Isis Pharmaceuticals, Inc has earned a US$ 5 million milestone payment from GlaxoSmithKline related to the designation as a development candidate of a drug to treat an undisclosed rare and serious disease. Isis will develop the drug to phase-2 proof-of-concept, at which time GSK has the option to license it.

"We are pleased to have achieved this milestone so early in our collaboration with GSK. This alliance is an important step in enhancing our focus on rare disease, and we are excited to move our third drug in this area into our pipeline," said Stanley T Crooke, chairman and chief executive officer of Isis. "Under this new model of partnership we are able to retain control of this asset to facilitate its rapid development, while benefiting from GSK's expertise and new focus in rare diseases, and financially, from milestone payments even before GSK exercises its license option at Phase 2 proof-of-concept."

In March 2010, Isis entered into a strategic alliance with GSK to develop RNA therapeutics for rare and infectious diseases. Under the terms of the agreement, which covers up to six programs, Isis received an upfront US$ 35 million payment from GSK and is eligible to receive license fees and milestone payments, totalling nearly US$ 1.5 billion, in the event all six programs are successfully developed for one or more indications and commercialized through to pre-agreed sales targets. The alliance applies Isis' antisense drug discovery platform to discover and develop new therapeutics against targets for rare and serious diseases, including infectious diseases and some conditions causing blindness.

Isis is exploiting its expertise in RNA to discover and develop novel drugs for its product pipeline and for its partners.