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First Scientific Meeting to Present Preclinical Proof-of-Concept Data for Next-Generation Pompe ERT and Proprietary Enzyme Targeting Technology
Encouraging Additional Post-Hoc Analyses of Interim Data from Phase 3 Fabry Monotherapy Study 011
CRANBURY, N.J., Feb. 12, 2014 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today summarized the key highlights from 2 oral platform presentations featuring its Pompe and Fabry programs at the Lysosomal Disease Network WORLD Symposium (
LDN WORLD) 2014. LDN WORLD is taking place in San Diego, CA from February 10-13, 2014.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, "At this year's WORLD Symposium we are highlighting data from several of our ongoing development programs. This is the first scientific meeting to feature our next-generation Pompe ERT and proprietary enzyme targeting technology, purchased through our acquisition of Callidus Biopharma. Together with our Chaperone-Advanced Replacement Therapy, or CHART, platform these technologies may provide a unique tool set to address some of the major challenges with currently marketed ERT products – enzyme activity and stability; targeting and uptake; and tolerability and immunogenicity. We are also pleased to present further, post-hoc analyses of 6-month data from our first of two ongoing Phase 3 Fabry monotherapy studies, which we believe demonstrate that migalastat HCl is having a positive impact in Fabry patients with amenable mutations."
Preclinical Proof-of-Concept Data for Next-Generation Pompe ERT and Proprietary Enzyme Targeting Technology
Preclinical proof-of-concept data for AT-B200, Amicus' proprietary recombinant human acid-alpha glucosidase (rhGAA) for Pompe disease, as well as an overview of the Company's proprietary peptide tagging technology (vIGF-2), will be presented to a scientific audience for the first time at LDN WORLD 2014. AT-B200 is differentiated from other Pompe ERTs by its unique carbohydrate structure. This ERT may be further optimized through co-formulation with Amicus' pharmacological chaperone AT2220 to improve enzyme stability and tolerability, and by applying the Company's peptide tagging technology for better targeting. Preclinical results have shown that AT-B200 and AT-B200 conjugated with vIGF-2 ("tagged AT-B200") were better than Lumizyme for clearing glycogen in skeletal muscles in GAA knock-out mice after 4 weekly IV bolus administrations
Hung Do, PhD, Senior Vice President, Amicus Therapeutics, Inc., stated, "After spending over a decade developing enzyme replacement therapies for Pompe and other lysosomal storage diseases at Genzyme and Novazyme, AT-B200 was developed to be a next-generation Pompe ERT with a unique carbohydrate structure that may better target skeletal muscles. Our preclinical studies to date demonstrate the potential for AT-B200 in Pompe disease, as well as the differences between our peptide tagging technology and other IGF-2 tagging approaches in development. We look forward to further preclinical development to evaluate tissue uptake and glycogen clearance with longer dosing schedules of AT-B200, with and without a pharmacological chaperone."