ISIS Pharmaceuticals Earns $5 Million from GSK for Initiation of Phase 1 Study of ISIS-GSK4-L Rx

CARLSBAD, Calif., Oct. 26, 2015 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $5 million milestone payment from GSK related to the initiation of a Phase 1 study of ISIS-GSK4-LRx (formerly ISIS-GSK4Rx) in healthy volunteers.  ISIS-GSK4-LRx is one of five drugs in development with GSK and is designed to treat patients with an undisclosed ocular disorder.

About ISIS and GSK
GSK and Isis have established an alliance focused on leveraging antisense technology to develop drugs to treat rare and infectious diseases.  This alliance provides GSK with access to Isis' expertise in RNA-targeted drug discovery and development, with Isis retaining responsibility for the discovery and development of drugs to the alliance targets from inception to proof-of-concept.  GSK has the option to license each antisense program at a particular stage in development.  Current development-stage programs include ISIS-HBVRx for patients with hepatitis B virus, ISIS-GSK4-LRx for patients with an undisclosed ocular disorder, ISIS-RHO-2.5Rx for patients with autosomal dominant retinitis pigmentosa, ISIS-GSK6-LRx for patients with an undisclosed viral disorder and ISIS-TTRRx, which is in Phase 3 development for patients with transthyretin amyloidosis.  To date, Isis has generated nearly $135 million from GSK as its partnered programs have advanced in development.

ABOUT ISIS PHARMACEUTICALS, INC.
Isis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases.  Using its proprietary antisense technology, Isis has created a large pipeline of first-in-class or best-in-class drugs, with over a dozen drugs in mid- to late-stage development.  Drugs currently in Phase 3 development include volanesorsen, a drug Isis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with familial chylomicronemia syndrome and familial partial lipodystrophy; ISIS-TTRRx, a drug Isis is developing with GSK to treat patients with all forms of TTR amyloidosis; and ISIS-SMNRx, a drug Isis is developing with Biogen to treat infants and children with spinal muscular atrophy.  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' alliance with GSK, the discovery, development, activity, therapeutic and commercial potential and safety of ISIS-GSK4-L.  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 December 31, 2014, 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.  Akcea Therapeutics™ is a trademark of Isis Pharmaceuticals, Inc. 

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Accelerate Biologics, Gene and Cell Therapy Product Development partnering with GenScript ProBio

GenScript ProBio is the bio-pharmaceutical CDMO segment of the world’s leading biotech company GenScript, proactively providing end-to-end service from drug discovery to commercialization with professional solutions and efficient processes to accelerate drug development for customers.

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