GSK boosts stake in Amicus as PhIII Fabry data looms

Twenty months after stepping up with $60 million in cash to invest in Amicus Therapeutics' ($FOLD) experimental therapy for Fabry disease, GlaxoSmithKline ($GSK) has stepped back up to the table with an added $18.6 million equity investment in the biotech to expand their rare disease pact.

In this new deal GSK and the Cranbury, NJ-based biotech will co-develop all formulations of migalastat HCl for Fabry disease, including a co-formulation of migalastat HCl with GSK/JCR Pharmaceutical's investigational enzyme replacement therapy; a split in commercialization, with Amicus holding on to the U.S. market as GSK shoulders the rest of the world; and a boost in GSK's stake to 19.9%, bringing it back to the level GSK had obtained in the original deal.

Migalastat HCl--dubbed Amigal--is now in two late-stage studies for Fabry disease, with pivotal data expected in the third quarter. And there's a mid-stage study of the drug combined with an ERT. This lead program fits neatly into GSK's plans to expand in rare diseases, with a heavy emphasis on partnerships like these. For Amicus, the pact helps position the biotech ahead of an attempt to transition into a commercial-stage company, taking the reins in the U.S. market.

"Amicus has a very successful track record as our development partner, and long-standing relationships with the Fabry community," said Marc Dunoyer, the global head of GSK Rare Diseases. "We look forward to their leadership in the U.S. commercialization of now several potential medicines for patients with Fabry disease. This is an important step in our strategic vision, allowing us to undertake and fund an enlarged scientific program with a view to turning molecules into medicines for rare diseases faster and more effectively than ever before."

GSK also committed to a package of milestones worth up to $170 million in the original pact, struck in October, 2010.

- here's the press release