This presentation includes forward-looking statements regarding our business, financial guidance and the therapeutic and commercial potential of SPINRAZA® (nusinersen), TEGSEDI® (inotersen), WAYLIVRA® (volanesorsen) and Ionis' technologies and products in development. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including those related to the impact COVID-19 could have on our business, and including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2020 and our most recent Form 10-Q quarterly filing, which are on file with the SEC. Copies of these and other documents are available at www.ionispharma.com.
In this presentation, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to Ionis Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics® is a registered trademark of Akcea Therapeutics, Inc. TEGSEDI® is a trademark of Akcea Therapeutics, Inc. WAYLIVRA® is a registered trademark of Akcea Therapeutics, Inc. SPINRAZA® is a registered trademark of Biogen.
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Introduction
Brett Monia, Ph.D. Chief Executive Officer
2021: Executing on our Key Strategic Objectives
Goals
Commercialize products of our own
Expand our drug discovery capabilities
12 or more marketed products in
2026
1H 2021 Progress
Completed integration of Akcea
Completed transition of Tegsedi and Waylivra to Sobi
Building capabilities to support multiple Ionis launches
Licensed transferrin receptor targeting technology from Bicycle
Advanced Genuity collaboration to identify novel targets
Broadened internal technology initiatives
Tofersen VALOR study placebo-controlled portion completed
Eplontersen NEURO-TTRansform study fully enrolled
Pelacarsen Lp(a) HORIZON study 50% enrolled
ION363 Phase 3 study for FUS-ALS initiated
IONIS-PKK-LRx positive Phase 2 study reported
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Ionis Pharmaceuticals Inc. published this content on 04 August 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 04 August 2021 16:15:04 UTC.
Ionis Pharmaceuticals, Inc. is engaged in ribonucleic acid (RNA)-targeted therapeutics. The Company has five marketed medicines and a pipeline in neurology, cardiology, and other areas of high patient need. It has nine medicines in Phase III development and multiple additional medicines in early and mid-stage development. The Companyâs products include SPINRAZA, QALSODY, WAINUA, TEGSEDI and WAYLIVRA. SPINRAZA is used for the treatment of patients with spinal muscular atrophy (SMA), a progressive, debilitating and often fatal genetic disease. QALSODY is an antisense medicine that is used for the treatment of adult patients with superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS), a rare, neurodegenerative disorder that causes progressive loss of motor neurons leading to death. WAINUA is a self-administered subcutaneous LIgand-Conjugated Antisense (LICA) medicine used for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN).
IONIS PHARMACEUTICALS, INC. 
