Akcea Therapeutics, Inc. (Nasdaq:AKCA), an affiliate of Ionis
Pharmaceuticals, Inc., today announced that data from inotersen’s
clinical development program for the treatment of hereditary ATTR
(hATTR) amyloidosis will be presented at the American Academy of
Neurology (AAN) Annual Meeting in Los Angeles, Calif., April 21-27,
2018.
“We are pleased to have the opportunity to present data from the
NEURO-TTR study in two oral presentations at this year’s American
Academy of Neurology Annual Meeting. These data, combined with the
sustained efficacy in the open label extension study, continue to
demonstrate that inotersen has the potential to significantly
benefit people living with hATTR amyloidosis,” said Sarah Boyce,
president of Akcea Therapeutics.
In addition to the oral presentations, two posters regarding
one-year follow-up results of an open-label extension of the Phase
3 NEURO-TTR study and the disease burden of hATTR amyloidosis will
also be presented.
AAN Oral Presentations
- Sunday, April 22, 2018 at 2:00pm PT – P. James B. Dyck, M.D.,
Mayo Clinic
- S5.006: Inotersen Improves Norfolk Quality of Life-Diabetic
Neuropathy Measures in Patients With Hereditary Transthyretin
Amyloidosis with Polyneuropathy in the Phase 3 Study
NEURO-TTR
- Monday, April 23, 2018 at 4:30pm PT – John Berk, M.D., Boston
University
- N2.001: Safety and Efficacy of Inotersen in Patients With
Hereditary Transthyretin Amyloidosis With Polyneuropathy
(NEURO-TTR)
AAN Poster Presentations
- Sunday, April 22, 2018 from 11:30am – 5:30pm PT
- P1.324: Open Label Extension of the Phase 3 Study NEURO-TTR to
Assess the Long-term Efficacy and Safety of Inotersen in Patients
With Hereditary Transthyretin Amyloidosis
- P1.331: Burden of Hereditary Transthyretin Amyloidosis With
Polyneuropathy in Patients Enrolled in the Phase 3 Study
NEURO-TTR
For the 2018 program and a full list of presentations, please
visit the AAN website at www.aan.com/conferences.
ABOUT INOTERSEN
Inotersen is an antisense drug designed to reduce the production
of transthyretin, or TTR protein, to treat ATTR amyloidosis, a
systemic, progressive and fatal disease. Inotersen is currently
under Priority Review for marketing authorization in the U.S. and
Accelerated Assessment in the EU. The U.S. Food and Drug
Administration has granted inotersen Orphan Drug Designation and
Fast Track Status, and the European Medicines Agency has granted
inotersen Orphan Drug Designation.
The NEURO-TTR study was a Phase 3 randomized (2:1),
double-blind, placebo-controlled, international study in 172
patients with polyneuropathy due to hATTR amyloidosis. The 15-month
study measured the effects of inotersen on neurological dysfunction
and on quality-of-life by measuring the change from baseline in the
modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk
Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN)
total score. The NEURO-TTR OLE is an ongoing study for patients who
completed the NEURO-TTR study and is intended to evaluate the
long-term efficacy and safety profile of inotersen.
Results from the Phase 3 NEURO-TTR study demonstrated that most
inotersen-treated patients experienced substantial reductions in
TTR. Nearly 90% of patients achieved >50% TTR reduction
and nearly 50% of patients achieved over 75% TTR reduction at 15
months. Median TTR reduction was 75-79% between weeks 13-65. These
TTR reductions were associated with positive outcomes compared to
placebo in both primary endpoints of the study: the Norfolk Quality
of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and the
modified Neuropathy Impairment Score +7 (mNIS+7) at both eight and
15 months of treatment. In addition, consistent positive outcomes
were observed in both the Norfolk QoL-DN and mNIS+7, independent of
disease stage, types of mutation or presence of cardiomyopathy at
the beginning of the study. Inotersen-treated patients showed
positive outcomes in the quality of life primary endpoint
with 50% of patients experiencing improved scores compared to
baseline and a mean difference in magnitude of 11.68 points,
compared to placebo-treated patients, at 15 months of treatment
(mean change from baseline of 0.99 vs. 12.67, p<0.001). In
addition, clinically meaningful positive outcomes compared to
placebo were observed in the SF-36 physical component score, a
measure of general health quality of life. Inotersen-treated
patients also was seen in the co-primary endpoint of disease
control, mNIS+7, with 37% of patients experiencing improved scores
compared to baseline and a mean difference in magnitude of 19.73
-points, compared to placebo-treated patients, at 15 months of
treatment, (p < 0.001). Whether or not a patient improved in
either Norfolk QOL-DN or mNIS+7 could not be predicted by TTR
reduction alone. Most patients achieved benefit regardless of
whether they were in the 50-75% reduction range or in the 75-95%
reduction range.
Positive outcomes were observed in inotersen-treated patients
with cardiac disease at baseline (interventricular septum
thickness, IVS ≥ 1.5 cm) in both primary endpoints (Norfolk QoL-DN,
p=0.036 and mNIS+7, p<0.001) and in the SF-36 Health Survey
endpoint (p=0.025) at 15 months, compared to placebo. Encouraging
outcomes also were observed in multiple cardiac measures, including
mean decreases in left ventricle mass (p=0.0288), IVS (p=0.0150)
and posterior wall thickness (p=0.0425), which increased, on
average, in placebo-treated patients.
Thrombocytopenia and safety signals related to renal function
were identified during the study. Enhanced monitoring was
implemented during the study to support early detection and
management of these issues. Serious platelet and renal events were
infrequent and easily managed with routine monitoring, which has
proven effective since implementation. Other serious adverse events
were observed in 24.1% of inotersen-treated patients and 21.7% of
placebo-treated patients. No cumulative toxicities have been
identified with long-term exposure.
Adverse events occurring in >=10% of patients and twice as
frequently in inotersen-treated patients compared with
placebo-treated patients, included thrombocytopenia/platelet count
decreases, nausea, pyrexia, chills, vomiting, and anemia. Injection
site reactions accounted for less than 1% of all injections and
were mild or moderate in severity. There were no discontinuations
due to injection site reactions. The overall mortality rate in the
NEURO-TTR study was 2.9% and was lower than overall mortality rates
reported in other studies in hATTR patients. There was a total of
five deaths in the study, five (4.7%) in the inotersen arm and zero
in the placebo arm. Four deaths in the inotersen arm were
associated with disease progression and considered unrelated to
treatment. As previously reported, there was one fatal intracranial
hemorrhage in conjunction with serious thrombocytopenia. No serious
thrombocytopenia was observed following implementation of more
frequent monitoring.
The inotersen expanded access program (EAP) has been initiated
for eligible patients in the U.S.
ABOUT HEREDITARY TRANSTHYRETIN (hATTR)
AMYLOIDOSIS
hATTR amyloidosis is a progressive, systemic and fatal genetic
disease caused by the inappropriate formation and aggregation of
TTR amyloid deposits in various tissues and organs throughout the
body, including in peripheral nerves, heart, intestinal tract,
eyes, kidneys, central nervous system, thyroid and bone marrow. The
progressive accumulation of TTR amyloid deposits in these tissues
and organs leads to sensory, motor and autonomic dysfunction often
having debilitating effects on multiple aspects of a patient's
life. Patients with hATTR amyloidosis often present with a mixed
phenotype and experience overlapping symptoms of polyneuropathy and
cardiomyopathy.
Ultimately, hATTR amyloidosis results in death within three to
fifteen years of symptom onset. Therapeutic options for the
treatment of patients with hATTR amyloidosis are limited and there
are currently no disease-modifying drugs approved for the disease.
There are an estimated 50,000 patients with hATTR amyloidosis
worldwide. Additional information on hATTR amyloidosis,
including a full list of organizations supporting the hATTR
amyloidosis community worldwide, is available at
www.hattrchangethecourse.com.
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 and rare diseases. Akcea is
advancing a mature pipeline of six novel drugs, including
inotersen, volanesorsen, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx,
AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, all with the potential to
treat multiple diseases. All six drugs were discovered by and are
being co-developed with Ionis, a leader in antisense therapeutics,
and are based on Ionis’ proprietary antisense technology. Inotersen
is under regulatory review in the U.S. and EU for the treatment of
hereditary amyloid transthyretin amyloidosis (hATTR) amyloidosis.
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. Akcea is a
global company headquartered in Cambridge, Massachusetts.
Additional information about Akcea is available at
www.akceatx.com.
AKCEA’S FORWARD-LOOKING STATEMENTThis press
release includes forward-looking statements regarding the business
of Akcea Therapeutics, Inc. and the therapeutic and commercial
potential of inotersen 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 annual report on Form 10-K for the year ended
December 31, 2017, 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 Akcea Therapeutics,
Inc.
Media and Investor Contact:Kathleen
Gallagher
Head of Communications and Investor Relations
(617)-207-8509kgallagher@akceatx.com
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