Akcea Announces Completion of Landmark Study to Assess Disease Burden in People Living with FCS

Twenty-two people with FCS participated in the Re-FOCUS survey. They were asked to assess the number and severity of a range of symptoms and daily life measures affected by FCS. The web-based global study was conducted among people with FCS treated with volanesorsen, Akcea's investigational therapy currently under regulatory review in the US, EU and Canada.

"FCS is an ultra-rare disease that has not been widely studied. This landmark study represents an important step in our efforts to identify and quantify the many ways that FCS can affect the physical, functional and emotional health of people living with FCS," said Michael W. Stevenson, R.Ph., Ph.D., global vice president, medical affairs at Akcea. "The Re-FOCUS study provides helpful, new information as we work to design and validate a tool that can accurately evaluate changes in disease burden with therapeutic intervention." 

The Re-FOCUS study represents another milestone in Akcea's ongoing efforts to understand the impact of FCS. In October 2017, Akcea announced results of the IN-FOCUS study, a survey of 166 people with FCS in 10 countries. The data provided new insight on the disease burden, including cognitive, emotional, and physical impairments that can affect daily life as well as the risk of recurrent, potentially fatal, acute pancreatitis.

"For people living with FCS, it impacts every part of daily life, often making it difficult to work, care for other family members or even join in life events with family and friends. Studies like IN-FOCUS and Re-FOCUS can enable patients and caregivers around the world to better understand and communicate about the devastating effects of this rare disease," said Lindsey Sutton, co-president of the FCS Foundation.

"We are working to increase understanding of FCS, through our clinical programs, scientific communications, and by bringing forward the voices of those who live with this disease," said Molly Harper, vice president, and global franchise head, cardiometabolics. "We would like to thank the FCS Foundation as well as all of the patients, caregivers and clinicians who participated in or supported this groundbreaking work, which will help build awareness and improve quality of care for patients with FCS and potentially other rare diseases."

ABOUT FCS
FCS is an ultra-rare 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. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognitions that affect their ability to work. People with FCS also 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.

ABOUT AKCEA THERAPEUTICS, INC.
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 TEGSEDI^TM (inotersen), WAYLIVRA^TM (volanesorsen), AKCEA-APO(a)-L_Rx, AKCEA-ANGPTL3-L_Rx, AKCEA-APOCIII-L_Rx, and AKCEA-TTR-L_Rx, 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. TEGSEDI is under regulatory review in the U.S., EU and Canada for the treatment of people with hereditary transthyretin amyloidosis (hATTR). WAYLIVRA 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 people with 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 WAYLIVRA^TM (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), TEGSEDI^TM and WAYLIVRA^TM are trademarks of Akcea Therapeutics, Inc.

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