New Prescient leadership to drive PTX-100 development in rare blood cancer

• Prescient Therapeutics is focused on development of lead compound PTX-100 to treat rare blood cancer
• New leadership to steer the company through next stage of clinical program
• First patient dosed in phase 2a trial with PTX-100 granted US FDA fast-track designation

Special Report: With a new leadership team and fast-track designation from the US Food and Drug Administration (FDA), clinical-stage oncology company Prescient Therapeutics is laser focused on development of lead compound PTX-100.

Prescient Therapeutics (ASX:PTX) recently announced the first patient had been dosed in its phase 2a clinical trial of PTX-100 in patients with relapsed or refractory cutaneous T-cell lymphoma (CTCL).

The trial will evaluate two dosage levels of PTX-100 in an open-label design in up to 40 patients with relapsed or refractory CTCL across multiple sites in Australia, the US and Europe.

CTCL is a rare cancer affecting white blood cells known as T cells, which normally help regulate the immune system.

In CTCL, these cancerous T cells migrate to the skin, where they grow, divide and attack uncontrollably.

Treatment options remain limited for patients whose disease has relapsed or become resistant to therapy.

“We have a dose optimisation committee which will examine the first 20 patients in the phase 2a trial, so 10 patients at each dose level,” Prescient Therapeutics CEO James McDonnell said.

“They may then go to the FDA if it looks like there is an optimal dose and good efficacy to say this is what we want to do to continue to the phase 2b trial.

“We’ll keep communicating with the FDA and are hopeful the phase 2b trial will be a registrational trial given PTX-100 has fast-track designation.”

The phase 2b trial will evaluate safety and efficacy of PTX-100.

A first-in-class targeted cancer therapy

McDonnell explained PTX-100 is a first-in-class compound with the ability to block an important cancer growth enzyme geranylgeranyl transferase-1 (GGT-1).

PTX-100 disrupts critical signalling pathways that cancer cells rely on for growth and survival.

Specifically, it targets the oncogenic Ras pathway, blocking the activity of Rho, Rac, and Ral circuits.

McDonnell said the interference triggers a form of programmed cell death known as apoptosis, leading to the elimination of cancer cells.

PTX-100 is believed to be the only GGT-1 inhibitor in the world in clinical development.

The compound has demonstrated safety and early clinical activity in a previous phase I study and recent PK/PD basket study of haematological and solid malignancies.

In a phase 1b expansion cohort study in T cell lymphomas, PTX-100 showed
encouraging efficacy and safety.

“Our results showed an overall response rate of 45%  which means they had a complete response or partial response to  PTX-100 treatment, and  64% experienced a clinical benefit,” McDonnell said.

Watch:  CEO James McDonnell on changing the game for T-Cell Lymphoma

 PTX-100 secures key FDA designations

The FDA’s designations for PTX-100 highlights its relevance to treat a high unmet need.

PTX-100 has been granted fast-track designation by the US FDA for treatment of adults with relapsed or refractory mycosis fungoides, the most common subtype of CTCL following the phase 1b results.

“These patients typically relapse and run out of options – it’s an unmet need and the clinical responses in our phase 1b looked really promising so the FDA provided fast-track designation,” McDonnell said.

The regulator also granted PTX-100 orphan drug designation (ODD) for all T-cell lymphomas.

An ODD is aimed at encouraging treatment for rare disease and provides a range of benefits including seven years of market exclusivity upon approval in the US.

In December the FDA cleared Prescient’s investigational new drug (IND) application, paving the way for human clinical trials of PTX-100 as a new drug to start in the US.

“It’s not very often you get into a phase 2 trial environment, and you have a fast-track designation from the FDA and have some really good phase 1B results, so it makes sense to be very focused,” McDonnell said.

To speed up time to market, McDonnell said Prescient would also start a rolling new drug application (NDA) with the FDA.

A rolling NDA is available for drugs with fast-track designation. It allows companies to submit parts of a NDA to the FDA as they’re completed, rather than waiting to file everything at once.

The process helps speed up review for therapies targeting serious or life-threatening conditions.

New Aussie leadership team to support clinical program

2025 has been a year of leadership and board changes for Prescient.  McDonnell – a registered pharmacist and biopharmaceutical leader with more than 25 years of experience in the global pharmaceutical industry – became head of the biotech in January.

He has significant involvement in blood disorders and haematological malignancies such as myeloma, myelodysplasia, and CML, holding numerous senior leadership positions at leading pharmaceutical companies.  

 Prescient also has a new board chairman with Dr James Campbell replacing Steven Engle at the end of March.

The seasoned biotechnology executive is also the chairman of AusBiotech, Australia’s biotechnology industry organisation.

In March Dr Marissa Lim became chief medical officer. Lim has more than 20 years of experience in the pharmaceutical and biotechnology industries.

And in April experienced governance and risk professional in the life sciences sector Melanie Farris was appointed an independent non-executive director.

Farris will also serve as chair of the company’s audit and risk committee.

“What I have learned in my experience is it is about the people and the team you create,” said McDonnell who worked with Lim at CSL Vifor and Campbell at ChemGenex Pharmaceuticals.

“We have some good experts now and I feel with James and Marissa we really do have the right team to progress these PTX-100 studies.”

Chasing partners for cell therapy platforms

Prescient also owns exclusive rights to two next-generation cell therapy platform technologies, OmniCAR and CellPryme.

While still a part of its clinical pipeline McDonnell said the company had pivoted to prioritising advancement of PTX-100 and partnering its cell therapy platforms.

“It’s great to have a number of assets but at some point, you’ve got to make a call about where to really focus,” McDonnell said.

He added Presicent has worked to resolve certain OmniCAR technical issues, while cell therapies in general have experienced a downturn globally.

“Cell therapies are really expensive, and we wouldn’t have the capability to take OmniCAR to market ourselves,” he said.

“So that is our goal to get those assets to a point where we can partner with a cell therapy expert.”

Prescient’s US manufacturing facility

US President Donald Trump’s return to the White House has brought renewed uncertainty for pharmaceutical companies looking to enter the world’s largest healthcare market.

Trump has flagged tariffs on pharmaceutical imports to the US in a bid to reshore up manufacturing locally.

With that in mind, McDonnell emphasised:

“We have a US manufacturing facility and we’re not likely to change that considering transport costs and possible tariffs.

“Our number one market is the US and then Europe so manufacturing in the US makes a lot of sense.”

This article was developed in collaboration with Prescient Therapeutics, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.