(RTTNews) - Abbisko Therapeutics Co., Ltd. (02256.HK) announced positive preliminary Phase II results for avengratinib (ABSK061), used for the treatment for achondroplasia, a genetic condition that affects bone growth and is the most common form of dwarfism. The early data showed improvements in growth rates among treated children while maintaining a favorable safety profile.
Achondroplasia is caused by abnormal activity of the FGFR3 pathway, which limits normal bone growth. Lavengratinib is an orally available small-molecule FGFR2/3 inhibitor designed to target this pathway and promote skeletal growth.
The ongoing ABSK061-202 study is a Phase II, multicenter, open-label, dose-escalation trial evaluating once-daily lavengratinib in children aged 3 to 12 years with achondroplasia. Participants are expected to receive treatment for 78 weeks.
Preliminary results from the first and lowest-dose cohort showed encouraging signs of growth promotion. Seven children aged 6 to 12 years who received lavengratinib at 0.064mg/kg once daily for 27 weeks achieved a mean increase of 2.4 cm per year in annualized height velocity from baseline, while the responder rate reached 100%. A responder was defined as a participant achieving at least a 25% improvement in annualized height velocity. Higher-dose cohorts remain under evaluation.
Lavengratinib was generally well tolerated. The company reported no serious adverse events, no treatment discontinuations due to adverse events, and no observed safety issues commonly associated with FGFR pathway inhibition, including hyperphosphatemia and corneal toxicity.
The therapy uses Abbisko's proprietary oral mini-tablet formulation, with tablets measuring less than 3 mm in diameter, allowing administration with food or drinks and simplifying use in pediatric patients.
Abbisko said preliminary safety evaluations across the first three dose cohorts have been completed without major safety concerns. Additional six-month efficacy data from higher-dose cohorts are expected by the end of 2026.
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