Amicus Up on Migalastat Study Update - Analyst Blog


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Amicus Therapeutics ( FOLD ) 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) − Sanofi 's ( SNY ) Fabrazyme and Shire 's ( SHPG ) 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 include Regeneron ( REGN ). The stock carries a Zacks Rank #1 (Strong Buy).

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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