Akcea Therapeutics’ Volanesorsen Receives Promis

LONDON, Oct. 10, 2017 (GLOBE NEWSWIRE) -- Akcea Therapeutics United Kingdom (UK) today announced that volanesorsen has been granted a Promising Innovative Medicine (PIM) Designation by the Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of familial chylomicronaemia syndrome (FCS), a rare genetic lipid disorder.  Akcea Therapeutics UK, the UK subsidiary of Akcea Therapeutics Inc. (NASDAQ: AKCA ), an affiliate of Ionis Pharmaceuticals, Inc., is focused on developing and commercialising drugs to treat patients with serious cardiometabolic lipid disorders.

A PIM Designation is an early indication that a medicinal product is a promising candidate for the Early Access to Medicines Scheme (EAMS) in the UK; intended for the treatment, diagnosis or prevention of a life-threatening or seriously debilitating condition, with the potential to address an unmet medical need.

“We are delighted about the MHRA’s decision to designate volanesorsen as a PIM. This designation demonstrates the potential to address clear unmet medical needs in the treatment of this debilitating condition,” comments Luke Robinson, general manager, Akcea Therapeutics, UK, Ireland & Nordics.

FCS is a severe, rare disorder characterised by extremely elevated levels of triglycerides, ¹ symptoms such as abdominal pain that affect daily living, ¹ and the risk of recurrent, potentially fatal, acute pancreatitis. ¹ People with FCS are unable to effectively metabolise large, triglyceride-rich, lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, ¹ an enzyme that helps to break down triglycerides.

“FCS is a significant burden on people affected by the condition – from their day to day health, to what they can eat, their employment, their sense of self-worth and their relationships with their friends and family,” says Dr. Louis O’Dea, chief medical officer, Akcea Therapeutics. “FCS patients have a high unmet need with potentially life-threatening consequences, and we seek to bring this new treatment to these patients as expeditiously as possible.”

Patients with FCS have triglyceride levels that can reach 20 to 30 times that of healthy individuals. ² This can often lead to repeated episodes of acute pancreatitis, which can be fatal. ¹ There is currently no effective therapy available. ³

Investor and Media Contact Kathleen Gallagher Head of Communications, Akcea Therapeutics, UK, Ireland & Nordics E: kgallagher@akceatx.com T: +1 617 207 8509

Notes to Editors

About Volanesorsen in FCS Volanesorsen, a product of Ionis’ proprietary antisense technology, is in development for rare metabolic disorders including familial chylomicronaemia syndrome (FCS). Volanesorsen is designed to reduce the production of ApoC-III, a protein produced in the liver that plays a central role in the regulation of plasma triglycerides and may also affect other metabolic parameters. ²

FCS is a severe, rare disorder characterised by extremely elevated levels of triglycerides, symptoms such as abdominal pain that affect daily living, and the risk of recurrent, potentially fatal, acute pancreatitis. ¹ People with FCS are unable to effectively metabolise large, triglyceride-rich lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to break down triglycerides. ¹ Patients with FCS have triglyceride levels that can reach 20 to 30 times that of healthy individuals, ² which can often lead to repeated episodes of acute pancreatitis, which can be fatal. ¹ There is currently no effective therapy available. ³

About Akcea Therapeutics UK Akcea Therapeutics UK is the UK subsidiary of Akcea Therapeutics Inc., an affiliate of Ionis Pharmaceuticals, Inc. Akcea is a biopharmaceutical company focused on transforming the lives of patients with serious cardiometabolic lipid disorders. Akcea has a robust portfolio of development-stage drugs covering multiple targets and diseases using advanced RNA-targeted antisense therapeutics.

Akcea is advancing a mature pipeline of four novel drugs, including volanesorsen, AKCEA-APO(a)-L _Rx , AKCEA-ANGPTL3-L _Rx and AKCEA-APOCIII-L _Rx , all with the potential to treat multiple diseases. All four drugs were discovered by and are being co-developed with Ionis, a leader in antisense therapeutics, and are based on Ionis’ proprietary antisense technology. The most advanced drug in its pipeline, volanesorsen, is under regulatory review in the U.S., EU and Canada for the treatment of familial chylomicronaemia syndrome, or FCS. Akcea is building the infrastructure to commercialise its drugs globally with a focus on lipid specialists as the primary call point. Akcea’s global headquarters is located 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 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 endeavour 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 final prospectus for its initial public offering and its most recent quarterly report on Form 10-Q, 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™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals, Inc.

References 1. Brunzell JD. Familial lipoprotein lipase deficiency. GeneReviews 2011. 2. Gaudet D, et al. Targeting APOC3 in the familial chylomicronemia syndrome. New England Journal of Medicine 2014; 371:2200-2206 3. NORD. The physician’s guide to lipoprotein lipase deficiency (LPLD). Available at: https://rarediseases.org/physician-guide/lipo...iency-lpld Accessed October 2017.