New Jersey, USA-based biopharmaceutical company Amicus Therapeutics says that a Phase I trial of its developmental Gaucher's disease treatment Plicera (isofagomine tartrate) has produced promising results. The firm added that the data, which were presented at the American College of Medical Genetics annual meeting in Nashville, Tennessee, indicate that the oral administration of the drug resulted in a significant elevation of the target enzyme level in healthy volunteers.
Plicera is designed to selectively bind to and stabilize the enzyme GCase, a deficiency in which is responsible for the cellular accumulation of glucocerebroside that characterizes the disease. The firm said that, based on these results, it has initiated a Phase II assessment of the drug.
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