NKGen Biotech Presents Data from Phase 1/2a Clinical Trial of Troculeucel in Moderate Alzheimer's Disease at the International Conference on Alzheimer's and Parkinson's Diseases (AD/PD™ 2025)
Moderate Alzheimer's disease (AD) patients in the trial received the highest dose of troculeucel to date at 6 billion cells per treatment.
After 12 months of treatment, both patients who completed 17 doses improved from moderate to mild AD within just 3 months. One patient stabilized at their improved score, while the other patient continued to show ongoing improvement.
Clinical development of troculeucel is ongoing and will be evaluated for safety and efficacy in the randomized, placebo-controlled Phase 2a part of the study.
SANTA ANA, Calif., April 07, 2025 (GLOBE NEWSWIRE) -- NKGen Biotech, Inc. (OTC:NKGN) ("NKGen" or the "Company"), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic natural killer (NK) cell therapeutics, today announced the oral presentation of updated Phase 1 clinical data from the Phase 1/2a trial evaluating troculeucel, cryopreserved expanded autologous NK cell therapy, in patients with moderate AD at the International Conference on Alzheimer's and Parkinson's Diseases and Related Neurological Disorders (AD/PD™ 2025) in Vienna, Austria.
Dr. Paul Y. Song, Chairman and Chief Executive Officer of NKGen Biotech, delivered an oral presentation to conference attendees which was focused on the six and twelve-month cognitive and biomarker results from the Phase 1 cohort of the Phase 1/2a clinical trial for troculeucel NK cell therapy in moderate Alzheimer's disease (NCT06189963).
Highlights from the presentation:
- Troculeucel was administered intravenously at a dose of 6 x 109 cells to three patients with moderate AD [median Clinical Dementia Rating – Sum of Boxes (CDR-SB) score of 11].
- Primary endpoint was safety, monitored for 21 days after the first dose for each patient.
- Patients were treated every three weeks. Two patients completed 17 doses while one patient completed 10 doses.
- Preliminary efficacy was measured using cognitive scales CDR-SB, Mini Mental State Examination (MMSE), Activities of Daily Living Scale (ADCS-ADL-Severe) and Alzheimer's Disease Assessment Scale-Cognitive (ADAS-Cog-11), with the calculated Alzheimer's Disease Composite Score (ADCOMS) score as an additional measure.
- Secondary endpoints included changes in protein aggregate and neuroinflammation biomarker levels in the CSF and blood at 6 and 12 months.
- After up to 12 months of treatment, with a dose of 6 x 109 cells every three weeks, the three patients had no drug-related adverse reactions.
- At 3 months, preliminary efficacy analyses for the three patients showed stable or improved cognitive scores on CDR-SB and ADCOMS; two of the three patients improved on all cognitive scales and ADCOMS score and downgraded their AD stage from moderate to mild (based on CDR-SB score).
- At 6 months, all three patients were stable/improved on CDR-SB, ADAS-Cog-11 and ADCOMS with two of the three patients stable/improved on all cognitive scales and ADCOMS score.
- At 12 months, the two patients completing the 17 doses continued to show stable or improved CDR-SB, ADAS-Cog-11, ADCS-ADL-Severe and ADCOMS scores, with one patient reaching a CDR-SB of 4.5 (considered the cutoff between mild cognitive impairment [MCI] and mild AD). While the initial cohort consisted of three patients, the third patient withdrew from the trial prior to reaching 12 months. This withdrawal was not related to any adverse effects or drug related issues.
- At 6 months, all three patients showed decreased levels in both CSF and plasma for Glial Fibrillary Acidic Protein (GFAP) while at 12 months, the remaining two patients in the study continue to show decreased levels in plasma.
- At 12 months, the CSF and plasma Amyloid Beta (Aβ) 42/40 ratio improved in both patients, while the CSF p-Tau 181 and p-Tau 217 levels remained relatively stable.
- Troculeucel at the highest dose given to moderate AD patients was well tolerated and in 2/3 patients showed clinical improvements after only 3 months on therapy; cognitive scores continued to show stability or improvement through the end of study period at 12 months. Troculeucel will be evaluated for safety and efficacy in the randomized, placebo-controlled Phase 2a part of the study.
"In our prior proof-of-concept Phase 1 dose escalation trial (NCT04678453) we were able to demonstrate that troculeucel was well tolerated and appears to cross the blood brain barrier to improve CSF levels of amyloid, p-tau, and alpha-synuclein proteins," said Paul Y. Song, M.D., Chairman and Chief Executive Officer of NKGen. "Despite the fact that 2/3 of the patients were sub-optimally dosed, 90% of patients had stable or improved cognitive functions (ADCOMS) after 11 weeks of treatment (4 doses)."
Dr. Song continued, "In this new Phase 1/2a trial, we are only treating moderate stage patients and at our highest dose of 6 x 109 cells given every three weeks for one full year. We are pleased again to see no drug related adverse events, and more importantly 100% stable or improved cognitive function using CDR-SB and ADCOMS scores. In fact, two of the first three patients treated at our highest dose have improved from moderate to mild disease including one patient who improved from a CDR-SB baseline score of 11 to 4.5. As GFAP has been shown by others to be a very strong independent biomarker, which correlates well with dementia severity and progression, we observed reductions in GFAP in CSF and plasma which appeared to correlate with cognitive improvement in our patients. We also found improvements in levels of Aβ 42/40. As we begin to rapidly enroll patients in our double-blind randomized Phase 2a trial, we expect to see significant cognitive and biomarker differences between the treatment arm and placebo group within as little as 6 months. We are very excited that troculeucel continues to be well tolerated and shows promising disease modifying activity."
A copy of the presentation will be added to the Scientific Publications page of the Company's website https://nkgenbiotech.com/ after the conference has concluded.
About Troculeucel
Troculeucel is a novel cell-based, patient specific, ex vivo expanded autologous NK cell immunotherapeutic drug candidate. NKGen is developing troculeucel for the treatment of neurodegenerative disorders and a broad range of cancers. Troculeucel is the International Nonproprietary Name ("INN") for SNK01 assigned by the World Health Organization ("WHO"). The WHO INN approval of troculeucel establishes a universally recognized nonproprietary drug name for SNK01 and marks a significant step on NKGen's journey toward bringing this therapy to market.
About NKGen Biotech
NKGen is a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic NK cell therapeutics. NKGen is headquartered in Santa Ana, California, USA. For more information, please visit www.nkgenbiotech.com.
Forward-Looking Statements
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