Phase 2 Study of 9-ING-41, a Glycogen Synthase Kinase 3 Beta (GSK 3β) Inhibitor, Plus Carboplatin in Patients With Advanced, Metastatic Salivary Gland Carcinoma

9-ING-41 is a small molecule potent selective GSK-3β inhibitor with antitumor activity. This study investigates 9-ING-41 in combination with carboplatin chemotherapy in patients with incurable, recurrent or metastatic salivary gland carcinomas (SGC). Patients with advanced SGC (including all histologic subtypes and adenoid cystic carcinoma [ACC]) will receive 9-ING-41 intravenously (IV) along with carboplatin IV at standard dosing together on Day 1, and 9-ING-41 alone on Day 4 of a 21-day cycle. Participants will be enrolled to two histologic cohorts: Cohort 1 will be comprised of those with ACC, and Cohort 2 will include patients with non-ACC SGC (or all other salivary gland cancer histologies). Treatment will continue until progression of disease, death, or discontinuation of therapy for any reason.

开始日期2030-12-18

申办/合作机构

Actuate Therapeutics, Inc.

NCT07714395

/ Not yet recruiting临床1/2期

Phase 1/2 Open-Label, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Anti-Tumor Activity of Elraglusib Oral Tablets in Adult Participants With Advanced Solid Tumors.

This study is being done to test the safety of elraglusib when taken once a day and to assess:
* How the body processes the drug (pharmacokinetics)
* Assess potential effects of the drug on heart rhythm
* Find the highest daily dose that can be given without causing side effects

开始日期2026-10-01

申办/合作机构

Actuate Therapeutics, Inc.

NCT06896188

/ Recruiting临床1期IIT

A Phase Ib Study of 9-ING-41 (Elraglusib), a Glycogen Synthase Kinase 3-beta (GSK-3β) Inhibitor, Combined With Retifanlimab, a PD-1 Inhibitor, Plus Modified FOLFIRINOX as Frontline Therapy for Patients With Advanced Pancreatic Adenocarcinoma (RiLEY)

This is a study of the combination of 9 ING-41 (elraglusib) and retifanlimab plus mFOLFIRINOX in patients with pancreatic cancer without prior systemic therapy for advanced disease. The safety lead-in cohort will consist of 6 patients, followed by dose de-escalation if necessary, based on safety assessments. After evaluating the safety and tolerability at the initial dose level, the study will proceed to an expansion cohort at the determined safe dose level, with the total maximum enrollment not exceeding 12 patients for the entire study.

开始日期2025-09-22

申办/合作机构

University of Pittsburgh

[+2]

100 项与 Elraglusib 相关的临床结果

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100 项与 Elraglusib 相关的转化医学

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100 项与 Elraglusib 相关的专利（医药）

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项与 Elraglusib 相关的文献（医药）

2026-05-01·NATURE MEDICINE

Elraglusib and chemotherapy in metastatic pancreatic ductal adenocarcinoma: a randomized controlled phase 2 trial

Article

作者: Sahai, Vaibhav ; Weiskittel, Taylor ; Ploquin, Anne ; Giles, Francis J ; Ji, Yan ; Mazar, Andrew P ; Jaros, Mark ; Kavan, Petr ; Cervantes, Andres ; Ugolkov, Andrey ; Hiret, Sandrine ; Pernot, Simon ; Carneiro, Benedito A ; Lopez, Charles D ; Percent, Ivor J ; Shroff, Rachna T ; Coveler, Andrew L ; Fine, Gil ; Carr, Ryan ; Seifarth, Chris ; Bekaii-Saab, Tanios S ; Mulcahy, Mary ; LoConte, Noelle K ; Mahalingam, Devalingam

Metastatic pancreatic ductal adenocarcinoma (mPDAC) is one of the leading causes of cancer-related mortality, but advances in therapeutic treatments remain limited. Elraglusib (9-ING-41), an inhibitor of GSK-3β, exhibits a multimodal mechanism of action based on antitumor activity in preclinical models of cancer, including pancreatic. The efficacy and safety of elraglusib with gemcitabine plus nab-paclitaxel (GnP) were assessed in patients with previously untreated mPDAC. In an open-label, international, multicenter, phase 2 study, patients were randomized 2:1 to weekly elraglusib/GnP or GnP alone. Primary endpoints were median overall survival (OS) and 1-year survival rate. The prespecified modified intention-to-treat population included 155 patients on elraglusib/GnP and 78 on GnP. As of the data cutoff of 27 April 2025, elraglusib/GnP improved median OS by 2.9 months and decreased the risk of death by 38% versus GnP (median OS 10.1 months versus 7.2 months, respectively (hazard ratio 0.62; 95% confidence interval 0.46 to 0.84; P = 0.01)). The 1-year survival rates were 44.1% versus 22.3%, respectively. The safety profile of elraglusib/GnP was manageable. The most common grade 3 or higher treatment-emergent adverse events (TEAEs) with elraglusib/GnP versus GnP alone were neutropenia (52.3% versus 30.8%), anemia (25.2% versus 29.5%) and fatigue (16.8% versus 5.1%). Explorative correlative analyses demonstrated that baseline circulating immune-related factors (that is, CXCL2 and TRAIL ligands) were associated with improved survival in the elraglusib/GnP arm. Treatment was accompanied by increases in intratumoral cytotoxic immune cell populations. Together, these findings support the clinical activity of elraglusib/GnP as first-line treatment in mPDAC and provide a biological context for the observed survival benefit. Based on the results of this phase 2 trial, a phase 3 trial is being planned. ClinicalTrials.gov registration: NCT03678883.

2026-01-06·CLINICAL CANCER RESEARCH

Elraglusib, a Glycogen Synthase Kinase 3β Inhibitor, plus Chemotherapy with or without Immunotherapy in Patients with Recurrent, Metastatic Salivary Gland Carcinoma

Article

作者: Hanna, Glenn J. ; Jo, Vickie Y. ; Haddad, Robert I. ; Ugolkov, Andrey ; Scarfo, Nicole ; Wong, Kristine ; Sehgal, Kartik ; Mazar, Andrew ; Dennis, Michael J. ; O’Neill, Anne ; Shin, Kee-Young ; Bedard, Veronica

Abstract:

Purpose::

Glycogen synthase kinase 3β (GSK-3β) is a known therapeutic target in cancer. Aberrant nuclear GSK-3β (nGSK-3β) expression has been shown in some salivary gland cancers. Elraglusib is a small-molecule GSK-3β inhibitor with immunomodulatory potential. We hypothesized that elraglusib plus platinum-based chemotherapy and immunotherapy priming would be a novel treatment approach for salivary gland cancer.

Patients and Methods::

This phase II, open-label trial enrolled patients with recurrent or metastatic salivary gland cancer [adenoid cystic carcinoma (ACC) vs. other subtypes] with disease progression in the preceding year. Cohort 1 received elraglusib (15 mg/kg i.v. on days 1 and 4) plus carboplatin (AUC = 5) or cisplatin (75 mg/m2) every 21 days. Cohort 2 received two cycles of pembrolizumab (200 mg i.v.) every 21 days prior to the same regimen. The primary endpoint included best overall response rate (ORR) by RECIST v1.1 (>5/32 in response to detect 25% ORR).

Results::

Thirty-two patients were enrolled, including 15 (47%) with ACC and 17 (53%) with non-ACC. The best ORR was 9.4% [3/32; 95% confidence interval (CI), 2–25; three partial responses, all non-ACC]. Nineteen (59%) patients achieved stable disease. The median duration of response was 6.9 months. Common treatment-related adverse events were anemia (22, 69%), nausea (16, 50%), and neutropenia (14, 44%). At the median follow-up of 18.3 months, the median progression-free survival was 6.4 months (95% CI, 2.3–8.8), and the median overall survival was 18.6 months (95% CI, 9.7–29.4) overall. The median nGSK-3β expression was 50% versus 2% for responders versus nonresponders.

Conclusions::

The trial did not meet its primary endpoint, although 18% of patients with non-ACC treated with immune priming followed by cisplatin plus elraglusib achieved a response. Higher nGSK-3β expression was observed in tumor samples obtained from responders.

2025-12-31·CANCER BIOLOGY & THERAPY

Potential therapeutic GSK-3β inhibitor 9-ING-41 is active in combination with venetoclax in double-hit lymphoma (DHL)

Article

作者: Guo, Xutao ; Zhang, Yunxia ; Wei, Xiaolei ; Shi, Pengcheng ; Zheng, Haiqing ; Lei, Haohao

BACKGROUND:

Double-hit lymphoma (DHL) exhibits aggressive behavior due to dysregulated proliferation and resistance to apoptosis. Current therapies, including R-CHOP, show limited efficacy, necessitating novel strategies. 9-ING-41, a novel ATP-competitive small-molecule inhibitor that targets glycogen synthase kinase-3β (GSK-3β), has emerged as a promising therapeutic agent because of its ability to disrupt oncogenic signaling pathways associated with tumor progression and treatment resistance. However, the antitumor effects of 9-ING-41 in DHL remain unclear.

MATERIALS AND METHODS:

DHL cell lines (Karpas-422 and SuDHL2) were treated with venetoclax and 9-ING-41, either alone or in combination. Cell viability in cytotoxicity assays was assessed using the CCK-8 assay, while apoptosis and cell cycle changes were analyzed via flow cytometry. Western blotting was employed to evaluate alterations in the levels of GSK-3β and WNT/β-catenin pathway proteins following treatment.

RESULTS:

In preclinical studies utilizing DHL cell models, the single agent 9-ING-41 demonstrated robust biological activity through inducing significant G1/S phase cell cycle arrest and triggering apoptosis. When coadministered with venetoclax, a clinically approved BCL-2 inhibitor, the combination exhibited marked synergistic cytotoxicity in DHL cells, achieving superior inhibitory effects compared to either agent alone. The combined treatment enhanced cell cycle arrest, significantly reducing the number of S-phase cells and reinforcing G0/G1 arrest. Further mechanistic studies revealed that the combination modulated key proteins in the GSK-3 pathway and downstream WNT/β-catenin pathway, revealing a potential synergistic mechanism.

CONCLUSION:

The demonstrated single-agent efficacy and combination synergy with venetoclax support the potential of 9-ING-41 as a novel therapeutic strategy for DHL. These findings provide a proof-of-concept that may serve as a basis for future preclinical investigations in DHL.

项与 Elraglusib 相关的新闻（医药）

2026-09-09

·BioSpace

Actuate Announces Promising Preclinical Data Showing Synergistic Activity of Elraglusib in Combination with RAS Inhibitors Zoldonrasib and Daraxonrasib

Independent studies conducted at Northwestern University and Mayo Clinic demonstrate synergistic activity of elraglusib in combination with RAS-targeted agents across multiple murine and human pancreatic cancer models Data further support the potential for combination therapy strategies across other RAS-driven cancers Additional studies are ongoing with results to be presented at upcoming medical conferences CHICAGO and FORT WORTH, Texas, Sept. 09, 2026 (GLOBE NEWSWIRE) -- Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Actuate” or the “Company”), a clinical-stage biopharmaceutical company, focused on developing novel therapies for high-impact, difficult-to-treat cancers, today announced new preclinical data evaluating elraglusib in combination with zoldonrasib (RMC-9805), an investigational RAS(ON) G12D-selective inhibitor, and daraxonrasib (RMC-6236), a RAS(ON) multi-selective inhibitor, in pancreatic ductal adenocarcinoma (PDAC), including models resistant to FOLFIRINOX chemotherapy. The studies were conducted in collaboration with investigators at Northwestern University and the Mayo Clinic. Across a panel of murine KPC pancreatic cancer cell lines, the addition of elraglusib to zoldonrasib resulted in greater inhibition of tumor-cell growth compared with zoldonrasib alone. Synergistic combination activity was also observed across human patient-derived xenograft (PDX)-derived pancreatic cancer cell lines, demonstrating the effect across multiple independent pancreatic cancer models. Studies of elraglusib in combination with daraxonrasib were also conducted, demonstrating synergistic anti-cancer activity compared with either agent alone. Together, the findings with two distinct RAS-targeted agents provide evidence that the combination effect of elraglusib extends beyond a single RAS inhibitor. “The emergence of direct RAS inhibitors represents a major advance in pancreatic cancer and creates an opportunity to identify combinations capable of further enhancing RAS-directed activity,” said Tanios “Tony” Bekaii-Saab, MD, FASCO - Chair for the Division of Hematology/Medical Oncology, Mayo Clinic in Arizona. “Importantly, the enhanced activity observed when elraglusib was combined with either zoldonrasib or daraxonrasib, two distinct RAS-targeted agents, highlights the potential for elraglusib to synergize with diverse RAS-directed therapies. Based on the unique and complementary mechanisms of action of elraglusib with these new RAS inhibition therapies, we are eager to evaluate the potential of combining elraglusib with RAS inhibition therapies in a clinical setting to expand treatment opportunities across RAS-driven diseases, including pancreatic cancer.” Separate studies evaluated the combination of elraglusib with zoldonrasib in FOLFIRINOX-resistant murine and human pancreatic cancer models. Both murine and human models demonstrated that the combination of elraglusib with zoldonrasib in vitro resulted in enhanced anti-tumor activity of the combination and greater reductions in tumor cell viability compared with either agent alone. “Resistance to frontline chemotherapy remains a major challenge in pancreatic cancer, underscoring the need for new therapeutic strategies to be used after patients progress on FOLFIRINOX,” said Dr. Deva Mahalingam, Professor of Medicine, Robert H. Lurie Comprehensive Cancer Center, Northwestern University. “Across multiple FOLFIRINOX-resistant pancreatic cancer models, we observed greater antitumor activity when elraglusib was combined with zoldonrasib than with either agent alone, with quantitative analysis supporting enhanced anti-tumor activity and potential synergistic interactions of the combination in the models evaluated. These findings provide a compelling rationale to further investigate whether combining GSK-3β (elraglusib) and RAS G12D inhibition can enhance therapeutic activity in treatment-resistant pancreatic cancer. We look forward to participating in advancing the development of elraglusib in upcoming clinical trials, including combination trials with these most promising RAS inhibitors.” Key preclinical findings Across murine KPC and human PDX-derived pancreatic cancer cell lines, the addition of elraglusib to zoldonrasib reduced cell growth beyond that observed with zoldonrasib alone across all models tested. Elraglusib in combination with daraxonrasib demonstrated enhanced activity across multiple murine/human PDAC models compared with daraxonrasib alone. In independent cell line models of FOLFIRINOX-resistant PDAC, both murine and human models of elraglusib plus zoldonrasib produced greater reductions in tumor-cell viability than either agent alone Additional studies are ongoing, with results planned for presentation at upcoming medical conferences. “RAS-targeted therapies are rapidly reshaping the treatment landscape for pancreatic cancer, highlighted by the recent FDA approval of daraxonrasib, the first broad RAS-targeted therapy approved for metastatic pancreatic cancer,” said Daniel Schmitt, President and Chief Executive Officer of Actuate Therapeutics. “What is particularly compelling about the synergy data with elraglusib is the breadth and consistency of these findings. Elraglusib enhanced RAS-targeted activity across multiple murine and human models, with two distinct RAS inhibitors, and in models resistant to FOLFIRINOX. We believe these independent findings strengthen the potential for elraglusib to work synergistically with RAS-directed therapies and establish an important new combination approach in pancreatic and potentially other RAS-driven cancers.” About Actuate Therapeutics, Inc. Actuate is a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers. Actuate’s lead investigational drug, elraglusib (a novel GSK-3β inhibitor), targets molecular pathways in cancer that are involved in promoting tumor growth and resistance to conventional cancer drugs such as chemotherapy through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and DNA Damage Response (DDR). Elraglusib may also mediate anti-tumor immunity through the regulation of multiple immune checkpoints and immune cell function. For additional information, please visit the Company’s website at or follow us on LinkedIn , X, and Facebook . Forward Looking Statements This press release contains forward-looking statements about us, including our and other parties’ preclinical and clinical trials and development plans, and our industry. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would,” or the negative of these terms or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. All statements, other than statements related to present facts or current conditions or of historical facts, contained in this press release are forward-looking statements, including statements concerning the potential for elraglusib to synergize with diverse RAS-directed therapies, our intention to advance the development of elraglusib in upcoming clinical trials in combination with RAS inhibitors and the potential for elraglusib in combination with RAS-directed therapies to establish an important new combination approach in pancreatic and potentially other RAS-driven cancers. Accordingly, these statements involve estimates, assumptions, substantial risks and uncertainties which could cause actual results to differ materially from those expressed in them, including but not limited to that preliminary and unpublished data may be subject to change and further interpretation following the availability of more data or following a more comprehensive review of the data and should not be relied upon as a final analysis; clinical and preclinical drug development involves a lengthy and expensive process with uncertain timelines and outcomes, results of prior preclinical studies, early clinical trials and sub-group studies are not necessarily predictive of future results and may not correlate with improved responses, and elraglusib may not achieve positive clinical results or favorable preclinical results or receive regulatory approval on a timely basis, if at all; that we may not successfully enroll additional patients or establish or advance plans for further development, including through conversations with the FDA or EMA and the standards such bodies may impose for such development; that elraglusib could be associated with side effects, adverse events or other properties or safety risks, which could delay or preclude regulatory approval, cause us to suspend or discontinue clinical trials or result in other negative consequences; our reliance on third parties to conduct our non-clinical studies and our clinical trials; our reliance on third-party licensors and ability to preserve and protect our intellectual property rights; that we face significant competition from other biotechnology and pharmaceutical companies; our ability to fund development activities, including because our financial condition raises substantial doubt as to our ability to continue as a going concern and we require additional capital to finance our operations beyond October 2026, and a failure to obtain this necessary capital in the near term on acceptable terms, or at all, could force us to delay, limit, reduce or terminate our development programs, commercialization efforts or other operations; and those other factors discussed under the heading “Item 1A. Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2025, filed with the SEC on March 26, 2026, and in our subsequent filed Quarterly Reports on Form 10-Q, and other filings with the SEC. Because the risk factors referred to above could cause actual results or outcomes to differ materially from those expressed in any forward-looking statements made by us or on our behalf, you should not place undue reliance on any forward-looking statements. Further, any forward-looking statement speaks only as of the date on which it is made. New factors emerge from time to time, and it is not possible for us to predict which factors will arise. In addition, we cannot assess the impact of each factor on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Unless legally required, we do not undertake any obligation to release publicly any revisions to such forward-looking statements to reflect events or circumstances after the date of this press release or to reflect the occurrence of unanticipated events. Investor Contact Mike Moyer Managing Director LifeSci Advisors, LLC mmoyer@lifesciadvisors.com Media Contact Ignacio Guerrero-Ros, Ph.D. Russo Partners, LLC Ignacio.guerrero-ros@russopartnersllc.com (858) 717-2310

上市批准

2026-09-09

·BioSpace

Actuate to Present at the H.C. Wainwright 28th Annual Global Investment Conference

CHICAGO and FORT WORTH, Texas, Sept. 09, 2026 (GLOBE NEWSWIRE) -- Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Actuate” or the “Company”), a clinical-stage biopharmaceutical company, focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers, today announced that it will present at the H.C. Wainwright 28 th Annual Global Investment Conference taking place September 14-16, 2026, in New York City. In addition, the management team will be available for one-on-one meetings during the conference. Details on the presentation: Format : Corporate Presentation Date : Monday, September 14, 2026 Time : 4:00 PM ET Webcast Link : click here The webcast of the presentation will be accessible in the Investor section of the Actuate website About Actuate Therapeutics, Inc. Actuate is a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers. Actuate’s lead investigational drug, elraglusib (a novel GSK-3β inhibitor), targets molecular pathways in cancer that are involved in promoting tumor growth and resistance to conventional cancer drugs such as chemotherapy through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and DNA Damage Response (DDR). Elraglusib may also mediate anti-tumor immunity through the regulation of multiple immune checkpoints and immune cell function. For additional information, please visit the Company’s website at or follow us on LinkedIn , X, and Facebook . Investor Contact Mike Moyer Managing Director LifeSci Advisors, LLC mmoyer@lifesciadvisors.com Media Contact Ignacio Guerrero-Ros, Ph.D. Russo Partners, LLC Ignacio.guerrero-ros@russopartnersllc.com (858) 717-2310

免疫疗法

2026-08-13

·CA幸存者

2026骨与软组织肉瘤新药年中盘点：100种方案全覆盖

骨与软组织肉瘤新药全整理（2026年8月更新版）数据来源：FDA官网、NMPA/CDE公开数据、Targeted Oncology、ASCO、ESMO等权威来源 | 数据截止2026年8月12日 | 全文覆盖约95种药物/方案核心前置提示（必读）： FDA（美国食品药品监督管理局）和NMPA（中国国家药品监督管理局）是中美两套完全独立的药品审批体系，互不通用。一个药拿到FDA认定，不代表在中国有任何进展；反之亦然。 FDA各类研发认定≠上市：孤儿药认定（ODD）、突破性疗法认定（BTD）、快速通道（FTD）、罕见儿科疾病认定（RPDD）等都是FDA鼓励药物研发的“加速通道”或“优惠政策”，不代表药物已经获批上市。真正能让患者用上药的，只有“FDA正式批准”或“NMPA正式批准”。本文中“FDA审评中”的药物（NDA/BLA已提交、PDUFA日期已定）已进入上市前最后一关，是除了“已获批”之外距离患者最近的阶段。一、已获FDA正式批准新药Afami-Cel（afamitresgene autoleucel，Tecelra，TCR-T细胞疗法）获批时间：2026年6月22日（完全批准）适应症：不可切除或转移性滑膜肉瘤，既往接受过化疗适用人群：12岁及以上作用机制：靶向MAGE-A4抗原的TCR-T细胞治疗，要求患者HLA-A*02:01阳性、肿瘤表达MAGE-A4 临床价值：首款获批用于实体瘤的工程化T细胞受体免疫疗法；2024年8月加速批准，2026年6月转为完全批准并扩大适用年龄二、已递交上市申请、处于FDA审评阶段药物审评阶段说明：NDA（New Drug Application，新药申请）是小分子药物的上市申请；BLA（Biologics License Application，生物制品许可申请）是大分子生物药的上市申请。PDUFA（Prescription Drug User Fee Act，处方药使用者付费法案）日期是FDA对该药物做出最终审批决定的截止日期——通俗理解：PDUFA日期就是“FDA最晚哪天给答复” 。1. Varegacestat（AL102，口服γ分泌酶抑制剂）申请类型：NDA（新药申请），2026年4月29日提交，2026年7月FDA受理适应症：进展型硬纤维瘤（侵袭性纤维瘤病）关键数据：对比安慰剂显著延长无进展生存期（PFS），56%患者肿瘤大幅缩小，疾病进展或死亡风险降低84% PDUFA目标日期：2027年4月28日2. Ozekibart（INBRX-109，四价DR5激动剂单抗）申请类型：BLA（生物制品许可申请），2026年4月提交，2026年6月15日FDA受理适应症：不可切除/转移性经典型软骨肉瘤关键数据：ChonDRAgon III期注册临床（206例患者）显示，ozekibart组中位PFS 5.52个月 vs 安慰剂2.66个月（HR 0.479, P<0.0001），DCR 54% vs 27.5% PDUFA目标日期：2027年4月14日拓展研究：联合伊立替康+替莫唑胺用于复发/难治尤文肉瘤，后线疗效优异3. Bezuclastinib（CGT9486）+ 舒尼替尼（GIST靶向组合）申请类型：NDA（新药申请），2026年5月获FDA受理并授予优先审评（Priority Review）适应症：伊马替尼治疗后进展的胃肠道间质瘤（GIST）加速资质：优先审评（非突破性疗法认定BTD） PDUFA目标日期：2026年11月30日三、获FDA特殊通道资格（一）孤儿药认定（ODD，Orphan Drug Designation）通俗解读：FDA为鼓励开发患者人数少于20万的罕见病药物，给予税收减免、7年市场独占权等优惠。不等于获批上市。药物授予时间适应症备注 FL118（小分子） 2026年5月20日骨肉瘤罕见儿科疾病认定（RPDD，Rare Pediatric Disease Designation）+孤儿药认定（ODD）双重资格 Eftilagimod Alfa（IMP321） 2026年4月15-16日软组织肉瘤 LAG-3免疫激动剂，联合帕博利珠单抗在新辅助治疗中ORR是单纯放疗历史数据3倍以上 LP-284（小分子） 2026年1月20日软组织肉瘤靶向DNA损伤修复（DDR）通路 Vactosertib 2021年8月（ODD）；后续获RPDD+FTD 骨肉瘤口服TGF-β受体抑制剂，获FDA三项认定 LSTA1（Certepetide） 2024年3-4月骨肉瘤获FDA ODD+RPDD，通过结合α-v整合素增强药物渗透 SOT106 2026年6月3日骨肉瘤靶向LRRC15的ADC，获FDA ODD，计划2026下半年启动I期 LIT1001 2025年7月骨肉瘤含多柔比星的小颗粒制剂，直接置入骨肿瘤内部，获FDA ODD DUNP19 2022年9月骨肉瘤靶向LRRC15的抗体，获FDA ODD Dovitinib 2023年骨肉瘤泛TKI，获FDA ODD+RPDD双重认定 Cinnamtannin B-1 2024年5月2日骨肉瘤获FDA ODD（二）快速通道（FTD，Fast Track Designation）通俗解读：FDA为加速严重疾病药物的开发与审评而设立的通道，允许滚动提交资料、优先审评。不等于获批上市。药物授予时间机制临床阶段 ADCE-D01（uPARAP靶向ADC） 2025年10月9日新型ADC（抗体偶联药物），载荷为拓扑异构酶Ⅰ抑制剂 Ⅰ/Ⅱ期ADCElerate1试验，广谱抑制间叶源性肿瘤 CLD-201 2025年7月29日 first-in-class同种异体脂肪干细胞装载溶瘤病毒，瘤内注射临床阶段 OST-HER2 多批次李斯特菌免疫疗法，获FDA ODD+RPDD+FTD三重认定 IIb完成，BLA滚动递交中，目标2026年9月冲刺FDA加速批准（三）突破性疗法认定（BTD，Breakthrough Therapy Designation）通俗解读：FDA授予的资格，基于初步临床证据显示该药较现有疗法有显著改善。获得BTD后FDA将全程指导开发、加速审评。不等于获批上市。 HS-20093（2025年1月获FDA BTD）：骨肉瘤领域唯一BTD药物 Letetresgene autoleucel（lete-cel）：靶向NY-ESO-1的TCR-T疗法，获FDA BTD，针对黏液样/圆细胞脂肪肉瘤（MRCLS）和滑膜肉瘤四、临床阶段潜力新药/联合治疗方案1. HS-20093（Risvutatug Rezetecan，B7-H3 ADC）——骨肉瘤首款破局新药 FDA状态：2025年1月获突破性疗法认定（BTD），暂无上市申报 NMPA状态：纳入NMPA（中国国家药品监督管理局）突破性治疗药物清单，2026年7月17日上市申请正式受理关键数据： 2026年7月28日：III期ARTEMIS-011达到PFS主要终点 2025 ESMO II期ARTEMIS-002：骨肉瘤12.0 mg/kg组cORR 17.2%（确认ORR 20.0%），mPFS 8.4个月，DCR 86.2%，中位DoR 19.8个月预计：2026年下半年在中国率先获批上市2. C019199（国产多靶点创新药） FDA状态：无FDA任何认定 NMPA状态：2026年7月正式启动骨肉瘤三线治疗III期注册临床（CTR20261818），是国内进度最快的自研骨肉瘤新药关键数据：2025年ASCO Ib期显示DCR达73.3%、mPFS达6个月，≥3级TRAE仅23.3%，安全性优异3. OST-HER2（减毒李斯特菌（Lm）载体肿瘤疫苗免疫疗法） FDA状态：获ODD+RPDD+FTD三重认定，BLA滚动递交中适应症：骨肉瘤术后肺转移复发预防关键进展：已完成IIb期临床，目标2026年9月冲刺FDA加速批准4. ALMB-0168（全球首创Cx43激活型单抗） FDA状态：早期临床阶段关键数据：2025年ASCO快速口头报告（摘要#11509），6mg/kg扩展剂量组DCR达90%（10例可评估：2例PR+7例SD），1例持续PR达17个月，≥3级TRAE仅3.7%5. SGN1（桑美威克®，全球首创溶瘤细菌） FDA状态：已获ODD+FTD 关键数据：2026年ASCO壁报（摘要#11530），25例≥2线化疗后晚期骨与软组织肉瘤总体DCR 76.0%，骨肉瘤亚组DCR 70.0%，软组织肉瘤亚组DCR 80.0%，全身联合局部给药组DCR达100%，中位PFS 11.5个月6. ABP1011T （多靶点 TKI） NMPA状态：国内开展局部晚期/转移性骨肉瘤IIb期临床（ChiCTR2500111060），仅中国临床布局申办方：上海爱博医药科技有限公司7. TAEST16001（国产TCR-T疗法）靶点：NY-ESO-1肿瘤抗原，适配HLA-A*02:01阳性的晚期软组织肉瘤患者关键进展：2026年4月正式启动确证性临床试验，是国内首个获批IND、首个纳入CDE突破性治疗品种、首个进入确证性临床的TCR-T产品覆盖范围：相较于仅覆盖滑膜肉瘤的Afami-cel，本品可覆盖NY-ESO-1阳性泛软组织肉瘤，适用人群更广8. Elraglusib（9-ING-41，新型GSK-3β抑制剂） FDA状态：2024年9月获软组织肉瘤ODD 临床进展：II期开放标签双队列试验（NCT04906876）正在入组，评估其联合吉西他滨+多西他赛用于≥10岁不可切除/转移性肉瘤患者9. Lifileucel（Amtagvi，TIL肿瘤浸润淋巴细胞疗法）数据公布：2026年2月24日适应症：晚期去分化脂肪肉瘤（DDLPS）和未分化多形性肉瘤（UPS）关键数据：首批6例可评估患者，单次输注确认ORR达50%；历史二线治疗ORR不足5% 后续计划：已启动专属注册性试验IOV-SAR-201，计划2026年Q3开启入组10. Tigilanol Tiglate（EBC-46，Stelfonta，瘤内注射） FDA状态：2024年2月获软组织肉瘤ODD 关键数据：Ⅱa期可注射病灶ORR达80%，其中52%病灶完全消融，6个月随访零复发11. 芦比替定双免疫三联方案（LINNOVATE）方案：芦比替定+纳武利尤单抗+伊匹木单抗关键数据：LINNOVATE I/II期研究（NCT05876715）数据于2024年公布，II期初治患者DCR、CBR均达100%，I期经治耐药患者DCR达80%12. Letetresgene autoleucel（lete-cel 自体 SPAR‑TCR‑T 细胞疗法）靶点：NY-ESO-1 适应症：黏液样/圆细胞脂肪肉瘤（MRCLS）和滑膜肉瘤关键进展：II期试验中期结果于2024年ASCO公布，已获FDA突破性疗法认定13. 卡博替尼+替莫唑胺（平滑肌肉瘤）关键数据：2026年1月《柳叶刀·肿瘤学》公布II期阳性结果，12周PFS率达74%14. 曲贝替定+纳武利尤单抗（NitraSarc）研究：德国GISG-15 II期研究（2023 ASCO 摘要11500），92例晚期STS患者关键数据：L型肿瘤（脂肪肉瘤+平滑肌肉瘤）6个月PFS率47.6%，延迟联合队列（先曲贝替定3周期再加免疫）PFS达9.8个月，优于早期联合队列的4.4个月15. 多柔比星+Zalifrelimab（CTLA-4）+Balstilimab（PD-1）研究：单臂II期（NCT04028063），35例晚期STS（2023 ASCO 摘要11501）关键数据：28例可评估患者6个月PFS率46.4%（95% CI: 27.0%-65.8%），ORR 36%，DCR 86%16. GLP-1通路激动剂（脂肪肉瘤新靶点）机制：可诱导脂肪肉瘤细胞终末分化；IGF-1/GLP-1通路为脂肪肉瘤全新治疗靶点状态：基础+转化研究，靶点验证阶段17. 卡博替尼单药维持（高级别子宫肉瘤）结果：阴性——无法延长生存，增加毒性五、退市/全球终止研发药物Tazemetostat（Tazverik，EZH2抑制剂）退市时间：2026年3月9日退市原因：SYMPHONY-1试验提示继发血液肿瘤安全风险超过获益范围：Ipsen自愿撤回所有FDA适应症，全球终止临床试验及商业化供应原适应症：上皮样肉瘤、滤泡性淋巴瘤、去分化脂肪肉瘤 CSCO（中国临床肿瘤学会）指南状态：曾在2022版CSCO指南中被推荐，2025版已移出核心推荐体系六、细分亚型专项突破① 脂肪肉瘤亚型精准突破二联方案（特瑞普利单抗+西达本胺）：2025 ESMO公布II期研究（NCT04025931），高分化/去分化脂肪肉瘤24例患者ORR达62.5%、DCR 95.8%、中位PFS 17.1个月、中位OS 43.0个月；其余亚型ORR仅13.3%、中位PFS仅3.6个月。整体人群ORR 30.4%、中位PFS 7.1个月。艾立布林+安罗替尼（ERAS研究，2025 ASCO 摘要11502 / 2025 ESMO 壁报2722P）：22例既往含蒽环类治疗后进展的晚期LPS/LMS患者，总体DCR达89.5%（17/19），脂肪肉瘤（LPS）亚组中位PFS达17.3个月，中位OS达25.8个月。三联进阶方案（西达本胺+特瑞普利单抗+安罗替尼）：国内多中心II期注册临床（ChiCTR213777），专门针对二线及以上失败的晚期软组织肉瘤。依托“表观遗传重编程+PD-1免疫激活+抗血管生成”三重协同机制。② ASPS腺泡状软组织肉瘤国产方案正式获批上市：2026年4月17日，正大天晴贝莫苏拜单抗（安得卫）+安罗替尼（福可维）联合方案获NMPA批准，用于晚期/不可切除腺泡状软组织肉瘤。进口免疫长期疗效实锤：2026年ASCO公布阿替利珠单抗II期研究（NCT03141684）3年超长随访数据，同步发表于J Clin Oncol。总人群（53例）ORR达35.8%，中位PFS为20.8个月，中位缓解持续时间（DOR）长达37.0个月。 ASPSCR1-TFE3融合亚型分层分析（47例可分型）：融合亚型占比 ORR 中位PFS 临床意义 Type1（TFE3基因的第4个外显子） 87%（41/47） 43.9% 28.3个月 TIL浸润高，免疫单药获益显著 Type2（TFE3基因的第3个外显子融合） 13%（6/47） 0% 7.5个月免疫应答差，进展风险高（HR=3.2）临床关键点：检测提示：FISH仅能检出TFE3重排，需RNA测序区分Type1/Type2亚型以预判免疫疗效治疗实操：治疗满2年无进展可考虑“药物假期”；进展后可换用阿替利珠单抗+贝伐珠单抗联合方案注：该分层为回顾性探索分析，Type2组样本量小（仅6例），不作绝对排除标准。 IBI110（抗LAG-3抗体）+信迪利单抗：2024年ASCO首次报告、2025年ASCO更新数据，在晚期ASPS中显示出疗效。纳武利尤单抗+伊匹木单抗：1例7岁ASPS患儿在帕唑帕尼停药后接受该双免疫方案，达到完全缓解（CR）。③ 平滑肌肉瘤卡博替尼+替莫唑胺：2026年1月《柳叶刀·肿瘤学》公布II期阳性结果，12周PFS率达74%，中位随访18个月，耐受性良好。 PARP抑制剂（子宫平滑肌肉瘤，BRCA2缺失）：2025年发表于JCO Precision Oncology的回顾性分析显示，在携带体细胞BRCA2缺失的子宫平滑肌肉瘤患者中显示出令人鼓舞的疗效。多柔比星+曲贝替定→曲贝替定维持（子宫平滑肌肉瘤）：该方案在一线治疗中显示出生存优势。④ NTRK融合肉瘤佐来曲替尼（ICP-723）：诺诚健华中国首款自主研发新一代TRK抑制剂，2025年底获批上市，不限癌种，全面覆盖携带NTRK融合的各类软组织肉瘤，儿童实体瘤适应症纳入优先审评。瑞普替尼（Repotrectinib）：2026年1月6日NMPA获批补充适应症，用于NTRK融合阳性晚期实体瘤，为多线治疗失败患者提供高阶备选方案。安瑞曲替尼（VC004，商品名：维迈妥®）：江苏威凯尔医药自主研发的新一代抗耐药TRK抑制剂。2026年6月4日，NMPA通过优先审评审批程序附条件批准其上市，用于成人和≥12岁青少年携带NTRK融合基因的局部晚期、转移性实体瘤患者。该药是全球第三款新一代TRK抑制剂，在关键注册临床试验中展现了卓越的有效性和安全性，可有效解决一代TRK抑制剂的耐药问题。⑤ 软骨肉瘤 Ozekibart（INBRX-109）：全球首款四价DR5激动剂单抗，斩获FDA ODD+FTD双认定。2026年4月向FDA递交BLA，6月15日正式受理，PDUFA目标日期2027年4月14日。注册性II期ChonDRAgon试验（NCT04950075）共入组206例经典型软骨肉瘤患者，ozekibart组中位PFS 5.52个月 vs 安慰剂2.66个月（HR 0.479, P<0.0001），DCR 54% vs 27.5%。软骨肉瘤目前尚无任何获批的系统性疗法。 IDH1抑制剂艾伏尼布（2023 ASCO）：13例IDH1突变晚期经典型软骨肉瘤，ORR 23.1%（1 CR + 2 PR + 7 SD），中位DoR 42.5个月，中位PFS 7.4个月，NCCN（美国国家综合癌症网络）指南已推荐用于IDH1突变软骨肉瘤。国产同步突破：海特生物埃普奈明DR5通路药物，2026年ASCO公布临床中期数据。⑥ 尤文肉瘤 Ozekibart联合伊立替康+替莫唑胺，后线疗效优异 Silmitasertib获批开展尤文肉瘤、骨肉瘤广谱临床研究国产KC1036多靶点药持续推进儿童青少年尤文肉瘤专属II期临床，入选CDE儿童肿瘤“星光计划”⑦ 泛软组织肉瘤一线治疗+维持双方案迭代一线方案（ALTN-III-04已获批）：安罗替尼联合表柔比星→安罗替尼维持，mPFS 8.57 vs 3.02个月（HR 0.30），2025年6月已获NMPA批准。 SAIS研究（信迪利单抗+多柔比星+异环磷酰胺，2024 ASCO 摘要11505）：复旦大学附属肿瘤医院罗志国教授团队。41例可评估患者（UPS、滑膜肉瘤、MLPS、DDLPS）ORR达68.3%（28/41）。亚组ORR：UPS 87.5%（7/8）、滑膜肉瘤65.0%（13/20）、MLPS 100%（3/3）、DDLPS 50%（5/10）。中位PFS 9.0个月，中位OS 19.9个月。一线后维持（TORANS II期，2026 ASCO）：特瑞普利单抗+安罗替尼维持治疗，51例患者24周PFS率74.5%、中位PFS 12个月、DCR 96.1%。七、早期/新靶点探索1. STEAP1 ADC（ADRX-0405）潜在首创STEAP1 ADC，2024年10月获FDA IND批准，2026年6月获NMPA IND批准。目前I期临床主要针对晚期实体瘤，STEAP1在尤文肉瘤中亦具靶向潜力。2. PRMT5/MAT2A代谢靶点抑制剂通过MTAP缺失合成致死机制发挥抗肿瘤作用，肉瘤中MTAP缺失率约28%。国内勤浩医药在该领域布局领先： GH56（MTA协同PRMT5抑制剂）：已获NMPA及FDA IND批准，正在中国开展I期临床 GH31（MAT2A抑制剂）：2026年1月获NMPA及FDA IND批准，已在中国启动I期临床试验（CTR20260195），全球开发权益已独家授权给美国吉利德科学公司 GH31与GH56联合：临床前研究显示两者联合在MTAP缺失的肿瘤中发挥了协同效应3. CD3免疫靶向双特异性抗体 Nivatrotamab（GD2×CD3双抗）：已在骨肉瘤中进入II期临床。AMG 509（STEAP1×CD3双抗）：在尤文肉瘤中具靶向潜力，仍处于I期临床阶段。4. 三特异性抗体（CS2009）基石药业全球首创PD-1/VEGF/CTLA-4三特异性抗体，后线经治晚期软组织肉瘤ORR 33.3%、DCR 66.7%。5. MDM2靶向（APG115+APG-2575）亚盛医药APG115联合Bcl-2抑制剂APG-2575，17例可评估复发难治儿童肉瘤中整体ORR 23.5%、DCR 70.6%。6. 艾普美妥司他（HH2853，上皮样肉瘤）国产EZH1/2双靶抑制剂，已获CDE纳入优先审评，拟用于不可手术的局部晚期或转移性经治上皮样肉瘤，为Tazemetostat退市后的国产替代方案。八、其他罕见肉瘤亚型年度小突破炎性肌纤维母细胞瘤：克唑替尼拓展1岁及以上儿童ALK阳性IMT适应症，儿童ORR达86%。卡波西肉瘤：纳武利尤单抗+伊匹木单抗双免疫方案在难治复发病例中展现治疗潜力。阿贝西利（2025 ASCO）在未经治卡波西肉瘤患者中亦显示出良好活性。 PEComa（血管周上皮样细胞瘤）：Nab-sirolimus是全球唯一获批的恶性PEComa系统治疗方案。 OVV-01（溶瘤病毒）：获FDA ODD+FTD双重认定，针对NY-ESO-1阳性晚期软组织肉瘤，4例可评估患者ORR 75%、2例CR，最长缓解超27个月。硬纤维瘤： Nirogacestat（Ogsiveo）：全球首款获批的硬纤维瘤口服靶向药，2025年ESMO肉瘤与罕见癌症大会公布III期DeFi研究4年长期随访数据，ORR进一步提升至45.7% Varegacestat（AL102）：NDA审评，PDUFA 2027.4.28 国产ES014（CD39/TGF-β双抗）：2026年2月获FDA ODD，7月完成II期首例入组，5例可评估患者ORR 40%、DCR 100% 腱鞘巨细胞瘤：匹米替尼（Pimicotinib）：全球III期MANEUVER研究，ORR达76.2%，已在中国获批 Vimseltinib：第25周ORR 40%，与已获批Pexidartinib的39%相当，肝脏安全性更优脊索瘤：2025 ASCO口头报告（摘要11503）公布卡瑞利珠单抗+阿帕替尼（“双艾”组合）治疗晚期/难治性脊索瘤的II期研究数据，ORR达24.2%（RECIST 1.1），DCR达90.9%，中位PFS为17.71个月。透明细胞肉瘤： Tebentafusp（TCR-CD3双特异性蛋白）：SARC045 II期研究（NCT06942442）正在评估其在HLA-A*02:01阳性晚期透明细胞肉瘤中的疗效伯瑞替尼（高选择性MET抑制剂）：2026年ASCO公布II期VEBrant研究结果，这是该亚型首次迎来有效的靶向治疗希望 DSRCT（促结缔组织增生性小圆细胞瘤）： T-DXd（德曲妥珠单抗，Enhertu）：2025 ASCO公布，在DSRCT中具有抗肿瘤活性 Enfortumab vedotin（Nectin-4 ADC）：2025年病例报告首次记录在1例非典型DSRCT患者中的持久缓解恩杂鲁胺（enzalutamide，AR靶向治疗）：研究显示雄激素受体是DSRCT的治疗靶点血管肉瘤： Botensilimab（Fc增强型CTLA-4抑制剂）+ Balstilimab（PD-1抗体）：2024年ESMO公布的I期研究中，血管肉瘤患者ORR达39%，中位缓解持续时间21.7个月硼中子俘获疗法（BNCT）：日本一项针对不可切除皮肤血管肉瘤的II期临床试验达到主要终点（2025 ESMO）泛罕见肉瘤双免疫 vs 帕唑帕尼（RAR-IMMUNE III期，2025 ESMO LBA98）：首个在罕见肉瘤亚型中头对头比较纳武利尤单抗+伊匹木单抗（N+I）vs 帕唑帕尼的随机III期试验。入组116例患者，涵盖16种罕见肉瘤亚型。N+I组ORR更高（15.3% vs 5.3%），安全性更优（≥3级TRAE 24% vs 47%），但PFS无显著差异。骨外黏液样软骨肉瘤（EMC）：舒尼替尼+纳武利尤单抗：2025年ASCO显示抗肿瘤活性帕唑帕尼：ORR 18%，中位PFS 19个月曲贝替定：II期亚组分析显示有效帕博利珠单抗：基于NGS指导，1例EMC患者达到近完全缓解 MPNST（恶性外周神经鞘膜瘤）： MEK+BET+CDK抑制剂三联方案：临床前研究显示在MPNST小鼠模型中显著缩小肿瘤 MTAP缺失靶点：MTAP在MPNST中缺失率约25%–50%，PRMT5抑制剂（TNG908/TNG462）在MPNST模型中展现出抗肿瘤活性免疫检查点抑制剂：2025年病例报告显示ICI对携带NF2突变的MPNST患者具有临床获益黏液纤维肉瘤（MFS）：2024年病例报告显示，伊匹木单抗+纳武利尤单抗双免疫联合治疗在转移性MFS中具有疗效 GIST（胃肠道间质瘤）： Bezuclastinib+舒尼替尼：优先审评，PDUFA 2026.11.30 奥雷巴替尼（Olverembatinib）：2024年ASCO口头报告显示，治疗SDH缺陷型GIST的临床获益率（CBR）达92.3%（26例中6例PR、18例SD）横纹肌肉瘤（RMS） AMI463资格认定：于2024年4月获得FDA授予的孤儿药认定（ODD），用于治疗软组织肉瘤。该药物的开发特别针对横纹肌肉瘤 ABSK012：一款靶向FGFR4突变的新药，已于2026年获得FDA批准，可开展针对包括FGFR4突变横纹肌肉瘤在内的晚期实体瘤的I期临床试验安罗替尼（Anlotinib）：作为中高危横纹肌肉瘤儿童的维持治疗，正在中国开展II期临床研究（ALTER-02）仑伐替尼（Lenvatinib）联合依维莫司（Everolimus）：一项I/II期研究在复发性/难治性儿童实体瘤中进行探索，在横纹肌肉瘤患者中观察到了部分缓解未分化多形性肉瘤（UPS）卡瑞利珠单抗联合化疗：在2026年ASCO公布的II期研究中，该方案一线治疗晚期UPS的ORR为29.2%，中位无进展生存期（PFS）为8.4个月帕博利珠单抗（Keytruda）：在SARC028试验中，UPS单药ORR“40%”只在最初10例小样本成立；“全UPS队列23%” 信迪利单抗联合化疗：信迪利单抗+多柔比星+异环磷酰胺一线治疗晚期UPS/SS/MLPS/DDLPS，全4亚型ORR 68.3%；其中UPS亚组7/8达87.5%，mPFS 9.0月——是UPS一线免疫化疗里目前应答最高的中国数据之一纳武利尤单抗（Opdivo）联合伊匹木单抗（Yervoy）：双免疫联合治疗的UPS，ORR可达16.6% Lifileucel（TIL疗法）：在针对晚期UPS的小规模试验中，ORR达50%。表柔比星+异环磷酰胺+纳武利尤单抗（Nivolumab）：一项研究显示，该联合方案作为晚期UPS的一线治疗“安全、可行且高度有效” 帕博利珠单抗（Pembrolizumab）联合放疗：在SU2C-SARC032试验中，对于局部高风险UPS患者，在术前放疗的基础上加用帕博利珠单抗，显著改善了无病生存期（DFS）九、补充行业趋势治疗模式转变：进展型硬纤维瘤临床共识逐步减少手术优先，转向全身靶向药物长期管理精准检测普及：基因融合检测、ctDNA动态监测成为晚期肉瘤分层、疗效预测核心手段疗法多元化：小分子靶向、双特异性抗体、ADC（抗体偶联药物）、TCR-T、TIL（肿瘤浸润淋巴细胞）、免疫激动剂多管线同步推进，软骨肉瘤、硬纤维瘤等既往无药亚型迎来首款潜在系统治疗退市风险警示：Tazemetostat因安全性问题全球退市，提醒关注药物长期安全性演变附：缩略语索引缩略语英文全称中文全称通俗理解 FDA Food and Drug Administration 美国食品药品监督管理局美国药品审批机构 NMPA National Medical Products Administration 中国国家药品监督管理局中国药品审批机构 CDE Center for Drug Evaluation 国家药监局药品审评中心 NMPA下属审评部门 ODD Orphan Drug Designation 孤儿药认定 FDA鼓励罕见病药物研发的优惠政策 RPDD Rare Pediatric Disease Designation 罕见儿科疾病认定儿童罕见病的加速通道 FTD Fast Track Designation 快速通道认定 FDA加速开发与审评的通道 BTD Breakthrough Therapy Designation 突破性疗法认定基于早期数据显著优于现有疗法 NDA New Drug Application 新药申请小分子药物的FDA上市申请 BLA Biologics License Application 生物制品许可申请生物药的FDA上市申请 PDUFA Prescription Drug User Fee Act 处方药使用者付费法案 PDUFA日期=FDA审评截止日 IND Investigational New Drug 新药临床试验申请获批后方可开展人体试验 ADC Antibody-Drug Conjugate 抗体偶联药物 “生物导弹”精准递送化疗药 TCR-T T Cell Receptor-Engineered T Cell T细胞受体工程化T细胞改造T细胞识别肿瘤抗原 TIL Tumor-Infiltrating Lymphocyte 肿瘤浸润淋巴细胞从肿瘤组织中提取的免疫细胞本文基于FDA官网、NMPA/CDE公开数据、Targeted Oncology、ASCO、ESMO等权威来源整理，仅作行业科普，不构成临床治疗建议。整理不易，希望看到最后的你别忘了“一键三连”！ *本文只作资料分享整理，不是治疗方案推荐，如需获得疾病治疗方案指导，请咨询正规医院医生。文末左下角，有原文链接寻找治疗机会、陪诊代诊病理送检、基因检测和药敏检测，找希希爸都OK 微信号：15015700173

100 项与 Elraglusib 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D12304	-	-	DB16047

研发状态

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适应症	最高研发状态	国家/地区	公司	日期
晚期恶性实体瘤	临床2期	-	Actuate Therapeutics, Inc.	2026-10-01
转移性胰腺导管腺癌	临床2期	美国	Mayo Clinic	2025-05-30
转移性黑色素瘤	临床2期	美国	Actuate Therapeutics, Inc.	2023-05-31
转移性黑色素瘤	临床2期	美国	Lantern Pharma, Inc.	2023-05-31
转移性胰腺腺癌	临床2期	美国	Actuate Therapeutics, Inc.	2022-03-21
转移性骨肉瘤	临床2期	美国	Actuate Therapeutics, Inc.	2022-03-01
小细胞肉瘤	临床2期	美国	Actuate Therapeutics, Inc.	2022-03-01
晚期胰腺腺癌	临床2期	美国	Actuate Therapeutics, Inc.	2022-01-26
晚期胰腺腺癌	临床2期	美国	Incyte Corp.	2022-01-26
胰腺导管腺癌	临床2期	美国	Actuate Therapeutics, Inc.	2022-01-26

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05010629 (CTgov)	临床2期	涎腺癌 \| 转移性肿瘤 \| 唾液腺癌复发 ... 更多	32	9-ING-41+Carboplatin (9-ING-41 + Carboplatin (Part I))	遞廠憲鹹繭壓構鬱製鹽(壓醖壓顧艱繭壓遞願鹽) = 築構遞遞窪製網蓋鑰構遞齋憲網窪鑰蓋夢膚憲 (鹹觸鹽鑰製觸醖壓膚願, 遞觸網願餘鹽選鹹餘範 ~ 觸選齋夢襯築蓋衊製憲) 更多	-	2026-08-07
				9-ING-41+Carboplatin+Pembrolizumab (Pembrolizumab + 9-ING-41 + Carboplatin (Part II))	遞廠憲鹹繭壓構鬱製鹽(壓醖壓顧艱繭壓遞願鹽) = 選簾膚鹹衊鑰選餘製鹹遞齋憲網窪鑰蓋夢膚憲 (鹹觸鹽鑰製觸醖壓膚願, 築鹹網齋顧衊窪襯獵淵 ~ 壓鹽繭築簾鏇醖構構齋) 更多
NCT05077800 (ASCO2026)	临床2期	转移性胰腺导管腺癌	49	FFX alone	憲窪簾鹽壓膚憲艱選膚(觸鹽鹹遞夢鹹襯簾齋襯) = 膚顧憲淵糧齋網製夢積窪獵範鏇鬱糧壓艱鏇繭 (簾蓋鹹網淵築淵選構壓, 1.1 ~ NR) 更多	积极	2026-05-29
				FFX plus LOS (50 mg daily)	憲窪簾鹽壓膚憲艱選膚(觸鹽鹹遞夢鹹襯簾齋襯) = 鏇蓋齋齋廠壓網鬱廠選窪獵範鏇鬱糧壓艱鏇繭 (簾蓋鹹網淵築淵選構壓, 1.6 ~ 9.9) 更多
NCT03678883 (1801 Part 3B, ASCO2026)	临床2期	转移性胰腺导管腺癌	233	Elraglusib plus Gemcitabine/Nab-paclitaxel	糧網醖構鹹網鹹鏇觸鹹(鹹鹽鹽範艱醖蓋簾醖夢) = 繭鹹襯製膚鹹艱鹽構壓製鏇選願衊鹹獵襯蓋齋 (壓夢艱夢窪遞築網顧衊 ) 更多	积极	2026-05-29
				Gemcitabine/Nab-paclitaxel	糧網醖構鹹網鹹鏇觸鹹(鹹鹽鹽範艱醖蓋簾醖夢) = 衊獵夢簾壓衊獵遞醖觸製鏇選願衊鹹獵襯蓋齋 (壓夢艱夢窪遞築網顧衊 ) 更多
NCT03678883 (Pubmed \| Nat Med)	临床2期	转移性胰腺导管腺癌一线	233	Elraglusib + Gemcitabine + Nab-paclitaxel	餘廠築選顧廠築齋繭衊(簾簾遞艱鏇遞簾壓範憲) = 醖觸衊積鹹製網齋廠築夢餘繭壓餘廠廠蓋淵獵 (鏇鬱醖積衊鏇醖網廠膚 ) 更多	积极	2026-04-14
				Gemcitabine + Nab-paclitaxel	餘廠築選顧廠築齋繭衊(簾簾遞艱鏇遞簾壓範憲) = 淵製憲範繭衊壓夢築餘夢餘繭壓餘廠廠蓋淵獵 (鏇鬱醖積衊鏇醖網廠膚 ) 更多
NCT03678883 (1801 Part 3B, ASCO_GI2026)	临床2期	转移性胰腺癌一线 KRAS \| TP53 \| CDKN2A	170	Elraglusib/Gemcitabine/nab-paclitaxel	襯觸餘鏇艱鬱艱膚壓鑰(觸鏇醖製簾願簾網淵築) = KRAS and TP53 gene mutations were associated with worse overall survival (OS) in patients treated with elraglusib/GnP (p<0.05) but not in GnP-treated patients. 願鏇壓觸觸襯鏇網鹹鏇 (鬱壓繭觸網膚蓋顧鹹選 )	积极	2026-01-08
				Gemcitabine/nab-paclitaxel
NCT04239092 (Actuate-1902, GlobeNewswire) 人工标引	临床1期	肉瘤 \| 尤文肉瘤	10	elraglusib	憲膚餘襯製餘蓋鹹襯衊(鹹廠遞鹽願獵鹽製製餘) = 繭構鏇選壓蓋築選夢壓齋選遞鹹膚憲憲艱衊鏇 (範淵廠構糧壓鹽鹹淵餘 ) 更多	积极	2025-07-17
NCT05239182 (RiLEY, CTgov)	临床2期	胰腺腺癌 \| 胰腺导管腺癌 \| 晚期胰腺腺癌	7	9-ING-41+Abraxane+Gemcitabine+Retifanlimab	廠膚廠築鏇製鏇餘獵網 = 艱鏇鏇鏇糧壓選淵選簾觸鬱醖夢鑰膚積蓋鏇鹽 (淵網衊壓願網艱醖鏇顧, 艱齋鹽鹽範艱鹹襯廠願 ~ 廠夢鑰網糧顧鬱醖鏇遞) 更多	-	2025-07-17
NCT03678883 (1801 part 3B, ASCO2025)	临床2期	转移性胰腺导管腺癌 CA 19-9 levels \| IFNβ \| PD-L1	-	Elraglusib 9.3 mg/kg IV once weekly + Gemcitabine/nab-paclitaxel	顧範憲壓構醖壓齋醖鹽(網鹹選願積衊淵膚鑰鏇) = 夢簾築壓窪壓範鹽獵壓鑰夢繭鏇蓋範製糧願簾 (遞遞簾夢遞廠廠鏇艱蓋 ) 更多	积极	2025-05-30
				Gemcitabine/nab-paclitaxel	顧範憲壓構醖壓齋醖鹽(網鹹選願積衊淵膚鑰鏇) = 遞廠襯憲選夢蓋廠鹽齋鑰夢繭鏇蓋範製糧願簾 (遞遞簾夢遞廠廠鏇艱蓋 ) 更多
NCT03678883 (Actuate-1801 Part 3B, GlobeNewswire) 人工标引	临床2期	转移性胰腺癌一线	-	elraglusib + gemcitabine/nab-paclitaxel (GnP)	廠鑰襯製願簾顧糧膚繭(獵衊蓋選蓋遞齋遞廠網) = elraglusib in combination with gemcitabine/nab-paclitaxel (GnP) met the primary endpoints and achieved statistical significance in topline results. 艱糧鏇壓膚鬱繭壓鹹願 (窪觸醖網蓋鏇壓淵鑰鹽 ) 达到	积极	2025-05-09
				gemcitabine/nab-paclitaxel (GnP)
NCT05010629 (AACR2025)	临床2期	唾液腺肿瘤 tumor PD-L1 \| GSK-3β expression	32	Elraglusib	襯範製艱蓋構鏇衊鹹築(衊淵顧顧壓築鬱憲壓艱) = 夢憲鬱夢壓醖壓鬱窪艱憲選願選選餘窪築製鏇 (憲壓顧淵蓋壓觸鏇選顧, 2 ~ 25) 更多	积极	2025-04-29