Amicus Therapeutics Inc said its experimental drug significantly reduced the abnormal accumulation of fat in body cells related to a rare genetic disorder that could lead to heart attack, stroke and kidney failure.
The company, whose shares rose as much as 36 percent, said it would file for U.S. marketing approval based on these results and those from an European trial, expected in the third quarter.
The late-stage trial evaluated the drug, migalastat, as a form of monotherapy for certain Fabry disease patients after 12 months of treatment.
The 24-month study began with a 6-month period where patients received either the drug or a placebo. All patients were then treated with migalastat for a 6-month follow-up period and a subsequent 12-month extension phase.
After the drug failed to show statistically significant reduction in kidney lipid levels at 6 months, Amicus said it would report 12-month efficacy and safety data to support its marketing application.
"It may be that (the drug) needs more time to work, as shown by today's results," Janney Montgomery Scott analyst Kimberly Lee said, adding that the European trial data would provide further evidence of whether the drug needed a longer duration to be effective.
Cowen & Co analyst Edward Nash said migalastat could satisfy the "significant need for an oral therapy for Fabry disease".
Sanofi SA's Fabrazyme, the first FDA-approved Fabry treatment, is administered intravenously.
Lee said she expected migalastat's use as a monotherapy could represent a $250 million global market opportunity and be worth up to double that size as a combination therapy.
Amicus is also testing migalastat in combination with the current standard-of-care for the disease.
GlaxoSmithKline returned the rights to migalastat to Amicus in November 2013.
Fabry disease is an inherited, potentially fatal disorder characterized by the buildup of a particular type of fat - most notably in the kidneys - called globotriaosylceramide, or GL-3, in the body's cells.
This progressive lipid accumulation, caused by the deficiency of the enzyme α-galactosidase A (α-Gal A), results in cell damage, leading to pain, hearing loss, kidney failure, heart attacks and strokes. As a monotherapy, migalastat works by binding to the α-Gal A enzyme, made in the patient's body, helping it break down the lipids. The Cranbury, New Jersey-based company's stock was up about 22 percent at $2.25 in afternoon trading.
(Reporting by Natalie Grover in Bangalore; Editing by Don Sebastian and Sriraj Kalluvila)