Yuhan Corporation Receives Approval for Phase 1 Clinical Trial of Gaucher Disease Treatment

Yuhan Corporation Receives Approval for Phase 1 Clinical Trial of Gaucher Disease Treatment

Yuhan Corporation has taken its first step towards developing a treatment for a rare disease. The company announced on the 1st that it has received approval from the Ministry of Food and Drug Safety for the Phase 1 clinical trial plan (IND) for ‘YH35995,’ a new drug being developed to treat Gaucher disease. Gaucher disease is a type of lysosomal storage disorder (LSD) caused by the deficiency of an enzyme responsible for processing unnecessary substances in the body.

Yuhan Corporation is developing YH35995 as an oral treatment to reduce the production of glucosylceramide (GL1), which accumulates in the bodies of patients with Gaucher disease.

The upcoming Phase 1 clinical trial will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of YH35995 when orally administered to healthy adult males.

Preclinical trials have demonstrated excellent efficacy and safety for this compound. Notably, the substance showed high blood-brain barrier (BBB) permeability and a longer suppression of GL1 levels in the brain compared to existing treatments, according to the company.

Yuhan Corporation anticipates that YH35995 will provide clinical benefits, particularly for patients with Type 3 Gaucher disease, who have limited treatment options for neurological symptoms. Gaucher disease is categorized into three types: Type 1, which involves increased risk of fractures, bleeding tendencies, and tumors; Type 2 and Type 3, which also involve seizures and neurological degeneration. Types 2 and 3 Gaucher disease have very few treatment options available globally, representing a high unmet medical need.

Kim Yeol Hong, President of R&D at Yuhan Corporation, stated, “After years of focusing on lead compound derivation, optimization, and preclinical development at Yuhan Research Institute, we have successfully advanced YH35995 to clinical development. This marks a significant milestone as it is Yuhan’s first drug developed for a rare disease, and we plan to begin recruiting trial participants soon.”