) provided updates and a final analysis plan for its second phase
III study (Study 012) on migalastat HCl as a monotherapy in Fabry
patients with amenable mutations. Amicus' shares jumped over 10%
following the announcement.
The study is comparing oral migalastat to standard-of-care enzyme
replacement therapies (ERTs) −
) Fabrazyme and
) Replagal − for Fabry disease. Renal function measurement at 18
months is the primary focus of the study. Amicus mentioned that it
has completed the 18-month primary treatment period of the Study
012. Top-line results from the study are expected in the third
quarter this year. Amicus plans to file for EU regulatory approval
for migalastat following positive results from the study.
Patients in the study were randomized 1.5:1 to switch to migalastat
or remain on ERT for the 18-month period. After the primary
treatment period, patients were eligible to receive migalastat in a
12-month extension phase.
Amicus is developing migalastat as a monotherapy in two phase III
studies (Study 011 and Study 012) for Fabry patients with amenable
mutations. Earlier in the year, Amicus had announced positive
results from its first phase III study (Study 011) on migalastat
monotherapy. Shares at that time gained over 20% on the news.
Migalastat showed a statistically significant reduction in kidney
interstitial capillary GL-3 at month 12 (p=0.013) and disease
substrate in plasma lyso-Gb3 in subjects who switched from placebo.
Amicus plans to meet the FDA in the fourth quarter of 2014 to
discuss results from both the studies on migalastat. They will also
discuss the future course of action regarding the candidate.
We are encouraged by Amicus' progress with migalastat. Results from
Study 012 are crucial for the candidate's future. We expect
investor focus to remain on migalastat going forward.
Amicus, a biopharmaceutical company, currently carries a Zacks Rank
#3 (Hold). Some better-ranked stocks in the biopharma sector
). The stock carries a Zacks Rank #1 (Strong Buy).
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