和誉医药FGFR4抑制剂依帕戈替尼完成全球多中心I期临床扩展阶段美国首例患者给药

目前，晚期或不可切除的HCC患者的标准治疗主要包括免疫检查点抑制剂（ICI）和多靶点激酶抑制剂（mTKI）等。研究表明，约30%的HCC患者存在FGF19过表达，该人群在现有ICI和mTKI治疗后的预后较差，存在显著未满足的临床需求。因此，针对FGF19/FGFR4信号通路的精准靶向治疗，被认为有望为该细分人群带来新的治疗突破。

依帕戈替尼是和誉医药自主研发的一款高选择性、口服小分子FGFR4抑制剂，专为FGF19过表达的晚期肝细胞癌患者设计。ABSK-011-101是一项开放标签的全球多中心I期临床研究（NCT04906434），旨在评估依帕戈替尼在晚期实体瘤患者中的安全性、耐受性及药代动力学特征。研究分为剂量递增和剂量扩展两个阶段，此次首例患者给药基于已完成的剂量递增阶段所确定的推荐剂量，进一步评估美国人群中依帕戈替尼在FGF19过表达晚期HCC患者中的安全性、耐受性及初步疗效。

在中国人群中，ABSK-011-101研究数据显示，依帕戈替尼单药治疗在FGF19过表达晚期HCC患者中展现出持续的抗肿瘤活性以及良好的安全性和耐受性。尤其在ICI和mTKI经治亚组人群中，依帕戈替尼单药疗效表现尤为突出，客观缓解率（ORR）达到46.7%，中位无进展生存期（mPFS）为5.5个月。上述研究成果已于2024年欧洲肿瘤内科学会（ESMO）年会期间正式公布。

2025年5月，依帕戈替尼获得中国国家药品监督管理局药品审评中心（CDE）授予的突破性疗法认定（BTD），并已启动覆盖全国50余家研究中心的关键注册临床研究（文末附临床招募信息）。中国科学院陈孝平院士对该项目给予高度评价，认为依帕戈替尼有望成为全球首个真正意义上的精准靶向肝癌药物，为患者带来长期生存获益。

此外，和誉医药在2025年欧洲肿瘤内科学会胃肠道肿瘤大会（ESMO-GI）上公布了依帕戈替尼联合阿替利珠单抗治疗HCC的II期临床研究最新进展。研究结果显示，在初治及ICI经治的FGF19过表达HCC患者中，该联合治疗方案均展现出显著的抗肿瘤活性，ORR超过50%，mPFS超过7个月。同时，联合用药整体安全性可控，未观察到新的安全性信号，提示依帕戈替尼在靶向FGF19/FGFR4通路的基础上，具备与免疫治疗形成协同增效的潜力，显示出冲击HCC一线治疗方案的临床价值与潜力。

未来，和誉医药将加速依帕戈替尼的全球临床开发与商业化布局，深化国际多中心研究网络建设，致力于为全球HCC患者带来更具突破性的创新治疗方案。

依帕戈替尼关键注册临床患者招募

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关于依帕戈替尼（Irpagratinib/ABSK-011）

依帕戈替尼（ABSK-011）是一种高选择性小分子FGFR4抑制剂，被开发用于治疗FGF19过表达的晚期肝细胞癌（aHCC）。研究表明，全球约30%的HCC患者存在FGF19过表达。开发针对该信号通路的靶向疗法代表了治疗HCC的一种新颖的创新方法。

当前，全球尚无FGFR4抑制剂获批上市，根据弗若斯特沙利文的分析，依帕戈替尼凭借其在竞争格局中的领先地位，有望成为首个治疗FGF19过表达HCC患者的突破性药物。

除单药之外，和誉医药同时在探索联合抗PD-L1抗体阿替利珠单抗（由F. Hoffmann-La Roche Ltd.及罗氏（中国）投资有限公司制造）的II期试验。在2025年ESMO-GI大会上，和誉医药发布了联合用药治疗aHCC患者的最新临床试验数据，其中，依帕戈替尼联用阿替利珠单抗队列在初治及既往接受过ICI治疗的FGF19过表达HCC患者中，客观缓解率（ORR）均超过50%，中位无进展生存期（mPFS）超过7个月。

Abbisko Therapeutics' FGFR4 Inhibitor Irpagratinib Completed First U.S. Patient Dosing in the Expansion Part of a Global Phase I Study

11 February 2026, Abbisko Therapeutics Co., Ltd. ("Abbisko Therapeutics" hereafter, HKEX code: 02256.HK) today announced that the first patient in the United States has been successfully dosed in the expansion part of the global, multicenter Phase I clinical study (ABSK-011-101) of its independently developed, highly selective small-molecule FGFR4 inhibitor irpagratinib (ABSK-011) for patients with FGF19 overexpression advanced hepatocellular carcinoma (HCC). Previously, irpagratinib was granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA), which is expected to further accelerate its global clinical development.

Currently, the standard therapy for patients with advanced or unresectable HCC include immune checkpoint inhibitors (ICIs) and multi-targeted kinase inhibitors (mTKIs), etc. Studies indicate that approximately 30% of HCC patients exhibit FGF19 overexpression, a subgroup associated with relatively poor outcomes following existing ICIs and mTKIs treatment, underscoring a substantial unmet medical need. Precision therapies targeting the FGF19/FGFR4 signaling pathway are therefore considered a promising strategy to deliver meaningful clinical benefit to this defined patient population.

Irpagratinib is a highly selective, oral small-molecule FGFR4 inhibitor developed by Abbisko Therapeutics for the treatment of advanced HCC with FGF19 overexpression. ABSK-011-101 is an open-label, global, multicenter Phase I study (NCT04906434) designed to assess the safety, tolerability, and pharmacokinetics of irpagratinib in patients with advanced solid tumors. The study consists of dose-escalation and dose-expansion parts. In the U.S., patients will be treated at the recommended dose determined from the completed dose-escalation part to further assess safety, tolerability, and preliminary efficacy in FGF19 overexpression advanced HCC.

Data from the China cohort of ABSK-011-101 demonstrated that irpagratinib monotherapy achieved durable antitumor activity with a favorable safety and tolerability profile in patients with FGF19 overexpression advanced HCC. Notably, in the subgroup of patients previously treated with ICIs and mTKIs, irpagratinib showed particularly encouraging efficacy, with an objective response rate (ORR) of 46.7% and a median progression-free survival (mPFS) of 5.5 months. These results were presented at the 2024 European Society for Medical Oncology (ESMO) Congress.

In May 2025, irpagratinib received Breakthrough Therapy Designation (BTD) from the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA), and a pivotal registration clinical study was initiated across more than 50 research centers nationwide. Academician Xiaoping Chen of the Chinese Academy of Sciences has previously spoken highly of the program, noting that irpagratinib has the potential to become the world's first truly precision-targeted therapy for liver cancer, offering long-term survival benefits to patients.

In addition, Abbisko presented the latest Phase II clinical data of irpagratinib in combination with atezolizumab for the treatment of HCC at the 2025 ESMO Gastrointestinal Cancers Congress (ESMO-GI). The combination demonstrated robust antitumor activity in both treatment-naïve and ICI-pretreated patients with FGF19 overexpression HCC, achieving an ORR exceeding 50% and an mPFS of more than 7 months. The overall safety profile was manageable, with no new safety signals observed, suggesting potential synergistic benefit with immunotherapy and supporting the regimen's promise as a candidate for first-line HCC treatment.

Looking ahead, Abbisko will accelerate the global clinical development and commercialization of irpagratinib, strengthen our international multi-center clinical network, and deliver transformative therapeutic options for patients with HCC worldwide.

About Irpagratinib (ABSK-011)

Irpagratinib is a highly-selective FGFR4 small molecule inhibitor designed to target overexpression of the FGF19 signaling pathway. Several epidemiological studies indicate that approximately 30% of HCC patients worldwide exhibit FGF19 overexpression. Development of targeted therapies against FGFR4 represent an innovative and novel approach to the treatment of HCC.

To date, no FGFR4 inhibitor has been granted regulatory approval globally. According to Frost & Sullivan, irpagratinib is expected to become the first breakthrough treatment for the treatment of HCC patients with FGF19 overexpression.

In addition to monotherapy, Abbisko Therapeutics is exploring irpagratinib in combination with atezolizumab, an anti-PD-L1 antibody manufactured by F. Hoffmann-La Roche and Roche (China), in a Phase II study. At the the 2025 ESMO GI Congress, Abbisko presented clinical data showing that the combination of irpagratinib and atezolizumab achieved an objective response rate (ORR) exceeding 50% and a median progression-free survival (mPFS) of more than 7 months in FGF19-overexpressing HCC patients previously treated with immune checkpoint inhibitors.

About Abbisko Therapeutics

Founded in April 2016, Abbisko Therapeutics Co., Ltd. (HKEX: 02256.HK), is an oncology-focused biopharmaceutical company based in Shanghai that is dedicated to the discovery and development of innovative medicines to treat unmet medical needs in China and globally. The Company was established by a group of seasoned drug hunters with rich research & development and managerial expertise from top multinational pharmaceutical companies. Since its founding, Abbisko Therapeutics has built an extensive pipeline of innovative programs focused on precision oncology and immuno-oncology.

Please visit www.abbisko.com for more information.

关于和誉

和誉医药（香港联交所代码：02256）成立于2016年，是一家立足中国，着眼全球的创新药研发公司。公司的创始人和管理团队拥有多年顶尖跨国药企的研发和管理经验，并参与了多个临床及上市新药的研发。和誉医药专注于肿瘤新药研发，以小分子肿瘤精准治疗和小分子肿瘤免疫治疗药物为核心，着眼病患及医药市场的需求，秉承国际新药开发的理念和标准，致力于开发新颖及高潜力药物靶点的潜在first-in-class或best-in-class创新药物，用于改善中国及全球病人的生活质量。自成立以来，和誉医药已经建立了丰富的创新产品管线，涵盖肿瘤精准治疗领域以及肿瘤免疫治疗领域。

更多信息，欢迎访问 www.abbisko.com。