Prescient Therapeutics prepares for transformative year with new CEO and key FDA clearance

With new CEO James McDonnell in place and FDA clearance for its Investigational New Drug application, 2025 could be a signpost year for Prescient Therapeutics as it continues to develop PTX-100. 

It was a big December quarter for the company after its IND application for the PTX-100 Phase 2 clinical trial in r/rCTCL was successfully cleared by the US Food and Drug Administration (FDA), allowing for the study's immediate initiation

Significant progress was also made in the clinical development of CellPryme and re-engineering of OmniCAR, with growing collaboration interest from channel and R&D partners.

The company had a cash and term deposit balance of $8.4 million at December 31, with an additional $3.7 million R&D Tax Incentive Rebate received on January 10.

FDA clearance clears a path forward

Prescient received clearance from the FDA for its IND application, allowing the Phase 2 clinical trial of PTX-100 to proceed. PTX-100 is a first-in-class inhibitor targeting the RAS-family pathway, with the trial focusing on relapsed and refractory cutaneous T-cell lymphomas (r/r CTCL). The IND clearance represents a key milestone for the company, enabling the formal initiation of the study.

During the reporting period, Prescient dedicated significant resources to preparing the IND application, engaging clinical and industry experts to refine the study design, trial implementation strategy, and responses to FDA inquiries.

The company is now moving forward with site selection, assessing clinical locations in Australia, the United States, and Europe, with up to 15 sites expected to be activated.

Additionally, Prescient remains interested in exploring PTX-100's potential in Peripheral T-Cell Lymphoma (PTCL) and is evaluating strategies to generate further clinical data in this patient population.

What is PTX-100?

PTX-100 is a first-in-class compound designed to inhibit geranylgeranyl transferase-1 (GGT-1), a key enzyme involved in cancer growth. By blocking GGT-1, PTX-100 disrupts oncogenic Ras pathways, preventing the activation of Rho, Rac, and Ral signalling circuits in cancer cells, ultimately inducing apoptosis. It is believed to be the only GGT-1 inhibitor currently in clinical development.

The compound has demonstrated safety and early clinical activity in a previous Phase 1 study and a recent pharmacokinetics/pharmacodynamics (PK/PD) basket study across hematological and solid malignancies. The United States Food and Drug Administration (FDA) has granted PTX-100 Orphan Drug Designation for all T-cell lymphomas and recently approved an Investigational New Drug (IND) application for a Phase 2 study focusing on cutaneous T-cell lymphomas.

Momentum-gaining platform

Prescient’s CellPryme platform continues to gain momentum in both preclinical and clinical development. During Q4 2024, the company presented data on cell therapy manufacturing and adjuvant enhancements at the CAR-TCR Summit in Boston and the American Society of Hematology conference in San Diego, generating increased interest in potential collaborations.

Following these engagements, Prescient has initiated multiple partnerships involving CellPryme-M, working with potential collaborators to explore ways to enhance their cell therapy programs. The company intends to provide investors with updates on the progress of these partnerships as developments allow.

Additionally, Prescient is in the early stages of evaluating first-in-human clinical trials for CellPryme-A. If initiated, these trials will investigate CellPryme-A’s potential in combination with CAR-T therapies.

Meanwhile, significant progress has been made in OmniCAR development following extensive troubleshooting of technical challenges and the engineering of new, optimised molecular variants. With encouraging results achieved, Prescient will continue development efforts, with further validation work planned.

What is CellPryme?

CellPryme-M: Prescient’s CellPryme-M is a novel, clinic-ready technology designed to enhance adoptive cell therapy by shifting T cells towards a central memory phenotype. This improves persistence and increases the ability of cells to locate and penetrate tumours. The process is a non-disruptive, 24-hour step within cell manufacturing. Cell therapies requiring enhanced manufacturing productivity or increased in vivo potency and durability are well-suited for CellPryme-M.

CellPryme-A: CellPryme-A is an adjuvant therapy administered alongside cellular immunotherapy to counteract the suppressive tumour microenvironment. It significantly reduces regulatory T cells, promotes CAR-T cell expansion in vivo, and enhances tumour penetration. These improvements result in better tumour cell killing and increased host survival. When used in combination with CellPryme-M pre-treated CAR-T cells, these benefits are further amplified.

New CEO

New CEO James McDonnell is a seasoned biopharmaceutical executive with over 25 years of experience in the global pharmaceutical industry, with a strong focus on blood disorders and haematological malignancies, including myeloma, myelodysplasia, and chronic myeloid leukaemia (CML). His strategic leadership has driven outstanding commercial success, underpinned by his ability to cultivate high-performing team cultures.