Pioneering antisense drug discovery

We are the leading company in RNA-targeted drug discovery and development, exploiting a proven novel drug discovery platform we created to generate a broad pipeline of first-in-class or best-in-class antisense drugs. The efficiency and broad applicability of our drug discovery platform allows us to discover and develop antisense drugs to treat a wide range of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, including neurological disorders, and cancer. The efficiency of our drug discovery technology allows us to employ a unique business strategy designed to maximize the value of our drugs and technology while maintaining an effective cost structure that limits our cash needs. Our business strategy is supported by our platform technology, our robust pipeline of drugs and our multifaceted partnering strategy, which have enabled us to focus on doing what we do best — to discover and develop novel antisense drugs.

We have created a mature and broad pipeline of 38 drugs in development that represents the potential for significant commercial opportunities in many therapeutic areas. We have a number of drugs in later-stage development that we believe represent significant near-term commercial opportunities. Volanesorsen (ISIS-APOCIIIRx) is a drug we designed to treat patients with severely high triglyceride levels, including patients with a severe and rare genetic condition called familial chylomicronemia syndrome, or FCS and patients with familial partial lipodystrophy, or FPLD, another severe and rare genetic condition. We initiated a Phase 3 study in patients with FCS that is designed to support a regulatory filing for marketing approval for volanesorsen.  We also plan to start this year a Phase 3 study in patients with FPLD.  In addition to volanesorsen, we are also evaluating ISIS-TTRRx and ISIS-SMNRx in Phase 3 studies. We designed these drugs to treat patients with severe and rare diseases, such as transthyretin amyloidosis, or TTR amyloidosis, and spinal muscular atrophy, or SMA, who have very limited therapeutic options. The significant unmet medical need and the severity of these diseases could warrant a rapid path to market. We believe all three of these drugs have the potential to reach the market in the next several years.

Our novel lipid-lowering product, KYNAMRO® (mipomersen sodium) injection, is on the market in the United States for patients with homozygous familial hypercholesterolemia, or HoFH. Patients with HoFH are at high cardiovascular risk and cannot reduce their low-density lipoprotein cholesterol, or LDL-C, sufficiently with currently available lipid-lowering therapies.

The efficiency and broad utility of our drug discovery technology supports the continued growth of our pipeline of antisense drugs. To maximize the value of our drugs and technologies, we have a multifaceted partnering strategy. Our partnering strategy provides us the flexibility to license each of our drugs at what we believe is the optimal time to maximize the near- and long-term value of our drugs. In this way, we can expand our and our partners’ pipelines with antisense drugs that we design to address significant medical needs while remaining small and focused.

Furthermore, as an innovator in RNA-targeted drug discovery and development, we have designed and executed a patent strategy that has provided us with strong and extensive protection for our drugs as well as many aspects of antisense drug discovery, development and manufacturing. We are continually making improvements to our drug discovery platform and our drugs and expanding our antisense technology to include new opportunities. We expect to remain the leader in all aspects of antisense technology advancement.

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