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Biotech / Medical : Alexion Pharmaceuticals, Inc. (ALXN) -- Ignore unavailable to you. Want to Upgrade?

To: Icebrg who wrote (387)	11/11/2003 2:27:35 PM
From: Icebrg	Read Replies (1) \| Respond to of 824

Eculizumab Could Be Paroxysmal Nocturnal Hemoglobinuria Breakthrough [This is an article from Medscape. Almost a year old, coming out following last year's ASH conference. But still worth reading giving some background ahead of this year's ASH conference, when further data will be presented]. Jane Salodof MacNeil Dec. 13, 2002 (Philadelphia) — Preliminary results from an ongoing open-label phase Ib clinical trial suggest an experimental drug called eculizumab could be the first to alleviate symptoms of paroxysmal nocturnal hemoglobinuria (PNH). Data on the first 11 patients show that three months of eculizumab therapy reduced the need for blood transfusions by 68%. An 81% reduction in biochemical parameters of hemolysis was also reported, and duration of clinical paroxysms went down by 90% from 2.1 days per patient per month to 0.2. "With leukemia trials you have to wait months and years to see if there's a benefit. You knew immediately with this drug," principal investigator Peter Hillmen, MB, ChB, PhD, told Medscape. "[The patients] came in the next week, and they were fine." Dr. Hillmen, a clinical hematologist at The General Infirmary at Leeds in the U.K., presented the results here at the American Society of Hematology annual meeting. The paper was chosen because eculizumab would be the first targeted therapy with relevance to PNH, according to Russell Ware, MD, PhD, of Duke University Medical Center, co-chair of the session on acquired disorders. Dr. Ware called the work "very exciting" in an interview with Medscape, but cautioned, "There's still a lot of work to be done. They need to study dosing, side effects — both short and long term — and risks as well as benefits to patients." An acquired genetic disorder, PNH occurs in roughly one in 200,000 people, according to Dr. Hillmen. Patients with PNH are deficient in the PIG-A enzyme and consequently their cells cannot protect themselves from complements in the blood. As a result, red blood cells are prone to lysis, producing blood in the person's plasma and urine, which often becomes dark red. cont...