YASTEST
LONDON, Oct. 10, 2017 (GLOBE
NEWSWIRE) -- Akcea Therapeutics United Kingdom (UK) today announced
that volanesorsen has been granted a Promising Innovative Medicine
(PIM) Designation by the Medicines and Healthcare products
Regulatory Agency (MHRA) for the treatment of familial
chylomicronaemia syndrome (FCS), a rare genetic lipid
disorder. Akcea Therapeutics UK, the UK subsidiary of Akcea
Therapeutics Inc. (NASDAQ:AKCA), an affiliate of Ionis
Pharmaceuticals, Inc., is focused on developing and commercialising
drugs to treat patients with serious cardiometabolic lipid
disorders.
A PIM Designation is an early indication that a
medicinal product is a promising candidate for the Early Access to
Medicines Scheme (EAMS) in the UK; intended for the treatment,
diagnosis or prevention of a life-threatening or seriously
debilitating condition, with the potential to address an unmet
medical need.
"We are delighted about the MHRA's decision to
designate volanesorsen as a PIM. This designation demonstrates the
potential to address clear unmet medical needs in the treatment of
this debilitating condition," comments Luke Robinson, general
manager, Akcea Therapeutics, UK, Ireland & Nordics.
FCS is a severe, rare disorder characterised by
extremely elevated levels of triglycerides,1 symptoms
such as abdominal pain that affect daily living,1 and the
risk of recurrent, potentially fatal, acute
pancreatitis.1 People
with FCS are unable to effectively metabolise large,
triglyceride-rich, lipid particles called chylomicrons due to a
deficiency in lipoprotein lipase,1 an
enzyme that helps to break down triglycerides.
"FCS is a significant burden on people affected by
the condition - from their day to day health, to what they can eat,
their employment, their sense of self-worth and their relationships
with their friends and family," says Dr. Louis O'Dea, chief medical
officer, Akcea Therapeutics. "FCS patients have a high unmet need
with potentially life-threatening consequences, and we seek to
bring this new treatment to these patients as expeditiously as
possible."
Patients with FCS have triglyceride levels that
can reach 20 to 30 times that of healthy individuals.2 This can
often lead to repeated episodes of acute pancreatitis, which can be
fatal.1 There is
currently no effective therapy available.3
Investor and Media
Contact
Kathleen Gallagher
Head of Communications, Akcea Therapeutics, UK, Ireland &
Nordics
E: kgallagher@akceatx.com
T: +1 617 207 8509
Notes to
Editors
About Volanesorsen in
FCS
Volanesorsen, a product of Ionis' proprietary antisense technology,
is in development for rare metabolic disorders including familial
chylomicronaemia syndrome (FCS). 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.2
FCS is a severe, rare disorder characterised by
extremely elevated levels of triglycerides, symptoms such as
abdominal pain that affect daily living, and the risk of recurrent,
potentially fatal, acute pancreatitis.1People with
FCS are unable to effectively metabolise large, triglyceride-rich
lipid particles called chylomicrons due to a deficiency in
lipoprotein lipase, an enzyme that helps to break down
triglycerides.1Patients with
FCS have triglyceride levels that can reach 20 to 30 times that of
healthy individuals,2 which
can often lead to repeated episodes of acute pancreatitis, which
can be fatal.1 There is
currently no effective therapy available.3
About Akcea Therapeutics
UK
Akcea Therapeutics UK is the UK subsidiary of Akcea Therapeutics
Inc., an affiliate of Ionis Pharmaceuticals, Inc. Akcea is a
biopharmaceutical company focused on transforming the lives of
patients with serious cardiometabolic lipid disorders. Akcea has a
robust portfolio of development-stage drugs covering multiple
targets and diseases using advanced RNA-targeted antisense
therapeutics.
Akcea is advancing a mature pipeline of four novel
drugs, including volanesorsen, AKCEA-APO(a)-LRx,
AKCEA-ANGPTL3-LRx and
AKCEA-APOCIII-LRx, all with
the potential to treat multiple diseases. All four drugs were
discovered by and are being co-developed with Ionis, a leader in
antisense therapeutics, and are based on Ionis' proprietary
antisense technology. The most advanced drug in its pipeline,
volanesorsen, is under regulatory review in the U.S., EU and Canada
for the treatment of familial chylomicronaemia syndrome, or FCS.
Akcea is building the infrastructure to commercialise its drugs
globally with a focus on lipid specialists as the primary call
point. Akcea's global headquarters is located 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 and other products in
development. 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 endeavour 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 final prospectus for its initial public
offering and its most recent quarterly report on Form 10-Q, 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(TM) is a trademark of Ionis
Pharmaceuticals, Inc. Akcea Therapeutics(TM) is a trademark of
Ionis Pharmaceuticals, Inc.
References
1. Brunzell JD. Familial lipoprotein lipase
deficiency. GeneReviews 2011.
2. Gaudet D, et al. Targeting APOC3 in the familial chylomicronemia
syndrome. New England Journal of Medicine 2014;
371:2200-2206
3. NORD. The physician's guide to lipoprotein lipase
deficiency (LPLD). Available
at: https://rarediseases.org/physician-guide/lipoprotein-lipase-deficiency-lpld Accessed
October 2017.
This
announcement is distributed by Nasdaq Corporate Solutions on behalf
of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: Akcea Therapeutics, Inc. via Globenewswire
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