Our Science

Proclara’s novel approach to treating protein misfolding diseases is based on our discovery that several toxic misfolded protein aggregates share a common characteristic – the amyloid fold – that represents a promising target for drug development.

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) and tau, characteristics of Alzheimer’s disease. Our therapies are designed to prevent the further accumulation of aggregates and clear existing aggregates from the brain, 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 capable of targeting multiple misfolded proteins for the treatment of a broad range of aging diseases and certain orphan indications, including its lead development candidate, NPT088, which is currently in clinical development for Alzheimer’s disease.

 

An Urgent Need

Proteins are large, exquisitely folded molecules that play essential and diverse roles in the human body. When normal protein folding is disrupted in progressive brain diseases, these misfolded proteins clump together – binding to form toxic aggregates of plaques and tangles that cause inflammation, and eventually, nerve cell death.

Protein misfolding is a well-characterized hallmark of severe neurodegenerative diseases, including Alzheimers’ and Parkinson’s, as well as certain rare systemic amyloidosis diseases.

There is a critical unmet need to develop novel treatments for protein misfolding diseases, for which no approved disease-modifying treatments currently exist.

Neurodegenerative diseases including Alzheimer’s and Parkinson’s place an enormous economic and social burden on patients, caregivers, and society. Alzheimer’s is the most common neurodegenerative disease and the leading cause of dementia, which currently affects approximately 47 million victims worldwide. That number is expected to grow to 75 million by 2030.

Meanwhile, Parkinson’s, the second most common neurodegenerative disease, affects an estimated 7 to 10 million worldwide.