Vertex Pharmaceuticals Inc. has begun Phase II clinical trialswith its compound, VX-105, for the treatment of sickle cell anemia.The trial will be conducted at several sites in the U.S. andCanada and will involve up to 45 patients. The Cambridge,Mass.-based company already is conducting Phase II trials withVX-105 for treatment of beta thalassemia.Richard Aldrich, Vertex's senior vice president and chiefbusiness officer, told BioWorld he expects to have results latethis year or early in 1995 for both Phase II trials. VX-105 is acompound designed to activate the gene responsible for hemoglobin F,whose production usually ends shortly after birth. The objectiveis to increase levels of hemoglobin F in sickle cell anemia and betathalassemia patients as a replacement for their lack of hemoglobin A.Aldrich said the studies will attempt to determine if VX-105 canincrease levels of hemoglobin F and if that result can reducesymptoms experienced by sickle cell anemia and beta thalassemia patients.In both Phase II trials of VX-105, patients receive one of threedoses intravenously and treatments span 14 days. Dosage levelsinvolve infusions of either 250 milligrams or 750 milligrams perkilogram of body weight at intervals of eight or 24 hours.The company also is conducting Phase I studies for a relatedcompound, VX-366, which is administered orally to sickle cellanemia and beta thalassemia patients._ Charles Craig
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