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Akcea Therapeutics Announces Presentations on Volanesorsen and FCS at the National Lipid Association Scientific SessionsCAMBRIDGE, Mass. and LAS VEGAS, April 26, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc., focused on developing and commercializing drugs to treat people with serious and rare diseases, today announced that researchers will present clinical data on volanesorsen and Familial Chylomicronemia Syndrome (FCS) at the National Lipid Association (NLA) Scientific Sessions taking place in Las Vegas, NV from April 26-29. 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 August 30, 2018. If approved, volanesorsen would be the first and only therapy indicated for people with FCS. 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: Friday, April 27, 2018 from 10:40 to 10:55 am PDT
Saturday, April 28, 2018 from 9:45 to 10:00 am PDT
Akcea is also hosting an Expert Theatre panel discussion at the meeting entitled “Hypertriglyceridemia-Induced Pancreatitis: How Lipidologists Can Partner to Optimize Complex Care.” Vikesh Singh, M.D., MSc, director of the Pancreatitis Center and associate professor of medicine at Johns Hopkins University, and David J. Davidson, M.D., a clinical lipidologist at NorthShore Medical Group in Bannockburn, IL, will participate in the panel and discuss their clinical experiences with FCS and management of patient care. The panel will take place on Saturday, April 28 from 11:55 am to 12:55 pm PDT in the Valencia room at the meeting. Additional results from the ReFOCUS study will be presented at the European Atherosclerosis Society (EAS) 2018 congress in Lisbon next month. Volanesorsen, a product of Ionis’ proprietary antisense technology, is under regulatory review in the U.S., EU and Canada as a treatment for familial chylomicronemia syndrome (FCS). The U.S. and EU regulatory agencies have granted Orphan Drug Designation to volanesorsen for the treatment of FCS. If approved, volanesorsen would be the first and only therapy indicated for people with FCS. 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 FCS Foundation at http://www.livingwithfcs.org and the LPLD Alliance at www.lpldalliance.org. For a full list of organizations supporting the FCS community worldwide, please click here. 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 FORWARD-LOOKING STATEMENT 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 Akcea Therapeutics, Inc. Investor and Media Contact |