"Despite currently available prophylactic therapies, we believe that there remains a significant need for patients with HAE. PKK is a protein produced in the liver that plays an important role in the activation of inflammatory mediators associated with acute attacks of HAE. Since the inappropriate activation of PKK leads to HAE, these data could be predictive of the potential benefit we hope to see in patients with HAE," said
The Phase 1 study of ISIS-PKKRx was a blinded, placebo-controlled, dose-escalation study in healthy volunteers. The study was designed to assess the safety, tolerability and pharmacokinetics of ISIS-PKKRx. ISIS-PKKRx was evaluated in single and multiple doses ranging from 50 mg per week up to 400 mg per week for the single dose and 100 mg up to 400 mg for the multiple doses. After only 3 weeks of dosing, subjects in the 100, 200, 300 and 400 mg multiple-dose cohorts displayed a mean reduction of PKK of 33, 69, 87 and 92 percent, respectively, from baseline. In this study, ISIS-PKKRx was generally well tolerated.
"ISIS-PKKRx is a new addition to our severe and rare disease franchise, and one that we plan to develop into later-stage clinical studies on our own. We have the internal resources and capabilities to easily and rapidly move this program forward in patients with HAE," said
Isis is exploiting its leadership position in RNA-targeted technology to discover and develop novel drugs for its product pipeline and for its partners. Isis' broad pipeline consists of 34 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, including neurological disorders, and cancer. Isis' partner, Genzyme, is commercializing Isis' lead product, KYNAMRO®, in
This press release includes forward-looking statements regarding the discovery, development, activity, therapeutic and commercial potential and safety of ISIS-PKKRx for the treatment of patients with HAE. Any statement describing Isis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Isis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Isis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Isis' programs are described in additional detail in Isis' annual report on Form 10-K for the year ended
In this press release, unless the context requires otherwise, "Isis," "Company," "we," "our," and "us" refers to
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D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741 or Amy Blackley, Ph.D., Associate Director, Corporate Communications, 760-603-2772