BioCentury
ARTICLE | Clinical News

Amicus falls on Phase III Fabry's subgroup data

February 16, 2013 1:30 AM UTC

Amicus Therapeutics Inc. (NASDAQ:FOLD) fell $1 (26%) to $2.89 on Friday after reporting post hoc subgroup data from Fabry's disease patients based on baseline disease burden in the Phase III FACETS (Study 011) trial of Amigal migalastat. In 35 patients with a lower disease burden, 32% of patients receiving Amigal achieved a 50% or greater reduction in kidney interstitial capillary globotriaosylceramide (GL-3) levels as measured in kidney biopsies from baseline to six months, the primary endpoint, vs. 44% for placebo. In 25 patients with a higher disease burden, 64% of patients receiving Amigal achieved the endpoint vs. 14% for placebo. The company did not disclose p-values. The double-blind, international trial enrolled Fabry's disease patients who had genetic mutations amenable to chaperone monotherapy in a cell-based assay. Data were presented at the Lysosomal Disease Network World Symposium in Orlando.

In December, Amicus reported top-line data showing that Amigal missed the primary endpoint in the trial (p=0.3). The news sent shares of Amicus down $2.71 (47%) to $3.06. The company, which expects 12-month data from patients who continued to receive Amigal or crossed over to receive Amigal in 2Q13, said it looks forward to reporting the 12-month results "to add to the entirety of the data that FDA has indicated would support a potential U.S. conditional approval." The company declined to disclose details, but said it plans to meet with FDA in mid-2013 to discuss an approval pathway. Data from the Phase III ATTRACT (Study 012) trial evaluating Amigal compared to enzyme replacement therapy (ERT) are expected in 3Q14 (see BioCentury Extra, Dec. 20, 2012). ...