Genzyme Corporation (NASDAQ: GENZ) today announced three-year follow-up data from patients enrolled in the phase 2 clinical trial for its investigational oral therapy for Gaucher disease type 1 known as eliglustat tartrate. Sustained or further improvements were observed across all endpoints, including bone disease, at the three-year timepoint. The results were presented for the first time this week at the Lysosomal Disease Network WORLD Symposium in Las Vegas, Nevada. Genzyme previously reported that the eliglustat tartrate phase 2 trial had met its primary endpoint at one year, and that data demonstrated continued improvement through two years. The primary composite endpoint was a clinically meaningful response in at least two of three endpoints: improvements in spleen size, hemoglobin and platelet levels. The study has continued with 19 patients through three years. The extension phase of this trial is still ongoing. Eliglustat tartrate continued to show robust clinical response through three years:
- Spleen volume decreased from baseline by a mean of 61 percent and liver volume decreased from baseline by 29 percent.
- Hemoglobin level increased from baseline by a mean of 2.6 grams per deciliter.
- Platelet count increased from baseline by a mean of 91 percent.
The three-year data also included analyses that suggest eliglustat tartrate positively impacts indicators of bone disease through three years of follow up. These indicators include bone mineral density in the lumbar spine, as measured by dual energy x-ray absorptiometry (DXA), and dark marrow signal in the femur, as visualized by magnetic resonance imaging (MRI). Dark marrow reflects the infiltration of lipid-laden Gaucher cells into bone marrow. Specifically:
- In the 18 patients at baseline with dark marrow in the femur visible by MRI, five improved by one year, seven by two years and 10 by three years, with the other eight patients remaining stable.
- In the 15 patients with results available at all time points, bone mineral density in the lumbar spine showed clinically and statistically significant improvements after one year of treatment (T score = +0.4) which further improved after 2 years (T score = +0.6) and were sustained after three years of treatment.
Eliglustat tartrate, a capsule taken orally, is being developed to provide a convenient treatment alternative for adult patients with Gaucher disease type 1, and to offer a broader range of treatment options for patients and physicians to achieve individual therapeutic goals. Genzyme is currently enrolling patients in three global, multi-center, phase 3 trials of eliglustat tartrate. This is the largest clinical program ever focused on Gaucher disease, with over 50 sites in more than 25 countries currently participating. Genzyme’s Gaucher disease portfolio also offers Cerezyme ® (imiglucerase for injection), the standard of care for patients with Gaucher disease type 1, which is administered through intravenous infusions.To learn more about the phase 3 trials of eliglustat tartrate, contact Genzyme Medical Information at firstname.lastname@example.org or 1-800-745-4447. More information can also be found at www.clinicaltrials.gov or www.explorerstudies.com. 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, acid 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 typically affect the nervous system and brain. About eliglustat tartrate Eliglustat tartrate, 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, developed with James A. Shayman, M.D. from the University of Michigan, has shown high potency and specificity. Based on its mechanism of action, which is independent of genotype, eliglustat tartrate may be a potential therapy for patients with Gaucher disease type 1. Initiation of the phase 2 and 3 studies of eliglustat tartrate in Gaucher disease followed completion of an extensive pre-clinical research effort and a phase 1 program. Over 300 subjects have now been treated in nine separate studies.
The data from the phase 2 trials with eliglustat tartrate were previously published in the journal Blood and the results can be found at the below references:
- Phase 2 data at the 1 year time point: Lukina et al. Blood, Aug 2010; Vol. 116: 893 - 899
- Phase 2 data at the 2 year time point: Lukina et al. Blood, Nov 2010; Vol 116: 4095 - 4098
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