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An announcement from Ribomic, Inc. ( (JP:4591) ) is now available.
Ribomic reported Phase II trial results for its anti-FGF2 aptamer umedaptanib pegol in Japanese paediatric patients with achondroplasia, showing a statistically significant mean increase of 1.4 cm per year in annualized height velocity after 26 weeks of treatment. The study, which tested low- and high-dose regimens in 12 children and found mainly mild, short-term adverse events, supports proof-of-concept for the drug and underpins the company’s plan to seek approval to begin a pivotal Phase III trial.
Responses were observed in both dose cohorts, with notable height gains in several subjects and sustained improvements in a long-term extension, including benefits in some children previously treated with Voxzogo. While secondary endpoints showed no consistent efficacy trends, the favorable safety and primary endpoint data strengthen Ribomic’s position in the emerging achondroplasia treatment market and mark a key step toward potential commercialization of its lead aptamer therapy.
The most recent analyst rating on (JP:4591) stock is a Sell with a Yen82.00 price target. To see the full list of analyst forecasts on Ribomic, Inc. stock, see the JP:4591 Stock Forecast page.
More about Ribomic, Inc.
Ribomic, Inc. is a Tokyo-based clinical-stage pharmaceutical company specializing in aptamer therapeutics. The company focuses on developing novel treatments targeting fibroblast growth factor pathways, with a current emphasis on rare skeletal disorders such as achondroplasia in paediatric patients.
Average Trading Volume: 2,027,873
Technical Sentiment Signal: Hold
Current Market Cap: Yen5.16B
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