Hemogenyx clears FDA hurdle for its novel CAR-T therapy for AML

Hemogenyx Pharmaceuticals, Cell & Gene Therapy, clinical hold, CAR-T

Hemogenyx Pharmaceuticals has moved a step closer to advancing its CAR-T therapy, HEMO-CAR-T, into clinical trials following the FDA’s acceptance of its plan to lift a clinical hold. This development comes after a summer of setbacks, during which the FDA raised concerns about a splicing issue in the lentivirus manufacturing process for CAR-T cell production.

The London-based biotech initially revealed the FDA’s decision to halt its planned early-phase trial in June. The specific concern centered on the manufacturing process of the lentivirus, which plays a crucial role in CAR-T cell production. However, in July, Hemogenyx announced that it had pinpointed the source of the lentivirus splicing problem and developed a strategy to address it.

In a recent announcement, the company disclosed that the detailed plan, accompanied by supporting laboratory tests, submitted to the FDA in August has successfully addressed the regulatory body’s concerns. 

“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. The team is now dedicated to completing the outlined plan promptly, with the goal of resubmitting the IND (Investigational New Drug) application as soon as possible to proceed with clinical trials of HEMO-CAR-T.”

– Vladislav Sandler, Ph.D., CEO of Hemogenyx

HEMO-CAR-T is a promising therapy in development for the treatment of acute myeloid leukemia. The path to the IND stage was acknowledged by Hemogenyx as more complex and time-intensive than originally anticipated. Nevertheless, the company had taken steps to mitigate potential delays, as indicated in a May update.

It’s noteworthy that Hemogenyx is not the only biotech company facing CAR-T therapy setbacks this summer. 2seventy bio encountered a pause in its CD33-targeted cell therapy due to a patient’s death, while Arcellx’s CAR-T therapy, developed in partnership with Gilead, was also placed on a two-month hold following a similar incident.

Hemogenyx’s progress in addressing the FDA’s concerns marks a positive turn of events, bringing renewed hope for advancing HEMO-CAR-T towards potential clinical breakthroughs in leukemia treatment.

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