in the Discovery, Development & Delivery of Life Transforming Genetic Medicines
Forward-Looking Statements
This presentation includes forward-looking statements regarding our business, financial guidance and the therapeutic and commercial potential of SPINRAZA® (nusinersen), TEGSEDI® (inotersen), WAYLIVRA® (volanesorsen), eplontersen, olezarsen, donidalorsen, ION363, pelacarsen, bepirovirsen, Ionis' technologies, and Ionis' other 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 but not limited to those related to our commercial products and the medicines in our pipeline, and particularly 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, 2022, which is on file with the SEC. Copies of this 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 registered 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. QALSODYTM is a trademark of Biogen. SPINRAZA® is a registered trademark of Biogen.
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Agenda
Topic
Speaker
Welcome
Wade Walke, Ph.D.
Introduction
Brett Monia, Ph.D.
ATTR & Eplontersen Program Overview
Eugene Schneider, M.D.
NEURO-TTRansformWeek-35 and Week-66 Data
Sami Khella, M.D.
ATTR Cardiomyopathy & CARDIO-TTRansform
Sam Tsimikas, M.D.
Commercial Readiness
Onaiza Cadoret
Concluding Remarks
Brett Monia, Ph.D.
Q&A
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Introduction
Brett Monia, Ph.D.
Chief Executive Officer
Every Moment Matters...
in the Discovery, Development & Delivery of Life Transforming Genetic Medicines
Eplontersen: Well Positioned to Address Underserved, Global ATTRv-PN Market1,2
Met co-primary and all secondary endpoints
Favorable safety and tolerability
Next steps: regulatory & commercial
Met all co-primary and secondary endpoints at week-35 and 66
Substantial number of patients improved in measures of neuropathy progression and quality of life compared to baseline
Favorable safety and tolerability profile comparable to placebo
Safety and tolerability consistent with LICA platform
Preparing to launch in the U.S.; PDUFA December 22, 2023
Preparing for additional oUS regulatory submissions this year
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1. Timing expectations based on current assumptions and subject to change. 2. Assuming approval.
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Ionis Pharmaceuticals Inc. published this content on 25 April 2023 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 25 April 2023 17:14:36 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. 
