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)
AMYLOIDOSIShATTR 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 THERAPEUTICSAkcea 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
STATEMENTThis 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™ is a trademark of Ionis Pharmaceuticals,
Inc. Akcea Therapeutics™, TEGSEDITM and WAYLIVRATM are trademarks
of Akcea Therapeutics, Inc.
Akcea Investor Contact:Kathleen GallagherVice
President of Communications and Investor
Relations617-207-8509kgallagher@akceatx.com
Akcea Media Contact:Liz BryanSpectrum
Communications 202-587-2526lbryan@spectrumscience.com
Ionis Investor Contact:D. Wade Walke, Ph.D.Vice
President, Investor Relations760-603-2741wwalke@ionisph.com
Ionis Media Contact:Roslyn PattersonVice
President, Corporate
Communications760-603-2681rpatterson@ionisph.com
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