The Agency Simon and Garfunkel has indicated that it supports
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The Agency shares Simon and Garfunkel has indicated simon and garfunkle imparts that it supports aPhase 3 clinical trial comparing Amigal to placebo based on a surrogateprimary endpoint of the change in the amount of kidney GL-3, the substratethat accumulates in the cells of Fabry patients. Amicus plans to continuediscussions with the FDA through a Special Protocol Assessment (SPA) procedurethat it commenced in the fourth quarter of 2008 to finalize how the primaryendpoint will be measured. Amicus expects the SPA process to be complete inthe second quarter of this year.Based on discussions with the European Medicines Agency (EMEA), Amicus expectsto initiate a separate clinical study designed to evaluate the safety andefficacy of Amigal versus ERT in Fabry patients. Amicus and its partner ShireHGT plan additional discussions with the EMEA in the first half of 2009 tofinalize the design of this study.In parallel with the Phase 3 regulatory discussions, 23 of the original 26patients from the Phase 2 studies continue to be treated in a voluntaryextension study to characterize the long-term safety and efficacy of Amigaland to evaluate additional doses and dose regimens.
Data from this extensionstudy are expected to be available in the first quarter of 2009 and theresults will be used to finalize the dose and regimen for the Phase 3 studies.Plicera(TM) (isofagomine tartrate) for the treatment of Gaucher DiseaseA Phase 2 clinical trial of Plicera in Gaucher patients is ongoing i am a rock simon and garfunkel . This 6month study is designed to evaluate safety as well as to demonstrate trends ofefficacy as measured by the standard endpoints in Gaucher disease garfunkel . Targetenrollment of 16 patients for this study is expected to be surpassed and theresults are expected to be available in the third quarter of 2009.In addition, Amicus is working closely with its partner Shire HGT to preparefor Phase 3 development of Plicera pending the results of the ongoing Phase 2trial.AT2220 (1-deoxynojirimycin HCl) for the treatment of Pompe DiseaseA Phase 2 clinical trial of AT2220 in adult Pompe patients is ongoing simon and garfunkel 'song for the asking . Thetrial includes an 11 week treatment period with an optional extension study.The objectives of the trial include the evaluation of the safety andpharmacodynamics of multiple doses and regimens of AT2220 simon and garfunkel 'song . The results of thisstudy are expected to be available in the second half of 2009.In addition, Amicus is continuing to conduct preclinical animal studies toevaluate the effects of administering AT2220 in combination with ERT.Encouraging preliminary results were announced at the American Society ofHuman Genetics conference in November 2008.
These results indicated thatAT2220 in combination with ERT significantly increases the stability andtissue uptake of ERT . Amicus is conducting additional preclinicalproof-of-concept studies to determine the feasibility of a combination thatmay be appropriate for Pompe patients who are not amenable to chaperonemonotherapy simon and gartfunkel . Amicus expects to announce additional results in the firstquarter of 2009.2009 FINANCIAL GUIDANCEAmicus expects to spend a total of approximately $70 million on 2009 cashoperating expenses, to be offset by $50 million in program cost-sharingreimbursements and clinical milestone payments from Shire simon and carfunkel . Amicus anticipatesending 2009 with approximately $100 million in cash.About Amicus Therapeutics Amicus Therapeutics is a biopharmaceutical company developing novel, oraltherapeutics known as pharmacological chaperones for the treatment of a rangeof human genetic diseases .

