In addition to chaperone-ERT co-administration in Fabry disease, Amicus and GSK are developing migalastat HCl co-formulated with JCR Pharmaceutical Co. Ltd's proprietary investigational ERT (JR-051, recombinant human alpha-Gal A enzyme). This chaperone-ERT co-formulated product has the potential to enter the clinic in late-2013 or early 2014.
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
, and will be archived for 30 days. The slide presentation for the conference call/webcast will also be available at
. 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.
About 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
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 patients with genetic mutations that are amenable to this chaperone in a cell-based assay. Migalastat HCl monotherapy is in Phase 3 development (
) for Fabry patients with genetic mutations that are amenable to this chaperone monotherapy in a cell-based assay. Study 011 is a placebo-controlled study intended primarily to support U.S. registration, and Study 012 compares 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 thereby allowing more active enzyme to reach tissues.
Migalastat HCl co-administered with ERT is in Phase 2 (
) 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 symptoms of Fabry disease, including pain, kidney failure, and increased risk of heart attack and stroke.