可穿越血脑屏障，创新NLRP3靶向小分子获积极进展；三特异性NK细胞衔接器在美国递交IND申请…… | 一周盘点

本期看点：

1. 具 良好 脑渗透性 的小分子NLRP 3抑制剂 BGE-102公布了 1期临床试验的新数据，给药 14天后， 具有心血管风险因素的肥胖受试者的关键炎症标志物水平下降86%，93%达到了正常水平。

2. GT Biopharma公司宣 布已向美国FDA提交了其新 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。

BGE-102：公布1期临床试验的新数据

BioAge公司公布了其候选药物BGE-102的1期临床试验的新数据。BGE-102是一种结构新颖、可口服的小分子NLRP3抑制剂，具有高活性及良好的脑渗透性，旨在用于治疗具有心血管风险因素的患者。NLRP3是驱动与年龄相关炎症的关键因子，与肥胖、神经退行性疾病及心血管疾病密切相关。该公司基于人类衰老队列分析发现，NLRP3活性降低与长寿相关，因此将其确立为潜在治疗靶点。BGE-102通过独特的结合位点和机制抑制NLRP3炎性小体，作用方式区别于其他在研NLRP3抑制剂。

在一项针对肥胖（BMI 32–42）且基线高敏C反应蛋白（hsCRP）升高（>3 mg/L）的受试者开展的多剂量递增（MAD）研究中，每日接受120 mg的BGE-102治疗受试者的炎症标志物显示出迅速且显著的减少。第14天时，受试者的中位hsCRP水平下降86%，93%（13/14）达到了正常水平（<2 mg/L）。此外，BGE-102还显著降低了IL-6（系统性炎症和心血管风险的关键驱动因子）和纤维蛋白原（心血管事件的独立预测因子）水平。安全性方面，BGE-102的安全性和耐受性良好。

GTB-5550：向FDA提交IND申请

GT Biopharma公司宣布已向美国FDA提交了其新型候选药物GTB-5550的IND申请。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7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。

ORKA-002：公布1期临床试验的中期数据

Oruka Therapeutics公司公布了其在研药物ORKA-002在1期临床试验中取得积极的中期数据。ORKA-002旨在阻断在银屑病及其他炎症性疾病发病机制中起关键作用的细胞因子，是一种靶向IL-17-A和IL-17-F的双特异性抗体。

该1期研究是一项随机、双盲、安慰剂对照试验，在24名健康成人受试者中评估了ORKA-002的安全性和药代动力学特征。截至2026年1月数据截止日期的结果，ORKA-002的半衰期达75–80天，支持银屑病患者实现每年两次的给药频率，以及化脓性汗腺炎（HS）患者每季度一次的给药频率。药效学分析显示，在体外IL-17刺激实验中，所有剂量组均能强效抑制IL-17信号传导，效果持续至末次随访（最长24周），进一步支持两年一次给药的潜力。安全性方面，ORKA-002在所有剂量水平下均耐受良好，安全性与抗IL-17类药物一致。ORKA-002的2期研究预计将于2026年1月启动，针对银屑病，下半年将启动针对化脓性汗腺炎的研究。

参考资料：

[1] Benitec Biopharma Provides Positive Long-Term Clinical Study Results for BB-301 Phase 1b/2a Clinical Trial Demonstrating Robust Efficacy and Continued Durability of Response. Retrieved January 16, 2026, from https://www.globenewswire.com/news-release/2026/01/11/3216469/0/en/Benitec-Biopharma-Provides-Positive-Long-Term-Clinical-Study-Results-for-BB-301-Phase-1b-2a-Clinical-Trial-Demonstrating-Robust-Efficacy-and-Continued-Durability-of-Response.html

[2] Ocugen Announces Publication of Phase 1 GARDian1 Trial Results for OCU410ST Modifier Gene Therapy. Retrieved January 16, 2026, from https://www.globenewswire.com/news-release/2026/01/12/3216661/0/en/Ocugen-Announces-Publication-of-Phase-1-GARDian1-Trial-Results-for-OCU410ST-Modifier-Gene-Therapy.html

[3] Tessera Therapeutics Announces FDA Clearance of IND Application for its Lead In Vivo Gene Editing Program TSRA-196 for AATD. Retrieved January 16, 2026, from https://www.globenewswire.com/news-release/2026/01/12/3216718/0/en/Tessera-Therapeutics-Announces-FDA-Clearance-of-IND-Application-for-its-Lead-In-Vivo-Gene-Editing-Program-TSRA-196-for-AATD.html

[4] Oruka Therapeutics Announces Positive Interim Phase 1 Data for ORKA-002 and Initiation of EVERLAST-B Trial of ORKA-001. Retrieved January 16, 2026, from https://www.globenewswire.com/news-release/2026/01/12/3216715/0/en/Oruka-Therapeutics-Announces-Positive-Interim-Phase-1-Data-for-ORKA-002-and-Initiation-of-EVERLAST-B-Trial-of-ORKA-001.html

[5] Lexeo Therapeutics Announces Positive Interim Phase I/II Data for LX2020 for the Treatment of PKP2-Associated Arrhythmogenic Cardiomyopathy. Retrieved January 16, 2026, from https://www.globenewswire.com/news-release/2026/01/12/3216688/0/en/Lexeo-Therapeutics-Announces-Positive-Interim-Phase-I-II-Data-for-LX2020-for-the-Treatment-of-PKP2-Associated-Arrhythmogenic-Cardiomyopathy.html

[6] Solid Biosciences Doses First Participant in First-in-Class Phase 1b FALCON Trial Evaluating SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia. Retrieved January 16, 2026, from https://www.globenewswire.com/news-release/2026/01/12/3216871/0/en/Solid-Biosciences-Doses-First-Participant-in-First-in-Class-Phase-1b-FALCON-Trial-Evaluating-SGT-212-Dual-Route-Gene-Therapy-for-the-Treatment-of-Friedreich-s-Ataxia.html

[7] Recludix Pharma Announces Dosing of First Subjects in a Phase 1 Study of REX-8756, a Potent and Selective Oral STAT6 Inhibitor, and Achieves Associated $20 Million Milestone Payment Under Collaboration with Sanofi. Retrieved January 16, 2026, from https://www.globenewswire.com/news-release/2026/01/12/3216802/0/en/Recludix-https://www.mareatx.com/press-releases/marea-therapeutics-unveils-positive-topline-phase-1-data-for-mar002-in-acromegaly-demonstrates-potential-for-best-in-disease-efficacy-and-dosing-profile/Pharma-Announces-Dosing-of-First-Subjects-in-a-Phase-1-Study-of-REX-8756-a-Potent-and-Selective-Oral-STAT6-Inhibitor-and-Achieves-Associated-20-Million-Milestone-Payment-U.html

[8] BioAge Announces Additional Positive Interim Phase 1 Data for BGE-102, a Novel Brain-Penetrant NLRP3 Inhibitor, Demonstrating Potential for Best-in-Class hsCRP Reduction in Participants with Elevated Cardiovascular Risk. Retrieved January 16, 2026, from https://www.globenewswire.com/news-release/2026/01/12/3216965/0/en/BioAge-Announces-Additional-Positive-Interim-Phase-1-Data-for-BGE-102-a-Novel-Brain-Penetrant-NLRP3-Inhibitor-Demonstrating-Potential-for-Best-in-Class-hsCRP-Reduction-in-Participa.html

[9] Curasight Announces Encouraging Preliminary Data from Phase 1 Trial Evaluating First-in-Class Radiopharmaceutical uTREAT in High-Grade Gliomas®. Retrieved January 16, 2026, from https://www.prnewswire.com/news-releases/curasight-announces-encouraging-preliminary-data-from-phase-1-trial-evaluating-first-in-class-radiopharmaceutical-utreat-in-high-grade-gliomas-302658795.html

[10] Marea Therapeutics Unveils Positive Topline Phase 1 Data for MAR002 in Acromegaly; Demonstrates Potential for Best-in-Disease Efficacy and Dosing Profile. Retrieved January 16, 2026, from https://www.mareatx.com/press-releases/marea-therapeutics-unveils-positive-topline-phase-1-data-for-mar002-in-acromegaly-demonstrates-potential-for-best-in-disease-efficacy-and-dosing-profile/

[11] Marea Therapeutics Unveils Positive Topline Phase 1 Data for MAR002 in Acromegaly; Demonstrates Potential for Best-in-Disease Efficacy and Dosing Profile. Retrieved January 16, 2026, from https://www.mareatx.com/press-releases/marea-therapeutics-unveils-positive-topline-phase-1-data-for-mar002-in-acromegaly-demonstrates-potential-for-best-in-disease-efficacy-and-dosing-profile/

[12] GT Biopharma Announces IND Submission for GTB-5550 TriKE®, a B7-H3-targeted natural killer (NK) cell engager for B7-H3 expressing solid tumor cancers. Retrieved January 16, 2026, from https://www.globenewswire.com/news-release/2026/01/15/3219554/0/en/GT-Biopharma-Announces-IND-Submission-for-GTB-5550-TriKE-a-B7-H3-targeted-natural-killer-NK-cell-engager-for-B7-H3-expressing-solid-tumor-cancers.html

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