CEO James McDonnell is excited:

Tackling a cancer of unmet need – why it matters

Cutaneous T-Cell Lymphoma (CTCL) is a rare but relentless form of non-Hodgkin lymphoma that primarily affects the skin, blood and internal organs. Unlike many cancers, CTCL begins in the skin and is often misdiagnosed in its early stages.

It progresses into painful, disfiguring lesions and tumors and for many patients, the quality of life can deteriorate dramatically as the cancer becomes more aggressive. Patients may endure years of physical discomfort, emotional isolation, and worsening symptoms with no clear end in sight.

Despite its devastating impact, CTCL remains a cancer of true unmet need with no known cure. Current treatment options have limited effectiveness, and these treatments can come with side effects 98% of the time – of which 36% are classified as serious, adding a heavy burden to an already difficult disease.

This combination of complexity and lack of options makes CTCL a compelling focus for new, better-tolerated treatments like PTX-100, which has shown outstanding results in the Phase 1b trials and is now in Phase 2.

Even though CTCL is classed as an orphan cancer, the market is US$1.8 billion which provides Prescient with a significant opportunity if it can get PTX-100 to market. Its outstanding Phase 1b results and FDA designations so far give them a lot of confidence.

A turning point – PTX-100 leads the way

PTX-100 is hoping to shift the balance on CTCL and save lives on the back of their outstanding Phase 1b clinical trials across the main three factors – response rate, duration and safety.

An incredible 64% of patients reported tumor halt or shrinkage and the duration of the patients’ response was longer than existing drugs.

The safety profile is vital for such fragile patients and this is where PTX-100 has excelled, showing only 4% serious adverse side effects (1 case of diarrhea) – a fraction of the 30% target.

The FDA is responding. They are looking for drugs that last longer and are safer than already approved products (like Lymphir) to improve patient lives for this cancer. Safety is particularly important, not just because the patients are so fragile but also to enable the drug to be safely paired up with other therapies like chemo.

PTX-100 comparison results (source Prescient Therapeutics)

The path to market for PTX-100

With over 27,000 new TCL cases every year, in 8 major centres, PTX is now aiming to get its first application into market as fast as possible.

FDA support is critical and has proven they are paying attention with two key designations.

Overview of FDA review pathways and designations (source FDA US Food and Drug Administration)

The first is Orphan Drug Designation, which is an incentive regulators give to companies developing drugs which are addressing an orphan disease which is serious but generally too small for big pharma. The main benefit is seven years of market exclusivity once a drug is in the market, which could be a huge boost for Prescient’s commercial strategy.

Second is Fast Track Designation, which PTX has recently received. As the name implies, it allows for a faster process with the FDA through increased access. This is key to moving things forward without delay; the increased communication provides the opportunity for PTX to turn Phase 2b into a registrational study, after which the drug could go straight into the market.

If the FDA deems it appropriate for Phase 2b to be the registrational study, PTX-100 could skip years of additional trials. Patients would then have quicker access to a drug that potentially has a better chance of improving and extending their lives and has the potential to be used alongside other key treatments like chemotherapy.

The impact these FDA approvals can have for a company can be seen in some biotech success stories. Mirati Therapeutics’ flagship drug transitioned from Phase 1 trials to commercialisation in as little as four years with FDA designations and was acquired by Bristol Myers Squibb for $4.8 billion – just 10 months after receiving accelerated approval.

This shows how close PTX may be to a rerate event as it progresses through Phase 2 – which is why CEO James McDonnell is excited:

“If Phase 2a trials show results anywhere near Phase 1b, then things will get very exciting. We could be in the market in very few years. The support we have received from the FDA so far plus our Phase 1 results give us a lot of confidence.”

The larger opportunities beyond CTCL

PTX-100’s Phase 2 trial is only focused on CTCL as it represents their most near-term, tangible opportunity showing significant results, but the company is no one-trick pony.

This is because PTX-100 is a first-in-class RAS Pathway Disruptor Platform technology which could apply to up to 22% of all cancers, including lung, bladder, ovarian and pancreatic cancers, to name a few.

So there is a broader potential to improve the lives of millions of patients worldwide.

On top of this, PTX is also advancing its proprietary OmniCAR and CellPryme platforms which are relevant to CAR-T therapies which are considered the next generation of cancer care.

A recent breakthrough immunotherapy trial using CAR-T cell therapy to wipe out solid tumours has just reported results that patients treated with the therapy lived on average approximately 40% longer. Dr John Haanen, of the Netherlands Cancer Institute, commented that he thinks this is the new generation of treatment, opening significant opportunities to contribute to future groundbreaking therapies.

OmniCar is Prescient’s next generation CAR-T platform designed to give unprecedented control over cell therapies. It has the potential to allow CAR-T therapy cells to be more targeted, safer, more effective, cost-effective and of longer duration.

CellPryme is a complementary application to OmniCar split into two components. CellPryme-M produces superior cells that are more potent and last longer, aiming to double tumour control. CellPryme- A act as an adjuvant therapy, increasing the expansion of CAR-T cells and enhancing their ability to penetrate into the tumour.

Multiple shots on goal for Prescient – one of which is now very close

Prescient is a company on a mission to change cancer care, thereby changing the lives of many thousands.

This provides the company with several shots on goal, but its most advanced drug is the one CEO James McDonnell is focussed on.

“I’ve got a drug in phase 2 for a horrible disease of unmet need where we have high confidence from existing data and FDA designations to take it into a US$1.8 billion market within a few years. That’s the near-term opportunity and 90% of my focus. But yes, the wider opportunity is compelling and with our other applications of PTX-100 plus our CAR-T platform we have a lot of shots on goal.”

With new global cancer cases projected to increase by 77% from 20 million in 2022 to over 35 million by 2050, there is ample need for companies like Prescient to make a difference.

Click here to join an upcoming webcast with Prescient Therapeutics CEO James McDonnell, to discuss how their advanced cancer therapy could improve thousands of lives by tackling a brutal cancer of unmet need.

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