- ULTOMIRIS demonstrated consistent efficacy
and safety through 52 weeks, with no cases of breakthrough
hemolysis (BTH) associated with incomplete C5 complement inhibition
-
- Nearly all surveyed patients preferred
ULTOMIRIS over SOLIRIS® (eculizumab) -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the
presentation of new data demonstrating that ULTOMIRIS®
(ravulizumab), the first and only long-acting C5 complement
inhibitor administered every eight weeks, provided consistent
efficacy and safety through 52 weeks in the extension1 of the Phase
3 study of ULTOMIRIS and SOLIRIS® (eculizumab) in complement
inhibitor-naïve, adult patients with paroxysmal nocturnal
hemoglobinuria (PNH).2 Sustained efficacy of ULTOMIRIS was observed
on the co-primary endpoints of transfusion avoidance and
normalization of lactate dehydrogenase (LDH) levels and the
secondary endpoints of LDH level reduction and breakthrough
hemolysis (BTH). In an additional sub-study, nearly all patients
preferred ULTOMIRIS over SOLIRIS.3 The data will be presented at
the Annual Congress of the European Hematology Association (EHA),
taking place June 13-19, 2019 in Amsterdam, Netherlands.
LDH level normalization and reduction are direct markers for
reduced hemolysis in PNH, a severe, ultra-rare disease
characterized by complement-mediated intravascular hemolysis. PNH
can cause a wide range of debilitating symptoms and complications,
including thrombosis, which can occur throughout the body and
result in organ damage and premature death.4,5,6,7,8,9,10,11
Incomplete inhibition of the C5 complement protein can increase the
risk of BTH and related serious complications.12,13,14,15
“The confirmation of consistent efficacy and safety through 52
weeks with only six or seven infusions per year instead of 26 with
SOLIRIS makes ULTOMIRIS a very compelling new therapy for patients
with PNH,” said Professor Hubert Schrezenmeier, M.D., Medical
Director of the Institute of Transfusion Medicine and the Institute
for Clinical Transfusion Medicine and Immunogenetics, German Red
Cross Blood Transfusion Service Baden-Württemberg-Hessen and
University Hospital Ulm, Germany, study investigator and presenting
author. “I am impressed with the continuing complete C5 inhibition
in all patients receiving ULTOMIRIS and the absence of breakthrough
hemolysis associated with incomplete C5 inhibition. This makes me
hopeful that we can reduce the potentially devastating consequences
of returning PNH symptoms.”
All patients in the initial ULTOMIRIS group of the extension
study maintained complete C5 inhibition through 52 weeks, and no
patient experienced BTH associated with incomplete C5 inhibition.
All patients who had experienced incomplete C5 inhibition while
receiving SOLIRIS in the first 26 weeks achieved complete C5
inhibition after the switch to ULTOMIRIS in the extension phase. No
patient experienced BTH associated with incomplete C5 inhibition
between weeks 27 and 52 after switching to ULTOMIRIS compared to
six percent while receiving SOLIRIS in the first 26 weeks.1
“We continue to expand the body of clinical evidence supporting
the potential of ULTOMIRIS to become the new standard of care for
patients with PNH,” said John Orloff, M.D., Executive Vice
President and Head of Research & Development at Alexion.
“SOLIRIS, the first approved therapy for PNH, was a breakthrough
for patients for whom only supportive care had been available
before. With ULTOMIRIS, we want to enable patients to live their
lives more freely thanks to maximal hemolysis control with
established safety and reduced treatment burden.”
The most common adverse events occurred less frequently in the
extension phase than during the initial treatment phase where the
safety profile of ULTOMIRIS was consistent with that of SOLIRIS.2
The most common treatment-emergent adverse events in the extension
phase were upper respiratory tract infection (in the initial
ULTOMIRIS arm) and nasopharyngitis (in the initial SOLIRIS arm).
The most frequently observed serious adverse event was pyrexia. One
patient in the initial SOLIRIS arm died from lung cancer (unrelated
to SOLIRIS treatment). There was no case of meningococcal infection
observed.1,2
ULTOMIRIS was studied in the largest-ever Phase 3 program in
PNH. The entire clinical development program for ULTOMIRIS in PNH
to date represents more than 800 patient years of experience.
Additional data to be presented at the EHA congress indicate a
very strong patient preference for ULTOMIRIS over SOLIRIS.3
According to results from a sub-study in the ULTOMIRIS Phase 3
extension in patients who had been stable on SOLIRIS before,16
nearly all patients (93%) preferred ULTOMIRIS due to reduced
infusion frequency, ability to plan activities, overall quality of
life, convenience of treatment, and effectiveness of medication
until the next infusion compared to SOLIRIS.3
New results from the International PNH Registry will also be
presented at the EHA congress. These data suggest that a change in
clone size does not change the increased risk of major adverse
vascular events in PNH,17 and that complement inhibition with
SOLIRIS does not change the effectiveness of concomitant
immunosuppressive therapy in patients with PNH and aplastic anemia
(AA).18
The U.S. Food and Drug Administration (FDA) approved ULTOMIRIS
for adult patients with PNH on December 21, 2018. The European
Commission (EC) is reviewing the recommendation by the European
Medicines Agency’s (EMA) Committee for Medicinal Products for Human
Use (CHMP) from April 26, 2019 to approve ULTOMIRIS as a treatment
for adult patients with PNH and typically delivers its final
decision within two months. The Japanese Ministry of Health, Labour
and Welfare (MHLW) is reviewing the recommendation by the
Pharmaceuticals and Medical Devices Agency’s (PMDA) Drug Committee
(BUKAI) to approve ULTOMIRIS as a treatment for patients with PNH
and is anticipated to deliver a decision in late June.
One-Year Efficacy of Ravulizumab (ALXN1210) in Adult Patients
with Paroxysmal Nocturnal Hemoglobinuria Naive to Complement
Inhibitors, EHA Congress, June 13-16, 2019 Amsterdam, Netherlands,
oral presentation, June 15, 2019, 12:00 p.m., abstract S863.1
Patient Preferences for the Treatment of Paroxysmal Nocturnal
Hemoglobinuria: Results of a Patient Survey of Ravulizumab
(ALXN1210) and Eculizumab, EHA Congress, June 13-16, 2019
Amsterdam, Netherlands, poster presentation, June 14, 2019, 5:30
p.m., abstract PF734.3
Change in Clone Size Does Not Predict Risk of Major Adverse
Vascular Events: Results from the International PNH Registry, EHA
Congress, June 13-16, 2019 Amsterdam, Netherlands, oral
presentation, June 15, 2019, 12:30 p.m., abstract S865.17
No Change in the Effectiveness of Immunosuppressive Therapy in
Patients with PNH and AA Receiving concomitant Eculizumab, EHA
Congress, June 13-16, 2019 Amsterdam, Netherlands, poster
presentation, June 15, 2019, 5:30 p.m., abstract PS1117.18
About the extension1 of the Phase 3 study in
complement-naïve, adult patients with PNH2At the end of
the 26-week Phase 3 study,2 all patients (246; 125 on ULTOMIRIS,
121 on SOLIRIS) had the option to receive ULTOMIRIS every eight
weeks for up to two years. The aim of this extension study is to
monitor the durability of efficacy in the initial ULTOMIRIS group,
the efficacy of ULTOMIRIS in the initial SOLIRIS group after the
switch to ULTOMIRIS and the safety of ULTOMIRIS in all patients. A
total of 243 patients (124 from the initial ULTOMIRIS group, 119
from the initial SOLIRIS group) were followed. Results for the
co-primary endpoints of transfusion avoidance and LDH level
normalization and the secondary endpoints of percentage change from
baseline in LDH levels and proportion of patients with BTH are
provided descriptively. Complete C5 inhibition was defined as
plasma levels of free C5 ≤0.5 μg/ml.1
About the patient preference study3At the end of
the 26-week Phase 3 study in adult patients with PNH who had been
clinically stable on SOLIRIS for at least 6 months,16 all patients
(195) were given the option to continue receiving ULTOMIRIS every
eight weeks for up to two years. The patient preference study
enrolled patients from the extension study who had received at
least two doses of ULTOMIRIS. Patient treatment preference was
evaluated at one time point using an 11-item PNH-specific Patient
Preference Questionnaire (PNH-PPQ©). The 11 questions included one
question on overall treatment preference, nine questions on
preference based on treatment characteristics, one question on the
most important treatment characteristic for the overall treatment
preference, four questions on aspects of treatment with SOLIRIS and
four matching questions for ULTOMIRIS. Of 98 patients enrolled, 95
patients from eight countries (European Union, North America, and
Australia) completed PNH-PPQs per protocol.3
About Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal
nocturnal hemoglobinuria (PNH) is a chronic, progressive,
debilitating and life-threatening ultra-rare blood disorder
characterized by intravascular hemolysis (destruction of red blood
cells) that is mediated by an uncontrolled activation of the
complement system, a part of the immune system.4,5,19 PNH can
strike men and women of all races, backgrounds and ages without
warning, with an average age of onset in the early 30s.4,20 PNH
often goes unrecognized, with delays in diagnosis ranging from one
to more than five years.21 Patients with PNH may experience a wide
range of signs and symptoms, such as fatigue, difficulty
swallowing, shortness of breath, abdominal pain, erectile
dysfunction, dark-colored urine and anemia.6,7,8,9,10,11,19 The
most devastating consequence of chronic intravascular hemolysis is
thrombosis, which can occur in blood vessels throughout the body,
damage vital organs and cause premature death.22 The first
thrombotic event can be fatal.4,20,23 Despite historical supportive
care, including transfusion and anticoagulation management, 20 to
35 percent of patients with PNH die within five to 10 years of
diagnosis.24,25 Patients with certain types of hemolytic anemia,
bone marrow disorders and unexplained venous or arterial thrombosis
are at increased risk of PNH.14,19,26,27,28,29
About ULTOMIRIS®ULTOMIRIS (ravulizumab-cwvz), the
first and only long-acting C5 inhibitor administered every eight
weeks, is approved in the U.S. as a treatment for adults with
paroxysmal nocturnal hemoglobinuria (PNH). ULTOMIRIS works by
inhibiting the C5 protein in the terminal complement cascade, a
part of the body’s immune system. The terminal complement cascade,
when activated in an uncontrolled manner, plays a role in severe
ultra-rare disorders like PNH, atypical hemolytic uremic syndrome
(aHUS), anti-acetylcholine receptor (AchR) antibody-positive
myasthenia gravis (MG) and anti-aquaporin-4 (AQP4)
auto-antibody-positive neuromyelitis optica spectrum disorder
(NMOSD). Regulatory authorities in the European Union (EU) and
Japan are reviewing applications for the approval of ULTOMIRIS as a
treatment for adult patients and patients with PNH, respectively.
In Phase 3 clinical studies in complement inhibitor-naïve patients
with PNH2 and patients with PNH who had been stable on SOLIRIS®
(eculizumab),16 intravenous treatment with ULTOMIRIS every eight
weeks demonstrated non-inferiority to intravenous treatment with
SOLIRIS every two weeks on all endpoints.
The Phase 3 study of ULTOMIRIS, administered intravenously every
eight weeks in adult patients with aHUS, met its primary objective.
Alexion has submitted a supplemental Biologics License Application
(sBLA) to the U.S. Food and Drug Administration (FDA) for approval
of ULTOMIRIS as a treatment for patients with aHUS and plans to
submit similar applications in the EU and Japan later in 2019.
ULTOMIRIS is also currently being evaluated in a Phase 3 clinical
study in children and adolescents with aHUS, administered
intravenously every eight weeks. Alexion has initiated a Phase 3
study of ULTOMIRIS, intravenously administered every eight weeks,
as a potential treatment for patients with generalized MG (gMG),
and is planning to initiate a Phase 3 in patients with NMOSD. In
addition, Alexion has initiated Phase 3 studies of ULTOMIRIS
delivered subcutaneously once per week as a potential treatment for
patients with PNH and aHUS.
ULTOMIRIS has received Orphan Drug Designation (ODD) for the
treatment of patients with PNH in the U.S. and Japan and for the
subcutaneous treatment of patients with aHUS in the U.S.
U.S. Indication of ULTOMIRIS®ULTOMIRIS
(ravulizumab-cwvz) is a prescription medicine called a monoclonal
antibody. ULTOMIRIS is used to treat adults with a disease called
paroxysmal nocturnal hemoglobinuria (PNH). It is not known if
ULTOMIRIS is safe and effective in children.
U.S. Important Safety Information for
ULTOMIRIS®ULTOMIRIS (ravulizumab-cwvz) is a medicine
that affects the immune system. ULTOMIRIS can lower the ability of
the immune system to fight infections. ULTOMIRIS 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 2 weeks before
the first dose of ULTOMIRIS if one has not already had this
vaccine. If one’s doctor decided that urgent treatment with
ULTOMIRIS is needed, meningococcal vaccination should be
administered as soon as possible. If one has not been vaccinated
and ULTOMIRIS therapy must be initiated immediately, 2 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 ULTOMIRIS. 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 with a stiff neck or stiff back, fever and a
rash, muscle aches with flu-like symptoms, headache and fever,
fever, confusion, and eyes sensitive to light.
ULTOMIRIS is only available through a program called the
ULTOMIRIS REMS.
ULTOMIRIS may also increase the risk of other types of serious
infections. People who take ULTOMIRIS may have an increased risk of
getting infections caused by Streptococcus pneumoniae and
Haemophilus influenzae. Certain people may also have an increased
risk of gonorrhea infection. To find out if one is at risk for
gonorrhea infection, about gonorrhea prevention, and regular
testing, talk to the healthcare provider. Call the healthcare
provider right away if one has any new signs or symptoms of
infection.
Before one receives ULTOMIRIS, 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 ULTOMIRIS will harm an unborn
baby. It is not known if ULTOMIRIS passes into the breast milk. One
should not breast feed during treatment and for 8 months after
one’s final dose of ULTOMIRIS.
Tell the doctor about all the medicines one takes, including
prescription and over-the-counter medicines, vitamins, and herbal
supplements. ULTOMIRIS and other medicines can affect each other
causing side effects. 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 stops receiving ULTOMIRIS, the doctor will need to
monitor closely for at least 16 weeks after one stops ULTOMIRIS.
Stopping ULTOMIRIS 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,
tiredness, blood in the urine, stomach-area (abdomen) pain, blood
clots, shortness of breath, trouble swallowing, and erectile
dysfunction (ED) in males.
ULTOMIRIS can cause serious side effects including infusion
reactions. Infusion reactions may happen during one’s ULTOMIRIS
infusion. Symptoms of an infusion reaction with ULTOMIRIS may
include lower back pain, pain with the infusion, or feeling faint.
Tell the doctor or nurse right away if these symptoms develop, or
any other symptoms during the ULTOMIRIS infusion that may mean one
is 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. One’s doctor will
treat the symptoms as needed. The most common side effects of
ULTOMIRIS are upper respiratory infection and headache.
For more information, please see the full U.S. Prescribing
Information and Medication Guide for ULTOMIRIS, including Boxed
WARNING regarding serious and life-threatening meningococcal
infections/sepsis, also available at: www.ultomiris.com.
U.S. Indication for SOLIRIS®SOLIRIS (eculizumab)
is a prescription medicine called a monoclonal antibody. SOLIRIS is
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]), and adults with a disease called
generalized Myasthenia Gravis (gMG) who are anti-acetylcholine
receptor (AchR) antibody positive. It is not known if SOLIRIS is
safe and effective in children with PNH or gMG.
U.S. Important Safety Information for
SOLIRIS®SOLIRIS (eculizumab) 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 2 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, 2 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. 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.
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.
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.
It is not known 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 for at
least 12 weeks after stopping SOLIRIS 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 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.
Please see the accompanying full U.S. Prescribing Information
and Medication Guide for SOLIRIS, including Boxed WARNING regarding
serious and life-threatening meningococcal infections, also
available at: www.soliris.net.
About AlexionAlexion 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) and anti-acetylcholine
receptor (AchR) antibody-positive generalized myasthenia gravis
(gMG), and is also developing it for patients with 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]
Forward-Looking StatementThis 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 efficacy and safety of
ULTOMIRIS as a treatment of PNH; that the Phase 3 extension study
data confirm consistent efficacy and safety of ULTOMIRIS; that six
or seven infusions per year instead of 26 with SOLIRIS make
ULTOMIRIS a very compelling new therapy for patients with PNH; that
patients prefer ULTOMIRIS over SOLIRIS and that ULTOMIRIS results
in a reduced treatment burden for patients as compared to SOLIRIS;
ULTOMIRIS has the potential to become the new standard of care for
patients with PNH; the expected timing of the final decision of the
EC with respect to pharmaceutical product approval including
ULTOMIRIS for PNH; Alexion’s future plans for submitting
supplemental Biologics License Application and similar applications
to the applicable regulatory authorities for ULTOMIRIS as a therapy
for certain indications; ULTOMIRIS is a potential treatment for
patients with gMG and NMOSD; the company is planning to initiate a
Phase 3 clinical trial in patients with NMOSD; the anticipated
timing of the review and decision of regulatory agencies with
respect to the potential approval of ULTOMIRIS as a treatment for
PNH in certain jurisdictions; future plans for the evaluation and
clinical trials of ULTOMIRIS in additional indications and patient
populations, including subcutaneous administration; and the
potential medical benefits of ULTOMIRIS for the treatment of PNH
and other diseases. 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: our inability to facilitate the timely
conversion of patients with PNH (and any future indications) from
SOLIRIS to ULTOMIRIS; payer, physician and patient acceptance of
ULTOMIRIS as an alternative to SOLIRIS; ULTOMIRIS does not gain
market acceptance and/or does not become the standard of care for
patients with PNH and/or is not recognized by patients and
physicians as the standard of care for patients with PNH; the
benefits (including safety and efficacy) of ULTOMIRIS evidenced in
clinical trials are not witnessed in a broader patient population;
any potential post-approval restrictions that the FDA or any other
regulatory agency may impose on ULTOMIRIS; ULTOMIRIS does not gain
regulatory approval from the EC or Japanese MHLW as a treatment for
PNH; ULTOMIRIS does not gain approval from regulatory agencies as a
treatment for indications beyond PNH, including aHUS; 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 (including ULTOMIRIS as a treatment for
PNH); 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)
future clinical trials due to safety issues, Institutional Review
Board (IRB) decisions, Chemistry, Manufacturing, and Controls
(CMC)-related issues, expense or unfavorable results from earlier
trials (among others); our dependence on sales from our principal
product (SOLIRIS); future competition from biosimilars and other
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 the FDA and other regulatory agencies; results in early
stage clinical trials may not be indicative of full results or
results from later stage or larger clinical trials (or broader
patient populations) and do not ensure regulatory approval; 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 halt trials, delay or prevent us from making regulatory
approval filings or result in denial of approval of our product
candidates; future product improvements may not be realized due to
expense or feasibility; uncertainty of long-term success in
developing, licensing or acquiring other product candidates or
additional indications for existing products; the possibility that
current rates of adoption of SOLIRIS in PNH, aHUS, gMG or other
diseases (and ULTOMIRIS in PNH in the U.S.) 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 and challenges against us
(including intellectual property lawsuits relating to ULTOMIRIS
brought by third parties against Alexion and inter partes review
petitions submitted by third parties); the risk that third party
payers (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; 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 (including intellectual property suits initiated
against Alexion and our products), 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
future 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
period ended March 31, 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.
References_______________________1 Schrezenmeier
H, Kulasekararaj AG, Mitchell L et al. 24th Congress of the
European Hematology Association (EHA), June 13-16, 2019 Amsterdam,
Netherlands, oral presentation, June 15, 2019, abstract S863.2 Lee
JW, Sicre de Fontbrune F, Lee LWL et al. Blood. December 3,
2018;doi:10.1182/blood-2018-09-876136.3 Peipert JD, Kulasekararaj
AG, Gaya A et al. 24th Congress of the European Hematology
Association (EHA), June 13-16, 2019 Amsterdam, Netherlands, poster
presentation, June 14, 2019, abstract PF734.4 Hill A, Richards SJ,
Hillmen P. Br J Haematol. 2007 May;137(3):181-92.5 Hillmen P, Lewis
SM, Bessler M, et al. N Engl J Med. 1995 Nov 9;333(19):1253-8.6
Schrezenmeier H, Muus P, Socié G, et al. Haematologica.
2014;99:922-929.7 Brodsky RA. Blood Rev. 2008;22:65-74.8 Weitz I,
Meyers G, Lamy T, et al. Intern Med J. 2013;43:298-307.9 Lee JW,
Jang JH, Kim JS, et al. Int J Hematol. 2013;97:749-757.10 Dacie JV,
Lewis SM. Ser Haemat. 1972;5:3-23.11 Nishimura J, Kanakura Y, Ware
RE, et al. Medicine (Baltimore) 2004 May;83(3):193-207.12
ULTOMIRIS® [package insert]. Boston: Alexion Pharmaceuticals Inc.;
201813 Nakayama H, Usuki K, Echizenet H, et al. Biol Pharm Bull.
2016;39:285-288.14 Hill A, Kelly RJ, Hillmen P. Blood.
2013;121(25):4985-4996.15 Peffault de Latour R, Fremeaux-Bacchi V,
Porcher R, et al. Blood. 2015;125:775-783.16 Kulasekararaj AG, Hill
A, Rottinghaus ST et al. Blood. doi:10.1182/blood-2018-09-876805.17
Peffault de Latour r, Kulasekararaj AG, Larratt L et al. 24th
Congress of the European Hematology Association (EHA), June 13-16,
2019 Amsterdam, Netherlands, poster presentation, June 15, 2019,
abstract S865.18 Hill A, Peffault de Latour R, Kulasekararaj AG 3
et al. 24th Congress of the European Hematology Association (EHA),
June 13-16, 2019 Amsterdam, Netherlands, poster presentation, June
15, 2019, abstract PS1117.19 Parker C, Omine M, Richards S, et al.
Blood. 2005 Dec;106(12):3699-3709.20 Socié G, Mary JY, de Gramont
A, et al. Lancet. 1996;348:573-577.21 Shammo JM, Mitchell RL,
Ogborn K et al. Blood. 2015;126:3264.22 Hillmen P, Muus P, Duhrsen
U, et al. Blood. 2007 Dec;110(12):4123-8.23 Hillmen P, Elebute MO,
Kelly R, et al. Blood. 2007;110: Abstract 3678.24 Hillmen P, Muus
P, R�th A, et al. Br J Haematol. 2013;162:62-73.25 Loschi M,
Porcher R, Barraco F, et al. Am J Hematol. 2016;91:366-370.26
Borowitz MJ, Craig FE, DiGiuseppe JA, et al. Cytometry B Clin
Cytom. 2010;78B:211-230.27 Rachidi S, Musallam KM, Taher AT. Eur J
Intern Med. 2010;21:260-267.28 Morado M, Freire Sanders A, Colado E
et al. Cytometry Part B (Clinical Cytometry). 2017;92B:361-370.29
Sharma VR. Clin Adv Hematol Oncol. 2013;11(suppl 13):1-11.
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Alexion Pharmaceuticals, Inc.MediaArne Naeveke, PhD, +1
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Altschuller, PhD, +1 857-338-8788
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