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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 Biogen Exercises Option with Ionis to Develop and Commercialize Investigational Treatment BIIB067 for a Subtype of Familial Amyotrophic Lateral Sclerosis (ALS) Based on Positive Phase 1 Data
Positive interim analysis demonstrated proof-of-biology and proof-of-concept for BIIB067 Biogen plans to advance BIIB067 to a pivotal clinical study Biogen paid Ionis Pharmaceuticals a $35 million one-time upfront payment and may pay potential milestone payments and royalties CAMBRIDGE, Mass.
Toggle Summary First-in-class therapies using advanced antisense chemistries highlighted at 2018 American Society of Hematology Annual Meeting
Wholly-owned LICA drug for beta-thalassemia, IONIS-TMPRSS6-L Rx, demonstrates therapeutic potential with positive modulation of important disease-related biomarkers and increase of serum hepcidin in initial clinical study Ionis oncology research to be recognized with American Society of Hematology
Toggle Summary Ionis Pharmaceuticals to Present at Jefferies 2018 London Healthcare Conference
CARLSBAD, Calif. , Nov. 13, 2018 /PRNewswire/ --  Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, today announced that management will present a company overview at the Jefferies 2018 London Healthcare Conference on Wednesday, November 14 in London, United Kingdom
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

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