In short
PTX-100, also known as N-[[(2S)-4-[(4-METHYL-1H-IMIDAZOL-5-YL)METHYL]-3-OXO-2-(PHENYLMETHYL)-1-PIPERAZINYL]CARBONYL]-L-LEUCINE TRIHYDRATE, is a chemically synthesised substance being studied as a potential treatment for cutaneous T-cell lymphoma (CTCL). It has no marketing authorisation and is an investigational medicine supplied by Prescient Therapeutics Ltd.
Key points
- PTX-100 is an investigational, chemically synthesised substance with no marketing authorisation.
- It is being studied in cutaneous T-cell lymphoma (CTCL), a rare cancer of the skin.
- The single registered trial is a Phase 2 study in patients with relapsed or refractory CTCL.
- The trial plans to enrol 65 participants and is currently authorised in the EU.
- PTX-100 is given as an intravenous infusion at a dose of 1000 mg/m².
- The main outcome measured is the objective response rate (ORR), meaning how many patients see their cancer shrink or disappear.
What is PTX-100?
PTX-100 is the short name for N-[[(2S)-4-[(4-METHYL-1H-IMIDAZOL-5-YL)METHYL]-3-OXO-2-(PHENYLMETHYL)-1-PIPERAZINYL]CARBONYL]-L-LEUCINE TRIHYDRATE, a chemically synthesised small-molecule substance. It belongs to the therapeutic area of neoplasms, meaning it is being investigated in cancer. The substance is not authorised as a medicine in any country and remains an investigational product; the company supplying it in the registered trial is Prescient Therapeutics Ltd. The product records list PTX-100 as the only name for this substance.
How does PTX-100 work?
The registry records do not state the molecular target or mechanism of action of PTX-100. The trial is designed to determine the efficacy of the substance in patients with relapsed or refractory cutaneous T-cell lymphoma, but no source in the provided data explains how PTX-100 acts on cells. As a chemical substance given by intravenous infusion, it is studied as a monotherapy, meaning it is given alone and not in combination with another cancer medicine.
Which conditions is PTX-100 studied for?
- Cutaneous T-cell lymphoma (CTCL) — a rare type of non-Hodgkin lymphoma that primarily affects the skin. The registered trial focuses on patients whose disease has returned after previous treatment or has not responded to it (relapsed or refractory).
How is PTX-100 given?
PTX-100 is prepared as a solution for infusion and is given directly into a vein (intravenous infusion). In the registered Phase 2 trial, the dose reported is 1000 mg/m², meaning the amount is calculated according to the patient's body surface area. No other pharmaceutical forms are mentioned in the available data.
What do the clinical trials show?
One clinical trial of PTX-100 is registered in the EU Clinical Trials Information System. It is a Phase 2 study, which means it is testing how well the treatment works in a specific patient group after earlier phases have suggested it may be active. The trial is currently authorised, meaning it has received regulatory approval to proceed in the EU. The study is interventional, so participants receive the treatment according to a defined plan rather than being observed without intervention.
Who can take part in the trial?
The trial enrols patients with relapsed or refractory cutaneous T-cell lymphoma. The registry record does not list additional details such as age limits, prior treatments allowed, or other inclusion and exclusion criteria. The planned number of participants is 65.
What are the trials measuring?
The primary outcome of the trial is the objective response rate (ORR). ORR is the proportion of patients whose cancer shrinks by a predefined amount or disappears completely after treatment. The brief summary states that the purpose of the study is to determine the efficacy of PTX-100. No secondary outcomes are reported in the available data.
What side effects are known?
The registry record for this trial does not provide information about side effects of PTX-100. No supplementary sources describing known adverse reactions were included in the data, so no side effect profile can be reported here.