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GRF6019 Shows Positive Phase 2 Results in Mild to Moderate Alzheimer Disease Subjects receiving GRF6019 maintained cognitive and functional status over a period of 6 months, and dosing was reported to
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
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.
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.”
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.
Alkahest Announces Positive Top-line Data from Phase 2 Study in Mild to Moderate Alzheimer’s Disease
About GRF6019 and GRF6021Alkahest, 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