Alexion issued key patent for Eculizumab monoclonal antibody in Japan.

Alexion Pharmaceuticals, Inc. (Cheshire, CT) announced that the Japanese Patent Office has issued Patent 3734266, entitled "Methods and Compositions for the Treatment of Glomerulonephritis and Other Inflammatory Diseases," which relates to the company's lead product, Soliris brand of eculizumab. The issued claims encompass the use of Alexion's lead drug candidate, eculizumab, to inhibit complement activation in humans. Complement inhibition is the key mechanism of action for eculizumab in paroxysmal nocturnal hemoglobinuria (PNH), which Alexion is currently developing under the brand name Soliris.

"The issuance of this Japanese patent represents a key element of our proprietary position in C5 complement inhibition. The patent both supports and protects the exclusive market position we expect to develop globally for Soliris for the treatment of PNH," stated Leonard Bell, M.D., Chief Executive Officer of Alexion. "This patent also comes on the heels of exciting positive results from TRIUMPH, our pivotal Phase III efficacy trial using Soliris in PNH patients. As previously reported, all pre-specified primary and secondary endpoints in that international trial were achieved with statistical significance," added Dr. Bell.

Corresponding patent claims have issued in the United States and additional corresponding patents are issued or are pending in Europe, Canada, Australia and other key markets.

"Alexion is keenly focused on progressing our regulatory applications for Soliris in PNH, and on preparing for global commercialization, starting in the United States, Europe, and Japan. Securing our proprietary intellectual property position in Japan now provides us an important foothold for commercialization of Soliris for PNH in Asia. Following regulatory approvals, we plan to launch Soliris for PNH in at least 40 countries within three years of our first commercial launch," stated Mr. David Keiser, President and Chief Operating Officer of Alexion.

PNH, a rare form of hemolytic anemia, is an acquired genetic blood disorder characterized by destruction of red blood cells by the body's complement system (a component of the immune system). Patients with PNH lack naturally-occurring complement inhibitors which prevent red blood cell destruction. Eculizumab, a long-acting C5 terminal complement inhibitor, is a monoclonal antibody drug that selectively blocks terminal complement activation. There currently is no therapy specifically available for treatment of PNH.

Based upon scientific investigations and presentations of the prevalence of patients diagnosed with abnormal PNH cells in their blood, it is currently estimated that approximately 8,000 - 10,000 people in North America and Western Europe suffer from PNH. However, this estimate may not be applicable to Asian populations, where reports suggest that the prevalence may be higher.

Patients with PNH often have a poor quality of life and may suffer from severe hemolysis, anemia, chronic fatigue, recurrent pain, pulmonary hypertension and intermittent episodes of dark colored urine, known as hemoglobinuria. Importantly, PNH patients are at increased risk of forming life-threatening blood clots, or thromboses, which are a leading cause of death in this disease.

Eculizumab, an investigational drug therapy, has been granted Orphan Drug Status in the PNH indication from both the United States and European regulatory agencies. If approved for PNH, Soliris (eculizumab) would represent the first drug from a new class of anti-inflammatory therapeutics-terminal complement inhibitors-as well as the first drug available specifically for patients suffering from PNH.

Alexion is engaged in the discovery and development of therapeutic products aimed at treating patients with a wide array of severe disease states, including hematologic and cardiovascular disorders, autoimmune diseases and cancer. Alexion's two lead product candidates, eculizumab and pexelizumab, are currently undergoing evaluation in several clinical development programs, including two Phase III trials of Soliris (eculizumab) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Under the Special Protocol Assessment (SPA) process, the FDA has agreed to the design of protocols for the two trials of Soliris (eculizumab) in PNH patients that could, if successful, serve as the primary basis of review for approval of a licensing application for eculizumab in the PNH indication. The company's Phase III trial of pexelizumab in coronary artery bypass graft (CABG) surgery patients undergoing cardiopulmonary bypass (CPB) failed to achieve its primary endpoint. The company has determined to finalize its ongoing Phase III trial of pexelizumab in acute myocardial infarction (AMI) patients with fewer patients than originally planned. The pexelizumab trials are conducted in collaboration with Procter and Gamble Pharmaceuticals. Preliminary results from the PRIMO-CABG2 trial of pexelizumab indicate that the trial is unlikely to support filing for licensing approval of pexelizumab in the CABG indication. The number of patients to be enrolled in the AMI trial may not be sufficient for the previously agreed pexelizumab SPA for AMI. Alexion is engaged in discovering and developing a pipeline of additional antibody therapeutics targeting severe unmet medical needs, through its wholly owned subsidiary, Alexion Antibody Technologies, Inc.

Alexion Pharmaceuticals, Inc.

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