Press Releases
Isis Announces Expedited Development Of ISIS-TTR Rx And Amendment To The Collaboration With GSK To Support Phase 2/3 Study
"In our Phase 1 study, we demonstrated that ISIS-TTRRx was generally well tolerated and produced dose-dependent significant reductions in TTR protein with several subjects reaching TTR protein levels that were below the limit of assay detection," said
Under the terms of the original collaboration agreement with GSK, which includes five programs in addition to the TTR program, Isis is eligible to receive on average up to
"We have a robust pipeline of novel new drugs. Many of which, including ISIS-TTRRx, could advance into registration studies in the next 12 to 18 months. Together with GSK, we have been able to rapidly advance the TTR program from research to late-stage clinical development in just over two years," said
ABOUT ISIS-TTRRx
Transthyretin amyloidosis is a genetic disease in which the patient inherits a mutant gene that produces a misfolded form of transthyretin (TTR) protein, which progressively accumulates in tissues, impairing their function. In patients with transthyretin amyloidosis, both the mutant and normal forms of TTR can build up as fibrils in tissues, including heart, peripheral nerves, and the gastrointestinal tract. The presence of TTR aggregates interferes with the normal functions of these tissues, and as the TTR protein aggregates enlarge more tissue damage occurs and the disease worsens. There are two common types of transthyretin amyloidosis, familial amyloid polyneuropathy(FAP), which affects more than 10,000 patients worldwide, and familial amyloid cardiomyopathy (FAC), which affects more than 40,000 patients worldwide. Patients with FAP have TTR build up in peripheral nerve tissue leading to the loss of nerve function and wasting. Patients with FAC have TTR build up in the heart muscle and succumb to heart failure five to six years after symptom onset. ISIS-TTRRx is an investigational drug that is designed to inhibit the production of all forms of TTR, and could offer an alternative approach to treat all types of transthyretin-related amyloidosis.
ABOUT
Isis is exploiting its leadership position in antisense technology to discover and develop novel drugs for its product pipeline and for its partners. Isis' broad pipeline consists of 25 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, and cancer. Isis' partner, Genzyme, plans to commercialize Isis' lead product, KYNAMRO™, following regulatory approval. Isis' patents provide strong and extensive protection for its drugs and technology. Additional information about Isis is available at www.isispharm.com.
This press release includes forward-looking statements regarding Isis' collaboration with GlaxoSmithKline and the development, activity, therapeutic potential and safety of ISIS-TTRRx. Any statement describing Isis' goals, expectations, financial or other projections, intentions or beliefs, including the planned commercialization of KYNAMRO, 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
Isis Pharmaceuticals® is a registered trademark of
SOURCE
D. Wade Walke, Ph.D., Executive Director, Corporate Communications and Investor Relations, +1-760-603-2741, or Amy Blackley, Ph.D., Associate Director, Corporate Communications, +1-760-603-2772