Deal includes up to six programs and $1.5 billion in milestones related to development and commercialization.
GlaxoSmithKline (GSK) is paying Isis Pharmaceuticals $35 million up front to discover new antisense drugs against serious rare disorders including infectious diseases and specific conditions causing blindness.
The agreement will exploit Isis’ antisense drug discovery platform and covers up to six program. The firm could receive $20 million in additional milestones per program that successfully negotiates Phase II trials.
GSK retains an option to license compounds at the proof-of-concept stage, after which it will be responsible for all further development and commercialization. Isis could potentially reap nearly $1.5 billion in license fees and milestones if all six programs were to be developed for one or more indications and commercialized through to predetermined sales targets.
“This alliance is exactly the type of deal we want to do,” comments Stanley T. Crooke, chairman and CEO at Isis. “We retain control of the discovery and early development of our drugs while working together with a very high quality partner to maximize the value of the drugs in late-stage development and commercialization.”
GSK says Isis’ antisense platform will allow it to address certain severe diseases in a manner that has previously not been possible. “Isis Pharmaceuticals is a leader in antisense technology, and this new alliance will enhance our discovery platform in this promising research area,” remarks Patrick Vallance, Ph.D., svp and head of drug discovery at GSK.
Isis currently has 22 drugs in its pipeline, including 17 in clinical development. Disease areas include oncology, metabolic diseases, cardiovascular and neurodegenerative disorders, as well as inflammation. The company is developing its pipeline both in-house and through collaborations with firms like Genzyme, Bristol Myers Squibb, Novartis, and Eli Lilly.
Isis’ marketed antisense drug, Vitravene, was approved by FDA in 1998 as a treatment for cytomegalovirus retinitis in AIDS patients. The drug was developed in collaboration with Novartis Ophthalmics, Isis’ worldwide distribution partner for the drug.
The firm’s lead investigational candidate is Mipomersen, a first-in-class apoB, synthesis inhibitor, currently in Phase III development to reduce LDL-C in patients with high cholesterol who are at high cardiovascular risk. Mipomersen is a second-generation antisense drug administered to patients through a once-weekly subcutaneous injection. In June 2008, Isis finalized a deal with Genzyme, in which it got $175 million up front and gave Genzyme an exclusive, worldwide license to the drug. The firm had three months earlier taken a $150 million equity investment in Isis.
The two companies are jointly funding development of Mipomersen and in February reported positive data from a second Phase III trial evaluating Mipomersen in patients with heterozygous familial hypercholesterolaemia. During the latter half of 2009, Isis and Genzyme reported positive data from the first Phase III trial in patients with homozygous FH. Data from the remaining two Phase III studies including a trial in patients with severe hypercholesterolemia are expected in mid-2010.
Isis says Genzyme’s initial U.S. and EU regulatory filings for Mipomersen will seek marketing approval for the homozygous FH indication and may include patients with severe hypercholesterolemia.