CAMBRIDGE, Mass. and CARLSBAD,
Calif., July 11, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc.
(NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), announced today that
TEGSEDITM (inotersen)
has received marketing authorization approval from the European
Commission (EC) for the treatment of stage 1 or stage 2
polyneuropathy in adult patients with hereditary transthyretin
amyloidosis (hATTR). This follows the positive opinion recommending
approval provided by the Committee for Medicinal Products for Human
Use (CHMP) of European Medicines Agency (EMA). Click
here to view the EC's decision.
"With the EC's decision, TEGSEDI is now the
world's first and only RNA-targeted therapeutic approved for
patients with hATTR amyloidosis. With subcutaneous delivery,
TEGSEDI puts treatment in the patients' hands while bringing the
significant benefits shown in the NEURO-TTR study in both measures
of neuropathy and quality of life for people living with this
serious and fatal disease. This is an important day for the hATTR
amyloidosis community as we believe TEGSEDI enables people and
their families impacted by this disease to move forward with their
lives," said Paula Soteropoulos, chief executive officer at Akcea
Therapeutics. "Today is a milestone for Akcea with our first drug
approval. It is an achievement we share with the courageous hATTR
patient community in Europe and around the globe. We are ready to
launch TEGSEDI along with our patient and physician support
services across Europe."
The abnormal formation and aggregation of
transthyretin (TTR) protein results in TTR amyloid deposits
throughout the body and is the underlying cause of hATTR
amyloidosis. TEGSEDI is designed to block production of the TTR
protein. In the NEURO-TTR study, treatment with TEGSEDI produced
substantial reductions in the levels of the TTR protein regardless
of mutation type or stage of disease.
"hATTR amyloidosis is an inherited, progressive
and fatal disease for which treatment options are limited. The
approval of TEGSEDI brings us into a new era of treatment with an
efficacious and disease modifying medicine that potentially allows
patients to achieve a greater degree of independence," said Teresa
Coelho, M.D., neurologist and neurophysiologist at Santo António
Hospital, Porto, Portugal. "TEGSEDI has demonstrated rapid and
sustained benefits in improving the course of this disease and
preserving quality of life."
The European Commission's approval of TEGSEDI was
based on results from the Phase 3 NEURO-TTR study in patients with
hATTR amyloidosis with symptoms of polyneuropathy. Results from
that study demonstrated that patients treated with TEGSEDI
experienced significant benefit compared to patients treated with
placebo across both co-primary endpoints: the Norfolk Quality of
Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and
modified Neuropathy Impairment Score +7 (mNIS+7), a measure of
neuropathic disease progression.
TEGSEDI is associated with risk of
thrombocytopenia and glomerulonephritis. Enhanced monitoring is
required to support early detection and management of these
identified risks.
"Today, we are thrilled to see our successful
research and development efforts result in the approval of an
important new medicine for patients with hATTR amyloidosis. Using
our antisense technology platform, we set out to design a therapy
to block the production of the underlying cause of this disease,
the TTR protein," said Brett P. Monia, Ph.D., chief operating
officer at Ionis Pharmaceuticals. "Approval of TEGSEDI further
establishes Ionis as a multi-product company. We are confident that
the experienced team at Akcea will deliver on the promise of
TEGSEDI. We are grateful to all of the physicians and patients who
participated in the TEGSEDI clinical program and who made this
landmark approval possible."
For important safety information for TEGSEDI,
including method of administration, special warnings, drug
interactions and adverse drug reactions, please see the European
Summary of Product Characterstics (SmPC), available from the EMA
website at www.ema.europa.eu.
TEGSEDI is also under regulatory review in the
United States and Canada. TEGSEDI's U.S. Prescription Drug User Fee
Act, or PDUFA, date is October 6, 2018.
In April, Akcea licensed the worldwide rights to
commercialize TEGSEDI from Ionis. Based on the EC authorization of
TEGSEDI, Ionis will receive a $40 million milestone payment from
Akcea payable in shares of Akcea common stock. Commercial profits
and losses from TEGSEDI will be split 60% to Ionis and 40% to
Akcea.
ABOUT TEGSEDITM (INOTERSEN)
TEGSEDITM (inotersen)
is an antisense oligonucleotide (ASO) inhibitor of human
transthyretin (TTR) production. TEGSEDI is approved in the E.U. for
the treatment of stage 1 or stage 2 polyneuropathy in adult
patients with hereditary transthyretin amyloidosis (hATTR) and is
currently under regulatory review in the U.S. and Canada.
The approval is based on data from the NEURO-TTR
study which was a Phase 3 randomized (2:1), double-blind,
placebo-controlled, international study in 172 patients with hATTR
amyloidosis with symptoms of polyneuropathy. The 15-month study
measured the effects of TEGSEDI on neurological function 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. TEGSEDI provided significant benefit on both of these
co-primary endpoints in the NEURO-TTR study, including improvement
in disease relative to baseline measurements in both co-primary
endpoints for a substantial portion of patients.
TEGSEDI is associated with risk of
thrombocytopenia and glomerulonephritis. Enhanced monitoring is
required to support early detection and management of these
identified risks. The most frequently observed adverse reactions
during treatment with TEGSEDI were events associated with injection
site reactions. The other most commonly reported adverse reactions
(over 10%) seen with TEGSEDI were nausea, anaemia, headache,
pyrexia, peripheral oedema, chills, vomiting, thrombocytopenia and
platelet count decreased.
The approval is also based on data from the
NEURO-TTR Open Label Extension (OLE) which is an ongoing study for
patients who completed the NEURO-TTR study, designed to evaluate
the long-term efficacy and safety of TEGSEDI.
The TEGSEDI expanded access program (EAP)
(NCT03400098) has been initiated in the U.S. and is currently
enrolling eligible patients. Click here for more
information on the TEGSEDI EAP. For more information on TEGSEDI,
please visit www.TEGSEDI.eu.
ABOUT HEREDITARY
TRANSTHYRETIN (hATTR) AMYLOIDOSIS
hATTR amyloidosis is a progressive, systemic and fatal inherited
disease caused by the abnormal formation of the TTR protein 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
THERAPEUTICS
Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals,
Inc., 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 TEGSEDITM (inotersen),
WAYLIVRATM (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. TEGSEDI is approved in the E.U. for the
treatment of stage 1 or stage 2 polyneuropathy in adult patients
with hereditary transthyretin amyloidosis (hATTR) and is currently
under regulatory review in the US and Canada. WAYLIVRA is under
regulatory review in the U.S., E.U. and Canada for the treatment of
familial chylomicronemia syndrome, or FCS, and is currently in
Phase 3 clinical development for the treatment of people with
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.
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 40 drugs in development. SPINRAZA® (nusinersen)
has been approved in global markets for the treatment of spinal
muscular atrophy (SMA). Biogen is responsible for commercializing
SPINRAZA. TEGSEDITM (inotersen)
and WAYLIVRATM (volanesorsen)
are two antisense drugs that Ionis discovered and successfully
advanced through Phase 3 studies. TEGSEDI is approved in the E.U.
for the treatment of stage 1 or stage 2 polyneuropathy in adult
patients with hereditary transthyretin amyloidosis, or hATTR, and
is currently under regulatory review in the U.S. and Canada.
WAYLIVRA is under regulatory review for marketing approval in the
U.S., E.U., and Canada for the treatment of patients with familial
chylomicronemia syndrome, or FCS. WAYLIVRA is also in a Phase 3
study in patients with familial partial lipodystrophy, or FPL.
Akcea Therapeutics, an affiliate of Ionis focused on developing and
commercializing drugs to treat patients with serious and rare
diseases, will commercialize TEGSEDI and WAYLIVRA, if approved.
Ionis' patents provide strong and extensive protection for its
drugs and technology. Additional information about Ionis is
available at www.ionispharma.com.
AKCEA'S AND IONIS'
FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding
the business of Akcea Therapeutics, Inc. and Ionis Pharmaceuticals,
Inc. and the therapeutic and commercial potential of
TEGSEDITM Any
statement describing Akcea's or Ionis' goals, expectations,
financial or other projections, intentions or beliefs, including
the commercial potential of TEGSEDI or other of Akcea's or Ionis'
drugs in development 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 and
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 Akcea's and Ionis'
forward-looking statements reflect the good faith judgment of its
management, these statements are based only on facts and factors
currently known by Akcea and Ionis. As a result, you are cautioned
not to rely on these forward-looking statements. These and other
risks concerning Ionis' and Akcea's programs are described in
additional detail in Ionis' and Akcea's quarterly reports on Form
10-Q and annual reports on Form 10-K, which are on file with the
SEC. Copies of these and other documents are available from each
company.
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(TM) is a trademark of Ionis
Pharmaceuticals, Inc. Akcea Therapeutics(TM), TEGSEDITM and
WAYLIVRATM are
trademarks of Akcea Therapeutics, Inc.
Akcea Investor
Contact:
Kathleen Gallagher
Vice President of Communications and Investor Relations
617-207-8509
kgallagher@akceatx.com
Akcea Media
Contact:
Liz Bryan
Spectrum Communications
202-587-2526
lbryan@spectrumscience.com
Ionis Investor
Contact:
D. Wade Walke, Ph.D.
Vice President, Investor Relations
760-603-2741
wwalke@ionisph.com
Ionis Media
Contact:
Roslyn Patterson
Vice President, Corporate Communications
760-603-2681
rpatterson@ionisph.com
This
announcement is distributed by Nasdaq Corporate Solutions on behalf
of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: Akcea Therapeutics, Inc. via Globenewswire
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