One of the most advanced cancer therapies on the ASX, Prescient Therapeutics’ (ASX: PTX) PTX-100 is currently in Phase 2a trials for its first targeted cancer, Cutaneous T-Cell Lymphoma (CTCL), which many clinicians see as a death sentence in the advanced stage.

Phase 1b trials remarkable results saw a 100% of evaluable CTCL patients with a halt or reversal of their cancer – even more remarkably, zero drug-related serious adverse events.

PTX has received both FDA and EMA Orphan Drug designations and FDA Fast Track Designation for PTX-100, with the goal of getting the therapy to market as fast as possible.

With Phase 2b trials holding the potential to serve as a registrational study, PTX is approaching a pivotal moment and the exact stage of development where typically big pharma come in and make a deal.

This gives Prescient a shot at becoming one of Australia’s biotech success stories.

PTX-100: A first-in-class therapy

PTX have developed a unique technology, PTX-100, that disrupts the RAS-pathway, implicated in approximately 22% of all cancers. It has the potential to act as a platform and reach beyond a single disease.

PTX has chosen to first focus on Cutaneous T-Cell Lymphoma (CTCL) as a rare, potentially aggressive blood cancer of genuine unmet need. Existing treatment options are poor with current drugs providing only modest efficacy and serious adverse side effects.

In Phase 2a, a Dose Optimisation Committee meeting is expected before the end of the year, following patient recruitment across Australia, US and Italy. The committee will provide guidance on the optimal dose for the next stage of the program and will form part of the Phase 2b trial design.

With several significant milestones ahead, that if positive, there is potential for the Phase 2b trial to be designated as an accelerated-approval pathway and registrational study – which could see PTX-100 reach market without running a separate Phase 3 study – and the development stage is where large pharmaceutical companies generally aim to get deals done.

At the stage where deals get done

The biotech industry is in a dealmaking supercycle driven by looming patent expiries and generic competition. Big pharma’s deal capacity was estimated at around US$1.3 trillion1 at the start of 2026 with biotech M&A hitting US$84 billion in Q1 2026 alone – nearly

double the same period last year.2

Large pharmaceutical companies need new, differentiated drugs that shore up their pipeline. The problem is funding a drug candidate from scratch means years of risk, and most never make it through the clinic. So rather than explore, large pharmaceuticals would rather buy de-risked assets with near-term commercial potential.

The logic mirrors mining – a major would rather acquire an asset with proven resources than fund high-risk exploration from scratch.

Most licensing deals and acquisitions in rare disease happen in pivotal studies, before registration or commercialisation. A Phase 2 asset has already generated favourable results in humans, making its regulatory and commercial potential far more visible. Sobi’s move on Innate Pharma may be an illustration of the type of deal that large pharma is hunting for.

Innate’s Lacutamab is a first-in-class antibody which received Breakthrough Designation after Phase 1 and showed promising late Phase 2 results in Sézary Syndrome, an aggressive subtype of CTCL. In August 2026, they announced a strategic global licence – before initiation of the confirmatory Phase 3 study – totalling US$580 million which included US$75 million upfront.

“We think we’re a natural fit for pharma looking for a differentiated drug, particularly focused on rare diseases, like CTCL. As we progress toward a potential registrational study, that’s what makes an asset like ours attractive to the larger players in this space.” – James McDonnell, CEO, Prescient Therapeutics

A major inflection point for PTX

A first-in-class technology, PTX-100 is understood to be the only drug of its kind in clinical development. Currently in Phase 2a, there is a tangible opportunity for PTX to enter a US target market estimated to be US$1.2 Billion by 20343, with several significant milestones ahead in the next 12 months.

With regulatory acknowledgment through Fast Track and Orphan Drug Designation, giving greater access to the FDA and market exclusivity in the US (7 years) and the EU (10 years).

PTX is set for its next stage of development, led by an experienced team of drug developers and deal-makers with a track record in blood cancers, and experience gained across global companies including Bristol Myers Squibb, GSK, Novartis and Amgen.

PTX offers a genuine first-in-class drug that could be applicable to 1 in 5 cancers, is backed by remarkable Phase 1b results and key FDA designations, and is positioned to take advantage of the exact stage of development at which the industry’s deals are done.