本期看点： 1. GT Biopharma公司宣布，其新型B7-H3靶向三特异性自然杀伤（NK）细胞衔接器GTB-5550的IND申请已获得美国FDA批准，将在7种不同的转移性癌症队列中开展研究。 2. Prelude Therapeutics公司宣布，其新型JAK2V617F突变选择性抑制剂PRT12396的IND申请已获美国FDA批准，1期临床试验将在高危真红细胞增多症（PV）以及中高危骨髓纤维化（MF）患者中开展。 GTB-5550：IND申请获得FDA许可 GT Biopharma公司宣布，其新型候选药物GTB-5550的IND申请已获得美国FDA批准。GTB-5550是一种靶向B7-H3的三特异性NK细胞衔接器，用于治疗表达B7-H3抗原的实体肿瘤。该分子由三种组分组成，通过柔性接头连接：1）靶向NK细胞表面CD16激活受体的纳米体臂；2）野生型IL-15（WT IL-15）连接臂，用于促进NK细胞的增殖、活化和存活；3）靶向肿瘤细胞上B7-H3抗原的纳米体臂，从而将NK细胞精准引导至肿瘤部位。计划开展的1期临床试验共分为两个部分：1a期为剂量递增阶段，将评估最多6个剂量水平以确定最大耐受剂量（MTD）；1b期为剂量扩展阶段，将在7种不同的转移性癌症队列中（包括去势抵抗性前列腺癌、卵巢癌、乳腺癌、头颈癌、非小细胞肺癌、胰腺癌和膀胱癌）验证1a期研究确定的MTD，并进一步评估药物的耐受性。 PRT12396：IND申请获得FDA许可 Prelude Therapeutics公司宣布，其新型JAK2V617F突变选择性抑制剂PRT12396的IND申请已获美国FDA批准，将启动一项1期临床研究。该研究为开放标签、多中心试验，旨在评估PRT12396在高危真红细胞增多症以及中高危骨髓纤维化患者中的安全性、初步疗效和药代动力学特征。公司预计将于2026年第二季度完成首例患者给药。 PRT12396是Prelude公司设计并验证的新型变构抑制剂，能够结合到V617F突变所在的JAK2 JH2“深口袋”。该突变是骨髓增殖性肿瘤（MPNs）的主要驱动突变，在约95%的PV、60%的原发性血小板增多症（ET）和55%的MF患者中存在。Prelude公司开发的这类变构抑制剂在MPN临床前模型中展现了突变特异性抑制作用，有望降低突变等位基因负荷、延缓甚至逆转疾病进展，从而改善MPN患者的治疗结果。 AMXT 1501：启动1b/2期联合治疗试验 Aminex Therapeutics公司宣布启动一项AMXT 1501联合二氟甲基鸟氨酸（DFMO）的1b/2期临床试验。这项多中心、开放标签研究将评估口服AMXT 1501与口服DFMO联合标准治疗在两类人群中的安全性、耐受性和初步疗效，包括既往治疗失败、绝经前/后、雌激素受体（ER）阳性、人表皮生长因子受体2（HER2）阴性乳腺癌和转移性黑色素瘤患者。 AMXT 1501是一种新型多胺转运抑制剂，旨在阻断肿瘤细胞摄取外源性多胺——这些分子对肿瘤生长和存活至关重要。DFMO是一种已知的多胺生物合成抑制剂。两者联合旨在全面抑制多胺代谢，从而抑制肿瘤生长。参考资料： [1] Onchilles Pharma Announces IND Clearance for N17350, Advancing the First Next-Generation Cytotoxic Therapeutic Leveraging the ELANE Pathway into the Clinic. Retrieved February 6, 2026, from https://www.onchillespharma.com/post/onchilles-pharma-announces-ind-clearance-for-n17350 [2] GT Biopharma Announces FDA Clearance of Investigational New Drug (IND) Application for GTB-5550 TriKE®, a B7-H3-Targeted Natural Killer (NK) Cell Engager for Solid Tumors Expressing B7-H3. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/03/3231077/0/en/GT-Biopharma-Announces-FDA-Clearance-of-Investigational-New-Drug-IND-Application-for-GTB-5550-TriKE-a-B7-H3-Targeted-Natural-Killer-NK-Cell-Engager-for-Solid-Tumors-Expressing-B7-H.html [3] Prelude Therapeutics Receives FDA Clearance of Investigational New Drug Application (IND) for PRT12396, a Mutant-selective JAK2V617F Inhibitor. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/03/3231175/0/en/Prelude-Therapeutics-Receives-FDA-Clearance-of-Investigational-New-Drug-Application-IND-for-PRT12396-a-Mutant-selective-JAK2V617F-Inhibitor.html [4] Affinia Therapeutics Announces FDA Acceptance of IND Application to Advance AFTX-201 to a Phase 1/2 Trial for the Treatment of BAG3-Associated Dilated Cardiomyopathy (DCM). Retrieved February 6, 2026, from https://affiniatx.com/affinia-therapeutics-announces-fda-acceptance-of-ind-application-to-advance-aftx-201-to-a-phase-1-2-trial-for-the-treatment-of-bag3-associated-dilated-cardiomyopathy-dcm/ [5] PharmaResearch Receives U.S. FDA Clearance to Initiate Phase 1 Clinical Trial of Nano Anticancer Drug, PRD-101. Retrieved February 6, 2026, from https://www.prnewswire.com/news-releases/pharmaresearch-receives-us-fda-clearance-to-initiate-phase-1-clinical-trial-of-nano-anticancer-drug-prd-101-302681109.html [6] Spur Therapeutics Presents New Phase 1/2 Data on Its Gene Therapy Candidate in Gaucher Disease at 22nd Annual WORLDSymposium™. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/04/3231939/0/en/Spur-Therapeutics-Presents-New-Phase-1-2-Data-on-Its-Gene-Therapy-Candidate-in-Gaucher-Disease-at-22nd-Annual-WORLDSymposium.html [7] Aminex Therapeutics Announces Multiple Sites Activated for Phase 1b/2 Clinical Trial of Novel Investigational Cancer Treatment AMXT 1501 and DFMO in Patients with Breast Cancer or Melanoma. Retrieved February 6, 2026, from https://www.prnewswire.com/news-releases/aminex-therapeutics-announces-multiple-sites-activated-for-phase-1b2-clinical-trial-of-novel-investigational-cancer-treatment-amxt-1501-and-dfmo-in-patients-with-breast-cancer-or-melanoma-302678969.html [8] Neomorph Announces First Patient Dosed in Phase 1/2 Trial Evaluating NEO-811 For the Treatment of Locally Advanced or Metastatic Non-Resectable Clear Cell Renal Cell Carcinoma. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/03/3231475/0/en/Neomorph-Announces-First-Patient-Dosed-in-Phase-1-2-Trial-Evaluating-NEO-811-For-the-Treatment-of-Locally-Advanced-or-Metastatic-Non-Resectable-Clear-Cell-Renal-Cell-Carcinoma.html [9] Ultragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome (MPS IIIA). Retrieved February 6, 2026, from https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-positive-longer-term-data-demonstrating [10] uniQure Announces Updated Preliminary AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/06/3233740/0/en/uniQure-Announces-Updated-Preliminary-AMT-191-Phase-I-IIa-Data-Showing-Sustained-Increases-in-%CE%B1-Gal-A-Enzyme-Activity-in-Patients-with-Fabry-Disease.html 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

2026-02-06

·微信

从“无药可用”到“73.7%的疾病控制”，NK细胞如何成为抗击胰腺癌的新“闪电部队”？

当晚期胰腺癌患者王磊的主治医生首次在病历上写下“建议尝试NK细胞联合疗法”时，一种曾被视作“科幻”的治疗方案，正依托清晰的国家政策路径，从实验室加速走向临床。 2025年，随着国务院《生物医学新技术临床研究和临床转化应用管理条例》的正式发布，中国为细胞治疗等前沿生物医学技术构建了从临床研究到转化应用的制度化高速路。该条例确立的“临床研究备案制”与“临床转化应用审批制”双轨体系，为如NK细胞疗法这类前沿探索松绑提速，同时将实施主体严格限定在三级甲等医院，确保了创新与风险控制的平衡。与此同时，国家药监局在“十五五”规划中明确将细胞与基因治疗列为重点支持的前沿领域，科技部发布的《创新药物研发国家科技重大专项2026年度项目申报指南》亦将胰腺癌等实体瘤的靶向治疗列为攻关方向。政策的强力驱动与临床的迫切需求在此交汇，将自然杀伤细胞疗法，特别是“现货型”疗法，推向了对抗“癌王”——胰腺癌的舞台中央。顶层设计：双轨并行的中国监管新纪元中国生物医药创新的浪潮正奔涌向前。2025年，中国共有76个创新药获批上市，审评审批标准与国际深度对接。在这一宏大背景下，针对细胞治疗等颠覆性技术的监管框架也完成了关键性重构。 2025年10月，国务院颁布的《生物医学新技术临床研究和临床转化应用管理条例》成为里程碑事件。这部被业界称为 “818号文” 的条例，首次在国家行政法规层面为包括NK细胞疗法在内的生物医学新技术，规划了一条有别于传统药品审批的清晰路径。条例的核心在于建立了 “临床研究备案制”与“临床转化应用审批制” 的双轨体系。这意味着，一项有前景的NK细胞新技术，在完成严谨的非临床研究后，可在符合资质的三甲医院通过学术与伦理审查后快速启动临床研究，极大加速了前沿探索的步伐。这一政策的深意在于鼓励创新与风险控制并举。它既为技术转化“松绑”，例如为治疗危重疾病的技术开辟优先审查绿色通道；同时也“筑牢底线”，明确仅限三级甲等医院等机构作为实施主体，确保研究的规范与安全。精准导航：国家战略与科研专项的强力聚焦政策东风不仅吹拂在监管层面，更体现在国家级的科研战略布局中。胰腺癌作为恶性程度最高、治疗手段最匮乏的实体瘤之一，其破局已被提升至国家科技攻关的层面。在科技部发布的《创新药物研发国家科技重大专项2026年度项目申报指南》中，“靶向治疗胰腺癌的化学创新药物研发”被列为明确的指南方向。这份由国家卫生健康委和科技部联合组织的权威指南，其导向意义重大。它如同一个精准的导航信号，引导全国的科研力量、资金与人才向胰腺癌治疗这一高地集结。虽然指南直接指向化学药物，但其对“实体肿瘤免疫治疗”的整体关注，无疑为包括NK细胞免疫疗法在内的所有创新模式提供了肥沃的土壤和协同发展的机遇。从国家药监局“密切关注前沿技术”的表态，到具体重大专项的指南设置，一条自上而下支持突破性疗法研发的链条已然清晰。这标志着，针对胰腺癌等未满足临床需求的攻坚，已从科学家和企业的自发探索，升级为有组织、有体系的国家创新行动。临床竞速：NK细胞“现货疗法”的中国突破在明确的政策预期下，中国NK细胞疗法的临床研发进入了爆发期。尤其令人瞩目的是，“现货型”异体NK细胞疗法因其“即取即用、可批量生产”的颠覆性优势，已成为国内创新药企攻坚实体瘤的主流方向。截至2025年底，我国已有16个NK细胞药物产品获得34个临床试验批件，其中异体来源产品占比显著。例如，由美欧赛奥金研发的ZMPB-NK006注射液，是国内首个面向实体瘤的“现货型”NK细胞产品，采用健康供体来源，无需基因改造，并且在胰腺癌中取得的积极信号，为NK细胞疗法应用于免疫抑制性更强的胰腺癌积累了宝贵经验，预示着在胰腺癌领域取得突破。未来之战：联合策略与胰腺癌微环境破译与胰腺癌的“未来之战”蓝图已经绘就。核心策略在于利用NK细胞的天然安全性，将其与现有疗法深度联合，形成协同攻击。目前，最前沿的探索已不局限于简单的细胞回输，而是通过基因工程对NK细胞进行“武装升级”。例如，通过敲除BCL11B等基因，可直接将外周血细胞重编程为攻击力更强的NK细胞，并使其高表达NKG2D等靶向分子。更具想象力的策略是开发“细胞衔接器”。例如，美国GT Biopharma公司最新获FDA临床试验许可的GTB-5550，就是一种双纳米抗体TriKE®衔接器，它能同时结合NK细胞上的激活受体和肿瘤细胞上的B7-H3抗原，将NK细胞精准“桥接”并激活至肿瘤细胞身边，其中胰腺癌正是其重点关注的适应症之一。另一方面，研究正深入胰腺癌内部，破解其抵御免疫细胞的“魔法盾”。最新研究发现，胰腺癌细胞会创造一种维生素B6缺乏的局部微环境，直接导致浸润的NK细胞“失活”。这一发现提示，未来通过营养干预或代谢调节药物与NK细胞疗法联用，有望解除肿瘤的免疫抑制，让疗法事半功倍。从国家层面的战略布局与法规护航，到企业与科研机构的快速临床推进，中国在NK细胞“现货疗法”治疗胰腺癌的征程上，已构建了从基础研究到临床转化的完整创新生态。这场对抗“癌王”的战役，正以前所未有的系统性和节奏加速进行。本文所有内容均不构成任何医疗指导。如有需求请通过正规就医渠道及时就医。如果您有任何需求，可以在留言区留言，也可以扫码咨询。扫码下方二维码专家免费解读报告

细胞疗法优先审批临床研究申请上市ASH会议

2026-02-04

·GlobeNewswire-Health Care

GT Biopharma Announces FDA Clearance of Investigational New Drug (IND) Application for GTB-5550 TriKE®, a B7-H3-Targeted Natural Killer (NK) Cell Engager for Solid Tumors Expressing B7-H3

GTB-5550 Phase 1 dose escalation basket trial expected to initiate mid-2026 Phase 1 protocol allows multiple solid tumor types known to express B7-H3 Unaudited proforma cash balance as of January 31, 2026 of approximately $9 million anticipated to extend cash runway through Q4 2026 Feb. 03, 2026 -- GT Biopharma, Inc. (the “Company”) (NASDAQ: GTBP), a clinical stage immuno-oncology company focused on developing innovative therapeutics based on the Company's proprietary TriKE® natural killer (NK) cell engager platform, today announced FDA clearance of its IND application for GTB-5550, allowing the company to proceed with a Phase 1 clinical trial, which is anticipated to initiate in mid-2026. “FDA clearance of our third TriKE® IND, GTB-5550, represents a defining moment for GT Biopharma as we bring another NK cell engager into the clinic”, said Michael Breen, Executive Chairman and Chief Executive Officer of GT Biopharma. “We expect to commence enrollment of the Phase 1 basket trial in mid-2026. While the phase I trial is open to patients with common solid tumors that express B7-H3, in the dose-escalation component we will prioritize enrollment for advanced prostate, ovarian, and pancreatic cancer patients who have failed standard therapies. Based on the encouraging trends we have seen from our ongoing Phase 1 trial with GTB-3650 in AML patients, we are even more enthusiastic about the potential benefits of GTB-5550 treatment in patients with solid tumors known to express B7-H3.” GTB-5550 is a camelid (cam) anti-CD16/WT IL-15/cam anti-B7-H3 tri-specific natural killer (TriKE) cell engager, with a single chain recombinant TriKE® comprised of three components joined by flexible linkers: 1) a nanobody arm that engages the CD16 activating receptor (camelid anti-CD16) on natural killer (NK) cells; 2) a wildtype IL-15 (WT IL-15) linker arm to drive NK cell proliferation, priming, and survival; and 3) a nanobody arm that specifically engages B7-H3 (camelid anti-B7-H3) to target the antigen expressed on tumor cells. “This clearance is an important step toward developing new immune-based therapies for patients with advanced prostate cancer and other solid tumor types”, said Dr. Emmanuel Antonarakis, MD, Associate Director, Translational Research at the University of Minnesota Masonic Cancer Center1. “Given the high expression of B7-H3 in over 90% of metastatic castration-resistant prostate cancers and the unmet need in the patient population, I look forward to evaluating GTB-5550’s TriKE® approach in the upcoming Phase 1 trial. As part of our team, I am excited that Dr. Nicholas Zorko will be leading this clinical trial. He is emerging as a national leader in early phase immune engager clinical trials across oncology.” The Phase 1 trial with GTB-5550 will be the first dual nanobody TriKE® tested with more patient-friendly subcutaneous dosing. The Phase 1a dose escalation portion of the trial will test up to 6 dose levels to identify the maximum tolerated dose (MTD). After the dose escalation phase , the Phase 1b expansion component of the trial will then confirm the MTD identified in the Phase 1a trial in up to 7 different possible metastatic disease cohorts (castration-resistant prostate cancer, ovarian cancer, breast cancer, head and neck cancer, non-small cell lung cancer, pancreatic cancer, and bladder cancer) and further evaluate its safety, tolerability and preliminary anti-tumor activity. GTB-5550 will be administered by subcutaneous (SQ) injection in the abdominal area for 5 consecutive days during Week 1 and Week 2 followed by 2 weeks of no treatment. One treatment cycle is 4 weeks in duration. A minimum of 2 cycles is planned, and patient-appropriate disease reassessment is performed after 2 cycles and every 8-12 weeks thereafter. Treatment may continue until disease progression, unacceptable toxicity, patient refusal, or treatment is no longer in the best interest of the patient. Patients are followed for 12 months to determine progression free survival (PFS) and overall survival (OS). GT Biopharma, Inc. is a clinical stage biopharmaceutical company focused on the development and commercialization of immuno-oncology therapeutic products based on our proprietary TriKE® NK cell engager platform. Our TriKE® platform is designed to harness and enhance the cancer killing abilities of a patient’s immune system’s natural killer cells. GT Biopharma has an exclusive worldwide license agreement with the University of Minnesota to further develop and commercialize therapies using TriKE® technology. 1 The University of Minnesota, pursuant to its license agreement with GT Biopharma, is entitled to receive royalties should commercial sales of product using the TriKE technology be realized, including GTB-3650 and GTB-5550. This interest has been reviewed and managed by the University of Minnesota in accordance with its conflict of interest policies. The content above comes from the network. if any infringement, please contact us to modify.

引进/卖出免疫疗法临床申请

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适应症	最高研发状态	国家/地区	公司	日期
实体瘤	临床申请批准	美国	GT Biopharma, Inc.	2026-02-03
头颈部肿瘤	临床前	美国	Xcell Biosciences, Inc.	2024-03-22
头颈部肿瘤	临床前	美国	University of Minnesota	2024-03-22
前列腺癌	临床前	美国	GT Biopharma, Inc.	2023-11-06
头颈部鳞状细胞癌	临床前	美国	GT Biopharma, Inc.	2022-04-11
多发性骨髓瘤	临床前	美国	GT Biopharma, Inc.	2022-03-21