本期看點：

1. 具 良好 腦滲透性 的小分子NLRP 3抑制劑 BGE-102公布了 1期臨床試驗的新數(shù)據(jù)，給藥 14天后， 具有心血管風(fēng)險因素的肥胖受試者的關(guān)鍵炎癥標(biāo)志物水平下降86%，93%達(dá)到了正常水平。

2. GT Biopharma公司宣 布已向美國FDA提交了其新 型 B7-H3 靶向 三 特異性自然殺傷（NK）細(xì)胞銜接器 GTB-5550的IND申請。

BGE-102：公布1期臨床試驗的新數(shù)據(jù)

BioAge公司公布了其候選藥物BGE-102的1期臨床試驗的新數(shù)據(jù)。BGE-102是一種結(jié)構(gòu)新穎、可口服的小分子NLRP3抑制劑，具有高活性及良好的腦滲透性，旨在用于治療具有心血管風(fēng)險因素的患者。NLRP3是驅(qū)動與年齡相關(guān)炎癥的關(guān)鍵因子，與肥胖、神經(jīng)退行性疾病及心血管疾病密切相關(guān)。該公司基于人類衰老隊列分析發(fā)現(xiàn)，NLRP3活性降低與長壽相關(guān)，因此將其確立為潛在治療靶點。BGE-102通過獨特的結(jié)合位點和機(jī)制抑制NLRP3炎性小體，作用方式區(qū)別于其他在研NLRP3抑制劑。

在一項針對肥胖（BMI 32–42）且基線高敏C反應(yīng)蛋白（hsCRP）升高（>3 mg/L）的受試者開展的多劑量遞增（MAD）研究中，每日接受120 mg的BGE-102治療受試者的炎癥標(biāo)志物顯示出迅速且顯著的減少。第14天時，受試者的中位hsCRP水平下降86%，93%（13/14）達(dá)到了正常水平（<2 mg/L）。此外，BGE-102還顯著降低了IL-6（系統(tǒng)性炎癥和心血管風(fēng)險的關(guān)鍵驅(qū)動因子）和纖維蛋白原（心血管事件的獨立預(yù)測因子）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申請

GT Biopharma公司宣布已向美國FDA提交了其新型候選藥物GTB-5550的IND申請。GTB-5550是一種靶向B7-H3的三特異性NK細(xì)胞銜接器，用于治療表達(dá)B7-H3抗原的實體腫瘤。該分子由三種組分組成，通過柔性接頭連接：1）靶向NK細(xì)胞表面CD16激活受體的納米體臂；2）野生型IL-15（WT IL-15）連接臂，用于促進(jìn)NK細(xì)胞的增殖、活化和存活；3）靶向腫瘤細(xì)胞上B7-H3抗原的納米體臂，從而將NK細(xì)胞精準(zhǔn)引導(dǎo)至腫瘤部位。

計劃開展的1期臨床試驗共分為兩個部分：1a期為劑量遞增階段，將評估最多7個劑量水平以確定最大耐受劑量（MTD）；1b期為劑量擴(kuò)展階段，將在7種不同的轉(zhuǎn)移性癌癥隊列中（包括去勢抵抗性前列腺癌、卵巢癌、乳腺癌、頭頸癌、非小細(xì)胞肺癌、胰腺癌和膀胱癌）驗證1a期研究確定的MTD，并進(jìn)一步評估藥物的耐受性。

ORKA-002：公布1期臨床試驗的中期數(shù)據(jù)

Oruka Therapeutics公司公布了其在研藥物ORKA-002在1期臨床試驗中取得積極的中期數(shù)據(jù)。ORKA-002旨在阻斷在銀屑病及其他炎癥性疾病發(fā)病機(jī)制中起關(guān)鍵作用的細(xì)胞因子，是一種靶向IL-17-A和IL-17-F的雙特異性抗體。

該1期研究是一項隨機(jī)、雙盲、安慰劑對照試驗，在24名健康成人受試者中評估了ORKA-002的安全性和藥代動力學(xué)特征。截至2026年1月數(shù)據(jù)截止日期的結(jié)果，ORKA-002的半衰期達(dá)75–80天，支持銀屑病患者實現(xiàn)每年兩次的給藥頻率，以及化膿性汗腺炎（HS）患者每季度一次的給藥頻率。藥效學(xué)分析顯示，在體外IL-17刺激實驗中，所有劑量組均能強(qiáng)效抑制IL-17信號傳導(dǎo)，效果持續(xù)至末次隨訪（最長24周），進(jìn)一步支持兩年一次給藥的潛力。安全性方面，ORKA-002在所有劑量水平下均耐受良好，安全性與抗IL-17類藥物一致。ORKA-002的2期研究預(yù)計將于2026年1月啟動，針對銀屑病，下半年將啟動針對化膿性汗腺炎的研究。

參考資料：

[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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