Neurotrope Inc. announced that it has initiated a Phase 2a clinical trial to evaluate bryostatin for the treatment of patients with Alzheimer’s disease (AD). Bryostatin is a potent modulator of the enzyme protein kinase C epsilon (PKCe). In preclinical in vivo models, this effect has been shown to play an important role in slowing or reversing AD and restoring cognition, memory and motor skills. The top-line analysis of this study is expected to be reported by the end of the first quarter of 2015.
“Advancing the clinical development of bryostatin in patients with AD is a significant milestone for Neurotrope,” said Charles Ramat and Paul Freiman, the company’s co-chairmen and co-chief executive officers. “We believe bryostatin may restore synaptic structures and functions damaged by AD, leading to improvements in cognition and memory, thereby improving quality of life in AD patients. With 36 million people worldwide estimated to have AD, there is significant commercial potential for a new therapeutic that is effective in delaying the progression of the disease.”
In this randomized, double-blind, placebo-controlled, single dose study, a total of 15 patients will be enrolled. Ten patients will be randomized to receive bryostatin by injection and five will receive a matching placebo control. The primary objective of the clinical trial will be to assess the safety and tolerability of a single dose of bryostatin in the treatment of patients with AD. The secondary objectives of the study are the preliminary evaluation of the efficacy of a single dose of bryostatin in the treatment of patients with AD, its pharmacokinetics and pharmacodynamics and to correlate the changes in PKCe with plasma levels of bryostatin and with improvement in cognitive function.
Bryostatin is a natural product produced by a marine invertebrate organism called Bugula neritina and is isolated from organic matter harvested from the ocean. Several variations of this complex product have been developed in recent years in several academic chemistry laboratories, including bryostatin derivatives being developed through an exclusive license with Stanford University and the existing license agreement with the Blanchette Rockefeller Neurosciences Institute (BRNI). These approaches may represent alternative sources of drug supply.
Date: July 29, 2014