本期看点：

1. 口服KRAS G12D（ON/OFF）抑制剂VS-7375用于治疗晚期KRAS G12D突变实体瘤，在 一项1/2期临床试验中获积极初步数据。 93%接受单药治疗的转移性胰腺导管癌（mPDAC）患者的肿瘤生物标志物CA19-9水平下降超过50%。

2. 在研溶酶体靶向药物GNS561在针对KRAS突变胆管癌患者的1b期临床试验中取得了积极结果。约半数患者在第6周时达到疾病稳定（SD），其中一名患者的SD状态维持至第30周。

打开网易新闻 查看精彩图片

VS-7375：公布1/2期临床试验的新数据

打开网易新闻 查看精彩图片

Verastem Oncology公司宣布了一项1/2期临床试验的积极初步数据，该试验旨在评估口服KRAS G12D（ON/OFF）抑制剂VS-7375在晚期KRAS G12D突变实体瘤患者中的表现。VS-7375是一款口服KRAS G12D抑制剂，可与KRAS G12D的活化状态和非活化状态结合，从而更全面地抑制KRAS G12D信号和肿瘤生长。此次公布的数据涵盖了mPDAC、转移性结直肠癌（mCRC）和晚期非小细胞肺癌（NSCLC）等多个瘤种，证实了该药物在多个剂量水平下均展现出令人鼓舞的临床活性及良好的安全性。此外，Verastem公司还宣布计划与Erasca公司合作，探索将VS-7375与Erasca在研的泛RAS分子胶ERAS-0015联合用于KRASG12D突变实体瘤的潜在临床评估。

此次公布的结果显示，VS-7375的安全性和耐受性良好，主要表现为低级别胃肠道反应，且多数在治疗第一周期后显著减轻；联合用药方案的耐受性也良好。在经治mPDAC患者中，900 mg VS-7375单药治疗使93%（13/14）患者的肿瘤标志物CA19-9下降超过50%；与西妥昔单抗的联合用药则带来了更深、更快的肿瘤缩小；与标准化疗（吉西他滨联合白蛋白结合型紫杉醇）也表现出良好的相容性。在mCRC和NSCLC队列中，该药物也观察到了积极的初步疗效。

GNS561：公布1b期联合治疗试验数据

打开网易新闻 查看精彩图片

GENFIT公司宣布其在研溶酶体靶向药物GNS561在针对KRAS突变胆管癌患者的1b期临床试验中取得了积极结果。GNS561是一种靶向PPT1的小分子药物，通过抑制自噬和诱导溶酶体功能障碍来破坏癌细胞生存机制，与MEK抑制剂联用可协同阻断自噬与MAPK信号通路。该研究旨在评估GNS561联合曲美替尼在既往接受过一至二线标准治疗失败、亟需新疗法的重度经治胆管癌患者中的安全性与耐受性。

试验结果显示，该联合疗法展现出良好的安全性和耐受性，未报告剂量限制性毒性（DLT），支持继续推进临床开发。在抗肿瘤活性方面，约半数患者在第6周时达到SD，其中一名患者的SD状态维持至第30周。基于这些积极数据，GENFIT决定在更高剂量水平下扩展1b期研究以进一步充实临床数据集，同时计划于今年夏季确定2期临床试验的推荐剂量及研究方案，2期临床试验仍按计划将于2026年下半年启动。

VK3019：启动1期临床试验

打开网易新闻 查看精彩图片

Viking Therapeutics公司宣布正式启动其在研胰淀素与降钙素受体双重激动剂（DACRA）VK3019的1期临床试验。VK3019正被开发为一种潜在的减重治疗选择，此次1期试验采用随机、双盲、安慰剂对照设计，纳入身体质量指数（BMI）≥30的健康成年受试者，主要评估VK3019单次皮下给药后的安全性、耐受性和药代动力学特征，同时探索单次给药后体重变化的药效动力学指标。

Viking内部开发的DACRAs的临床前研究结果显示，与接受对照治疗的动物相比，该类化合物对健康大鼠和饮食诱导肥胖（DIO）小鼠的体重、食物摄入及代谢产生了显著影响。在单次给药后的0至72小时内，这些化合物有效减少了瘦大鼠的食物摄入量；在给药72小时时，与对照组相比，受试动物的体重降幅最高达8%。

参考资料：

[1] TScan Therapeutics Announces Positive Initial Data from Cohort C of Ongoing ALLOHA™ Phase 1 Study Evaluating TSC-101 in Patients with Heme Malignancies Undergoing Allogeneic Hematopoietic Cell Transplantation. Retrieved June 26, 2026, from https://www.globenewswire.com/news-release/2026/06/22/3315162/0/en/tscan-therapeutics-announces-positive-initial-data-from-cohort-c-of-ongoing-alloha-phase-1-study-evaluating-tsc-101-in-patients-with-heme-malignancies-undergoing-allogeneic-hematop.html

[2] Taysha Gene Therapies Announces Completion of Dosing in REVEAL Pivotal Trial and Reports Longer-Term Clinical Data from Part A of REVEAL Phase 1/2 Trials Evaluating TSHA-102 for Rett Syndrome. Retrieved June 26, 2026, from https://www.globenewswire.com/news-release/2026/06/22/3315248/0/en/taysha-gene-therapies-announces-completion-of-dosing-in-reveal-pivotal-trial-and-reports-longer-term-clinical-data-from-part-a-of-reveal-phase-1-2-trials-evaluating-tsha-102-for-re.html

[3] Pheno Therapeutics Granted FDA IND Clearance for Lead Multiple Sclerosis Therapeutic Candidate PTD802. Retrieved June 26, 2026, from https://secure.businesswire.com/news/home/20260622772175/en/Pheno-Therapeutics-Granted-FDA-IND-Clearance-for-Lead-Multiple-Sclerosis-Therapeutic-Candidate-PTD802

[4] Prolium Bioscience Announces First Patient Dosed in its Multinational, Phase 1/2 Study of PRO-203 in Systemic Sclerosis, and Last Patient, Last Visit in an Investigator-Led Study of PRO-203 in Refractory Lupus Nephritis. Retrieved June 26, 2026, from https://www.globenewswire.com/news-release/2026/06/22/3315254/0/en/prolium-bioscience-announces-first-patient-dosed-in-its-multinational-phase-1-2-study-of-pro-203-in-systemic-sclerosis-and-last-patient-last-visit-in-an-investigator-led-study-of-p.html

[5] Kither Biotech successfully completes Phase 1 clinical trial of KIT2014, a novel inhaled peptide therapy for respiratory diseases. Retrieved June 26, 2026, from https://www.globenewswire.com/news-release/2026/06/19/3314633/0/en/kither-biotech-successfully-completes-phase-1-clinical-trial-of-kit2014-a-novel-inhaled-peptide-therapy-for-respiratory-diseases.html

[6] GENFIT: Positive Phase 1b Data with GNS561 in Combination Therapy in Heavily Pretreated Patients with Cholangiocarcinoma. Retrieved June 26, 2026, from https://www.globenewswire.com/news-release/2026/06/23/3316274/0/en/genfit-positive-phase-1b-data-with-gns561-in-combination-therapy-in-heavily-pretreated-patients-with-cholangiocarcinoma.html

[7] ProQR Announces Positive Phase 1 Target Engagement Data for AX-0810, Establishing First Clinical Validation of the Axiomer RNA Editing Platform. Retrieved June 26, 2026, from https://www.globenewswire.com/news-release/2026/06/25/3317367/33039/en/proqr-announces-positive-phase-1-target-engagement-data-for-ax-0810-establishing-first-clinical-validation-of-the-axiomer-rna-editing-platform.html

[8] Viking Therapeutics Announces Initiation of Phase 1 Study to Evaluate Novel Amylin Receptor Agonist VK3019 in Healthy Adults. Retrieved June 26, 2026, from https://www.prnewswire.com/news-releases/viking-therapeutics-announces-initiation-of-phase-1-study-to-evaluate-novel-amylin-receptor-agonist-vk3019-in-healthy-adults-302808417.html

[9] VS-7375 Demonstrates Clinical Activity with a Favorable Safety and Tolerability Profile in TARGET-D 101 Phase 1/2 Clinical Trial in Patients with Advanced KRAS G12D-Mutated Solid Tumors. Retrieved June 26, 2026, from https://secure.businesswire.com/news/home/20260623312556/en/VS-7375-Demonstrates-Clinical-Activity-with-a-Favorable-Safety-and-Tolerability-Profile-in-TARGET-D-101-Phase-12-Clinical-Trial-in-Patients-with-Advanced-KRAS-G12D-Mutated-Solid-Tumors

[10] Araris Biotech AG and Taiho Oncology Announce Dosing of First Patient in Phase 1 Trial of ARC-02, a Novel ADC for the Treatment of Non-Hodgkin Lymphoma. Retrieved June 26, 2026, from https://secure.businesswire.com/news/home/20260623627184/en/Araris-Biotech-AG-and-Taiho-Oncology-Announce-Dosing-of-First-Patient-in-Phase-1-Trial-of-ARC-02-a-Novel-ADC-for-the-Treatment-of-Non-Hodgkin-Lymphoma

[11] Leal Therapeutics Doses First Patient in NeurALS Phase 1/2 Trial of LTX-002 for Amyotrophic Lateral Sclerosis (ALS). Retrieved June 26, 2026, from https://www.prnewswire.com/news-releases/leal-therapeutics-doses-first-patient-in-neurals-phase-12-trial-of-ltx-002-for-amyotrophic-lateral-sclerosis-als-302807297.html

[12] Mediar Therapeutics Announces First Cohorts Dosed in Phase 1 Clinical Trial of MTX-439 for Fibrosis Associated with Chronic Kidney Disease (CKD) and Additions to Clinical Advisory Board. Retrieved June 26, 2026, from https://www.prnewswire.com/news-releases/mediar-therapeutics-announces-first-cohorts-dosed-in-phase-1-clinical-trial-of-mtx-439-for-fibrosis-associated-with-chronic-kidney-disease-ckd-and-additions-to-clinical-advisory-board-302807065.html

[13] Trace Neuroscience Initiates Global Clinical Development Program for TRCN-1023, an Antisense Oligonucleotide Designed to Restore UNC13A Function for the Treatment of ALS. Retrieved June 26, 2026, from https://secure.businesswire.com/news/home/20260622451252/en/Trace-Neuroscience-Initiates-Global-Clinical-Development-Program-for-TRCN-1023-an-Antisense-Oligonucleotide-Designed-to-Restore-UNC13A-Function-for-the-Treatment-of-ALS

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。