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Highlights
- FDA Clears Phase 2 IND: The U.S. FDA has approved Prescient Therapeutics' Investigational New Drug (IND) application for its Ras pathway inhibitor, PTX-100, targeting advanced cutaneous T-cell lymphomas (r/r CTCL).
- Strong Phase 1b Results: Data from the Phase 1b trial shows a 42% overall response rate, with responders achieving a median progression-free survival of 12.2 months.
- Stock Soars: PTX shares gained 24% in morning trade on December 23 following the announcement.
Shares of Prescient Therapeutics Limited (ASX:PTX) surged 24% during morning trading on 23 December 2024 after the clinical-stage oncology company announced that the U.S. Food and Drug Administration (FDA) had cleared its Investigational New Drug (IND) application for the Phase 2 clinical trial of PTX-100. This development marks a significant milestone for the company as it advances its innovative cancer therapy for relapsed and refractory cutaneous T-cell lymphomas (r/r CTCL).
FDA Approval and Phase 2 Trial Details
The FDA’s IND clearance allows Prescient to initiate its Phase 2 trial of PTX-100, the company’s first-in-class Ras pathway inhibitor. This trial builds on the promising results from the Phase 1b study, which reported an overall response rate of 42% in T-cell lymphoma (TCL) patients and demonstrated PTX-100’s excellent safety profile across doses ranging from 500 to 2,000 mg/m².
In the Phase 1b trial, five out of seven evaluable CTCL patients showed clinical benefit, with responders achieving a median progression-free survival (PFS) of 12.2 months—significantly exceeding the typical PFS of 3.1 months associated with the current standard of care, vorinostat.
The upcoming Phase 2 trial will involve up to 115 r/r CTCL patients globally across 15 sites, including Australia, the United States, and eventually Europe. The trial will be led by global TCL expert Professor H. Miles Prince, AM, out of Melbourne.
Trial Protocol and Objectives
The Phase 2 trial will be conducted in two parts:
- Phase 2a: Dose optimization in up to 40 patients, with randomization between two dosing groups (500 mg/m² and 1,000 mg/m²). A safety review committee will determine the optimal dose for the next phase, with the possibility of interim analysis.
- Phase 2b: Efficacy and safety evaluation in approximately 75 patients.
The primary endpoint is the objective response rate (ORR), which measures the proportion of patients achieving partial or complete responses. Secondary endpoints include progression-free survival, duration of response, overall survival, and quality of life, among others.
Prescient remains optimistic that the Phase 2b data could be considered for FDA registration, which would accelerate the availability of PTX-100 for patients in need.
Market Reaction
Investors reacted positively to the news, driving PTX shares up 24% to 18 cents in morning trade on 23 December 2024. The FDA clearance validates the company’s progress and reinforces confidence in its innovative oncology pipeline
