Akcea and Ambry Genetics to Launch hATTR Compass, a Genetic Testing Program for People with Suspected Hereditary ATTR Amyloidosis

CAMBRIDGE, Mass., June 12, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. AKCA, an affiliate of Ionis Pharmaceuticals, Inc., and Ambry Genetics Corporation ("Ambry"), a Konica Minolta company, today announced the launch of hATTR Compass™, a no-cost, confidential genetic testing and genetic counseling program for people with suspected hereditary ATTR (hATTR) amyloidosis.

Akcea and Ambry created the hATTR Compass program to empower people with accurate genetic information so they can make informed decisions about their healthcare. Through hATTR Compass, Ambry's genetic testing will allow people to learn if they carry any of the TTR gene mutations associated with hATTR amyloidosis. Confidential genetic counseling will be provided through partnership with PWNHealth, a virtual care company that enables confidential access to diagnostic testing, treatment and professional guidance. Genetic testing results will only be provided to people and their healthcare providers to ensure the protection of confidential patient information.

"Akcea is proud to bring this empowering service to people who may suspect that they have hATTR amyloidosis and their families at no-cost. We have developed a program that quickly and conveniently provides those people and their healthcare professionals with thorough genetic results to help with diagnosis and treatment decisions," said Sarah Boyce, President at Akcea Therapeutics. "Under this program, people will receive rapid genetic test results and supportive, confidential, genetic counseling to help interpret genetic test results and what those results mean for themselves and their family."

hATTR Compass screens for up to 80 genes that cause hereditary polyneuropathies and up to 85 genes associated with hereditary cardiomyopathies, including hATTR amyloidosis. To be eligible for hATTR Compass, people must be in the US and Canada, 18 years and older, and are either experiencing red flag symptoms of hATTR amyloidosis or are aware of a family history of hATTR amyloidosis, including polyneuropathies and cardiomyopathies. hATTR Compass will provide both participants and healthcare professionals with precise and reliable results through Ambry's trusted and comprehensive genetic testing.

Currently the road to diagnosis can be long and challenging for people suffering from hATTR amyloidosis. It can take upwards of five years and visits to multiple physicians across many specialties, before people finally receive an accurate diagnosis. The goal of the hATTR Compass program is to help accelerate or confirm the diagnosis of hATTR amyloidosis while providing support and resources to people and healthcare professionals throughout the genetic testing process.

"We are very excited to partner with Akcea to bring our comprehensive genetic testing to a patient population desperately in need of finding answers," stated Brigette Tippin Davis, Ph.D., FACMG, VP, Research & Development at Ambry Genetics Corporation. "It is a privilege to leverage our advanced genomic profiling technology to assist in accelerating the diagnosis of individuals with hereditary ATTR amyloidosis so that they can work with their healthcare professionals toward earlier diagnosis to better manage their disease."

"It is critical that hATTR amyloidosis patients are diagnosed early in the course of their disease. Access to genetic testing combined with well supported counselling services is vital for symptomatic patients to get an accurate diagnosis, and is an important first step toward getting the care they need," said Isabelle Lousada, President and CEO of the Amyloidosis Research Consortium.

Akcea has also partnered with Backpack Health to provide an additional resource for people and their families. As part of hATTR Compass, Backpack Health, a mobile and web-based app that helps people keep track of their symptoms, procedures, diagnostic tests and ongoing treatment for hATTR amyloidosis, will be available at no-cost for people. The tool also makes it easy for individuals and families navigating the hATTR amyloidosis journey to share important medical information with healthcare professionals.

"Our secure, cloud-based platform helps people with hATTR amyloidosis manage health details, care and communication between caregivers and often with multiple healthcare providers," said Jim Cavan, founder, president and CEO of Backpack Health.  "Now, people will have ready access to diagnostic test results and other personalized health summaries, which is essential to those who need to manage and share that information with others."

To learn more about hATTR Compass and the testing process, please visit www.hattrcompass.com.

ABOUT HEREDITARY TRANSTHYRETIN (hATTR) AMYLOIDOSIS

hATTR amyloidosis is a rare, progressive, systemic and fatal hereditary disease caused by the abnormal formation and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including peripheral nerves, heart, intestinal tract, eyes, kidneys, central nervous system, thyroid and bone marrow.^1-6 The progressive accumulation of TTR amyloid deposits in these tissues and organs leads to sensory, motor and autonomic dysfunction often having debilitating effects on multiple aspects of a patient's life. People with hATTR amyloidosis often present with a mixed phenotype and experience overlapping symptoms of polyneuropathy and cardiomyopathy.

Ultimately, hATTR amyloidosis results in death within three to fifteen years of symptom onset. Therapeutic options for the treatment of people with hATTR amyloidosis are limited, and there are currently no disease-modifying drugs approved for the disease. There are an estimated 50,000 people with hATTR amyloidosis worldwide.⁴ Additional information on hATTR amyloidosis, including a full list of organizations supporting the hATTR amyloidosis community worldwide, is available at www.hattrchangethecourse.com and www.hattrrguide.com. 

ABOUT AKCEA THERAPEUTICS

Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc. IONS, is a biopharmaceutical company focused on developing and commercializing drugs to treat people with serious and rare diseases. Akcea is advancing a mature pipeline of six novel drugs, including TEGSEDI™ (inotersen), WAYLIVRA™ (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.

ABOUT AMBRY GENETICS CORPORATION
Ambry is both College of American Pathologists (CAP)-accredited and Clinical Laboratory Improvement Amendments (CLIA)-certified. Ambry is an established leader in clinical genetic diagnostics and genetics software solutions, combining both to offer the most comprehensive testing menu in the industry. Ambry has established a reputation for sharing data while safeguarding patient privacy, unparalleled service, and responsibly applying new technologies to the clinical molecular diagnostics market. For more information about Ambry, visit https://www.ambrygen.com/.

ABOUT BACKPACK HEALTH

Backpack Health, LLC builds secure-cloud-based innovative information tools to help people manage their health journey – wellness and chronic health conditions. The Backpack Health mission is to make it easy for everyone to access, own and control all their health information to support better health for themselves, their loved ones and their communities. Backpack Health provides a platform for organizations to engage patients, collect up-to-date data and build communities around the globe. Learn more at www.backpackhealth.com.

AKCEA'S 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 its quarterly report on Form 10-Q and other documents, which are 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™, TEGSEDI™, WAYLIVRA™ and hATTR Compass™ 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

Ambry PR Contact:
Dina Scaglione
Director, Marketing
(945) 457-4355
dscaglione@ambrygen.com

References:

1. Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis. 2013;8:31.
2. Gertz MA. Hereditary ATTR amyloidosis: burden of illness and diagnostic challenges. Am J Manag Care. 2017;23(suppl 7):S107-S112.
3. Coelho T, Maurer MS, Suhr OB. THAOS—The Transthyretin Amyloidosis Outcomes Survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis. Curr Med Res Opin. 2013;29(1):63-76.
4. Hawkins PN, Ando Y, Dispenzeri A, Gonzalez-Duarte A, Adams D, Suhr OB. Evolving landscape in the management of transthyretin amyloidosis. Ann Med. 2015;47(8):625-638.
5. Coelho T, Ericzon B-G, Falk R, et al. A guide to transthyretin amyloidosis. Amyloidosis Foundation. http://www.amyloidosis.org/wp-content/uploads/2017/05/2017-ATTR-guide.pdf. Accessed February 14, 2018.
6. Johnson SM, Connelly S, Fearns C, Powers ET, Kelly JW. The transthyretin amyloidosis: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory agency approved drug. J Mol Biol. 2012;421(2-3):185-203.