Troculeucel Gets Fast Track Status for Moderate Alzheimer Disease

The Food and Drug Administration (FDA) has granted Fast Track designation to troculeucel, an autologous natural killer (NK) cell therapy, for the treatment of moderate Alzheimer disease (AD). 

The Fast Track designation is supported by data from the open-label phase 1 SNK01-MX04 trial (ClinicalTrials.gov Identifier: NCT04678453), as well as preliminary data from the phase 1 portion of the phase 1/2a SNK01-AD01 trial (ClinicalTrials.gov Identifier: NCT06189963). 

The SNK01-MX04 trial enrolled 10 participants with AD (median age, 79 years) to evaluate the safety, tolerability, and preliminary efficacy of 4 varying doses of troculeucel (SNK01). Findings showed even with 70% of participants receiving low doses of troculeucel, 90% of all evaluable patients had either stable or improved (±0.1) composite Alzheimer Disease Composite Score (ADCOMS) 1 week after the final dose (week 11). 

Additionally at week 11, improvements were also seen in various cerebrospinal fluid biomarkers such as phosphorylated tau protein 181, beta-amyloid 42/40 ratio, glial fibrillary acidic protein, growth differentiation factor 15, latent transforming growth factor beta-binding protein 2, and neurofilament light chain.

The phase 1 portion of SNK01-AD01 evaluated troculeucel in 3 participants with moderate AD. Early findings showed after 3 months of troculeucel 6×10⁹ cells every 3 weeks, 2 out of the 3 study participants saw clinical improvements and went from a moderate to mild AD rating on the Clinical Dementia Rating-Sum of Boxes scale; all 3 patients had either a stable or improved ADCOMS score. 

In both studies, troculeucel was well tolerated, with no treatment-related adverse reactions.

“We are pleased with the FDA’s decision to grant Fast Track designation for troculeucel,” said Paul Y. Song, MD, Chairman and Chief Executive Officer of NKGen. “This decision underscores the significant unmet need for effective treatments for patients with moderate AD.”

According to NKGen, the phase 2a trial is currently enrolling patients with moderate AD; updated clinical data are expected by the end of 2025.

This article originally appeared on MPR