NeuroPhage’s NPT088 Universally Targets Misfolded Proteins in Preclinical Studies Highlighted in Oral Session at the Alzheimer’s Association International Conference
Cambridge, MA, July 19, 2015 – NeuroPhage Pharmaceuticals, Inc. today announced that the discovery, preclinical development and clinical trial approach for its lead candidate, NPT088, were highlighted in an oral session at the Alzheimer’s Association International Conference (AAIC) taking place July 18–23, 2015, in Washington, D.C. NPT088 contains the general amyloid interaction motif (GAIM), which allows the compound to universally recognize and disrupt the shape of misfolded proteins and target them for degradation through the body’s natural mechanisms. In the data presented at AAIC, administration of NPT088 led to broad beneficial effects in animal models of Alzheimer’s and Parkinson’s diseases, with improvements in memory and cognition and reductions in toxic protein aggregates.
“Multiple kinds of toxic misfolded proteins can appear together in a single disease, for example amyloid beta, tau, and alpha-synuclein deposits in brains of people afflicted with Alzheimer’s or Parkinson’s disease,” said Richard Fisher, PhD, Chief Scientific Officer of NeuroPhage. “NPT088, therefore, has tremendous therapeutic potential, because it has demonstrated ability to prevent the formation of and disrupt existing toxic aggregates of amyloid beta, tau and alpha-synuclein, leading to functional benefits in animal models. We look forward to extending this work into clinical studies and anticipate initiating Phase 1 trials in early 2016.”
In the research presented at AAIC, NPT088 was found to potently bind toxic aggregates of amyloid beta, tau and alpha-synuclein. In addition, NPT088 was able to block aggregate formation of amyloid beta and tau in vitro. NPT088 was also found to effectively treat Alzheimer’s and Parkinson’s disease models in vivo. In the Tg2576 hAPP model of Alzheimer’s disease, NPT088 significantly improved cognition, reduced brain amyloid beta toxic fragments and amyloid beta plaques, as well as amyloid beta in the cerebral spinal fluid (CSF). In the rTg4510 tau model of Alzheimer’s disease, NPT088 significantly improved cognition, reduced levels of toxic tau and lowered tau levels in the CSF. Finally, in the mThy1-Hα-synuclein model of Parkinson’s disease, NPT088 significantly reduced proteinase K-resistant alpha-synuclein and increased tyrosine hydroxylase levels, a measure of activity of the brain cells affected by Parkinson’s. Together, these results support the clinical development of NPT088. NeuroPhage expects to initiate first-in-human clinical studies of NPT088 in 2016 for the treatment of Alzheimer’s disease, where proof-of-activity can be measured with PET amyloid imaging for both amyloid beta and tau deposits.
NeuroPhage Pharmaceuticals, Inc. is a privately funded company headquartered in Cambridge, Massachusetts. NeuroPhage’s mission is to advance the treatment of misfolded protein diseases through GAIM therapy, simultaneously targeting multiple, toxic, misfolded proteins. NeuroPhage is initially developing drug candidates to treat Alzheimer’s and Parkinson’s diseases and several rare systemic amyloidosis diseases.