NKGen’s Autologous NK Cell Therapy Troculeucel Fast Tracked for Alzheimer Disease
Troculeucel is currently being evaluated in a phase 1/2a trial (NCT06189963) for moderate AD.
Paul Y. Song, MD
Credit: NKGen
NKGen Biotech's troculeucel (also known as SNK01), an investigational autologous natural killer (NK) cell therapy, has been granted fast track designation by the FDA for the treatment of moderate Alzheimer disease (AD).1
Troculeucel is currently being evaluated in a phase 1/2a trial (NCT06189963) for moderate AD. In May 2024, NKGen announced that the trial would be progressing to its phase 2 portion following clearance from a Safety Review Committee that looked at data from the phase 1 portion.2 The phase 2a portion is actively recruiting participants and NKGen anticipates that it will announce updated clinical data before the end of this year.1 Notably, troculeucel was originally developed for oncology indications and is also being evaluated for the treatment of Parkinson disease.
“We are pleased with the FDA’s decision to grant Fast Track designation for troculeucel,” Paul Y. Song, MD, the chairman and chief executive officer of NKGen, said in a statement.1 “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 AD cases and most, if not all, of the current focus has been on early/mild patients. 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.”
Troculeucel is an autologous NK cell therapy that is not genetically engineered, but has enhanced cytotoxicity and activating receptor expression.2 It was previously evaluated for AD in an earlier phase 1 clinical trial (ASK-AD; NCT04678453).3 The study was an open label, 3+3 dose escalation study that administered 4 doses of 1, 2, or 4 billion cells administered intravenously. The study was terminatedearly after NKGen gained clearance of an investigational new drug application for a new trial with an improved manufacturing process for the therapy.
Data from ASK-AD were presented at the 2024 Alzheimer’s Association International Conference (AAIC), held July 28 to August 1, in Philadelphia, Pennsylvania. The therapy was well-tolerated, with no treatment-related adverse events observed. Even at lower doses (70% of participants were treated with the trial’s low dose) in a safety-focused study, troculeucel does seem to have demonstrated some clinical activity. At week 11, 1 week after the final dose, 90% of all evaluable subjects had either stable or improved (±0.1) composite AD Composite Score scores and 60% had a decrease in cerebrospinal fluid α-syn compared to baseline values.
“We've now dosed almost, I would say, 100 patients between all of our cancer trials, AD trials, and our compassionate use trials, and [we’re] not seeing any side effect related to our treatment at all,” Song told CGTLive® in a previous request for comment on troculeucel. “There is no pretreatment, unlike a lot of cell therapies where you have to give some lymphodepletion or some type of low dose chemotherapy or even some cytokine support. We do not have to do any pretreatment, because it's your own natural killer cells that are not genetically modified. So, it's a very simple outpatient infusion in and out. And we find our patients, thus far, have been really, really tolerating [troculeucel] extremely well with no side effects.”
