Data from the International PNH Registry Also to Be Presented at the ASH Meeting
Abstracts summarizing these presentations can be accessed on the ASH
website. The data will be presented in a poster session on
Abstract 3482: "Optimization of Dose Regimen for ALXN1210, a Novel
Complement C5 Inhibitor, in Patients with Paroxysmal Nocturnal
Hemoglobinuria (PNH): Results of 2 Phase 1/2 Studies," Roeth A,
Rottinghaus T, Hill A, et al.
Accessible at: https://ash.confex.com/ash/2017/webprogram/Paper100715.html
Abstract 3486: "Interim Analysis of Safety Outcomes During Treatment
With Eculizumab: Results from the International Paroxysmal Nocturnal
Hemoglobinuria Registry," Hill A, Roeth A, Socié G, et al.
Accessible at: https://ash.confex.com/ash/2017/webprogram/Paper101230.html
Abstract 3487: "Efficacy of Eculizumab in Patients with Paroxysmal
Nocturnal Hemoglobinuria (PNH) and High Disease Activity with or
Without History of Aplastic Anemia in the International PNH Registry,"
Lee JW, Peffault de Latour R, Brodsky RA, et al.
Accessible at: https://ash.confex.com/ash/2017/webprogram/Paper101237.html
Abstract 3488: "Analysis of Baseline Clinical Characteristics and
Disease Burden in Patients Enrolled in the International Paroxysmal
Nocturnal Hemoglobinuria Registry," Schrezenmeier H, Maciejewski JP,
Roeth A, et al.
Accessible at: https://ash.confex.com/ash/2017/webprogram/Paper101263.html
Abstract 3472: "Comparison of Baseline Clinical Characteristics
between Asian and non-Asian Patients with Paroxysmal Nocturnal
Hemoglobinuria (PNH) from the International PNH Registry," Jang JH,
Okamoto S, Sakurai M, et al.
Accessible at: https://ash.confex.com/ash/2017/webprogram/Paper106034.html
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Nocturnal Hemoglobinuria (PNH) is a chronic, progressive, debilitating and potentially life-threatening ultra-rare blood disorder that can strike men and women of all races, backgrounds and ages without warning, with an average age of onset in the early 30s.1,2,3 PNH often goes unrecognized, with delays in diagnosis ranging from one to more than 10 years.2 In patients with PNH, chronic, uncontrolled activation of the complement system, a component of the body's immune system, results in hemolysis (the destruction of red blood cells)4, which in turn can result in progressive anemia, fatigue, dark urine and shortness of breath.5,6,7 The most devastating consequence of chronic hemolysis is thrombosis (the formation of blood clots), which can damage vital organs and cause premature death.8 Historically, it had been estimated that one in three patients with PNH did not survive more than five years from the time of diagnosis.2 PNH is more common among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndromes (MDS).9,10,11 In certain patients with thrombosis of unknown origin, PNH may be an underlying cause.12
ALXN1210 is an innovative, long-acting anti-C5 antibody discovered and
ALXN1210 has received Orphan Drug Designation (ODD) for the intravenous
treatment of patients with PNH in the
About Soliris® (eculizumab)
Soliris® is a first-in-class complement inhibitor that works by
inhibiting the terminal part of the complement cascade, a part of the
immune system that, when activated in an uncontrolled manner, plays a
role in serious 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
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
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.
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.
This press release contains forward-looking statements, including
statements related to the potential medical benefits of ALXN1210 for the
treatment of PNH. Forward-looking statements are subject to factors that
1 Hill A, Richards SJ, Hillmen P. Recent developments in the understanding and management of paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2007 May;137(3):181-92.
2 Hillmen P, Lewis SM, Bessler M, et al. Natural history of
paroxysmal nocturnal hemoglobinuria. NEngl J Med. 1995
3 Socié G, Mary JY, de Gramont A, et al. Paroxysmal nocturnal
haemoglobinuria: long-term follow-up and prognostic factors.
4 Rosse W. Paroxysmal Nocturnal Hemoglobinuria. Hoffman:
Hematology: Basic Principles and Practice. 3rd ed.
5 Nishimura J, Kanakura Y, Ware RE, et al. Clinical course
and flow cytometric analysis of paroxysmal nocturnal hemoglobinuria in
6 Weitz I, Meyers G, Lamy T, et al. Cross-sectional validation study of patient-reported outcomes in patients with paroxysmal nocturnal haemoglobinuria. Intern Med J. 2013;43:298-307.
7 Parker C, Omine M, Richards S, et al. Diagnosis and
management of paroxysmal nocturnal hemoglobinuria. Blood. 2005
8 Hillmen P, Muus P, Duhrsen U, et al. Effect of the
complement inhibitor eculizumab on thromboembolism in patients with
paroxysmal nocturnal hemoglobinuria. Blood. 2007
9 Wang H, Chuhjo T, Yasue S, et al. Clinical significance of a minor population of paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure syndrome. Blood. 2002;100 (12):3897-3902.
10 Iwanga M, Furukawa K, Amenomori T, et al. Paroxysmal
nocturnal haemoglobinuria clones in patients with myelodysplastic
11 Maciejewski JP, Rivera C, Kook H, et al. Relationship
between bone marrow failure syndromes and the presence of
glycophosphatidyl inositol-anchored protein-deficient clones. Br
12 Hill A, Kelly RJ, Hillmen P. Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood. 2013;121:4985-4996.
13 Sahelijo L, Mujeebuddin A, Mitchell D, et al. First in human single-ascending dose study: safety, biomarker, pharmacokinetics and exposure-response relationships of ALXN1210, a humanized monoclonal antibody to C5, with marked half-life extension and potential for significantly longer dosing intervals. Blood. 2015;126 (23):4777.
Arne Naeveke, PhD, 475-230-3774
Vice President, Investor Relations
Director, Investor Relations
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