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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' Inotersen NDA Accepted for Priority Review by the FDA
PDUFA date of July 6, 2018 CARLSBAD, Calif. , Jan. 8, 2018 /PRNewswire/ --  Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that its New Drug Application (NDA) for inotersen has been accepted for Priority Review by the U.S. Food and Drug Administration ( FDA ).
Toggle Summary Akcea Initiates Phase 2b Study of AKCEA-APOCIII-LRx in Patients with Hypertriglyceridemia and Established Cardiovascular Disease
CAMBRIDGE, Mass. , Jan. 05, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc. , focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders, today announced the
Toggle Summary Ionis Pharmaceuticals Announces the Initiation of a Clinical Study of its First Orally Delivered Antisense Drug for the Treatment of Gastrointestinal Disorders
- Ionis Earns $5 Million Milestone Payment from Janssen CARLSBAD, Calif. , Dec. 21, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced that it has earned a $5 million milestone payment associated with initiation and enrollment in a newly initiated Phase 1 clinical study
Toggle Summary Biogen and Ionis Enter into New Collaboration to Identify Novel Therapies for the Treatment of Spinal Muscular Atrophy
CAMBRIDGE, Mass. & CARLSBAD, Calif. --(BUSINESS WIRE)--Dec. 19, 2017-- Biogen (Nasdaq: BIIB) and Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that they have entered into a new collaboration agreement to identify new antisense oligonucleotide drug candidates for the treatment of spinal

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