Top-Line Efficacy Data Presented from Phase 2 Trial of Alemtuzumab in Multiple Sclerosis

Three-Year Analysis Demonstrates Robust, Highly Statistically Significant Treatment Effect of Alemtuzumab Compared to Rebif(R)

 CAMBRIDGE, Mass., Oct. 15 /PRNewswire-FirstCall/ -- Genzyme Corporation (Nasdaq: GENZ) today announced that top-line, three-year data from a completed Phase 2 clinical trial comparing alemtuzumab with Rebif(R) (interferon beta- 1a) for the treatment of multiple sclerosis were presented this weekend at the 23rd Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) in Prague.

The results come from an analysis conducted after 36 months of treatment for 334 patients in the three-year trial.

Efficacy Results

Overall efficacy results demonstrate that alemtuzumab provides a significant treatment effect that has been found to last three years among patients in the study. Analysis of the first co-primary endpoint showed that patients taking alemtuzumab experienced at least a 73 percent reduction in the risk for relapse after three years of follow up when compared to patients treated with interferon beta-1a.

This difference was highly statistically significant in favour of the alemtuzumab patients with a p-value less than the pre-specified value (p=0.00396) assigned for the three-year analysis. Analysis of the other co-primary endpoint showed that patients taking alemtuzumab experienced at least a 70 percent reduction in the risk for progression of clinically significant disability when compared to patients treated with interferon beta-1a.  This difference was statistically significant in favour of the alemtuzumab patients with a p-value less than the pre-specified value (p=0.01646) assigned for the three-year analysis.

Results for the secondary endpoints support the findings seen in the co-primary endpoints. Full, detailed efficacy and safety data from the study are expected to be presented at the 60th Annual Meeting of the American Academy of Neurology next spring. "These results demonstrate the durability of the previously reported effect of alemtuzumab for the treatment of multiple sclerosis that, by our analysis, exceeds any current marketed products and anything that we can see in development," said Richard A. Moscicki, MD, chief medical officer for Genzyme. "We are very impressed with the consistency of these data and feel that they continue to point to the strong likelihood of alemtuzumab being approved for MS patients."

Safety Data: No New Cases of ITP A total of six patients have been diagnosed with ITP associated with Grade 3 or 4 thrombocytopenia in this trial and there have been no new cases of ITP reported in the past year in this study.

Common non-serious adverse events included infusion reactions in the alemtuzumab patients and flu-like symptoms in patients using interferon beta- 1a. Severe infections were infrequent in the alemtuzumab patients and were resolved with or without an intervention. The incidence of all thyroid adverse events, including Graves' disease, was less than expected compared to early reports in the literature on the use of alemtuzumab in MS.

Two Phase 3 studies have recently begun examining the safety and efficacy of alemtuzumab for the treatment of multiple sclerosis. Genzyme and Bayer Schering Pharma AG, Germany announced last month the start of the CARE-MS I trial (Comparison of Alemtuzumab and Rebif Efficacy in Multiple Sclerosis), a randomized, rater-blinded study that will compare alemtuzumab to Rebif(R) as first-line therapy for patients with relapsing-remitting multiple sclerosis (MS).

The second Phase 3 study, CARE-MS II, also has begun and will enroll patients who have continued to experience relapse episodes while on currently available disease-modifying therapies.

Phase 2 Trial Design

The phase 2 trial randomized 334 patients with active relapsing-remitting multiple sclerosis at 49 medical centers in Europe and the United States. Patients in the trial were treated with alemtuzumab at one of two doses (12 or 24/mg per day intravenously for five days at initial treatment, and three days of re-treatment after 12 months with an option to treat again at 24 months), or interferon beta-1a (44 mcg administered by subcutaneous injection three times per week, as indicated in its product label). The randomized trial compared the efficacy of alemtuzumab with interferon beta-1a using two primary endpoints: the rate of relapse of MS symptoms, and the time to Sustained Accumulation of Disability over six months as measured by Expanded Disability Status Scale [EDSS]. Efficacy assessments were made by independent neurologists blinded to therapy.

Alemtuzumab is an investigational drug for the treatment of MS and must not be used outside of a formal clinical trial setting in MS patients. Physicians or patients seeking additional information about the recently-initiated CARE-MS I Phase 3 trial should contact Genzyme Medical Information at 1-800-745-4447, option 2 in the United States, + 31 35 6991499 in Europe, or visit http://www.clinicaltrials.gov.