* Migalastat as effective as standard therapies for Fabry
* Started submitting data to EMA, to meet FDA later this
* Drug already proven effective against placebo in previous
* Amicus is an attractive takeover target - analyst
* Shares jump 21.8 pct in morning trading
(Adds analyst comments; updates shares)
By Natalie Grover
Aug 20 Amicus Therapeutics Inc is a
step closer to bringing its first drug to the market after trial
data paved the way for its lead drug to become the first oral
treatment for patients with a disorder that causes an abnormal
build-up of fat.
The company's stock jumped as much as 21.8 percent to a
20-month high on Wednesday after it released results from a
second late-stage trial of its drug, migalastat.
The treatment was as effective as enzyme replacement
therapies (ERTs) - the standard of care - over an 18-month
period in 60 patients with a form of Fabry disease. It had a
comparable effect to ERTs on kidney function - the main goal of
the study, Amicus said.
Amicus has begun submitting data to the European regulator
and plans to meet the U.S. Food and Drug Administration later
this year to advance the drug's development, the company said.
The data significantly increases migalastat's chances of
approval, making Amicus an attractive acquisition target, Janney
Montgomery Scott LLC analyst Kimberly Lee said.
Cowen and Co analyst Ritu Baral expects a European launch
for migalastat in 2016 and a U.S. launch in 2017.
The United States, European Union and Japan have granted
migalastat orphan drug status, allowing Amicus certain
incentives including a period of marketing exclusivity.
A prior study showed the drug significantly reduced fat
accumulation compared with a placebo after 12 months of
Lee expects the therapy to cost $200,000 per patient per
year and estimates peak sales of $400 million worldwide.
Fabry disease is a potentially fatal inherited disorder
involving the build up of a type of fat, most notably in the
kidneys, caused by the deficiency of the alpha-Gal A enzyme.
The accumulation damages cells, leading to pain, kidney
failure, heart attack and stroke.
Migalastat, tailored to treat 30-50 percent of Fabry
patients with a specific cellular mutation, needs to be orally
administered every other day, giving it an edge over Sanofi SA's
Fabrazyme and Shire Plc's Replagal, which
require bi-weekly infusions.
GlaxoSmithKline Plc returned the rights to
migalastat last November, after the drug failed to significantly
reduce kidney lipid levels at six months.
The results make Amicus an attractive target that could
elicit bids with a 30-40 percent premium, Lee said.
Companies focused on orphan drugs, including BioMarin
Pharmaceuticals Inc, could benefit from Amicus's
armory, which includes drugs undergoing testing against Pompe
disease and Parkinson's.
Amicus is also testing migalastat in combination with
current treatments for patients who are not amenable to the oral
The company will likely grab the attention of "big pharma",
including drugmakers with rival therapies such as Sanofi and
Shire, Lee said.
Up to Tuesday's close, Amicus' stock had risen about 148
percent since the results of the earlier study were released in
April. The stock was up about 21 percent at 1125 ET.
(Editing by Savio D'Souza and Simon Jennings)