Last week, NeuroPhage Pharmaceuticals announced positive pre-clinical data from studies into NPT001, a compound targeting protein aggregates in the brains of people with both Alzheimer’s and Parkinson’s disease (PD).
In PD, alpha-synuclein is the targeted culprit: All people with the disease experience clumping of the protein in their brains. While there remain questions as to whether alpha-synuclein plays a direct causative role in PD, targeting its aggregation has been of major interest to researchers aiming to develop disease-modifying drugs.
Since its discovery in PD in 1997, several different approaches to addressing alpha-synuclein build-up have emerged, including reducing alpha-synuclein gene expression, blocking its build-up, and targeting its toxicity in the cell. NPT001’s technology is designed to clear these protein aggregates.
According to the Cambridge, Massachusetts-based biotech, these recent studies showed that NPT001 did in fact reduce alpha-synuclein deposits in the brain, while also restoring dopamine-producing cells to normal function.
The next potential steps for NPT001 could be more advanced pre-clinical testing for efficacy and safety, and then readying the drug for clinical trials.
NeuroPhage is also seeking to develop the compound for Alzheimer’s (AD), by targeting two proteins implicated in that disease, beta amyloid and tau.