By Natalie Grover
(Reuters) - 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 treatment.
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 formulation.
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)