Virginia Commonwealth University Leads Global Clinical Trial of PTX-100 for Cutaneous T-Cell Lymphoma Treatment.

Virginia Commonwealth University's Massey Comprehensive Cancer Center is conducting a global Phase 2a clinical trial of PTX-100, a first-in-class inhibitor of geranylgeranyl transferase-1, for the treatment of cutaneous T-cell lymphoma. The trial aims to evaluate the efficacy and safety of PTX-100 in patients with relapsed or refractory disease. PTX-100 has shown encouraging signs of efficacy and a favorable safety profile in a Phase 1 study and has received orphan drug and fast-track designations from the FDA.

Virginia Commonwealth University's Massey Comprehensive Cancer Center has launched a global Phase 2a clinical trial to evaluate PTX-100, a first-in-class inhibitor of geranylgeranyl transferase-1 (GGT-1), for the treatment of cutaneous T-cell lymphoma (CTCL). This trial aims to assess the efficacy and safety of PTX-100 in patients with relapsed or refractory disease.

PTX-100, also known as GGTI-2418, is a novel small molecule inhibitor that targets the enzyme GGT-1, which plays a critical role in cancer biology by activating molecular switches that mediate the oncogene RAS. Sebti, a co-inventor of PTX-100 and scientific founder at Prescient Therapeutics, is leading the trial at Massey. The trial will enroll patients across leading academic cancer centers in the United States, Australia, and Europe.

The clinical trial follows promising results from a Phase 1 study, which demonstrated PTX-100's safety and early signs of clinical activity in patients with T-cell lymphomas. PTX-100 has received orphan drug designation and fast-track designation from the FDA for the treatment of relapsed or refractory mycosis fungoides, the most common form of CTCL. This designation highlights the urgent need for new therapies in CTCL, a rare and often treatment-resistant cancer.

CTCL is a type of non-Hodgkin lymphoma where immune cells called T cells become cancerous and primarily affect the skin, causing itchy, red, scaly patches. The global Phase 2a trial will help provide an extension of care for patients diagnosed with this rare cancer, where new treatments are urgently needed.

The trial's primary endpoints include evaluating the efficacy and safety of PTX-100 in patients with relapsed or refractory CTCL. The trial is expected to provide valuable insights into the potential of PTX-100 as a targeted therapy for CTCL.

References:
[1] https://www.news-medical.net/news/20250726/VCU-Massey-pioneers-clinical-trial-of-novel-GGT-1-inhibitor-PTX-100.aspx
[2] https://www.targetedonc.com/view/fda-grants-fast-track-designation-to-vs-7375-for-kras-g12d-mutated-pancreatic-cancer