On April 30, 2025 Ingenium Therapeutics, a clinical-stage biotechnology company advancing allogeneic natural killer (NK) cell therapies, reported that it has received positive regulatory feedback from the U.S. Food and Drug Administration (FDA) following a Pre-Investigational New Drug (Pre-IND) meeting (Press release, Ingenium Therapeutics, APR 30, 2025, View Source [SID1234652401]).
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Ingenium has conducted multiple investigator-initiated trials in South Korea, enrolling over 140 patients with acute myeloid leukemia (AML), including a Phase 2 randomized clinical trial with long-term overall survival follow-up. These studies have generated robust non-clinical and clinical data supporting the safety and efficacy profile of Gengleucel.
Based on its review of these 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 multicenter study will be conducted at leading cancer centers across the United States, including a world-renowned institution in Texas. 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.
"We are thrilled with the FDA’s guidance, which reflects the rigor of our clinical research and the significant therapeutic potential of Gengleucel, especially in targeting minimal residual disease," said Kevin Koh, CEO of Ingenium Therapeutics. "Launching our U.S. Phase 2 trial at premier cancer centers marks a major milestone in our mission to deliver novel immunotherapies to AML patients with MRD."
The trial is expected to begin in early 2026 and 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.
About Gengleucel
Gengleucel is a non-engineered, allogeneic NK cell therapy composed of memory NK cells with enhanced activating receptor expression and increased secretion of IFN-gamma, granzyme, and perforin. These features drive potent anti-cancer activity and sustained in vivo persistence. Clinical trials have demonstrated Gengleucel’s favorable safety, tolerability, and efficacy in high-risk AML and MDS populations. Gengleucel has been granted Orphan Drug Designation by the U.S. FDA for the treatment of AML.