Massey becomes first U.S. site to enroll patients in global Phase 2a trial of first-in-class therapy for T-cell lymphoma

VCU Massey Comprehensive Cancer Center is the first site in the United States to be activated in a global Phase 2a clinical trial evaluating PTX-100 (AKA GGTI-2418), a first-in-class small molecule inhibitor of geranylgeranyl transferase-1 (GGT-1), for the treatment of cutaneous T-cell lymphoma (CTCL), a rare and often treatment-resistant T-cell lymphoma.

Said Sebti, Ph.D., associate director for basic research and Lacy Family Chair in Cancer Research at Massey and professor in the Department of Pharmacology and Toxicology at the VCU School of Medicine, is the co-inventor of PTX-100 and the scientific founder at Prescient Therapeutics, which is sponsoring the trial.

“This trial reflects Massey’s long-standing commitment to translating basic science discoveries into innovative treatments that can directly benefit patients,” said Sebti.

Thinking about the journey of PTX-100, Sebti remarked, “As a cancer researcher, there is no greater fulfillment than seeing a therapy you co-invented in the laboratory begin to reach those who need it most. With the launch of this global Phase 2 trial of PTX-100 in CTCL, where new treatments are urgently needed, that long-held dream feels deeply real. To see my academic home, Massey, lead the way as the first U.S. site to open enrollment to this trial, is one of the most exhilarating and humbling moments of my career, and is a powerful reminder of what science can achieve when driven by purpose and passion.”

The newly launched phase 2a global trial will enroll patients with CTCL across leading academic cancer centers in the United States, Australia and Europe, with the aim of further evaluating the efficacy and safety of PTX-100 in patients with relapsed or refractory disease.

PTX-100 is currently believed to be the only GGT-1 inhibitor in clinical development globally, representing a novel class of targeted therapies. After demonstrating safety and early signals of clinical activity in a first-in-human Phase 1 study, PTX-100 advanced into a Phase 1b expansion cohort focused on patients with T-cell lymphomas, where it showed encouraging signs of efficacy and a favorable safety profile. Based on these results, PTX-100 received orphan drug designation from the United States Food and Drug Administration for all T-cell lymphomas, as well as fast track designation for the treatment of relapsed or refractory mycosis fungoides, the most common form of CTCL.

CTCL is a rare type of non-Hodgkin lymphoma where immune cells called T cells become cancerous and  primarily affect the skin causing itchy, red, scaly patches that may resemble eczema or psoriasis.

The enzyme GGT-1 plays a critical role in cancer biology, as it is essential for the activation of Rho, Rac, and Ral proteins, molecular switches that mediate the cancer-causing activity of the oncogene RAS, one of the most commonly mutated oncogenes in human cancers. Sebti and his colleagues have pioneered the development of GGT-1 inhibitors designed to block these downstream RAS effector pathways, thereby inducing programmed cell death and halting tumor growth in a variety of cancer models.

"This global Phase 2a clinical trial of PTX-100 will help provide an extension of care for our patients diagnosed with this rare cancer, for which new therapies are urgently needed," Robert A. Winn, M.D., director and Lipman Chair in Oncology at Massey said. "We are incredibly proud of Dr. Sebti and his team for bridging the gap between world-class scientific innovation and the communities we serve, ultimately making significant strides in the fight against cancer." 

For more information about the study or to inquire about eligibility, patients and referring physicians may contact Massey’s Clinical Trials Office at 804-628-6430 or by filling out a clinical trial request form.

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