Prescient focuses on advancing lead cancer drug toward pivotal trial

Prescient Therapeutics is entering a critical stage with clinical progress, regulatory tailwinds and commercial opportunity converging.

Prescient is at a critical stage of development with its cancer therapy PTX-100. Pic via Getty Images.

• Prescient at critical stage of development with phase 2a trial underway for PTX-100

• PTX-100 granted orphan drug and fast track designations by the US FDA, unlocking benefits

• Focus on advancing PTX-100 to potential registrational phase 2b trial to open commercial opportunities

Special Report: Prescient Therapeutics has entered a critical stage where clinical data, regulatory support, and commercial potential begin to converge in a defining moment for a biotech company aiming to bring a new therapy to market.

Prescient Therapeutics (ASX:PTX) is in phase 2a trial for its lead compound PTX-100 in patients with rare blood cancer relapsed/refractory Cutaneous T-Cell Lymphoma (r/r CTCL) with the potential for its phase 2b arm to be registrational.

PTX-100 is a first-in-class compound with the ability to block an important cancer growth enzyme geranylgeranyl transferase-1 (GGT-1). It is believed to be the only GGT-1 inhibitor in the world in clinical development.

The US Food and Drug Administration (FDA) has granted PTX-100 orphan drug designation (ODD) for all T-cell lymphomas and fast-track designation for treatment of adults with relapsed or refractory mycosis fungoides, the most common subtype of CTCL.

Fast Track designation provides an expedited pathway to approval, with orphan drug designation offering market exclusivity for a period of seven years in the US.

“The aim is to improve the quality of life for people suffering from this terrible form of cancer and getting them the therapy they need as fast as possible,” CEO James McDonnell said.

“It's also an incentive for larger pharmaceuticals who are looking to add to their portfolio as the drug progresses through clinical development.”

Potential regulatory trial for PTX-100

McDonnell is focused on getting PTX-100 through the phase 2a trial with the potential to make the second phase 2b arm a registration study.

“Fast Track designation opens a dialogue with the FDA for companies to provide ongoing trial updates, instead of a lengthy review at the end, to expedite therapies to patients who desperately need them,” he said.

“All we need to do is replicate the Phase 1b results, if the FDA agrees, they could potentially authorise Phase 2b as a registration study.”

In the phase 1b trial PTX-100 delivered a 45% overall response rate and 64% clinical benefit in T-cell lymphoma, with just 4% serious side effects – well below the 30% benchmark.

“The first step now is to progress Phase 2a where we can define a dose which will also have some efficacy and safety data,” McDonnell said

“The registrational study means we are closer to approval and the momentum for PTX-100 really starts to accelerate and excitement grows on the commercial front.”

Watch: James McDonnell discusses the first US site for the Phase 2a trial of PTX-100.

Eyes on the commercialisation prize

With over 27,000 new TCL cases every year, in eight major centres, PTX is targeting a market worth ~US$1.8 billion.

Commercialisation of PTX-100 will become a key focus for Prescient if it advances to a registrational study, with all options on the table – including partnership, licensing, or taking the drug to market independently.

“We are confident in how PTX-100 could change the lives of thousands, we need the data now to back that confidence and provide the market a clear indication of the value the therapy can provide,” he said.

“PTX-100 is a first-in-class therapy with a unique mechanism of action which has shown very good results in the Phase 1b study.

“These results have exceeded our benchmarks and compare well to the available therapies on efficacy, duration and a safety perspective.“

McDonnell said that Dimerix (ASX:DXB) was a great example of what a potential commercial pathway could look like for Prescient if Phase 2b became a registrational trial.

Dimerix is advancing its lead candidate DMX-200 through the ACTION3 phase 3 trial for the rare kidney disease FSGS and has secured four regional licensing agreements to drive commercialisation.

“They were able to take orphan drug designated therapy DMX-200, which is currently in a phase 3 registration study, into significant licensing agreements worth approximately $1.4 billion,” he said.

“Dimerix have highlighted how these milestones, FDA designation and registrational study, can open commercialisation opportunities and drive value creation.”

McDonnell said in biotech it often comes down to two questions with the first being: can you get a product to market? The second is: what kind of market is waiting at the end?

Phase 2 trials usually provide clearer insights into a drug’s potential, with data readouts serving as critical indicators that can drive value inflection points.

Source: Prescient Therapeutics

A series of upcoming catalyst

Investors have plenty to watch out for in H2 CY25, with Prescient advancing its phase 2a study.

“We are excited for the release, once we reach 20 patients, of the initial data from the dose optimisation committee review, which will be a key event in determining the next steps for development of PTX-100,” McDonnell said.

A share purchase plan aimed at raising $7 million to support Prescient’s clinical program closed on July 25.

“There are several milestones we are looking to hit in the coming months and if we can potentially get that Phase 2b into a registration study we can start to shift our focus toward commercialisation opportunities,” McDonnell said.

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.