Healthcare Industry News:  gene therapy  


 News Release - May 16, 2007

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

CAMBRIDGE, Mass., May 16 (HSMN NewsFeed) -- 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.

About Genz-112638

Genz-112638, a novel ceramide analog given orally, 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 Fabry disease, and Genzyme intends to explore studies in this area.

Initiation of the Phase 2 program followed completion of an extensive pre- clinical research effort and a Phase 1 program that involved more than 120 subjects in three separate studies.

Genzyme's Commitment to Gaucher Disease

Development of Genz-112638 reflects Genzyme's long-standing commitment to improving the care of patients with Gaucher disease. Since introducing Ceredase in 1991, Genzyme has engaged in extensive basic science research to further improve the management of Gaucher patients. Post-approval experience has helped to demonstrate the positive and enduring impact Ceredase and Cerezyme can have on different manifestations of the disease, such as anemia, thrombocytopenia, hepatosplenomegaly, and bone involvement. Data generated by the Gaucher Registry, the largest database of its kind, have yielded insights that have shaped how physicians and their patients manage Gaucher disease. Genzyme has also conducted extensive research into gene therapy for single gene disorders like Gaucher disease.

About Genzyme

One of the world's leading biotechnology companies, Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. Since 1981, the company has grown from a small start-up to a diversified enterprise with more than 9,000 employees in locations spanning the globe and 2006 revenues of $3.2 billion. Genzyme has been selected by FORTUNE as one of the "100 Best Companies to Work for" in the United States. With many established products and services helping patients in nearly 90 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant, and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as immune disease, infectious disease, and other areas of unmet medical need.

Forward Looking Statements

This press release contains forward-looking statements regarding Genzyme's business plans and strategies, including: Genzyme's intention to meet with regulatory authorities to discuss an expedited regulatory strategy and the expected timing of that meeting; the potential of Genz-112638 to treat Gaucher disease; and Genzyme's intention to pursue studies of Genz-112638 for treatment of other diseases. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those forecast in these forward-looking statements. These risks and uncertainties include, among others, Genzyme's ability to successfully complete clinical development of, and secure regulatory approvals for, Genz-112638 for the treatment of Gaucher disease; that early improvements in patients are not observed in new patients and/or over a longer period of time; the willingness of regulatory authorities to meet with Genzyme and approve an expedited regulatory strategy; and the risks and uncertainties described in Genzyme's SEC reports filed under the Securities Exchange Act of 1934, including the factors discussed under the caption "Risk Factors" in Genzyme's Quarterly Report on Form 10-Q for the period ended March 31, 2007. Genzyme cautions investors not to place substantial reliance on the forward-looking statements contained in this press release. These statements speak only as of the date of this press release and Genzyme undertakes no obligation to update or revise the statements.

Genzyme® is a registered trademark of Genzyme Corporation. All rights reserved.

Genzyme's press releases and other company information are available at and by calling Genzyme's investor information line at 1-800-905-4369 within the United States or 1-703-797-1866 outside the United States.

Source: Genzyme

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