針對多種血液癌癥，多款創新療法展現積極臨床試驗進展…… | 一周盤點

本期看點：

1. 抗半乳糖凝集素-9（galectin-9）單克隆抗體 LYT-200 用于治 療復發/難治性急性髓系白血病（AML）和高風險骨髓增生異常綜合征（MDS），在一項早期臨床試驗中 聯合標準療法，使患者的初步總生存期（OS）較同類患者延長近10個月。

2. 新型在研變構型BCR-ABL1抑 制劑TERN-701在既往接受過治療的慢性髓系白血病（CML）患者中取得令人鼓舞的1期臨床試驗數據，75%接受 劑量＞320 mg/天的患者 在24周內達到主要分子學緩解（MMR）。

3. 創新BTK降解劑bexobrutideg（NX-5948） 用于治療復發或難治性慢性淋巴細胞白血病（CLL）和小淋巴細胞淋巴瘤（SLL）， 早期臨床試驗數據積極， 患者的總緩解率（ORR）達到83%。

LYT-200：公布1b期臨床試驗的初步數據

Gallop Oncology公司公布了其在研藥物LYT-200在1b期臨床試驗中的初步頂線數據。LYT-200是一種潛在“first-in-class”的抗半乳糖凝集素-9單克隆抗體，正在被評估用于治療復發/難治性AML和高風險MDS患者。

該研究納入了既往接受過多種治療的晚期患者，旨在評估LYT-200作為單藥或聯合標準療法的療效與安全性。結果顯示，LYT-200在推薦的2期劑量下表現出良好的耐受性和顯著的抗腫瘤活性。在聯合治療組中，初步的中位OS達到13.2個月，遠超此類晚期患者通常預期的＜2.5個月的生存期。此外，LYT-200在攜帶高風險基因突變的患者中也觀察到治療應答，提示其可能具有廣泛的適用人群。

TERN-701：公布1期臨床試驗數據

Terns Pharmaceuticals公司宣布，在正在進行的CARDINAL臨床試驗中，其新型在研變構型BCR-ABL1抑制劑TERN-701在既往接受過治療的CML患者中展現出令人鼓舞的療效和安全性數據。數據顯示，在所有可評估療效的患者中，64%在24周內達到MMR；而在劑量＞320 mg/天的患者亞組中，這一比例提升至75%。此外，隨著治療時間延長，TERN-701繼續保持良好的安全性和耐受性。

Bexobrutideg（NX-5948）：公布1a/1b期臨床試驗的新數據

Nurix Therapeutics公司公布了其開發的創新BTK降解劑bexobrutideg，用于治療復發或難治性CLL和SLL的積極早期臨床試驗數據。數據顯示，1a期臨床試驗中，在47名可進行療效評估的患者中，接受bexobrutideg治療的患者的ORR達到83%，中位無進展生存期為22.1個月，中位緩解持續時間為20.1個月。值得一提的是，這些患者此前接受過多種治療，包括共價和/或非共價BTK抑制劑。在1b期患者隊列中，早期數據顯示接受劑量為600 mg的bexobrutideg治療的患者ORR為83.3%。Bexobrutideg在所有劑量水平下耐受良好，符合先前披露的研究結果。

Pociredir：公布1b期臨床試驗的初步數據

Fulcrum Therapeutics公司公布了其在研療法pociredir在鐮狀細胞病（SCD）患者中開展的1b期PIONEER臨床試驗的初步結果。Pociredir是一種靶向胚胎外胚層發育蛋白（EED）的口服小分子抑制劑。通過抑制EED，pociredir可有效下調關鍵的胎兒珠蛋白阻遏蛋白（包括BCL11A），從而導致胎兒血紅蛋白（HbF）水平升高。

此次公布的結果顯示，在第6周時，20 mg劑量組患者的平均絕對HbF水平較基線增加9.9%；其中58%（7/12）的患者HbF水平≥20%。截至2025年11月11日的數據，在完成第12周訪視的患者中（n=6），20 mg組HbF平均誘導倍數＞3.75倍。此外，研究還觀察到泛細胞型HbF誘導、溶血和貧血標志物改善，以及血管阻塞危象（VOC）減少的積極趨勢。Pociredir總體耐受性良好，未報告與治療相關的嚴重不良事件（SAEs）。

KT-621：公布1b期臨床試驗數據

Kymera Therapeutics公司宣布，其在研口服、潛在“first-in-class”的STAT6降解劑KT-621在BroADen特應性皮炎（AD）臨床1b期試驗中取得積極結果。結果顯示，在100 mg和200 mg劑量組中，KT-621在皮膚和血液中分別實現了中位94%和98%的STAT6降解。同時，KT-621在血液中顯著降低多項與疾病相關的2型炎癥生物標志物水平，包括TARC（中位下降74%）、Eotaxin-3、IL-31和IgE，并在皮損中下調核心2型炎癥及與AD相關的基因。

在臨床療效方面，KT-621在多項疾病評估終點上均表現出強勁療效，所有患者平均濕疹面積和嚴重度指數（EASI）評分降低63%，瘙癢NRS峰值評分平均降低40%。對于合并哮喘的患者，中位部分呼出一氧化氮（FeNO）水平下降56%，哮喘控制獲得有意義改善；合并過敏性鼻炎的患者在癥狀及生活質量方面也獲得顯著獲益。安全性方面，KT-621耐受性良好，未報告嚴重不良事件或治療相關不良事件，未出現結膜炎病例，生命體征、實驗室檢查及心電圖亦未見臨床相關異常。

REC-4881：公布1b/2期臨床試驗數據

Recursion公司宣布，其在研變構型MEK1/2抑制劑REC-4881在家族性腺瘤性息肉病（FAP）患者中開展的1b/2期TUPELO臨床試驗取得積極數據。自然病程分析顯示，在55例未接受治療、且符合TUPELO試驗入組標準的FAP患者中，87%的患者每年息肉負荷呈增加趨勢，10%保持穩定，僅有3%出現輕微減少，展現了該疾病的進行性發展特征。

此次公布的結果顯示，接受REC-4881 4 mg/天治療的患者在12周內即展現出快速臨床活性：在12例可評估患者中，75%出現結腸息肉負荷減少，中位減少幅度達43%。停藥12周后（研究第25周），11例可評估患者中有9例（82%）仍維持療效，息肉負荷較基線中位減少53%。此外，40%的患者（4/10）實現Spigelman分期改善≥1分，表明上消化道疾病嚴重程度減輕。安全性方面，REC-4881的安全性特征與MEK1/2抑制一致，大多數治療相關不良事件為1級或2級，未報告4級及以上的治療相關不良事件。

參考資料：

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