▎药明康德本期看点1. CUE-101联用pembrolizumab一线治疗HPV阳性复发性转移性头颈鳞状细胞癌(HNSCC)的1期试验结果积极,与单用pembrolizumab的历史研究数据相比,患者的中位总生存期(OS)延长近20个月。2. 新型口服小分子白细胞介素-2(IL-2)调节剂GL-IL2-138获FDA批准开展临床试验,该药物通过调控内源性IL-2 mRNA表达,促进产生天然IL-2蛋白。CUE-101:公布1期联合治疗试验数据Cue Biopharma公司公布了CUE-101治疗头颈鳞状细胞癌的1期联合治疗试验的积极新数据。CUE-101是该公司开发的一款基于IL-2的T细胞诱导剂,旨在选择性调节和激活HPV阳性T细胞,以提高疗效,同时减少传统免疫疗法的副作用。截至2025年7月14日的数据,接受CUE-101联用PD-1抑制剂pembrolizumab一线治疗的HPV阳性复发性转移性HNSCC患者的客观缓解率(ORR)为50%,12个月时的总生存率为88%,中位OS为32个月。在历史研究KEYNOTE-048中,pembrolizumab单药治疗的ORR为19%,12个月时的总生存率为57%,中位OS为12.3个月。在肿瘤PD-L1表达综合阳性评分(CPS)≥1的患者中,2例患者达到完全缓解(CR),10例患者达到部分缓解(PR)。此外,接受该一线联合治疗的PD-L1低表达患者的ORR也达到了50%。GL-IL2-138:IND申请获得FDA许可Genetic Leap宣布,其新型小分子IL-2调节剂GL-IL2-138的IND申请已获得美国FDA许可。GL-IL2-138是一种小分子药物,通过结合内源性的IL-2 mRNA,促使免疫细胞系统性地产生天然IL-2蛋白。传统的重组IL-2蛋白(rIL-2)在临床上有重要地位,但由于毒副作用大、改良困难,IL-2曾一度被视为是“难以成药”的靶点。相比之下,GL-IL2-138具有更强的效力和持久性,并可通过口服方式给药,耐受性更高。此外,它具备广泛的应用潜力,涵盖肿瘤、自身免疫疾病及神经退行性疾病等多个领域。该药物即将进入一项随机、双盲、安慰剂对照的1期临床试验,以评估其在人体中的安全性、药代动力学和药效学特性。MOMA-341:1期临床试验完成首例患者给药MOMA Therapeutics公司宣布,其旨在评估MOMA-341的1期临床试验已完成首例患者给药。MOMA-341是一种高度差异化、潜在“best-in-class”的口服共价Werner解旋酶抑制剂,具有新型化学支架。MOMA-341正在被开发为单药疗法,或与化疗或免疫治疗联合使用,用于治疗微卫星不稳定性高(MSI-H)和/或DNA错配修复缺陷(dMMR)的晚期或转移性实体瘤,包括结直肠癌、胃癌和子宫内膜癌等。由于Werner解旋酶是MSI-H/dMMR肿瘤细胞存活所必需的,这使其成为一个有前景的新兴药物靶点。MOMA公司预计在2026年年中初步公布单药治疗数据。参考资料(可上下滑动查看)[1] Imugene公布异体CAR-T疗法Azer-cel治疗三线以上DLBCL的1b期试验优异缓解率数据. Retrieved July 15, 2025, from https://www.prnasia.com/story/496465-1.shtml[2] Hipp, J.F., Bacino, C.A., Bird, L.M. et al. The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a phase 1 trial. Nat Med (2025). https://doi.org/10.1038/s41591-025-03784-7[3] AAVantgarde Receives FDA Clearance to Progress Stargardt Disease Asset, AAVB-039, into CELESTE, a Phase 1/2 Clinical Trial. Retrieved July 15, 2025, from https://www.globenewswire.com/news-release/2025/07/15/3115443/0/en/AAVantgarde-Receives-FDA-Clearance-to-Progress-Stargardt-Disease-Asset-AAVB-039-into-CELESTE-a-Phase-1-2-Clinical-Trial.html[4] Garmezy, B., Grossman, J.E., Buell, J. et al. Salvage therapy with allogeneic invariant natural killer cells in a heavily pre-treated germ cell tumor. Oncogene (2025). https://doi.org/10.1038/s41388-025-03491-0[5] Earendil Labs announces initiation of a phase 1 study of a half-life extended novel anti-TL1A antibody. Retrieved July 18, 2025, from https://www.prnewswire.com/news-releases/earendil-labs-announces-initiation-of-a-phase-1-study-of-a-half-life-extended-novel-anti-tl1a-antibody-302502297.html[6] Genetic Leap receives FDA IND clearance for first-in-class, oral natural IL-2 modulator. Retrieved July 18, 2025, from https://www.prnewswire.com/news-releases/genetic-leap-receives-fda-ind-clearance-for-first-in-class-oral-natural-il-2-modulator-302506647.html[7] Medera and University of Kansas Medical Center Announce First Patient Treated in Pioneering Gene Therapy Trial for Duchenne Muscular Dystrophy Associated Cardiomyopathy. Retrieved July 18, 2025, from https://www.medera.bio/medera-biopharm-s-sardocor-showcases-positive-interim-data-from-heref-gene-therapy-phase-1/medera-and-university-of-kansas-medical-center-announce-pioneering-gene-therapy-trial[8] Lisata Therapeutics and WARPNINE Announce iLSTA Trial Enrollment Completion and Provide Preliminary Data Update. Retrieved July 18, 2025, from https://www.globenewswire.com/news-release/2025/07/17/3117132/18623/en/index.html[9] MOMA Therapeutics Announces First Patient Dosed in Phase 1 Clinical Trial for MOMA-341, a Highly Potent and Selective Werner Helicase Inhibitor. Retrieved July 18, 2025, from https://momatx.com/posts/moma-therapeutics-announces-first-patient-dosed-in-phase-1-clinical-trial-for-moma-341/[10] Cue Biopharma Reports New Complete Response and Confirmed 50% Overall Response Rate in Ongoing Phase 1 Trial of CUE-101 and Pembrolizumab in Recurrent/Metastatic HPV+ Head and Neck Cancer. Retrieved July 18, 2025, from https://www.cuebiopharma.com/news/#news-section免责声明:本文仅作信息交流之目的,文中观点不代表药明康德立场,亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导,请前往正规医院就诊。版权说明:欢迎个人转发至朋友圈,谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”,获取转载须知。分享,点赞,在看,聚焦全球生物医药健康创新