Red Cell Disorders

February 6, 2006

Alexion Pharmaceuticals has issued positive results of a phase III trial, dubbed TRIUMPH, of eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria ("PNH"). Trial data met their co-primary efficacy endpoints, significantly reducing median hemoglobin transfusion rate (from 10 units/patient to 0 units/patient; p<0.000000001) and increasing the portion of patients with hemoglobin stabilization (49% vs. 0% for placebo; p<0.0000001). Secondary safety and efficacy endpoints, including reduction in lactate dehydrogenase levels, quality of life measures and transfusion avoidance, were also met. This placebo-controlled study enrolled 87 patients across 45 sites in the US, Canada, Europe and Australia. These results were seen to support BLA filing in the near future.

December 16, 2002

Celgene reported positive results from a phase II trial investigating Thalomid (thalidomide) in combination with prednisone for the treatment of myelofibrosis. The data demonstrated that 13 of 21 subjects (62%) achieved an objective clinical response that was defined by an increase in red blood cells or platelets. Seven of ten subjects (70%) who were red cell transfusion dependent responded to the combination therapy and four of the ten subjects (40%) became transfusion independent. Six of eight subjects (75%) with thrombocytopenia experienced a more than 50% increase in their platelet count. The most commonly reported adverse events were constipation, leukocytosis, mild neuropathy and mild sedation. The study enrolled 21 subjects with myelofibrosis who received 50 mg/day of Thalomid and a tapering regimen of prednisone.

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