Ribomic Expands Clinical Trial Sites for Achondroplasia Treatment umedaptanib pegol

TOKYO, Jun 29 (Pulse News Wire) – RIBOMIC Inc. (4591.T) announced today that it has secured IRB approval at six medical institutions for its Phase III clinical trial of umedaptanib pegol, an anti-FGF2 aptamer drug aimed at treating children aged two to fourteen with achondroplasia (ACH).

With these approvals, patient recruitment for the trial can now begin. The company initiated the trial in early June, receiving initial IRB approval on June 3. As the disease affects a limited number of patients and involves minors undergoing growth processes, high-specialized expertise and careful handling are essential. RIBOMIC expresses gratitude towards participating institutions and commits to working diligently to develop a treatment for ACH patients.

Umedaptanib pegol, designated as a rare disease drug by the Ministry of Health, Labour and Welfare, is expected to offer a fundamental treatment approach targeting the underlying mechanism of ACH by strongly inhibiting FGF2 function. The trial's objective is to cover the entire country through a total of sixteen planned sites. Further details on the trial can be found in the March 18, 2026 press release titled “Notice of Commencement of Domestic Phase III Clinical Trial Application for umedaptanib pegol.” There will be no impact on the fiscal year ending March 2027 earnings due to this expansion. Achondroplasia, characterized by genetic mutations affecting FGFR3 receptors leading to excessive FGF signaling and impaired bone development, occurs in approximately one out of every 25,000 newborns, making it a rare condition requiring effective treatments.