- Five abstracts accepted, including new
long-term data from PREVENT trial of SOLIRIS® (eculizumab)
in adults with anti-AQP4 antibody-positive NMOSD -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
five abstracts from the Phase 3 PREVENT study of SOLIRIS®
(eculizumab) in neuromyelitis optica spectrum disorder (NMOSD) have
been accepted for presentation at the 35th Congress of the European
Committee for Treatment and Research in Multiple Sclerosis
(ECTRIMS) in Stockholm, September 11 to 13, 2019. Key data will
include new long-term safety and efficacy findings through 192
weeks of SOLIRIS treatment, as well as data on the impact of
SOLIRIS on relapse-related hospitalizations, quality of life (QoL)
and disability. Collectively, the breadth of the data to be
presented at ECTRIMS support the robust treatment effect of SOLIRIS
on relapse prevention demonstrated in the PREVENT study and help to
further characterize the benefits of SOLIRIS for adult patients
with anti-aquaporin-4 (AQP4) antibody-positive NMOSD.
The accepted abstracts are listed below and are now available on
the ECTRIMS website:
Impact of eculizumab on hospitalization rates and relapse
treatment in patients with aquaporin-4 antibody-positive
neuromyelitis optica spectrum disorder: findings from the phase 3
PREVENT study. Abstract ID#: P604 – Poster Presentation
1, September 11, 2019, 17:15-19:15 CET, Poster Exhibition
Subgroup analyses from the Phase 3 PREVENT study in patients
with aquaporin-4 antibody-positive neuromyelitis optica spectrum
disorder. Abstract ID#: P605 – Poster Presentation 1,
September 11, 2019, 17:15-19:15 CET, Poster Exhibition
Impact of eculizumab on reported quality of life in patients
with aquaporin-4 antibody-positive neuromyelitis optica spectrum
disorder: findings from the PREVENT study. Abstract ID#:
P612 – Poster Presentation 1, September 11, 2019, 17:15-19:15 CET,
Poster Exhibition
Long-term safety and effectiveness of eculizumab in
neuromyelitis optica spectrum disorder. Abstract ID#:
142 – Free Communication, September 12, 2019, 09:06-09:18 CET, Hall
A
Impact of eculizumab on disability measures in patients with
aquaporin-4 antibody-positive neuromyelitis optica spectrum
disorder: phase 3 PREVENT study. Abstract ID#: P1343 –
Poster Presentation 3, September 13, 2019, 12:15-14:15 CET, Poster
Exhibition
SOLIRIS is approved in the EU for the treatment of NMOSD in
adult patients who are anti-AQP4 antibody positive with a relapsing
course of the disease. In the U.S., SOLIRIS is approved for the
treatment of adults with NMOSD who are anti-AQP4 antibody positive.
A supplemental New Drug Application currently is under review by
regulatory authorities in Japan. SOLIRIS received Orphan Drug
Designation (ODD) for the treatment of NMOSD in the U.S., EU and
Japan.
About NMOSD
NMOSD is a rare and severe, autoimmune,
inflammatory disorder that attacks the central nervous system
(CNS), in which complement activation due to anti-aquaporin-4
(AQP4) antibodies plays a significant role in the disease process.
Patients with NMOSD experience unpredictable attacks, also referred
to as relapses, which can cause irreversible damage predominantly
to the optic nerve and spinal cord and can lead to long-term
disability. The most common symptoms of NMOSD are optic neuritis
and transverse myelitis. Optic neuritis can cause visual problems
including blindness; transverse myelitis can cause mobility
problems including paralysis.
The disease primarily affects women, often in the prime of their
lives, with an average age of onset of 39 years. The prevalence of
NMOSD may be more common and more severe in non-Caucasian
populations worldwide.
Complement activation by anti-AQP4 antibodies can cause
destruction of vital cells in the CNS, leading to demyelination and
to the death of neurons, predominantly in the spinal cord and optic
nerve. Approximately three quarters (73%) of all patients with
NMOSD have AQP4 antibodies. In patients with anti-AQP4
antibody-positive NMOSD, the body’s own immune system can turn
against itself to produce antibodies against AQP4, a protein on
certain cells in the optic nerves, brain and spinal cord that are
critical for the survival of nerve cells. The binding of these
anti-AQP4 antibodies activates the complement cascade, another part
of the immune system.
About SOLIRIS
SOLIRIS® (eculizumab) is a first-in-class complement inhibitor
that works by inhibiting the C5 protein in the terminal part of the
complement cascade, a part of the immune system. The terminal
complement cascade, when activated in an uncontrolled manner, plays
a role in severe rare and ultra-rare disorders. SOLIRIS, an
intravenously administered therapy, is approved in the U.S., EU,
Japan and other countries as a treatment for adult patients with
PNH and for adults and children with aHUS. SOLIRIS is not indicated
for the treatment of patients with Shiga-toxin E. coli-related
hemolytic uremic syndrome (STEC-HUS). In the U.S., SOLIRIS is also
approved for the treatment of generalized MG (gMG) in adult
patients who are anti-AchR antibody-positive and for the treatment
of neuromyelitis optica spectrum disorder (NMOSD) in adult patients
who are anti-AQP4 antibody-positive, in the EU as the first and
only treatment of refractory gMG in adults who are anti-AchR
antibody-positive and for the treatment of NMOSD in adult patients
who are anti-AQP4 antibody-positive with a relapsing course of the
disease, and in Japan for the treatment of patients with gMG who
are anti-AChR antibody-positive and whose symptoms are difficult to
control with high-dose intravenous immunoglobulin (IVIG) therapy or
plasmapheresis (PLEX).
INDICATIONS & IMPORTANT SAFETY INFORMATION FOR SOLIRIS®
(eculizumab)
U.S. INDICATIONS
What is SOLIRIS?
SOLIRIS is a prescription medicine called a monoclonal antibody.
SOLIRIS is used to treat patients with a disease called Paroxysmal
Nocturnal Hemoglobinuria (PNH). SOLIRIS is used to treat 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). SOLIRIS is
used to treat adults with a disease called generalized myasthenia
gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody
positive. SOLIRIS is used to treat 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.
U.S. IMPORTANT SAFETY INFORMATION
SOLIRIS is a medicine that affects the immune system. SOLIRIS
can lower the ability of the immune system to fight infections.
SOLIRIS increases the chance of getting serious and
life-threatening meningococcal infections. Meningococcal infections
may quickly become life-threatening and cause death if not
recognized and treated early. Meningococcal vaccines must be
received at least two weeks before the first dose of SOLIRIS if one
has not already had this vaccine. If one’s doctor decided that
urgent treatment with SOLIRIS is needed, meningococcal vaccination
should be administered as soon as possible. If one has not been
vaccinated and SOLIRIS therapy must be initiated immediately, two
weeks of antibiotics should also be administered with the
vaccinations. If one had a meningococcal vaccine in the past,
additional vaccination might be needed before starting SOLIRIS.
Patients should ask their doctor if an additional meningococcal
vaccination is needed. Meningococcal vaccines reduce the risk of
meningococcal infection but do not prevent all meningococcal
infections. Call one’s doctor or get emergency medical care right
away if any of these signs and symptoms of a meningococcal
infection occur: 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.
One’s doctor will provide a Patient Safety Card about the risk
of meningococcal infection. Carry the card at all times during
treatment and for 3 months after the last SOLIRIS dose.
SOLIRIS is only available through a program called the SOLIRIS
REMS.
SOLIRIS may also increase the risk of other types of serious
infections. If one’s child is treated with SOLIRIS, make sure that
the child receives vaccinations against Streptococcus pneumoniae
and Haemophilus influenzae type b (Hib). Certain people may be at
risk of serious infections with gonorrhea. Talk to the doctor about
whether one is at risk for gonorrhea infection, about gonorrhea
prevention, and regular testing. Certain fungal infections
(Aspergillus) may also happen if one takes SOLIRIS and has a weak
immune system or a low white blood cell count.
Do not receive SOLIRIS if one has a meningococcal infection, or
has not been vaccinated against meningitis infection unless one’s
doctor decides that urgent treatment with SOLIRIS is needed.
Before one receives SOLIRIS, tell the doctor about all of the
medical conditions, including if one: has an infection or fever, is
pregnant or plans to become pregnant, and is breastfeeding or plans
to breastfeed. It is not known if SOLIRIS will harm an unborn baby
or if SOLIRIS passes into the breast milk.
Tell the doctor about all the medicines one takes, including
prescription and over-the-counter medicines, vitamins, and herbal
supplements. SOLIRIS and other medicines can affect each other,
causing side effects.
It is important that one: has all recommended vaccinations
before starting SOLIRIS, receives 2 weeks of antibiotics if one
immediately starts SOLIRIS, and stays up-to-date with all
recommended vaccinations during treatment with SOLIRIS. Know the
medications one takes and the vaccines one receives. Keep a list of
them to show the doctor and pharmacist when one gets a new
medicine.
If one has PNH, the doctor will need to monitor closely for at
least 8 weeks after stopping SOLIRIS. Stopping treatment with
SOLIRIS may cause breakdown of the red blood cells due to PNH.
Symptoms or problems that can happen due to red blood cell
breakdown include: drop in the number of the red blood cell count,
drop in the platelet counts, confusion, kidney problems, blood
clots, difficulty breathing, and chest pain. If one has aHUS, the
doctor will need to monitor 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, swellings in
arms or legs, and a drop in the platelet count.
SOLIRIS can cause serious side effects including serious
allergic reactions. Serious allergic reactions can happen during
one’s SOLIRIS infusion. Tell the doctor or nurse right away if one
gets any of these symptoms during the SOLIRIS infusion: chest pain,
trouble breathing or shortness of breath, swelling of the face,
tongue, or throat, and feeling faint or pass out. If one has an
allergic reaction to SOLIRIS, the 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 the 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 the 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 the nose or throat (nasopharyngitis); diarrhea; back
pain; dizziness; flu like symptoms (influenza) including fever,
headache, tiredness, cough, sore throat, and body aches; joint pain
(arthralgia); throat irritation (pharyngitis), and bruising
(contusion).
Please see the accompanying 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
therapies. 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) as well as the first
and only approved complement inhibitor to treat atypical hemolytic
uremic syndrome (aHUS), 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). In addition, the company is developing several
mid-to-late-stage therapies, including a second complement
inhibitor, a copper-binding agent for Wilson disease and an
anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G
(IgG)-mediated diseases as well as several early-stage therapies,
including one for light chain (AL) amyloidosis and a second
anti-FcRn therapy. 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, and metabolic disorders. Alexion has been named to the
Forbes’ list of the World’s Most Innovative Companies seven years
in a row and is headquartered in Boston, Massachusetts’ Innovation
District. The company also 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-G]
For patient or advocacy inquiries please contact
patientadvocacy@alexion.com.
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: the
data to be presented at ECTRIMS support the robust treatment effect
of SOLIRIS on relapse prevention and help to further characterize
the benefits of SOLIRIS for adult patients with anti-aquaporin-4
(AQP4) antibody-positive neuromyelitis optica spectrum disorder
(NMOSD), the potential approval of SOLIRIS as a treatment for NMOSD
in Japan; SOLIRIS can provide benefits for patients with NMOSD; and
the anticipated timing of the review and decision of regulatory
agencies with respect to the potential approval of SOLIRIS as a
treatment for NMOSD. 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 benefits of SOLIRIS for
NMOSD patients may not be realized (and the results of the clinical
trials may not be indicative of the results once approved for use
in the European Union); results of clinical trials may not be
sufficient to satisfy the European Commission or any other
regulatory authority in order to approve SOLIRIS as a treatment for
NMOSD (or they may request additional trials or additional
information); results in clinical trials may not be indicative of
results from later stage or larger clinical trials (or in broader
patient populations once the product is approved for use by
regulatory agencies); 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); 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 the European Commission and other regulatory agencies
regarding 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 and inter
partes review petitions submitted 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; uncertainties surrounding legal
proceedings, company investigations and government investigations,
including investigations of Alexion by the U.S. Securities and
Exchange Commission (SEC) and U.S. Department of Justice; 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 acquisition of Syntimmune 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 quarter ended June 30, 2019 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20190828005397/en/
Media Megan Goulart, 857-338-8634 Senior Director,
Corporate Communications Investors Susan Altschuller, Ph.D.,
857-338-8788 Vice President, Investor Relations
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