#China #Shanghai #Abbisko #achondroplasia #Lavengratinib

Abbisko Therapeutics Reports Encouraging Phase 2 Findings

Abbisko Therapeutics Co., Ltd., trading under HKEX code 02256.HK, recently unveiled promising preliminary results from its Phase 2 clinical trial of lavengratinib (ABSK061), a targeted therapy aimed at addressing achondroplasia (ACH) in pediatric patients. The announcement, made on September 21, 2026, reveals significant advancements in growth metrics among treatment participants.

Study Overview

The ABSK061-202 study is a multicenter, open-label, dose-escalating trial evaluating the safety and efficacy of lavengratinib administered orally. The key focus is on children aged 3 to 12 years diagnosed with achondroplasia. In the initial cohort, children aged 6 years and older were given the drug at a low dose of 0.064 mg/kg once daily for 27 weeks, yielding remarkable results.

Growth Outcomes

After the treatment period, children reported a mean increase of +2.4 cm/year in their annualized height velocity (AHV) compared to baseline measurements. Impressively, this outcome came with a 100% responder rate, defined as participants achieving at least a 25% improvement in their height velocity. This effectiveness was observed without any recorded adverse events linked to FGFR1 or FGFR2, indicating that lavengratinib was well tolerated by the children involved.

Safety and Tolerability

Considering the inherent risks associated with FGFR pathway inhibitors, the study has so far indicated favorable safety and tolerability profiles for lavengratinib. To date, there have been no reports of serious adverse events or treatment discontinuations attributable to adverse effects. Notably, the investigators noted that there are no safety signals of concern linked to FGFR inhibition, which is often a pressing issue with other therapies.

Innovative Formulation

What sets lavengratinib apart is Abbisko's innovative mini-tablet formulation, designed for ease of administration in younger patients. With each tablet measuring less than 3mm in diameter—significantly smaller than the conventional tablets—administration becomes much simpler, improving compliance among pediatric patients. The mini-tablets can be easily ingested with food and liquids, enhancing their usability.

Future Expectations

As the clinical trial continues, participants in higher dose cohorts remain under treatment, with six-month efficacy and safety results expected to be available by the end of 2026. These upcoming results will serve as crucial indicators for the drug's clinical potential and may further the understanding of lavengratinib as a viable treatment for children afflicted with achondroplasia.

About Lavengratinib

Lavengratinib represents a novel approach as a highly selective, orally bioavailable small-molecule inhibitor targeting FGFR2 and FGFR3, independently developed and fully owned by Abbisko Therapeutics. This groundbreaking compound is recognized as the first FGFR2/3 inhibitor entering clinical trials globally. Unlike older pan-FGFR inhibitors, which often show limited efficacy due to adverse side effects associated with FGFR1 inhibition, lavengratinib aims to offer improved therapeutic outcomes by selectively minimizing FGFR1 activity while maintaining its effectiveness against FGFR2 and FGFR3.

Recently, lavengratinib has also garnered significant recognition, having received Rare Pediatric Disease Designation and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA). As the Phase 2 clinical trial progresses, there is a growing optimism surrounding its potential benefits for young patients living with achondroplasia, making it a pivotal point of interest in pediatric healthcare advancements.