Akcea Presents Data Demonstrating Significant Burden of Illness in Familial Chylomicronemia Syndrome
October 16 2017 - 7:00AM
Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis
Pharmaceuticals, Inc., focused on developing and commercializing
drugs to treat patients with serious cardiometabolic diseases
caused by lipid disorders, today reported that new data from the
global IN-FOCUS study were presented at the National Organization
for Rare Diseases (NORD) Rare Diseases and Orphan Products
Breakthrough Summit in Washington, D.C.
The full data set is derived from 166 respondents with familial
chylomicronemia syndrome (FCS) in 10 countries in North America,
Europe, South America and South Africa. The data demonstrate the
disease burden patients with FCS experience, including cognitive,
emotional, and physical impairments that affect daily life. In
addition to the day-to-day complications of the disease, patients
with FCS are at risk of recurrent, potentially fatal, acute
pancreatitis. Findings from IN-FOCUS show these
manifestations diminish quality of life, can impact career choices
and affect the ability to secure and maintain employment.
The full data set from the IN-FOCUS study were presented at the
NORD summit in a poster titled: “The Investigation of Findings and
Observations Captured in Burden of Illness Survey in Patients with
Familial Chylomicronemia Syndrome – Final Results from the IN-FOCUS
Study.” The data confirmed an earlier interim analysis of the
IN-FOCUS study published in the Expert Review of Cardiovascular
Therapy. There was no discernible regional difference in
patient–reported disease characteristics.
Dr. Michael Davidson, director, Preventive Cardiology,
Department of Medicine, University of Chicago, said: “These data
illuminate the range and depth of impact that patients with FCS
experience on a daily basis. Despite considerable efforts in trying
to manage their symptoms through strict diet, patients live in
ongoing fear of hospitalization from acute pancreatitis. Due to
chronicity and severity of symptoms, these patients are
under-employed, and isolated from family and friends. These data
underscore a significant unmet need in FCS.”
“The IN-FOCUS study is the first comprehensive assessment of the
patient experience of living with FCS and increases our
understanding of the disease. This study gives us and the medical
community important insights into aspects of the disease from a
patient perspective that have been under-recognized and not
previously characterized in literature,” said Dr. Louis O’Dea,
chief medical officer of Akcea.
“The results of the IN-FOCUS study represent a true achievement
in giving patients a voice to articulate the symptoms, burdens and
outcomes of living with FCS. We are encouraged further by the
recent increase in disease awareness. We are hopeful that
these collective efforts will lead to faster diagnosis, better
understanding, and eventually, treatment options for patients with
FCS,” said Lindsey Sutton, co-president of the FCS Foundation.
To view data from the interim analysis of the IN-FOCUS study,
please click here.
ABOUT FCSFCS is a severe, rare disorder
characterized by extremely high levels of triglycerides, daily
symptoms such as abdominal pain, and the risk of recurrent,
potentially fatal, acute pancreatitis. People with FCS are unable
to effectively metabolize large, triglyceride-rich lipid particles
called chylomicrons due to a deficiency in lipoprotein lipase, an
enzyme that helps to break down triglycerides. There is no
effective therapy 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.
ABOUT AKCEA THERAPEUTICSAkcea Therapeutics, an
affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical
company focused on developing and commercializing drugs to treat
patients with serious cardiometabolic diseases caused by lipid
disorders. 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. Akcea is
building the infrastructure to commercialize its drugs globally
with a focus on lipid specialists as the primary call point. Akcea
is located in Cambridge, Massachusetts. Additional information
about Akcea is available at www.akceatx.com.
FORWARD-LOOKING STATEMENTThis 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 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 final prospectus for its initial public offering and its most
recent quarterly report on Form 10-Q, which are on file with the
SEC.
In this press release, unless the context requires otherwise,
“Akcea,” “Company,” “we,” “our,” and “us” refers to Akcea
Therapeutics.
Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc. Ionis Pharmaceuticals™ is a trademark of Ionis
Pharmaceuticals, Inc.
Media and Investor Contact:Kathleen
Gallagher
Head of Communications, Akcea
Therapeutics(617)-207-8509kgallagher@akceatx.com
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