About Ionis Pharmaceuticals

Pioneering antisense drug discovery

Ionis Pharmaceuticals, Inc. is the leading company in RNA-targeted drug discovery and development, exploiting a proven novel drug discovery platform created to generate a broad pipeline of first-in-class or best-in-class antisense drugs. The efficiency and broad applicability of the drug discovery platform allows Ionis Pharmaceuticals 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 this drug discovery technology allows the company to employ a unique business strategy designed to maximize the value of its drugs and technology while maintaining an effective cost structure that limits cash needs. The company’s business strategy is supported by its platform technology, robust pipeline of drugs and multifaceted partnering strategy, which has enabled Ionis Pharmaceuticals to focus on doing what it does best — to discover and develop novel antisense drugs.

Ionis Pharmaceuticals has created a mature and broad pipeline of 38 drugs in development that represent the potential for significant commercial opportunities in many therapeutic areas. The pipeline consists of a number of drugs in later-stage development that could represent significant near-term commercial opportunities. Volanesorsen is a drug 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. The company initiated a Phase 3 study in patients with FCS that is designed to support a regulatory filing for marketing approval for volanesorsen , and also plans to start a Phase 3 study in patients with FPLD this year.  In addition to volanesorsen, they are also evaluating IONIS-TTRRx and nusinersen in Phase 3 studies.  These drugs are designed 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.  Ionis Pharmaceuticals believes all three of these drugs have the potential to reach the market in the next several years.

Ionis Pharmaceutical’s 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 their drug discovery technology supports the continued growth of the company’s pipeline of antisense drugs. To maximize the value of the drugs and technologies, Ionis Pharmaceuticals have a multifaceted partnering strategy. Its partnering strategy provides the flexibility to license each drug at the optimal time to maximize the near- and long-term value of the drug. In this way, the comapny can expand theirs and their partners’ pipelines with antisense drugs that are designed to address significant medical needs while remaining a small and focused company.

Furthermore, as an innovator in RNA-targeted drug discovery and development, Ionis Pharmaceuticals has designed and executed a patent strategy that has provided strong and extensive protection for their drugs as well as many aspects of antisense drug discovery, development and manufacturing. It is continually making improvements to the drug discovery platform, drugs and expanding antisense technology to include new opportunities. Ionis Pharmaceuticals expects to remain the leader in all aspects of antisense technology advancement.

Ionis Pharmaceuticals, Inc.