- First Conference Presentation of Results for
ALXN1210 in Patients on Soliris® (Eculizumab) at ASH -
- Publications in Blood of Results for ALXN1210
in Complement Inhibitor-Naïve Patients and Patients on Soliris®
-
- Presentation at ASH of New Results from
Sensitivity Analyses in Inhibitor-Naïve Patients, and Analyses of
C5 Inhibition and Breakthrough Hemolysis in Inhibitor-Naïve
Patients and Patients on Soliris® -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the
presentation of comprehensive positive Phase 3 data for ALXN1210,
the company’s investigational long-acting C5 complement inhibitor,
at the American Society of Hematology (ASH) Annual Meeting, taking
place December 1-4, 2018. The presentations included both
previously announced and new data from the two large Phase 3
studies in patients with paroxysmal nocturnal hemoglobinuria (PNH)
who had either never been treated with a complement inhibitor
before or who had been stable on Soliris® treatment. The conference
presentations coincided with publications in Blood of the positive
results on all primary and key secondary endpoints from these two
studies.
“We are excited by the increasing body of data from our two
active comparator-controlled Phase 3 studies, the largest PNH Phase
3 program ever conducted, on clinically meaningful endpoints in
this devastating and potentially life-threatening disease. We are
particularly pleased by the positive data in patients converting to
ALXN1210 from Soliris®,” said John Orloff, M.D., Executive Vice
President and Head of Research & Development at Alexion. “Our
ambition is to make ALXN1210 the new standard of care for patients
with PNH.”
Results from a Phase 3, Multicenter, Non-Inferiority Study of
Ravulizumab (ALXN1210) Versus Eculizumab in Adult Patients with
Paroxysmal Nocturnal Hemoglobinuria Currently Treated with
Eculizumab – ASH, Session 101, Oral Presentation 625,
Abstract ID# 119147, December 3, 20181 The conference
presentation of these previously announced data coincided with
their peer-reviewed publication in Blood.2
A Phase 3 Study of Ravulizumab (ALXN1210) versus Eculizumab
in Adults with Paroxysmal Nocturnal Hemoglobinuria Naïve to
Complement Inhibitors: Results of a Subgroup Analysis with Patients
Stratified by Baseline Hemolysis Level, Transfusion History, and
Demographics – ASH, Session 101, Oral Presentation 627,
Abstract ID# 110623, December 3, 20183These new results add to
previously announced results on the co-primary and key secondary
endpoints of this study, which have now also been published in
Blood.4
A Prospective Analysis of Breakthrough Hemolysis in 2 Phase 3
Randomized Studies of Ravulizumab (ALXN1210) versus Eculizumab in
Adults with Paroxysmal Nocturnal Hemoglobinuria – ASH, Oral
and Poster Abstracts, Poster 2330, Abstract ID# 110874, December 2,
20185
Ravulizumab (ALXN1210) versus Eculizumab in Adults with
Paroxysmal Nocturnal Hemoglobinuria: Pharmacokinetics and
Pharmacodynamics Observed in Two Phase 3 Randomized, Multicenter
Studies – ASH Session 101, Oral Presentation 626, Abstract
ID# 110858, December 3, 20186
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic,
progressive, debilitating and life-threatening ultra-rare blood
disorder characterized by hemolysis (destruction of red blood
cells) that is mediated by an uncontrolled activation of the
complement system, a component of the body’s immune system.7,8,9
PNH can strike men and women of all races, backgrounds and ages
without warning, with an average age of onset in the early 30s.7,10
PNH often goes unrecognized, with delays in diagnosis ranging from
one to more than five years.11 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.9,12,13,14,15,16,17 The
most devastating consequence of chronic hemolysis is thrombosis,
which can occur in blood vessels throughout the body, damage vital
organs and cause premature death.18 The first thrombotic event can
be fatal.8,10,19 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.20,21
Patients with certain types of hemolytic anemia, bone marrow
disorders and unexplained venous or arterial thrombosis are at
increased risk of PNH.9,22,23,24,25,26
About ALXN1210
ALXN1210 is an innovative, investigational, long-acting C5
inhibitor discovered and developed by Alexion that works by
inhibiting the C5 protein in the terminal complement cascade, a
part of the body’s immune system that, when activated in an
uncontrolled manner, plays a role in severe ultra-rare disorders
like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic
uremic syndrome (aHUS), and anti-acetylcholine receptor (AchR)
antibody-positive myasthenia gravis (MG). In Phase 3 clinical
studies in complement inhibitor-naïve patients with PNH, and
patients with PNH who had been stable on Soliris®, intravenous
treatment with ALXN1210 every eight weeks demonstrated
non-inferiority to intravenous treatment with Soliris® every two
weeks, with numeric results for all primary and key secondary
endpoints favoring ALXN1210. ALXN1210 is also currently being
evaluated in a Phase 3 clinical study in complement inhibitor-naïve
patients with aHUS, administered intravenously every eight weeks.
In addition, Alexion plans to initiate a Phase 3 clinical study of
ALXN1210 delivered subcutaneously once per week as a potential
treatment for patients with PNH and aHUS. Alexion is also planning
to initiate the development of ALXN1210, intravenously administered
every eight weeks, as a potential treatment for patients with
generalized MG (gMG).
ALXN1210 has received Orphan Drug Designation (ODD) for the
treatment of patients with PNH in the U.S., EU, and Japan, and for
the subcutaneous treatment of patients with aHUS in the U.S.
About Soliris® (eculizumab)
Soliris® 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 that, when activated in an
uncontrolled manner, plays a role in severe rare and ultra-rare
disorders like paroxysmal nocturnal hemoglobinuria (PNH), atypical
hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor
(AchR) antibody-positive myasthenia gravis (MG). Soliris® is
approved in the U.S., EU, Japan, and other countries as the first
and only treatment for patients with PNH and aHUS, in the EU as the
first and only treatment of refractory generalized MG (gMG) in
adults who are anti-AchR antibody-positive, in the U.S. for the
treatment of adult patients with gMG who are anti-AchR
antibody-positive, and in Japan for the treatment of patients with
gMG who are AChR antibody-positive and whose symptoms are difficult
to control with high-dose intravenous immunoglobulin (IVIG) therapy
or plasmapheresis (PLEX). Soliris® is not indicated for the
treatment of patients with Shiga-toxin E. coli-related hemolytic
uremic syndrome (STEC-HUS).
Soliris® has received Orphan Drug Designation (ODD) for the
treatment of patients with PNH in the U.S., EU, Japan, and many
other countries, for the treatment of patients with aHUS in the
U.S., EU, and many other countries, for the treatment of patients
with MG in the U.S. and EU, for the treatment of patients with
refractory gMG in Japan, and for the treatment of patients with
neuromyelitis optica spectrum disorder (NMOSD) in the U.S., EU, and
Japan. Alexion and Soliris® have received some of the
pharmaceutical industry's highest honors for the medical innovation
in complement inhibition: the Prix Galien USA (2008, Best
Biotechnology Product) and France (2009, Rare Disease
Treatment).
For more information on Soliris®, please see full prescribing
information for Soliris®, including BOXED WARNING regarding risk of
serious meningococcal infection, available at www.soliris.net.
Important Soliris® Safety Information
The U.S. prescribing information for Soliris® includes the
following warnings and precautions: Life-threatening and fatal
meningococcal infections have occurred in patients treated with
Soliris®. Meningococcal infection may become rapidly
life-threatening or fatal if not recognized and treated early.
Comply with the most current Centers for Disease Control (CDC)’s
Advisory Committee on Immunization Practices (ACIP) recommendations
for meningococcal vaccination in patients with complement
deficiencies. Immunize patients with meningococcal vaccines at
least two weeks prior to administering the first dose of Soliris®,
unless the risks of delaying Soliris® therapy outweigh the risk of
developing a meningococcal infection. Monitor patients for early
signs of meningococcal infections and evaluate immediately if
infection is suspected. Soliris® is available only through a
restricted program under a Risk Evaluation and Mitigation Strategy
(REMS). Under the Soliris® REMS, prescribers must enroll in the
program. Enrollment in the Soliris® REMS program and additional
information are available by telephone: 1-888-SOLIRIS
(1-888-765-4747) or at www.solirisrems.com.
Patients may have increased susceptibility to infections,
especially with encapsulated bacteria. Aspergillus infections have
occurred in immunocompromised and neutropenic patients. Children
treated with Soliris® may be at increased risk of developing
serious infections due to Streptococcus pneumoniae and Haemophilus
influenza type b (Hib). Soliris® treatment of patients with PNH
should not alter anticoagulant management because the effect of
withdrawal of anticoagulant therapy during Soliris® treatment has
not been established. Administration of Soliris® may result in
infusion reactions, including anaphylaxis or other hypersensitivity
reactions.
In patients with PNH, the most frequently reported adverse
events observed with Soliris® treatment in clinical studies were
headache, nasopharyngitis, back pain, and nausea. In patients with
aHUS, the most frequently reported adverse events observed with
Soliris® treatment in clinical studies were headache, diarrhea,
hypertension, upper respiratory infection, abdominal pain,
vomiting, nasopharyngitis, anemia, cough, peripheral edema, nausea,
urinary tract infections, and pyrexia. In patients with gMG who are
anti-AchR antibody-positive, the most frequently reported adverse
reaction observed with Soliris® treatment in the placebo-controlled
clinical study (≥10%) was musculoskeletal pain.
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 the first and only approved complement inhibitor to
treat patients with paroxysmal nocturnal hemoglobinuria (PNH),
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. 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 Statement
This press release contains “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of 1995
that involve risks and uncertainties relating to future events and
the future performance of Alexion, including statements related to:
the Company’s ambition to make ALXN1210 the new standard of care
for patients with PNH; Alexion plans to initiate a Phase 3 clinical
study of ALXN1210 delivered subcutaneously once per week as a
potential treatment for patients with PNH and aHUS; Alexion is
planning to initiate the development of ALXN1210, intravenously
administered every eight weeks, as a potential treatment for
patients with generalized MG (gMG); the Company is developing a
complement inhibitor for patients with neuromyelitis optica
spectrum disorder (NMOSD); future plans to initiate a clinical
studies of ALXN1210 delivered subcutaneously once per week as a
potential treatment for patients with PNH and for studies of
ALXN1210 for other indications; and the potential medical benefits
of ALXN1210 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 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 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 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; unexpected delays in
clinical trials; 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; inability to complete
planned acquisitions due to failure of regulatory approval or
material changes in the target or otherwise; inability to complete
acquisitions and investments due to increased competition for
technology; the possibility that current rates of adoption of
Soliris® in PNH, aHUS, gMG or other diseases 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;
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; the possibility that expected tax benefits will not
be realized; assessment of impact of recent accounting
pronouncements; 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, 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 September 30, 2018 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
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2018;Session 101:625
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online ahead of print December 3, 2018]. Blood.
doi:10.1182/blood-2018-09-876805
3 Weitz IC, Kulagin AD, Nakao S et al. American Society of
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Alexion Pharmaceuticals, Inc.MediaArne Naeveke, PhD,
857-338-8597OrInvestorsSusan Altschuller, PhD, 857-338-8788
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