Hemogenyx Pharmaceuticals PLC (LSE:HEMO, OTC:HOPHF) hailed a "major milestone" as it opened the first trial site and began recruiting patients for the clinical study of its lead drug as a potential treatment for recurrent leukaemia.
This Phase I trial of HG-CT-1 in relapsed or refractory acute myeloid leukaemia (R/R AML) in adults is designed as a dose-escalation study to evaluate the safety profile of the drug.
Secondary objectives will also be assessed, including what effects HG-CT-1 has on certain AML-specific criteria, overall survival rates among participants, the rate of progression-free survival and the duration of response in those who achieve clinical responses.
The company said these objectives are "pivotal" for assessing the overall clinical impact of the drug on patients with R/R AML, as there are few remaining therapeutic options for these people.
Hemogenyx also noted that the trial is being carried out at one of the world's most prestigious cancer research institutions.
Dr Vladislav Sandler, chief executive and co-founder, said the opening of our first clinical site "is a milestone in our mission to revolutionize the treatment landscape for relapsed and refractory acute myeloid leukaemia.
"This trial offers hope to patients who currently face limited treatment options by exploring the safety and potential efficacy of HG-CT-1. We are excited to start this pivotal study."
AML is the most common type of acute leukaemia in adults and has a five-year survival rate of less than 30% in adults.
With chemotherapy currently the main form of treatment, HG-CT-1 is based on a potentially more benign and effective CAR-T therapy, which uses and modifies a patient's own immune cells to recognize and kill the patient's cancer cells.
The procedure involves: isolating T-cells from the patient; modifying the isolated T-cells in a laboratory using a CAR gene construct (which allows the cells to recognize the patient's cancer); amplifying (growing to large numbers) the newly modified cells; and re-introducing the cells back into the patient.