A Phase 2, Open-label, Multicenter Multi-cohort Study to Evaluate the Efficacy and Safety of Anitocabtagene Autoleucel in Participants With Newly Diagnosed Multiple Myeloma

The goal of this clinical trial is to learn if anitocabtagene autoleucel following induction therapy works to treat adult participants with newly diagnosed multiple myeloma. The main objectives of this clinical trial are:
* To determine the incidence and severity of all adverse events (AEs).
* To determine the proportion of patients achieving undetectable minimal residual disease (uMRD) negative-CR rate (minimum 10 to -5) at 12 months (+/- 3 months) after enrollment.
Participants will receive induction therapy with a quadruplet regimen including a proteasome inhibitor (Bortezomib [V]), immunomodulatory drug (Lenalidomide [R]), dexamethasone [d] and anti-CD38 monoclonal antibody (Daratumumab [D] or Isatuximab [Isa]) followed by anitocabtagene autoleucel. Participants in Cohorts A and B will receive lenalidomide maintenance therapy following infusion with anitocabtagene autoleucel.

开始日期2025-06-30

申办/合作机构-

NCT06626919

/ Recruiting临床1期

A Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects With Non-oncology Plasma Cell-related Diseases

A Phase 1 dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of anito-cel in subjects with generalized myasthenia gravis (GMG). Anitocabtagene autoleucel (anito-cel) is a BCMA-directed CAR-T cell therapy.

开始日期2025-04-30

申办/合作机构

Arcellx, Inc.

NCT06413498

/ Recruiting临床3期

A Phase 3, Randomized, Open-Label Study to Compare the Efficacy and Safety of Anitocabtagene Autoleucel Versus Standard of Care Therapy in Participants With Relapsed/Refractory Multiple Myeloma

The goal of this study (iMMagine-3) is to compare the study drug, anitocabtagene autoleucel to standard of care therapy (SOCT) in participants with relapsed/refractory multiple myeloma who have received 1 to 3 prior lines of therapy, including an anti-CD38 monoclonal antibody and an immunomodulatory drug.
The primary objective of this study is to compare the efficacy of anitocabtagene autoleucel versus SOCT in participants with RRMM.

开始日期2024-08-23

申办/合作机构

Kite Pharma, Inc.

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项与阿尼托卡布他基因自体淋巴细胞相关的文献（医药）

2023-03-14·Blood Advances2区 · 医学

Phase 1 study of CART-ddBCMA for the treatment of subjects with relapsed and refractory multiple myeloma

2区 · 医学

Article

作者: Shaw, Kit ; Raje, Noopur ; Heery, Christopher ; Bishop, Michael R. ; O’Donnell, Elizabeth K. ; Daley, Heather ; Frigault, Matthew J. ; Jakubowiak, Andrzej ; Rosenblatt, Jacalyn ; Maus, Marcela V. ; Griffin, Faith ; Yee, Andrew J. ; Nikiforow, Sarah ; Logan, Emma ; Cook, Daniella ; Avigan, David E. ; Cornwell, Christine ; Shen, Angela

Abstract:

Relapsed and refractory multiple myeloma (RRMM) is a plasma cell neoplasm defined by progressively refractory disease necessitating chronic and increasingly intensive therapy. Despite recent advances, limited treatment options exist for RRMM. This single-arm, open label phase 1 study aimed to evaluate the safety of novel B-cell maturation antigen (BCMA)-targeting chimeric antigen receptor (CAR) T construct that leverages a completely synthetic antigen-binding domain (CART-ddBCMA), which was specifically engineered to reduce immunogenicity and improve CAR cell surface stability. Thirteen patients ≥18 years with RRMM who received at least 3 prior regimens of systemic therapy were enrolled in the study. Patients received a single dose of 100 × 106 CART-ddBCMA (DL1) or 300 × 106 CART-ddBCMA (DL2) following standard lymphodepleting chemotherapy. The primary endpoints of the study were to evaluate the incidence of treatment emergent adverse events, including dose-limiting toxicities, and establish a recommended phase 2 dose. Results showed that CART-ddBCMA was well tolerated and demonstrated a favorable toxicity profile. Only 1 case of grade ≥3 cytokine release syndrome and 1 case of immune effector cell–associated neurotoxicity were reported; both were at DL2 and were manageable with standard treatment. No atypical neurological toxicities and Parkinson disease-like movement disorders were observed. The maximum tolerated dose was not reached. All infused patients responded to CART-ddBCMA, and 9/12 (75%) patients achieved complete response/stringent complete response. Responses deepened over time, and at the time of last data-cut (median follow-up 56 weeks), 8/9 (89%) evaluable patients achieved minimal residual disease negativity. In conclusion, the findings demonstrate the safety of CART-ddBCMA cells and document durable responses to CART-ddBCMA in patients with RRMM. This trial was registered at www.clinicaltrials.gov as #NCT04155749.

2022-07-05·Molecular cancer therapeutics

Preclinical Efficacy of BCMA-Directed CAR T Cells Incorporating a Novel D Domain Antigen Recognition Domain

Article

作者: Richman, Laura K. ; Tice, David A. ; Witter, Alexandra R. ; Zaritskaya, Liubov ; Hilbert, David M. ; Buonato, Janine M. ; Edwards, Justin P. ; Gupta, Ankit ; LaFleur, David W.

Abstract:

Chimeric antigen receptor (CAR) T-cell therapies directed against B-cell maturation antigen (BCMA) have shown compelling clinical activity and manageable safety in subjects with relapsed and refractory multiple myeloma (RRMM). Prior reported CAR T cells have mostly used antibody fragments such as humanized or murine single-chain variable fragments or camelid heavy-chain antibody fragments as the antigen recognition motif. Herein, we describe the generation and preclinical evaluation of ddBCMA CAR, which uses a novel BCMA binding domain discovered from our D domain phage display libraries and incorporates a 4-1BB costimulatory motif and CD3-zeta T-cell activation domain. Preclinical in vitro studies of ddBCMA CAR T cells cocultured with BCMA-positive cell lines showed highly potent, dose-dependent measures of cytotoxicity, cytokine production, T-cell degranulation, and T-cell proliferation. In each assay, ddBCMA CAR performed as well as the BCMA-directed scFv-based C11D5.3 CAR. Furthermore, ddBCMA CAR T cells demonstrated in vivo tumor suppression in three disseminated BCMA-expressing tumor models in NSG-immunocompromised mice. On the basis of these promising preclinical data, CART-ddBCMA is being studied in a first-in-human phase I clinical study to assess the safety, pharmacokinetics, immunogenicity, efficacy, and duration of effect for patients with RRMM (NCT04155749).

Turkish Journal of Hematology

2025 Update of Cellular Immunotherapy for Plasma Cell Disorders

Review

作者: Beksac, Meral ; Vural, Ece

Despite progression-free survival in multiple myeloma (MM) patients extending to 17 years due to contemporary quadruplet induction therapies, there remains a necessity for novel products in the treatment of high-risk patients. BCMA, GPRC5D, FcRH5, SLAMF7, and TACI are the principal chimeric antigen receptor T (CAR-T) cell target molecules, with dual-target treatments under development to enhance treatment efficacy. Ide-cel and cilta-cel are CAR-T cells directed against BCMA, having received approval from the U.S. Food and Drug Administration for relapsed/refractory MM based on the phase 2 KarMMa and CARTITUDE trials, respectively. Research is currently being conducted on the administration of these products in newly diagnosed patients and for maintenance therapy. Additional anti-BCMA targeted medicines, including LCAR-B38M, completely humanized CAR-T (FHVH-T), P-BCMA-ALLO-1, ALLO-715, and anti-BCMA CAR-NK, provide promising treatment options. Moreover, the anti-CD19 Fast-CAR, designed to shorten production time, and PHE885, which possesses in vivo proliferation capability, are regarded as very efficacious. Arlo-cel, developed for the significant target GPRC5D, has demonstrated efficacy compared to conventional treatments. The development of academic CAR-T products such as ARI0002h, HBI0101, eque-cel, zevor-cel, anito-cel, and Sleeping Beauty (utilizing a non-viral vector) have importance due to their accessibility and cost-effectiveness. Real-world data have demonstrated comparable efficacy and safety outcomes in both academic and commercial CAR-T research. CAR-T cell studies are also being undertaken for smoldering MM and amyloid light-chain (AL) amyloidosis. CAR-PRISMM and CAR-HiRiSMM are regarded as extremely effective and safe therapies for patients with high-risk smoldering MM. NXC-201, which targets BCMA, has been developed for AL amyloidosis. Notwithstanding these promising outcomes, numerous difficulties still confront CAR-T therapy. These factors may be related to the tumor, the patient, and/or the CAR-T product. To overcome these issues, new strategies are being implemented, including combination therapy and the incorporation of gamma-secretase inhibitors. In conclusion, CAR-T treatments have evolved into an effective therapy modality and are anticipated to be utilized in earlier treatment phases in the future. The CRISPR gene editing method contributes to future perspectives.

282

项与阿尼托卡布他基因自体淋巴细胞相关的新闻（医药）

2026-03-24

·FierceBiotech

Gilead\'s $2.2B Ouro buyout delivers autoimmune T-cell engager, new purpose for Galapagos

Gilead Sciences\' chief medical officer said the deal “underscores our commitment to ... serious autoimmune diseases.” \n Gilead Sciences is paying $1.67 billion for a T-cell engager (TCE) company via a front-loaded deal that could also give new meaning to its partner Galapagos.By acquiring GSK-founded Ouro Medicines, Gilead will get its hands on OM336, also known as gamgertamig, a BCMAxCD3 TCE aimed at autoimmune disorders. Ouro has taken OM336 into phase 1/2 studies, which have “demonstrated transformative efficacy and a differentiated safety profile” in conditions like autoimmune hemolytic anemia and immune thrombocytopenia—which both involve the autoimmune destruction of circulating blood cells—after a single treatment cycle, the pharma explained.As well as the $1.675 billion in upfront cash, Gilead has pledged up to $500 million in potential milestone payments for Ouro. The front-loaded nature of the deal means Gilead is handing over a lot of money before OM336 has even proven its worth in a phase 2 test. But the pharma has a plan—to rope in its partner Galapagos to pay half of the upfront and milestone payments.Galapagos’ retreat from cell therapy last year had left the company without a purpose. But CEO Henry Gosebruch explained to Fierce Biotech in November that Gilead—which owns 25% of Galapagos—expected the Belgian biopharma to continue to seek new assets to rebuild the company.Now, Galapagos could have a meaning once again. Gilead said yesterday that it is in advanced discussions for Galapagos to absorb “substantially all of Ouro Medicines’ operating assets and retain its employees.”Under these terms, Galapagos would be responsible for developing OM336 through to the launch of registrational studies, which Gilead hopes to start in 2027. Gilead will retain worldwide commercialization rights—except in China, where Keymed Biosciences already has the license.If OM336 makes it to market, Gilead would pay Galapagos royalties of between 20% and 23% of net sales.The Ouro buyout comes a month after Gilead went big for another cell therapy company, striking a $7.8 billion deal for Arcellx in February. That acquisition brought Gilead full control of anitocabtagene autoleucel, a BCMA-directed CAR-T on the cusp of approval as a direct challenger to Johnson & Johnson and Legend Biotech’s multiple myeloma blockbuster Carvykti.Gilead acquired cell therapy operations through its $11.9 billion Kite Pharma takeover in 2017 and already sells the CD19-directed treatments Tecartus and Yescarta. But the unit has struggled, with Gilead’s cell therapy sales falling 7% to $1.8 billion last year. BMO Capital Markets analysts said in a recent note to investors that the launch of anitocabtagene autoleucel could help Gilead’s cell therapy unit “regain its footing.” What sets yesterday’s deal apart is that Ouro’s TCE is being specifically targeted at autoimmune diseases—a hot area of cell therapy development.Gilead Chief Medical Officer Dietmar Berger, M.D., Ph.D., said buying Ouro “underscores our commitment to advancing transformative therapies for people living with serious autoimmune diseases.”“BCMA is a validated target with emerging data demonstrating potentially transformative outcomes in autoimmune diseases,” Dietmer said. “BCMA targeted T-cell engagers represent a differentiated approach with the potential to induce durable disease control.”“This novel framework complements our expanding inflammation pipeline and reflects our strategy to invest in innovative science that may redefine standards of care,” he added.Galapagos’ CEO Gosebruch also celebrated the chance for “meaningful flexibility in our relationship with Gilead.”“As Galapagos established a relationship with Ouro this past year, we have been impressed with the emerging clinical profile of gamgertamig, its clinically de-risked lead program, and the capability of the Ouro team,” Gosebruch said in a statement. “This is an exciting day in the transformation of Galapagos.”Ouro was launched by GSK and Monograph Capital in January 2025. The biotech arrived on the scene with $120 million in funds and the rights to OM336, which it licensed from China’s Keymed.Ouro’s CEO Jaideep Dudani, Ph.D., a portfolio principal at Monograph, explained at the time that the company’s conviction in OM336 stemmed from the successful off-label use of BCMA-directed TCEs in treating B-cell-mediated diseases.

$Gilead\'s $2.2B Ouro buyout delivers autoimmune T-cell engager, new purpose for Galapagos$

细胞疗法并购临床2期引进/卖出免疫疗法

2026-03-24

·BioSpace

Gilead Chief Gets 20% Pay Bump, Still Falls Short of $30M Club

Gilead Sciences CEO Daniel O’Day (Gilead) Gilead Gilead’s median employee compensation in 2025 was $238,979. Daniel O’Day’s compensation package is 119 times larger. Like his peers, Gilead CEO Daniel O’Day got a raise last year, with his total compensation coming out to $28.44 million, a 20% increase from the previous year. O’Day’s base pay in 2025 was $1.795 million, according to the pharma’s proxy statement released Monday. The bulk of his take-home package came from stock and option awards, which together totaled nearly $19.74 million. The exec also received more than $6.9 million in incentives and other benefits. Gilead’s median employee compensation last year was $238,979, meaning O’Day was paid 119 times more than the median employee, the filing noted. O’Day assumed leadership of Gilead in March 2019 , jumping ship after more than 30 years at Roche , including three at the helm of its pharma unit. Under O’Day, Gilead’s shares have risen some 117% . 2025 turned out to be a good year for Gilead. In June, the pharma won a historic approval for its twice-yearly HIV prevention pill Yeztugo, which BMO Capital Markets in a June 19 note said “marks a major win for Gilead in driving meaningful growth in its PrEP business.” Mizuho Securities was similarly effusive about the approval, saying in a note the same day that Yeztugo “has the potential to ‘redefine’ the PrEP market.” Infectious disease Gilead Eyes HIV Market Domination With Twice-Yearly Yeztugo as GSK Trails Analysts believe that Gilead’s new PrEP drug Yeztugo could reach peak sales of $4.5 billion. Not if GSK has anything to say about it. July 2, 2025 · 4 min read · Nick Paul Taylor Read more In its first few months on the market, Yeztugo made $150 million in sales, including $96 million in the fourth quarter of 2025. Gilead projects the pill to hit $800 million this year—an achievable sales target given the pharma’s direct-to-consumer marketing efforts and the coverage of all major insurance providers, executives said during the company’s 2025 earnings call last month. Outside of HIV, Gilead also strengthened its position in cell therapies in 2025, standing strong even as many of its big pharma peers move away from the modality . In May last year, just weeks before Yeztugo’s approval, Gilead partner Arcellx released mid-stage data for the CAR T therapy anitocabtagene autoleucel (anito-cel), touting a 97% overall response rate when used in patients who had undergone at least three prior lines of treatment. In February of this year, Gilead went on to acquire Arcellx for $7.8 billion . The companies have also filed for anito-cel’s accelerated approval, for which the FDA has a target action date of Dec. 23, 2026. Cell therapy Gilead Gears Up To Challenge J&J in $20B Multiple Myeloma CAR -T Market J&J has a multi-year head start, but Gilead believes it can win market share by delivering a drug with better safety and at least as good efficacy. June 4, 2025 · 5 min read · Nick Paul Taylor Read more Aside from O’Day, CEOs of other top pharmas also saw hefty pay bumps last year. Earlier this month, a proxy statement for AbbVie showed that its chief, Robert Michael, received a 75% raise to bring his total compensation package to $32.5 million. He joins Johnson & Johnson CEO Joaquin Duato, who last year took home $32.76 million, in the $30-million-plus club. Also in this circle is Eli Lilly CEO David Ricks, who in 2025 was paid $36.7 million, according to the pharma’s proxy filing .

上市批准高管变更并购细胞疗法加速审批

2026-03-23

·药事纵横

2026孤儿药报告解读：五款创新疗法如何改写罕见病治疗格局

近日，行业权威机构Evaluate发布《2026年孤儿药报告》（2026 Orphan Drug Report），报告明确指出，尽管全球医药市场环境复杂多变，但孤儿药领域仍保持稳健增长态势，成为医药创新最具活力的赛道之一。报告重点梳理了5款值得关注的在研孤儿药，涵盖RAS抑制剂、CAR-T细胞疗法、双重激酶抑制剂、双特异性抗体等多个前沿领域，这些疗法凭借独特的作用机制、优异的临床数据，有望打破现有治疗瓶颈，改写罕见病及难治性肿瘤的治疗格局。来自参考2一、RAS抑制剂破局胰腺癌，靶向治疗迎来“关键一跃” 在2026年孤儿药报告列出的重点疗法中，Revolution Medicines公司的Daraxonrasib（RMC-6236）尤为引人注目。作为一款口服选择性RAS抑制剂，该药凭借靶向激活状态RAS蛋白的技术突破，打破了RAS突变肿瘤“难靶向、疗效差”的困境，尤其在胰腺导管腺癌（PDAC）治疗中展现出强劲潜力，成为RAS靶向治疗领域的“关键一跃”。 Daraxonrasib的核心技术突破，在于其独特的作用机制——能够精准靶向与GTP结合、处于激活状态的突变型及野生型RAS蛋白。长期以来，RAS突变被认为是“不可成药”靶点，核心原因在于RAS蛋白表面缺乏明确的小分子结合位点，且其与GTP的结合亲和力极强，传统抑制剂难以竞争结合。而Daraxonrasib通过结构优化，实现了对激活态RAS蛋白的特异性结合，能够有效抑制RAS蛋白的信号传导，阻断肿瘤细胞的增殖与存活，从根源上发挥抗肿瘤作用。2025年6月，该药获美国FDA授予突破性疗法认定，用于治疗既往接受治疗、携带KRAS G12突变的转移性胰腺导管腺癌患者，彰显了其临床价值。胰腺导管腺癌（PDAC）是一种恶性程度极高的罕见肿瘤，也是RAS突变的高发领域，其中KRAS G12突变占比极高，患者预后极差，5年生存率不足10%，一线治疗手段有限，临床需求极为迫切。Daraxonrasib在早期临床试验中展现出的优异数据，为PDAC患者带来了新的希望。2025年公布的临床试验结果显示，无论是单药治疗还是联合化疗，该药在一线转移性PDAC患者中均表现出积极疗效。在单药治疗队列中，Daraxonrasib每日一次300 mg给药，40例患者总体耐受性良好；在具有足够随访时长的38例患者中，客观缓解率（ORR）达47%，疾病控制率（DCR）达89%，中位随访时间为9.3个月，多数患者在数据截止时仍在接受治疗，展现出持久的疗效。更值得关注的是其联合治疗方案，Daraxonrasib每日一次200 mg联合吉西他滨和白蛋白紫杉醇（GnP）标准化疗，在40例RAS突变患者中总体安全性良好；在31例随访充分的患者中，ORR达到55%，DCR为90%，中位随访时间为6.9个月，缓解率较单药治疗进一步提升，体现出显著的协同效应。 Daraxonrasib的研发进展，不仅为PDAC患者提供了全新的治疗选择，更对KRAS突变型肿瘤的治疗格局产生深远影响。此前，KRAS突变肿瘤的治疗多依赖化疗、免疫治疗等非特异性手段，疗效有限，而Daraxonrasib作为口服RAS抑制剂，实现了对KRAS突变的精准靶向，且联合化疗能够进一步提升疗效，有望成为KRAS突变型PDAC的一线标准治疗方案。目前，该药正在多项3期临床试验中用于治疗PDAC，同时其用于治疗非小细胞肺癌（NSCLC）的3期临床试验也在推进中，未来有望覆盖更多RAS突变型肿瘤，推动靶向治疗进入“RAS可及”的新时代。二、CAR-T再进阶，anito-cel如何实现疗效与安全性的“双赢” CAR-T细胞疗法自问世以来，彻底改变了多发性骨髓瘤（MM）等血液系统罕见病的治疗格局，但传统CAR-T疗法往往面临疗效与安全性难以平衡的困境——要么缓解率不足，要么易引发严重免疫毒性。而吉利德科学/Arcellx联合开发的Anitocabtagene autoleucel（anito-cel），通过创新的结构设计，实现了疗效与安全性的“双赢”，成为2026年孤儿药报告中的焦点CAR-T疗法。 anito-cel的核心优势，在于其采用了Arcellx开发的创新紧凑结合结构域——D-Domain。与传统CAR-T细胞的结合结构域相比，D-Domain体积更小、稳定性更强，能够在避免持续信号传导的情况下，实现较高水平的嵌合抗原受体（CAR）表达；同时，其被设计为可快速从B细胞成熟抗原（BCMA）靶点解离，这一独特特征能够有效避免CAR-T细胞的过度激活，减少细胞因子释放综合征（CRS）、神经毒性等严重免疫毒性的发生，同时保证对多发性骨髓瘤细胞的有效清除，实现“疗效最大化、毒性最小化”。优异的结构设计，转化为了突出的临床疗效。在2025年12月美国血液学会（ASH）年会上公布的关键性2期临床试验iMMagine-1数据显示，中位随访时间为15.9个月时，接受anito-cel治疗的复发/难治性多发性骨髓瘤患者，总缓解率（ORR）高达96%，完全缓解/严格完全缓解率（CR/sCR）达74%，这一缓解率显著高于现有CAR-T疗法。更重要的是，该药展现出良好的长期生存获益，患者24个月无进展生存率（PFS）为61.7%，24个月总生存率（OS）为83.0%，在复发/难治性MM患者中表现出卓越的持久疗效。 anito-cel的巨大潜力，也吸引了吉利德科学的重金布局。2026年2月，吉利德科学宣布将斥资约78亿美元收购Arcellx公司，核心目的便是将anito-cel纳入自身肿瘤管线，强化其在多发性骨髓瘤治疗领域的竞争力。目前，FDA已受理anito-cel的生物制品许可申请（BLA），用于治疗复发/难治性MM，若顺利获批，将成为一款兼具高疗效与高安全性的CAR-T疗法，改写复发/难治性MM的治疗格局。从行业竞争来看，anito-cel的优势在于其差异化的结构设计的带来的安全性优势——相较于现有CAR-T疗法，其免疫毒性发生率更低，患者耐受性更好，更适合用于多线治疗失败的复发/难治性MM患者。随着吉利德科学的收购与推广，anito-cel有望快速抢占市场份额，同时推动CAR-T疗法的技术升级，引领血液系统罕见病治疗向“高效、安全”的方向发展。三、TYK2/JAK1双重抑制，brepocitinib能否终结皮肌炎“无药可用”困境皮肌炎（DM）是一种罕见的自身免疫性疾病，以皮肤和肌肉炎症为主要特征，患者常表现为肌肉无力、皮肤红斑、关节疼痛等症状，严重时可累及内脏器官，甚至危及生命。长期以来，皮肌炎缺乏针对性的靶向治疗药物，临床治疗主要依赖糖皮质激素、免疫抑制剂等非特异性手段，疗效有限且不良反应较多，患者面临“无药可用”的困境。而Priovant Therapeutics公司的Brepocitinib，作为一款选择性TYK2和JAK1双重抑制剂，有望终结这一困境，成为首个获批用于治疗皮肌炎的靶向疗法。 Brepocitinib的核心作用机制，在于其能够双重抑制TYK2和JAK1激酶，进而阻断与自身免疫相关的多重关键细胞因子的信号传导，包括I型IFN、II型IFN、IL-6、IL-12和IL-23等。这些细胞因子在皮肌炎的发病机制中发挥关键作用，过度激活会导致皮肤和肌肉的异常炎症反应。通过双重抑制TYK2/JAK1，brepocitinib能够从源头抑制炎症反应，缓解患者症状，且相较于单一JAK抑制剂，其作用更广泛、更精准，能够减少单一靶点抑制带来的不良反应。该药的3期VALOR临床试验数据，充分印证了其在皮肌炎治疗中的优异疗效。VALOR是一项全球多中心3期临床试验，241例皮肌炎患者按1：1：1比例随机分配至brepocitinib 30 mg组、15 mg组或安慰剂组。研究结果显示，30 mg剂量组在第52周时，主要终点肌炎总改善评分（TIS）相较安慰剂达到统计学显著且具有临床意义的改善——TIS作为涵盖6项疾病活动指标的综合终点，能够全面评估患者的皮肤、肌肉受累情况，其改善意味着患者整体病情的缓解。更值得关注的是，brepocitinib的治疗获益具有“快速、持久”的特点，最早在第4周即已显现，且在整个为期一年的双盲治疗期间持续保持。除主要终点外，30 mg剂量组在全部9项关键次要终点上同样优于安慰剂，包括皮肤受累、肌肉受累及糖皮质激素减量等指标，其中超过三分之二接受30 mg治疗的患者达到TIS40标准（即TIS≥40分），这一改善幅度为最小临床重要差异的两倍；更有超过一半患者在达到TIS40的同时，将类固醇依赖剂量降低至每日≤2.5 mg，有效减少了长期使用糖皮质激素带来的不良反应。目前，美国FDA已受理brepocitinib用于治疗皮肌炎的新药申请（NDA），并授予优先审评资格，若顺利获批，将成为首个获批用于治疗皮肌炎的靶向疗法，彻底填补该领域的治疗空白。此外，brepocitinib的适应症布局正在持续拓展，目前还在3期临床试验中用于治疗非感染性葡萄膜炎（NIU），治疗皮肤结节病的2期临床试验也在进行中，未来有望覆盖更多自身免疫性罕见病，成为自身免疫领域的“重磅疗法”。四、双特异性抗体再发力，etentamig组合疗法重塑MM治疗边界双特异性抗体作为近年来的研发热点，凭借其能够同时结合两个靶点、精准杀伤肿瘤细胞的优势，在多发性骨髓瘤（MM）等罕见血液肿瘤治疗中展现出巨大潜力。艾伯维公司的Etentamig，作为一款靶向BCMA和CD3的双特异性T细胞衔接器，通过与泊马度胺和地塞米松（POM+DEX）的联合策略，在多线治疗失败的复发/难治性MM患者中展现出显著疗效，有望重塑MM的治疗边界。 Etentamig的作用机制具有高度特异性：其一端结合多发性骨髓瘤细胞表面的BCMA抗原，另一端结合T细胞表面的CD3分子，通过“桥梁作用”将T细胞招募至肿瘤细胞周围，激活T细胞的杀伤功能，从而特异性清除MM细胞。与传统抗体药物相比，双特异性T细胞衔接器能够直接激活内源性T细胞，无需体外培养，给药更便捷，且对肿瘤细胞的杀伤效率更高，尤其适合用于复发/难治性患者。考虑到复发/难治性MM患者多经过多线治疗，肿瘤细胞易产生耐药性，艾伯维采用了“etentamig+POM+DEX”的联合治疗策略，通过机制协同提升疗效。2025年12月ASH年会上公布的1b期临床试验结果显示，该联合疗法在既往接受过多种治疗（至少3线既往治疗）的复发/难治性MM患者中表现出显著疗效：来自剂量递增阶段（n=82）的初步数据显示，中位随访时间为23个月时，联合治疗的总缓解率（ORR）达81%，达到非常好部分缓解或更佳（≥VGPR）的患者比例为72%，在多线治疗失败的患者群体中，这一数据尤为亮眼。更重要的是，该联合疗法展现出持久的缓解趋势，截至数据分析时，缓解持续时间（DoR）尚未达到，意味着多数患者能够长期获益，有效延长了患者的生存期。此外，该联合疗法的安全性良好，未出现不可耐受的严重不良反应，患者耐受性较好，适合长期治疗。艾伯维预计在2026年获得etentamig组合疗法治疗复发/难治性MM患者的3期临床试验结果，并计划在2027年递交该疗法的监管申请。若顺利获批，etentamig组合疗法将为多线治疗失败的复发/难治性MM患者提供全新的治疗选择，打破现有治疗瓶颈，重塑MM的治疗格局。从市场潜力来看，复发/难治性MM患者群体庞大，且现有治疗手段有限，etentamig组合疗法凭借优异的疗效，有望成为该领域的核心治疗方案，具备广阔的市场前景。五、从SM到GIST，bezuclastinib如何打通KIT突变“双通道” 系统性肥大细胞增多症（SM）和胃肠道间质瘤（GIST）均为与KIT基因突变相关的罕见疾病，其中KIT D816V突变是SM的关键驱动因素，而KIT第17外显子突变也常见于晚期GIST患者。长期以来，针对这些突变的靶向治疗药物有限，患者疗效不佳、生存期较短。Cogent Biosciences公司的Bezuclastinib，作为一种选择性酪氨酸激酶抑制剂（TKI），能够强效抑制KIT D816V及第17外显子的其他突变，成功打通SM与GIST治疗的“双通道”，成为该领域的突破性疗法。 Bezuclastinib的核心优势在于其高度的选择性——专门针对KIT D816V突变以及KIT第17外显子的其他突变，对正常细胞的KIT激酶影响较小，因此不良反应发生率更低，患者耐受性更好。与传统TKI药物相比，bezuclastinib对KIT突变的抑制活性更强，能够更精准地阻断肿瘤细胞的信号传导，从而实现更好的治疗效果。在系统性肥大细胞增多症（SM）领域，bezuclastinib已取得重要突破。Cogent Biosciences已于2025年12月向美国FDA递交新药申请，寻求批准bezuclastinib用于治疗非进展期系统性肥大细胞增多症，为这类患者提供了针对性的靶向治疗选择。而在胃肠道间质瘤（GIST）领域，该药的表现更为亮眼：2026年1月，bezuclastinib获得FDA授予的突破性疗法认定，与sunitinib联用，治疗既往接受过imatinib治疗的GIST患者。这一突破性疗法认定的授予，主要基于PEAK试验的研究结果。该试验在主要终点无进展生存期（PFS）方面显示出显著且具有高度统计学意义的临床获益：与当前标准治疗相比，bezuclastinib联合治疗组的疾病进展或死亡风险降低了50%（HR=0.50，95% CI：0.39–0.65）；经盲法独立中心评估（BICR）判定，联合治疗组的中位无进展生存期为16.5个月，而活性对照组仅为9.2个月，疗效提升显著，为既往接受过imatinib治疗的GIST患者带来了新的希望。 Bezuclastinib的研发进展，不仅填补了SM与GIST领域KIT第17外显子突变靶向治疗的空白，更体现了精准治疗时代下“突变导向”的治疗理念。Cogent Biosciences公司表示，预计在2026年4月完成递交bezuclastinib用于治疗GIST的NDA，若顺利获批，将成为首个专门针对KIT第17外显子突变的靶向疗法，打通SM与GIST治疗的“双通道”，为两类罕见病患者提供精准、高效的治疗选择，推动相关领域的治疗进入“精准靶向”新时代。结语：五款在研疗法，五个不同的技术路径，却共同指向同一个方向——为罕见病患者带来更具针对性、更安全的治疗选择。在政策鼓励、技术突破与资本加持的多重推动下，孤儿药正从“边缘”走向“主流”。未来，我们有理由相信，更多曾被忽视的疾病将迎来属于它们的“高光时刻”。参考资料： [1] Evaluate Releases 2026 Orphan Drug Report. Retrieved March 13, 2026, from https://www.evaluate.com/press-release/2026-orphan-drug-report/ [2] Orphan Drugs Outlook 2032: Sales, Pipeline & Policy for Rare Diseases. Retrieved March 13, 2026, from https://www.evaluate.com/thought-leadership/orphan-drugs-outlook-2032/ [3] AbbVie to Feature New Data at ASH 2025 Showcasing Continued Advances Across Novel Treatment Modalities in Multiple Blood Cancers. Retrieved March 13, 2026, from https://news.abbvie.com/2025-12-02-AbbVie-to-Feature-New-Data-at-ASH-2025-Showcasing-Continued-Advances-Across-Novel-Treatment-Modalities-in-Multiple-Blood-Cancers [4] Cogent Biosciences Highlights Additional Data with Six Bezuclastinib Posters from SUMMIT Trial at 2026 AAAAI Annual Meeting. Retrieved March 13, 2026, from https://investors.cogentbio.com/news-releases/news-release-details/cogent-biosciences-highlights-additional-data-six-bezuclastinib [5] Cogent Biosciences to Initiate New Drug Application (NDA) Submission for Bezuclastinib Under Real-Time Oncology Review (RTOR). Retrieved March 13, 2026, from https://investors.cogentbio.com/news-releases/news-release-details/cogent-biosciences-initiate-new-drug-application-nda-submission 立即扫码加入药事纵横交流群

100 项与阿尼托卡布他基因自体淋巴细胞相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
难治性多发性骨髓瘤	申请上市	美国	Gilead Sciences, Inc.	2026-02-23
复发性多发性骨髓瘤	申请上市	美国	Gilead Sciences, Inc.	2026-02-23
多发性骨髓瘤	临床3期	美国	Arcellx, Inc.	2024-08-23
多发性骨髓瘤	临床3期	美国	Kite Pharma, Inc.	2024-08-23
多发性骨髓瘤	临床3期	日本	Kite Pharma, Inc.	2024-08-23
多发性骨髓瘤	临床3期	日本	Arcellx, Inc.	2024-08-23
多发性骨髓瘤	临床3期	澳大利亚	Arcellx, Inc.	2024-08-23
多发性骨髓瘤	临床3期	澳大利亚	Kite Pharma, Inc.	2024-08-23
多发性骨髓瘤	临床3期	奥地利	Kite Pharma, Inc.	2024-08-23
多发性骨髓瘤	临床3期	奥地利	Arcellx, Inc.	2024-08-23

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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


NCT05396885 (iMMagine-1, ASH2025 \| NEWS 1 \| NEWS 2) 人工标引	临床2期	复发性多发性骨髓瘤 \| 难治性多发性骨髓瘤	117	Anitocabtagene autoleucel	選壓廠餘衊獵衊膚選憲(網膚鏇顧築選鏇鏇鹽襯) = 醖觸顧齋築鑰網顧鹹窪淵鹹廠壓繭餘衊選願觸 (鬱壓蓋鬱積範積鑰淵網 ) 更多	积极	2025-12-06
NCT05396885 (iMMagine-1, EHA2025)	临床2期	复发性多发性骨髓瘤 BCMA	86	Anitocabtagene autoleucel (anito-cel)	壓獵糧鹽遞鑰鑰憲壓範(觸簾網鑰鑰餘淵鑰憲憲) = 獵壓簾構蓋廠膚構夢鬱窪築廠遞積鹽願選淵製 (憲觸網構製艱選築鑰鏇 ) 更多	积极	2025-05-14
NCT04155749 (ASH2024) 人工标引	临床1期	多发性骨髓瘤	38	Anitocabtagene autoleucel (anito-cel)	壓艱窪襯願鑰醖遞積鬱(顧蓋鬱顧鬱選鬱顧獵壓) = 範鹹鏇膚獵遞蓋餘鹹鏇鬱餘鬱夢齋選襯壓選淵 (願簾窪蓋鬱遞齋選餘廠 ) 更多	积极	2024-12-09
NCT05396885 (iMMagine-1, ASH2024) 人工标引	临床2期	多发性骨髓瘤末线	58	Anitocabtagene autoleucel	願夢繭齋壓糧夢築簾製(選齋糧衊餘醖觸鹹鏇遞) = 選鹹繭願簾淵鑰遞鹽襯壓醖膚鹹鹹選齋獵廠淵 (餘獵願鑰網襯鹽鏇糧製 ) 更多	积极	2024-12-09
NCT05396885 (iMMagine-1, PipelineReview) 人工标引	临床2期	多发性骨髓瘤	98	anitocabtagene autoleucel	繭憲夢鑰衊淵衊艱齋鹽(遞鑰顧艱醖膚窪製淵簾) = 齋餘觸憲衊餘網醖範窪壓餘襯餘網選廠鏇餘繭 (鏇範壓憲範築膚網鬱製 ) 更多	积极	2024-12-09
NCT04155749 (IMS2024 \| NEWS) 人工标引	临床1期	多发性骨髓瘤	38	anitocabtagene autoleucel	選淵鬱醖製簾築蓋鏇願(窪鹹廠網醖鹽獵艱齋齋) = One grade 5 adverse event (AE) of unrelated cardiac arrest attributed to a non-study drug overdose occurred post study. The most common grade 3/4 hematologic AEs (n = 38) that were not attributable to cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome were decreased neutrophil count (81.6%), anemia (57.9%), and thrombocytopenia (42.1%). Among non-hematologic AEs, the most common were hypertension (7.9%), increased aspartate aminotransferase levels (5.3%), and cellulitis (5.3%). 構鹹選鑰壓衊憲選簾構 (構廠顧廠鹹鏇積壓蓋繭 )	积极	2024-09-24
NCT04155749 (IMS2024 \| NEWS) 人工标引	临床1期	多发性骨髓瘤	38	anitocabtagene autoleucel (patients with extramedullary disease)		积极	2024-09-24
NCT06413498 (iMMagine-3, SOHO2024)	临床3期	多发性骨髓瘤	-	Anito-cel	蓋衊糧襯鑰簾願積願簾(鹹觸顧襯淵夢鏇積襯廠) = 壓鏇網製蓋壓簾夢夢鏇選獵願鑰衊簾夢衊襯獵 (製窪蓋獵夢選鬱願築夢 ) 更多	积极	2024-09-04
NCT04155749 (EHA 12024 \| EHA 22024) 人工标引	临床1期	多发性骨髓瘤末线	40	Anitocabtagene autoleucel (anito-cel)	鑰夢夢鏇窪鹽餘鑰鹽簾(夢鏇餘願選鏇構獵廠簾) = 築壓廠淵襯積醖網廠膚鏇糧衊夢網積築衊衊糧 (構遞構範夢淵製窪廠選 ) 更多	积极	2024-05-14
NCT04155749 (ASH2023) 人工标引	临床1期	多发性骨髓瘤末线	38	CART-ddBCMA	壓淵製範網壓廠鏇鏇艱(鑰淵蓋憲選鬱願廠窪製) = 製築淵蓋衊網築夢願鬱蓋獵衊淵顧獵積鑰觸齋 (範襯選築壓醖壓襯艱憲 ) 更多	积极	2023-12-11
NCT04155749 (PRNewswire) 人工标引	临床1期	多发性骨髓瘤	38	CART-ddBCMA	選鹽艱繭蓋觸範蓋壓網(廠築鬱襯遞獵製簾築積) = 願獵鹽鏇鏇餘觸積醖淵顧餘壓觸鑰艱製鑰醖築 (窪願網衊鹽蓋齋繭築鏇 ) 更多	积极	2023-12-08