First Patient Enrolled In Phase 3 Trials Of Genzyme Oral Capsule For Gaucher Disease

Main Category: Genetics
Also Included In: Clinical Trials / Drug Trials
Article Date: 22 Oct 2009 - 0:00 PDT

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Genzyme Corporation (NASDAQ: GENZ) 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."

About Genz-112638

Genz-112638, a novel glucosylceramide analog given orally, is designed to partially inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucosylceramide. Glucosylceramide is the substance that builds up in the cells and tissues of people with Gaucher disease. In preclinical studies, the molecule has shown high potency and specificity. In February, Genzyme reported that the phase 2 clinical trial of Genz-112638 met its composite primary efficacy 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. The safety analysis for the phase 2 trial demonstrated that Genz-112638 was well tolerated. Related adverse events in the phase 2 study included infrequent abdominal discomfort and diarrhea, as well as transient palpitations and headache. Patients continue to be treated in the extension phase of the study and have now completed two years of treatment.

Based on its mechanism of action, which is independent of genotype, Genz-112638 is a potential therapy for all adult patients with Gaucher disease type 1. The compound was developed with James A. Shayman, M.D. from the University of Michigan.

Source
Genzyme Corporation