The granting of an Orphan Drug Designation for PTX-100 gives Prescient guaranteed market exclusivity of 7 years.

The US FDA has just granted Prescient Therapeutics (ASX:PTX)’s PTX-100 additional Orphan Drug Designation (ODD) for the treatment of all T-cell lymphomas (TCL), including cutaneous TCL (CTCL).

Prescient had applied for an approval for CTCL after having separately received an Orphan Drug Designation  for peripheral TCL in 2022.

The FDA has now granted a broader designation than the one Prescient requested, which encompasses all TCLs – a group of lymphomas that develop when a group of white blood cells called lymphocytes grow out of control.

There are different groups of TCL, CTCL and Peripheral T-cell lymphoma (PCTL), each with several distinct subtypes. Collectively, TCLs represent an area of unmet or poorly-met patient need, especially in patients with relapsed or refractory disease.

The Orphan Drug Designation today covers all TCLs and their subtypes.

“Prescient is delighted to be granted this Orphan Drug Designation by the FDA, and is pleasantly surprised for the granting of the designation that is broader than our request,” commented Prescient CEO, Steven Yatomi-Clarke

“This now confers the certainty of 7 years of market exclusivity for PTX-100 in a broader range of diseases with unmet or poorly met clinical need. We look forward to sharing updates on the PTX-100 trial shortly.”

Considerable benefits of having ODD status

PTX-100 is Prescient’s first-in-class prenylation inhibitor that disrupts oncogenic Ras pathways in cancer cells.

Relapsed and refractory TCLs are the focus of the current expansion cohort of the PTX-100 Phase-1b study under the leadership of globally-renowned lymphoma expert, Professor H. Miles Prince, with an update due imminently.

The FDA approval today is a crucial milestone for Prescient as an Orphan Drug Designation will allow the company to benefit from incentives that can assist the development of PTX-100.

The ODD program provides orphan status to drugs which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the US.

It is designed to provide benefits to incentivise drug development in less common diseases.

The benefits of obtaining an ODD  status are considerable,  and include guaranteed market exclusivity of seven years from granting of regulatory approval.

A designation also allows for a waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs, which has a value of over US$3.1 million in 2022.

A major opportunity beckons

Prescient believes that PTX-100 represents a major opportunity to address the unmet needs of a patient group with few options available.

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

The drug has demonstrated safety and early clinical activity in a previous Phase-1 study and recent PK/PD basket study of hematological and solid malignancies.

PTX-100 is now in a Phase-1b expansion cohort study in T cell lymphomas, where it has shown encouraging efficacy signals and safety.

Meanwhile, Prescient and its partners continue to demonstrate how its CAR platform was more effective at targeting a wider range of cancer tumours compared to current first generation CAR-T treatments.

Several of its OmniCAR studies have now advanced through a number of important preclinical proof of concept trials, as the company progresses this next-generation cell therapy platform.

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.