What's Happening?
RIBOMIC, a clinical-stage pharmaceutical company, has announced positive results from its Phase 2 clinical trial of 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. The results showed significant increases in height growth rates, with some patients achieving growth rates of up to +5.0 cm/year. These findings demonstrate the efficacy, safety, and durability of the drug, establishing proof-of-concept as a treatment for achondroplasia. The company plans to initiate a Phase 3 trial to further verify the drug's efficacy.
Why It's Important?
The positive results from the Phase 2 trial of umedaptanib pegol are significant for the treatment of achondroplasia, a rare genetic disorder that affects bone growth. Current treatments are limited, and the development of effective new drugs is urgently needed. The promising growth rates observed in the trial suggest that umedaptanib pegol could offer a viable alternative to existing treatments, potentially improving the quality of life for patients. The drug's orphan designation in Japan could expedite its approval process, providing faster access to treatment for those affected by achondroplasia.
What's Next?
RIBOMIC plans to conduct a Phase 3 clinical trial, increasing the weekly dose to approximately 1 mg/kg and lowering the age of participating patients to around two years. This trial is expected to begin in the first quarter of fiscal year 2026 and conclude within fiscal year 2027. The company aims to utilize preferential measures associated with the drug's orphan designation to obtain approval by the end of fiscal year 2028. The successful completion of the Phase 3 trial could lead to the drug's availability as a new treatment option for achondroplasia.
