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Study of Genzyme Oral Compound for Gaucher Disease Meets Primary Endpoint
News

Study of Genzyme Oral Compound for Gaucher Disease Meets Primary Endpoint

Study of Genzyme Oral Compound for Gaucher Disease Meets Primary Endpoint
News

Study of Genzyme Oral Compound for Gaucher Disease Meets Primary Endpoint

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Genzyme Corp. has reported that the Phase 2 clinical trial of its investigational oral therapy Genz-112638 for Gaucher disease type 1 met its primary endpoint. Results from this study, which evaluated the efficacy, safety and pharmacokinetics of the compound, were presented at the Lysosomal Disease Network WORLD meeting in San Diego, Calif.

The company is developing Genz-112638, a capsule taken orally, to provide a treatment alternative for adult 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.

“Given Cerezyme’s unique safety and efficacy profile, we set a high threshold for success, and the results were better than anticipated, indicating a potent, highly-specific and well-tolerated molecule with an ability to reduce substrate in patients, independent of their mutation,” said Genzyme Senior Vice President Geoff McDonough.

The study had a 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 data for the 52-week analysis indicate that Genz-112638 demonstrated robust efficacy results in that 91 percent of those who completed 52 weeks achieved the primary endpoint:

• At 12 months, spleen volumes had decreased from baseline by a mean of 39 percent and liver volumes had decreased from baseline by 17 percent.

• At 12 months, hemoglobin levels had increased from baseline by a mean of 1.62 grams per deciliter of blood.

• At 12 months, platelet counts increased from baseline by a mean of 40 percent with Genz-112638.

• At 12 months, chitotriosidase levels decreased from baseline by a median of 51 percent, among the 20 patients treated with chitotriosidase. Chitotriosidase is commonly monitored by physicians as a biomarker of Gaucher disease burden and response to treatment.

These results approach those observed in patients after one year of Cerezyme treatment. Patients in the trial continue to be treated and longer-term efficacy data are currently being collected.

Genzyme is currently finalizing protocols for two Phase 3 trials that it expects to initiate in mid-2009. One trial will include untreated patients with Gaucher disease type 1, and the other a switch trial in which patients who have achieved their therapeutic goals with Cerezyme will be maintained.

McDonough continued, “As we progress through the clinical development plan, we believe that Genz-112638 will become a meaningful treatment option for the management of Gaucher disease type 1, offering patients and physicians more flexibility to individualize therapy for optimal management of the disease.”
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