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Amicus Therapeutics Advances Chaperone-Enzyme Replacement Therapy (ERT) Combination Platform In Pompe Disease

Stocks in this article: FOLD

Phase 2 AT2220-ERT Co-Administration and Preclinical AT2220-ERT Co-Formulation Studies Presented at LDN WORLD Symposium Next-Generation ERT Advancing in Preclinical Studies Conference Call on February 15, 2013 at 11:30am ET

CRANBURY, N.J., and ORLANDO, Fla., Feb. 14, 2013 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD) today announced positive results from clinical and preclinical studies of the pharmacological chaperone AT2220 (duvoglustat HCl) in combination with ERT for Pompe disease at the Lysosomal Disease Network WORLD Symposium ( LDN WORLD).

John F. Crowley, Chairman and Chief Executive Officer of Amicus stated, "The advancement of our core platform technology in Pompe and other lysosomal storage disorders is a continued great step forward for Amicus. Across these serious genetic diseases, we are leveraging this chaperone-ERT combination platform to work towards improving currently marketed ERTs and to develop our own proprietary next-generation ERTs that incorporate our small molecule chaperones. These chaperone stabilizers have the potential to enhance ERT activity and tissue uptake while also significantly reducing the immunogenicity of the ERTs. Through these programs we hope to offer new benefits and treatment options for patients with lysosomal storage diseases."

Chaperone-ERT Combinations for Pompe Disease

AT2220 Co-Administered with Marketed ERTs

Positive results from a Phase 2 study ( Study 010) established human proof-of-concept that co-administration of AT2220 just prior to infusing ERT (Myozyme/Lumizyme, or rhGAA enzymes) increases GAA enzyme activity in muscle tissue compared to ERT alone. These results appear in a poster 1 and will be featured in an oral platform presentation at LDN WORLD on Friday, February 15 at 9:15 am ET.

Based on these results, Amicus plans to initiate a repeat-dose clinical study in the third quarter of 2013 to evaluate a novel intravenous formulation of AT2220 (AT2220-IV) co-administered with Myozyme/Lumizyme. AT2220-IV when co-administered with ERT is designed to have an improved pharmacokinetic (PK) profile compared to oral AT2220 for all Pompe patients, many of whom are unable to swallow an oral small molecule. The upcoming clinical study will investigate multiple doses of AT2220-IV co-administered with Myozyme/Lumizyme every two weeks in Pompe patients. Objectives of the study are to characterize safety and PK for later evaluation of infants and special populations. Key parameters are expected to include GAA enzyme activity and AT2220 levels in plasma and muscle, as well as rhGAA antibody titers.

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