Researchers found improvements in SIB and NPI score between high-dose and placebo groups.
Data from a phase 2, double-blind, parallel-group, placebo-controlled, 6-month randomized clinical trial published in Alzheimer's Research and Therapy suggest that GV1001 (GemVax) is efficacious and well-tolerated in patients with Alzheimer disease (AD).1
Researchers found that patients treated with 1.12 mg of GV1001 showed less decreases in Severe Impairment Battery (SIB) score at 12 and 24 weeks compared with placebo (P <.05), while patients treated with 0.56 mg showed significantly better improvements in Neuropsychiatric Inventory (NPI) score compared to the higher-dose cohort.
Principal investigator Seong-Ho Koh, MD, professor, Department of Neurology, Hanyang University College of Medicine, said in a statement that "we are very pleased to have our research being recognized by this world-renowned journal for Alzheimer's disease research. As recognition of GV1001's research performance as a treatment for Alzheimer's disease continues in Korea and abroad, interest in future clinical trials will be higher both domestically, and globally."2
Koh and colleagues analyzed data from 96 patients with moderate-to-severe AD recruited from 13 centers in South Korea between September 2017 and September 2019. The patients had an average age of 70.9 years (SD, 9.0) and were randomized 1:1:1, with 31 patients in the placebo group, 33 in the 0.56-mg GV1001 group, and 32 in the 1.12-mg GV1001 group.
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The treatment was administered weekly for 4 weeks, then biweekly the next 20 weeks. Endpoints assessed included SIB, Clinical Dementia Rating Sum of Box (CDR-SOB), Alzheimer’s Disease Cooperative Study-Activities of Daily Living (ADCS-ADL), Mini-Mental State Examination (MMSE), and Global Deterioration Scale (GDS) scores. Safety endpoints were also assessed.
At week 24, the high-dose group differed in SIB score by 6.6 points in the full analysis set (FAS; P = .027) and by 7.1 points in the per-protocol set (PPS; P = .018) as compared to placebo. There was also a statistically significant difference at week 12 between high-dose and placebo in both FAS (P = .030) and PPS (P = .016). There were no statistically significant differences between low-dose and placebo on SIB score.
The high-dose cohort had significant improvements in NPI at week 12, with a least-square mean (LSM) of change from baseline of − 5.1 (standard error [SE], 2.6) as compared to placebo (LSM, 4.6 [SE, 2.6]; P = .017). No NPI differences were significant between placebo and low-dose, and other secondary endpoints including CDR-SOB, ADCS-ADL, MMSE, and GDS had no significant differences.
“The following clinical trials should consider evaluating additional biomarkers, considering other drugs for the treatment of AD, analyzing sample size based on this study, and including diverse ethnic populations. Based on these results, another phase 2a clinical trial for moderate AD has already been approved in the USA after a thorough review by the FDA (NCT03959553), and a phase 3 clinical trial in South Korea will be performed soon,” Koh and colleagues wrote in the paper.
Safety profiles were similar between placebo, low-dose, and high-dose cohorts. No differences were seen between groups in treatment-emergent adverse events (TEAEs). Severe TEAEs occurred in the placebo group, with 1 patient fracturing an arm after a fall and another incidentally diagnosed with stomach cancer.
"The publication of GV1001 phase II Alzheimer's disease clinical trial results in the highly prestigious journal 'Alzheimer's Research & Therapy' is an undisputable success and scientifically meaningful achievement. We will continue to strive in the upcoming clinical trials domestically and abroad to develop what we hope be a truly breakthrough treatment of AD,” a GemVax official added to the statement.