Genzyme begins phase 2 trial of oral treatment for Gaucher disease

By Elaine Rigoli

Tampa, Fla., July 26 - Genzyme Corp. has started treating patients in a phase 2 clinical trial of Genz-112638, an oral therapy being developed for the treatment of Gaucher disease.

Genzyme said it will conduct the study in patients with type 1 Gaucher disease at medical centers in Europe, Israel, South America and the United States.

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, the company said in a news release.

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. Other endpoints to be investigated include change in liver volume and a series of biomarkers and quality of life indicators relevant to Gaucher disease.

Genzyme is a biotechnology company located in Cambridge, Mass.

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