Amicus will receive an initial licensing payment of $50 million and is eligible to receive an additional $150 million tied to clinical and regulatory approvals, $240 million in sales-based milestones and tiered double-digit royalties. According to the agreement, U.K.-based Shire will have the rights to commercialize the products outside of the U.S., and New Jersey-based Amicus will have the right to commercialize inside the U.S.
The companies will share equally the costs toward global approval of the Amicus compounds: Amigal, Plicera and AT2220. Amigal is in phase II development for Fabry disease, Plicera is in phase II clinical trials for Gaucher disease, and AT2220 is in phase I trials for Pompe disease. (Shire also owns Replagal to treat the genetic disorder Fabry disease.)
The diseases belong to a class of lysosomal storage disorders, which are caused by a mutation in the genes that control enzymes responsible for breaking down lipids, sugars and other materials that can build up to harmful levels in the body.Genzyme (GENZ) makes Cerezyme for Gaucher disease and said in October that it completed enrollment for the phase II trial of an oral therapy for Gaucher disease and has requested a meeting with the FDA to discuss an expedited development strategy, hoping to bring the drug to market in three to four years. Genzyme also reported $53.6 million in sales from Myozyme therapy for Pompe disease and $104.6 million in sales of Fabrazyme, a recombinant form of a human enzyme to treat Fabry disease. It submitted an application to the FDA for approval of a larger-scale manufacturing process to supply Myozyme in the U.S., and it expects a decision from the agency in the first quarter of next year. Amicus shares, which surged about 12% in early trading, were up 53 cents, or 3.3% at $16.61 around midday.