Heyu-B (02256): Clinical trial of ABSK061, an FGFR2/3 inhibitor for the treatment of achondroplasia completed the first patient

Zhitong Finance App News, Heyu-B (02256) announced that on December 16, 2025, Shanghai Heyu Biomedical Technology Co., Ltd. (“Heyu Pharmaceutical”) announced that its highly selective small molecule FGFR2/3 inhibitor ABSK061 has successfully completed the first patient administration in a phase II clinical trial for children with achondroplasia (“ACH”) aged 3-12.

ACH is an autosomal hereditary rare disease that causes severe growth and development impairment in children. Studies have shown that the pathogenesis of ACH stems from abnormal activation of FGFR3 caused by fibroblast growth factor receptor 3 (“FGFR3”) gene mutations, thereby inhibiting the normal ossification process of cartilage. Targeted inhibitors are expected to bring more accurate and effective treatment options for ACH patients.

ABSK061 is a highly active and highly selective small molecule FGFR2/3 inhibitor independently developed by Heyu Pharmaceutical. In preclinical studies, it has shown significant target inhibitory activity, good pharmacokinetic characteristics, and safety advantages. Its oral administration method has significant advantages in terms of convenience and treatment compliance, especially for pediatric patients, making ABSK061 a potentially valuable candidate for the treatment of pediatric and adolescent ACH patients.

The first patient administration was completed as part of an open-label phase II clinical study aimed at comprehensively evaluating the safety, tolerability, pharmacokinetics, and initial efficacy of ABSK061 in children aged 3 to 12 with ACH. The study was approved for clinical trial drugs (“IND”) by the China National Drug Administration (“NMPA”) Drug Evaluation Center (“CDE”) in March 2025.