Akcea Initiates Phase 2 Study of AKCEA-ANGPTL3-LRx in Patients with Hypertriglyceridemia, Type 2 Diabetes Mellitus and Nonalc...
December 14 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 announced the initiation of a
Phase 2 program of AKCEA-ANGPTL3-LRx in patients with
hypertriglyceridemia, type 2 diabetes mellitus and nonalcoholic
fatty liver disease (NAFLD). The primary goal of the study is to
determine the dose level and frequency for use of AKCEA-ANGPTL3-LRx
in future registration studies.
AKCEA-ANGPTL3-LRx is designed to target and reduce the
production of angiopoietin-like 3, or ANGPTL3, a protein produced
and secreted by the liver. The congenital absence of ANGPTL3 is
associated with lower lipid levels and reduced risk of insulin
resistance, diabetes mellitus and cardiovascular disease relative
to individuals with normal production of ANGPTL3 protein.
“To date, we have seen that ANGPTL3 modulates multiple lipid
parameters that are implicated in a variety of significant
metabolic disorders like NAFLD and diabetes, where currently
physicians are only able to manage symptoms, placing a major burden
on individuals and the healthcare system,” said Louis O’Dea,
chief medical officer at Akcea Therapeutics. “By conducting
Phase 2 studies in patients with these multifactorial diseases with
AKCEA-ANGPTL3-LRx we hope to gain critical insights into the role
of ANGPTL3 in these diseases.”
The multicenter, randomized, double-blind, placebo-controlled,
dose-ranging Phase 2b study will evaluate the safety and efficacy
of AKCEA-ANGPTL3-LRx administered at different doses and
dosing intervals to approximately 144 patients with
hypertriglyceridemia, type 2 diabetes mellitus and NAFLD. Akcea
plans to report top-line data from this study in 2019. For further
study information, please visit www.clinicaltrials.gov and
search for AKCEA-ANGPTL3-LRx.
ABOUT AKCEA-ANGPTL3-LRx
AKCEA-ANGPTL3-LRx is a ligand conjugated antisense (LICA) drug
designed to reduce angiopoietin-like 3 protein, or ANGPTL3. People
with congenital absence of ANGPTL3 have lower LDL-C and
triglyceride levels and lower risk of cardiovascular disease. Akcea
and Ionis are developing AKCEA-ANGPTL3-LRx to treat patients
with multiple lipid disorders, including rare hyperlipidemias and
NAFLD with metabolic complications.
In a Phase 1/2 clinical study in volunteers with elevated
triglycerides, published in The New England Journal of Medicine,
treatment with multiple doses of AKCEA-ANGPTL3-LRx resulted in
dose-dependent reductions in ANGPTL3 protein greater than 85% after
six weeks of treatment. Treatment with AKCEA-ANGPTL3-LRx also
resulted in substantial and dose-dependent reductions in
triglycerides, LDL cholesterol, VLDL cholesterol, non-HDL
cholesterol, apolipoprotein B and apolipoprotein C-III protein.
AKCEA-ANGPTL3-LRx was well tolerated in the study. No serious
adverse events, no platelet count reductions and no injection site
reactions were reported. Further, there were no discontinuations
during the treatment period. LICA technology can provide greater
patient convenience by allowing for significantly lower doses and
less frequent administration, compared to non-LICA drugs. Phase 1
studies of all three of Akcea’s LICA drugs have shown that doses up
to 30 fold lower than non-LICA drugs result in consistent target
reductions and a favorable safety and tolerability profile.
ABOUT AKCEA THERAPEUTICSAkcea Therapeutics, an
affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), 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. The most
advanced drug in its pipeline, volanesorsen, is under regulatory
review in the U.S., EU and Canada for the treatment of familial
chylomicronemia syndrome, or FCS, and is currently in Phase 3
clinical development for the treatment of familial partial
lipodystrophy, or FPL. 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, AKCEA-ANGPTL3-LRx 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 is on file
with the SEC.
Media and Investor Contact: Kathleen
Gallagher
Head of Communications and Investor Relations, Akcea
Therapeutics (617)-207-8509 kgallagher@akceatx.com
Ionis Pharmaceuticals (NASDAQ:IONS)
Historical Stock Chart
From Mar 2024 to Apr 2024
Ionis Pharmaceuticals (NASDAQ:IONS)
Historical Stock Chart
From Apr 2023 to Apr 2024