Genzyme announces publication of phase 2 clinical trial results of eliglustat tartrate for Gaucher disease

Genzyme Corporation (NASDAQ: GENZ) announced today that the two-year follow-up results from the phase 2 clinical trial of its investigational therapy known as eliglustat tartrate have been accepted for publication in the journal Blood. The results have been pre-published on the journal's website and are available to subscribers.

“Through this robust phase 3 program, we look forward to increasing our understanding of the compound as we work to offer patients and physicians more treatment choices to help optimize the management of Gaucher disease.”

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. Current treatments, including Genzyme's Cerezyme^® (imiglucerase for injection), the standard of care for patients with Gaucher disease type 1, are administered through intravenous infusions.

Genzyme reported last year that the 52-week phase 2 trial of eliglustat tartrate 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. The two-year data to be published in Blood indicate continued improvements across all endpoints. After two years, most patients (85%) had met at least three of the four hematologic and visceral therapeutic goals established for enzyme replacement therapy. These data also suggest that eliglustat tartrate may positively impact two indicators of bone disease, bone mineral density and the presence of dark marrow.

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

These two-year results were presented at the Lysosomal Disease Network WORLD Symposium earlier this year.

Phase 3 Program

Genzyme is currently enrolling patients in three global, multi-center, phase 3 trials of eliglustat tartrate. Combined, more than 450 patients are expected to participate, making this the largest clinical program ever focused on Gaucher disease. Over 50 sites in more than 25 countries are currently participating, with additional centers planned.

The first phase 3 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 enzyme replacement therapy 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 received enzyme replacement therapy within 9 months or substrate reduction therapy within 6 months of participation may qualify for this study. Genzyme recently launched a third trial, known as EDGE, to compare once-daily dosing of eliglustat tartrate with twice-daily dosing.

"We remain very enthusiastic about the potential of eliglustat tartrate," said Genzyme's President of Personalized Genetic Health, John Butler. "Through this robust phase 3 program, we look forward to increasing our understanding of the compound as we work to offer patients and physicians more treatment choices to help optimize the management of Gaucher disease."

Complete results from the one-year analysis period for eliglustat tartrate were previously published in Blood. The study and accompanying commentary can be found here.