Yuhan said on the 8th that it recently announced in Italy the results of a phase 1 clinical trial of a candidate treatment for Gaucher disease. Gaucher disease is a hereditary rare disease that causes symptoms such as enlargement of the liver and spleen, anemia, and a decrease in platelets.
Yuhan introduced the candidate YH35995 at the 3rd International Gaucher Disease Working Group Symposium held in Italy. Designed to cross the blood-brain barrier, this oral treatment is raising expectations for improving central nervous system symptoms. In preclinical studies, it showed high brain permeability and long-lasting efficacy.
In the phase 1 stage, overall safety and tolerability were also demonstrated. The clinical trial was conducted by administering a single dose of the drug to healthy adult men. No serious adverse events were identified.
Based on these results, the company projected that dosing at four-week intervals would be possible. Kim Yeolhong, Yuhan's head of R&D and president, said, "We will speed up clinical development to provide a treatment alternative for patients with Gaucher disease."