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FDA Accepts Hemogenyx Pharmaceuticals' Plan to Address Clinical Hold for AML CAR T-Cell Therapy

NEW YORK – The US Food and Drug Administration has accepted how Hemogenyx Pharmaceuticals plans to address the agency's concerns about its investigational acute myeloid leukemia CAR T-cell therapy, the company said Thursday.

The FDA placed a clinical hold on an investigational new drug (IND) application for the firm's autologous HEMO-CAR-T product in June due to a splicing issue that occurred during the manufacturing of the lentivirus used to produce the CAR T-cells. Hemogenyx responded to the FDA with a plan to address its concerns last month, and the agency has indicated it is satisfied with the firm's proposed course of action.

In a July announcement, Hemogenyx said it had identified the source of the splicing deficiency and developed a way to fix it and was already remanufacturing the lentivirus. The London-headquartered company said the FDA has also provided suggestions for improving the safety of HEMO-CAR-T that are unrelated to the clinical hold and "can be dealt with readily."

Early in the development of HEMO-CAR-T, Hemogenyx partnered with the University of Pennsylvania to conduct proof-of-concept studies in acute myeloid leukemia.

According to Hemogenyx CEO Vladislav Sandler, the firm is now implementing its plan to address the splicing deficiency and will resubmit its IND to the FDA seeking permission to begin a Phase I trial of HEMO-CAR-T.