Prescient Therapeutics gets FDA orphan drug status for PTX-100 in PTCL


Prescient Therapeutics has secured the orphan drug designation for its PTX-100 compound from the US Food and Drug Administration (FDA) for the treatment of peripheral T-cell lymphomas (PTCL).

According to the Australian oncology company, the FDA orphan drug designation will enable it in getting incentives that can help the development of PTX-100, which is a prenylation inhibitor that is designed to disrupt oncogenic Ras pathways in cancer cells.

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In the dose escalation of a phase 1b basket study, PTX-100 displayed an encouraging efficacy signal in peripheral T-cell lymphomas, said Prescient Therapeutics. Currently, the compound is being assessed in an expansion cohort made up of 12 patients who have relapsed and refractory T cell lymphomas (TCL), including peripheral T-cell lymphomas.

Prescient Therapeutics gets FDA orphan drug status for PTX-100 in PTCL

Prescient Therapeutics gets FDA orphan drug status for PTX-100 in PTCL. Photo courtesy of Bluerasberry/

The expansion cohort of the PTX-100 phase 1b trial is scheduled to fully recruit patients this year.

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Steven Yatomi-Clarke — Prescient Therapeutics Managing Director and CEO said: “The granting of Orphan Drug Designation by the FDA is significant for Prescient’s development of PTX-100. Orphan drugs often enjoy shorter and cheaper development pathways. Additionally, the Company now has the certainty of 7 years of market exclusivity in the event of regulatory approval of PTX-100 for PTCL.

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“PTCL is a disease of unmet need, where safer and more effective therapies 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.”

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