Prescient Therapeutics Doses First Patient in Pivotal PTX-100 CTCL Trial
Prescient Therapeutics has reached a pivotal milestone by dosing the first patient in its Phase 2a clinical trial of PTX-100 for relapsed/refractory Cutaneous T-Cell Lymphoma (CTCL), aiming to evaluate safety and efficacy across multiple international sites.
- First patient dosed in Phase 2a PTX-100 trial for CTCL
- Study targets up to 40 patients across Australia, USA, and Europe
- PTX-100 holds FDA orphan drug and fast track designations
- Trial focuses on relapsed/refractory Cutaneous T-Cell Lymphoma
- Investor briefing scheduled for May 29, 2025
A Significant Step Forward in CTCL Treatment
Prescient Therapeutics Limited (ASX: PTX) has announced the dosing of the first patient in its Phase 2a clinical trial of PTX-100, a novel targeted therapy for relapsed or refractory Cutaneous T-Cell Lymphoma (CTCL). This milestone marks a critical advancement in the company’s efforts to develop new treatment options for this rare and challenging form of cancer.
CTCL is a rare type of cancer affecting T cells, a subset of white blood cells integral to immune function. In CTCL, malignant T cells accumulate in the skin, leading to progressive and often debilitating symptoms. Treatment options for patients who relapse or do not respond to existing therapies remain limited, underscoring the urgent need for innovative approaches.
The Phase 2a Trial Design and Scope
The open-label Phase 2a study aims to enroll up to 40 patients across multiple sites in Australia, the United States, and Europe. It will evaluate various dosage levels of PTX-100, focusing primarily on assessing efficacy and safety. Secondary endpoints will further explore the drug’s clinical benefits and tolerability.
PTX-100 is a first-in-class compound that inhibits geranylgeranyl transferase-1 (GGT-1), an enzyme involved in cancer cell growth pathways. By disrupting oncogenic signaling, PTX-100 induces apoptosis, or programmed cell death, in cancer cells. The drug has already demonstrated encouraging safety and activity signals in earlier Phase 1 and Phase 1b studies, including a recent expansion cohort targeting T-cell lymphomas.
Regulatory Recognition and Strategic Positioning
The U.S. Food and Drug Administration (FDA) has granted PTX-100 orphan drug designation and fast track status for the treatment of T-cell lymphomas, including CTCL. These designations reflect the unmet medical need and the potential for PTX-100 to offer a meaningful therapeutic advance.
Prescient’s Chief Medical Officer, Dr. Marissa Lim, highlighted the importance of this milestone, noting the dosing took place at the Linear Clinical Research site in Perth under the care of Dr. Dejan Radeski, a specialist in T-cell lymphoma. The company’s CEO, James McDonnell, will provide further insights during an online investor briefing scheduled for May 29, 2025.
Looking Ahead
As the trial progresses, the oncology community and investors alike will be watching closely for data that could validate PTX-100’s promise as a new treatment avenue for CTCL. The company’s broader pipeline, including innovative cell therapy platforms, positions Prescient as a notable player in personalised cancer medicine.
Bottom Line?
With the first patient dosed, Prescient Therapeutics sets the stage for pivotal data that could reshape CTCL treatment options.
Questions in the middle?
- How quickly will Prescient Therapeutics achieve full enrollment of 40 patients?
- What interim efficacy and safety signals will emerge from the Phase 2a trial?
- How might regulatory feedback influence the development timeline and commercial prospects?
