Comprehensive Positive Phase 3 Data for Alexion’s ALXN1210 in Patients with Paroxysmal Nocturnal Hemoglobinuria Presented at American Society of Hematology (ASH) Annual Meeting and Published in Blood
- 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® -
“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®,”
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,
The conference presentation of these previously announced data coincided with their peer-reviewed publication in Blood.2
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,
These 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
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,
(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,
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
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
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
Soliris® has received Orphan Drug Designation (ODD) for the treatment of
patients with PNH in the U.S., EU,
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
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.
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
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
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Alexion Pharmaceuticals, Inc.
Arne Naeveke, PhD, 857-338-8597
Susan Altschuller, PhD, 857-338-8788