Study of Genzyme Oral Compound for Gaucher Disease Meets Primary Endpoint

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genzyme Corp. (NASDAQ: GENZ) today reported that the Phase 2 clinical trial of its investigational oral therapy Genz-112638 for Gaucher disease type 1 met its primary endpoint. Results from this study, which evaluated the efficacy, safety and pharmacokinetics of the compound, were presented today at the Lysosomal Disease Network WORLD meeting in San Diego, Calif.

The company is developing Genz-112638, a capsule taken orally, to provide a convenient treatment alternative for adult patients with Gaucher disease type 1, and to provide a broader range of treatment options for patients and physicians to achieve individual therapeutic goals. Currently, Genzyme’s Cerezyme® (imiglucerase for injection), the standard of care for patients with Gaucher disease type 1, is administered through intravenous infusions.

“Given Cerezyme’s unique safety and efficacy profile, we set a high threshold for success, and the results were better than anticipated, indicating a potent, highly-specific and well-tolerated molecule with an ability to reduce substrate in patients, independent of their mutation,” said Genzyme Senior Vice President Geoff McDonough.

The study had a composite primary efficacy endpoint: a clinically meaningful response in at least two of three endpoints (improvements in spleen size, hemoglobin and platelet levels) in individual patients after the 52-week study period. The ongoing open-label study concluded in October 2008. Twenty-two of 26 study participants completed at least 52 weeks of treatment. After the study concluded, all 20 of the patients who were eligible chose to remain on treatment. More than half of the participants have completed more than two years of treatment. Medical centers in North America, South America, Europe, Asia and the Middle East are participating in this study. The data for the 52-week analysis indicate that Genz-112638 demonstrated robust efficacy results in that 91 percent of those who completed 52 weeks achieved the primary endpoint:

• At 12 months, spleen volumes had decreased from baseline by a mean of 39 percent and liver volumes had decreased from baseline by 17 percent.

• At 12 months, hemoglobin levels had increased from baseline by a mean of 1.62 grams per deciliter of blood.

• At 12 months, platelet counts increased from baseline by a mean of 40 percent with Genz-112638.

• At 12 months, chitotriosidase levels decreased from baseline by a median of 51 percent, among the 20 patients treated with chitotriosidase. Chitotriosidase is commonly monitored by physicians as a biomarker of Gaucher disease burden and response to treatment.

These results approach those observed in patients after one year of Cerezyme treatment. Patients in the trial continue to be treated and longer-term efficacy data are currently being collected.

The safety analysis demonstrated that Genz-112638 was well tolerated and has a promising safety profile. Ninety-one percent of adverse events (AEs) were reported to be unrelated to drug treatment. Drug-related AEs occurred in a small number of patients early in treatment, were mild and transient in nature, and did not require any medical intervention. Related adverse events in the Phase 2 study included infrequent abdominal discomfort and diarrhea, as well as transient palpitations and headache. Asymptomatic non-sustained ventricular tachycardia detected on study assessments have occurred when drug levels were below quantifiable limits. Subsequent reviews by three expert cardiologists reveal an unlikely relationship to Genz-112638.

Genzyme is currently finalizing protocols for two Phase 3 trials that it expects to initiate in mid-2009. One trial will include untreated patients with Gaucher disease type 1, and the other a switch trial in which patients who have achieved their therapeutic goals with Cerezyme will be maintained.

McDonough continued, “As we progress through the clinical development plan, we believe that Genz-112638 will become a meaningful treatment option for the management of Gaucher disease type 1, offering patients and physicians more flexibility to individualize therapy for optimal management of the disease.”

About Gaucher disease

Gaucher disease is an inherited condition affecting fewer than 10,000 people worldwide. People with Gaucher disease do not have enough of an enzyme, β-glucosidase (glucocerebrosidase), that breaks down a certain type of fat molecule. As a result, lipid engorged cells (called Gaucher cells) amass in different parts of the body, primarily the spleen, liver and bone marrow. Accumulation of Gaucher cells may cause spleen and liver enlargement, anemia, excessive bleeding and bruising, bone disease and a number of other signs and symptoms. The most common form of Gaucher disease, type 1, does not affect the brain or nervous system.

About Genz-112638

Genz-112638, a novel glucosylceramide analog given orally, is designed to partially inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucosylceramide. Glucosylceramide 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. Based on its mechanism of action, which is independent of genotype, Genz-112638 is a potential therapy for all patients with Gaucher disease type 1. Initiation of the Phase 2 study of Genz-112638 in Gaucher disease followed completion of an extensive pre-clinical research effort and a Phase 1 program that involved more than 120 subjects in three separate studies. The compound was developed with James A. Shayman, M.D. from the University of Michigan.

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 11,000 employees in locations spanning the globe and 2008 revenues of $4.6 billion. In 2007, Genzyme was chosen to receive the National Medal of Technology, the highest honor awarded by the President of the United States for technological innovation.

With many established products and services helping patients in nearly 100 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 immune disease, and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as cardiovascular disease, neurodegenerative diseases, and other areas of unmet medical need.

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 1-678-999-4572 outside the United States.

This press release contains forward-looking statements regarding Genzyme’s development plans for Genz-112638, including that it expects to initiate Phase 3 trials of Genz-112638 in treated and untreated patients in mid-2009, and that it can develop Genz-112638 into a meaningful treatment option for management of Gaucher disease type 1. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those forecasted. These risks and uncertainties include, among others: Genzyme’s ability to pursue its Phase 3 studies expeditiously and in a broad group of Gaucher disease type 1 patients, Genzyme’s ability to develop Genz-112638 into a meaningful treatment option for Gaucher disease type 1 patients, Genzyme’s ability to obtain regulatory approvals for Genz-112638 once it has completed development of the molecule, 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 10Q for the quarter ended September 30, 2008. Genzyme cautions investors not to place substantial reliance on the forward-looking statements contained in this press release. These statements speak only as of today’s date and Genzyme undertakes no obligation to update or revise the statements.

Genzyme® and Cerezyme® are registered trademarks of Genzyme Corporation or its subsidiaries. All rights reserved.

Genzyme Corp.
Media Contact:
Lori Gorski, 617-768-9344
Investor Contact:
Patrick Flanigan, 617-768-6563

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