The phase 1 study of HEMO-CAR-T in patients with acute myeloid leukemia can proceed following the lifted clinical hold.
The FDA has lifted the clinical hold on the investigational new drug (IND) application for HEMO-CAR-T, a proprietary CAR T-cell therapy for the treatment of AML following the issue of a clinical hold letter.1 The phase 1 trial of HEMO-CAR-T can now proceed.
Homogenyx submitted its IND seeking to start a phase 1 trial in May 2023. The FDA issued the clinical hold letter in July 2023 and identified manufacturing issues as the cause—specifically, issues with a “splicing deficiency” during the production of lentivirus.2
The FDA then accepted Hemogenyx Pharmaceuticals’ plan to resolve the clinical hold placed on the HEMO-CAR-T IND application in September 2023.3
"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. The removal of the clinical hold was made possible by the hard work and dedication of the entire Hemogenyx Pharmaceuticals team and its board of directors and advisors,” said Vladislav Sandler, PhD, chief executive officer and co-founder of Hemogenyx Pharmaceuticals, in a press release.1
HEMO-CAR-T is a proprietary humanized monoclonal antibody targeted against the surface of AML cells.4 While CAR T-cell therapy is an established treatment option in B-cell malignancies, it is still in its incipient stages for AML treatment.5 If successful, HEMO-CAR-T could provide a new treatment pathway for patients with AML, which has a 5-year survival rate of less than 30% in adults.1