本期看點(diǎn)：

1. 抗半乳糖凝集素-9（galectin-9）單克隆抗體 LYT-200 用于治 療復(fù)發(fā)/難治性急性髓系白血病（AML）和高風(fēng)險(xiǎn)骨髓增生異常綜合征（MDS），在一項(xiàng)早期臨床試驗(yàn)中 聯(lián)合標(biāo)準(zhǔn)療法，使患者的初步總生存期（OS）較同類患者延長(zhǎng)近10個(gè)月。

2. 新型在研變構(gòu)型BCR-ABL1抑 制劑TERN-701在既往接受過(guò)治療的慢性髓系白血?。–ML）患者中取得令人鼓舞的1期臨床試驗(yàn)數(shù)據(jù)，75%接受 劑量＞320 mg/天的患者 在24周內(nèi)達(dá)到主要分子學(xué)緩解（MMR）。

3. 創(chuàng)新BTK降解劑bexobrutideg（NX-5948） 用于治療復(fù)發(fā)或難治性慢性淋巴細(xì)胞白血?。–LL）和小淋巴細(xì)胞淋巴瘤（SLL）， 早期臨床試驗(yàn)數(shù)據(jù)積極， 患者的總緩解率（ORR）達(dá)到83%。

LYT-200：公布1b期臨床試驗(yàn)的初步數(shù)據(jù)

Gallop Oncology公司公布了其在研藥物L(fēng)YT-200在1b期臨床試驗(yàn)中的初步頂線數(shù)據(jù)。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(píng)估用于治療復(fù)發(fā)/難治性AML和高風(fēng)險(xiǎn)MDS患者。

該研究納入了既往接受過(guò)多種治療的晚期患者，旨在評(píng)估LYT-200作為單藥或聯(lián)合標(biāo)準(zhǔn)療法的療效與安全性。結(jié)果顯示，LYT-200在推薦的2期劑量下表現(xiàn)出良好的耐受性和顯著的抗腫瘤活性。在聯(lián)合治療組中，初步的中位OS達(dá)到13.2個(gè)月，遠(yuǎn)超此類晚期患者通常預(yù)期的＜2.5個(gè)月的生存期。此外，LYT-200在攜帶高風(fēng)險(xiǎn)基因突變的患者中也觀察到治療應(yīng)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(yàn)數(shù)據(jù)

Terns Pharmaceuticals公司宣布，在正在進(jìn)行的CARDINAL臨床試驗(yàn)中，其新型在研變構(gòu)型BCR-ABL1抑制劑TERN-701在既往接受過(guò)治療的CML患者中展現(xiàn)出令人鼓舞的療效和安全性數(shù)據(jù)。數(shù)據(jù)顯示，在所有可評(píng)估療效的患者中，64%在24周內(nèi)達(dá)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(shí)間延長(zhǎng)，TERN-701繼續(xù)保持良好的安全性和耐受性。

Bexobrutideg（NX-5948）：公布1a/1b期臨床試驗(yàn)的新數(shù)據(jù)

Nurix Therapeutics公司公布了其開(kāi)發(fā)的創(chuàng)新BTK降解劑bexobrutideg，用于治療復(fù)發(fā)或難治性CLL和SLL的積極早期臨床試驗(yàn)數(shù)據(jù)。數(shù)據(jù)顯示，1a期臨床試驗(yàn)中，在47名可進(jìn)行療效評(píng)估的患者中，接受bexobrutideg治療的患者的ORR達(dá)到83%，中位無(wú)進(jìn)展生存期為22.1個(gè)月，中位緩解持續(xù)時(shí)間為20.1個(gè)月。值得一提的是，這些患者此前接受過(guò)多種治療，包括共價(jià)和/或非共價(jià)BTK抑制劑。在1b期患者隊(duì)列中，早期數(shù)據(jù)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(jié)果。

Pociredir：公布1b期臨床試驗(yàn)的初步數(shù)據(jù)

Fulcrum Therapeutics公司公布了其在研療法pociredir在鐮狀細(xì)胞?。⊿CD）患者中開(kāi)展的1b期PIONEER臨床試驗(yàn)的初步結(jié)果。Pociredir是一種靶向胚胎外胚層發(fā)育蛋白（EED）的口服小分子抑制劑。通過(guò)抑制EED，pociredir可有效下調(diào)關(guān)鍵的胎兒珠蛋白阻遏蛋白（包括BCL11A），從而導(dǎo)致胎兒血紅蛋白（HbF）水平升高。

此次公布的結(jié)果顯示，在第6周時(shí)，20 mg劑量組患者的平均絕對(duì)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(shù)據(jù)，在完成第12周訪視的患者中（n=6），20 mg組HbF平均誘導(dǎo)倍數(shù)＞3.75倍。此外，研究還觀察到泛細(xì)胞型HbF誘導(dǎo)、溶血和貧血標(biāo)志物改善，以及血管阻塞危象（VOC）減少的積極趨勢(shì)。Pociredir總體耐受性良好，未報(bào)告與治療相關(guān)的嚴(yán)重不良事件（SAEs）。

KT-621：公布1b期臨床試驗(yàn)數(shù)據(jù)

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(yīng)性皮炎（AD）臨床1b期試驗(yàn)中取得積極結(jié)果。結(jié)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(shí)現(xiàn)了中位94%和98%的STAT6降解。同時(shí)，KT-621在血液中顯著降低多項(xiàng)與疾病相關(guān)的2型炎癥生物標(biāo)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(diào)核心2型炎癥及與AD相關(guān)的基因。

在臨床療效方面，KT-621在多項(xiàng)疾病評(píng)估終點(diǎn)上均表現(xiàn)出強(qiáng)勁療效，所有患者平均濕疹面積和嚴(yán)重度指數(shù)（EASI）評(píng)分降低63%，瘙癢NRS峰值評(píng)分平均降低40%。對(duì)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(guò)敏性鼻炎的患者在癥狀及生活質(zhì)量方面也獲得顯著獲益。安全性方面，KT-621耐受性良好，未報(bào)告嚴(yán)重不良事件或治療相關(guān)不良事件，未出現(xiàn)結(jié)膜炎病例，生命體征、實(shí)驗(yàn)室檢查及心電圖亦未見(jiàn)臨床相關(guān)異常。

REC-4881：公布1b/2期臨床試驗(yàn)數(shù)據(jù)

Recursion公司宣布，其在研變構(gòu)型MEK1/2抑制劑REC-4881在家族性腺瘤性息肉?。‵AP）患者中開(kāi)展的1b/2期TUPELO臨床試驗(yàn)取得積極數(shù)據(jù)。自然病程分析顯示，在55例未接受治療、且符合TUPELO試驗(yàn)入組標(biāo)準(zhǔn)的FAP患者中，87%的患者每年息肉負(fù)荷呈增加趨勢(shì)，10%保持穩(wěn)定，僅有3%出現(xiàn)輕微減少，展現(xiàn)了該疾病的進(jìn)行性發(fā)展特征。

此次公布的結(jié)果顯示，接受REC-4881 4 mg/天治療的患者在12周內(nèi)即展現(xiàn)出快速臨床活性：在12例可評(píng)估患者中，75%出現(xiàn)結(jié)腸息肉負(fù)荷減少，中位減少幅度達(dá)43%。停藥12周后（研究第25周），11例可評(píng)估患者中有9例（82%）仍維持療效，息肉負(fù)荷較基線中位減少53%。此外，40%的患者（4/10）實(shí)現(xiàn)Spigelman分期改善≥1分，表明上消化道疾病嚴(yán)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(shù)治療相關(guān)不良事件為1級(jí)或2級(jí)，未報(bào)告4級(jí)及以上的治療相關(guān)不良事件。

參考資料：

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[27] Tenaya Therapeutics Reports Positive Interim Data from Cohort 1 of RIDGE?-1 Phase 1b/2 Clinical Trial of TN-401 Gene Therapy for Adults with PKP2-associated ARVC. Retrieved December 12, 2025, from https://www.globenewswire.com/news-release/2025/12/11/3204284/0/en/Tenaya-Therapeutics-Reports-Positive-Interim-Data-from-Cohort-1-of-RIDGE-1-Phase-1b-2-Clinical-Trial-of-TN-401-Gene-Therapy-for-Adults-with-PKP2-associated-ARVC.html

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