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.