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Akcea Therapeutics Recognizes Rare Disease Day by Spotlighting Several Historic "Firsts" for People Living with Familial Chylomicronemia Syndrome


Upcoming global FCS Summit will enable advocates to connect and build a stronger FCS community

CAMBRIDGE, Mass., Feb. 28, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc., focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders, today announced the company's support for Rare Disease Day 2018, including many first-time efforts to connect and support the global community for individuals and families affected by familial chylomicronemia syndrome (FCS).

"As with many individuals who are diagnosed with a rare disease, people living with FCS and their caregivers often express feelings of isolation. Most have never met another person with FCS," said Paula Soteropoulos, president and chief executive officer of Akcea. "We are proud to support efforts to help more people living with FCS build the connections they need for information, support and advocacy. There has been a lot of progress over the past several years, but there is still much more work ahead. With a stronger and more connected FCS community, we can raise more awareness of the burden of this disease, help patients receive a diagnosis faster, and work to improve the lives of more patients in need around the world."

Established in 2008 by EURORDIS, Rare Disease Day takes place on the last day of February each year, with the goal to raise awareness about rare diseases and their impact on patients and their caregivers.

Among the "firsts" for the FCS community in 2017 or planned for 2018:

  • Akcea will sponsor the FCS Global Connection Summit, a first-ever global advocacy meeting taking place in March 2018 in The Netherlands. The program will support efforts to connect, share learnings and build a stronger FCS advocacy community.
  • "Connection and Hope," a new video sponsored by Akcea featuring an FCS patient named Aaron and his community of caregivers, will be distributed to global audiences. Aaron's story helps to demonstrate the value of connecting with others with similar journeys when diagnosed with a rare disease. To view this video, click here.
  • Akcea sponsored a meeting in Canada for the FCS community that included a demonstration of customized meal preparation in collaboration with The Culinary Institute.
  • A new outline of nutritional resources specifically designed for people living with FCS, developed by dieticians and other experts and supported by Akcea, is now available at on
  • A series of webinars led by registered dieticians, nutritionists and psychologists with information about healthy eating for people living with FCS was launched.
  • Results of the first study of the impact of FCS on patient quality of life - known as the IN-FOCUS Study - were presented at the National Organization for Rare Diseases (NORD) Rare Diseases and Orphan Products Breakthrough Summit in Washington, D.C.

FCS is a severe, rare disorder characterized by extremely high levels of triglycerides, daily symptoms such as abdominal pain, and the risk of recurrent, potentially fatal, acute pancreatitis. People with FCS are unable to effectively metabolize large, triglyceride-rich lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to break down triglycerides. There is no effective therapy available. Additional information on FCS is available at, through the FCS Foundation at and the LPLD Alliance at For a full list of organizations supporting the FCS community worldwide, please click here.

Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders. Akcea is advancing a mature pipeline of four novel drugs, including volanesorsen, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx and AKCEA-APOCIII-LRx, all with the potential to treat multiple diseases. All four drugs were discovered by and are being co-developed with Ionis, a leader in antisense therapeutics, and are based on Ionis' proprietary antisense technology. The most advanced drug in its pipeline, 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 with a focus on lipid specialists as the primary call point. Akcea is located in Cambridge, Massachusetts. Additional information about Akcea is available at

This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. and the therapeutic and commercial potential of volanesorsen and other products in development. 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(TM) is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics(TM) is a trademark of Ionis Pharmaceuticals, Inc.

Investor and Media Contact
Kathleen Gallagher
Head of Communications, Akcea Therapeutics
T: +1 617 207 8509

This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Akcea Therapeutics, Inc. via Globenewswire

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