本期看點：

1. 口服KRAS G12D（ON/OFF）抑制劑VS-7375用于治療晚期KRAS G12D突變實體瘤，在 一項1/2期臨床試驗中獲積極初步數據。 93%接受單藥治療的轉移性胰腺導管癌（mPDAC）患者的腫瘤生物標志物CA19-9水平下降超過50%。

2. 在研溶酶體靶向藥物GNS561在針對KRAS突變膽管癌患者的1b期臨床試驗中取得了積極結果。約半數患者在第6周時達到疾病穩定（SD），其中一名患者的SD狀態維持至第30周。

VS-7375：公布1/2期臨床試驗的新數據

Verastem Oncology公司宣布了一項1/2期臨床試驗的積極初步數據，該試驗旨在評估口服KRAS G12D（ON/OFF）抑制劑VS-7375在晚期KRAS G12D突變實體瘤患者中的表現。VS-7375是一款口服KRAS G12D抑制劑，可與KRAS G12D的活化狀態和非活化狀態結合，從而更全面地抑制KRAS G12D信號和腫瘤生長。此次公布的數據涵蓋了mPDAC、轉移性結直腸癌（mCRC）和晚期非小細胞肺癌（NSCLC）等多個瘤種，證實了該藥物在多個劑量水平下均展現出令人鼓舞的臨床活性及良好的安全性。此外，Verastem公司還宣布計劃與Erasca公司合作，探索將VS-7375與Erasca在研的泛RAS分子膠ERAS-0015聯合用于KRASG12D突變實體瘤的潛在臨床評估。

此次公布的結果顯示，VS-7375的安全性和耐受性良好，主要表現為低級別胃腸道反應，且多數在治療第一周期后顯著減輕；聯合用藥方案的耐受性也良好。在經治mPDAC患者中，900 mg VS-7375單藥治療使93%（13/14）患者的腫瘤標志物CA19-9下降超過50%；與西妥昔單抗的聯合用藥則帶來了更深、更快的腫瘤縮小；與標準化療（吉西他濱聯合白蛋白結合型紫杉醇）也表現出良好的相容性。在mCRC和NSCLC隊列中，該藥物也觀察到了積極的初步療效。

GNS561：公布1b期聯合治療試驗數據

GENFIT公司宣布其在研溶酶體靶向藥物GNS561在針對KRAS突變膽管癌患者的1b期臨床試驗中取得了積極結果。GNS561是一種靶向PPT1的小分子藥物，通過抑制自噬和誘導溶酶體功能障礙來破壞癌細胞生存機制，與MEK抑制劑聯用可協同阻斷自噬與MAPK信號通路。該研究旨在評估GNS561聯合曲美替尼在既往接受過一至二線標準治療失敗、亟需新療法的重度經治膽管癌患者中的安全性與耐受性。

試驗結果顯示，該聯合療法展現出良好的安全性和耐受性，未報告劑量限制性毒性（DLT），支持繼續推進臨床開發。在抗腫瘤活性方面，約半數患者在第6周時達到SD，其中一名患者的SD狀態維持至第30周。基于這些積極數據，GENFIT決定在更高劑量水平下擴展1b期研究以進一步充實臨床數據集，同時計劃于今年夏季確定2期臨床試驗的推薦劑量及研究方案，2期臨床試驗仍按計劃將于2026年下半年啟動。

VK3019：啟動1期臨床試驗

Viking Therapeutics公司宣布正式啟動其在研胰淀素與降鈣素受體雙重激動劑（DACRA）VK3019的1期臨床試驗。VK3019正被開發為一種潛在的減重治療選擇，此次1期試驗采用隨機、雙盲、安慰劑對照設計，納入身體質量指數（BMI）≥30的健康成年受試者，主要評估VK3019單次皮下給藥后的安全性、耐受性和藥代動力學特征，同時探索單次給藥后體重變化的藥效動力學指標。

Viking內部開發的DACRAs的臨床前研究結果顯示，與接受對照治療的動物相比，該類化合物對健康大鼠和飲食誘導肥胖（DIO）小鼠的體重、食物攝入及代謝產生了顯著影響。在單次給藥后的0至72小時內，這些化合物有效減少了瘦大鼠的食物攝入量；在給藥72小時時，與對照組相比，受試動物的體重降幅最高達8%。

參考資料：

[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

免責聲明：本文僅作信息交流之目的，文中觀點不代 表藥 明康德立場，亦不代 表藥明康德支持或反對文中觀點。本文也不是治療方案推薦。如需獲得治療方案指導，請前往正規醫院就診。

版權說明：歡迎個人轉發至朋友圈，謝絕媒體或機構未經授權以任何形式轉載至其他平臺。轉載授權請在「藥明康德」微信公眾號回復“轉載”，獲取轉載須知。