US-based Neurotrope has reported positive top-line results from the Phase II clinical trial of bryostatin-1 for the treatment of moderate-to-severe Alzheimer's disease (AD).
Bryostatin-1 activates protein kinase C epsilon and targets synaptic growth factors, anti-amyloid and anti-tangle signalling pathways in the brain.
The results indicated that the pre-specified primary endpoint was met in the completer population, comprising patients within the modified intent-to-treat (mITT) population with a completed 13-week assessment.
The repeat dose, randomised, double-blind, placebo-controlled Phase II trial evaluated the safety and preliminary efficacy of two dose levels of bryostatin-1 compared to placebo in 147 late-stage AD patients for a period of 12 weeks.
Approximately 135 out of the total number of patients were recruited into the mITT population and 113 into the complete population.
Neurotrope president and chief scientific officer Dr Daniel Alkon said: "These results, which show improvement in patients with moderate-to-severe Alzheimer's disease, the population that is generally recognised as the most difficult to treat, provide exciting evidence of a new therapeutic approach that potentially could rejuvenate synaptic networks in the brain.
"Improvements across the range of important manifestations of the underlying neurodegenerative disease, as shown in this Phase II study, could potentially represent a shift in the paradigm to treat Alzheimer's disease."
The pre-specified primary endpoint was the measure of cognition using severe impairment battery (SIB) for 20µg and 40µg doses of the drug at week 13.
The trial's secondary endpoint was Alzheimer's disease cooperative study activities of daily living inventory severe impairment version (ADCS-ADL-SIV).
Bryostatin-1 is being further studied as potential treatment for genetic disorders, Fragile X Syndrome, Niemann-Pick Type C disease and Rett Syndrome.
It also secured orphan drug designation from the US Food and Drug Administration (FDA) for Fragile X Syndrome.