PTX-100

PTX-100 is a first-in-class, small molecule synthetic peptidomimetic inhibitor of geranylgeranyl transferase I (GGTase I or GGT‑1), an enzyme critical for the post-translational modification and activation of several oncogenic proteins, including members of the Ras superfamily. By inhibiting GGT‑1, PTX‑100 disrupts oncogenic Ras pathways through inhibition of Rho, Rac, and Ral circuits in cancer cells. This leads to apoptosis (programmed cell death) in malignant cells. The drug is being developed primarily for hematological malignancies such as cutaneous T-cell lymphoma (CTCL) and other T-cell lymphomas. It has received Orphan Drug Designation and Fast Track status from the US FDA for all T-cell lymphomas. Clinical studies have shown promising safety and efficacy signals in relapsed/refractory CTCL patients[1][3][5][10].

Other names

2-{[2-benzyl-4-(5-methyl-3H-imidazol-4-ylmethyl)-3-oxo-piperazine-1-carbonyl]-amino}-4-methyl-pentanoic acidPTX 100PTX100PTX-100