- Accepted abstracts include interim Phase 2
extension data demonstrating reduced duration of infusion with
ULTOMIRIS® (ravulizumab-cwvz) 100 mg/mL formulation -
- Data from five abstracts demonstrate
Alexion’s continued commitment to advancing the understanding of
PNH with ongoing real-world studies and emerging clinical data
-
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
five abstracts have been accepted for presentation at the 25th
Congress of the European Hematology Association (EHA), which will
be held virtually from June 11 to 14, 2020. During the meeting,
results will be presented from an interim analysis of the Phase 2
ULTOMIRIS® (ravulizumab-cwvz) 100 mg/mL formulation open-label
study extension period. The 100 mg/mL formulation of ULTOMIRIS—a
long-acting C5 inhibitor administered every eight weeks offering
immediate, complete and sustained complement inhibition—was
evaluated to determine if participants with paroxysmal nocturnal
hemoglobinuria (PNH) could achieve greater convenience by reducing
infusion times, thus lessening demands on the healthcare
system.
“ULTOMIRIS is an important new standard of care for patients
with PNH and atypical hemolytic uremic syndrome,” said John Orloff,
M.D., Executive Vice President and Head of Research and Development
at Alexion. “An interim analysis of a PNH study evaluating the
higher concentration formulation showed a 78-minute reduction in
infusion time for adult patients in the 60-100kg cohort
(representative of the majority of patients treated for PNH), and
comparable safety, pharmacokinetics, and immunogenicity to the
current formulation. This would reduce the time patients spend
receiving their infusion—in clinic or in-home settings—lessening
the overall burden on healthcare systems. We look forward to the
opportunity to present these data during the virtually-held EHA
meeting.”
The U.S. Food and Drug Administration (FDA) is currently
reviewing the company’s supplemental biologics application for the
ULTOMIRIS 100mg/mL formulation for the treatment of atypical
hemolytic uremic syndrome (aHUS) and for adults with PNH. The
agency has set a Prescription Drug User Fee Act (PDUFA) target
action date of October 11, 2020. An application is also under
review with the European Medicines Agency. The interim analysis
that will be presented at EHA is from the extension period of the
Phase 2 study evaluating the higher concentration of ULTOMIRIS in
patients with PNH and is not inclusive of the aHUS patient
population.
Additional data being presented at the meeting include analyses
from the ongoing, observational real-world study of SOLIRIS®
(eculizumab) as a treatment for PNH, along with a study evaluating
the use of ALXN2040 (danicopan, formerly ACH-4471)—an
investigational, oral, factor D inhibitor—used as an add-on therapy
for the small, subgroup of PNH patients who are also diagnosed with
extravascular hemolysis.
The accepted abstracts are listed below and are now available on
the EHA website. All e-poster presentations will be available on
the EHA website for the duration of the Congress.
e-Poster Presentations
An Interim Analysis of A Phase 2 Study Evaluating The Efficacy,
Safety, and Pharmacokinetics of Intravenous Ravulizumab 100 mg/mL
Formulation in Patients with Paroxysmal Nocturnal Hemoglobinuria.
e-poster presentation, abstract ID#: EP862.
Development of A Composite Endpoint to Evaluate Treatment
Benefit in Patients with Paroxysmal Nocturnal Hemoglobinuria.
e-poster presentation, abstract ID#: EP845.
Effectiveness of Eculizumab Treatment in Patients Aged ≥65 Years
with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from The
International PNH Registry. e-poster presentation, abstract ID#:
EP853.
Use of Prophylactic Antibiotics in Patients with Paroxysmal
Nocturnal Hemoglobinuria Treated with Eculizumab. e-poster
presentation, abstract ID#: EP866.
Effects of Oral, Factor D Inhibitor Danicopan on Transfusion
Rates in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria
(PNH) Patients with A Sub-Optimal Response to Eculizumab: Phase 2
Study. E-poster presentation, abstract ID#: EP855.
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal nocturnal hemoglobinuria (PNH) is a serious ultra-rare
blood disorder with devastating consequences. It is characterized
by the destruction of red blood cells, which is also referred to as
hemolysis. PNH occurs when the complement system—a part of the
body’s immune system—over-responds, leading the body to attack its
own red blood cells. PNH often goes unrecognized, with delays in
diagnosis from one to more than five years. Patients with PNH may
experience a range of symptoms, such as fatigue, difficulty
swallowing, shortness of breath, abdominal pain, erectile
dysfunction, dark-colored urine and anemia. The most devastating
consequence of chronic hemolysis is the formation of blood clots,
which can occur in blood vessels throughout the body, damage vital
organs, and potentially lead to premature death. PNH can strike men
and women of all races, backgrounds and ages without warning, with
an average age of onset in the early 30s.
About Atypical Hemolytic Uremic Syndrome (aHUS) Atypical
HUS is an ultra-rare disease that can cause progressive injury to
vital organs, primarily the kidneys, via damage to the walls of
blood vessels and blood clots. aHUS occurs when the complement
system—a part of the body’s immune system—over-responds, leading
the body to attack its own healthy cells. aHUS can cause sudden
organ failure or a slow loss of function over time—potentially
resulting in the need for a transplant, and in some cases, death.
aHUS affects both adults and children, and many patients present in
critical condition, often requiring supportive care, including
dialysis, in an intensive care unit. The prognosis of aHUS can be
poor in many cases, so a timely and accurate diagnosis—in addition
to treatment—is critical to improving patient outcomes. Available
tests can help distinguish aHUS from other hemolytic diseases with
similar symptoms.
About ULTOMIRIS® (ravulizumab‑cwvz) ULTOMIRIS®
(ravulizumab-cwvz) is the first and only long-acting 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 complement cascade
over-responds, leading the body to attack its own healthy cells.
ULTOMIRIS is administered intravenously every eight weeks or every
four weeks for pediatric patients less than 20 kg, following a
loading dose. ULTOMIRIS is approved in the United States (U.S.),
European Union (EU) and Japan as a treatment for adults with
paroxysmal nocturnal hemoglobinuria (PNH) and in the U.S. for
atypical hemolytic uremic syndrome (aHUS) to inhibit
complement-mediated thrombotic microangiopathy (TMA) in adult and
pediatric (one month of age and older) patients. To learn more
about the regulatory status of ULTOMIRIS in the countries that we
serve, please visit www.alexion.com.
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 ULTOMIRIS®
(ravulizumab-cwvz) AND SOLIRIS® (eculizumab)
INDICATIONS ULTOMIRIS and SOLIRIS are prescription
medicines called monoclonal antibodies. ULTOMIRIS and SOLIRIS are
used to treat adults with a disease called Paroxysmal Nocturnal
Hemoglobinuria (PNH). ULTOMIRIS and SOLIRIS are also used to treat
adults and children with a disease called atypical Hemolytic Uremic
Syndrome (aHUS). Neither ULTOMIRIS nor SOLIRIS is for use in
treating people with Shiga toxin E. coli related hemolytic uremic
syndrome (STEC-HUS).
In addition, SOLIRIS is used to treat adults with a disease
called generalized myasthenia gravis (gMG) who are
anti-acetylcholine receptor (AChR) antibody positive. SOLIRIS is
also 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 ULTOMIRIS is safe and effective in children
with PNH or in children younger than one month of age in aHUS. It
is also not known if SOLIRIS is safe and effective in children with
PNH, gMG, or NMOSD.
IMPORTANT SAFETY INFORMATION ULTOMIRIS and SOLIRIS are
medicines that affect the immune system. These medicines can lower
the ability of the immune system to fight infections. Both
medicines increase 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 ULTOMIRIS or SOLIRIS if the patient has
not already had this vaccine. If the patient’s doctor decided that
urgent treatment is needed, meningococcal vaccination should be
administered as soon as possible. If the patient has not been
vaccinated and therapy must be initiated immediately, two weeks of
antibiotics should also be administered with the vaccinations. If
the patient had a meningococcal vaccine in the past, additional
vaccination might be needed before starting ULTOMIRIS or 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. Patients should be instructed to call their 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. The doctor will
provide a Patient Safety Card about the risk of meningococcal
infection. This card must be carried at all times during treatment
and for 8 months after the last ULTOMIRIS dose or 3 months after
the last SOLIRIS dose.
Before a patient can receive ULTOMIRIS or SOLIRIS, their doctor
must: enroll in the corresponding ULTOMIRIS REMS or SOLIRIS REMS
program; counsel the patient about the risk of meningococcal
infection; give the patient information and a Patient Safety Card
about the symptoms and risk of meningococcal infection (as
discussed above); and make sure that the patient is vaccinated with
a meningococcal vaccine.
ULTOMIRIS and SOLIRIS may also increase the risk of other types
of serious infections. Patients should talk to their doctor right
away if they have any new signs or symptoms of infection.
Patients must not receive ULTOMIRIS or SOLIRIS if they have a
meningococcal infection, or if they have not been vaccinated
against meningococcal infection, unless their doctor decides that
urgent treatment is needed.
Before a patient receives ULTOMIRIS or SOLIRIS, they should tell
their doctor about all of their medical conditions, including if
they: have an infection or fever, are pregnant or plan to become
pregnant, and are breastfeeding or plan to breastfeed. It is not
known if ULTOMIRIS or SOLIRIS will harm an unborn baby or if these
medicines pass into the breast milk.
Patients should tell their doctor about all the medicines they
take, including prescription and over-the-counter medicines,
vitamins, and herbal supplements. ULTOMIRIS and SOLIRIS can affect
how other medicines work, causing side effects.
For patients with PNH, the doctor will need to monitor the
patient closely for at least 16 weeks after stopping ULTOMIRIS or 8
weeks after stopping SOLIRIS. Stopping treatment with these
medicines may cause breakdown of the red blood cells due to PNH.
Symptoms or problems that can happen due to red blood cell
breakdown include: a drop in red blood cell count, tiredness, blood
in the urine, stomach-area (abdomen) pain, shortness of breath,
blood clots, trouble swallowing, and erectile dysfunction (ED) in
males.
For patients with aHUS, the doctor will need to monitor closely
during and for at least 12 months after stopping ULTOMIRIS, or 12
weeks after stopping SOLIRIS, for signs of worsening aHUS symptoms
or problems related to abnormal clotting and breakdown of red blood
cells called thrombotic microangiopathy (TMA). Symptoms or problems
that can happen with TMA may include: confusion or loss of
consciousness, seizures, chest pain (angina), difficulty breathing
and blood clots or stroke.
ULTOMIRIS can cause serious side effects including infusion
reactions. Symptoms of an infusion reaction with ULTOMIRIS may
include lower back pain, pain with the infusion, feeling faint or
discomfort in the arms or legs. Patients should tell their doctor
or nurse right away if they develop these symptoms, or any other
symptoms during their ULTOMIRIS infusion that may mean they are
having a serious infusion reaction, including: chest pain, trouble
breathing or shortness of breath, swelling of the face, tongue, or
throat, and feel faint or pass out.
The most common side effects of ULTOMIRIS in people treated for
PNH are upper respiratory infection and headache. The most common
side effects of ULTOMIRIS in people with aHUS are upper respiratory
infection, diarrhea, nausea, vomiting, headache, high blood
pressure and fever.
SOLIRIS can cause serious side effects including serious
allergic reactions. Serious allergic reactions can happen during
the SOLIRIS infusion. Patients should tell their doctor or nurse
right away if they get 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 a patient 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).
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
ULTOMIRIS or SOLIRIS. For more information, ask your doctor or
pharmacist. Call your doctor right away if you miss an ULTOMIRIS or
SOLIRIS infusion or for medical advice about side effects. You may
report side effects to FDA at 1-800-FDA-1088.
Please refer to the full U.S. Prescribing Information and
Medication Guide for ULTOMIRIS and SOLIRIS available via the links
below, including the BOXED WARNING regarding serious and
life-threatening meningococcal infections for both medicines.
ULTOMIRIS Full Prescribing Information and Medication
Guide
SOLIRIS Full Prescribing Information and Medication
Guide
About Alexion Alexion is a global biopharmaceutical
company focused on serving patients and families affected by rare
and devastating 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). 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: the anticipated benefits of the
ULTOMIRIS 100 mg/mL formulation (including to the patients and the
healthcare system); the Company’s commitment to advancing the
understanding of PNH with ongoing real-world studies and emerging
clinical data; and the anticipated timing of the review and
decision of regulatory agencies with respect to the potential
approval of certain of our product candidates. 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: ULTOMIRIS 100 mg/mL
formulation may not generate the expected benefits to patients or
the healthcare system that are anticipated; anticipated regulatory
approvals may be delayed or refused; results of clinical trials may
not be sufficient to satisfy regulatory authorities to approve
ULTOMIRIS 100 mg/mL formulation as a treatment for PNH and/or aHUS
(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); the
severity of the impact of the COVID-19 pandemic on Alexion’s
business, including on commercial and clinical trial 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 complement inhibitors; 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 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 products and product
candidates; uncertainty of long-term success in developing,
licensing or acquiring other product candidates or additional
indications for existing products; proposed acquisition of Portola
by Alexion may not be completed; 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 parties 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; 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 the
coronavirus; 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 restructurings;
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 March 31, 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20200514005533/en/
Alexion: Media Megan Goulart, 857-338-8634 Senior
Director, Corporate Communications
Investors Chris Stevo, 857-338-9309 Head of Investor
Relations
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