- Accepted abstracts include data
assessing the long-term efficacy and safety of SOLIRIS®
(eculizumab) in neuromyelitis optica spectrum disorder (NMOSD)
-
- Data from 11 abstracts demonstrate Alexion’s
commitment to continuing to advance the understanding of the burden
of NMOSD for patients and the efficacy and safety of SOLIRIS in
real-world settings -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
11 abstracts will be presented at MSVirtual2020, the 8th joint
Americas Committee for Treatment and Research in Multiple Sclerosis
and European Committee for Treatment and Research in Multiple
Sclerosis (ACTRIMS-ECTRIMS) meeting, taking place virtually from
September 11 to 13, 2020. The data being presented include two oral
presentations of additional findings from the Phase 3 PREVENT study
of SOLIRIS® (eculizumab) in anti-aquaporin-4 (AQP4) antibody
positive neuromyelitis optica spectrum disorder (NMOSD) – one on
long-term safety and efficacy data through 192 weeks of treatment
with SOLIRIS as monotherapy, and another demonstrating the risk of
adjudicated relapse was significantly lower with SOLIRIS than
placebo in patients who had previously received rituximab. An
additional analysis reinforces the safety of SOLIRIS as a treatment
for patients with AQP4 antibody positive NMOSD and for adult
patients with generalized Myasthenia Gravis (gMG) who are
anti-acetylcholine receptor (AchR) antibody positive, based on data
from the Phase 3 PREVENT and REGAIN studies and their
extensions.
The totality of data being presented at MSVirtual2020 reinforces
Alexion’s commitment to conducting research that helps increase
awareness and understanding of the burden of disease. Additionally,
the data further strengthen the evidence supporting the substantial
efficacy and durability of SOLIRIS in reducing relapse risk in
patients with AQP4 antibody positive NMOSD and illustrate the
benefits of SOLIRIS in decreasing healthcare resource utilization
and use of concomitant immunosuppressive therapies (ISTs).
All accepted abstracts are listed below and are now available on
the MSVirtual2020 website:
Long-term efficacy and safety of eculizumab monotherapy in
AQP4+ neuromyelitis optica spectrum disorder. Oral: FC01.01 –
Sunday, September 13, 2020, 2:00 PM-2:12 PM ET; Free Communications
1
Efficacy and safety of eculizumab in patients with
neuromyelitis optica spectrum disorder previously treated with
rituximab: findings from PREVENT. Oral: FC01.02 – Sunday,
September 13, 2020, 2:12 PM-2:24 PM ET; Free Communications 1
Safety of eculizumab in NMOSD and MG – analysis of the phase
3 studies PREVENT and REGAIN and their extensions Poster: P0752
– e-Poster presentation
Long-term efficacy and safety of eculizumab in AQP4+
neuromyelitis optica spectrum disorder Poster: P0727– e-Poster
presentation
Benefit of eculizumab for a broad range of patients with
aquaporin-4 antibody-positive neuromyelitis optica spectrum
disorder: findings from PREVENT Poster: P0692 – e-Poster
presentation
Burden of disease in patients with neuromyelitis optica
spectrum disorder: insights from the CIRCLES study cohort
Poster: P0695 – e-Poster presentation
Relapse-associated visual impairment and disability in
patients with neuromyelitis optica spectrum disorder Poster:
P0748 – e-Poster presentation
Patients with neuromyelitis spectrum disorder who experience
relapses take more chronic pain medication Poster: P0743 –
e-Poster presentation
Cost of neuromyelitis optica spectrum disorder in US clinical
practice. Poster: P0706 – e-Poster presentation
Relapses and associated healthcare utilization among patients
with neuromyelitis optica spectrum disorder in US clinical
practice. Poster: P0749 – e-Poster presentation
Impact of relapse on disability and quality of life in
patients with neuromyelitis optica spectrum disorder: findings from
the phase 3 PREVENT study. Poster: P0718 – e-Poster
presentation
About Neuromyelitis Optica Spectrum Disorder (NMOSD)
Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare
autoimmune disease of the central nervous system (CNS).
Approximately three-quarters of NMOSD patients have anti-AQP4
antibody-positive NMOSD. In patients with these antibodies, NMOSD
occurs when the complement system—a part of the body’s immune
system—over-responds—leading the body to primarily attack the optic
nerves and/or spinal cord in the CNS. People living with NMOSD
often experience unpredictable attacks, also referred to as
relapses, which tend to be severe and recurrent and may result in
permanent disability. The most common symptoms of NMOSD are optic
neuritis, which can cause visual problems including blindness, and
transverse myelitis, which can cause mobility problems including
paralysis. The disease primarily affects women, with an average age
of onset of 39 years. NMOSD is more common and more severe in
non-Caucasian populations worldwide.
About SOLIRIS® (eculizumab)
SOLIRIS® (eculizumab) is a first-in-class C5 complement
inhibitor. The medication works by inhibiting the C5 protein in the
terminal complement cascade, a part of the body’s immune system.
When activated in an uncontrolled manner, the terminal complement
cascade over-responds, leading the body to attack its own healthy
cells. SOLIRIS is administered intravenously every two weeks,
following an introductory dosing period. In many countries around
the world, SOLIRIS is approved to treat paroxysmal nocturnal
hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS),
adults with generalized myasthenia gravis (gMG) who are
acetylcholine receptor (AchR) antibody positive and/or adults with
neuromyelitis optica spectrum disorder (NMOSD) who are
anti-aquaporin-4 (AQP4) antibody positive. SOLIRIS is not indicated
for the treatment of patients with Shiga-toxin E. coli-related
hemolytic uremic syndrome (STEC-HUS). To learn more about the
regulatory status of SOLIRIS in the countries that we serve, please
visit www.alexion.com.
INDICATIONS & IMPORTANT SAFETY INFORMATION FOR SOLIRIS®
(eculizumab) injection for intravenous use, 300 mg/30 mL
vial
INDICATION(S) What is SOLIRIS? SOLIRIS is a prescription
medicine used to treat:
- patients with a disease called Paroxysmal Nocturnal
Hemoglobinuria (PNH).
- adults and children with a disease called atypical Hemolytic
Uremic Syndrome (aHUS). SOLIRIS is not for use in treating people
with Shiga toxin E. coli related hemolytic uremic syndrome
(STEC-HUS)
- adults with a disease called generalized myasthenia gravis
(gMG) who are anti-acetylcholine receptor (AChR) antibody
positive.
- adults with a disease called neuromyelitis optica spectrum
disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody
positive.
It is not known if SOLIRIS is safe and effective in children
with PNH, gMG, or NMOSD.
IMPORTANT SAFETY INFORMATION What is the most important
information I should know about SOLIRIS? SOLIRIS is a medicine that
affects your immune system and can lower the ability of your immune
system to fight infections.
- SOLIRIS increases your chance of getting serious and
life-threatening meningococcal infections that may quickly become
life-threatening and cause death if not recognized and treated
early.
- You must receive meningococcal vaccines at least 2 weeks before
your first dose of SOLIRIS if you are not vaccinated.
- If your doctor decided that urgent treatment with SOLIRIS is
needed, you should receive meningococcal vaccination as soon as
possible.
- If you have not been vaccinated and SOLIRIS therapy must be
initiated immediately, you should also receive two weeks of
antibiotics with your vaccinations.
- If you had a meningococcal vaccine in the past, you might need
additional vaccination. Your doctor will decide if you need
additional vaccination.
- Meningococcal vaccines reduce but do not prevent all
meningococcal infections. Call your doctor or get emergency medical
care right away if you get any of these signs and symptoms of a
meningococcal infection: headache with nausea or vomiting, headache
and fever, headache with a stiff neck or stiff back, fever, fever
and a rash, confusion, muscle aches with flu-like symptoms, and
eyes sensitive to light.
Your doctor will give you a Patient Safety
Card about the risk of meningococcal infection. Carry it with
you at all times during treatment and for 3 months after your last
SOLIRIS dose. It is important to show this card to any doctor or
nurse to help them diagnose and treat you quickly.
SOLIRIS is only available through a
program called the SOLIRIS REMS. Before you can receive
SOLIRIS, your doctor must enroll in the SOLIRIS REMS program;
counsel you about the risk of meningococcal infection; give you
information and a Patient Safety Card about the symptoms and
your risk of meningococcal infection (as discussed above); and make
sure that you are vaccinated with the meningococcal vaccine and, if
needed, get revaccinated with the meningococcal vaccine. Ask your
doctor if you are not sure if you need to be revaccinated.
SOLIRIS may also increase the risk of
other types of serious infections. Make sure your child
receives vaccinations against Streptococcus pneumoniae and
Haemophilus influenzae type b (Hib) if treated with SOLIRIS.
Certain people may be at risk of serious infections with gonorrhea.
Certain fungal infections (Aspergillus) may occur if you take
SOLIRIS and have a weak immune system or a low white blood cell
count.
Who should not receive SOLIRIS? Do not
receive SOLIRIS if you have a meningococcal infection or have not
been vaccinated against meningitis infection unless your doctor
decides that urgent treatment with SOLIRIS is needed.
Before you receive SOLIRIS, tell your
doctor about all of your medical conditions, including if you:
have an infection or fever, are pregnant or plan to become
pregnant, and are breastfeeding or plan to breastfeed. It is not
known if SOLIRIS will harm your unborn baby or if it passes into
your breast milk.
Tell your doctor about all the vaccines
you receive and medicines you take, including prescription and
over-the-counter medicines, vitamins, and herbal supplements which
could affect your treatment. It is important that you have all
recommended vaccinations before you start SOLIRIS, receive 2 weeks
of antibiotics if you immediately start SOLIRIS, and stay
up-to-date with all recommended vaccinations during treatment with
SOLIRIS.
If you have PNH, your doctor will need to
monitor you closely for at least 8 weeks after stopping SOLIRIS.
Stopping treatment with SOLIRIS may cause breakdown of your red
blood cells due to PNH. Symptoms or problems that can happen
due to red blood cell breakdown include: drop in the number of your
red blood cell count, drop in your platelet count, confusion,
kidney problems, blood clots, difficulty breathing, and chest
pain.
If you have aHUS, your doctor will need to
monitor you closely during and for at least 12 weeks after stopping
treatment for signs of worsening aHUS symptoms or problems related
to abnormal clotting (thrombotic microangiopathy). Symptoms or
problems that can happen with abnormal clotting may include:
stroke, confusion, seizure, chest pain (angina), difficulty
breathing, kidney problems, swelling in arms or legs, and a drop in
your platelet count.
What are the possible side effects of
SOLIRIS? SOLIRIS can cause serious side effects including serious
allergic reactions. Tell your doctor or nurse right away if you
get any of these symptoms during your SOLIRIS infusion: chest pain,
trouble breathing or shortness of breath, swelling of your face,
tongue, or throat, and feel faint or pass out. If you have an
allergic reaction to SOLIRIS, your doctor may need to infuse
SOLIRIS more slowly, or stop SOLIRIS.
The most common side effects in people
with PNH treated with SOLIRIS include: headache, pain or
swelling of your nose or throat (nasopharyngitis), back pain, and
nausea.
The most common side effects in people
with aHUS treated with SOLIRIS include: headache, diarrhea,
high blood pressure (hypertension), common cold (upper respiratory
infection), stomach-area (abdominal) pain, vomiting, pain or
swelling of your nose or throat (nasopharyngitis), low red blood
cell count (anemia), cough, swelling of legs or feet (peripheral
edema), nausea, urinary tract infections, and fever.
The most common side effects in people
with gMG treated with SOLIRIS include: muscle and joint
(musculoskeletal) pain.
The most common side effects in people
with NMOSD treated with SOLIRIS include: common cold (upper
respiratory infection), pain or swelling of your nose or throat
(nasopharyngitis), diarrhea, back pain, dizziness, flu like
symptoms (influenza) including fever, headache, tiredness, cough,
sore throat, and body aches, join pain (arthralgia), throat
irritation (pharyngitis), and bruising (contusion).
Tell your doctor about any side effect that bothers you or that
does not go away. These are not all the possible side effects of
SOLIRIS. For more information, ask your doctor or pharmacist. Call
your doctor for medical advice about side effects. You are
encouraged to report negative side effects of prescription drugs to
the FDA. Visit MedWatch, or call 1-800-FDA-1088.
Please see the full Prescribing Information and Medication
Guide for SOLIRIS, including Boxed WARNING regarding serious and
life-threatening meningococcal infections.
About Alexion Alexion is a global biopharmaceutical
company focused on serving patients and families affected by rare
diseases through the discovery, development and commercialization
of life-changing medicines. As the global leader in complement
biology and inhibition for more than 20 years, Alexion has
developed and commercializes two approved complement inhibitors to
treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and
atypical hemolytic uremic syndrome (aHUS), as well as the first and
only approved complement inhibitor to treat anti-acetylcholine
receptor (AchR) antibody-positive generalized myasthenia gravis
(gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion
also has two highly innovative enzyme replacement therapies for
patients with life-threatening and ultra-rare metabolic disorders,
hypophosphatasia (HPP) and lysosomal acid lipase deficiency
(LAL-D), as well as the first and only approved Factor Xa inhibitor
reversal agent. In addition, the company is developing several
mid-to-late-stage therapies, including a copper-binding agent for
Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for
rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D
inhibitor as well as several early-stage therapies, including one
for light chain (AL) amyloidosis, a second oral Factor D inhibitor
and a third complement inhibitor. Alexion focuses its research
efforts on novel molecules and targets in the complement cascade
and its development efforts on the core therapeutic areas of
hematology, nephrology, neurology, metabolic disorders and
cardiology. Headquartered in Boston, Massachusetts, Alexion has
offices around the globe and serves patients in more than 50
countries. This press release and further information about Alexion
can be found at: www.alexion.com.
[ALXN-P]
Forward-Looking Statement
This press release contains forward-looking statements that
involve risks and uncertainties relating to future events and the
future performance of Alexion, including statements related to:
Alexion’s commitment to continue to advance the understanding of
the burden of NMOSD for patients and the efficacy and safety of
SOLIRIS in real-world settings; the presented data will demonstrate
the risk of adjudicated relapse was significantly lower with
SOLIRIS than placebo in patients who had previously received
rituximab; and that data being presented at MSVirtual2020
reinforces Alexion’s commitment to conducting research that helps
increase awareness and understanding of the burden of disease; the
data further strengthen the evidence supporting the substantial
efficacy and durability of SOLIRIS in reducing relapse risk in
patients with AQP4 antibody positive NMOSD and illustrate the
benefits of SOLIRIS in decreasing healthcare resource utilization
and use of concomitant ISTs. Forward-looking statements are subject
to factors that may cause Alexion's results and plans to differ
materially from those expected by these forward looking statements,
including for example: the anticipated safety profile and the
benefits of SOLIRIS for adult patients with AQP4 antibody-positive
NMOSD may not be realized (and the results of the clinical trials
may not be indicative of future results); results of clinical
trials may not be sufficient to satisfy regulatory authorities;
results in clinical trials may not be indicative of results from
later stage or larger clinical trials (or in broader patient
populations);; the possibility that results of clinical trials are
not predictive of safety and efficacy and potency of our products
(or we fail to adequately operate or manage our clinical trials)
which could cause us to discontinue sales of the product (or halt
trials, delay or prevent us from making regulatory approval filings
or result in denial of approval of our product candidates); the
severity of the impact of the COVID-19 pandemic on Alexion’s
business, including on commercial and clinical development
programs; unexpected delays in clinical trials; unexpected concerns
regarding products and product candidates that may arise from
additional data or analysis obtained during clinical trials or
obtained once used by patients following product approval; future
product improvements may not be realized due to expense or
feasibility or other factors; delays (expected or unexpected) in
the time it takes regulatory agencies to review and make
determinations on applications for the marketing approval of our
products; inability to timely submit (or failure to submit) future
applications for regulatory approval for our products and product
candidates; inability to timely initiate (or failure to initiate)
and complete future clinical trials due to safety issues, IRB
decisions, CMC-related issues, expense or unfavorable results from
earlier trials (among other reasons); our dependence on sales from
our principal product (SOLIRIS); future competition from
biosimilars and novel products; decisions of regulatory authorities
regarding the adequacy of our research, marketing approval or
material limitations on the marketing of our products; delays or
failure of product candidates to obtain regulatory approval; delays
or the inability to launch product candidates due to regulatory
restrictions, anticipated expense or other matters; interruptions
or failures in the manufacture and supply of our products and our
product candidates; failure to satisfactorily address matters
raised by regulatory agencies regarding our products and product
candidates; uncertainty of long-term success in developing,
licensing or acquiring other product candidates or additional
indications for existing products; inability to complete
acquisitions or grow the product pipeline through acquisitions
(including due to failure to obtain antitrust approvals); the
possibility that current rates of adoption of our products are not
sustained; the adequacy of our pharmacovigilance and drug safety
reporting processes; failure to protect and enforce our data,
intellectual property and proprietary rights and the risks and
uncertainties relating to intellectual property claims, lawsuits
and challenges against us (including intellectual property lawsuits
relating to ULTOMIRIS brought by third parties); the risk that
third party payors (including governmental agencies) will not
reimburse or continue to reimburse for the use of our products at
acceptable rates or at all; failure to realize the benefits and
potential of investments, collaborations, licenses and
acquisitions; the possibility that expected tax benefits will not
be realized; potential declines in sovereign credit ratings or
sovereign defaults in countries where we sell our products; delay
of collection or reduction in reimbursement due to adverse economic
conditions or changes in government and private insurer regulations
and approaches to reimbursement; adverse impacts on our supply
chain, clinical trials, manufacturing operations, financial
results, liquidity, hospitals, pharmacies and health care systems
from natural disasters and global pandemics, including COVID-19;
uncertainties surrounding legal proceedings, company investigations
and government investigations; the risk that estimates regarding
the number of patients with PNH, aHUS, gMG, NMOSD, HPP and LAL-D
and other indications we are pursuing are inaccurate; the risks of
changing foreign exchange rates; risks relating to the potential
effects of the Company's restructuring; risks related to the
acquisitions of Portola Pharmaceuticals, Achillion and other
companies and co-development efforts; and a variety of other risks
set forth from time to time in Alexion's filings with the SEC,
including but not limited to the risks discussed in Alexion's
Quarterly Report on Form 10-Q for the period ended June 30, 2020
and in our other filings with the SEC. Alexion disclaims any
obligation to update any of these forward-looking statements to
reflect events or circumstances after the date hereof, except when
a duty arises under law.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200909005334/en/
Alexion Contacts: Media Megan Goulart,
857-338-8634 Senior Director, Corporate Communications
Investors Chris Stevo, 857-338-9309 Head of Investor
Relations
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