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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 Reports Second Quarter 2018 Financial Results
Year-to-date revenues increased 15%, driven by increased SPINRAZA revenue TEGSEDI approved and on track to launch in the EU Positive FDA Advisory Committee vote recommending approval of WAYLIVRA Conference call and webcast today, August 7, 2018, at 11:30 a.m. Eastern Time CARLSBAD, Calif. , Aug.
Toggle Summary IONIS-HTT Rx (RG6042) Granted PRIME Designation by the European Medicines Agency for the Treatment of People with Huntington's Disease
IONIS-HTT Rx is the first and only drug to demonstrate reduction of mutant huntingtin protein, the underlying cause of Huntington's disease, in patients Roche plans to initiate a pivotal study of IONIS-HTT Rx (RG6042) CARLSBAD, Calif. , Aug. 2, 2018 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
Toggle Summary Akcea Therapeutics and PTC Therapeutics Collaborate to Commercialize Two Rare Disease Drugs in Latin America
- PTC in-licenses regional rights to TEGSEDI™ and WAYLIVRA™ from Akcea - SOUTH PLAINFIELD, N.J. and CAMBRIDGE, Mass. , Aug. 2, 2018 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT), and Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc.
Toggle Summary Ionis Pharmaceuticals to Hold Second Quarter 2018 Financial Results Webcast
Webcast scheduled for Tuesday, August 7th at 11:30 a.m. Eastern Time CARLSBAD, Calif. , July 24, 2018 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it will host a live webcast on Tuesday, August 7 th at 11:30 a.m. Eastern Time to discuss its second quarter 2018

Upcoming Events

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Date Event Details
Summary Toggle 10/16/18
Summary Toggle 10/08/18 - 10/10/18

Location

La Jolla, CA

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©1999 - 2017 Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals, Inc.