Study 011 results from Stage 2 are anticipated in the second quarter of 2013. The U.S. Food and Drug Administration (FDA) has indicated that it will consider the entirety of the Stage 1 and Stage 2 efficacy and safety data from Study 011. A meeting with the agency is anticipated in mid-2013 to discuss the U.S. conditional approval pathway for migalastat HCl under subpart H.
A second Phase 3 global registration study ( Study 012) is also underway to compare open-label migalastat HCl to ERT to primarily support global registration. Study 012 (The ATTRACT, or FAB-AT1001-012 Study) is a randomized, open-label 18-month Phase 3 study investigating the safety and efficacy of oral migalastat HCl 150 mg QOD compared to standard-of-care infused therapy using ERTs (Fabrazyme® and Replagal®). This study achieved final enrollment of 60 total patients in December 2012.
Conference Call and Webcast
John F. Crowley, Chairman and Chief Executive Officer, and members of the Amicus executive team will host a conference call and live audio/visual webcast on Friday, February 15, 2013 at 11:30am ET to discuss the data from several programs presented at LDN WORLD. Interested participants and investors may access the conference call at 11:30 a.m. ET by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international). A live audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicustherapeutics.com/events.cfm , and will be archived for 30 days. The slide presentation for the conference call/webcast will also be available at http://ir.amicustherapeutics.com/events.cfm . Web participants are encouraged to go to the web site 15 minutes prior to the start of the call to register, download and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 2:30 p.m. ET on February 15, 2013. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); participant code 97505816.Study 011 Design Study 011 - also referred to as FACETS - is one of 2 ongoing Phase 3 studies of migalastat HCl monotherapy being conducted by Amicus and GlaxoSmithKline (GSK). This study was designed based on feedback from the U.S. Food and Drug Administration (FDA), and is primarily intended to support U.S. registration. Study 011 randomized 67 patients (24 males and 43 females) diagnosed with Fabry disease who had genetic mutations considered amenable to chaperone monotherapy in a cell-based assay. For the 6-month, double-blind period (Stage 1) patients were randomized to migalastat HCl 150 mg or placebo on an every-other-day (QOD) oral dosing schedule. During a 6-month open-label follow up period (Stage 2), patients continued treatment with migalastat HCl or switched from placebo to migalastat HCl. The primary analysis compared the number of responders in the migalastat HCl versus placebo groups in Stage 1, based on a 50% or greater reduction in interstitial capillary globotriaosylceramide (GL-3) as measured in kidney biopsies. Pathologists blinded to biopsy sequence are using the published, quantitative Barisoni Lipid Inclusion Scoring System with virtual microscopy ( BLISS-VM) 2 for the histological evaluation of interstitial capillary GL-3 in kidney biopsies from baseline to month 6 (Stage 1) and from baseline to month 12 (Stage 2). Secondary endpoints for Study 011 include safety and tolerability, urine GL-3 and kidney function. About Amicus Therapeutics Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of therapies for rare and orphan diseases. The Company is developing orally-administered, small molecule drugs called pharmacological chaperones, a novel, first-in-class approach to treating a broad range of human genetic diseases. Amicus' late-stage programs for lysosomal storage disorders include migalastat HCl monotherapy in Phase 3 for Fabry disease; migalastat HCl co-administered with enzyme replacement therapy (ERT) in Phase 2 for Fabry disease; and AT2220 co-administered with ERT in Phase 2 for Pompe disease. About Migalastat HCl Amicus in collaboration with GlaxoSmithKline (GSK) is developing the investigational pharmacological chaperone migalastat HCl for the treatment of Fabry disease. Amicus has commercial rights to all Fabry products in the United States and GSK has commercial rights to all of these products in the rest of world. As a monotherapy, migalastat HCl is designed to bind to and stabilize, or "chaperone" a patient's own alpha-galactosidase A (alpha-Gal A) enzyme in those patients with genetic mutations that are amenable to this chaperone in a cell-based assay. Oral migalastat HCl monotherapy is in Phase 3 development ( Study 011 and Study 012) for Fabry patients with amenable mutations. Study 011 is a placebo-controlled study intended primarily to support U.S. registration, and Study 012 is comparing open-label migalastat HCl to ERT to primarily support global registration. For patients currently receiving ERT for Fabry disease, migalastat HCl in combination with ERT may improve ERT outcomes by keeping the infused alpha-Gal A enzyme in its properly folded and active form. Migalastat HCl co-administered with ERT is in Phase 2 ( Study 013) and migalastat HCl co-formulated with JCR Pharmaceutical Co. Ltd's proprietary investigational ERT (JR-051, recombinant human alpha-Gal A enzyme) is in preclinical development. About Fabry Disease Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A). The role of alpha-Gal A within the body is to break down specific lipids in lysosomes, including globotriaosylceramide (GL-3, also known as Gb3). Lipids that can be degraded by the action of α-Gal are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity leads to the accumulation of GL-3 in the affected tissues, including the kidneys, heart, central nervous system, and skin. This accumulation of GL-3 is believed to cause the various manifestations of Fabry disease, including pain, kidney failure, and increased risk of heart attack and stroke. It is currently estimated that Fabry disease affects approximately 5,000 to 10,000 people worldwide. However, several literature reports suggest that Fabry disease may be significantly under diagnosed, and the prevalence of the disease may be much higher.