Six months of treatment with GemVax & KAEL’s investigational GV1001 provided benefits in the cognition and daily life activities of adults with moderate to severe Alzheimer’s disease, who take Aricept (donepezil) for dementia, according to data from a Phase 2 clinical trial.
The results were published in an article, “Efficacy and safety of GV1001 in patients with moderate-to-severe Alzheimer’s disease already receiving donepezil: a phase 2 randomized, double-blind, placebo-controlled, multicenter clinical trial,” in the journal Alzheimer’s Research & Therapy.
“The publication of GV1001 Phase 2 Alzheimer’s disease clinical trial results in the highly prestigious journal ‘Alzheimer’s Research & Therapy‘ is an undisputable success and scientifically meaningful achievement,” a GemVax official said in a press release.
Larger clinical studies are needed to confirm these findings and clarify the therapeutic potential of GV1001, the researchers noted. The launch of a Phase 2a trial (NCT03959553) in people with moderate Alzheimer’s has already been approved in the U.S. and a Phase 3 trial in South Korea is planned to start later this year.
“We will continue to strive in the upcoming clinical trials domestically and abroad to develop what we hope will be a truly breakthrough treatment of Alzheimer’s disease,” the company official said.
Originally developed as a cancer vaccine, GV1001 mimics a specific portion/fragment of the telomerase reverse transcriptase (TERT), an enzyme highly produced by many cancers.
As such, GV1001 is thought to prime the immune system to better recognize and kill cancer cells that contain TERT, and in 2014, it was conditionally approved in South Korea for pancreatic cancer, under the brand name RIAVAX.
In addition, the therapy was shown to have neuroprotective properties, mediated through anti-cell death, anti-inflammatory, anti-aging, and antioxidant effects.
“These findings suggest that GV1001 has diverse modes of action against [Alzheimer’s disease]; thus, we hypothesized that these protective effects would make GV1001 a promising therapeutic option” for Alzheimer’s, the researchers wrote.
The Phase 2 clinical trial (NCT03184467) evaluated the safety and effectiveness of GV1001 in 96 adults (59 women and 37 men) with moderate to severe Alzheimer’s, who were on a stable dose of Aricept.
Participants were recruited at 13 hospitals across South Korea and had a mean age of 70.9 years (range of 56–85 years).
They were randomly assigned to receive an under-the-skin injection of 0.56 mg or 1.12 mg of GV1001 (32 patients in each dose group) or a placebo (31 patients), once a week for four weeks, and every other week until week 24 (nearly six months), totaling 14 injections.
The study’s main goal was to assess changes in patients’ cognitive function using the Severe Impairment Battery (SIB) scores. Secondary goals included safety assessments and other validated measures of several aspects of disease severity. These included the Clinical Dementia Rating Sum of Box (CDR-SOB), the Neuropsychiatric Inventory (NPI), and the Alzheimer’s Disease Cooperative Study-Activities of Daily Living (ADCS-ADL).
Efficacy evaluations were conducted at study start, and at week 12 and 24. Efficacy analyses were performed in the entire treated patient population and in the group of 73 (76%) participants who completed the six-month treatment period.
Results showed that while patients on placebo showed a mean decrease of 6.9 to 7.3 points in the SIB scores (indicating cognitive function worsening) after six months, those treated with the high dose of GV1001 had stable scores, with a mean drop of 0.1 to 0.3 points.
Notably, GV1001’s significant, beneficial effect on cognition was already observed at week 12, both for the entire patient population and for those who completed treatment. No significant differences in the SIB score were observed between the low-dose group and the placebo group throughout the study.
These findings meant that the trial met its main goal for GV1001’s high dose, which also resulted in significant reductions in dementia-related behaviors at week 12, as assessed with the NPI, compared with a placebo.
While trends of improvement were also observed in the ADCS-ADL and CDR-SOB scores over time for treated patients, differences relative to the placebo group did not reach statistical significance.
“The relatively small number of subjects might have restricted us from showing statistical significance,” the researchers wrote, adding that the effect of GV1001 on secondary measures and in the levels of biomarkers needs “to be confirmed in a larger clinical trial.”
GV1001 was generally well-tolerated, with rates of most common adverse events similar to those reported in the placebo group, and no reports of severe or clinically significant adverse events. The number of patients discontinuing treatment was not significantly different between the three groups.
“In this study, we demonstrated that GV1001 might have beneficial effects on the cognition and activities of daily living in patients with moderate-to-severe [Alzheimer’s] already receiving [Aricept],” the researchers wrote.
Among the study’s limitations, the team noted the inclusion of a single ethnic population and the short follow-up.
“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,” said Seong-Ho Koh, MD, the trial’s principal investigator, from the department of neurology at Hanyang University College of Medicine.
The U.S.-based Phase 2a trial will evaluate the safety and effectiveness of the two doses of GV1001, against a placebo, in up to 90 adults, 55–85 years of age, with moderate Alzheimer’s disease.
Participants will receive treatment for 24 weeks and be assessed for safety and effectiveness measures up to 26 weeks (six months).
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