Hemogenyx has received the all-clear from the US Food and Drug Administration (FDA) to begin a Phase I clinical trial of its acute myeloid leukaemia (AML) chimeric antigen receptor (CAR) T-therapy in the US.

In June 2023, the US FDA placed a clinical hold on the trial for HEMO-CAR-T, requesting additional information from Hemogenyx after a splicing deficiency during manufacture of the lentivirus used to produce CAR-T cells. Hemogenyx produced a plan, supported by laboratory tests to address FDA’s concerns in August 2023, which was accepted the following month. In January, the London, UK-based biopharma issued a complete response to the agency to lift the hold.

Hemogenyx’s stock price has increased by 62% following the announcement of the lifting of the hold today (9 February) since the market close yesterday.

CAR-T therapies have been hitting the headlines recently after the FDA launched an investigation into the incidence of potentially associated secondary malignancies in November. A month later, Johnson & Johnson and Legend Biotech added a boxed warning to jointly-developed therapy Carvykti (ciltacabtagene autoleucel; cilta-cel) after Phase II data from the CARTITUDE-1 trial (NCT03548207) showed that myeloid neoplasms occurred in 10 out of 97 patients post treatment. Last month, the FDA added similar boxed warnings on additional CAR-T therapies.

Despite this, the CAR-T cell therapy market is still expected to grow, with many professionals arguing that the benefits outweigh the risks associated with the therapies. According to a report on GlobalData’s Pharma Intelligence Center, the total market value for both cell therapies and established/traditional AML therapies, is expected to increase from $3.1bn in 2021 to $6.62bn in 2031 globally.

AML, a cancer that affects the bone marrow and blood, is characterised by the rapid growth of abnormal white blood cells, which interfere with the production of normal blood cells. The current standard of care for AML is chemotherapy, with the cancer generally having a bad prognosis.

HEMO-CAR-T comprises T cells genetically engineered to express chimeric antigen receptors, targeting cells expressing Fms-like receptor tyrosine kinase 3 (FLT3). T cells are extracted from the patient, modified to target specific proteins on cancer cells, and then infused back into the patients’ bloodstream.

In the announcement accompanying the announcement, CEO of Hemogenyx Vladislav Sandler said: “We are extremely pleased with the FDA’s decision to lift the clinical hold. We now look forward to accelerating clinical development of HEMO-CAR-T and to offering patients a potentially life-saving treatment.”