Genzyme Corp.Genzyme Corp. (NASDAQ: GENZ) reported that a preliminary analysis of data from an ongoing open-label Phase 2 clinical trial of its investigational oral therapy Genz-112638 showed that the compound produced a meaningful impact on key clinical manifestations of Gaucher disease. The results highlight the potential of this compound to become an innovative treatment option for Gaucher disease.

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

The primary analysis period of the Phase 2 study of Genz-112638 is scheduled to conclude later this year, and the results for all trial participants will be available in the first quarter of 2009. The study included 26 adults with Type 1 Gaucher disease at medical centers in North America, South America, Europe and Israel. It was designed to evaluate the efficacy, safety and pharmacokinetics of the compound over one year. Nearly all study participants had completed six months of treatment when the preliminary analysis was conducted, and approximately half of the participants had completed one year of treatment.

The preliminary analysis showed that Genz-112638 produced promising efficacy results at six months and that these results continued to improve through one year of treatment:

  • At six months, spleen volumes had decreased from baseline by a mean of 27 percent among the 21 patients for whom data were available. Spleen volumes had decreased by 40 percent among 11 patients with available data at one year.
  • At six months, hemoglobin levels had increased from baseline by a mean of 0.9 grams per deciliter of blood among 17 patients for whom data were available. Hemoglobin levels had increased by 1.3 grams per deciliter among 13 patients with available data at one year.
  • Platelet counts increased from baseline by a mean of 18 percent among 17 patients treated for six months and by 34 percent among 13 patients with available data at one year.
  • Chitotriosidase levels decreased from baseline by a mean of 30 percent at six months among 20 patients and by 50 percent among 12 patients treated for one year. Chitotriosidase commonly serves as a biomarker of Gaucher disease burden, allowing physicians to monitor patient response to treatment.

These preliminary results are consistent with results observed for patients beginning enzyme replacement therapy.

The analysis showed that drug-related adverse events seen in the trial occurred in a small number of patients, were mild and transient in nature, and did not require any medical intervention. The drug-related adverse events were diarrhea, abdominal pain, tachycardia, and headache.

Genzyme is currently developing protocols for two Phase 3 trials that it expects to initiate early next year. One trial is expected to include untreated Gaucher patients, and the other is expected to include patients previously treated with Cerezyme.

"We have set a very high bar in trying to develop an oral therapy for Gaucher disease given the remarkable impact that Cerezyme has had," said David P. Meeker, M.D., Genzyme’s president of Lysosomal Storage Disorder Therapeutics. "We are excited by the potential of Genz-112638. The data we collect from this study and from the Phase 3 program will provide guidance on the roles that this compound may play in treating and maintaining patients with Gaucher disease."

Genzyme reported the results from the preliminary analysis of the Phase 2 study of Genz-112638 at its Analyst Day meeting held today in Boston.

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 that breaks down a certain type of fat molecule called glucocerebrosidase. As a result, cells fill up with the undigested fat in different parts of the body, primarily the liver, spleen 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 inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucocerebroside. Glucocerebrosidase 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 Type 1 Gaucher disease. 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. In addition to Gaucher disease, there are a variety of other conditions including Fabry disease that can be caused by malfunctions in the pathway targeted by this molecule. The compound is exclusively licensed from the University of Michigan and was developed with James A. Shayman, M.D.

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 10,000 employees in locations spanning the globe and 2007 revenues of $3.8 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 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, cardiovascular disease, and other areas of unmet medical need.

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