Vertex Pharmaceuticals Inc. has started Phase I trials withVX-366, an orally delivered compound being developed fortreatment of sickle cell anemia and beta thalassemia.The Cambridge, Mass., company's compound will betested in 20 to 30 healthy volunteers to determinepharmacokinetics and overall tolerability, Joshua Boger,president and CEO of Vertex, told BioWorld. He expectstrial data to be available in a few months, and that it will setup Phase II trials for each indication, which Boger hopes tobegin later this year.Another compound, VX-105, an intravenouslyadministered drug related to VX-366, already is in Phase IItrials for beta thalassemia. Boger said it is expected to be ina Phase II trial for sickle cell anemia in the next fewmonths. Both developed from arginine butyrate, whichVertex licensed from the Children's Hospital Oakland inCalif.The compounds are believed to act as transcriptionalactivators to upregulate the fetal hemoglobin gene. _ JimShrine
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