Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis
Pharmaceuticals, Inc.(NASDAQ: IONS), announced today that they are
working together with Accredo® specialty pharmacy, a subsidiary of
Express Scripts (NASDAQ: ESRX), to distribute
TEGSEDI™ (inotersen) subcutaneous injection for the treatment
of the polyneuropathy of hereditary transthyretin-mediated (hATTR)
amyloidosis in adults.
Approved by the U.S. Food and Drug Administration (FDA) today,
TEGSEDI is the first and only subcutaneous RNA-targeting
therapeutic offering patients an effective treatment for the
polyneuropathy of hATTR amyloidosis. Subcutaneous delivery
represents a significant new option for the patients, caregivers,
and healthcare professionals in the U.S. and making TEGSEDI widely
available to patients is now a critical next step. Akcea chose
Express Scripts’ Accredo because of its experience supporting the
unique needs of rare disease communities and its proven track
record for simplifying access to therapy.
“Accredo is entirely focused on supporting the needs of patients
with rare and complex conditions like hATTR amyloidosis, and we are
prepared and ready to serve patients and physicians by distributing
TEGSEDI,” said Bill Martin, Vice President, GM Accredo and
CuraScript SD. “In addition, our Rare Disease Therapeutic Resource
Center (TRC)®, provides patient education and proactive outreach to
help improve health outcomes while reducing time to therapy and
overall healthcare costs. Working together with Akcea, we are
collectively united in our commitment to simplifying the patient
journey and providing timely access to therapy.”
Accredo's robust team of specialty clinicians, pharmacists and
over 600 field-based nurses located throughout the U.S. will
augment the AKCEA CONNECT™ team of nurse case managers to provide
support and address the needs of the hATTR community.
“TEGSEDI is a weekly self-administered injection, that should
reduce administration costs and drug wastage. By introducing
competition into the marketplace this should drive down the total
cost of care,” said Steve Miller, MD, and Chief Medical Officer at
Express Scripts.
“Akcea and Accredo share a common goal to simplify the patient
experience while improving access to therapies that can transform
lives, like TEGSEDI. This strategic collaboration and partnership
with Express Scripts and Accredo allows us to consider innovative
access strategies such as value and outcomes-based access
arrangements in addition to other contracting strategies and
risk-based approaches that address access and affordability
concerns of patients, providers, and payers,” said Kyle
Jenne, head of U.S. commercial at Akcea Therapeutics.
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™ (inotersen), 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 (hATTR).
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 affects 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 AKCEA CONNECT™Akcea Therapeutics is
committed to ensuring that patients have access to TEGSEDI. Our
patient assistance program called AKCEA CONNECT offers assistance
to qualified patients at no cost. AKCEA CONNECT offers personalized
and dedicated support to patients and their care teams through
best-in-class services. The program focuses on sharing knowledge,
enabling access and empowering patients in order to optimize care
in rare diseases, improve patient outcomes, and enhance patients’
overall experiences. For more information please visit
www.AkceaConnect.com or call 1-866-AKCEATX (1-866-252-3289).
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 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.
ABOUT EXPRESS SCRIPTSExpress Scripts puts
medicine within reach of tens of millions of people by aligning
with plan sponsors, taking bold action and delivering
patient-centered care to make better health more affordable and
accessible.
Headquartered in St. Louis, Express Scripts provides integrated
pharmacy benefit management services, including network-pharmacy
claims processing home delivery pharmacy care, specialty pharmacy
care, specialty benefit management, benefit-design consultation,
drug utilization review, formulary management, and medical and drug
data analysis services. Express Scripts also distributes a
full range of biopharmaceutical products and provides extensive
cost-management and patient-care services.
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
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 annual report on Form 10-K for the
year ended December 31, 2017, and most recent Form 10-Q quarterly
filing, which are on file with the SEC. Copies of this 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™, WAYLIVRA™ are trademarks of
Akcea Therapeutics, Inc.
Akcea Media and Investor Contact:Kathleen
GallagherVice President of Communications and Investor
Relations617-207-8509kgallagher@akceatx.com
© 2018. Akcea Therapeutics, Inc. All rights reserved.
US-TEG-1800147
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