Prescient Therapeutics’ PTX-100 Progresses to Phase 2a Trials after Outstanding Phase 1b Results

Clinical-stage oncology company Prescient Therapeutics (ASX: PTX) is looking to disrupt a US$1.8 billion market on the back of outstanding Phase 1b clinical trial data for lead candidate PTX-100 to treat cutaneous T-cell lymphoma (CTCL).

Now in its Phase 2a trial, the advanced drug has delivered a 64% tumour halt or shrinkage in patients with the rare but relentless cancer, with only 4% reporting serious adverse effects.

This compares to incumbent therapies which have demonstrated a 36% chance of any relief and adverse effects in 98% of patients.

Success Story

Chief executive officer James McDonnell said the trial results give Prescient a shot at becoming one of Australia’s biotech success stories.

“PTX-100 has given us highly-encouraging clinical data and we are now looking to advance our clinical program toward registration,” he said.

“It is an exciting development which could potentially be applied to 22% of all cancers, so there is a significant opportunity for us to touch millions of lives with this potentially life-changing drug.”

FDA Designations

Prescient has received key designations from the US Food and Drug Administration, including orphan drug and fast track status, potentially speeding up commercialisation.

The FDA grants orphan drug designation to drug companies that are addressing a serious disease within a market that is generally considered too small for “big pharma”.

It provides seven years of market exclusivity, which Mr McDonnell said could be a huge boost for Prescient’s commercial strategy.

Fast track designation provides the opportunity for Prescient to turn its Phase 2b trial into a registrational study, after which the drug could bypass years of additional trials and go straight to market.

Painful Disease

CTCL is a form of non-Hodgkin’s lymphoma that primarily affects the skin, blood and internal organs.

Unlike many cancers, it begins in the skin and is often misdiagnosed in its early stages, progressing into painful and often disfiguring lesions and tumors.

Patients may endure years of physical discomfort and worsening symptoms and their quality of life can rapidly deteriorate as CTCL becomes more aggressive.

Unmet Need

Despite its devastating impact, CTCL remains a cancer of unmet need with no known cure and current treatment options of only limited effectiveness with often serious side effects.

“The combination of complexity and lack of options makes CTCL a compelling focus for new and better tolerated treatments like PTX-100,” Mr McDonnell said.

“Even though it is classed as an orphan cancer, it has a huge addressable market and the designations we have received from the FDA gives us a lot of confidence moving forward.”

Safety Profile

Mr McDonnell said PTX-100 had excelled in its safety profile for TCL patients.

“The FDA is looking for drugs that last longer and are safer than already-approved products in order to improve patient lives,” he said.

“Safety of PTX-100 is particularly important not just because patients are fragile but also to enable the drug to be safely paired up with other treatments such as chemotherapy.”

Cell Therapy Platforms

Prescient is also advancing its proprietary OmniCAR and CellPryme platforms relevant to CAR-T therapies, considered to be the next generation of cancer care.

OmniCar offers unprecedented control over cell therapies and has the potential to allow CAR-T therapy cells to be more targeted, safer and more cost-effective.

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

A recent breakthrough immunotherapy trial using CAR-T cell therapy to wipe out solid tumours showed that treated patients could survive approximately 40% longer on average than untreated patients.

Changing Cancer Care

Mr McDonnell said Prescient was working hard on its mission to change cancer care.

“PTX-100 is the near-term opportunity for us and consumes 90% of our focus,” he said.

“But the wider opportunity is compelling and with our CAR-T platform, we believe we have a lot of opportunities.”