Data Support Commencement of Longer-term Phase 2 Switching Study
CRANBURY, N.J., March 13 /PRNewswire/ -- Amicus Therapeutics, a biopharmaceutical company developing small-molecule, orally administered pharmacological chaperones for the treatment of human genetic diseases, announced today that the Company will present positive results from a Phase 2 clinical study of Plicera(TM) (isofagomine tartrate) for Gaucher disease at the American College of Medical Genetics (ACMG) Annual Meeting from March 12-16 in Phoenix, AZ. Results from the fully enrolled Phase 2 trial support the previously reported interim findings that Plicera was generally safe and well tolerated at all doses and increased target enzyme activity levels in a majority of patients.
Phase 2 Plicera data presented at ACMG
The primary objective of this study was to evaluate safety and tolerability of different doses and dosing regimens of Plicera. The secondary objective was to evaluate certain pharmacodynamic measures of treatment, including effects on GCase (the enzyme deficient in individuals with Gaucher disease) levels in white blood cells.
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 4 week duration of the study.
The key findings from the trial were as follows:
-- Plicera was generally well-tolerated at all doses evaluated, and no
serious adverse events were reported.
-- 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
|SOURCE Amicus Therapeutics|
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