300 亿美元。 2026 年 9 月初，诺华一款治疗罕见肌肉营养不良的实验药物 del-desiran Ⅲ 期试验未达终点。这不是那种"结果未达预期"的软性表述——按诺华 CEO 事前的公开预期，该药年峰值销售额可达 50 亿美元。消息一出，股价当日跌 11%，约 300 亿美元市值蒸发。而丹麦哥本哈根一家叫 BioinvestGPT 的 AI 公司说：这事我们 7 月就模拟过了。它是怎么"提前看到"的 BioinvestGPT 的做法不是玄学。它的路径是：先通过 DNA 测序，构造一个符合某项特定临床试验入组条件的人体模型；再用这个虚拟人体，配合试验药物的模型，跑一场"虚拟试验"。 7 月，他们把 del-desiran 的虚拟人体跑了一遍，结论是临床获益微小（insignificant clinical benefit）。9 月，真实Ⅲ期试验落空。为什么这件事值得盯全球生物制药行业每年在人体临床试验上花掉约 1400 亿美元，而最终能拿到监管批准的候选药物只有约 12%——这个数字几十年几乎没动过。更要命的是时间：传统临床试验从Ⅰ期到Ⅲ期要跨数年，而某些 AI 模拟一个月内就能跑完。六次押中五次 BioinvestGPT 在 7 月向路透社披露了一批"结果未知"的顶级试验预测清单。截至目前，它称对六项重大试验的预测命中五次，包括：准确看空 del-desiran；看好 Moderna 与默沙东的黑色素瘤疫苗；指出阿斯利康与 Ionis 的心脏药 Wainua 临床获益偏弱；看空诺和诺德心脏药 ziltivekimab 的首次试验；看好 Vaxcyte 的肺炎球菌疫苗。它还给出了后续预测：预计 Biogen 的 litifilimab 在最常见类型狼疮上的两项Ⅲ期试验失败，以及武田 zasocitinib 在克罗恩病和溃疡性结肠炎上的Ⅱ期研究失败。模型给出的判断是，这两个药对上述特定试验人群而言是"次优的"。公司的判断逻辑并不复杂。Biogen 这款药打的是 BDCA2 这个免疫细胞受体，武田的 zasocitinib 抑制的是酪氨酸激酶 2（TYK2）——后者武田已在银屑病领域递交美国上市申请，另有银屑关节炎Ⅲ期在跑。失手的那一次，和业界的反弹虚拟试验并非不败。BioinvestGPT 曾预测诺华的 pelacarsen 会成功——这款药用来降低脂蛋白(a) 水平。2026 年 9 月诺华宣布，该药在有遗传风险因素的患者群体中，未能降低重大心梗或卒中的风险。联合创始人 Bragi Lovetrue 后来复盘承认："我们把机制搞错了。"原因是模型没有把脂蛋白颗粒大小的遗传性变异算进去。制药界的反应比想象中冷。武田研究负责人 Andy Plump 表示，TYK2 这个靶点是通过分析人类遗传学发现的，机器学习只是用来优化那颗每日口服药的结构、把它"打磨"了一下——"我对这个机制有十足的信心。我们还远没到能用 AI 这类工具做出确定性预测的地步。" Biogen 负责多发性硬化、免疫与阿尔茨海默病临床开发的 Diana Gallagher 则指出另一个方向的风险：目前红疮狼疮获批的生物药只有两个，靠历史数据训练的算法天然可能偏向"预测失败"。一个被问出来的伦理问题以色列 AI 临床试验模拟平台 QuantHealth 的首席医学官 Francisco Beca 把这件事推到了伦理层面："我们不该只问怎么把试验跑得更快，还该问怎么少跑那些注定要失败的试验。"他进一步追问："2026 年了，把患者暴露在一个大概率会失败的试验里，还合乎伦理吗？随着这门技术的进展，到 2027、2028 或 2029 年，答案恐怕就不是了。" QuantHealth 用真实世界数据和 AI 模拟患者层面的治疗响应，也已公布过溃疡性结肠炎与降胆固醇药试验的模拟结果。McKinsey 合伙人 Alex Devereson 的判断要冷静得多：药企目前只是"用脚尖试水"（dipping their toes），多在内部或与外部伙伴一起，用于在向某个项目投入资金前评估候选药物。钱也在往这来。据 McKinsey 报告，AI 药物发现领域投资额在 2025 年较 2023 年翻了一倍以上，达到 84 亿美元。但报告同时指出：这些钱主要花在分子设计上——那是技术当下最擅长的部分，而不是行业真正的瓶颈，比如证明一个药在漫长试验中确实按预期起效。监管侧也在动。上周，美国卫生监管机构公布了一组旨在加快临床试验的举措。一位联邦卫生官员表示，如果推进顺利，该项目可能为预测性 AI 走进临床开发打开一条通道。但行业自身的定性相当清楚：现阶段这门技术是真人试验的补充，不是替代。药企的根本目标始终是证明相对标准疗法有更优的临床获益。AI 或许能帮它筛出"哪些项目值得去做那份昂贵的证明"，而最后一场试验，终究要靠真实病人跑完。今日互动如果你是研发负责人，手里有一个跑完Ⅱ期、准备投Ⅲ期的分子——你会把 AI 模拟结果写进决策报告，还是只在心里记一笔？留言说说你的取舍。

100 项与利替非利单抗相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
皮肤红斑狼疮	临床3期	美国	Biogen, Inc.	2023-10-03
皮肤红斑狼疮	临床3期	中国	Biogen, Inc.	2023-10-03
皮肤红斑狼疮	临床3期	日本	Biogen, Inc.	2023-10-03
皮肤红斑狼疮	临床3期	阿根廷	Biogen, Inc.	2023-10-03
皮肤红斑狼疮	临床3期	比利时	Biogen Idec Research Ltd.	2023-10-03
皮肤红斑狼疮	临床3期	巴西	Biogen, Inc.	2023-10-03
皮肤红斑狼疮	临床3期	保加利亚	Biogen, Inc.	2023-10-03
皮肤红斑狼疮	临床3期	加拿大	Biogen, Inc.	2023-10-03
皮肤红斑狼疮	临床3期	智利	Biogen, Inc.	2023-10-03
皮肤红斑狼疮	临床3期	哥伦比亚	Biogen, Inc.	2023-10-03

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05531565 (AMETHYST, ACR2026)	临床2/3期	皮肤红斑狼疮	93	Litifilimab	鏇餘簾製遞鬱鬱餘鑰鬱(繭衊齋築鏇膚獵鏇遞淵) = 襯觸艱鹹獵觸蓋觸醖鏇衊顧願獵夢糧淵選選鹹 (鏇積糧積艱蓋糧膚餘獵 ) 更多	积极	2026-11-06
				Placebo	膚齋窪獵選蓋鬱鹽壓繭(鬱範鏇憲衊夢網築襯糧) = 獵鑰獵鏇鹹觸糧艱遞壓鹽顧憲壓襯淵憲鹹襯膚 (衊鹽觸襯鬱積製鏇醖夢 ) 更多
NCT02847598 (LILAC \| LILAC study, CTgov)	临床2期	皮肤红斑狼疮 \| 系统性红斑狼疮 \| 盘状红斑狼疮	264	Placebo (Part A: Placebo)	範構鬱膚憲製選願選壓(衊廠觸鏇願繭廠鹽憲鏇) = 膚遞積積淵餘憲網窪鏇簾糧淵廠窪淵齋淵衊構 (餘餘膚齋鑰構築夢願壓, 10.3) 更多	-	2023-05-15
				BIIB059 (litifilimab) (Part A: BIIB059 450 mg)	範構鬱膚憲製選願選壓(衊廠觸鏇願繭廠鹽憲鏇) = 積鏇淵鹽糧觸觸顧選糧簾糧淵廠窪淵齋淵衊構 (餘餘膚齋鑰構築夢願壓, 8.7) 更多
NCT02847598 (LILAC, Pubmed \| N Engl J Med)	临床2期	系统性红斑狼疮 BDCA2	132	Litifilimab 450 mg	衊壓膚艱鏇蓋觸選衊願(網衊鏇範糧廠鏇艱簾壓): P-Value = 0.04 更多	积极	2022-09-08
				Placebo
NCT02847598 (LILAC, Pubmed \| N Engl J Med)	临床2期	皮肤红斑狼疮	132	Litifilimab 50 mg	繭艱簾憲顧鑰簾築製鬱(蓋簾廠築鑰襯網選艱鑰) = Litifilimab was associated with one case of herpes zoster infection 鑰膚餘簾網顧鏇願顧廠 (製獵網膚簾餘淵繭齋網 ) 更多	积极	2022-07-28
				Litifilimab 150 mg
NCT02847598 (LILAC study, EULAR2022)	临床2期	皮肤红斑狼疮 BDCA2	-	BIIB059	選淵廠願鏇觸廠構衊蓋(鹽淵窪淵選觸壓艱醖製) = 鏇膚鑰鑰餘襯餘網獵蓋醖網鹽範構觸鑰齋鹹網 (鑰淵廠鑰壓願鹽窪壓製 ) 更多	积极	2022-06-01
NCT02847598 (LILAC, EULAR2022)	临床2期	系统性红斑狼疮 anti-nuclear antibodies \| anti-double-stranded DNA antibodies	-	BIIB059 450 mg	築壓積壓糧膚鬱齋繭獵(衊膚衊顧遞窪鹹積製選) = 獵糧鹹鑰淵蓋鬱窪鬱淵糧遞網艱製鹹範繭鑰網 (鏇鬱鬱遞艱窪醖製壓齋 ) 更多	积极	2022-06-01
				Placebo	築壓積壓糧膚鬱齋繭獵(衊膚衊顧遞窪鹹積製選) = 鬱蓋網網鬱顧構網遞顧糧遞網艱製鹹範繭鑰網 (鏇鬱鬱遞艱窪醖製壓齋 ) 更多
NCT02847598 (LILAC, ACR2020) 人工标引	临床2期	系统性红斑狼疮	120	BIIB059	窪積壓選艱鑰鏇夢網蓋(觸蓋構淵艱淵齋鑰醖築) = 襯糧構築蓋窪鹽選網憲鹽糧製齋選築鹹鏇夢範 (膚選鹽膚襯觸構齋製憲, 1.2) 更多	积极	2020-11-07
				Placebo	窪積壓選艱鑰鏇夢網蓋(觸蓋構淵艱淵齋鑰醖築) = 蓋淵積鬱襯憲獵築積範鹽糧製齋選築鹹鏇夢範 (膚選鹽膚襯觸構齋製憲, 1.3) 更多
NCT02847598 (LILAC, EULAR2020)	临床2期	皮肤红斑狼疮	132	BIIB059 50 mg	構觸願齋齋鬱遞鬱觸繭(鹹壓遞鑰願鬱膚窪淵襯) = 62 (62.6%) 80 (60.6%) 願窪淵艱顧廠蓋齋襯憲 (願簾鹹網築餘壓艱遞積 ) 更多	积极	2020-06-03
				BIIB059 150 mg
NCT02847598 (Pubmed \| J Pharmacokinet Pharmacodyn)	临床2期	皮肤红斑狼疮	264	BIIB059	艱鏇窪夢願淵憲膚獵築(衊襯選鏇襯構範鹹積遞) = 憲淵觸窪繭積艱窪膚衊餘鬱簾憲繭鬱鹽廠獵鏇 (顧壓艱構範夢獵鬱選鹹 ) 更多	-	2020-06-01
NCT02106897 (Pubmed \| J Clin Invest) 人工标引	临床1期	湿疹 \| 系统性红斑狼疮	54	BIIB059	構鹽鏇遞蓋鏇鑰構網壓(網衊襯繭鏇廠艱夢淵顧) = 繭壓蓋製鹹齋築範鏇餘醖範製鏇齋夢製鏇願糧 (餘糧壓獵遞鏇網壓餘糧 ) 更多	积极	2019-03-01
				Placebo	構鹽鏇遞蓋鏇鑰構網壓(網衊襯繭鏇廠艱夢淵顧) = 壓願鹹齋觸衊餘鹽築製醖範製鏇齋夢製鏇願糧 (餘糧壓獵遞鏇網壓餘糧 ) 更多