Genzyme Announces Positive Initial Observations in Trial Evaluating Novel Oral Treatment for Gaucher Disease

Genzyme Corp. (Nasdaq: GENZ) announced today that it has completed enrollment in the ongoing Phase 2 trial of Genz-112638, a novel oral therapy being developed for the treatment of Gaucher disease. Based on positive results seen in the trial to date, Genzyme intends to meet with regulatory agencies in the coming weeks to discuss an expedited development strategy.

Initial observations from the first five patients suggest that Genz-112638 may produce a rapid and meaningful impact on important clinical endpoints including reductions in spleen and liver volume, and an increase in platelet counts and hemoglobin concentration. Safety observations from all patients enrolled to date suggest that the only drug-related adverse events seen in the trial have been mild and transient in nature, including one possibly related serious adverse event that is currently being investigated. These early findings will be presented today at Genzyme’s Analyst Day, and full trial results will be available in mid-2008.

If these early improvements continue and are observed in other patients enrolled in the trial, Genz-112638 may represent a promising novel approach to treating patients with Gaucher disease.

“We are very encouraged by our first observations from this trial,” said David Meeker MD, president of the Lysosomal Storage Disorder business. “Cerezyme has had a remarkable effect on the lives of patients with Gaucher Disease. We have set a very high bar for ourselves in trying to develop a convenient oral therapy that can provide a safe and effective choice for patients. We look forward to developing this molecule further and exploring the role it may play in the treatment of patients with Gaucher disease.”

The open-label trial has enrolled patients with Type 1 Gaucher disease at medical centers in Europe, Israel, North America and South America.