Healthcare Industry News: cardiopulmonary
News Release - January 4, 2006
Alexion Completes Patient Treatment in Its Phase III Pivotal TRIUMPH Efficacy Trial of Eculizumab in PNH PatientsTrial Results Expected to be Available During First Quarter 2006
CHESHIRE, Conn., Jan. 4 (HSMN NewsFeed) -- Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN ) announced today that it has completed treatment of the final patient in the pivotal Phase III TRIUMPH efficacy trial of eculizumab in patients with the chronic orphan blood disorder Paroxysmal Nocturnal Hemoglobinuria ("PNH"), a rare form of hemolytic anemia characterized by destruction of red blood cells by the immune system. In accordance with the trial's design, eligible patients were randomized to receive either eculizumab or placebo in a six-month treatment phase. Eculizumab is a monoclonal antibody drug that selectively blocks terminal complement, a part of the immune system that is implicated in PNH red blood cell destruction. There currently is no drug specifically available for treatment of PNH, in which patients' blood cells are deficient in natural inhibitors of terminal complement.
Approximately 85 PNH patients were randomized into the six month treatment phase in TRIUMPH, which exceeded the patient requirements agreed upon with the U.S. Food and Drug Administration (FDA) as part of the Special Protocol Assessment (SPA) process. Having completed treatment of the final patient, it is anticipated that top line clinical results will be available later in the first quarter of this year.
"We are pleased to have completed the final protocol-specified treatment in the TRIUMPH trial, on target with our earlier announced schedule," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "Additionally, we are encouraged that every eligible TRIUMPH patient to date has opted to enroll in an open-label extension trial to receive eculizumab. We are keenly focused on progressing our internal efforts to compile our regulatory applications for submission, pending clinical results, to U.S. and European regulatory authorities later this year to market eculizumab for use in PNH patients."
TRIUMPH is a double-blind, randomized, placebo-controlled multi-center pivotal Phase III trial, examining the effects of eculizumab on the co-primary endpoints of hemoglobin stabilization and blood transfusion requirement in hemolytic, transfusion-dependent PNH patients during six months of therapy. The study enrolled patients in the US, Canada, Europe, and Australia. TRIUMPH is designed to be a single pivotal efficacy trial for eculizumab therapy in PNH.
In addition to TRIUMPH, the pivotal Phase III clinical program includes the SHEPHERD trial. SHEPHERD is an open-label, non-placebo-controlled, multi-center clinical trial primarily aimed at generating additional safety data with eculizumab in a broader population of hemolytic PNH patients with a history of transfusions. Efficacy measures will also be obtained during the study. The SHEPHERD protocol includes 12 months of treatment with a six month interim analysis.
Alexion previously reached an agreement with the FDA on the design of TRIUMPH and SHEPHERD under the FDA's SPA process. It is expected that, if successful, the combined trials will comprise the application that will serve as the primary basis of review for the approval of a Biologics License Application (BLA) for eculizumab in the PNH indication. Pending analyses of results from TRIUMPH and SHEPHERD and subsequent discussions with U.S. and European regulatory authorities, Alexion anticipates submitting applications later this year to both U.S. and European regulatory authorities for approval to market eculizumab for use in PNH patients.
PNH is a rare blood disorder characterized by the onset of severe hemolytic anemia, chronic fatigue and intermittent episodes of dark colored urine, known as hemoglobinuria. PNH patients are also at increased risk of forming life-threatening blood clots, or thromboses, which are a leading cause of death in this disease. People with PNH have an acquired deficiency of proteins that normally protect red blood cells from a component of the body's natural defense system, known as the complement cascade. Lack of these complement inhibitor proteins leaves PNH red blood cells susceptible to destruction (hemolysis) by terminal complement, causing patients to become anemic. Eculizumab is designed to block production of terminal complement, thereby preventing PNH red blood cell destruction. There currently is no drug specifically available for treatment of patients with 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 may suffer 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, pexelizumab and eculizumab, are currently undergoing evaluation in several clinical development programs, including two Phase III trials of 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 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. Eculizumab has also been studied in rheumatoid arthritis and membranous nephritis. 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, and the Company is assessing the impact of the results of this study, known as PRIMO-CABG2, on its ongoing Phase III trial of pexelizumab in acute myocardial infarction (AMI) patients. The pexelizumab trials are conducted in collaboration with Procter and Gamble Pharmaceuticals. Under the SPA process, the FDA has agreed to the design of protocols for the Phase III pexelizumab trials that could, if successful, serve as the primary basis of review for approval of licensing applications for the two indications. 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. 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. This press release and further information about Alexion Pharmaceuticals, Inc. can be found at: http:/www.alexionpharm.com.
This news release contains forward-looking statements, including statements related to timing of announcement of clinical trial results, timing of anticipated regulatory submissions and the progression of Alexion's drug candidates towards commercial sales. Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ from those expected, including the results of pre-clinical or clinical studies (including termination or delay in clinical programs), the need for additional research and testing, decision of the FDA not to approve (or to materially limit) marketing of one or both of Alexion's two drug candidates, delays in arranging satisfactory manufacturing capability, inability to acquire funding on timely and satisfactory terms, delays in developing or adverse changes in commercial relationships, the possibility that results of earlier clinical trials are not predictive of safety and efficacy results in later clinical trials, dependence on Procter & Gamble Pharmaceuticals for development and commercialization of pexelizumab, the risk that third parties won't agree to license any necessary intellectual property to us on reasonable terms, and a variety of other risks set forth from time to time in Alexion's filings with the Securities and Exchange Commission, including but not limited to the risks discussed in Alexion's Annual Report on Form 10-K for the year ended July 31, 2005 and in our other filings with the Securities and Exchange Commission. P&GP retains the development rights and the termination rights discussed in Alexion's Form 10-K referred to above. Alexion does not intend to update any of these forward- looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.
Source: Alexion Pharmaceuticals
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