GRF6019 Shows Positive Phase 2 Results in Mild to Moderate Alzheimer Disease

Karoly Nikolich, PhD, Alkahest

Karoly Nikolich, PhD

Alkahest reported positive top-line data from its phase 2 clinical trial studying GRF6019 which is under investigation for mild-to-moderate Alzheimer disease.

Investigators reported that dosing with GRF6019 was safe and well-tolerated. Subjects receiving GRF6019 experienced no decline in cognition measured by the Alzheimer’s Disease Assessment Scale-Cognitive Subscale (ADAS-Cog11) and the Mini-Mental State Examination (MMSE), and minor decline in function 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). These data demonstrate that subjects maintained cognitive and functional status throughout the 6-month period.

“These top-line results indicate a potential benefit of this plasma protein fraction in slowing the progression of cognitive decline in patients with mild to moderate Alzheimer disease,” Karoly Nikolich, PhD, chief executive officer of Alkahest, said in a statement. “Developing impactful treatments for this disease and other neurodegenerative diseases of aging has proven unsuccessful for decades. GRF6019 represents an innovative approach to effectively treating Alzheimer disease by targeting multiple underlying mechanisms of disease, and these data support the continued development of GRF6019 and other plasma protein fractions in Alzheimer disease.”

The randomized, double-blind, dose comparison phase 2 trial (NCT03520998) evaluated the safety, tolerability, and feasibility of GRF6019, a plasma-derived product, administered as an IV, in 40 subjects with mild to moderate Alzheimer disease for 6 months. Subjects eligible for the study were 60—90 years of age, received a diagnosis of probable Alzheimer disease, had a MMSE score of 12–24 inclusive, and a Modified Hachinski ischemia score of ≤4.

Subjects were randomized 1:1 to 1 infusion per day of 100 mL or 250 mL of active treatment for 5 consecutive days at weeks 1 and 13, with a treatment-free interval of 12 weeks following each dose, for a total of 10 doses per subject.

The primary outcome measure included the incidence of treatment-emergent adverse effects, while secondary measures included changes in scores on the MMSE, ADAS-Cog11, the Grooved Pegboard Test, the Category Fluency Test, the CDR-SB, the ADCS-ADL₂₃, the Alzheimer’s Disease Cooperative Study-Clinical Global Impression of Change (ADCS-CGIC), the Neuropsychiatric Inventory Questionnaire (NPI-Q), and the Savonix Neurocognitive Assessments and Digit Span.

Alkahest also intends to study GRF6019 in severe Alzheimer disease.

REFERENCE

Alkahest Announces Positive Top-line Data from Phase 2 Study in Mild to Moderate Alzheimer’s Disease [news release]. San Carlos, Calif.: Alkahest, Inc.; Aug. 5, 2019. https://finance.yahoo.com/news/alkahest-announces-positive-top-line-113000742.html. Accessed Aug. 6, 2019.