Amicus Up on Positive Data - Analyst Blog

Shares of Amicus Therapeutics FOLD 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 amenable mutations.

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 of 2014.

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 18-24 months.

We remind investors that GlaxoSmithKline plc GSK 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 Regeneron REGN and Gilead Sciences Inc. GILD. Both these stocks carry a Zacks Rank #1 (Strong Buy).