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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 Enters New Collaboration with Partner to Develop IONIS-FB-L Rx for Complement-Mediated Diseases
Transaction valued at up to $760 million, including $75 million upfront payment, plus royalties up to 20% Planned Phase 2 program to begin in early 2019 CARLSBAD, Calif. , Oct. 10, 2018 /PRNewswire/ --  Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced a new collaboration with Roche to
Toggle Summary Akcea Announces Its Access and Distribution Strategy for TEGSEDI™ (inotersen)
Express Scripts’ Accredo® selected as specialty pharmacy for its expertise in providing timely access and support services for patients living with rare diseases BOSTON , Oct. 05, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc.
Toggle Summary Akcea and Ionis Receive FDA Approval of TEGSEDI™ (inotersen) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults
TEGSEDI provides powerful knockdown of TTR protein, delivering significant and sustained benefits in neuropathy and quality of life Hereditary Transthyretin (hATTR) Amyloidosis Infographic AKCEA CONNECT™ Infographic TEGSEDI™ (inotersen) Product Shot Ionis Pharmaceuticals Logo TEGSEDITM(inotersen)
Toggle Summary Akcea and Ionis Announce Approval of TEGSEDI™ (inotersen injection) in Canada
First and only treatment of its kind for Canadians with Hereditary Transthyretin-mediated Amyloidosis (hATTR amyloidosis) with Polyneuropathy – a rare and inherited disease OTTAWA and BOSTON and CARLSBAD, Calif., Oct. 04, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics Inc.

Upcoming Events

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Date Event Details
Summary Toggle 10/16/18
Summary Toggle 12/07/18 7:30 am EST
Marriott East Side, 525 Lexington Avenue, New York, NY 10017

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