本期看點(diǎn)：

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

2. 在研溶酶體靶向藥物GNS561在針對(duì)KRAS突變膽管癌患者的1b期臨床試驗(yàn)中取得了積極結(jié)果。約半數(shù)患者在第6周時(shí)達(dá)到疾病穩(wěn)定（SD），其中一名患者的SD狀態(tài)維持至第30周。

VS-7375：公布1/2期臨床試驗(yàn)的新數(shù)據(jù)

Verastem Oncology公司宣布了一項(xiàng)1/2期臨床試驗(yàn)的積極初步數(shù)據(jù)，該試驗(yàn)旨在評(píng)估口服KRAS G12D（ON/OFF）抑制劑VS-7375在晚期KRAS G12D突變實(shí)體瘤患者中的表現(xiàn)。VS-7375是一款口服KRAS G12D抑制劑，可與KRAS G12D的活化狀態(tài)和非活化狀態(tài)結(jié)合，從而更全面地抑制KRAS G12D信號(hào)和腫瘤生長(zhǎng)。此次公布的數(shù)據(jù)涵蓋了mPDAC、轉(zhuǎn)移性結(jié)直腸癌（mCRC）和晚期非小細(xì)胞肺癌（NSCLC）等多個(gè)瘤種，證實(shí)了該藥物在多個(gè)劑量水平下均展現(xiàn)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(jì)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(lián)合用于KRASG12D突變實(shí)體瘤的潛在臨床評(píng)估。

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

GNS561：公布1b期聯(lián)合治療試驗(yàn)數(shù)據(jù)

GENFIT公司宣布其在研溶酶體靶向藥物GNS561在針對(duì)KRAS突變膽管癌患者的1b期臨床試驗(yàn)中取得了積極結(jié)果。GNS561是一種靶向PPT1的小分子藥物，通過(guò)抑制自噬和誘導(dǎo)溶酶體功能障礙來(lái)破壞癌細(xì)胞生存機(jī)制，與MEK抑制劑聯(lián)用可協(xié)同阻斷自噬與MAPK信號(hào)通路。該研究旨在評(píng)估GNS561聯(lián)合曲美替尼在既往接受過(guò)一至二線標(biāo)準(zhǔn)治療失敗、亟需新療法的重度經(jīng)治膽管癌患者中的安全性與耐受性。

試驗(yàn)結(jié)果顯示，該聯(lián)合療法展現(xiàn)出良好的安全性和耐受性，未報(bào)告劑量限制性毒性（DLT），支持繼續(xù)推進(jìn)臨床開(kāi)發(fā)。在抗腫瘤活性方面，約半數(shù)患者在第6周時(shí)達(dá)到SD，其中一名患者的SD狀態(tài)維持至第30周。基于這些積極數(shù)據(jù)，GENFIT決定在更高劑量水平下擴(kuò)展1b期研究以進(jìn)一步充實(shí)臨床數(shù)據(jù)集，同時(shí)計(jì)劃于今年夏季確定2期臨床試驗(yàn)的推薦劑量及研究方案，2期臨床試驗(yàn)仍按計(jì)劃將于2026年下半年啟動(dòng)。

VK3019：?jiǎn)?dòng)1期臨床試驗(yàn)

Viking Therapeutics公司宣布正式啟動(dòng)其在研胰淀素與降鈣素受體雙重激動(dòng)劑（DACRA）VK3019的1期臨床試驗(yàn)。VK3019正被開(kāi)發(fā)為一種潛在的減重治療選擇，此次1期試驗(yàn)采用隨機(jī)、雙盲、安慰劑對(duì)照設(shè)計(jì)，納入身體質(zhì)量指數(shù)（BMI）≥30的健康成年受試者，主要評(píng)估VK3019單次皮下給藥后的安全性、耐受性和藥代動(dòng)力學(xué)特征，同時(shí)探索單次給藥后體重變化的藥效動(dòng)力學(xué)指標(biāo)。

Viking內(nèi)部開(kāi)發(fā)的DACRAs的臨床前研究結(jié)果顯示，與接受對(duì)照治療的動(dòng)物相比，該類(lèi)化合物對(duì)健康大鼠和飲食誘導(dǎo)肥胖（DIO）小鼠的體重、食物攝入及代謝產(chǎn)生了顯著影響。在單次給藥后的0至72小時(shí)內(nèi)，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(shí)時(shí)，與對(duì)照組相比，受試動(dòng)物的體重降幅最高達(dá)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(zé)聲明：本文僅作信息交流之目的，文中觀點(diǎn)不代 表藥 明康德立場(chǎng)，亦不代 表藥明康德支持或反對(duì)文中觀點(diǎn)。本文也不是治療方案推薦。如需獲得治療方案指導(dǎo)，請(qǐng)前往正規(guī)醫(yī)院就診。

版權(quán)說(shuō)明：歡迎個(gè)人轉(zhuǎn)發(fā)至朋友圈，謝絕媒體或機(jī)構(gòu)未經(jīng)授權(quán)以任何形式轉(zhuǎn)載至其他平臺(tái)。轉(zhuǎn)載授權(quán)請(qǐng)?jiān)凇杆幟骺档隆刮⑿殴娞?hào)回復(fù)“轉(zhuǎn)載”，獲取轉(zhuǎn)載須知。