-Additional Data, Including Late-Breaking
Results from Phase 2 Trial of ALXN1007 in Patients with GI-GVHD,
Also to be Presented-
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
researchers will present late-breaking data from a Phase 1/2
dose-escalating study of ALXN1210, the Company’s highly innovative
longer-acting C5 antibody, in patients with paroxysmal nocturnal
hemoglobinuria (PNH). In another late-breaking poster, researchers
will present additional results from a Phase 2 trial evaluating
ALXN1007 in patients with acute graft-versus-host disease involving
the lower gastrointestinal tract (GI-GVHD). Data will also be
presented from an observational study in Japan (OPTIMA) evaluating
change in PNH clone size in patients with bone marrow failure.
These findings will be presented at the 21st Congress of the
European Hematology Association (EHA), being held June 9-12,
2016, in Copenhagen, Denmark.
PNH is a debilitating, ultra-rare and life-threatening blood
disorder characterized by complement-mediated hemolysis
(destruction of red blood cells).1 Acute GI-GVHD is a severe and
life-threatening rare auto-immune disease that can occur as a
complication of stem cell transplantation.2,3
Abstracts summarizing these presentations were published on the
EHA website and can be accessed using the links below.
The following late-breaking abstracts will be presented in a
poster session available from Friday, June 10, 2016, at 9:30
a.m., Central European Summer Time (CEST) to Saturday, June 11,
2016, at 7:00 p.m., CEST:
- Abstract LB2247: “ALXN1210, A
Long-Acting C5 Inhibitor, Results in Rapid and Sustained Reduction
of LDH with a Monthly Dosing Interval in Patients with PNH:
Preliminary Data from a Dose-Escalation Study,” Lee, et al.
- Accessible
at: http://learningcenter.ehaweb.org/eha/2016/21st/135358/jong.wook.lee.alxn1210.a.long-acting.c5.inhibitor.results.in.rapid.and.html?f=m3
- Abstract LB2269: “Phase 2A Study of
ALXN1007, A Novel C5A Inhibitor, in Subjects with Newly Diagnosed
Acute Graft-Versus-Host Disease (GVHD) Involving the Lower
Gastrointestinal Tract,” Alousi, et al.
- Accessible
at: http://learningcenter.ehaweb.org/eha/2016/21st/135380/amin.majid.alousi.phase.2a.study.of.alxn1007.a.novel.c5a.inhibitor.in.subjects.html?f=m3
The following abstract also will be presented during the poster
session from Friday, June 10, 2016, at 9:30 a.m., CEST to
Saturday, June 11, 2016, at 7:00 p.m., CEST:
- Abstract P634: “A Clinical Significance
and Time-Dependent Change of PNH Clone Size in Patients with Bone
Marrow Failure Syndrome: Japanese Multi-Centre Prospective Study,”
Shirasugi, et al.
- Accessible at:
http://learningcenter.ehaweb.org/eha/2016/21st/133522/yukari.shirasugi.a.clinical.significance.and.time-dependent.change.of.pnh.html?f
About PNH
PNH is an ultra-rare blood disorder in which chronic,
uncontrolled activation of complement, a component of the normal
immune system, results in hemolysis (destruction of the patient's
red blood cells). PNH strikes people of all ages, with an average
age of onset in the early 30s.1 Approximately 10 percent of
all patients first develop symptoms at 21 years of age or
younger.4 PNH develops without warning and can occur in men
and women of all races, backgrounds and ages. PNH often goes
unrecognized, with delays in diagnosis ranging from one to more
than 10 years.5 In the period of time before Soliris was
available, it had been estimated that approximately one-third of
patients with PNH did not survive more than five years from the
time of diagnosis.1 PNH has been identified more commonly
among patients with disorders of the bone marrow, including
aplastic anemia (AA) and myelodysplastic syndromes (MDS).6,7,8 In
patients with thrombosis of unknown origin, PNH may be an
underlying cause.1
About ALXN1210
ALXN1210 is a highly innovative, longer-acting C5 antibody being
evaluated by Alexion in a Phase 1/2 and Phase 2 study for the
treatment of patients with PNH.
About GI-GVHD
Acute GI-GVHD is an immune-mediated disease and a complication
of stem cell transplantation occurring in 10 to 12 percent of
allogeneic hematopoietic stem cell transplants.2,3 Patients with
severe acute GI-GVHD have a 30 to 40 percent mortality rate within
the first six months post-transplant.9 There are no approved
treatments for GI-GVHD.
About ALXN1007
ALXN1007 is a novel anti-inflammatory antibody targeting
complement protein C5a being evaluated in a Phase 2 trial for
patients with acute GI-GVHD.
About Alexion
Alexion is a global biopharmaceutical company focused on
developing and delivering life-transforming therapies for patients
with devastating and rare disorders. Alexion is the global leader
in complement inhibition and has developed and commercializes the
first and only approved complement inhibitor to treat patients with
paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic
uremic syndrome (aHUS), two life-threatening ultra-rare disorders.
In addition, Alexion’s metabolic franchise includes two highly
innovative enzyme replacement therapies for patients with
life-threatening and ultra-rare disorders, hypophosphatasia (HPP)
and lysosomal acid lipase deficiency (LAL-D). Alexion is advancing
the most robust rare disease pipeline in the biotech industry with
highly innovative product candidates in multiple therapeutic areas.
This press release and further information about Alexion can be
found at: www.alexion.com.
[ALXN-G]
References
1Socié G, Mary JY, de Gramont A, et al. Paroxysmal nocturnal
haemoglobinuria: long-term follow-up and prognostic factors.
Lancet. 1996: 348:573-577.
2Jagasia, M., M. Arora, M. E. D. Flowers, N. J. Chao, P. L.
Mccarthy, C. S. Cutler, A. Urbano-Ispizua, S. Z. Pavletic, M. D.
Haagenson, M.-J. Zhang, J. H. Antin, B. J. Bolwell, C. Bredeson,
J.-Y. Cahn, M. Cairo, R. P. Gale, V. Gupta, S. J. Lee, M. Litzow,
D. J. Weisdorf, M. M. Horowitz, and T. Hahn. Risk Factors for Acute
GVHD and Survival after Hematopoietic Cell Transplantation. Blood
119.1 (2012): 296-307.
3MacMillan, M. L., DeFor, T. E. and Weisdorf, D. J. (2012), What
predicts high risk acute graft-versus-host disease (GVHD) at
onset?: identification of those at highest risk by a novel acute
GVHD risk score. British Journal of Haematology, 157: 732–741
10
4Parker C, Omine M, Richards S, et al. Diagnosis and management
of paroxysmal nocturnal hemoglobinuria. Blood.
2005;106(12):3699-3709.
5Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural
history of paroxysmal nocturnal hemoglobinuria. N Engl J Med 1995
Nov 9;333(19):1253-8.
6Wang H, Chuhjo T, Yasue S, Omine M, Naka S. Clinical
significance of a minor population of paroxysmal nocturnal
hemoglobinuria-type cells in bone marrow failure syndrome. Blood.
2002;100 (12):3897-3902.
7Iwanga M, Furukawa K, Amenomori T, et al. Paroxysmal nocturnal
haemoglobinuria clones in patients with myelodysplastic syndromes.
Br J Haematol. 1998;102(2):465-474.
8Maciejewski JP, Rivera C, Kook H, Dunn D, Young NS.
Relationship between bone marrow failure syndromes and the presence
of glycophosphatidyl inositol-anchored protein-deficient clones. Br
J Haematol. 2001;115:1015-1022.
9Bolanos- Meade, J. et al. Blood. 2014; 124 (22); 3221-3227.
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version on businesswire.com: http://www.businesswire.com/news/home/20160519005595/en/
Alexion Pharmaceuticals, Inc.MediaStephanie Fagan,
203-271-8223Senior Vice President, Corporate CommunicationsorAmanda
Fahey, 203-699-7240Associate Director, Corporate
CommunicationsorInvestorsElena Ridloff, CFA, 203-699-7722Vice
President, Investor Relations
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