What's Happening?
RIBOMIC, a clinical-stage pharmaceutical company, has announced the successful completion of its Phase 2 clinical trial for umedaptanib pegol, an anti-FGF2 aptamer, in pediatric patients with achondroplasia. The trial involved two cohorts, with Cohort 2 receiving a higher dose of 0.6 mg/kg biweekly, demonstrating significant improvements in annualized growth rates. Five out of six subjects in Cohort 2 showed increased height growth rates, with two subjects achieving growth rates of +5.0 cm/year and +2.0 cm/year. These results are comparable to the growth rates achieved by Voxzogo®, a currently approved treatment for achondroplasia. The trial confirmed the efficacy, safety, and durability of umedaptanib pegol, establishing proof-of-concept as a treatment for achondroplasia.
Why It's Important?
The successful Phase 2 trial of umedaptanib pegol is a significant development in the treatment of achondroplasia, a rare genetic disorder characterized by short stature. The promising results suggest that umedaptanib pegol could offer a viable alternative to existing treatments, potentially improving the quality of life for pediatric patients with achondroplasia. The drug's ability to achieve comparable results to Voxzogo® with less frequent dosing could reduce the burden of treatment on patients and caregivers. As achondroplasia affects approximately 1 in 25,000 newborns, the development of effective new drugs is crucial for addressing this unmet medical need.
What's Next?
Following the successful Phase 2 trial, RIBOMIC plans to initiate a Phase 3 clinical trial in the first quarter of fiscal year 2026. This trial will aim to further verify the efficacy of umedaptanib pegol as an achondroplasia treatment, with an increased weekly dose and a lower age range for participating pediatric patients. The Phase 3 trial is expected to conclude within fiscal year 2027, with the goal of obtaining approval by the end of fiscal year 2028. The drug has received orphan drug designation in Japan, which may facilitate expedited approval processes.
Beyond the Headlines
The development of umedaptanib pegol highlights the potential of aptamer therapeutics in addressing rare genetic disorders. Aptamers, as nucleic acid medicines, offer a novel approach to drug development, targeting specific molecular pathways involved in disease progression. The success of umedaptanib pegol could pave the way for further research and development of aptamer-based treatments for other conditions with unmet medical needs.
