CARLSBAD, Calif. and
CAMBRIDGE, Mass., July 22, 2015 /PRNewswire/ -- Isis
Pharmaceuticals, Inc. (NASDAQ: ISIS), the leader in RNA-targeted
therapeutics, and Akcea Therapeutics announced today that The
Lancet has published clinical data evaluating
ISIS-APO(a)Rx in healthy volunteers with elevated
lipoprotein(a) or Lp(a). An accompanying editorial was also
published. ISIS-APO(a)Rx is a Generation 2.0+
antisense drug that is part of Isis' lipid franchise, which is
being developed and commercialized by Akcea Therapeutics, Isis'
wholly owned subsidiary.
"Although not as widely recognized as LDL-cholesterol, a growing
body of scientific and medical evidence has identified Lp(a) as an
important and independent, genetically caused risk factor for
cardiovascular disease," said Joseph Witztum, M.D.,
distinguished professor of medicine and director of the
atherosclerosis research group at the University of
California, San Diego and
co-author on the paper. "There is no drug available today to
specifically and effectively lower elevated Lp(a) levels in
patients with high Lp(a) levels. The publication of these study
results today in The Lancet shows the therapeutic potential
of antisense drugs, such as ISIS-APO(a)Rx, to
specifically affect important, newly identified therapeutic targets
like Lp(a)."
"The introduction of a selective Lp(a)-lowering therapy is very
exciting," said Erik Stroes, M.D.,
Ph.D., professor of internal medicine, department of vascular
medicine, University of Amsterdam.
"It is estimated that up to 20% of the population have elevated
Lp(a) levels that put them at higher risk for cardiovascular
disease but because there has been no therapeutic option for
lowering elevated Lp(a) levels, most healthcare providers have not
been monitoring this important risk factor. However, the
availability of a therapeutic agent capable of lowering Lp(a)
should lead to the widespread testing of Lp(a) levels."
"Several seminal publications over the past five years,
including human genetic studies, have established Lp(a) as an
important risk factor for cardiovascular disease. It is a testament
to the efficiency of antisense technology that we were able to
create ISIS-APO(a)Rx and advance it into human clinical
trials so rapidly. We are looking forward to the results from our
Phase 2 clinical trials in patients with very high Lp(a) later this
year," said Rosanne M. Crooke, vice
president of cardiovascular research at Isis Pharmaceuticals.
"ISIS-APO(a)Rx is a Generation 2.0+ antisense drug that
specifically and potently reduces Lp(a) without affecting
other lipoproteins."
"ISIS-APO(a)Rx, as well as its next generation LICA
version, ISIS-APO(a)-LRx, are the first drugs in
development to directly target Lp(a), and among the various drugs
in the Akcea pipeline to treat patients with serious
cardiometabolic lipid disorders," said Paula Soteropoulos, president and chief
executive officer of Akcea Therapeutics. "Akcea is uniquely
positioned to drive development and rapidly advance these drugs to
market for these patients who are underserved with today's
therapies."
The paper titled "Antisense therapy targeting apolipoprotein(a):
a randomized, double-blind, placebo-controlled phase 1 study"
(Tsimikas et al, The Lancet 2015: published online today),
reported data from the Phase 1 study evaluating single and multiple
ascending doses of ISIS-APO(a)Rx in healthy volunteers
with elevated Lp(a) concentrations of 25 nmol/L (100 mg/L) or more.
Results of this study demonstrated potent, dose-dependent,
selective reductions of plasma Lp(a) up to 89% (mean reduction up
to 78%) in patients treated with ISIS-APO(a)Rx. In
addition, up to 90 percent reduction (mean reduction up to 84%) was
observed in Lp(a) associated oxidized phospholipids, which play an
important role in proinflammatory and proatherogenic processes. The
Lp(a) knockdown, together with safety and tolerability support
continued clinical development of ISIS-APO(a)Rx as a
potential therapeutic drug to reduce the risk of cardiovascular
disease and calcific aortic valve stenosis in patients with
elevated Lp(a) concentration. Isis and Akcea are currently
evaluating ISIS-APO(a)Rx in a Phase 2 study in
patients with elevated Lp(a) levels and plan to report data from
this study around the year end. Isis and Akcea are also
developing ISIS-APO(a)-LRx.
ABOUT Lp(a)
Lp(a) is considered an independent risk factor for
cardiovascular disease due to its association with an increased
risk of coronary heart disease, atherosclerotic plaque formation
and calcific aortic valve stenosis. Lp(a) is a lipoprotein
particle that is assembled in the liver and consists of the
apolipoprotein(a) protein covalently linked to
LDL-cholesterol. Lp(a) levels in blood can vary greatly
between individuals due primarily to genetic variations in the gene
that encodes for apolipoprotein(a). As a result, Lp(a) levels
are genetically determined and remain constant throughout the life
of the individual. Diet and lifestyle changes have little
impact on Lp(a) levels and current therapies are not able to
adequately reduce elevated levels of Lp(a) to acceptable levels in
patients who have severely elevated Lp(a). As a general
guideline for ideal Lp(a) levels, the European Atherosclerosis
Society recommends that Lp(a) levels be less than or equal to 50
mg/dL.
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-Cholesterol, 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
ISIS-APO(a)Rx, 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.
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SOURCE Isis Pharmaceuticals, Inc.