FDA Lifts Clinical Hold on CAR T Study in AML


The phase 1 study of HEMO-CAR-T in patients with acute myeloid leukemia can proceed following the lifted clinical hold.

CAR T-cell attack cancer cell and healthy cells © LASZLO - stock.adobe.com

CAR T-cell attack cancer cell and healthy cells © LASZLO - stock.adobe.com

  • HEMO-CAR-T is a proprietary chimeric antigen receptor (CAR) T-cell therapy.
  • The agent is being developed as a treatment for acute myeloid leukemia (AML).
  • A clinical hold letter was issued in July 2023 pointing to issues with the agent’s manufacturing.

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

1. Hemogenyx Pharmaceuticals PLC announces FDA consents to phase I trials of HEMO-CAR-T. News release. Hemogenyx Pharmaceuticals. February 9, 2024. Accessed February 12, 2024. http://tinyurl.com/mt9zkyzm
2. FDA puts clinical hold on Hemogenyx’s CAR-T AML Therapy. Hemogenyx Pharmaceuticals. News release. July 10, 2023. Accessed February 12, 2024. http://tinyurl.com/4wkc9hda
3. Hemogenyx Pharmaceuticals PLC - clinical hold lift plan is accepted by FDA. News release. Hemogenyx Pharmaceuticals. September 14, 2023. Accessed February 12, 2024. https://tinyurl.com/3em74th2
4. HEMO-CAR-T Immunotherapy. Hemogenyx Pharmaceuticals. Accessed February 12, 2024. https://hemogenyx.com/technology/hemo-car-t/
5. Atilla E, Benabdellah K. The black hole: CAR T cell therapy in AML. Cancers (Basel). 2023;15(10):2713. Published 2023 May 11. doi:10.3390/cancers15102713
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