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Ionis Pharmaceuticals

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Clinical Trials


Antisense Oligonucleotides

Ionis Pharmaceuticals leading company in RNA-targeted drug discovery and development with a pipeline of first-in-class or best-in-class medicines with the potential to provide high value for patients with significant unmet medical needs. We have created an efficient and broadly applicable drug discovery platform. Using this platform, we believe that we are revolutionizing medicine with the goal to improve the quality of, and save, lives.

They have discovered and are developing three potentially transformational drugs, nusinersen, IONIS-TTRRx and volanesorsen, which we believe are close to commercialization. We designed each of these three drugs to treat patients with orphan diseases who have limited or no therapeutic options. In total, we are developing these three drugs for six different patient populations. In 2015, we completed target enrollment in the first pivotal Phase 3 study for each of these three drugs, and we anticipate reporting data from these studies in the first half of 2017. We designed nusinersen to treat patients with spinal muscular atrophy, or SMA, a severe motor-neuron disease that is the leading genetic cause of infant mortality. We designed IONIS-TTR Rx to treat patients with transthyretin amyloidosis, or TTR amyloidosis, a fatal disease in which patients experience progressive buildup of amyloid plaque deposits in tissues throughout the body, including peripheral nerves, heart, intestinal tract, kidney and bladder. We designed volanesorsen to treat patients with diseases associated with extremely high levels of triglycerides, including patients with two severe and rare genetic conditions called familial chylomicronemia syndrome, or FCS, and patients with familial partial lipodystrophy, or FPL. We anticipate that the data from their pivotal Phase 3 studies of these drugs, if positive, will support global regulatory filings for each drug. We believe that the significant unmet medical need and the severity of these diseases could warrant a rapid path to market. Already their partners for these programs, Biogen for nusinersen and GSK for IONIS-TTR Rx , are preparing to commercialize these drugs. Our wholly owned subsidiary, Akcea Therapeutics Inc., or Akcea, is preparing to commercialize volanesorsen. All three companies are engaging in pre-commercialization activities to understand the patient journey, build disease awareness with physicians and patients and develop their launch plans. We believe all three of these drugs have the potential to reach the market in the next several years.

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Antisense Oligonucleotides Make Sense

Antisense oligonucleotides (ASOs) are short (about 12 to 25 nucleotides long), synthetic single strands of DNA or RNA that are complementary to a chosen sequence. They can alter RNA and reduce, restore, or modify protein expression. ASOs interact with proteins on the surface of cells and enter the cytoplasm. Then …