Amicus Open to Future Glaxo Bid If Fabry Drug Succeeds in Study

Amicus Therapeutics Inc., a U.S. company developing medicines for rare diseases, is open to a future takeover offer from partner GlaxoSmithKline Plc, Chief Executive Officer John Crowley said.

Glaxo agreed Oct. 29 to pay Amicus $30 million upfront and took a $31 million stake in the Cranbury, New Jersey-based company to license the rights to Amigal, a novel treatment for the rare inherited disorder Fabry disease. Sixty patients are enrolled in a trial in the final stage of human testing needed for U.S. regulatory approval. The company expects data from the study in the second half of next year, Crowley said in a Dec. 17 interview.

“With success some of those options may come in time,” he said, referring to a possible bid. “Right now we are focused on the partnership we have with GSK and other potential partnerships with Parkinson’s and other programs. We will look at different ways to maximize shareholder value over time.”

Fabry disease is an inherited disorder caused by low levels of certain enzymes needed to break down fats that can build up in the body’s organs. Amigal may win market share from Genzyme Corp.’s Fabrazyme and Shire Plc’s Replagal enzyme-replacement treatments that reach between 3,000 to 4,000 people, together generating about $800 million a year in sales, Crowley said.

“It’s going to be a drug that replaces enzyme therapy for roughly half the people living with Fabry disease,” he said. “For the other half of the people it will be a complement” to their existing treatment.

Amicus shares have risen 21 percent over the last year, closing yesterday at $4.48, giving the company a market value of $123.8 million. Glaxo holds a 25 percent stake in Amicus, according to Bloomberg data.

Shire Partnership

Amicus mutually terminated its two-year partnership with Shire, based in Dublin with headquarters in Basingstoke, England, in November 2009, said Crowley. The Shire partnership had covered three experimental treatments for rare illnesses, including Amigal, Plicera for Gaucher disease and AT2220 for Pompe disease.

“We had multiple term sheets for this program from other big pharmas. By and away, GSK’s was the most impressive,” Crowley said. “It was a very competitive process.”

Jo Revill, a spokeswoman for London-based Glaxo, declined to comment.

Amicus said in October 2009 that it wouldn’t pursue final stage-testing of Plicera after a study showed the treatment failed to provide “clinically meaningful improvements.”

Amicus received a $500,000 grant from the Michael J. Fox Foundation for Parkinson’s Research on Dec. 8 to fund pre- clinical research into an orally administered treatment of Parkinson’s and Alzheimer’s diseases.

“We have enough cash now to continue to invest in all of our programs, including Amigal, and to get to the U.S. commercial launch without the need to raise additional funds,” Crowley said.

To contact the reporter on this story: Chris Kay in London at

To contact the editor responsible for this story: Phil Serafino at

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