2025年12月16日，上海和誉生物医药科技有限公司（以下简称“和誉医药”，港交所代码：02256）今日宣布，其自主研发的高选择性小分子FGFR2/3抑制剂ABSK061针对3-12岁软骨发育不全（ACH）儿童患者的II期临床试验已顺利完成首例患者给药。
ACH是导致儿童严重生长发育障碍的常染色体遗传性罕见病，目前缺乏有效治疗方法。研究表明，ACH的发病机制主要源于成纤维细胞生长因子受体3（FGFR3）基因突变引起的FGFR3异常激活，从而抑制软骨的正常骨化过程1。因此，针对ACH的分子病因开发靶向抑制剂有望为ACH患者带来精准治疗新希望。
ABSK061是和誉医药自主研发的高活性、高选择性小分子FGFR2/3抑制剂，在临床前研究中已显示出显著的靶点抑制活性、良好的药代动力学特征以及安全性优势。其口服给药方式有望极大提升患者，尤其是儿童患者的用药便利性和治疗依从性，在儿童及青少年ACH患者的治疗中具备良好潜力。
此次完成首例给药的是一项开放标签II期临床研究，旨在系统评估ABSK061在3-12岁ACH儿童患者中的安全性、耐受性、药代动力学特征及初步有效性。该研究于2025年3月获得中国国家药品监督管理局（NMPA）药品审评中心（CDE）的临床试验许可。
关于ABSK061
ABSK061是和誉医药独立自主研发并拥有全球知识产权的一款新一代口服、高活性、高选择性小分子FGFR2/3抑制剂，也是全球范围内第一款进入临床的FGFR2/3选择性抑制剂。首代泛FGFR抑制剂已在针对多种携带FGFR2/3变异的肿瘤中展现出临床疗效并在全球范围内逐步获批上市，但安全窗及药效均受限于FGFR1抑制相关副作用。通过降低对FGFR1的抑制以及保持对FGFR2/3的高活性，ABSK061作为第二代FGFR抑制剂有望在临床上取得更好的安全窗及疗效。
参考文献
1. Savarirayan R, et al. International Consensus Statement on the diagnosis, multidisciplinary management and lifelong care of individuals with achondroplasia. Nat Rev Endocrinol. 2022 Mar;18(3):173-189.
Abbisko Therapeutics Completes First Patient Dosing in Phase II Clinical Study of ABSK061, FGFR2/3 Inhibitor, for the Treatment of Achondroplasia
16 December 2025, Abbisko Therapeutics Co., Ltd. ("Abbisko Therapeutics" hereafter, HKEX code: 02256.HK) today announced that the first patient has been successfully dosed in the Phase II clinical study evaluating ABSK061, a highly selective small-molecule FGFR2/3 inhibitor, in children ages 3 to 12 with achondroplasia (ACH).
ACH is a rare autosomal genetic disorder that causes severe growth and developmental impairments. Research has shown that the pathogenesis of ACH is driven by aberrant activation of the fibroblast growth factor receptor 3 (FGFR3) caused by FGFR3 gene mutations, which suppress normal endochondral ossification1. Targeted inhibitors offer the potential to deliver more precise and effective treatment options for ACH patients.
ABSK061, independently developed by Abbisko Therapeutics, is a highly potent and selective small-molecule FGFR2/3 inhibitor. It has demonstrated robust target inhibitory activity, favorable pharmacokinetic properties, and a promising safety profile in preclinical studies. Its oral administration offers significant advantages in terms of convenience and treatment compliance—particularly for pediatric patients—and positions ABSK061 as a potentially valuable therapeutic candidate for children and adolescents with ACH.
The first-patient dosing was completed as part of an open-label Phase II clinical study designed to comprehensively evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of ABSK061 in children ages 3 to 12 with ACH. The study received Investigational New Drug (IND) clearance from the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) in March 2025.
About ABSK061
ABSK061 is a novel, orally bioavailable, highly potent and selective small molecule inhibitor of FGFR2 and FGFR3 independently discovered and wholly-owned by Abbisko Therapeutics. It is the first FGFR2/3 inhibitor to enter clinical trials globally. First-generation pan-FGFR inhibitors demonstrated clinical efficacy in multiple tumors carrying FGFR2/3 variants and have steadily gained regulatory approval globally. However, the therapeutic window of pan-FGFRs and their clinical efficacy have been limited by side effects associated with FGFR1 inhibition. By reducing FGFR1 activity while maintaining potency against FGFR2 and FGFR3, ABSK061 is expected to achieve a wider therapeutic window with improved clinical efficacy as a new-generation of FGFR 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.
Reference
1. Savarirayan R, et al. International Consensus Statement on the diagnosis, multidisciplinary management and lifelong care of individuals with achondroplasia. Nat Rev Endocrinol. 2022 Mar;18(3):173-189.
