April 2025 - In a recent press release Prescient Therapeutics Limited (ASX: PTX), a clinical stage oncology company developing personalized therapies for cancer, announced the U.S. FDA has given PTX-100 Fast Track Designation for the treatment of adults with relapsed or refractory (r/r) mycosis fungoides, the most common subtype of CTCL. This marks another milestone for PTX-100 on the pathway to treating patients suffering with r/r Cutaneous T Cell Lymphoma (CTCL).
Fast Track is a process designed by the FDA to expedite the review of therapies that treat serious conditions with a high unmet need, with the aim of getting therapies to patients earlier. It provides several benefits including increased access to the FDA, the possibility of rolling submissions of New Drug Applications and a pathway to Accelerated Approvals – a key element of Prescient’s PTX-100 strategy.
PTX-100: is a first in class compound with the ability to block an important cancer growth enzyme known as geranylgeranyl transferase-1 (GGT-1). It disrupts oncogenic Ras pathways by inhibiting the activation of Rho, Rac and Ral circuits in cancer cells, leading to apoptosis (death) of cancer cells. PTX- 100 is believed to be the only GGT-1 inhibitor in the world in clinical development.
PTX-100 demonstrated safety and early clinical activity in a previous Phase 1 study and recent PK/PD basket study of hematological and solid malignancies. PTX-100 has recently completed a Phase 1b expansion cohort study in T cell lymphomas, where it showed encouraging efficacy and safety. The US FDA has granted PTX-100 Orphan Drug Designation for all T Cell Lymphomas. A Phase 2 study in Cutaneous T cell lymphoma (CTCL) has completed the first study site enrollment and now expects the first patient in and dosed in the coming weeks.