GENZYME INITIATES PHASE II TRIAL OF GAUCHER DISEASE TREATMENT
Genzyme announced that it has begun to treat patients in a Phase II trial of Genz-112638, an oral therapy being developed for the treatment of Gaucher disease, a genetic disorder. Genzyme will conduct the study in patients with Type 1 Gaucher disease at centers in Europe, Israel, South America and the U.S.
Genz-112638, a ceramide analog given as an oral capsule, is designed to inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucocerebroside. This is the substance that builds up in the cells and tissues of people with Gaucher disease. In preclinical studies, the molecule has shown high potency and specificity. In addition to Gaucher disease, there are a variety of other conditions that can be caused by malfunctions in the pathway targeted by this molecule, such as Tay-Sachs, Sandhoff and Fabry diseases.
The open-label trial will evaluate patient response for one year according to several primary endpoints, including changes in hemoglobin, platelet levels and spleen volume.