US-based Isis Pharmaceuticals has started a Phase I clinical trial of ISIS-PKK, an antisense drug, being developed for treatment of patients with hereditary angioedemia (HAE), a rare genetic disease.
HAE is characterised by rapid and painful attacks of inflammation in the hands, feet, limbs, face, abdomen, larynx and trachea.
The disease affects an estimated 20,000 patients in the US and Europe and can be fatal if swelling occurs in the larynx.
The company said that ISIS-PKK is designed to alter the course of HAE, so has the potential to be best-in-class to treat HAE.
Isis senior vice-president of antisense drug discovery Brett Monia said ISIS-PKK 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," Monia said.
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"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.
"By inhibiting production of PKK, we believe that ISIS-PKK could be a best-in-class prophylactic treatment for patients with HAE."
The company said that patients with HAE have acute, frequent, debilitating and sometimes fatal attacks.
ISIS-PKK is designed to reduce the production of PKK in the liver, so alter the course of disease in patients who have severe HAE attacks.
The company intends to complete the Phase I trial of ISIS-PKK and begin a Phase II trial in patients with HAE.