On March 15, 2023 Prescient Therapeutics (ASX: PTX), reported that it has received an expanded Orphan Drug Designation (ODD) from the Food and Drug Administration (FDA) for its targeted cell therapy PTX-100 that now extends to all T-cell lymphomas (TCLs) (Press release, Prescient Therapeutics, MAR 15, 2023, View Source;utm_medium=rss&utm_campaign=ptx-receives-expanded-orphan-drug-designation-for-all-t-cell-lymphomas [SID1234628794]). This is an encouraging signal from the US regulatory agency, who had already awarded the asset ODD for its potential to treat Peripheral T-cell Lymphoma – and aggressive cancer with an average 5-year survival rate of 32%.

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Prescient had applied for ODD due to the asset’s potential to treat Cutaneous T-cell Lymphoma (CTCL). In a pleasant surprise, the FDA granted the designation broader than PTX’s request. There are over 20 different types of TCL, so this significantly broadens the scope of PTX-100’s potential.

The FDA grants ODD status to drugs and biologics that are intended to treat rare diseases or conditions affecting fewer than 200,000 people in the United States. While drugs with ODD designation still have to go through the same rigorous scientific review process that any other drug must complete for approval or licensing, there is a correlation between receiving the designation and approval: between 2010 and 2016, 75% of all novel cancer drugs that received approval in the United States also qualified as orphan products. During the same time period, the average time between receiving ODD and approval was 5.3 years.

Investors tend to place value in the awarding of an ODD on a therapy. Specifically for smaller biotech companies, the average increase in share price or cumulative abnormal return following an ODD announcement has been found to be 8.87% for nano-caps (

An update on the study, which is being expanded to include up to 12 additional patients with TCL, is due imminently.

The focus of the current expansion cohort are patients with relapsed and refractory TCLs. The company has already achieved a partial response in a patient with aggressive PTCL. The response endured for over 32 months before the disease progressed. Importantly, this response was achieved in a patient that had failed five prior lines of therapy.

At the October 2022 update, one of the patients with CTCL, who had failed four prior lines of therapy, had a very good partial response (VGPR) that had endured beyond 6 months. Visually evident reduction in skin abnormalities were recorded and described by the Principle Investigator of the study, haematologist Professor H. Miles Prince, as "impressive responses". The patient remained on therapy. Two other patients with CTCL that failed three prior lines of therapy have experienced stable disease (SD) that had endured for 4 months. PTX-100 is a first in class compound that blocks an important cancer growth enzyme known as geranylgeranyl transferase-1 (GGT-1). This disrupts oncogenic Ras pathways by inhibiting the activation of Rho, Rac and Ral circuits in cancer cells which leads to apoptosis (death) of the cell. More than 30% of all human cancers, including 95% of pancreatic cancers, 45% of colorectal cancers, 35% of lung cancers and 15% of acute myeloid leukaemia – are driven by mutations of the RAS family of genes.