Akcea Therapeutics, Inc. (NASDAQ:AKCA), today announced it had
completed enrollment of a Phase 2b clinical study of
investigational drug AKCEA-APO(a)-LRx. Akcea is conducting the
study in patients with high Lp(a) and established cardiovascular
disease (CVD) to determine the dose level and frequency of
administration for a future planned Phase 3 cardiovascular outcome
study and to determine the safety and tolerability profile of
AKCEA-APO(a)-LRx. Akcea plans to report top-line data from the
Phase 2b study in the second half of 2018. Akcea is an affiliate of
Ionis Pharmaceuticals Inc. focused on developing and
commercializing drugs to treat patients with serious
cardiometabolic diseases caused by lipid disorders.
AKCEA-APO(a)-LRx is part of a strategic collaboration with
Novartis. If Novartis exercises its option to license
AKCEA-APO(a)-LRx after successful completion of the Phase 2b study,
it will be responsible for all future development activities for
AKCEA-APO(a)-LRx including a global Phase 3 cardiovascular outcome
study and, if approved, global commercialization activities. As
part of the agreement, Akcea retains the right to co-commercialize
globally.
“Elevated Lp(a) is a genetic trait that is present in
approximately 20-30% of the population and is recognized as a risk
factor for cardiovascular disease. There are currently no
treatment options available that specifically target Lp(a).
AKCEA-APO(a)-LRx is the only program in clinical development for
this indication that has been shown to substantially lower Lp(a),”
said Mustafa Noor, MD, chief development officer of Akcea
Therapeutics. “We now look forward to completing the study and
beginning analysis of the study data. The results of this study
will help us better understand the efficacy and safety profile of
AKCEA-APO(a)-LRx, so that this program can advance into a pivotal
cardiovascular outcome study to determine the potential benefit of
lowering Lp(a).”
AKCEA-APO(a)-LRx is an antisense drug that uses Ionis’ advanced
LIgand Conjugated
Antisense, or LICA technology. AKCEA-APO(a)-LRx
inhibits the production of apolipoprotein(a), or Apo(a), protein,
thereby reducing lipoprotein(a), or Lp(a). Lp(a) is made up
of apo(a) protein bound to LDL cholesterol resulting in an
atherogenic and thrombogenic lipoprotein that has been genetically
validated as an independent risk factor for coronary artery
disease, heart attack, stroke, calcific aortic valve disease and
peripheral arterial disease. 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.
The randomized, double-blind, placebo-controlled, dose-ranging
Phase 2b study is evaluating the safety and efficacy of different
doses of AKCEA-APO(a)-LRx. The study enrolled over 270
patients with high Lp(a) and established cardiovascular
disease. For further study information, please visit
www.clinicaltrials.gov and search for AKCEA-APO(a)-LRx.
ABOUT THE AKCEA AND NOVARTIS COLLABORATION
In January 2017, Akcea and Ionis entered into an exclusive,
worldwide option and collaboration agreement with Novartis to
develop and commercialize AKCEA-APOCIII-LRx and
AKCEA-APO(a)-LRx. Akcea is conducting a Phase 2 dose-ranging study
for AKCEA-APO(a)-LRx in patients with high lipoprotein(a), or
Lp(a), with established cardiovascular disease with data planned in
2018 and a Phase 2b dose-ranging study for AKCEA-APOCIII-LRx in
patients with hypertriglyceridemia and established cardiovascular
disease with data planned in 2019. The goal of both studies is to
choose the optimal dose and evaluate alternative dose schedules,
such as monthly dosing, for Phase 3 cardiovascular outcomes
studies. Novartis has the option to license each drug after
successful completion of the Phase 2 dose-ranging study and
end-of-Phase 2 meeting with FDA. Upon option exercise for each
drug, Novartis will pay Akcea a $150 million license fee
of which 50% will be paid to Ionis. If licensed, Novartis plans to
conduct a global Phase 3 cardiovascular outcome study in high-risk
patients. Novartis will be responsible for worldwide development
and, if approved, co-commercialization activities. Akcea retains
the right to co-commercialize any successful drug through its
specialty sales force focused on lipid specialists on terms and
conditions to be agreed with Novartis.
ABOUT Lp(a) Elevated Lp(a) is recognized as an
independent, genetic cause of cardiovascular disease. Lp(a) levels
are determined at birth and, therefore, lifestyle modification,
including diet and exercise, do not impact Lp(a) levels. Currently,
there is no effective drug therapy to target and reduce elevated
levels of Lp(a). Additional information is available from the
Lipoprotein(a) Foundation at
http://www.lipoproteinafoundation.org/.
In a Phase 1/2 study with AKCEA-APO(a)-LRx in patients with
elevated levels of Lp(a), significant and sustained reductions in
Lp(a) of up to 97% were observed, with a mean reduction of 79%
after only a single, small volume dose of AKCEA-APO(a)-LRx. With
multiple doses of AKCEA-APO(a)-LRx, even greater reductions of
Lp(a) of up to 99% were observed, with a mean reduction of 92%.
AKCEA-APO(a)-LRx was generally safe and well tolerated in the
study, which supported continued development. Out of 165
injections, there were no injection site reactions or flu-like
symptoms reported. To review the full study results published in
The Lancet, click here.
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
AKCEA-APO(a)-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.
In this press release, unless the context requires otherwise,
“Ionis”, “Akcea,” “Company,” “Companies” “we,” “our,” and “us”
refers to Ionis Pharmaceuticals and/or Akcea Therapeutics.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals,
Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc.
Media and Investor Contact: Kathleen
Gallagher
Head of Communications and Investor Relations, Akcea
Therapeutics (617)-207-8509 kgallagher@akceatx.com
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