FoldRx Pharmaceuticals Corporate Overview

FoldRx Pharmaceuticals is a development and discovery company focusing on first-in-class, disease-modifying, small molecule therapeutics to treat diseases of protein misfolding.

The folding and maintenance of proteins in a correctly folded active form is essential to normal cellular function. Accumulation of misfolded proteins, due to mutations or to defects in cellular quality control mechanisms, can prevent them from performing their normal function or result in toxic intermediates.

With an initial clinical program and a proprietary discovery platform, the company is establishing a leading position in this area. FoldRx’s lead drug candidate, tafamidis  (Fx-1006A), is being developed for two fatal diseases, TTR Amyloid Polyneuropathy (ATTR-PN) and TTR Amyloid Cardiomyopathy (ATTR-CM). FoldRx’s proprietary discovery platform uses a yeast-based model suitable as a powerful model system to discover drugs and drug targets for the treatment of neurodegenerative and other diseases resulting from protein misfolding. Using this broad discovery proprietary platform, the company has already built a pipeline of proprietary preclinical programs to treat neurological diseases such as Parkinson’s disease, Huntington’s disease and protein trafficking diseases, such as cystic fibrosis. In the near future, the company intends to further develop the platform to other neurodegenerative and protein trafficking diseases.