Akcea Therapeutics Announces Presentations on Volanesorsen and FCS at the National Lipid Association Scientific Sessions
Results to be presented show significant reduction in triglyceride levels associated with reductions of pancreatitis and burden of disease in patients with FCS

CAMBRIDGE, Mass. and LAS VEGASApril 26, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc., focused on developing and commercializing drugs to treat people with serious and rare diseases, today announced that researchers will present clinical data on volanesorsen and Familial Chylomicronemia Syndrome (FCS) at the National Lipid Association (NLA) Scientific Sessions taking place in Las Vegas, NV from April 26-29.

Volanesorsen is an investigational drug designed to prevent build-up of chylomicrons and other triglyceride-rich lipoproteins and has been studied for use as an adjunct therapy to diet for the treatment of adult patients with FCS. Results from the phase 3 APPROACH trial show that volanesorsen achieved a statistically significant mean reduction in triglycerides of 77% from baseline and decreased the risk of associated pancreatitis in patients with FCS. The most common adverse events in the APPROACH study were injection site reactions and platelet declines. The FDA Prescription Drug User Fee Act, or PDUFA, goal date for volanesorsen is August 30, 2018. If approved, volanesorsen would be the first and only therapy indicated for people with FCS.

FCS is a potentially life-threatening disease with significant disease burden caused by impaired function of the enzyme lipoprotein lipase (LPL) and resulting build-up of chylomicrons, which are large, triglyceride-rich particles in the plasma. FCS is associated with the risk of unpredictable acute pancreatitis, which is potentially fatal and can lead to chronic complications due to permanent organ damage, in addition to symptoms that severely impact the ability to work and participate in daily activities.  

Following are the four studies to be presented in poster presentations in the Exhibit Hall at the NLA meeting:

Friday, April 27, 2018 from 10:40 to 10:55 am PDT

  • ‘Treatment with Volanesorsen (VLN) Reduced Triglycerides and Pancreatitis in Patients with Familial Chylomicronemia Syndrome (FCS) and Severe Hypertriglyceridemia (sHTG) vs Placebo: Results of the APPROACH and COMPASS Studies’ an encore poster presentation by Karren Williams, Ph.D., Akcea Therapeutics (Monitor 11)
  • ‘Examining the High Disease Burden and Impact on Quality of Life in Familial Chylomicronemia Syndrome’ an encore poster presentation by Andrew Hsieh, PharmD, Akcea Therapeutics (Monitor 9)
  • ‘Evaluating the Impact of Peer Support and Connection on the Quality of Life of Patients with Familial Chylomicronemia Syndrome’ by Valerie Salvatore, BS, Akcea Therapeutics (Monitor 14)

Saturday, April 28, 2018 from 9:45 to 10:00 am PDT

  • ‘Assessing the Disease Burden Among Patients with Familial Chylomicronemia Syndrome (FCS) on Volanesorsen: Results of the ReFOCUS Study’ poster presentation by Andrew Hsieh, PharmD, Akcea Therapeutics (Monitor 6). 

Akcea is also hosting an Expert Theatre panel discussion at the meeting entitled “Hypertriglyceridemia-Induced Pancreatitis:  How Lipidologists Can Partner to Optimize Complex Care.” Vikesh Singh, M.D., MSc, director of the Pancreatitis Center and associate professor of medicine at Johns Hopkins University, and David J. Davidson, M.D., a clinical lipidologist at NorthShore Medical Group in Bannockburn, IL, will participate in the panel and discuss their clinical experiences with FCS and management of patient care. The panel will take place on Saturday, April 28 from 11:55 am to 12:55 pm PDT in the Valencia room at the meeting.

Additional results from the ReFOCUS study will be presented at the European Atherosclerosis Society (EAS) 2018 congress in Lisbon next month.


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, and through the FCS Foundation at and the LPLD Alliance at 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.

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

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.

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