Hemogenyx Sends the FDA a Complete Response Related to the IND Hold on AML CAR-T HEMO-CAR-T

Hemogenyx Pharmaceuticals has submitted a complete response to the FDA regarding the clinical hold that was placed on the company’s investigational new drug (IND) application for HEMO-CAR-T, an autologous chimeric antigen receptor T-cell (CAR-T) therapy developed with the use of Hemogenyx’s proprietary humanized monoclonal antibody against an acute myeloid leukemia (AML) target.^1,2

Hemogenyx originally submitted the IND to the FDA in May 2023 with the intention of gaining clearance for a phase 1 clinical trial.³ The hold on the IND was placed by the FDA shortly after, in June 2023, with agency citing a splicing that occurs during the manufacturing process of the lentivirus vector as the reason for the hold.⁴

“We are pleased to have filed a complete response to the FDA addressing their concerns that resulted in a clinical hold of the HEMO-CAR-T IND,” Vladislav Sandler, PhD, the CEO and cofounder of Hemogenyx, said in a statement.¹ “We look forward to becoming a clinical stage biopharmaceutical company once consent is received from the FDA.”

In July 2023, Hemogenyx pointed out that it had identified the source of the splicing issue, had developed a method to eliminate it, and was in the process of remanufacturing the lentivirus.⁴ The FDA's full review letter regarding the clinical hold also included several suggestions on how to improve the safety of HEMO-CAR-T that were not related to the hold. Hemogenyx noted in July 2023 its intention to address those additional suggestions promptly. In September 2023, the company announced that it had come to an accord with the FDA on its plan to resolve the clinical hold.²

"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,” Sandler said in a September 2023 statement.² “We are now working hard to complete the schedule of work set out in the plan and to resubmit the IND as expeditiously as possible in order to move forward with clinical trials of HEMO-CAR-T.”

Just a few weeks ago, on January 8, 2024, Hemogenyx announced that it had successfully completed all tests across a process qualification run of the end-to-end manufacturing process for HEMO-CAR-T.⁵ This process qualification run constituted a key piece of the plan agreed upon with the FDA for resolving the issue that lead to the clinical hold.

“We are pleased that we have now completed the necessary process qualification run,” Sandler said in a statement at the time.⁵ “We are now working hard to resubmit the IND as expeditiously as possible to move forward with clinical trials of HEMO-CAR-T.“

In addition to HEMO-CAR-T, Hemogenyx’s pipeline also includes Hu-PHEC, a preclinical stage cell therapy made from postnatal human hemogenic endothelial cells, which the company states require less manipulation before use compared to other types of cell therapy.⁶ Hu-PHEC is being investigated in preclinical studies for bone marrow or hematopoietic stem cell transplant.

REFERENCES
1. Submission of complete response to clinical hold for HEMO-CAR-T IND. News release. Hemogenyx Pharmaceuticals plc. January 16, 2024. Accessed January 18, 2024. https://hemogenyx.com/investors/investors/announcements/announcement/2024/submission-of-complete-response-to-clinical-hold-for-hemo-car-t-ind/
2.Hemogenyx Pharmaceuticals PLC - clinical hold lift plan is accepted by FDA. News release. Hemogenyx Pharmaceuticals. September 14, 2023. Accessed January 18, 2024. https://www.accesswire.com/783926/hemogenyx-pharmaceuticals-plc-clinical-hold-lift-plan-is-accepted-by-fda
3. Submission of IND for HEMO-CAR-T. News release. Hemogenyx Pharmaceuticals plc. May 9, 2023. Accessed January 18, 2024. https://hemogenyx.com/investors/investors/announcements/announcement/2023/submission-of-ind-for-hemo-car-t/
4.Hemogenyx Pharmaceuticals PLC Announces Full FDA Review for HEMO-CAR-T IND. News release. Hemogenyx Pharmaceuticals. July 10, 2023. Accessed January 18, 2024. https://www.biospace.com/article/releases/hemogenyx-pharmaceuticals-plc-announces-full-fda-review-for-hemo-car-t-ind/
5. HEMO-CAR-T process qualification run completed. News release. Hemogenyx Pharmaceuticals plc. January 8, 2024. Accessed January 18, 2024. https://hemogenyx.com/investors/investors/announcements/announcement/2024/hemo-car-t-process-qualification-run-completed/
6. Pipeline. Hemogenyx Pharmaceuticals plc. Website. Accessed January 18, 2024. https://hemogenyx.com/pipeline/