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Isis Selects Development Candidate, Isis-AATRx, to Treat AATD-Associated Liver Disease
Date:10/11/2011

CARLSBAD, Calif., Oct. 11, 2011 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has designated the second development candidate, ISIS-AATRx, in its collaboration with GlaxoSmithKline (NYSE: GSK).   Isis earned a $5 million milestone payment from GSK for the selection of ISIS-AATRx.  ISIS-AATRx is an antisense drug designed to treat liver disease caused by alpha-1 antitrypsin deficiency, or AATD, by inhibiting the production of an abnormal protein, which accumulates and causes damage in the liver.  AATD-associated liver disease is a rare disease that can lead to severe and sometimes fatal liver dysfunction.  Isis will develop ISIS-AAT Rx to Phase 2 proof-of-concept, at which time GSK has the option to license the drug.  

"Our collaboration with GSK is off to a very successful start.  Within less than 2 years we have identified two development candidates, with the first, ISIS-TTRRx, expected to finish our Phase 1 study this year and poised to start Phase 2 next year.  This collaboration exemplifies our ability to quickly select targets and discover drugs with promise to treat severe and rare diseases," said Brett P. Monia, Vice President of Drug Discovery and Corporate Development.  "We are pleased to continue to benefit from GSK's expertise as we further expand the breadth of our severe and rare disease franchise and broaden our focus on additional life-threatening diseases for patients who have very limited treatment options."

ISIS-AATRx is an antisense drug designed to inhibit the production of alpha-1 antitrypsin, or AAT, for the treatment of liver disease in patients with AATD.  AATD is a genetic disease that affects 1 out of every 2,500 people in the United States and can lead to severe liver disease, including liver scarring, cirrhosis, and liver cancer. There are currently no available
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SOURCE Isis Pharmaceuticals, Inc.
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