Telik announced that ezatiostat HCl (Telintra) has been granted orphan drug designation by the FDA for the treatment of myelodysplastic syndrome (MDS). Telintra is a novel inhibitor of the enzyme glutathione S-transferase P1‑1, leading to activation of Jun kinase, a key regulator of cellular growth and differentiation of blood precursor cells.
Telintra has been shown to cause clinically significant and sustained reduction in red blood cell transfusions, transfusion independence and multilineage responses in MDS patients. The results of four clinical trials of Telintra in MDS have been reported.
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