October 7, 2025 — Leads & Copy — RIBOMIC, Inc. (TOKYO:4591) has announced positive results from its Phase 2 clinical trial of umedaptanib pegol (anti-FGF2 aptamer) in pediatric patients (ages 5-14) with achondroplasia (ACH). The trial demonstrated a positive impact on patient growth rate in the high-dose (0.6 mg/kg) subcutaneous injection group (once biweekly).
The Phase 2 clinical trial included Cohort 1, which received weekly subcutaneous injections of a low dose (0.3 mg/kg), and Cohort 2, which received a high dose (0.6 mg/kg) every other week. The trial aimed to confirm the efficacy, safety, and durability of umedaptanib pegol.
In Cohort 2, all 6 subjects completed the trial, and 5 subjects showed increased height growth rate, with 2 showing significant increases of +5.0 cm/year and +2.0 cm/year. The mean height growth rates for Cohorts 1 and 2 were +1.5 cm/year and +1.4 cm/year, respectively, comparable to Voxzogo®. No safety concerns arose during the trial.
RIBOMIC is planning a Phase 3 clinical trial to further verify the efficacy of umedaptanib pegol. The Phase 3 trial is scheduled to begin in the first quarter of fiscal year 2026 and conclude within fiscal year 2027, with the goal of obtaining approval by the end of fiscal year 2028.
Umedaptanib pegol has received orphan drug designation from the Ministry of Health, Labor and Welfare in Japan.
RIBOMIC specializes in the discovery and development of aptamer therapeutics. The company’s core drug discovery platform, the RiboART system, can be used to discover many types of aptamer drugs.
ABOUT UMEDAPTANIB PEGOL:
Umedaptanib pegol is a novel oligonucleotide-based aptamer formerly designated RBM-007, with potent anti-FGF2 (fibroblast growth factor 2) activity. This drug has received orphan drug designation from the Ministry of Health, Labour and Welfare.
ABOUT ACHONDROPLASIA:
Achondroplasia is a disease caused by a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene, which makes FGFR3 more easily activated. This leads to excessive influx of FGF signals, inhibiting the normal development of cartilage and other tissues, resulting in short stature accompanied by limb shortening. It is a rare disease with an incidence of approximately 1 in 25,000 newborns and is designated as an intractable disease. The development of effective new drugs is urgently needed.
Source: RIBOMIC
