FDA Grants Fast Track Designation to Troculeucel for Moderate Alzheimer Disease
Data has shown the investigational cell therapy is well-tolerated in patients and showed preliminary impacts on cognition.
The FDA granted fast track designation to troculeucel, an investigational, ex vivo expanded autologous natural killer (NK) cell therapy, for the treatment of moderate Alzheimer disease (AD).1
Approximately 90% had either stable or improved composite ADCOMS scores at week 11—only 1 week after the final dose. | Image Credit: SewcreamStudio | stock.adobe.com
“This decision underscores the significant unmet need for effective treatments for patients with moderate AD. We specifically targeted the moderate stage population as they represent about 30% of all Alzheimer cases, and most, if not all, of the current focus has been on early/mild patients,” Paul Y. Song, MD, chairman and CEO of NKGen, said in a news release.1 “This designation comes after promising safety and efficacy results from our phase 1 trial, which shows early signs of clinical benefit in patients treated with troculeucel. Receiving fast track designation will significantly accelerate the drug development process, bringing us one step closer to delivering this promising therapy to AD patients in need and ensuring faster access to a potentially life-changing treatment.”
In a dose-escalation phase 1 pilot study, all 4 treatments with suboptimal doses were well-tolerated in patients and had preliminary impacts on cognition. There were also improvements in protein and neuroinflammation biomarkers. In the study, there were 11 patients enrolled, but only 10 were evaluable and had a median age of 79 years. Approximately 70% of patients were treated at low doses of troculeucel, and approximately 90% had either stable or improved composite ADCOMS scores at week 11—only 1 week after the final dose.2
One week after the final dose, investigators found improvements in cerebrospinal fluid (CSF) biomarkers, including 70% for p-Tau181, 60% for AB42/40 ratio, 60% for GFAP, 40% for GDF-15, 30% for LTBP2, and 30% for NF-L. There were also no treatment-related adverse events observed, and the results suggest that the drug could potentially be safe for individuals at high risk for AD.2
In a second poster presented at the Clinical Trials on Alzheimer’s Disease Annual Meeting 2024, 3 patients were enrolled in the phase 1 cohort, with early data showing that after 3 months of treatment, clinical improvements were seen in 2 patients who went from moderate AD to mild AD. Further, all patients had either stable or improved ADCOMS scores.2
“We have since initiated a phase 1/2a trial using our highest dose of cryopreserved troculeucel in moderate AD subjects. We observed stable or improved cognitive function and no treatment-related adverse events after just 3 months of treatment. Notably, 2 of the 3 enrolled subjects in the phase 1 cohort showed clinical improvement from moderate to mild AD based on ADCOMS and CDR-SB scores,” Song said in a news release.2 “Based on our promising data to date, we have already dosed the first 2 subjects in our double-blind, randomized phase 2 cohort; and, look forward to sharing data updates when available. We are excited about the potential of troculeucel as a treatment for AD as well as the potential utility for it across other tau- and synuclein-related neurodegenerative diseases.”
A phase 2a trial for the drug is currently enrolling patients, with expected clinical data by the end of 2025.1
READ MORE: Neurology Resource Center
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