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Amicus Therapeutics Announces Fourth Quarter and Full Year 2007 Financial Results & Program Advancements
Date:1/29/2008

ERT) with Cerezyme(R) (imiglucerase) to the pharmacological chaperone Plicera(TM). Thirty patients were enrolled in this trial, which was designed to demonstrate safety and to evaluate various doses and dose regimens of Plicera. The study subjects on average were on ERT with Cerezyme(R) for ten years prior to entering this study.

Preliminary data are available for the first twenty study subjects. Key findings include the following:

-- Plicera was generally safe and well-tolerated at all doses evaluated

and no serious adverse events have been reported

-- The level of the enzyme deficient in people with Gaucher disease

(GCase) as measured in white blood cells increased in 15 of the 20

patients

-- In the cohort with the highest Plicera exposure, 5 out of 5 patients

responded to Plicera with, on average, a near tripling of their GCase

levels from baseline

-- The 5 patients without a clear increase in GCase levels from baseline

were in either the lowest dose cohort or the cohort dosed least

frequently

-- 16 of 20 patients in this study had at least one copy of the N370S

mutation, the most common mutation known to cause Type 1 Gaucher

disease.

"As the Gaucher program at Amicus advances, it has increasing significance and value for our company. Plicera has the potential to address the needs of the vast majority of people living with Gaucher disease and may offer a new standard of care in the treatment and maintenance of their disease. We expect to move forward aggressively in 2008 with additional studies as we explore the full potential of switching Gaucher patients from Cerezyme(R) to Plicera," said John F. Crowley, Amicus President & CEO.

Amicus expects that the results for all thirty patients enrolled in the first Plicera Phase 2 study to be presented at the ACMG meeting in March.

Pompe Disease:

During the fourth quarte
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SOURCE Amicus Therapeutics
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