Data provide support for expected Phase 3 program
CRANBURY, N.J., March 28 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD) announced today positive results from its ongoing Phase 2 extension study of its investigational drug, Amigal(TM) (migalastat HCL) for Fabry disease. The results will be presented at the American College of Medical Genetics (ACMG) 2009 Annual Meeting in Tampa, FL.
Phase 2 Extension Study Overview:
Twenty-six subjects completed either 12 or 24 weeks of treatment during Phase 2 studies. Twenty-three of the 26 subjects continue to receive treatment in an ongoing extension study designed to evaluate the long term safety and efficacy of Amigal. Ten of the 23 subjects have been on treatment for at least 2 years and 4 subjects have been on treatment for more than 3 years.
Preliminary Results:
Treatment with Amigal was generally well-tolerated, with no drug-related serious adverse events. The most common adverse events were headache, arthralgia and diarrhea.
Subjects identified as responders to Amigal at the completion of the Phase 2 studies continued to maintain elevated levels of the target enzyme (a-Gal A), as measured in white blood cells, and reduced levels of the target substrate (kidney GL-3), as measured in urine.
A reduction of GL-3 levels was also observed in interstitial capillary cells from kidney biopsies. Previously reported Phase 2 results indicated that little to no GL-3 was detected in these cells in most subjects prior to treatment with Amigal. The new data were obtained from the retesting of biopsies using an improved methodology.
Preliminary results from the evaluation of modified doses and a new dosing regimen were also presented.
Derralynn Hughes, MA, DPhil, MRCPath, Senior Lecturer in the Haematology Department Academic Haematology, Royal Free & University College Medical School, London, UK, stated, 'The data with migalastat continue to be encouraging. I believe migalastat has the potential to be an important new treatment option for Fabry patients.'
John F. Crowley, President and CEO of Amicus Therapeutics, added, 'We are very pleased with this additional set of Phase 2 data and are very confident we have a solid basis for a successful Phase 3 program.
