CARLSBAD, Calif. and
CAMBRIDGE, Mass., July 29, 2015 /PRNewswire/ -- Isis
Pharmaceuticals, Inc. (NASDAQ: ISIS), the leader in RNA-targeted
therapeutics, and Akcea Therapeutics, its wholly owned subsidiary,
announced today that The New England Journal of Medicine
(NEJM) has published positive clinical results from
a Phase 2 clinical study evaluating volanesorsen (formerly
ISIS-APOCIIIRx) in patients with very high to severely
high triglycerides. This publication follows the December 2014 publication in the NEJM of the
positive Phase 2 results from a clinical study of volanesorsen in
patients with familial chylomicronemia syndrome (FCS).1
Volanesorsen is part of Isis' lipid franchise, which is
being developed and commercialized by Akcea Therapeutics.
"These publications emphasize the significant interest from the
medical community in the therapeutic potential of antisense drugs,
such as volanesorsen, to substantially affect novel cardiometabolic
targets, like apoC-III," said Sekar
Kathiresan, director of preventive cardiology at
Massachusetts General Hospital and researcher in medical and
population genetics at the Broad Institute of Massachusetts Institute of Technology and Harvard
University. "These and other findings from the medical and
scientific communities are confirming the importance of apoC-III
and triglycerides as risk factors for cardiovascular and metabolic
disease. The recent results with volanesorsen indicate that an
RNA-targeted antisense approach is proving to be an ideal way to
target these risk factors."
"We have consistently demonstrated significant reductions in
apoC-III and triglycerides in patients treated with
volanesoren. We have also reported significant improvements
in glucose parameters in patients with high triglycerides and type
2 diabetes treated with volanesorsen. These data highlight the
potential therapeutic value that volanesorsen can bring to patients
with serious cardiometabolic lipid disorders. Akcea is
building a world class team focused on the development and
commercialization of drugs to treat rare lipid disorders.
Preparations for global commercialization of volanesorsen are
underway," said Paula Soteropoulos,
president and chief executive officer of Akcea Therapeutics.
"Volanesorsen is the most advanced drug in Akcea's portfolio of
lipid drugs. Together with ISIS-APOCIII-LRx, our more
potent LICA follow on drug for patients with severely high
triglycerides, and the other drugs in our pipeline, we have the
opportunity to provide complementary therapeutic solutions to
physicians treating patients with lipid disorders."
"We continue to see growing interest in the therapeutic
potential of our antisense drugs to specifically affect important,
genetically validated targets, like apoC-III and Lp(a), that are
often not approachable with other therapeutic modalities," said
Richard Geary, Ph.D., senior vice
president of development at Isis Pharmaceuticals. "The
publication of four manuscripts in the last few months in either
The New England Journal of Medicine (3
papers) or The Lancet (one paper), represents
substantial recognition from the medical community of our
technology and its broad applicability to address newly identified
targets."
The paper, titled "Antisense Inhibition of APOC3 in Patients
with Hypertriglyceridemia" (Gaudet et al, N Engl J Med 2015:
published online July 29), reported
data from the Phase 2 study of volanesorsen, a double-blind,
randomized, placebo-controlled 13-week study designed to assess the
safety and activity of volanesorsen in patients as a monotherapy
and as an add-on to fibrates. Patients treated with volanesorsen
achieved mean reductions of up to 80 percent in apoC-III and up to
71 percent in triglycerides and average increases of up to 46
percent in HDL-C. The efficacy, safety and tolerability of
volanesorsen in patients with very high to severely high
triglycerides to date support continued development.
ABOUT VOLANESORSEN, FCS and FPL
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 plasma
triglycerides.2 Humans who do not produce apoC-III
have lower levels of plasma triglycerides and a lower prevalence of
cardiovascular disease.3 Humans with elevated levels of
apoC-III have high triglycerides associated with multiple metabolic
abnormalities, including insulin resistance and/or metabolic
syndrome.4 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. FCS is a rare genetic disorder
characterized by extremely high levels of triglycerides and a
history or risk of pancreatitis. Patients with FCS are unable
to effectively clear chylomicrons, and as such, have high levels of
triglycerides, which increase their risk of pancreatitis, which can
be life-threatening, type 2 diabetes and other serious
illnesses.
A second Phase 3 study of volanesorsen is planned to begin later
this year in patients with familial partial lipodystrophy (FPL).
FPL is a rare lipid disorder characterized by both selective loss
of fat from various areas of the body and a range of metabolic
abnormalities, including elevated apoC-III and triglycerides, as
well as diabetes. Patients with FPL are unable to store fat
or triglycerides in normal fat stores so excess triglycerides are
stored in the liver and muscle and accumulate at high levels in the
bloodstream. Patients with FPL are at increased risk of
pancreatitis, hepatic steatosis and NASH, enlarged livers, elevated
liver enzymes and premature cardiovascular disease.
For more information about this clinical trial program for
volanesorsen, please visit www.apociii.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.
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 (FCS) and familial partial lipodystrophy
(FPL). Akcea is located in Cambridge, Massachusetts. Additional
information about Akcea is available at www.akceatx.com.
ISIS PHARMACEUTICALS' FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding
Isis Pharmaceuticals and Isis' business and the commercial
potential of Isis' technology and lipid franchise drugs, including
the development, activity, therapeutic potential and safety of
volanesorsen, and the business of Akcea Therapeutics and the
commercial potential of drugs and technologies Akcea
develops. Any statement describing Isis' 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. Isis' 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 Isis' 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 Isis' programs
are described in additional detail in Isis' 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 from the Company.
References:
1. Gaudet, D. et al. (2014). Targeting APOC3 in the
familial chylomicronemia syndrome. N Engl J Med, 374(23),
2200-2206.
2. 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.
3. Jørgensen, A.B., Frikke-Schmidt, R., Nordestgaard, B.G.
& Tybjærg-Hansen, A. (2014) Loss-of-function mutations in APOC3
and risk of ischemic vascular disease. N Engl J Med, 371(1),
32-41
4. 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.
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SOURCE Isis Pharmaceuticals, Inc.