Genzyme announces 2-year data from eliglustat tartrate Phase 2 trial for Gaucher disease

Genzyme Corporation (NASDAQ: GENZ) announced today two-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 (formerly Genz-112638). Continued improvements were observed across all endpoints, including bone disease, at the two-year timepoint, compared with baseline. The two-year results were presented for the first time today at the Lysosomal Disease Network WORLD Symposium in Miami, Fla.

“We have set a high threshold for success for eliglustat tartrate, and are very encouraged by the results we continue to observe.”

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’s Cerezyme® (imiglucerase for injection), the standard of care for patients with Gaucher disease type 1, is administered through intravenous infusions.

Genzyme reported last year that the phase 2 trial had met its primary composite 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. Twenty-two of 26 study participants completed at least one year of treatment, and 20 patients completed two years of treatment. The study is continuing with 19 patients in their third year. Medical centers in North America, South America, Europe and the Middle East participated in this study.

Continued improvement was observed through two years for patients who received eliglustat tartrate:

  • Spleen volumes decreased from baseline by a mean of 52 percent and liver volumes decreased from baseline by 24 percent.
  • Hemoglobin levels increased from baseline by a mean of 2.1 grams per deciliter.
  • Platelet counts increased from baseline by a mean of 81 percent.
  • Chitotriosidase levels decreased from baseline by a median of 63 percent (only 18 month data are available to date), among the 17 patients with chitotriosidase. Chitotriosidase is commonly monitored by physicians as a biomarker of Gaucher disease burden and response to treatment.

“We remain committed to building our Gaucher health portfolio by developing clinically meaningful solutions to support individualized care for this community,” said Genzyme Senior Vice President Geoff McDonough, MD. “We have set a high threshold for success for eliglustat tartrate, and are very encouraged by the results we continue to observe.”

According to the ICGG Gaucher Registry, an international multi-center program sponsored by Genzyme that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status, over 80 percent of Gaucher patients have radiologic evidence of bone disease. The data presented today also included pre-planned analyses that suggest eliglustat tartrate may positively impact some indicators of bone disease through two years of follow up. These indicators include bone mineral density, as measured by dual energy x-ray absorptiometry (DXA), and the proportion of dark marrow on magnetic resonance imaging (MRI). Dark marrow reflects the infiltration of diseased cells into the bone marrow. Specifically:

  • Bone mineral density (BMD) in the lumbar spine showed clinically and statistically significant improvements (Z score = +0.60, T score = +0.56) in 16 patients with available data at baseline, one and two years. Four out of six of these patients who were below the normal range at baseline improved to have normal BMD Z scores at two years.
  • In the 18 patients with dark marrow visible on MRI at baseline, six improved by one year, an additional two improved by two years and 10 remained stable.

“Bone disease is a primary cause of morbidity for patients with Gaucher disease,” continued Dr. McDonough. “These data suggest that eliglustat tartrate may have a significant effect on bone mineral density, which could make a positive impact on the lives of patients.”

Eliglustat tartrate was generally well tolerated. The most common adverse events (AEs) reported in greater than 10 percent of patients by two years included viral infections (six patients), urinary tract infections, increased blood pressure, and abdominal pain (three patients each). Eight drug-related AEs, including one serious event, were reported in six patients. All were mild in severity.

Genzyme has begun enrollment in two global, multi-center, phase 3 trials of eliglustat tartrate. The first trial, ENCORE, is a randomized, open-label study for adult patients with Gaucher disease type 1 designed to compare eliglustat tartrate to Cerezyme. Adult patients who have previously received Cerezyme for at least three years and have reached their therapeutic goals may qualify for this trial. The second trial, ENGAGE, is a randomized, double-blind, placebo-controlled study for patients with a confirmed diagnosis of Gaucher disease type 1. Patients who have not been treated in the last 12 months for Gaucher disease may qualify for this study. Over 30 centers in more than 20 countries are participating in these trials. Genzyme is also initiating a trial comparing once-daily dosing of eliglustat tartrate with twice-daily dosing.




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