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Amicus Therapeutics Presents Additional Results From Phase 2 Chaperone-Enzyme Replacement Therapy (ERT) Study For Fabry Disease At LDN World Symposium

CRANBURY, N.J. and ORLANDO, Fla., Feb. 13, 2013 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), today announced further results from an open-label Phase 2 drug-drug interaction study ( Study 013) to evaluate a single oral dose of migalastat HCl (150 mg or 450 mg) co-administered prior to enzyme replacement therapy (ERT) in males with Fabry disease. Preliminary results were announced during 2012. Results for the migalastat HCl 450 mg dose group are being presented for the first time in a poster 1 at the Lysosomal Disease Network WORLD Symposium ( LDN WORLD).

Dr. David G. Warnock, University of Alabama-Birmingham, stated, "When co-administered with ERT, both doses of migalastat HCl appeared to increase enzyme activity compared to Fabrazyme and Replagal alone in this study. We believe that these results support further clinical studies in Fabry patients to investigate the use of a pharmacological chaperone to maintain infused alpha Gal-A enzymes in optimally active form through this chaperone-ERT combination approach."

Amicus, in collaboration with GlaxoSmithKline (GSK), is developing the investigational pharmacological chaperone migalastat HCl as a monotherapy and in combination with ERT for the treatment of Fabry disease. When co-administered with ERT , migalastat HCl binds to and stabilizes infused enzyme in the circulation.

Migalastat HCl (150 mg and 450 mg) Co-Administered with ERT (Fabrazyme and Replagal)

Each patient in Study 013 received their current dose and regimen of ERT (Fabrazyme® or Replagal®, infused alpha-Gal A enzymes) at one infusion. A single oral dose of migalastat HCl (150 mg or 450 mg) was co-administered 2 hours prior to the next infusion of the same ERT at the same dose and regimen. Among 20 total males who enrolled, 3 opted to participate in the migalastat 150 mg and then again in the 450 mg treatment arms.

  • Safety: finalized safety data are expected in 2Q13. As currently evaluated, 2 serious adverse events (SAEs) occurred in one patient (transient ischemic attack and hospitalization for acute pain and acroparesthesia due to Fabry disease) and were considered by the investigator to be unrelated to migalastat HCl when co-administered with ERT. Of the remaining treatment emergent adverse events the majority were unrelated to study drug.
  • Active enzyme in plasma: active alpha-Gal A enzyme levels were measured in plasma (total area under the curve, or AUC) during and after each infusion. Following co-administration, active enzyme levels in plasma increased in 22 out of 23 instances and were unchanged in 1 instance compared to ERT alone. These PK results demonstrated a drug-drug interaction between both doses of migalastat HCl and both ERTs.
  • Enzyme uptake into skin: alpha-Gal A enzyme levels were also measured in skin following each infusion. Increased levels of alpha-Gal A enzyme measured in skin biopsy samples on day 2 and, to a lesser extent, on day 7, showed increased enzyme uptake into tissue compared to ERT alone. This exploratory assessment showed that the greatest increases in active alpha-Gal A enzyme levels were observed following migalastat HCl 450 mg co-administered with ERT. 
  Alpha-Gal A Levels of Active Enzyme 
  Mean-Fold Increase in Plasma Area Under Curve (AUC) vs. ERT Alone (Range) Mean-Fold Increase in Skin at Day 2 vs. ERT Alone (Range) Mean-Fold Increase in Skin at Day 7 vs. ERT Alone (Range)
Migalastat HCl 150 mg + Replagal 0.2 mg/kg (n = 4) 4.3 (3.2 to 5.0) 1.8 (1.4 to 2.3) 1.4 (1.1 to 1.8)
Migalastat HCl 150 mg + Fabrazyme 0.5 mg/kg (n = 5)* 3.0 (2.0 to 4.2) 2.6 (1.1 to 3.9) 1.4 (0.7 to 2.8)
Migalastat HCl 150 mg + Fabrazyme 1.0 mg/kg (n = 3) 2.0 (1.6 to 2.2) 1.9 (1.6 to 2.1) 1.4 (1.2 to 1.7)
Migalastat HCl 450 mg + Replagal 0.2 mg/kg (n = 4) 3.1 (2.3 to 5.0) 2.3 (1.6 to 3.7) 2.1 (1.0 to 2.8)
Migalastat HCl 450 mg + Fabrazyme 0.5 mg/kg (n = 1)* 2.4 3.7 1.9
Migalastat HCl 450 mg + Fabrazyme 1.0 mg/kg (n = 6) 2.0 (1.0 to 3.2) 1.8 (1.0 to 2.5) 1.5 (0.6 to 3.3)

* Due to Fabrazyme supply difficulties during enrollment, subjects had been receiving 0.5 mg/kg Fabrazyme infused every two weeks

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics said, "Study 013 was the first clinical study to investigate the chaperone-ERT combination approach. This study has been a major catalyst for our further development of chaperone-ERT combinations across the lysosomal storage diseases, including chaperones co-administered with marketed ERTs as well as proprietary chaperone-ERT co-formulated products in preclinical development as next-generation ERTs."

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