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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 Presents New Data from NEURO-TTR Study and Highlights Programs from Its Neurological Disease Franchise at ANA Congress
Inotersen treatment resulted in a 20-point benefit in mNIS+7 compared to placebo Ionis antisense programs featured in 11 platform and poster presentations at ANA CARLSBAD, Calif. , Oct. 15, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, today
Toggle Summary Ionis Pharmaceuticals Initiates Clinical Study of IONIS-MAPT Rx in Patients with Alzheimer's Disease
Antisense drug designed to reduce tau protein in the CNS Ionis earns $10 million milestone payment from Biogen CARLSBAD, Calif. , Oct. 13, 2017 /PRNewswire/ --  Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced that it has initiated a Phase 1/2a clinical study of IONIS-MAPT Rx in patients
Toggle Summary Biogen’s SPINRAZA® (nusinersen) Data Show Earlier Treatment Initiation May Lead to Improved Motor Function Across a Broad Population of People Living with Spinal Muscular Atrophy
New data from Phase 3 ENDEAR study demonstrated earlier initiation of treatment with SPINRAZA may improve motor function outcomes in infants with Spinal Muscular Atrophy (SMA) Phase 2 EMBRACE interim analysis showed greater motor milestone achievement in infants and children treated with SPINRAZA,
Toggle Summary Akcea and Ionis Announce Filing of New Drug Submission for Volanesorsen to Health Canada
Health Canada grants priority review for volanesorsen NDS filing Volanesorsen could be the first approved treatment for patients with FCS CAMBRIDGE, Mass. and CARLSBAD, Calif., Sept. 12, 2017 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc.

Upcoming Events

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Date Event Details
Summary Toggle 10/15/17 - 10/17/17

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