) shot up 20.7% after the company announced positive data from
its first phase III study (Study 011) of the oral small molecule
chaperone migalastat HCl monotherapy in Fabry patients with
Amicus Therapeutics is developing migalastat HCl as a
monotherapy in two phase III studies (Study 011 and Study
012) for Fabry patients with amenable mutations.
Study 011 is a 24-month study consisting of a 6-month,
double-blind, placebo-controlled treatment period (Stage 1);
a 6-month, open-label, follow-up period (Stage 2); and a
12-month, open-label, extension phase. The study was designed to
measure the reduction of disease substrate
(Globotriaosylceramide, or GL-3) following treatment with
migalastat. Data from Study 012 is expected in the third quarter
Migalastat demonstrated a statistically significant reduction
in kidney interstitial capillary GL-3 at month 12 (p=0.013). The
reduction in disease substrate was also observed in plasma
lyso-Gb3 in subjects who switched from placebo to migalastat (p
< 0.0001). Also, kidney function remained stable over
We remind investors that
) amended its 2010 Fabry disease agreement with Amicus in Nov
2013. As per the agreement, Amicus will have the sole worldwide
rights to develop and commercialize migalastat HCl (both as
monotherapy and in combination with enzyme replacement therapy)
for Fabry disease.
We are encouraged by the positive data on migalastat HCl
considering that it is one of the key candidates at Amicus.
Amicus currently carries a Zacks Rank #3 (Hold). Investors
looking for better-ranked stocks in the biopharma sector may
consider companies like
Gilead Sciences Inc.
). Both these stocks carry a Zacks Rank #1 (Strong Buy).
AMICUS THERAPT (FOLD): Free Stock Analysis
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