Plicera was generally well-tolerated at all doses evaluated, and no serious adverse events were reported. Glucocerebrosidase (GCase) activity as measured in white blood cells was increased in 20 of the 26 patients with evaluable GCase data, and 5 of the 6 patients without a clear increase were either in the lowest dose cohort or the cohort dosed least frequently. The levels of relevant markers of Gaucher disease including platelet counts, hemoglobin levels, glucocerebroside levels, chitotriosidase activity and pulmonary activation-related chemokine levels were maintained.
Thirty patients with Gaucher disease (8 men and 22 women between the ages of 18 and 63) were enrolled, and there were 12 unique alleles represented including the most common N370S and L444P mutations. Patients were on enzyme replacement therapy (ERT) with imiglucerase for an average of 9 years prior to entering the trial, and they temporarily discontinued ERT to receive Plicera for the four-week duration of the study.
John Crowley, president and CEO of Amicus, said: “In addition to a six-month Phase II study in individuals naive to ERT, which is currently underway, we plan to initiate a longer-term study in individuals switching from enzyme replacement therapy to Plicera in the second half of 2008.”