Akcea Announces New Pricing and Reimbursement of WAYLIVRA® (volanesorsen) in Germany

BOSTON, Aug. 13, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today announced that the German Association of Statutory Sick Funds has agreed upon new pricing and reimbursement of WAYLIVRA(®) (volanesorsen) as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate. WAYLIVRA has been commercially available in Germany since August 15, 2019. As part of reimbursement and market access protocol in Germany, Akcea was required to renegotiate the WAYLIVRA launch price 12 months after first commercialization, which will go into effect on August 15, 2020.

"We are pleased that renegotiations in Germany were swiftly completed, demonstrating that payers in the region continue to recognize the value that WAYLIVRA offers to FCS patients. While WAYLIVRA has been commercially available to FCS patients in the region for some time, achieving this new, 2-year mutually agreed upon pricing and reimbursement is a significant milestone for the global FCS community of patients and caregivers, who are currently underserved," said Michael Pollock, SVP, head of Europe at Akcea. "We continue to build on our momentum in Europe and are on track to launch WAYLIVRA in additional European countries with the goal of bringing this much needed therapy to all eligible patients living with FCS."

"FCS patients in Germany as well as their families have been living with the significant burden of this disease for years, including physical, social, emotional and financial effects, and I am pleased to be able to offer appropriate patients a treatment option supported by extensive clinical data including results from the Phase 3 APPROACH study," said Professor Ioanna Gouni-Berthold from University of Cologne.

FCS is a debilitating genetic disease caused by impaired function of the enzyme lipoprotein lipase (LPL), which results in elevated triglyceride levels and significant risk and disease burden, including unpredictable and potentially fatal acute pancreatitis as well as chronic complications due to permanent organ damage. It is estimated that there are between 3,000 to 5,000 people living with FCS worldwide. WAYLIVRA is designed to reduce the production of apolipoprotein C-III (apoC-III), a protein produced in the liver that plays a central role in the regulation of plasma triglycerides.

About WAYLIVRA(®) (volanesorsen)
With conditional marketing authorization from the European Commission (EC) as an adjunct to diet in adult patients with genetically confirmed FCS and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate, WAYLIVRA is now the only therapy indicated for people with familial chylomicronemia syndrome (FCS). Akcea is working to confirm a path forward for WAYLIVRA in the U.S. and Canada.

WAYLIVRA, a product of Ionis' proprietary antisense technology, is designed to reduce the production of apoC-III, a protein that regulates plasma triglycerides and may also affect other metabolic parameters.

The European Commission's marketing authorization of WAYLIVRA is based on results from the Phase 3 APPROACH study and the ongoing APPROACH Open Label Extension (OLE) study and is supported by results from the Phase 3 COMPASS study. Results from the Phase 3 APPROACH trial, the largest study ever conducted in patients with FCS, show that in comparison to placebo, treatment with WAYLIVRA reduced triglycerides 77% (-94% when compared to placebo). All patients in the trial maintained a low-fat diet.

WAYLIVRA is associated with risk of thrombocytopenia. Enhanced monitoring is required to support early detection and management of thrombocytopenia. The most frequently observed adverse reactions (more than 10%) during treatment with WAYLIVRA were events associated with injection site reactions, immunogenicity and reduction in platelet levels.

The WAYLIVRA Early Access Program (EAP) has been initiated in Europe, the U.S. and Canada and is currently enrolling eligible patients. Click here for more information on the WAYLIVRA EAP. For more information on WAYLIVRA, please visit www.WAYLIVRA.eu.

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. Additional information on FCS is available at www.fcsfocus.com, through the LPLD Alliance at www.lpldalliance.org and through The FCS Foundation at http://www.livingwithfcs.org. For a full list of organizations supporting the FCS community worldwide, please click here.

Akcea Therapeutics, Inc., a majority-owned affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), is a biopharmaceutical company focused on developing and commercializing medicines 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 medicines, including AKCEA-APO(a)-L(Rx), vupanorsen (AKCEA-ANGPTL3-L(Rx)), AKCEA-APOCIII-L(Rx), and AKCEA-TTR-L(Rx), with the potential to treat multiple diseases. All six medicines 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, and WAYLIVRA is approved in the E.U. Akcea is headquartered in Boston, Massachusetts, and is building the infrastructure to commercialize its medicines globally. Additional information about Akcea is available at www.akceatx.com and you can follow us on Twitter at @akceatx.

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 TEGSEDI(®), WAYLIVRA(®) and Akcea's other medicines 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 medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. 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. In particular, we caution you that our forward-looking statements are subject to the ongoing and developing circumstances related to the COVID-19 pandemic, which may have a material adverse effect on our business, operations and future financial results. 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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SOURCE Akcea Therapeutics, Inc.