Volanesorsen is an investigational drug designed to prevent build-up of chylomicrons and other triglyceride-rich lipoproteins and has been studied for use as an adjunct therapy to diet for the treatment of adult patients with FCS. Results from the phase 3 APPROACH trial show that volanesorsen achieved a statistically significant mean reduction in triglycerides of 77% from baseline and decreased the risk of associated pancreatitis in patients with FCS. The most common adverse events in the APPROACH study were injection site reactions and platelet declines. The FDA Prescription Drug User Fee Act, or PDUFA, goal date for volanesorsen is
FCS is a potentially life-threatening disease with significant disease burden caused by impaired function of the enzyme lipoprotein lipase (LPL) and resulting build-up of chylomicrons, which are large, triglyceride-rich particles in the plasma. FCS is associated with the risk of unpredictable acute pancreatitis, which is potentially fatal and can lead to chronic complications due to permanent organ damage, in addition to symptoms that severely impact the ability to work and participate in daily activities.
Following are the four studies to be presented in poster presentations in the Exhibit Hall at the NLA meeting:
- ‘Treatment with Volanesorsen (VLN) Reduced Triglycerides and Pancreatitis in Patients with Familial Chylomicronemia Syndrome (FCS) and Severe Hypertriglyceridemia (sHTG) vs Placebo: Results of the APPROACH and COMPASS Studies’ an encore poster presentation by
Karren Williams, Ph.D., Akcea Therapeutics(Monitor 11)
- ‘Examining the High Disease Burden and Impact on Quality of Life in Familial Chylomicronemia Syndrome’ an encore poster presentation by
Andrew Hsieh, PharmD, Akcea Therapeutics(Monitor 9)
- ‘Evaluating the Impact of Peer Support and Connection on the Quality of Life of Patients with Familial Chylomicronemia Syndrome’ by
Valerie Salvatore, BS, Akcea Therapeutics(Monitor 14)
- ‘Assessing the Disease Burden Among Patients with Familial Chylomicronemia Syndrome (FCS) on Volanesorsen: Results of the ReFOCUS Study’ poster presentation by
Andrew Hsieh, PharmD, Akcea Therapeutics(Monitor 6).
Akcea is also hosting an
Additional results from the ReFOCUS study will be presented at the
ABOUT VOLANESORSEN AND FCS
Volanesorsen, a product of Ionis’ proprietary antisense technology, is under regulatory review in the U.S., EU and
FCS is an under-recognized 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 and can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognitions that affect their ability to work and also often 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
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. It is also currently in Phase 3 clinical development for the treatment of patients with familial partial lipodystrophy, or FPL. Akcea anticipates reporting top-line data from this study in 2019.
ABOUT AKCEA THERAPEUTICS
This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. and the therapeutic and commercial potential of volanesorsen. 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 annual report on Form 10-K for the year ended
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