FDA Lifts Clinical Hold on IND for HEMO-CAR-T in Acute Myeloid Leukemia

The FDA previously issued a clinical hold letter in response to the IND for HEMO-CAR-T in July 2023.

The FDA has lifted a clinical hold on an investigational new drug application (IND) for HEMO-CAR-T as a treatment for those with acute myeloid leukemia (AML), according to a press release from Hemogenyx Pharmaceuticals.¹

“We are extremely pleased with the FDA's decision to lift the clinical hold,” Vladislav Sandler, PhD, chief executive officer and co-founder at Hemogenyx Pharmaceuticals, said in the press release. “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.”

The FDA previously issued a clinical hold letter in response to the IND for HEMO-CAR-T in July 2023.² According to the letter, the regulatory agency placed a clinical hold on the application due to a splicing issue about the company’s lentivirus manufacturing process used for producing CAR T cells. The letter also gave several suggestions for improving the product’s safety, which did not contribute to the clinical hold.

Developers announced that they submitted a complete response to the FDA’s letter in January 2024.³ Moreover, they had successfully addressed the splicing deficiency and all other concerns described in the regulatory agency’s clinical hold letter. As a result, investigators are now able to proceed with their evaluation of HEMO-CAR-T in AML as part of a phase 1 clinical study.

“We are pleased to have filed a complete response to the FDA addressing their concerns that resulted in a CH of the HEMO-CAR-T IND. We look forward to becoming a clinical stage biopharmaceutical company once consent is received from the FDA,” Sandler said at the time of the complete response submission.³

Developers announced that they had successfully completed a process qualification run for the end-to-end process of HEMO-CAR-T cell manufacturing in January 2024.⁴ The process qualification run was organized as part of the company’s strategy for addressing the concerns highlighted in the FDA’s clinical hold letter.

The process qualification run, which was initiated in the company’s Good Manufacturing Practice compliant clean rooms, was the only manufacturing run necessary for submitting a complete response to the FDA’s clinical hold letter.

“We are pleased that we have now completed the necessary [process qualification] run. We are now working hard to re-submit the IND as expeditiously as possible to move forward with clinical trials of HEMO-CAR-T,” Sandler said at the time.⁴

An IND for HEMO-CAR-T as a treatment for patients with AML was originally submitted to the FDA in May 2023.⁵ The application submission followed successful development of the product’s manufacturability, quality, and safety.

“We are pleased to have reached this milestone with HEMO-CAR-T,” Sandler said in a press release at the time of the IND submission.⁵ “We are committed to advancing therapies for blood diseases, and our work to address AML, which currently has poor survival rates, is an essential part of that commitment.”

According to its developers, HEMO-CAR-T is designed with a proprietary humanized monoclonal antibody that works to target the surface of AML cells.⁶ It is believed that HEMO-CAR-T may introduce a potentially more benign, effective treatment option that can influence survival rates. Developers stated that HEMO-CAR-T has exhibited an ability to convert human T cells so that they can identify and attack human AML-derived cells in vitro.

References

1. Hemogenyx Pharmaceuticals PLC announces FDA consents to phase I trials of HEMO-CAR-T. News release. Hemogenyx Pharmaceuticals PLC. February 9, 2024. Accessed February 12, 2024. http://tinyurl.com/bde8r5hb
2. Hemogenyx Pharmaceuticals PLC announces full FDA review for HEMO-CAR-T IND. News release. Hemogenyx Pharmaceuticals PLC. July 10, 2023. Accessed February 12, 2024. http://tinyurl.com/43ek2ynj
3. Hemogenyx Pharmaceuticals PLC announces submission of complete response to clinical hold. News release. Hemogenyx Pharmaceuticals PLC. January 16, 2024. Accessed February 12, 2024. http://tinyurl.com/jbhsunw5
4. Hemogenyx Pharmaceuticals PLC announces HEMO-CAR-T process qualification run completed. News release. Hemogenyx Pharmaceuticals PLC. January 8, 2024. Accessed February 12, 2024. http://tinyurl.com/muvzyf9d
5. Submission of IND for HEMO-CAR-T. News release. Hemogenyx Pharmaceuticals PLC. May 9, 2023. Accessed February 12, 2024. http://tinyurl.com/3zpdee6h
6. HEM-CAR-T immunotherapy. Hemogenyx Pharmaceuticals PLC. Accessed February 12, 2024. http://tinyurl.com/3en7b899