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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 Phase 2 Results on AKCEA-APO(a)-LRx Presented in Late-Breaking Clinical Trial Presentation at AHA Scientific Sessions
Approximately 98% of patients receiving highest dose achieved reductions in Lp(a) levels below the established threshold of risk for CVD events Favorable safety and tolerability profile observed in largest study ever conducted in patients with cardiovascular disease and elevated Lp(a) BOSTON and
Toggle Summary Ionis Pharmaceuticals to Host Investor Day
Webcast on Friday, December 7 at 7:30 a.m. ET at www.ionispharma.com CARLSBAD, Calif. , Nov. 9, 2018 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, today announced that management will host an Investor Day on Friday, December 7 th at 7:30 a.m.
Toggle Summary Akcea and Ionis Announce Late-Breaking Presentation of Data From AKCEA-APO(a)-LRx at the American Heart Association Scientific Sessions
Results to be presented show a significant reduction of Lp(a) levels, favorable safety and tolerability profile BOSTON and CARLSBAD, Calif., Nov. 08, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals,
Toggle Summary Ionis Pharmaceuticals to Present at Upcoming Investor Conferences
CARLSBAD, Calif. , Nov. 7, 2018 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, today announced that management will present a company overview at the following investor conferences: Stifel 2018 Healthcare Conference on Wednesday, November 14, 2018

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