Hemogenyx Pharmaceuticals plc (LSE: HEMO) has announced the advancement of its lead asset, HG-CT-1 (also known as HEMO-CAR-T), into Phase I clinical trials. The trial, designed as a dose escalation study, aims to evaluate the safety of HG-CT-1 in adult patients with relapsed/refractory (R/R) acute myeloid leukaemia (AML). The company has successfully completed the Site Initiation Visit at the first clinical site, marking a significant milestone in the development of this innovative therapy.
Patient recruitment is poised to commence once the clinical site finalizes details with Prevail Infoworks, the company's clinical trials manager. This study is a crucial step in addressing the unmet medical needs of patients with R/R AML, a condition characterized by limited treatment options and poor prognosis.
Clinical Trial Objectives
The Phase I trial's primary objective is to assess the safety profile of HG-CT-1 in adult patients with R/R AML. Secondary objectives include:
- Evaluating the efficacy of HG-CT-1 based on AML-specific response criteria.
- Assessing overall survival (OS) rates among participating subjects.
- Measuring progression-free survival (PFS) in evaluable subjects.
- Determining the duration of response (DoR) in those who achieve clinical responses.
These objectives are critical for determining the clinical impact of HG-CT-1 on patients with R/R AML, a population with few remaining therapeutic options.
Addressing Unmet Needs in AML Treatment
AML, the most common type of acute leukemia in adults, is associated with poor survival rates, with a five-year survival rate of less than 30%. Current treatments primarily involve chemotherapy, which can be harsh and often ineffective in R/R cases. Hemogenyx Pharmaceuticals aims to provide a more benign and effective therapy through the development of HG-CT-1.
CAR-T Therapy: A Novel Approach
CAR-T therapy involves modifying a patient's own T-cells to recognize and kill cancer cells. The process includes:
- Isolating T-cells from the patient.
- Modifying the T-cells in a laboratory using a CAR gene construct.
- Amplifying the modified cells to large numbers.
- Re-introducing the cells back into the patient.
This innovative approach offers a potential alternative to traditional chemotherapy, with the goal of improving treatment outcomes and survival rates for AML patients.
Management Commentary
Dr. Vladislav Sandler, CEO & Co-Founder of Hemogenyx Pharmaceuticals, stated, "This step marks a significant milestone for the Company as we advance our lead asset, HG-CT-1, into clinical trials. This study represents our commitment to developing innovative therapies for patients with R/R AML, a condition with limited treatment options. We are excited to see patient recruitment set to begin and take the next steps in assessing the safety and treatment potential of HEMO-CAR-T. We remain dedicated to delivering transformative therapies that address critical unmet medical needs."
