First Clinical Experience With Soliris(R) in Treating Patients With Two Rare Complement-Mediated Diseases Presented at ASH Annual Meeting

Monday, December 8, 2008 General News
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CHESHIRE, Conn., Dec. 7 /PRNewswire-FirstCall/-- Soliris(R) (eculizumab),a terminal complement inhibitor developed by Alexion Pharmaceuticals, Inc.(Nasdaq: ALXN) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH),was associated with an improvement in hematologic parameters in a limitednumber of patients with two other rare and severe complement-mediated diseasestreated outside of a clinical trial, according to patient cases presentedtoday at the 50th Annual Meeting of the American Society of Hematology (ASH).

Physicians reported on the first clinical experience with Soliris inpatients with atypical Hemolytic Uremic Syndrome (aHUS) and Cold AgglutininDisease (CAD), two rare and serious diseases. Like patients with PNH, patientswith aHUS are missing or have defective complement inhibitors that help toregulate the body's immune system. These patients suffer from hemolysis, bloodclotting which can be measured in part as reduced number of circulatingplatelets, and kidney damage. In two patients with aHUS, Solirisadministration was associated with improvements in platelet levels andreduction in hemolysis. Patients with CAD suffer from an autoimmune attack ontheir red blood cells leading to severe complement activation and hemolysis(destruction of those blood cells), anemia, and poor quality of life. In onepatient with CAD, Soliris treatment was associated with reduced hemolysis,absence of the need for blood transfusions and improved symptoms of fatigueand anemia. Eculizumab appeared to be well tolerated in these patients, withsafety observations that were consistent with those reported from controlledtrials in patients with PNH.

"We are encouraged by this initial clinical experience with eculizumab ina very limited number of aHUS and CAD patients. These observations and otherknowledge of the mechanisms at work in these disorders lead us to believe thatthere are strong scientific rationales for undertaking controlled clinicaltrials to investigate the role of complement inhibition in these twoconditions," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "Aspreviously announced, we are working closely and urgently with clinicalexperts from Europe and North America to design clinical trials of eculizumabfor the treatment of aHUS and CAD."

Initial Experience with Eculizumab in aHUS

A poster titled "Successful Treatment of Atypical Hemolytic UremicSyndrome with the Complement Inhibitor Eculizumab" was presented today at theASH annual meeting by Dr. Jens Nuernberger of the Department of Nephrology atUniversity Duisburg-Essen in Essen, Germany.

Dr. Nuernberger and his colleagues investigated the potential effect ofeculizumab in two patients with aHUS, a rare disease usually caused by agenetic mutation in the complement system. In patients with aHUS, this defectresults in hemolysis, formation of tiny blood clots (microvascular thrombosis)and inflammation of blood vessels, often causing acute kidney injury andprogressing to end-stage kidney disease. Those who survive aHUS often livewith irreversible kidney disease and are dependent upon dialysis to stayalive.

"Our first clinical experience with eculizumab in patients with aHUS isvery promising, especially given the devastating nature of this disease andthe lack of effective treatments," said Dr. Nuernberger. "Initial data suggestthat eculizumab reduced hemolysis, microvascular thrombosis, and blood vesselinflammation, thereby appearing to improve the course of aHUS. If clinicaltrials confirm these results, eculizumab may then improve our ability to carefor these patients."

Initial Experience with Eculizumab in CAD

A poster titled "Long-term Efficacy of the Terminal Complement InhibitorEculizumab in a Patient with Cold Agglutinin Disease" was presented today atthe ASH annual meeting by Dr. Alexander Roeth of the Department of Hematologyat the University Hospital of Essen in Essen, Germany.

Dr. Roeth and his colleagues investigated the potential effect ofeculizumab therapy in one patient with CAD, a rare and severe diseasecharacterized by the production of circulatory cold agglutinins, a type ofmonoclonal antibody that lead to hemolysis. Conventional treatments for CAD,including corticosteroids and immunosuppressive drugs, are ineffective in manypatients.

"We currently lack effective treatment options for patients with CAD,"noted Dr. Roeth. "Given these promising initial observations, clinicalresearch is needed to determine the therapeutic potential of eculizumab inpatients with CAD."

About Soliris

Soliris was approved in March 2007 by the U.S. Food and DrugAdministration (FDA) as the first treatment for PNH, a rare, debilitating andlife-threatening blood disorder defined by hemolysis, or the destruction ofred blood cells. In June 2007, the European Commission (EC) also approved theuse of Soliris for the treatment of patients with PNH. Soliris is the firsttherapy approved in Europe for the treatment of PNH and was the firstmedicinal product to receive EC approval under the EMEA Accelerated AssessmentProcedure. Soliris is not approved for the treatment of atypical HemolyticUremic Syndrome (aHUS) and Cold Agglutinin Disease (CAD).

Important Safety Information

Soliris is generally well tolerated. The most frequent adverse eventsobserved in clinical studies were headache, nasopharyngitis (a runny nose),back pain and nausea. Treatment with Soliris should not alter anticoagulantmanagement because the effect of withdrawal of anticoagulant therapy duringSoliris treatment has not been established.

The U.S. product label for Soliris also includes a boxed warning: "Solirisincreases the risk of meningococcal infections. Vaccinate patients with ameningococcal vaccine at least two weeks prior to receiving the first dose ofSoliris; revaccinate according to current medical guidelines for vaccine use.Monitor patients for early signs of meningococcal infections, evaluateimmediately if infection is suspected, and treat with antibiotics ifnecessary." During clinical studies, two out of 196 vaccinated PNH patientstreated with Soliris experienced a serious meningococcal infection.

Prior to beginning Soliris therapy, all patients and their prescribingphysicians are enrolled in the Soliris Safety Registry, which is part of aspecial risk management program that involves initial and continuing educationand long-term monitoring for detection of new safety findings.

Please see full prescribing information at www.soliris.net.

About Alexion

Alexion Pharmaceuticals, Inc. is a biopharmaceutical company working todevelop and deliver life-changing drug therapies for patients with serious andlife-threatening medical conditions. Alexion is engaged in the discovery,development and commercialization of therapeutic products aimed at treatingpatients with a wide array of severe disease states, including hematologicdiseases, cancer and autoimmune disorders. In March 2007, the FDA grantedmarketing approval for Alexion's first product, Soliris, for all patients withPNH, and Alexion began commercial sale of Soliris in the U.S. during April2007. In June 2007, the EC granted marketing approval for Soliris in theEuropean Union for all patients with PNH. Alexion is evaluating otherpotential indications for Soliris as well as other formulations of eculizumabfor additional clinical indications, and is pursuing development of otherantibody product candidates in early stages of development. This press releaseand further information about Alexion Pharmaceuticals, Inc. can be found atwww.alexionpharm.com.

Safe Harbor Statement

This news release contains forward-looking statements, includingstatements related to potential health and medical benefits from Soliris.Forward-looking statements are subject to factors that may cause Alexion'sresults and plans to differ from those expected, including for example,decisions of regulatory authorities regarding marketing approval or materiallimitations on the marketing of Soliris, delays in arranging satisfactorymanufacturing capability and establishing commercial infrastructure, delays indeveloping or adverse changes in commercial relationships, the possibilitythat results of clinical trials are not predictive of safety and efficacyresults of Soliris in broader patient populations, the possibility thatinitial results of commercialization are not predictive of future rates ofadoption of Soliris, the risk that third parties won't agree to license anynecessary intellectual property to Alexion on reasonable terms or at all, therisk that third party payors will not reimburse for the use of Soliris atacceptable rates or at all, the risk that estimates regarding the number ofPNH patients are inaccurate, the risk that pending litigation may be resolvedadversely, and a variety of other risks set forth from time to time inAlexion's filings with the Securities and Exchange Commission, including butnot limited to the risks discussed in Alexion's Quarterly Report on Form 10-Qfor the period ended September 30, 2008, and in Alexion's other filings withthe Securities and Exchange Commission. Alexion does not intend to update anyof these forward-looking statements to reflect events or circumstances afterthe date hereof, except when a duty arises under law.

Posters: II-388/II-976[ALXN-G]

SOURCE Alexion Pharmaceuticals, Inc.


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