BOSTON, July 6, 2015 /PRNewswire/ -- Akcea Therapeutics,
a wholly-owned subsidiary of Isis Pharmaceuticals, Inc. (NASDAQ:
ISIS), announced today that the U.S. Food and Drug Administration
has granted Orphan Drug Designation to volanesorsen
(ISIS-APOCIIIRx) for the treatment of patients with
Familial Chylomicronemia Syndrome (FCS). FCS is a rare
genetic disease characterized by extremely high triglyceride levels
and risk of pancreatitis. In a Phase 2 study published in the NEJM
in December 2014, patients with FCS
treated with volanesorsen achieved substantial reductions in
apoC-III, triglycerides, chylomicrons and apoC-III-associated very
low density lipoprotein-cholesterol (VLDL-C)
particles.1 Akcea is currently conducting an
international multi-center, randomized, double-blind,
placebo-controlled Phase 3 study in patients with FCS.
"FCS is a rare and very serious genetic disorder associated with
very high triglyceride levels that put patients at risk of
potentially life-threatening pancreatitis. People with FCS
cope with daily consequences of their disease, including persistent
abdominal pain and life-altering food restrictions. Unfortunately,
current treatment options do not reduce triglyceride levels enough
to reduce the risk of serious illness in patients with FCS," said
Paula Soteropoulos, President and
Chief Executive Officer at Akcea Therapeutics. "Orphan drug
designation for FCS underscores the need for improved therapies to
treat patients with FCS and is an important benchmark as we
complete the ongoing Phase 3 study in patients with FCS and prepare
for commercialization."
The Orphan Drug Act provides for economic incentives to
encourage the development of drugs for diseases affecting fewer
than 200,000 people in the United States. Orphan drug
designation entitles Akcea Therapeutics to seven years of market
exclusivity in the United States
if market approval is granted for volanesorsen for the treatment of
patients with FCS. Additional incentives include tax credits
related to clinical trial expenses, an exemption from the FDA-user
fee, and FDA assistance in clinical trial design.
About FCS
FCS is a rare genetic disorder
characterized by extremely high levels of triglycerides that
affects an estimated one to two out of a million people. FCS
may also be called familial chylomicronemia, Frederickson Type I
hyperlipoproteinemia, familial lipoprotein lipase deficiency
(LPLD), or familial hypertryglyceridemia. Some people with FCS may
live with recurrent stomach pain, and a high risk or prior history
of hospitalization for pancreatitis. FCS is primarily caused
by mutations in genes that produce proteins involved in the
clearance of particles that carry triglycerides, called
chylomicrons. Patients with FCS are unable to effectively
clear chylomicrons, and as such, have high levels of triglycerides,
which increase their risk of pancreatitis, type 2 diabetes and
other serious illnesses.
About Volanesorsen
Volanesorsen
(ISIS-APOCIIIRx) is an antisense drug in development
intended to treat patients with severely high triglycerides either
as a single agent or in combination with other
triglyceride-lowering agents. Volanesorsen is designed to
target apoC-III, a protein produced in the liver that plays a
central role in the regulation of serum
triglycerides.2 Humans who do not produce apoC-III
have lower levels of triglycerides and lower instances of
cardiovascular disease.3 Humans with elevated levels of
apoC-III have high triglycerides associated with multiple metabolic
abnormalities, such as insulin resistance and/or metabolic
syndrome.4 In addition, the prevalence of type 2
diabetes is increased in patients with elevated
triglycerides.5 Humans with severely elevated
levels of triglycerides are at risk of many serious health
conditions, including pancreatitis,4 which can be
life-threatening and require hospitalization. Volanesorsen is
currently being evaluated in a Phase 3 study in patients with
FCS. A second Phase 3 study of volanesorsen is planned to
begin later this year in patients with Familial Partial
Lipodystrophy, another severe and rare lipid disorder. For
more information about this clinical trial program, please visit
www.apociii.com
ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics is a
development and commercialization company focused on transforming
the lives of patients with serious cardiometabolic lipid disorders.
Established as a wholly owned subsidiary of Isis Pharmaceuticals,
Inc, Akcea has a robust portfolio of development-stage drugs
covering multiple targets and disease states using advanced
RNA-targeted antisense therapeutics. Akcea's drug pipeline
includes novel antisense drugs designed to address a number of
lipid risk factors, including LDL-C, ApoC-III, triglycerides and
Lp(a). Akcea's most advanced program, volanesorsen, is in
Phase 3 development to treat patients with ultra-orphan lipid
disorders that are characterized by extremely high triglycerides
and ApoC-III, including familial chylomicronemia syndrome and
familial partial lipodystrophy. Akcea is located in
Cambridge, Massachusetts.
Additional information about Akcea is available at
www.akceatx.com.
ABOUT ISIS PHARMACEUTICALS, INC.
Isis is exploiting
its leadership position in RNA-targeted technology to discover and
develop novel drugs for its product pipeline and for its
partners. Isis' broad pipeline consists of 38 drugs to treat
a wide variety of diseases with an emphasis on cardiovascular,
metabolic, severe and rare diseases, including neurological
disorders, and cancer. Isis' partner, Genzyme, is
commercializing Isis' lead product, KYNAMRO®, in
the United States and other
countries for the treatment of patients with homozygous FH.
Isis has numerous drugs in Phase 3 development in severe/rare
diseases and cardiovascular diseases. These include
volanesorsen, a drug Isis is developing and plans to commercialize
through its wholly owned subsidiary, Akcea Therapeutics, to treat
patients with familial chylomicronemia syndrome and familial
partial lipodystrophy; ISIS-TTRRx, a drug Isis is
developing with GSK to treat patients with the polyneuropathy and
cardiomyopathy forms of TTR amyloidosis; and ISIS-SMNRx,
a drug Isis is developing with Biogen to treat infants and children
with spinal muscular atrophy, a severe and rare neuromuscular
disease. Isis' patents provide strong and extensive
protection for its drugs and technology. Additional
information about Isis is available at www.isispharm.com.
FORWARD-LOOKING STATEMENT
This press release includes
forward-looking statements regarding the business of Akcea
Therapeutics, Inc., a subsidiary of Isis Pharmaceuticals and the
therapeutic and commercial potential of Akcea's technologies and
products in development, including 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
Isis. 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 Akcea's
parent company, Isis Pharmaceuticals, Inc.,'s annual report on Form
10-K for the year ended December 31,
2014, and its most recent quarterly report on Form 10-Q,
which are on file with the SEC. Copies of these and other
documents are available at www.isispharm.com.
In this press release, unless the context requires otherwise,
"Akcea," "Company," "we," "our," and "us" refers to Akcea
Therapeutics.
Isis Pharmaceuticals® is a registered trademark of Isis
Pharmaceuticals, Inc. Akcea Therapeutics™ is a trademark of
Isis Pharmaceuticals, Inc. KYNAMRO® is a registered trademark
of Genzyme Corporation.
References:
- Gaudet, D. et al. (2014). Targeting APOC3 in the familial
chylomicronemia syndrome. N Engl J Med, 374(23),
2200-2206.
- Zheng, C. (2014). Updates on apolipoprotein CIII: fulfilling
promise as a therapeutic target for hypertriglyceridemia and
cardiovascular disease. Curr Opin Lipidol, 25(1),
35-39.
- Jorgensen, A.B., Frikke-Schmidt, R., Nordestgaard, B.G. &
Tybaerg-Hansen, A. (2014) Loss-of-function mutations in APOC3 and
risk of ischemic vascular disease. N Engl J Med, 371(1),
32-41
- Christian, J.B., Arondekar, B., Buysman, E.K., Jacobson, T.A.,
Snipes, R.G., Horwitz, R. (2014). Determining triglyceride
reductions needed for clinical impact in severe
hypertriglyceridemia. Am J Med, 127(1), 36-44.
- Mooradian, A.D. (2009). Dyslipidemia in type 2 diabetes
mellitus. Nat Clin Pract Endocrinol Metab, 5(3),
150-159.
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