Amicus Therapeutics (FOLD) Reports Patient Screening Profiles From Phase 3 Fabry Study
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Amicus Therapeutics (Nasdaq: FOLD) recently presented updated information on the number and type of patients screened for and enrolled in the global Phase 3 registration study (Study 011) of the investigational pharmacological chaperone migalastat HCl, being studied as a potential monotherapy for Fabry disease.
Amicus and its collaborator GlaxoSmithKline (GSK) have enrolled 67 (24 males and 43 females) out of 180 total subjects screened in Study 011. The study completed enrollment in December 2011. All patients enrolled in the study have been diagnosed with Fabry disease, and were naïve to enzyme replacement therapy (ERT) or had not received ERT for at least six months prior to study entry.
In addition, each patient enrolled in Study 011 met further entry criteria, which included having alpha-Gal A mutations amenable to migalastat HCl monotherapy in vitro. For study purposes, all patients also needed to have urine globotriaosylceramide (GL-3) at least four times the upper limit of normal. A number of patients screened had amenable mutations but did not have sufficiently elevated urine GL-3 at baseline to qualify for enrollment into Study 011.
Elevated levels of GL-3 in urine have been shown to correlate with GL-3 levels in kidney tissue, including the interstitial capillaries. The primary efficacy endpoint for Study 011 is a change in interstitial capillary globotriaosylceramide (GL-3) as measured in kidney biopsies. Patients in Study 011 with a reduction of GL-3 deposits per capillary of at least 50% at six months will be considered responders.
John F. Crowley, Chairman and Chief Executive Officer of Amicus stated, "We are pleased that the 67 Fabry patients enrolled in Study 011 include a range of patients by gender, disease burden and genotype who are living with Fabry disease today. Study 011 is the first randomized, controlled Fabry study to ever include a large number of female patients, who represent more than half of all patients in Fabry disease registries. We look forward to seeing these study results in the third quarter of this year."
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Amicus and its collaborator GlaxoSmithKline (GSK) have enrolled 67 (24 males and 43 females) out of 180 total subjects screened in Study 011. The study completed enrollment in December 2011. All patients enrolled in the study have been diagnosed with Fabry disease, and were naïve to enzyme replacement therapy (ERT) or had not received ERT for at least six months prior to study entry.
In addition, each patient enrolled in Study 011 met further entry criteria, which included having alpha-Gal A mutations amenable to migalastat HCl monotherapy in vitro. For study purposes, all patients also needed to have urine globotriaosylceramide (GL-3) at least four times the upper limit of normal. A number of patients screened had amenable mutations but did not have sufficiently elevated urine GL-3 at baseline to qualify for enrollment into Study 011.
Elevated levels of GL-3 in urine have been shown to correlate with GL-3 levels in kidney tissue, including the interstitial capillaries. The primary efficacy endpoint for Study 011 is a change in interstitial capillary globotriaosylceramide (GL-3) as measured in kidney biopsies. Patients in Study 011 with a reduction of GL-3 deposits per capillary of at least 50% at six months will be considered responders.
John F. Crowley, Chairman and Chief Executive Officer of Amicus stated, "We are pleased that the 67 Fabry patients enrolled in Study 011 include a range of patients by gender, disease burden and genotype who are living with Fabry disease today. Study 011 is the first randomized, controlled Fabry study to ever include a large number of female patients, who represent more than half of all patients in Fabry disease registries. We look forward to seeing these study results in the third quarter of this year."
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