三特异性NK细胞衔接器、创新JAK2变构抑制剂获FDA批准进入临床…… | 一周盘点

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。