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CHART Programs Advancing in Lysosomal Storage Diseases
12-Month Results from Ongoing Phase 3 Fabry Disease Monotherapy Study Expected 3Q13
CRANBURY, N.J., March 12, 2013 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced financial results for the full-year ended December 31, 2012. The Company also summarized recent and upcoming milestones and reiterated full-year 2013 operating expense guidance.
Key Highlights and Upcoming Milestones:
Stage 1 (6-month) results from first ongoing Phase 3 Fabry monotherapy study ( Study 011) – Stage 2 (12-month) data anticipated 3Q13. FDA will consider entirety of Stage 1 and Stage 2 data for potential U.S. approval of migalastat HCl monotherapy.
Positive results from Phase 2 study ( Study 010) of AT2220 co-administered with ERT (Myozyme®/Lumizyme®) in Pompe patients – repeat-dose clinical study on track to begin 3Q13.
Results from Phase 2 study ( Study 013) of migalastat HCl co-administered with ERT (Fabrazyme® and Replagal®) in Fabry patients – IND submission planned for chaperone-ERT co-formulated product by year-end 2013 for entry into clinic in early 2014.
Next-generation ERTs for Pompe disease and other LSDs advancing in preclinical studies.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics stated, "During 2012 we announced encouraging 6-month results from our first ongoing Phase 3 Fabry monotherapy study, or Study 011, and established initial human proof-of-concept for our Chaperone-Advanced Replacement Therapy, or CHART, platform. Throughout 2013 our strong financial position will allows us to advance our CHART programs for lysosomal storage diseases and to work toward a potential NDA submission for migalastat HCl monotherapy for Fabry disease. Given our dialogue with the Food and Drug Administration regarding the pre-specified analysis plan for Study 011, we will remain blinded to the 12-month results until the third quarter of this year. We look forward to our continued interactions with the agency to support a potential U.S. approval of migalastat HCl monotherapy. We believe that our pharmacological chaperones, in particular our CHART platform, have the potential to deliver next-generation treatments to patients and create significant shareholder value for many years to come."