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.

GRF6019, which is being developed with clinical development partner Grifols, represents a second-generation product of earlier clinical research infusing young adult plasma to treat age-related neurodegenerative disease.

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Participants will receive an intravenous infusion of either GRF6019 or placebo every day for 5 days in a row. The total study duration is approximately 9 weeks.

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While there continues to be debate as to what drives AD, Braithwaite said the plasma approach could be effective regardless of what protein is deemed the underlying cause of the disease.

 “Our approach is very agnostic to the specific driver, where we think we are modulating multiple age-related and disease-related biological processes,” he said. “This really is a whole mechanistically novel approach to treating Alzheimer’s disease.”

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 The rationale behind GRF6019 is that the components of young blood that counteract inflammatory and other age-related degenerative processes in the brain can be enriched and delivered as a plasma-derived product that does not require matching of donor and recipient blood types.

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Alkahest Announces Positive Top-line Data from Phase 2 Study in Mild to Moderate Alzheimer’s Disease

About GRF6019 and GRF6021
Alkahest, and clinical development partner Grifols, are studying GRF6019 and GRF6021 for the treatment of age-related diseases.  GRF6019 and GRF6021, proprietary plasma fractions, are developed and provided 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 Alzheimer’s disease and Parkinson’s disease, respectively, with other age-related indications being explored.

Sources: https://www.alzforum.org/therapeutics/grf6019

https://www.clinicaltrials.gov/ct2/show/NCT03765762?term=GRF6019&draw=2&rank=1

https://www.mdmag.com/medical-news/plasma-treatment-alzheimer-disease