Akcea to Present Volanesorsen Data at NLA Sessions

Over the course of the past year, Rare Disease Report has covered the journey of Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, and the journey of its investigational therapy volanesorsen.

The drug, intended for the treatment of individuals with familial chylomicronemia syndrome (FCS), has been specifically developed 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 patients in this indication.

In March 2017, the company announced that its pivotal Phase 3 APPROACH study met its primary endpoints. In August 2017, a New Drug Application (NDA) was submitted for the drug to the U.S. Food and Drug Administration (FDA). In November 2017, marketing applications were accepted for the potential treatment option in the United States, Canada and Europe.

At the National Lipid Association (NLA) Scientific Sessions in Las Vegas, NV this weekend, the company will present clinical data on volanesorsen and FCS.

The Phase 3 APPROACH study, a randomized, double-blind, placebo-controlled, 52-week trial, enrolled 66 patients with FCS, and administered volanesorsen to half of them (n=33) and placebo to those remaining (n=33). At the study’s initiation, patients had an average triglyceride level of 2,209 mg/dL; after only 3 months of treatment, a reduction in triglyceride levels was achieved, as the volanesorsen-treated group exhibited a 77% reduction versus the placebo-treated group who reported an 18% increase in triglyceride level (P < .0001).

Additionally, the company reported that the reduction in triglycerides was sustained for a year and the instance of pancreatitis, a common symptom experienced by patients with FCS, was also decreased.

On Friday, Akcea will present 3 studies via poster presentations in the Exhibit Hall at the NLA Sessions:

• ‘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)

On Saturday, the company will present a fourth poster:

• ‘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).

Also on Saturday, from 11:55 am to 12:55 pm in the Valencia room, Akcea will be hosting an Expert Theatre panel discussion 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 company has announced its intentions to present results from the ReFOCUS study at the European Atherosclerosis Society (EAS) 2018 congress in Lisbon next month.

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