Ezatiostat (TER199, TLK199, Telintra)[1][2] is a peptidomimetic inhibitor of Glutathione S-transferase P1-1 (GSTP1-1)[2] and a tripeptide analog of glutathione[1]. It is being studied as a potential treatment for severe chronic neutropenia in clinical trials[3] and has been shown to stimulate the formation of bone marrow cells that are precursors to granulocytes and monocytes (white blood cells)[4][5].

It undergoes de-esterification to TLK235 and TLK236, which are its active forms[4]. Ezatiostat has a novel mechanism of action and beneficial effects on bone marrow[2][3]. It promotes maturation of hematopoietic stem cells and increases their survival rate[3][5].

Ezatiostat (also known as Telintra) is an investigational drug being studied in clinical trials for the treatment of myelodysplastic syndrome (MDS)[1][2]. A Phase 1 study of ezatiostat tablets was conducted in patients with MDS[3], while a Phase 2 study of ezatiostat hydrochloride liposomes for injection resulted in a clinically significant and sustained reduction of red blood cell transfusions[1][2]. The dose schedule for the Phase 2 trial was three weeks treatment with Telintra 2000 mg per day in two divided doses followed by one week with no treatment per four week cycle[4].

It has been granted Orphan Drug designation by the US FDA in January 2013 for the treatment of myelodysplastic syndromes[2] and Telik, Inc. announced that its product candidate, ezatiostat hydrochloride (Telintra), has been granted orphan drug designation by the FDA[3]. Ezatiostat is currently in clinical development (Ezatiostat HCl) for the treatment of various diseases and the potential for FDA approval and registration of this drug awaits further clinical trials[4][5].

Reference

1
nih.gov
2
springer.com
3
ascopost.com
4
sciencedirect.com
5
selleckchem.com

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