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Genzyme Phase 3 Study Of Oral Compound Eliglustat Tartrate For Gaucher Disease Meets Primary Endpoint

Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), today announced that ENGAGE, the first Phase 3 trial of its investigational oral therapy, eliglustat tartrate, in previously untreated patients with Gaucher disease type 1, met its primary endpoint. Patients treated with eliglustat tartrate had a statistically significant improvement in spleen size at nine months, compared with placebo.

Spleen volumes in eliglustat tartrate treated patients decreased from baseline by a mean of 28 percent versus a mean increase of two percent in placebo patients, for an absolute difference of 30 percent (p<0.0001). In addition, all secondary endpoints were met, including improvements in hemoglobin levels and platelet levels, as well as liver volumes compared with placebo-treated individuals.

The initial safety analysis from ENGAGE suggests that eliglustat tartrate was well tolerated. There were no serious adverse events reported in the primary analysis period and no clinically meaningful differences in the related adverse events reported between the two treatment groups.

“The efficacy and safety data from our ENGAGE trial are consistent with what were observed in our Phase 2 study, continuing to suggest that eliglustat tartrate is a potent, well tolerated oral compound that may become a meaningful option for patients and physicians,” said Genzyme President and Chief Executive Officer, David Meeker, M.D. “The development of eliglustat tartrate has been underway for more than a decade and is the largest clinical program ever focused on Gaucher disease, demonstrating our ongoing commitment to innovation on behalf of this community.”

Full results from the ENGAGE study are planned for presentation at the Lysosomal Disease Network WORLD meeting, February 12-15, 2013, in Orlando, Fla. Top-line data from Genzyme’s second Phase 3 trial, ENCORE, are expected in early 2013.

The company is developing eliglustat tartrate, a capsule taken orally, to provide a convenient treatment alternative for patients with Gaucher disease type 1, and to provide a broader range of treatment options for patients and physicians to achieve individual therapeutic goals. Currently, Genzyme’s Cerezyme ® (imiglucerase for injection), the standard of care for patients with Gaucher disease type 1, is administered through intravenous infusions.

ENGAGE is a randomized, double-blind, placebo controlled study in treatment-naïve patients with Gaucher disease type 1 and evaluated the efficacy, safety and pharmacokinetics of twice-daily dosing of eliglustat tartrate in 40 patients untreated for at least six months. The study had a primary efficacy endpoint of improvement in spleen size in individual patients treated with eliglustat tartrate compared with treatment with placebo, after the nine month study period. Patients were stratified at baseline by their spleen volume. Thirty-nine out of 40 study participants completed at least nine months of treatment. One patient in the eliglustat treated group discontinued at six months for personal reasons. At the end of nine months, patients who were on placebo were transitioned to eliglustat tartrate. After the primary analysis period concluded, all 39 patients chose to remain on treatment. Eighteen medical centers in 12 countries in North America, South America, Europe, Asia and the Middle East are participating in this study.

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