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.