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TOKYO - Japanese pharmaceutical company Ribomic Inc. (TOKYO:4591), a small-cap biotech firm valued at $28.45 million with a strong financial health score according to InvestingPro, announced Tuesday that its experimental drug umedaptanib pegol demonstrated positive growth rate impacts in pediatric patients with achondroplasia, a rare genetic disorder causing dwarfism.
In the completed Phase 2 clinical trial, five out of six patients receiving high-dose biweekly injections (0.6 mg/kg) showed increased height growth rates. Two patients exhibited significant increases of 5.0 cm/year and 2.0 cm/year, exceeding the average growth rate of 1.7 cm/year seen with Voxzogo, a currently approved treatment. With a robust current ratio of 25.95 and substantial cash reserves, as highlighted in InvestingPro’s analysis, Ribomic appears well-positioned to fund its clinical development program.
The mean height growth rates across both dosing cohorts were 1.5 cm/year and 1.4 cm/year, comparable to Voxzogo’s results but with less frequent administration. The trial included children aged 5-14 years and reported no safety concerns throughout the study period.
"These results demonstrate the efficacy, safety and durability of umedaptanib pegol in Phase 2 clinical trials, successfully establishing proof-of-concept as an achondroplasia treatment," according to the company’s press release statement.
Ribomic plans to advance to Phase 3 trials in the first quarter of fiscal year 2026, increasing the weekly dose to approximately 1 mg/kg and enrolling younger patients around age two. The company aims to obtain approval by the end of fiscal year 2028, leveraging the drug’s orphan designation status from Japan’s Ministry of Health, Labor and Welfare.
Achondroplasia affects approximately 1 in 25,000 newborns and is caused by a mutation in the fibroblast growth factor receptor 3 gene. Umedaptanib pegol is an aptamer-based therapeutic that targets fibroblast growth factor 2, a protein involved in the condition’s underlying mechanism.
The company believes the biweekly or weekly dosing schedule could significantly benefit pediatric patients who require long-term treatment, compared to daily injections needed with current therapies.
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