The FDA has accepted the company’s plan to address its current IND clinical hold on HEMO-CAR-T.
Hemogenyx Pharmaceuticals has reached an accord with the FDA on its plan to resolve the agency’s current clinical hold on its HEMO-CAR-T investigational new drug application (IND) for treating acute myeloid leukemia (AML).1
"We are pleased that the FDA has agreed to our plan and preliminary test results to address their concerns regarding our HEMO-CAR-T IND application. We are now working hard to complete the schedule of work set out in the plan and to re-submit the IND as expeditiously as possible in order to move forward with clinical trials of HEMO-CAR-T,” Vladislav Sandler, PhD, chief executive officer and cofounder, Hemogenyx Pharmaceuticals, and Research Assistant Professor, State University of New York Downstate said in a statement.1
HEMO-CAR-T is an autologous chimeric antigen receptor (CAR) T-cell therapy developed with the use of Hemogenyx’sproprietary humanised monoclonal antibody against an AML target. Hemogenyx submitted the IND for HEMO-CAR-T in May 2023 and was first notified of the clinical hold in June.2,3 The FDA later provided Hemogenyx with a full review letter sharing the reasoning behind the hold, which relates to a splicing that occurs during the manufacturing process of the lentivirus vector. At the time, Hemogenyx stated that it had identified the source of the splicing issue, developed a method to eliminate it, and is remanufacturing the lentivirus. The company also stated that it planned to address several suggestions included in the FDA’s letter on how to improve the safety of HEMO-CAR-T that are not related to the hold.
"We are confident that we will be able to address the FDA's questions and concerns regarding the IND. AML has poor survival rates and we are eager to resolve this hold and continue down the treatment development pathway toward saving lives,” Sandler said in a statement at that time.2
Hemogenyx’s has another preclinical cell therapy candidate in the pipeline, Hu-PHEC, which is made from postnatal human hemogenic endothelial cells. The company believes these cells require less manipulation before use compared to other types of celltherapy. Hu-PHEC is being investigated in preclinical studies for bone marrow or hematopoietic stem cell transplant (HSCT). The company is also developing a bispecific antibody indicated for patient conditioning for bone marrow or HSCT and/or patients with relapsed/refractory AML.