Press Releases

Akcea® Recognizes Rare Disease Day Through Efforts to Increase Awareness of Hereditary Transthyretin Amyloidosis and Familial Chylomicronemia Syndrome

February 28, 2019
Planned activities to highlight wide-ranging impact of rare diseases on patients and challenges in disease management, including important perspectives from caregivers

BOSTON, Feb. 28, 2019 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., focused on developing and commercializing drugs to treat patients with serious and rare diseases, today announced the company’s support for Rare Disease Day 2019 for patients living with hereditary transthyretin (hATTR) amyloidosis and familial chylomicronemia syndrome (FCS) and their caregivers, including bringing several of them together to participate in a live panel discussion related to their disease.

“For most people living with a rare disease, management of their condition is often a significant challenge and can impact many aspects of their daily lives including the ability to perform at work or school or take care of their families,” said Paula Soteropoulos, chief executive officer at Akcea. “At Akcea, we are connecting patients and caregivers with one another to share their stories and build stronger rare disease communities so that those impacted by a rare disease can better advocate for themselves and others in the years ahead.”

Established in 2008 by EURORDIS, Rare Disease Day takes place on the last day of February each year, with the goal to raise awareness of many different rare diseases and their impact on both patients and caregivers. Akcea has several activities planned globally around Rare Disease Day this year, engaging employees as well as patients, patient advocates, families and caregivers affected by hATTR amyloidosis and FCS.

“hATTR amyloidosis has placed a significant burden on my family and me both mentally and physically, especially as my symptoms have progressed, which include severe numbness in my hands and feet, GI distress, substantial weight loss, dizziness and exhaustion,” said Chuck, a patient living with hATTR amyloidosis with polyneuropathy. “Not only did this disease impact every aspect of my daily life, my wife Sharell took on new responsibilities of caring for me, which also came with their own adjustments and challenges. I am honored to be supporting Akcea’s efforts around Rare Disease Day and to continue to raise awareness of hATTR amyloidosis and provide support to other patients and their families.”

Akcea, along with members of the global hATTR amyloidosis and FCS communities, invite patients, caregivers, clinicians, friends and family to participate in Rare Disease Day on social media using hashtags #RareDiseaseDay, #ShowYourRare, #ShowYourStripes, #hATTR, #amyloidosis, #ActionFCS, #LivingWithFCS and #RareReality.

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. Additional information on hATTR amyloidosis, including a full list of organizations supporting the hATTR amyloidosis community worldwide, is available at www.hattrchangethecourse.com or by visiting www.hATTRGuide.com.

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, including chronic pancreatitis and pancreatogenic diabetes. 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

Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases. Akcea is commercializing TEGSEDI® (inotersen) and advancing a mature pipeline of novel drugs, including WAYLIVRA™ (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. TEGSEDI is approved in the U.S., E.U. and Canada. WAYLIVRA is under regulatory review 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 Boston, Massachusetts. Additional information about Akcea is available at www.akceatx.com and you can follow us on twitter at @akceatx.

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 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 other documents, 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.

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

Akcea Media Contact:
Bill Berry
Berry & Company
T: 212 253-8881
bberry@berrypr.com

Lynn Granito
Berry & Company
T: 212 253-8881
lgranito@berrypr.com

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Source: Akcea Therapeutics, Inc.