Genzyme Enrolls First Patient In Phase 3 Trail Of Genz-112638

Genzyme has begun enrollment in the first of two global, multi-center, phase 3 trials of Genz-112638, a new oral therapy for Gaucher disease type 1. Reportedly, 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.

Geoff McDonough, senior vice president of Genzyme, said: “We remain very enthusiastic about this potential new therapy for Gaucher disease, which could offer flexibility and choice to patients and to physicians. 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.”

Genz-112638, a novel glucosylceramide analog given orally, is designed to partially inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucosylceramide.