Most current therapies being investigated for neurodegenerative diseases target only a single type of misfolded protein, not addressing the fact that these diseases are often characterized by the buildup of multiple types of pathologic protein aggregates. Our scientists have developed a novel proprietary technology known as GAIM, or General Amyloid Interaction Motif, that simultaneously targets multiple misfolded proteins implicated in neurodegenerative diseases, potentially creating a more robust response that could be suitable for patients at all stages of disease progression.
Our GAIM-based therapies use a novel mechanism to bind to the amyloid fold of toxic protein aggregates of various types, including amyloid-β (Aβ), Tau, and a-synuclein aggregates, which accumulate in brain to cause Alzheimer’s and Parkinson’s disease, and antibody light chain (AL) or transthyretin (TTR) aggregates, which accumulate in peripheral organs to cause systemic amyloidosis orphan diseases. Our therapies are designed to prevent the further accumulation of aggregates and clear existing aggregates from the brain and peripheral organs, while also blocking further cell-to-cell spread of misfolded proteins. We believe these therapies offer a breakthrough approach to treating protein misfolding diseases.
Proclara is developing several product candidates for the treatment of a broad range of aging diseases and certain peripheral orphan indications. These include its lead development candidate, NPT088, which is currently in clinical development for Alzheimer’s disease, and the 2nd generation GAIM fusion NPT189, which is undergoing IND enabling studies to initiate clinical trials in 2018 for orphan systemic amyloidoses.