Isis receives $5M milestone payment from GSK for initiating ISIS-TTRRx Phase 1 study

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $5 million milestone payment from GlaxoSmithKline (GSK) related to the initiation of a Phase 1 clinical study for ISIS-TTRRx.  ISIS-TTRRx is designed to treat transthyretin amyloidosis, a severe and rare genetic disease characterized by slow degeneration of peripheral nerve and/or heart tissues. ISIS-TTRRx is the first antisense drug to enter development under Isis' strategic alliance with GSK.

"We are very pleased with the successes of our collaboration with GSK, a partner with significant expertise in the development of drugs to treat severe and rare diseases.  In a little over a year, we have begun clinical development on the first drug in this collaboration, and we look forward to moving additional drugs from this collaboration into our pipeline," said B. Lynne Parshall, Chief Operating Officer and CFO of Isis.   "The progress we have made with GSK is representative of the value this type of alliance can provide to us and to our partners.  The innovative structure of this alliance allows us to control and facilitate rapid development of drugs while working together with a high-quality partner to maximize the value of the drugs in late-stage development and commercialization.  Already we have earned $48 million in payments from GSK, including $8 million this year.  This year, we anticipate making significant progress in our drugs to treat severe and rare diseases, such as transthyretin amyloidosis."

ISIS-TTRRx is an antisense drug that targets transthyretin, or TTR, for the treatment of transthyretin amyloidosis, a fatal disease that affects one in 100,000 people or about 50,000 people worldwide.  ISIS-TTRRx will initially be developed for patients with familial amyloid polyneuropathy, or FAP, who have TTR build up in their peripheral nerves and experience the loss of motor functions, such as walking.  Although these patients experience debilitating nerve damage throughout their body, they typically die from wasting due to the accumulation of TTR in the intestinal tract, which prevents the proper absorption of nutrients.  Currently there are no approved drugs to treat transthyretin amyloidosis, and the only approved available option is liver transplant.  Unfortunately, availability of donor livers is very limited and only a fraction of patients are eligible for this very expensive and invasive procedure.  

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, Isis received an upfront $35 million payment from GSK and is eligible to receive license fees and milestone payments, totaling nearly $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.  With the initiation of the Phase 1 study of ISIS-TTRRx, Isis has earned $10 million in milestone payments under the collaboration.

SOURCE Isis Pharmaceuticals, Inc.