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Press Release

Akcea Announces Expansion of Global Early Access Program to Provide Volanesorsen to People Living with FCS


Posted on: 02 May 18

Program currently available in the U.S. and select countries in Europe; initiating additional countries throughout 2018

CAMBRIDGE, Mass., May 02, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA) today announced it has expanded its global early access program (EAP) to the United States for people with familial chylomicronemia syndrome (FCS). Volanesorsen is under regulatory review in the United States (US), European Union (EU) and Canada for the treatment of FCS. Akcea, an affiliate of Ionis Pharmaceuticals, Inc., is focused on developing and commercializing drugs to treat patients with serious and rare diseases.

Early access, also called “compassionate use,” is the availability of an investigational medicinal product outside of a clinical trial that is intended to treat a serious or life-threatening condition.

“Our work is driven by a steadfast commitment to the FCS community, and we are pleased to make volanesorsen available through our global early access program to people who may benefit while our regulatory applications are being reviewed,” said Paula Soteropoulos, chief executive officer of Akcea Therapeutics. “FCS is a serious condition with a variety of symptoms, including potentially fatal acute pancreatitis, that can severely impact quality-of-life. As there are no currently approved treatments for FCS, we are working closely with lipid specialists to responsibly enable patient access.”

The EAP program is currently available in the U.S. and certain countries in Europe. It continues to be initiated on a country-by-country basis globally.

Physicians who are interested in learning more about the early access program for their patients should contact volanesorsenEAP@akceatx.com.

ABOUT VOLANESORSEN AND FCS
Volanesorsen, a product of Ionis’ proprietary antisense technology, is under regulatory review in the U.S., EU and Canada as a treatment for familial chylomicronemia syndrome (FCS). The U.S. and EU regulatory agencies have granted Orphan Drug Designation to volanesorsen for the treatment of FCS. If approved, volanesorsen would be the first and only therapy indicated for people with FCS.

FCS is an under-recognized disease caused by impaired function of the enzyme lipoprotein lipase (LPL) and characterized by severe hypertriglyceridemia (>880mg/dL) and a risk of unpredictable and potentially fatal acute pancreatitis. Because of limited LPL function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90% triglycerides. In addition to pancreatitis, FCS patients are at risk of chronic complications due to permanent organ damage and can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognitions that affect their ability to work and also often report major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog. There is no effective therapy for FCS currently available. Additional information on FCS is available at www.fcsfocus.com, and through the FCS Foundation at http://www.livingwithfcs.org and the LPLD Alliance at www.lpldalliance.org. For a full list of organizations supporting the FCS community worldwide, please click here.

Volanesorsen is designed to reduce the production of ApoC-III, a protein produced in the liver that plays a central role in the regulation of plasma triglycerides and may also affect other metabolic parameters. It is also currently in Phase 3 clinical development for the treatment of patients with familial partial lipodystrophy, or FPL. Akcea anticipates reporting top-line data from this study in 2019.

ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases. Akcea is advancing a mature pipeline of six novel drugs, including inotersen, volanesorsen, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx, AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, all with the potential to treat multiple diseases. All six drugs were discovered by and are being co-developed with Ionis, a leader in antisense therapeutics, and are based on Ionis’ proprietary antisense technology. Inotersen is under regulatory review in the U.S. and EU for the treatment of hereditary transthyretin amyloidosis (hATTR). Volanesorsen is under regulatory review in the U.S., EU and Canada for the treatment of familial chylomicronemia syndrome, or FCS, and is currently in Phase 3 clinical development for the treatment of familial partial lipodystrophy, or FPL. Akcea is building the infrastructure to commercialize its drugs globally. Akcea is a global company headquartered in Cambridge, Massachusetts. Additional information about Akcea is available at www.akceatx.com.

FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc., and the therapeutic and commercial potential of volanesorsen. Any statement describing Akcea’s 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, 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 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 forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Akcea’s programs are described in additional detail in its annual report on Form 10-K for the year ended December 31, 2017, which is on file with the SEC.

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.

Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics™ is a trademark of Akcea Therapeutics, Inc.

Media and Investor Contact:
Kathleen Gallagher                                                                         
Head of Communications and Investor Relations, Akcea Therapeutics
(617)-207-8509
kgallagher@akceatx.com

GlobeNewswire
globenewswire.com

Last updated on: 02/05/2018

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