Abbisko Therapeutics has reported preliminary results from a Phase II trial of lavengratinib (ABSK061), an oral FGFR2/3 inhibitor, in children with achondroplasia. In the first and lowest dose cohort, seven participants aged 6 to 12 who received 0.064 mg/kg once daily for 27 weeks recorded a mean rise of 2.4 cm/year in annualized height velocity from baseline, the company said. All seven met the trial's definition of a responder, which required an improvement of at least 25% in annualized height velocity from baseline. No FGFR1- or FGFR2-associated adverse events were observed in the cohort, and the drug was reported as well tolerated.
The trial, ABSK061-202, is a Phase II, multicenter, open-label, dose-escalation study in children aged 3 to 12 with achondroplasia. Participants take lavengratinib orally once a day for a planned 78 weeks. Abbisko said all seven participants in the first cohort, who are aged 6 to 12, have completed six months of treatment, while participants in higher dose cohorts are still receiving treatment.
No serious adverse events or treatment discontinuations because of adverse events have been reported in the study so far, and no cases of hyperphosphatemia or corneal toxicity have been observed. The preliminary safety evaluation of the first three dose cohorts has been completed. Six-month efficacy and safety results are expected by the end of 2026.
The drug is given as mini-tablets measuring less than 3mm in diameter, compared with conventional tablets of roughly 8mm to 10mm, a format the company says makes administration easier for children and allows the tablets to be taken with food and drink. Lavengratinib holds Rare Pediatric Disease Designation and Orphan Drug Designation from the U.S. Food and Drug Administration, and the company describes it as the first FGFR2/3 inhibitor to enter clinical trials.
