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Isis Initiates Phase 1 Study of ISIS-TTRRx and Earns $5 Million Milestone Payment From GlaxoSmithKline
Date:5/19/2011

e 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.

ABOUT ISIS-TTRRx

Transthyretin amyloidosis is a genetic disease in which the patient inherits a mutant gene that produces a misfolded form of TTR, which progressively accumulates in tissues, impairing their function.  In patients with transthyretin amyloidosis, both the mutant and normal forms of TTR can build up as fibrils in tissues, including heart, peripheral nerves, and the gastrointestinal tract.  The presence of fibrils interferes with the normal functions of these tissues, and as the fibril deposits enlarge more tissue damage occurs and the disease worsens.  There are two common types of transthyretin amyloidosis, familial amyloid cardiomyopathy, or FAC, which affects more than 40,000 patients worldwide, and F
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SOURCE Isis Pharmaceuticals, Inc.
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