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).
- 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)|