Akcea and Ionis to Present Phase 2 Clinical Data of Vupanorsen (AKCEA-ANGPTL3-L (Rx) ) at ESC Congress 2020
Vupanorsen was developed using Ionis' proprietary LIgand Conjugated Antisense (LICA) technology platform to reduce the production of angiopoietin-like 3 (ANGPTL3) protein, a key regulator of triglyceride and cholesterol metabolism, in the liver. The Phase 2 dose-ranging clinical trial evaluated the safety and efficacy of vupanorsen in patients with hypertriglyceridemia, type 2 diabetes, and non-alcoholic fatty liver disease (NAFLD).
In November 2019, Akcea and Ionis announced the closing of the worldwide exclusive licensing agreement with Pfizer Inc. for vupanorsen (AKCEA-ANGPTL3-LRx). Under terms of the agreement, Akcea and Ionis received a $250 million upfront license fee, which was split equally between the two companies. Pfizer is responsible for all development and regulatory activities and costs beyond those associated with this Phase 2 study.
Following are details about the late-breaking virtual presentation at
- Late-Breaking Science in Lipids: ANGPTL3 Antisense Oligonucleotide to Lower Triglycerides by
Daniel Gaudet, M.D., professor of medicine, Department of Medicine, University of Montreal
Saturday, August 29, 2020, 3:00 a.m. ET/ 9:00 a.m. CEST
ABOUT VUPANORSEN (AKCEA-ANGPTL3-LRx)
AKCEA-ANGPTL3-LRx (vupanorsen) is an investigational antisense therapy being developed to treat patients with certain cardiovascular diseases. This antisense medicine is designed to reduce the production of angiopoietin-like 3 (ANGPTL3) protein, a key regulator of triglyceride and cholesterol metabolism, in the liver. AKCEA-ANGPTL3-LRx was developed using Ionis' advanced LIgand Conjugated Antisense (LICA) technology platform. The potential therapeutic benefits of ANGPTL3 reduction are supported by the discovery that people with a genetic deficiency in ANGPTL3 have reduced levels of low-density lipoprotein cholesterol (LDL-C) and triglycerides, and a decreased risk of cardiovascular disease.1 In a previous Phase 1 study, patients treated with AKCEA-ANGPTL3-LRx achieved robust, dose-dependent reductions in ANGPTL3, triglycerides, LDL-C, non-HDL-C and total cholesterol with a positive safety and tolerability profile.2 AKCEA-ANGPTL3-LRx was discovered by Ionis and has been co-developed by Akcea and Ionis.
ABOUT AKCEA THERAPEUTICS
ABOUT IONIS PHARMACEUTICALS, INC.
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to potentially treat a broad range of disease, including neurological, cardio-renal, metabolic, infectious, and pulmonary diseases. To learn more about Ionis visit www.ionispharma.com and follow us on Twitter @ionispharma.
AKCEA AND IONIS FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. and Ionis Pharmaceuticals, Inc. and the therapeutic and commercial potential of AKCEA-ANGPTL3-LRx. Any statement describing Akcea's or Ionis' goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of AKCEA-ANGPTL3-LRx or other of Akcea's or Ionis' drugs in development is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Akcea's and 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 Akcea's and Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea and Ionis. In particular, we caution you that our forward-looking statements are subject to the ongoing and developing circumstances related to the COVID-19 pandemic, which may have a material adverse effect on our business, operations and future financial results. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Akcea's and Ionis' programs are described in additional detail in Akcea's and Ionis' quarterly reports on Form 10-Q and annual reports on Form 10-K, which are on file with the SEC. Copies of these and other documents are available from each company.
In this press release, unless the context requires otherwise, "Ionis," "Akcea," "Company," "Companies," "we," "our," and "us" refers to Ionis Pharmaceuticals and/or Akcea Therapeutics.
1. JAMA Cardiol. 2018
2. N Engl J Med. 2017 Jul 20;377(3):222-232.
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Akcea Investor Contact, Matt Roache, Director, Investor Relations, (617)-841-9535, firstname.lastname@example.org; Akcea Media Contact, Angelyn Lowe VP, Corporate Communications & Investor Relations, 442-287-0470, email@example.com; Ionis Investor Contact, D. Wade Walke, Ph.D., Vice President, Investor Relations, 760-603-2741, firstname.lastname@example.org; Ionis Media Contact, Roslyn Patterson, Vice President, Corporate Communications, 760-603-2681, email@example.com