Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis
Pharmaceuticals, Inc., today announced that it will present the
long-term analyses of the NEURO-TTR open-label extension study and
TEGSEDI’s impact on the lives of patients living with
polyneuropathy caused by hereditary transthyretin amyloidosis
(hATTR) at the 60th American Society of Hematology Annual Meeting
and Exposition in San Diego, California, December 1-4, 2018.
In addition to the oral presentation, a poster on the benefit of
TEGSEDI to patients from the NEURO-TTR study will be presented.
Sunday, December 2, 2018: 5:45 PM PT
- Oral: Long-Term Update from the Open-Label Extension of the
NEURO-TTR Study in Patients with Hereditary Transthyretin
Amyloidosis by Dr. Morie A. Gertz (Session Name: 903. Outcomes
Research—Non-Malignant Hematology: Health Outcomes in Hemophilia,
Thrombosis, ITP, and Hereditary Transthyretin Amyloidosis. San
Diego Convention Center, Room 24B)
Monday, December 3, 2018: 6:00 PM – 8:00 PM
PT
- Poster 4812: Impact of Inotersen on Functioning and Activities
of Daily Living for Patients with Hereditary TTR Amyloidosis:
Results from a Double-Blind Placebo-Controlled Trial (Session Name:
903. Outcomes Research – Non-Malignant Hematology: Poster III. San
Diego Convention Center, Hall GH)
Akcea will have an on-site presence at the meeting at booth
#2159 in the San Diego Convention Center where attendees can learn
more about TEGSEDI and hATTR amyloidosis with polyneuropathy.
ABOUT TEGSEDI™ (INOTERSEN)TEGSEDI was
approved by the U.S. Food and Drug Administration (FDA) for the
treatment of the polyneuropathy of hereditary
transthyretin-mediated (hATTR) amyloidosis in adults. TEGSEDI,
discovered and developed by Ionis Pharmaceuticals, is the world’s
first and only subcutaneous RNA-targeting drug designed to reduce
the production of human transthyretin (TTR) protein. TEGSEDI also
received marketing authorization in the European Union and Canada
for the treatment of stage 1 or stage 2 polyneuropathy in adult
patients with hereditary transthyretin amyloidosis.
The approval is based on data from the NEURO-TTR study that was
a Phase 3 randomized (2:1), double-blind, placebo-controlled,
15-month, international study in 172 patients with hATTR
amyloidosis with symptoms of polyneuropathy. In NEURO-TTR, TEGSEDI
demonstrated significant benefit compared to placebo in measures of
neuropathy and quality of life as measured by the modified
Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality
of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total
score. Patients treated with TEGSEDI experienced similar benefit
regardless of subgroups such as age, sex, race, region, Neuropathy
Impairment Score (NIS), Val30Met mutation status, and disease
stage.
The approval is also based on data from the NEURO-TTR Open Label
Extension (OLE) that is an ongoing study for patients who completed
the NEURO-TTR study, designed to evaluate the long-term efficacy
and safety of TEGSEDI.
For TEGSEDI’s full prescribing information, please visit
www.TEGSEDI.com.
IMPORTANT SAFETY INFORMATION
TEGSEDI can cause serious side effects including:Low platelet
counts (thrombocytopenia): TEGSEDI may cause the number of
platelets in your blood to be reduced. This is a common side effect
of TEGSEDI. When your platelet count is too low, your body cannot
form clots. You could have serious bleeding that could lead to
death. Call your healthcare provider immediately if you have:
- Unusual bruising or a rash of tiny reddish-purple spots, often
on the lower legs
- Bleeding from skin cuts that does not stop or oozes
- Bleeding from your gums or nose
- Blood in your urine or stools
- Bleeding into the whites of your eyes
- Sudden severe headaches or neck stiffness
- Vomiting or coughing up blood
- Abnormal or heavy periods (menstrual bleeding)
Kidney inflammation (glomerulonephritis): Your kidneys may stop
working properly. Glomerulonephritis can lead to severe kidney
damage and kidney failure that need dialysis. Call your healthcare
provider immediately if you have:
- Puffiness or swelling in your face, feet, or hands
- New onset or worsening shortness of breath and coughing
- Blood in your urine or brown urine
- Foamy urine (proteinuria)
- Passed less urine than usual
Because of the risk of serious bleeding caused by low platelet
counts and because of the risk of kidney problems, TEGSEDI is
available only through a restricted program called the TEGSEDI Risk
Evaluation and Mitigation Strategy (REMS) Program. Talk to your
healthcare provider about how to enroll in the TEGSEDI REMS
Program.
Do not use TEGSEDI if you have:
- A platelet count that is low
- Had kidney inflammation (glomerulonephritis) caused by
TEGSEDI
- Had an allergic reaction to inotersen or any of the ingredients
in TEGSEDI. See the end of the Medication Guide for a complete list
of ingredients in TEGSEDI
Before you start TEGSEDI, tell your healthcare provider about
all of your health issues, including if you:
- Have or had bleeding problems
- Have or had kidney problems
- Are pregnant or plan to become pregnant. It is not known if
TEGSEDI can harm your unborn baby
- Are breastfeeding or plan to breastfeed. It is not known if
TEGSEDI can pass into your breast milk or harm your baby. Talk with
your healthcare provider about the best way to feed your baby while
you are taking TEGSEDI
Tell your healthcare provider about all the medicines you take,
including prescription and over-the-counter medicines, vitamins,
and herbal supplements. Especially tell your healthcare provider if
you take vitamin A or beta-carotene supplements, blood thinners
(anticoagulants), or drugs that affect blood clotting.
Required monitoringYour healthcare provider will test your blood
and urine to check your platelet counts and kidney and liver
function before you start TEGSEDI. While you are receiving TEGSEDI,
you will be monitored closely for symptoms, which includes checking
your platelet counts every week (or more frequently as needed),
kidney function every 2 weeks, and liver function every 4 months.
If your healthcare provider has you stop taking TEGSEDI, you will
need to continue to get your blood and urine tested for 8 more
weeks after treatment.
TEGSEDI may cause serious side effects, including:Stroke.
TEGSEDI may cause a stroke. One person taking TEGSEDI had a stroke,
which occurred within 2 days after the first dose. Get emergency
help immediately if you have symptoms of stroke, including sudden
numbness or weakness, especially on one side of the body; severe
headache or neck pain; confusion; problems with vision, speech, or
balance; droopy eyelids.
Inflammatory and immune system problems. Some people taking
TEGSEDI had serious inflammatory and immune system problems.
Symptoms of inflammatory and immune system problems included
unexpected change in walking, weakness and spasms in legs, back
pain, weight loss, headache, vomiting, and problems with
speech.
Liver effects. TEGSEDI may cause liver problems. Your healthcare
provider should do laboratory tests to check your liver before you
start TEGSEDI and while you are using it. Tell your healthcare
provider if you have symptoms that your liver may not be working
right, which could include unexpected nausea and vomiting, stomach
pain, being not hungry, yellowing of the skin, or having dark
urine.
Allergic reactions. TEGSEDI may cause serious allergic
reactions. These allergic reactions often occur within 2 hours
after injecting TEGSEDI. Get emergency help immediately if you have
any symptoms of a serious allergic reaction, including joint pain,
chills, redness on palms of hands, muscle pain, chest pain,
flushing, tremor or jerking movements, flu-like symptoms, high
blood pressure, or difficulty swallowing.
Eye problems (low vitamin A levels). Treatment with TEGSEDI will
lower the vitamin A levels in your blood. Your healthcare provider
will tell you how much supplemental vitamin A to take every day;
only take the amount they tell you to take. Call your healthcare
provider if you get eye problems, such as having difficulty seeing
at night or in low-lit areas (night blindness).
The most common side effects of TEGSEDI include injection site
reactions (such as redness or pain at the injection site), nausea,
headache, tiredness, low platelet counts (thrombocytopenia), and
fever. These are not all of the possible side effects of TEGSEDI.
Talk to your healthcare provider about any side effects you may be
experiencing.
You are encouraged to report negative side effects of
prescription drugs to the FDA. Visit www.fda.gov/medwatch or call
1-800-FDA-1088.
Please see Medication Guide and full Prescribing Information,
including boxed WARNING.
ABOUT HEREDITARY TRANSTHYRETIN (hATTR)
AMYLOIDOSIS.hATTR amyloidosis is a severe, progressive,
and life-threatening 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 and intestinal tract. The progressive accumulation of
TTR amyloid deposits in these organs often leads to intractable
peripheral sensorimotor neuropathy, autonomic neuropathy, and/or
cardiomyopathy, as well as other disease manifestations. hATTR
amyloidosis causes significant morbidity and progressive decline in
quality of life, severely impacting activities of daily living. The
disease often progress rapidly and can lead to premature death. The
median survival is 4.7 years following diagnosis. Additional
information on hATTR amyloidosis, including a full list of
organizations supporting the hATTR amyloidosis community worldwide,
is available at www.hattrchangethecourse.com or by visiting
www.hATTRGuide.com.
ABOUT AKCEA THERAPEUTICSAkcea Therapeutics,
Inc., 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 TEGSEDI™ (inotersen), WAYLIVRA™ (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 U.S.,
E.U. and Canada. WAYLIVRA is under regulatory review 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.
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 TEGSEDI™ (inotersen). Any statement describing
Akcea’s goals, expectations, financial or other projections,
intentions or beliefs, including the commercial potential of
TEGSEDI or other of Akcea’s 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
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 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 the 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™, TEGSEDI™ and WAYLIVRA™ are
trademarks of Akcea Therapeutics, Inc.
For More
Information:
Akcea Investor Contact:Kathleen GallagherVice
President of Communications and Investor Relations
(617)-207-8509kgallagher@akceatx.com
Akcea Media Contact:Liz BryanSpectrumT:
202.587.2526lbryan@spectrumscience.com
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