Isis Announces That ISIS-TTR Rx Was Granted Fast Track Designation For The Treatment Of Patients With FAP

CARLSBAD, Calif., Dec. 13, 2012 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that the United States Food and Drug administration has granted ISIS-TTR_Rx fast track designation for the treatment of familial amyloid polyneuropathy (FAP).  ISIS-TTR_Rx is an antisense drug in development with GlaxoSmithKline (GSK) for the treatment of transthyretin (TTR) amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues.  Isis and GSK recently amended the clinical development plan and financial terms relating to ISIS-TTR_Rx to support a registration-directed Phase 2/3 clinical study on ISIS-TTR_Rx, which is expected to start this month.

"We are pleased to have received orphan drug status and fast track designation for ISIS-TTR_Rx for patients with FAP.  ISIS-TTR_Rx is our most advanced drug from our severe and rare disease franchise and represents a significant near-term commercial opportunity for us," said B. Lynne Parshall, chief operating officer and chief financial officer at Isis.  "We look forward to continuing to move ISIS-TTR_Rx toward the market for patients who have very limited therapeutic options."

ISIS-TTR_Rx is part of the Isis-GSK strategic alliance to develop RNA therapeutics for rare and infectious diseases.  Upon initiation of the Phase 2/3 study, Isis will receive a $7.5 million milestone payment and is eligible to earn an additional $50 million in pre-licensing milestone payments to support the Phase 2/3 study of ISIS-TTR_Rx­.  In addition, Isis is eligible to receive regulatory and sales milestones and double-digit royalties on sales of ISIS-TTR_Rx.

ABOUT ISIS-TTR_Rx

Transthyretin amyloidosis is a genetic disease in which the patient inherits a mutant gene that produces a misfolded form of TTR, 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 cardiomyopathy, or FAC, which affects more than 40,000 patients worldwide, and FAP, which affects more than 10,000 patients worldwide.  Patients with FAC have TTR build up in the heart muscle and succumb to heart failure five to six years after symptom onset.  Patients with FAP have TTR build up in peripheral nerve tissue leading to the loss of nerve function and wasting.  ISIS-TTR_Rx 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 PHARMACEUTICALS, INC.

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™, in the United States and Europe 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.

ISIS PHARMACEUTICALS' FORWARD-LOOKING STATEMENT

This press release includes forward-looking statements regarding Isis' collaboration with GlaxoSmithKline, the discovery, development and potential of drugs for severe and rare diseases, and the development, activity, therapeutic potential and safety of ISIS-TTR_Rx. 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 December 31, 2011 and its most recent quarterly report on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from the Company.

In this press release, unless the context requires otherwise, "Isis," "Company," "we," "our," and "us" refers to Isis Pharmaceuticals and its subsidiaries.

Isis Pharmaceuticals® is a registered trademark of Isis Pharmaceuticals, Inc.  KYNAMRO™ is a trademark of Genzyme Corporation.

SOURCE Isis Pharmaceuticals, Inc.