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Company Profile

Ionis 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, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over three dozen drugs in development. SPINRAZA® (nusinersen) has been approved in the U.S., Europe, Japan, Canada and Brazil for the treatment of spinal muscular atrophy (SMA). Biogen is responsible for commercializing SPINRAZA. Drugs that have successfully completed Phase 3 studies include inotersen , an antisense drug Ionis is developing to treat patients with TTR amyloidosis, and volanesorsen, an antisense drug discovered by Ionis and co-developed by Ionis and Akcea Therapeutics to treat patients with either familial chylomicronemia syndrome or familial partial lipodystrophy. Akcea, an affiliate of Ionis, is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders. If approved, volanesorsen will be commercialized through Ionis' affiliate, Akcea. Volanesorsen filings for marketing approval have been submitted in the U.S., EU and Canada. Inotersen is progressing toward regulatory filings for marketing authorization. Ionis' patents provide strong and extensive protection for its drugs and technology.

Recent Press Releases

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Date Title and Summary
Toggle Summary Ionis Licences New Antisense Drug for Kidney Disease to AstraZeneca
Ionis earns $30 million license fee CARLSBAD, Calif. , Feb. 20, 2018 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has licensed IONIS-AZ5-2.5 Rx (AZD2373) to the global, science-led biopharmaceutical company AstraZeneca (NYSE: AZN).
Toggle Summary The New England Journal of Medicine Publishes SPINRAZA® (nusinersen) Phase 3 Study Results in Individuals with Later-Onset Spinal Muscular Atrophy
SPINRAZA demonstrated stabilization or improvement in motor function in individuals with spinal muscular atrophy (SMA) where the natural history of the disease is a decline in motor function over time The majority of individuals treated with SPINRAZA demonstrated benefits in upper limb and general
Toggle Summary Ionis Pharmaceuticals to Hold 2017 Financial Results Conference Call
Webcast scheduled for Tuesday, February 27 at 11:30 a.m. Eastern Time CARLSBAD, Calif. , Feb. 13, 2018 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it will host a live webcast on Tuesday, February 27 at 11:30 a.m. Eastern Time to discuss its 2017 financial results
Toggle Summary Akcea Completes Enrollment in Phase 2b Study of AKCEA-APO(a)-LRx
CAMBRIDGE, Mass. , Feb. 07, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), today announced it had completed enrollment of a Phase 2b clinical study of investigational drug AKCEA-APO(a)-L Rx . Akcea is conducting the study in patients with high Lp(a) and established cardiovascular

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