Ingenium Therapeutics, a South Korea-based clinical-stage biotechnology company advancing allogeneic natural killer (NK) cell therapies, announced on Wednesday that it has received positive regulatory feedback from the US Food and Drug Administration (FDA) following a Pre-Investigational New Drug (Pre-IND) meeting.

Ingenium has conducted multiple investigator-initiated trials in South Korea, enrolling over 140 patients with acute myeloid leukaemia (AML), including a Phase 2 randomised clinical trial with long-term overall survival follow-up. The company says that these studies have generated robust non-clinical and clinical data supporting the safety and efficacy profile of Gengleucel.

Gengleucel has been granted Orphan Drug Designation by the FDA for the treatment of AML.

Based on its review of the trial data, the FDA has provided positive feedback on Ingenium's proposal to initiate a Phase 2 trial of Gengleucel in the United States without a preceding Phase 1 study. This regulatory milestone may streamline Gengleucel's clinical development pathway and may accelerate access for patients with measurable residual disease-positive (MRD+) AML.

The planned study will be conducted at cancer centres across the United States. Gengleucel is positioned to become the first NK cell therapy in AML to use MRD negativity as a primary endpoint, underscoring its potential to improve outcomes by eradicating minimal residual disease and reducing relapse risk.

Expected to begin in early 2026, the trial will evaluate Gengleucel's ability to achieve MRD negativity and reduce relapse and mortality. To ensure reliable clinical operations and product supply, Ingenium is transferring its manufacturing technology to a Texas-based manufacturing facility.