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Akcea Therapeutics Canada celebrates official launch of first-of-its-kind research grant program

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Young Investigator Grant Program Call for Proposals issued to Canadian researchers

OTTAWA, March 13, 2020 /CNW/ - Akcea Therapeutics Canada, a subsidiary of Akcea Therapeutics Inc. (NASDAQ:AKCA), congratulates Hereditary Amyloidosis Canada (HAC) and Regroupement québécois des maladies orphelines (RQMO) on issuing their official Call for Proposals for a new initiative designed to build capacity and foster further research in the field of transthyretin (TTR) amyloidosis in Canada. Akcea Therapeutics, Inc. is a majority-owned affiliate of Ionis Pharmaceuticals, Inc.

Funded by a three-year grant from Akcea Therapeutics Canada, the Unfolding Transthyretin Amyloidosis: Akcea Canada Young Investigator Grant Program is the first Canadian research grant program for the disease. It was created in partnership by HAC and RQMO to stimulate early career researchers and clinician scientists to study this complex, debilitating and life-limiting rare disease, and to drive knowledge translation with the aim of improving patient experiences and overall quality of life.

Transthyretin amyloidosis is a rare, progressive, genetic disease that occurs when there is a build-up of abnormal (misfolded) transthyretin proteins in the body's tissues and/or organs. Most commonly it affects the peripheral nerves, heart, intestinal tract, eyes, kidneys, central nervous system, thyroid and bone marrow. The progressive accumulation of 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.

"We are very proud to support this important initiative and help ensure deep and, importantly, sustained investigation into this rare disease in Canada," said Jared Rhines, Senior Vice President & Head, Americas, Akcea Therapeutics Inc., and General Manager, Akcea Therapeutics Canada. "At Akcea, we envision a future in which people with rare diseases can lead full and happy lives despite the inherent challenges of their conditions. We believe this opportunity to support young researchers will help bring that vision to life."

"As we recognize Amyloidosis Awareness Month this month, we're thrilled to see support for focused Canadian research into this disease which affects families in profound ways," said Anne Marie Carr, Founder and Executive Director of HAC. "We look forward to receiving and reviewing the proposals for novel research, ideally in areas ranging from biomedical to health services to population health, with our esteemed Scientific Advisory Committee which will lead the peer review process."

"We very much appreciate Akcea's support with this exciting initiative," said Gail Ouellette, a geneticist, genetic counsellor, and President and Executive Director of Regroupement québécois des maladies orphelines (RQMO). "This grant program will continue to build on the momentum we have in Canada by stimulating research into improving the patient experience and health outcomes in TTR amyloidosis."

Complete details about the program, including criteria and application instructions can be found at https://madhattr.ca/updates-developments, and by visiting www.madhattr.ca or www.rqmo.org. The deadline for applications is Friday, May 29, 2020 at 5:00 pm EST.

About Akcea Therapeutics Canada
Akcea Therapeutics Canada, based in Ottawa Ontario, is the Canadian subsidiary of Akcea Therapeutics, Inc. Akcea is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases. Akcea Therapeutics Canada is a member of Innovative Medicines Canada, the industry association representing Canada's research-based pharmaceutical companies.

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. 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, 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. 

SOURCE Akcea Therapeutics Canada

Cision View original content to download multimedia: http://www.newswire.ca/en/releases/archive/March2020/13/c0920.html

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