Preliminary Results from Migalastat - Fabry RCT Are Not Promising


Another day, another report that an orphan drug fails to be effective - and another drop in the company’s stock price. Yesterday we wrote about Allon therapeutics’ drug failing to meet its endpoint. As a consequence, Allon’s stocks dropped 98% to an astonishing $0.01 close.  

Today, it is Amicus Therapeutics’ turn.  In a statement posted Wednesday afternoon, Amicus announced that their phase 3 study comparing migalastat HCl to placebo did not find a statistically signficant difference in the endpoint following 6 months of treatment for patients with Fabry disease. The company's stock price immediately dropped and as of this writing, their stock is approximately 45% lower ($3) than it was yesterday ($5.70). Amicus’s partner in this study, GlaxoSmithKline, had a 1% drop in their stock  between Wednesday and Thursday.

Fabry disease is an inherited lysosomal storage disorder caused by deficiency of the enzyme alpha-galactosidase A. The role of alpha-galactosidase A is to break down specific lipids in lysosomes, including globotriaosylceramide (GL-3). Reduced or absent levels of alpha-galactosidase A activity leads to the accumulation of GL-3 in various tissues, including the kidneys, heart, central nervous system, and skin. The accumulation of GL-3 is believed to be responsible for the pain, kidney failure, and increased risk of heart attack and stroke that are associated with Fabry disease.

crowley_0.jpgFollowing 6 months of treatment with migalastat HCl, 13 of the 32 patients showed a >50% reduction in GL-3 levels while 9 of the 32 patients receiving placebo achieved the same goal. Based on those results, investors were quick to label this trial a failure.

Amicus’ CEO, John Crowley remains optimistic. In the press release, he stated that the current results only encompass 6 months of treatment and “Once we have the 12-month data, we intend to meet with the FDA to discuss a U.S. approval pathway. We continue to believe that migalastat HCl may become an important treatment option as an oral monotherapy drug for both men and women with Fabry disease.”

The press release also noted that results from this study will be presented at the Lysosomal Disease Network World Symposium being held in Orlando Florida, February 12-15, 2013. Rare Disease Report plans to be in attendance at that meeting.

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