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
- ‘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)
Saturday, April 28, 2018 from 9:45 to 10:00 am
PDT
- ‘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 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.
ABOUT VOLANESORSEN AND FCS
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 Akcea
Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc.
(NASDAQ:IONS), is a biopharmaceutical company focused on developing
and commercializing drugs to treat patients with serious and rare
diseases. Akcea is advancing a mature pipeline of six novel drugs,
including inotersen, volanesorsen, AKCEA-APO(a)-LRx,
AKCEA-ANGPTL3-LRx, AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, all with
the potential to treat multiple diseases. All six drugs were
discovered by and are being co-developed with Ionis, a leader in
antisense therapeutics, and are based on Ionis’ proprietary
antisense technology. Inotersen is under regulatory review in the
U.S. and EU for the treatment of hereditary transthyretin
amyloidosis (hATTR). Volanesorsen is under regulatory review in the
U.S., EU and Canada for the treatment of familial chylomicronemia
syndrome, or FCS, and is currently in Phase 3 clinical development
for the treatment of familial partial lipodystrophy, or FPL. Akcea
is building the infrastructure to commercialize its drugs globally.
Akcea is a global company headquartered 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. 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 December 31, 2017, 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 Akcea Therapeutics, Inc.
Investor and Media Contact Kathleen Gallagher
Head of Communications, Akcea Therapeutics
E: kgallagher@akceatx.com T: +1 617 207 8509
Ionis Pharmaceuticals (NASDAQ:IONS)
Historical Stock Chart
From Apr 2024 to May 2024
Ionis Pharmaceuticals (NASDAQ:IONS)
Historical Stock Chart
From May 2023 to May 2024