In short
PM54 is an investigational anticancer drug, a chemically synthesised substance being studied for the first time in humans. It is currently in a Phase 1 basket trial in combination with the immunotherapy pembrolizumab in 148 adults with advanced malignancies. The trial is authorised and is testing the safety, tolerability and recommended dose of PM54 when given by intravenous infusion.
Key points
- PM54 is an experimental anticancer drug with no marketing authorisation in the product records.
- The only registered trial is a Phase 1 basket trial in advanced malignancies.
- The trial combines PM54 with pembrolizumab, an immunotherapy, both given by intravenous infusion.
- 148 participants are planned for enrolment in the authorised trial.
- The main goals are safety, tolerability and finding the recommended dose of PM54.
- PM54 is supplied by Pharma Mar, S.A. as a powder for concentrate for solution for infusion.
What is PM54?
PM54 is a chemically synthesised substance being developed as an anticancer drug. It is an investigational medicine, meaning it has not received a marketing authorisation and is not yet approved for routine use. The product records list Pharma Mar, S.A. as the company supplying the substance. In the registered trial, PM54 is the test substance, which means the trial is designed to investigate its effects. PM54 is described in research documents as an experimental anticancer drug that has been shown to kill cancer cells in animal studies.
How does PM54 work?
The exact mechanism of action of PM54 is not stated in the available registry data or the supplementary source. What is known is that PM54 has shown the ability to kill cancer cells in several animal studies, and the current clinical trial aims to find out whether this effect is also present in humans. The trial combines PM54 with pembrolizumab, an immunotherapy. Pembrolizumab is a type of medicine that helps the body's own immune system to recognise and attack cancer cells. The combination is being tested to see whether PM54 and pembrolizumab can be given safely together.
Which conditions is PM54 used or studied for?
- Advanced malignancies — the registered trial includes adults with advanced cancers. A basket trial means that participants with different types of advanced cancer can be included in one study.
The earlier research summary describes a study in patients with selected advanced solid tumours, where the second stage would enrol patients into at least five tumour-specific groups.
How is PM54 given?
PM54 is prepared as a powder for concentrate for solution for infusion and is given by intravenous infusion, which means it is delivered directly into a vein. In the registered trial, pembrolizumab is also given by intravenous infusion.
What do the clinical trials show?
There is one registered clinical trial of PM54. It is a Phase 1 basket trial in adult participants with advanced malignancies, with a planned enrolment of 148 people. The trial is authorised, which means it has received regulatory approval to begin.
The trial is divided into two parts. Parts 1 and 2 evaluate the safety and tolerability of PM54 in combination with pembrolizumab. Part 1 is specifically designed to determine the recommended dose of PM54 when given together with pembrolizumab.
The main measurements in the trial are treatment-emergent adverse events, serious treatment-emergent adverse events, dose-limiting toxicities, and treatment-emergent adverse events that lead to treatment discontinuation or dose modifications. A dose-limiting toxicity is a side effect severe enough to prevent increasing the dose further. These measurements are used to determine the recommended dose. Part 1 also specifically measures dose-limiting toxicities during the dose-limiting toxicity assessment period.
The earlier research summary describes a similar study design in which small groups of people receive increasing doses of PM54 until the highest safe dose is found, followed by a second stage with larger groups of patients in at least five tumour-specific groups.
What side effects are known?
The registered trial is designed to measure side effects, but no specific side effects of PM54 are listed in the registry data. The research summary notes that some patients may experience important or serious side effects, and that these are more likely to occur as the dose of PM54 is increased. The dose will stop being increased when certain side effects are seen.