Preliminary Results from Phase 2 Genzyme Study Highlight Potential of Novel Oral Compound for Gauch...(able. Spleen volumes had decrease...),Health

volumes had decreased by 40 percent among 11 patients with available

data at one year.

-- At six months, hemoglobin levels had increased from baseline by a mean

of 0.9 grams per deciliter of blood among 17 patients for whom data

were available. Hemoglobin levels had increased by 1.3 grams per

deciliter among 13 patients with available data at one year.

-- Platelet counts increased from baseline by a mean of 18 percent among

17 patients treated for six months and by 34 percent among 13 patients

with available data at one year.

-- Chitotriosidase levels decreased from baseline by a mean of 30 percent

at six months among 20 patients and by 50 percent among 12 patients

treated for one year. Chitotriosidase commonly serves as a biomarker

of Gaucher disease burden, allowing physicians to monitor patient

response to treatment.

These preliminary results are consistent with results observed for patients beginning enzyme replacement therapy.

The analysis showed that drug-related adverse events seen in the trial occurred in a small number of patients, were mild and transient in nature, and did not require any medical intervention. The drug-related adverse events were diarrhea, abdominal pain, tachycardia, and headache.

Genzyme is currently developing protocols for two Phase 3 trials that it expects to initiate early next year. One trial is expected to include untreated Gaucher patients, and the other is expected to include patients previously treated with Cerezyme.

"We have set a very high bar in trying to develop an oral therapy for Gaucher disease given the remarkable impact that Cerezyme has had," said David P. Meeker, M.D., Genzyme's president of Lysosomal Storage Disorder Therapeutics. "We are excited by the potential of Genz-112638. The data we collect from this study and from the Phase 3 program will provide
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