Genzyme Corporation (NASDAQ: GENZ) today announced that the company has begun enrollment in the first of two global, multi-center, phase 3 trials of Genz-112638, a potential new oral therapy for Gaucher disease type 1. The two multi-national, multi-center trials are being conducted to evaluate the safety and efficacy of the small molecule.
The first trial, known as ENCORE, has enrolled its first patient. ENCORE is a randomized, open-label study for adult patients with Gaucher disease type 1 designed to compare Genz-112638 to Cerezyme® (imiglucerase for injection). Adult patients who have previously received Cerezyme for at least three years and have reached their therapeutic goals may qualify for this trial. Genzyme expects to enroll nearly 200 patients in the ENCORE trial, with a treatment duration of one year.
The ENGAGE trial is a randomized, double-blind, placebo-controlled study for patients with a confirmed diagnosis of Gaucher disease type 1. Patients with intact spleens who have not been treated in the last 12 months for Gaucher disease may qualify for this study. Genzyme expects to enroll 36 patients in the ENGAGE trial, with a treatment duration of nine months.
Currently over 35 centers in more than 20 countries are participating in these trials. Genzyme expects that the number of recruiting centers will expand, as more sites receive regulatory approval to proceed. At the conclusion of both trials, patients will be offered the option of continuing on therapy with Genz-112638 as part of the extension phase of the program.
“We remain very enthusiastic about this potential new therapy for Gaucher disease, which could offer flexibility and choice to patients and to physicians,” said Genzyme Senior Vice President Geoff McDonough, M.D. “Our studies to date for Genz-112638 suggest a potent and highly-specific therapy that was well-tolerated and that we believe could impact patients in a meaningful, positive way.”