Neurotrope has completed enrolment and initiated dosing in the last patient of a Phase II clinical trial evaluating Bryostatin-1 for the treatment of moderate to severe Alzheimer’s disease.
The confirmatory trial enrolled a total of 108 participants.
According to the company, Bryostatin-1 activates protein kinase C, triggers synaptic growth factors, amyloid-β degrading enzymes and prevents transformation of tau into neurofibrillary tangles.
Activation of protein kinase C is expected to improve synaptic function, allow new synapse formation, repair damaged synapses, and avoid neuronal death.
The multi-centre, randomised, double-blind, placebo-controlled Phase II trial evaluates the safety and efficacy of Bryostatin-1 as a treatment for cognitive deficits.
Participants received seven doses of 20μg Bryostatin-1 or placebo over 12 weeks. The trial only recruited patients who were not on memantine, an antagonist of NMDA receptor.
The trial’s primary efficacy endpoint is the change in the Severe Impairment Battery (SIB) score from the baseline to week 13, while secondary endpoints include repeated SIB changes from baseline at weeks five, nine, 13 and 15.
Neurotrope president and chief scientific officer Daniel Alkon said: “Completion of enrolment in our confirmatory Phase II trial is an important step toward understanding the transformative potential of Bryostatin in moderate to severe Alzheimer’s disease, a neglected area of AD research with no effective treatment options.
“Bryostatin uses a novel, multi-modal mechanism of action which has demonstrated the ability to generate new, mature synaptic connections and prevent neuronal death in AD models.”
In a prior exploratory Phase II trial, the drug candidate is said to have demonstrated greater than baseline improvements in SIB scores for patients treated with 20µg Bryostatin-1.
The company expects to report top-line results from the confirmatory trial in the second half of 2019.
Neurotrope also studied Bryostatin-1 in preclinical models of rare diseases and brain injury such as Fragile X syndrome, multiple sclerosis, stroke, Niemann-Pick Type C disease, Rett syndrome and traumatic brain injury.