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.

Go deeper with GlobalData

Premium Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

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.

"By inhibiting production of PKK, we believe that ISIS-PKK could be a best-in-class prophylactic treatment for patients with 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.

See Also:

"This is a well-validated pathway and one that is easily accessible using our antisense technology," Monia said.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

"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.