The granting of an Orphan Drug Designation by the US FDA will grant PTX-100 market exclusivity and other commercial benefits in blood cancer PTCL.

Clinical stage oncology company Prescient Therapeutics (ASX:PTX) announced that the US FDA has granted an Orphan Drug Designation to PTX-100, for the treatment of peripheral T-cell lymphomas (PTCL).

The Orphan Designation is used by the FDA to provide status to certain drugs which show promise in the treatment of rare diseases, defined as those that affect less than 200,000 persons in the US.

It’s effectively designed to provide benefits to incentivise drug development in less common diseases.

The benefits of being granted this Designation are considerable, and include guaranteed market exclusivity of seven years from the time of approval.

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

Prescient says today’s announcement adds tremendous value to  PTX-100, a first-in-class prenylation inhibitor that disrupts oncogenic Ras pathways in cancer cells.

In a Phase 1b basket study, a dose escalation of PTX-100 has shown an encouraging efficacy signal, as well as an excellent safety profile, in PTCL patients.

The clinical trial is now in an expansion cohort of 12 patients with relapsed and refractory T cell lymphomas (TCL), including PTCL.

This expansion cohort is due to fully recruit this year, and is led by globally-renowned lymphoma expert, Professor H. Miles, at the Epworth Hospital in Melbourne.

Faster route to market

PTCL is a blood cancer with substantial unmet need for new therapies, and represents an exciting clinical and commercial opportunity for PTX-100.

According to available data, survival following relapse of PTCL is poor, and has not significantly improved in the last 20 years.

Whilst it’s not a common malignancy, the nature of the disease and the lack of current effective treatment options for these patients creates a potentially shorter regulatory path and unique market opportunity for PTX-100.

Prescient believes that this will allow PTX-100 a faster route to market in a high value area of unmet clinical need.

PTX-100 works by blocking an important cancer growth enzyme called GGT-1. It was co-invented by Prescient’s scientific founder, Professor Said Sebti, and is exclusively licensed by Prescient from Yale University.

Strong early indicators for PTX-100 as a T-cell lymphoma treatment offer significant market potential. Its excellent safety profile also gives it a possible use-case as a treatment for patients who are unable to tolerate higher toxicities.

Big step towards treating PTCL

Currently, available therapies for PTCL are typically characterised by high occurrence of serious toxicities.

For example, they have low response rates (<30%) and short duration of responses (3-4 months).

PTX-100 on the other hand is exhibiting a favourable safety profile with encouraging efficacy signals, including one PTCL patient with a durable response that is still on therapy after 28 months.

Prescient CEO Steven Yatomi-Clarke said the granting today is a big step forward towards developing a new treatment for this disease.

“The granting of Orphan Drug Designation by the FDA is significant for Prescient’s development of PTX-100,” he said.

“Orphan drugs often enjoy shorter and cheaper development pathways. Additionally, the company now has the certainty of seven years of market exclusivity in the event of regulatory approval of PTX-100 for PTCL,” he added.

Yatomi-Clarke also said that more effective therapies for PTCL will be welcomed by patients and clinicians.

“As our expansion cohort unfolds, ultimately it will be supporting clinical data and Orphan Drug Designation that will combine to help bring PTX-100 to patients with this challenging disease.”

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.