Akcea Therapeutics : Supports Rare Disease Day 2020 and Global Efforts to 'Reframe Rare'

BOSTON - Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today announced the company's support for Rare Disease Day 2020 and the effort to help more people learn about the impact of rare diseases on patients, their families and their caregivers.

Throughout February, Akcea has highlighted important issues related to hereditary transthyretin (hATTR) amyloidosis, familial chylomicronemia syndrome (FCS) and other rare diseases on social media. In addition, the company will host a special event for employees with hATTR amyloidosis and FCS patients and caregivers at Akcea's offices, providing the opportunity to discuss challenges in managing these diseases and the ways that companies like Akcea can help build awareness and better support rare disease communities.

'There are more than 300 million people living with a rare disease worldwide, and yet oftentimes there is limited understanding of the wide-ranging impact that these diseases can have on people and families, caregivers and friends. This Rare Disease Day, we are focused on reframing what it means to live with and to be affected by a rare disease, including unique challenges like lack of access to diagnostic tools, treatment, medical care and support services,' said Damien McDevitt, Ph.D., interim chief executive officer at Akcea. 'We are so grateful to members of the hATTR amyloidosis and FCS communities for meeting with us and sharing their insights so that we can continue to identify opportunities to provide more effective support in the years ahead.'

Rare Disease Day was established by EURORDIS in 2008 and takes place on the last day of February each year with the goal to raise awareness of many different rare diseases worldwide.

'Living with hATTR amyloidosis with polyneuropathy has had a significant impact on my physical, emotional and social health and quality of life, as well as placed a burden on my wife and family,' said Gerry Harper, a hATTR amyloidosis patient. 'It is a devastating disease, and what's more, after I was diagnosed my wife and I had our children genetically tested and they also received a positive diagnosis of hATTR amyloidosis. We are grateful to companies like Akcea highlighting the daily burden that affected families experience and their efforts to support patients, including bringing an approved treatment option, that gives us hope for the future.'

ABOUT HEREDITARY TRANSTHYRETIN (hATTR) AMYLOIDOSIS

hATTR amyloidosis is a severe, progressive, and life-threatening disease caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, the heart and intestinal tract. The progressive accumulation of TTR amyloid deposits in these organs often leads to intractable peripheral sensorimotor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations. hATTR amyloidosis causes significant morbidity and progressive decline in quality of life, severely impacting activities of daily living. The disease often progresses rapidly and can lead to premature death. The median survival is 4.7 years following diagnosis.

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 or 10mmol/L) and a risk of unpredictable and potentially fatal acute pancreatitis. Because of limited LPL production or 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, including chronic pancreatitis and pancreatogenic (type 3c) diabetes. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognition that affect their ability to work. People with FCS also report major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog.

ABOUT AKCEA THERAPEUTICS

Akcea Therapeutics, Inc., a majority-owned 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 commercializing TEGSEDI (inotersen) and WAYLIVRA (volanesorsen), as well as advancing a mature pipeline of novel drugs, including AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx, AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, with the potential to treat multiple diseases. All six drugs were discovered by Ionis, a leader in antisense therapeutics, and are based on Ionis' proprietary antisense technology. TEGSEDI is approved in the U.S., E.U., Canada and Brazil. WAYLIVRA is approved in the E.U. 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 Boston, Massachusetts.

FORWARD-LOOKING STATEMENT

This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. Any statement describing Akcea's goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of Akcea's 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 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 Akcea's 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 the 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.

Ionis Pharmaceuticals is a trademark of Ionis Pharmaceuticals, Inc., Akcea Therapeutics, TEGSEDI and WAYLIVRA are trademarks of Akcea Therapeutics, Inc.

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