AstraZeneca and Isis Pharmaceuticals to Co-Develop Targeted Oligonucleotide Delivery Methods

CARLSBAD, Calif., Nov. 13, 2014  -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) and AstraZeneca today announced a strategic alliance to discover and develop novel delivery methods for antisense oligonucleotides. The new delivery approaches seek to target the desired tissue more effectively. The agreement builds on an existing collaboration between AstraZeneca and Isis Pharmaceuticals, a leader in the field of antisense, and supports AstraZeneca's research and development capabilities in the area of antisense oligonucleotide-based therapeutics and RNA biology. Initial project areas will be oncology and cardiovascular and metabolic diseases (CVMD).

Antisense oligonucleotides are short, single strands of DNA or RNA molecules. Rather than modulating the activity of already-formed proteins, antisense oligonucleotides act before proteins are produced at the level of messenger RNA in the cell, thus opening up new opportunities for therapeutic intervention. The new delivery methods will aim to enhance the access of antisense oligonucleotides into specific organs and cells. The methods build on Isis Pharmaceuticals' successful Ligand Conjugation Antisense (LICA) technology. The first example of this technology being Isis' GalNac-conjugated antisense oligonucleotides targeting liver hepatocytes, which lowers the therapeutic dose needed for liver targets by approximately 10-fold.

Under the terms of the agreement, each party will fund its own contribution and commit investigators to the collaboration. In line with AstraZeneca's open innovation approach, the companies will work together on an agreed programme and share rights to the results. Isis can apply learnings from this collaboration broadly across its antisense technology platform and AstraZeneca can similarly apply learnings across its broader RNA-based, small molecule and antibody research and development activities. 

Isis Pharmaceuticals and AstraZeneca entered into a collaboration, development and license agreement in 2012 in oncology, subsequently expanded in 2013 to include CVMD. Under the 2012 agreement, one of the molecules being developed is AZD9150 (ISIS-STAT3Rx), a first-in-human, first-in-class, antisense oligonucleotide inhibitor of STAT3, which is being developed as an immunomodulatory agent in combination with MEDI4736, AstraZeneca's investigational anti-PD-L1 immune checkpoint inhibitor. A second product of that collaboration, an antisense oncology compound targeting the androgen receptor AZD5312 (ISIS-ARRx), is in Phase I trials. The new delivery collaboration announced today builds on this existing relationship and is expected to also bring benefits to these programs.

"This exciting collaboration very much supports AstraZeneca's research and development in the area of RNA-based therapeutics. If successful, we'll have a way to selectivity modulate therapeutic targets in specific cell types that are intractable to small molecules and antibodies. This could lead to a number of ground breaking drugs for both oncology and cardiovascular and metabolic diseases," saidSusan Galbraith, Head of the Oncology Innovative Medicines Unit, AstraZeneca.

Brett Monia, Senior Vice President of Antisense Drug Discovery, Isis Pharmaceuticals said, "The collaboration expansion announced today builds upon an already successful agreement between Isis and AstraZeneca. Together, we have advanced ISIS-STAT3Rx and ISIS-ARRx in clinical development, both of which are being evaluated in patients with cancer. This opportunity also complements our internal efforts to expand the use of our technology and develop drugs with broad therapeutic applicability."

"RNA molecules play an increasingly important role in our research portfolio. We are delighted to be expanding our existing, strong collaboration with Isis Pharmaceuticals, who are leading players in RNA biology, with the aim of improving the delivery of antisense oligonucleotides to specific cardiovascular and metabolic tissue targets," said Marcus Schindler, Head of the CVMD Innovative Medicines Unit, AstraZeneca.

About AstraZeneca

AstraZeneca is a global, innovation-driven biopharmaceutical business that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of cardiovascular, metabolic, respiratory, inflammation, autoimmune, oncology, infection and neuroscience diseases. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information please visit:


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 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 the United States and other countries for the treatment of patients with homozygous FH.  Isis has numerous drugs in Phase 3 development in severe and rare and cardiovascular diseases.  These include a novel triglyceride lowering drug, ISIS-APOCIIIRx, for patients with familial chylomicronemia syndrome; ISIS-TTRRx, which Isis is developing with GSK to treat patients with the polyneuropathy form of TTR amyloidosis; and, ISIS-SMNRx, which Isis is developing with Biogen Idec to treat infants and children with spinal muscular atrophy, a severe and rare neuromuscular disease.  Isis' patents provide strong and extensive protection for its drugs and technology.  Additional information about Isis is available at


This press release includes forward-looking statements regarding Isis' collaboration with AstraZeneca and the discovery, development, distribution, delivery, activity, therapeutic potential, safety and commercialization of antisense drugs arising from the collaboration.  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 endedDecember 31, 2013, 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.

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