Alkahest presented data this weekend from its phase 2a study, AKST6019-201, in mild-to-moderate Alzheimer’s Disease (AD). The study met its primary endpoint of safety and tolerability and key secondary endpoints measuring cognitive and functional performance indicated limited-to-no declines over the six-month treatment period.
“These data are encouraging and indicate that the GRF6019 plasma fraction is safe and well-tolerated, and that it may slow the progression of mild-to-moderate AD in patients who would have otherwise been expected to experience cognitive and functional decline over this period,” said Dr. Karoly Nikolich, chief executive officer of Alkahest. “The results of this trial support the continued study of plasma fractions in Alzheimer’s disease in a larger, placebo-controlled clinical trial, in collaboration with our partner, Grifols.”
The primary endpoint of the study was safety and tolerability, and the drug was found to be safe and well-tolerated in all subjects randomized to dosing with 100ml or 250ml of study drug GRF6019. There was no significant difference between 100 mL (n=21) and 250 mL (n=22) arms in adverse events. Most adverse events experienced by subjects were mild, and consisted of headaches, transient lab changes, infusion site extravasations, and transient blood pressure changes.
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Secondary endpoints included measures of cognition and function, with no significant difference between the two dose levels. Subjects experienced no clinically detectable decline in cognition as measured by the 11-item AD assessment scale-cognitive subscale (ADAS-Cog11) and the Mini-Mental State Examination (MMSE) over the six-month study period. Additionally, subjects demonstrated only a small decline in function as measured by the AD Cooperative Study Activities of Daily Living scale 23-item version (ADCS-ADL23) and the Clinical Dementia Rating scale Sum-of-Boxes (CDR-SB) score.
All data below are presented for both treatment arms combined. Data are presented for evaluable subjects (those receiving at least 5 doses of GRF6019, n=43) and for completers (those receiving all 10 doses of GRF6019 and completing visit 19; n=39).
The Phase 2a ALK6019-201 trial was designed to evaluate the safety, tolerability, and potential therapeutic effects of multiple doses of GRF6019 in patients with mild-to-moderate AD over six months. Subjects were randomized to either 100 mL or 250 mL of GRF6019 given by IV daily for five consecutive days during Week 1 and again for five consecutive days during Week 13, with a treatment-free interval of 11 weeks following each dose.
Alkahest and clinical and development partner Grifols are studying GRF6019 and GRF6021 for the treatment of age-related diseases. GRF6019 and GRF6021, proprietary plasma fractions, are manufactured by Grifols. In animal models, these plasma fractions enhance neurogenesis, improve age-related deficits in learning and memory, and reduce neuroinflammation. Phase 2 clinical trials with GRF6019 and GRF6021 are ongoing in severe Alzheimer’s disease, Parkinson’s disease with cognitive impairment, and post-operative recovery, with other indications being explored.