Replies to post #131406 on Biotech Values

[turtlepower]

Amicus Therapeutics Announces Positive Preliminary Results From Ongoing Phase 2 Chaperone-Enzyme Replacement Therapy (ERT) Study for Fabry Disease
Co-Administration Increases Levels of Active Enzyme Compared to ERT Alone in First Six Patients

http://finance.yahoo.com/news/Amicus-Therapeutics-Announces-pz-1532247443.html?x=0

CRANBURY, N.J., Jan. 6, 2012 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD - News), today announced preliminary results from an ongoing, open-label Phase 2 drug-drug interaction study (Study 013) to evaluate the safety and pharmacokinetic (PK) effects of two doses of migalastat HCl (150 mg and 450 mg) co-administered with ERT (agalsidase beta or agalsidase alfa) in up to 24 males diagnosed with Fabry disease.
Amicus and GSK are developing migalastat HCl, an investigational oral pharmacological chaperone, as part of a global collaboration for Fabry disease. Migalastat HCl is in Phase 3 development (Study 011 and Study 012) for use as a monotherapy for patients with Fabry disease identified as having alpha-galactosidase A mutations amenable to chaperone therapy.
When co-administered with ERT, migalastat HCl is designed to bind to and stabilize the enzyme in the circulation, in any patient receiving ERT. In preclinical studies, the co-administration of migalastat HCl and ERT led to stabilization of the ERT and increased uptake of active enzyme into key organs of disease, including kidney, heart, and skin, when compared to ERT alone. This increased enzyme uptake in Fabry mouse models also led to further reductions in globotriaosylceramide (GL-3), the substrate that accumulates in kidney, heart and skin in Fabry disease.
Data are currently available for the first six subjects in Study 013, who received their current dose and regimen of agalsidase beta alone at one infusion followed by oral migalastat HCl 150 mg administered two hours prior to agalsidase beta at their next infusion. Due to the supply shortage of agalsidase beta, four of these subjects had been receiving 0.5 mg/kg infused every two weeks and two subjects had been receiving a dose of 1.0 mg/kg infused every four weeks.
Preliminary Results - Migalastat HCl 150 mg Co-Administered with Agalsidase Beta (n=6)
Increases in levels of active enzyme in plasma and skin demonstrate a positive drug-drug interaction between migalastat HCl 150 mg and agalsidase beta
In the four patients who received agalsidase beta at 0.5 mg/kg co-administered with migalastat HCl 150 mg, levels of active enzyme in plasma ranged from 2.0 to 4.2-fold higher than with ERT alone, as measured by total area under the curve (AUC). In skin biopsies from three patients, increases in levels of active enzyme in the skin ranged from 1.1 to 3.9-fold higher at day two, but no higher at day seven, following co-administration compared to ERT alone.
In the two patients who received agalsidase beta at 1.0 mg/kg co-administered with migalastat HCl 150 mg, levels of active enzyme in plasma were 1.6 and 2.2-fold higher than with ERT alone, as measured by total AUC. In skin biopsies, increases in levels of active enzyme in the skin were 1.6 and 2.1-fold higher at day two, and 1.2 and 1.7-fold higher at day seven, following co-administration compared to ERT alone.
Preliminary data to be presented as a "late breaking" abstract at 8th Annual Lysosomal Disease Network WORLD Symposium (LDN WORLD) in San Diego, February 8-10, 2012.
Independent data safety monitoring board approved dose escalation to migalastat HCl 450 mg as per study protocol. Amicus and GSK expect to complete Study 013 in the first half of 2012.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics said, "These very encouraging data in Fabry patients represent an important first step in validating the potential of pharmacological chaperones to enhance the stability and tissue uptake of enzyme replacement therapy products in Fabry disease. We look forward to completing Study 013, including obtaining data on the higher dose of migalastat HCl as well as data on co-administration with agalsidase alfa. We also look forward to evaluating the complete data set and collaborating with GSK on the best path forward for extending this co-administration use of our core technology as a treatment option for people living with Fabry disease."