NKGen Biotech's Alzheimer's Drug Moves to Phase 2 After Positive Phase 1 Results

NKGen Biotech, Inc., a clinical-stage biotechnology company, has announced promising early interim data from the Phase 1 cohort of its Phase 1/2a clinical trial of troculeucel, a cryopreserved autologous enhanced natural killer (NK) cell therapy for moderate Alzheimer’s disease (AD). The company also noted the dosing of the first patient in the Phase 2 cohort of the trial.

The Phase 1 cohort has shown positive interim results. An independent review of the data revealed that after only three months of treatment with a dose of 6 billion cells per treatment, two of the first three patients in this cohort experienced a clinical upgrade in their CDR-SB cognitive scores, moving from moderate to mild AD. These findings align with NKGen's previous MX04 Phase 1 AD study, where a single moderate AD patient on a dose of 4 billion cells also improved from moderate to mild AD on the CDR-SB scale.

Furthermore, NKGen plans to disclose six-month interim cognitive data from the Phase 1 cohort at a national Alzheimer’s conference in Q4 2024. With these encouraging results, the company has initiated the Phase 2 randomized, double-blind, placebo-controlled trial, aiming to further assess the efficacy and safety of troculeucel in a broader cohort of 30 patients with moderate AD. This trial will involve 20 patients randomly assigned to the treatment group and 10 to the placebo group. The goal is to gain a comprehensive understanding of the potential benefits and limitations of troculeucel for treating Alzheimer’s disease.

Paul Y. Song, MD, Chairman, and CEO of NKGen, expressed optimism: “We continue to make great progress with our troculeucel clinical program in AD. In the Phase 1 cohort, two of our first three patients were clinically upgraded from moderate to mild AD after only three months of treatment with troculeucel. Additionally, we have dosed the first patient in the Phase 2 cohort, marking a significant milestone, particularly since the dose used is cryopreserved and at our highest dosing of 6 billion cells per treatment. With encouraging cognitive improvements and a favorable safety profile from the Phase 1 interim analysis, dosing our first patient in Phase 2 is a crucial step towards providing a much-needed treatment option for patients facing this challenging condition. We are particularly eager to explore the enhanced cognitive benefits that ongoing usage of the higher dosing may bring in our Phase 2 trial.”

Troculeucel, formerly known as SNK01, is an innovative cell-based, patient-specific ex vivo expanded autologous NK cell immunotherapy drug candidate. NKGen is developing troculeucel for treating neurodegenerative disorders and various cancers. The World Health Organization (WHO) has assigned the International Nonproprietary Name (INN) troculeucel to SNK01, marking a significant step toward bringing this therapy to market with a universally recognized name.

NKGen Biotech, headquartered in Santa Ana, California, focuses on developing and commercializing innovative autologous and allogeneic NK cell therapeutics. The company has activated four clinical sites across North America and expects to increase enrollment in the coming months. The Phase 2 trial's randomized, double-blind, placebo-controlled design aims to validate the therapeutic efficacy of troculeucel and provide insight into its potential benefits and limitations in treating moderate Alzheimer's disease.