CAMBRIDGE, Mass., Nov. 15, 2016 /PRNewswire/ -- Akcea
Therapeutics, a wholly-owned subsidiary of Ionis Pharmaceuticals,
Inc. (NASDAQ: IONIS), today announced positive results from an
interim analysis of a Phase 1/2a study of
IONIS-ANGPTL3-LRx. In this study, subjects with
elevated triglycerides achieved substantial and statistically
significant mean reductions in angiopoietin-like 3 (ANGPTL3),
triglycerides and LDL-cholesterol of up to 83 percent, 66 percent,
and 35 percent, respectively. The study was presented today
at the American Heart Association Scientific Sessions by
Sotirios Tsimikas, M.D., professor
of medicine and director of vascular medicine at the University of California, San Diego, and vice
president of clinical development at Ionis Pharmaceuticals.
"These data provide solid support for the therapeutic potential
of ANGPTL3 to reduce multiple lipid risk factors in patients with
cardiovascular disease and to affect liver fat accumulation in
patients with NASH," said Dr. Tsimikas. "Pre-clinical studies
in animal models of atherosclerosis and obesity demonstrate that
lowering ANGPTL3 levels with antisense oligonucleotides
significantly reduces progression of atherosclerosis and liver fat
accumulation. The potential of ANGPTL3 reduction to improve
cardiovascular risk is also supported by genetic studies in
humans with both heterozygous and homozygous loss-of-function
mutations in their ANGPTL3 gene showing that individuals with lower
or absent plasma levels of ANGPTL3 protein exhibit low plasma
levels of triglycerides and LDL cholesterol, which are both risk
factors for cardiovascular disease."
Subjects who received multiple doses of 10 mg, 20 mg, 40 mg, or
60 mg of IONIS-ANGPTL3-LRx achieved dose-dependent,
statistically significant mean reductions at Day 37 in ANGPTL3 of
up to 83 percent (p ≤0.001). These subjects also experienced
statistically significant mean reductions in triglycerides of up to
66 percent (p ≤0.001), in LDL-C of up to 35 percent (p ≤0.001), and
in total cholesterol of up to 36 percent (p ≤0.001). In this
study, IONIS-ANGPTL3-LRx displayed a favorable safety
and tolerability profile. There were no discontinuations due
to adverse events and no clinically meaningful platelet
declines.
"These study results support the potential to develop
IONIS-ANGPTL3-LRx to treat multiple diseases, such as
severe dyslipidemias and NASH," said Paula
Soteropoulos, president and chief executive officer of Akcea
Therapeutics. "With its focus on cardio-metabolic disorders,
Akcea is uniquely positioned to advance the therapeutic and
commercial potential of IONIS-ANGPTL3-LRx."
IONIS-ANGPTL3-LRx is a LIgand Conjugated
Antisense (LICA) drug designed to reduce the production of the
ANGPTL3 protein, a key regulator of plasma lipids, such as
triglycerides and LDL-cholesterol. Ionis' LICA technology is
designed to enhance the potency of its drugs by increasing
efficient drug uptake in target tissues, which provides the
potential for improved therapeutic margin, improved tolerability
and dosing schedule flexibility.
"In this study, IONIS-ANGPTL3-LRx, demonstrated,
consistent with the data we have observed with our other LICA
drugs, the ability to robustly lower its intended target at much
lower doses than previously used to achieve similar target
reduction with our non-LICA drugs," said Richard Geary, Ph.D., senior vice president of
development at Ionis Pharmaceuticals. "These data further
confirm that the profile conferred by our LICA technology
demonstrate the potential of these advanced antisense drugs to
address broader patient populations in cardiovascular and in
non-cardiovascular indications because of the very low volume,
infrequent and well-tolerated subcutaneous dosing these drugs can
provide."
ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics is focused on developing and commercializing
drugs for patients with serious cardiometabolic diseases caused by
lipid disorders. Established as a wholly owned subsidiary of
Ionis Pharmaceuticals, Inc., Akcea has a robust portfolio of
development-stage drugs covering multiple targets and disease
states. The drugs in Akcea's pipeline are designed using
Ionis' advanced RNA-targeted antisense technology to address a
number of lipid risk factors, including, ApoC-III, triglycerides,
Lp(a) and LDL-cholesterol. Akcea's most advanced program,
volanesorsen, is in Phase 3 development to treat patients with
either familial chylomicronemia syndrome (FCS) or familial partial
lipodystrophy (FPL), two orphan lipid disorders that are
characterized by extremely high triglycerides and ApoC-III.
Akcea is located in Cambridge,
Massachusetts. Additional information about Akcea is
available at http://akceatx.com.
ABOUT IONIS PHARMACEUTICALS, INC.
Ionis is the leading company in RNA-targeted drug discovery and
development focused on developing drugs for patients who have the
highest unmet medical needs, such as those patients with severe and
rare diseases. Using its proprietary antisense technology,
Ionis has created a large pipeline of first-in-class or
best-in-class drugs, with over a dozen drugs in mid- to late-stage
development. Drugs currently in Phase 3 development include
volanesorsen, a drug Ionis is developing and plans to commercialize
through its wholly owned subsidiary, Akcea Therapeutics, to treat
patients with either familial chylomicronemia syndrome or familial
partial lipodystrophy; IONIS-TTRRx, a drug Ionis is
developing with GSK to treat patients with TTR amyloidosis; and
SPINRAZA (nusinersen), a drug Ionis is developing with Biogen to
treat infants and children with spinal muscular atrophy.
Ionis' patents provide strong and extensive protection for its
drugs and technology. Additional information about Ionis is
available at www.ionispharma.com.
IONIS' FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding
the development, activity, therapeutic and commercial potential and
safety of IONIS-ANGPTL3-LRx. Any statement
describing Ionis' 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. Ionis' 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 Ionis' forward-looking statements reflect the good faith
judgment of its management, these statements are based only on
facts and factors currently known by Ionis. As a result, you
are cautioned not to rely on these forward-looking
statements. These and other risks concerning Ionis' programs
are described in additional detail in Ionis' annual report on Form
10-K for the year ended December 31,
2015, 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.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our," and "us" refers to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals,
Inc. Akcea Therapeutics™ is a trademark of Ionis
Pharmaceuticals, Inc. SPINRAZA™ is a trademark of Biogen.
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SOURCE Ionis Pharmaceuticals, Inc.