本期看點(diǎn)：

1. GSPT1靶向分子膠降解劑用于治療 轉(zhuǎn)移性去勢(shì)抵抗性前列腺癌（mCRPC）的早期臨床試驗(yàn)結(jié)果積極， 攜帶雄激素受體（AR）突變患者的靶病灶均觀察到縮小，疾病控制率達(dá)100%。

2. PRO-X TEN雙掩蔽CD3 T細(xì)胞銜接器（TCE） VIR-5500用于治療 mCRPC，在一項(xiàng)1期臨床試驗(yàn)中使 82%的可評(píng)估 高劑量隊(duì)列組患者實(shí)現(xiàn)前列腺特異性抗原（PSA）下降≥50%（PSA50）。

MRT-2359：公布1/2期聯(lián)合治療試驗(yàn)數(shù)據(jù)

Monte Rosa Therapeutics公司公布了一項(xiàng)進(jìn)行中的1/2期臨床研究的。MRT-2359是一款高效、高選擇性、口服生物利用度良好的在研GSPT1靶向分子膠降解劑。包括前列腺癌在內(nèi)的MYC驅(qū)動(dòng)型腫瘤依賴增強(qiáng)的致癌蛋白翻譯以支持快速生長(zhǎng)。MRT-2359通過選擇性降解翻譯終止因子GSPT1以破壞蛋白翻譯過程，從而產(chǎn)生治療效果。MRT-2359治療可降低多種與前列腺癌相關(guān)的致癌蛋白水平，包括AR、MYC及細(xì)胞周期蛋白D1-E2F，并在多種mCRPC臨床前模型中顯示出顯著的抗腫瘤活性。

該研究評(píng)估其在研分子膠降解劑MRT-2359聯(lián)合恩扎盧胺（enzalutamide），在既往接受多線治療的mCRPC患者中的療效。分析顯示，整體15例可評(píng)估患者的RECIST疾病控制率達(dá)到67%（10/15），包含10例患者靶病灶出現(xiàn)縮小。攜帶AR突變患者的靶病灶均觀察到縮小，疾病控制率達(dá)100%。數(shù)據(jù)顯示治療效果具有持續(xù)性，尤其是在AR突變患者或既往未接受AR抑制劑治療的患者中更為明顯。在AR突變患者中，5例中有2例治療持續(xù)10個(gè)周期或更長(zhǎng)，且截至數(shù)據(jù)截止日仍有2例繼續(xù)接受治療。安全性方面，MRT-2359聯(lián)合恩扎盧胺總體耐受性良好，不良事件可控。公司計(jì)劃于2026年第三季度啟動(dòng)一項(xiàng)新的驗(yàn)證性2期研究，針對(duì)攜帶AR突變的患者開展評(píng)估。

VIR-5500：公布1期臨床試驗(yàn)數(shù)據(jù)

Vir Biotechnology公司公布了其在研藥物VIR-5500用于治療晚期mCRPC的1期臨床試驗(yàn)數(shù)據(jù)。VIR-5500是一款靶向前列腺特異性膜抗原（PSMA）的PRO-XTEN雙掩蔽CD3 T細(xì)胞銜接器，可在到達(dá)腫瘤微環(huán)境前保持非活性狀態(tài)，從而降低脫靶效應(yīng)并提升治療指數(shù)。近日，安斯泰來（Astellas Pharma）與Vir Biotechnology達(dá)成超17億美元的，將共同推進(jìn)VIR-5500的開發(fā)與商業(yè)化。

此次公布的數(shù)據(jù)顯示，IR-5500單藥具有良好的安全性和耐受性，迄今未觀察到劑量限制性毒性。截至2026年1月9日的數(shù)據(jù)，在≥3,000 μg/kg的高劑量隊(duì)列中，82%（14/17）的可評(píng)估患者達(dá)到PSA50，53%（9/17）達(dá)到PSA下降≥90%（PSA90）；在RECIST可評(píng)估患者中，客觀緩解率（ORR）達(dá)45%（5/11），其中4例已確認(rèn)緩解。PSMA-PET影像也證實(shí)了多發(fā)腫瘤病灶（包括內(nèi)臟轉(zhuǎn)移灶）的顯著縮小。

Pociredir：公布1b期臨床試驗(yàn)的新數(shù)據(jù)

Fulcrum Therapeutics公司公布了其在研療法pociredir在鐮狀細(xì)胞?。⊿CD）患者中開展的1b期PIONEER臨床試驗(yàn)的新數(shù)據(jù)。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。

此次公布的數(shù)據(jù)顯示，pociredir治療表現(xiàn)出快速、顯著且具有臨床意義的療效：12例患者在第12周時(shí)平均HbF的絕對(duì)水平從7.1%提升至19.3%（增加12.2個(gè)百分點(diǎn)），表達(dá)HbF的紅細(xì)胞比例從31%升至63%，顯示泛細(xì)胞型HbF誘導(dǎo)。58%（7/12）患者的HbF水平≥20%，所有患者的HbF水平均至少提升6.5個(gè)百分點(diǎn)。同時(shí)，溶血標(biāo)志物改善、紅細(xì)胞生成增強(qiáng)，并實(shí)現(xiàn)總血紅蛋白平均升高超1 g/dL。截至2025年12月23日的數(shù)據(jù)，pociredir總體耐受性良好，未出現(xiàn)治療相關(guān)嚴(yán)重不良事件（SAEs），亦無患者因治療相關(guān)不良事件而停藥。58%（7/12）的患者在治療期間未發(fā)生任何血管阻塞危象（VOC）。

參考資料：

[1] Vir Biotechnology Reports Positive Updated Phase 1 Results for PSMA-targeting, PRO-XTEN? Dual-masked T-Cell Engager VIR-5500 in Patients with Metastatic Prostate Cancer. Retrieved February 27, 2026, from https://investors.vir.bio/news/news-details/2026/Vir-Biotechnology-Reports-Positive-Updated-Phase-1-Results-for-PSMA-targeting-PRO-XTEN-Dual-masked-T-Cell-Engager-VIR-5500-in-Patients-with-Metastatic-Prostate-Cancer/default.aspx

[2] Fulcrum Therapeutics Announces Positive 12-Week Results from the 20 mg Dose Cohort of the Phase 1b PIONEER Trial of Pociredir in Sickle Cell Disease. Retrieved February 27, 2026, from https://www.globenewswire.com/news-release/2026/02/24/3243339/0/en/Fulcrum-Therapeutics-Announces-Positive-12-Week-Results-from-the-20-mg-Dose-Cohort-of-the-Phase-1b-PIONEER-Trial-of-Pociredir-in-Sickle-Cell-Disease.html

[3] SCYNEXIS Announces First Participants Dosed in a Phase 1 Single Ascending Dose and Multiple Ascending Dose Trial of Intravenous SCY-247. Retrieved February 27, 2026, from https://www.globenewswire.com/news-release/2026/02/26/3245564/0/en/SCYNEXIS-Announces-First-Participants-Dosed-in-a-Phase-1-Single-Ascending-Dose-and-Multiple-Ascending-Dose-Trial-of-Intravenous-SCY-247.html

[4] TScan Therapeutics Completes Enrollment in Cohort C of Phase 1 ALLOHA? Trial and Announces FDA Clearance of Investigational New Drug Applications for Heme Candidates TSC-102-A01 and TSC-102-A03. Retrieved February 27, 2026, from https://www.globenewswire.com/news-release/2026/02/26/3245412/0/en/TScan-Therapeutics-Completes-Enrollment-in-Cohort-C-of-Phase-1-ALLOHA-Trial-and-Announces-FDA-Clearance-of-Investigational-New-Drug-Applications-for-Heme-Candidates-TSC-102-A01-and.html

[5] Zelluna Receives UK MHRA and Ethics Approval to Initiate ZIMA-101 First-in-Human Clinical Trial. Retrieved February 27, 2026, from https://www.globenewswire.com/news-release/2026/02/20/3241927/0/en/Zelluna-Receives-UK-MHRA-and-Ethics-Approval-to-Initiate-ZIMA-101-First-in-Human-Clinical-Trial.html

[6] EuBiologics' shingles vaccine EuHZV matches active control in phase 1 interim data. Retrieved February 27, 2026, from https://www.koreabiomed.com/news/articleView.html?idxno=30721

[7] Opus Genetics Announces Initial Clinical Data from Phase 1/2 OPGx-BEST1 Gene Therapy Study at the Macula Society Annual Meeting. Retrieved February 27, 2026, from https://www.globenewswire.com/news-release/2026/02/27/3246599/0/en/Opus-Genetics-Announces-Initial-Clinical-Data-from-Phase-1-2-OPGx-BEST1-Gene-Therapy-Study-at-the-Macula-Society-Annual-Meeting.html

[8] Mahzi Therapeutics Announces First Patient Dosed in Phase 1/2 UNITE Study of MZ-1866 for Pitt Hopkins Syndrome. Retrieved February 27, 2026, from https://www.prnewswire.com/news-releases/mahzi-therapeutics-announces-first-patient-dosed-in-phase-12-unite-study-of-mz-1866-for-pitt-hopkins-syndrome-302696349.html

[9] Cartography Biosciences Doses First Patient in Phase 1 Trial of CBI-1214, a Highly Specific T-Cell Engager for the Treatment of Colorectal Cancer. Retrieved February 27, 2026, from https://www.businesswire.com/news/home/20260224232925/en/Cartography-Biosciences-Doses-First-Patient-in-Phase-1-Trial-of-CBI-1214-a-Highly-Specific-T-Cell-Engager-for-the-Treatment-of-Colorectal-Cancer

[10] IDEAYA Biosciences Announces First-Patient-In for Phase 1 Trial of IDE034, a Potential First-In-Class B7H3/PTK7 Bispecific TOP1 ADC. Retrieved February 27, 2026, from https://www.prnewswire.com/news-releases/ideaya-biosciences-announces-first-patient-in-for-phase-1-trial-of-ide034-a-potential-first-in-class-b7h3ptk7-bispecific-top1-adc-302696480.html

[11] AC Immune Initiates Phase 1 Clinical Trial of NLRP3 Inhibitor. Retrieved February 27, 2026, from https://www.globenewswire.com/news-release/2026/02/24/3243363/0/en/AC-Immune-Initiates-Phase-1-Clinical-Trial-of-NLRP3-Inhibitor.html

[12] Monte Rosa Therapeutics Presents Updated Clinical Data from Phase 1/2 Study of MRT-2359 in Combination with Enzalutamide in Heavily Pretreated Metastatic Castration-Resistant Prostate Cancer Patients at ASCO Genitourinary Cancers Symposium (ASCO GU). Retrieved February 27, 2026, from https://www.globenewswire.com/news-release/2026/02/24/3243353/0/en/Monte-Rosa-Therapeutics-Presents-Updated-Clinical-Data-from-Phase-1-2-Study-of-MRT-2359-in-Combination-with-Enzalutamide-in-Heavily-Pretreated-Metastatic-Castration-Resistant-Prost.html

[13] Bloom Science Initiates Phase 1b Obesity Trial of BL-001, Advancing a Differentiated Approach Beyond GLP-1. Retrieved February 27, 2026, from https://www.prnewswire.com/news-releases/bloom-science-initiates-phase-1b-obesity-trial-of-bl-001-advancing-a-differentiated-approach-beyond-glp-1-302694371.html

[14] QurAlis Demonstrates Effects on Disease Progression and Target Engagement in ANQUR Clinical Trial of QRL-201, a First-in-Class Precision Medicine in Development for Sporadic ALS. Retrieved February 27, 2026 from https://www.quralis.com/news/quralis-demonstrates-effects-on-disease-progression-and-target-engagement-in-anqur-clinical-trial-of-qrl-201-a-first-in-class-precision-medicine-in-development-for-sporadic-als/

[15] ADARx Pharmaceuticals Presents Onvuzosiran Data Supporting Potential for Sustained Attack-Free Rates with Reduced Treatment Burden in Patients with HAE. Retrieved February 27, 2026 from https://www.adarx.com/adarx-pharmaceuticals-presents-onvuzosiran-data-supporting-potential-for-sustained-attack-free-rates-with-reduced-treatment-burden-in-patients-with-hae/

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