Soliris(R) Therapy Provided Clinical Improvements In PNH Patients Regardless Of Disease Severity

Soliris(R)
(eculizumab) therapy reduced hemolysis, fatigue, thromboses (blood clots)
and transfusion requirements in patients with a rare blood disorder called
paroxysmal nocturnal hemoglobinuria (PNH), including those who might have
been expected to have less severe disease, according to data from an
ongoing open-label clinical study presented at the 49th Annual
Meeting of the American Society of Hematology Meeting in Atlanta.


The data were highlighted in an oral presentation titled, "High
Incidence of Progression to Significant Disease Burden in Paroxysmal
Nocturnal Hemoglobinuria Patients with Lower Levels of Hemolysis, Mild
Anemia and Minimal Transfusion: Clinical Improvement with Eculizumab
Therapy."



In the study, (1) Soliris was associated with significant long-term
clinical improvements in patients with PNH, regardless of baseline degree
of hemolysis, anemia or transfusion requirements. The study also
demonstrated that patients who might have been expected to have less severe
disease, considering their baseline clinical characteristics, suffered from
significant disease burden.



"PNH patients once thought to have less severe disease based on their
clinical characteristics actually face significant disease burden from
anemia, fatigue, impaired quality of life, blood transfusion requirements
and blood clot risk," said Monica Bessler, MD, PhD, lead author of the
study and Professor of Medicine, Professor of Pharmacology and Molecular
Biology, Washington University in St. Louis School of Medicine. "The
results presented today show that, in this study group, regardless of
disease severity, long- term Soliris treatment provides important clinical
improvements in their disease signs, symptoms and complications."



"We continue to observe that the PNH patient population includes a wide
range of patients with a broad clinical profile," said Leonard Bell, MD,
Chief Executive Officer of Alexion Pharmaceuticals. "The results presented
today provide further evidence for the utility of Soliris therapy in
patients with diverse manifestations of PNH. We remain committed to our
goal that all patients who can benefit from Soliris will have access to
it."



Soliris, developed by Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN), is
the first therapy approved for the treatment of patients with PNH, a rare,
debilitating and life-threatening blood disorder defined by the destruction
of red blood cells, or hemolysis. Soliris is a complement inhibitor
indicated for the treatment of patients with PNH to reduce hemolysis. In
patients with PNH, hemolysis can cause thromboses, kidney disease, liver
dysfunction, disabling fatigue, impaired quality of life, recurrent pain,
shortness of breath, pulmonary hypertension, intermittent episodes of dark
colored urine (hemoglobinuria), and anemia. (2-4)
















Clinical Data



In the ongoing open-label clinical study, investigators examined the
long- term clinical effect of Soliris in patient subgroups, including those
with lower levels of hemolysis, mild anemia and minimal transfusion
requirements.



Data were analyzed by levels of baseline hemolysis (as indicated by
baseline quartiles of lactate dehydrogenase, LDH), anemia (Hgb < 10.5 g/L
vs greater than or equal to 10.5 g/L), and transfusion requirements
(transfusion episodes in prior year greater than 1 vs 1 or 0 episodes).
Hemolysis, fatigue and transfusion requirements were examined using a
ranking test to measure efficacy during both the first and the most recent
six months of Soliris therapy; patients received a median of 22 months of
treatment.



Hemolysis was significantly reduced with eculizumab treatment in
patients with all levels of baseline hemolysis. In patients with the lowest
quartile of hemolysis (< 1490 U/L), LDH was reduced from 1077 +/- 42 U/L
pre-eculizumab to 323 +/- 22 U/L during the first six months and to 347 +/-
47 U/L during the most recent six months of Soliris therapy (P