Isis Pharmaceuticals starts Phase 1 clinical study of ISIS-PKKRx to treat patients with HAE

Published on May 21, 2014 at 1:25 AM · No Comments

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it initiated a Phase 1 clinical study of ISIS-PKKRx.  ISIS-PKKRx is an antisense drug in development to treat patients with hereditary angioedemia (HAE).  HAE is a rare genetic disease that is characterized by rapid and painful attacks of inflammation in the hands, feet, limbs, face, abdomen, larynx and trachea.  HAE affects approximately 20,000 patients in the United States and Europe and can be fatal if swelling occurs in the larynx.  ISIS-PKKRx is designed to alter the course of HAE and therefore has the potential to be best-in-class for the treatment of HAE.  

"ISIS-PKKRx targets the prekallikren (PKK), a protein produced in the liver that plays an important role in the activation of inflammatory mediators associated with acute attacks of HAE.  This is a well-validated pathway and one that is easily accessible using our antisense technology.  In our preclinical models of HAE, we observed significant reductions of PKK that were associated with the amelioration of symptoms of disease, including the prevention of tissue swelling," said Brett Monia, Ph.D., senior vice president of antisense drug discovery at Isis.  "By inhibiting the production of PKK, we believe that ISIS-PKKRx could be a best-in-class prophylactic treatment for patients with HAE."

ISIS-PKKRx is a wholly owned Isis drug in development for the prevention of HAE attacks.  HAE is a rare and severe disease in which patients have acute, frequent, debilitating and sometimes fatal attacks.  ISIS-PKKRx­ is an antisense drug designed to reduce the production of PKK in the liver and therefore alter the course of disease in patients who have severe HAE attacks.  Isis plans to complete the Phase 1 study of ISIS-PKKRx and initiate a Phase 2 study in patients with HAE.

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Isis Pharmaceuticals, Inc.

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