FoldRx’s Collaboration with Cystic Fibrosis Foundation Therapeutics

In 2007, FoldRx and Cystic Fibrosis Foundation Therapeutics (CFFT) announced a collaboration in which FoldRx will receive up to $22 million over five years to discover and develop new compounds aimed at treating a core defect in cystic fibrosis. The research, development and commercialization agreement — one of the largest of its kind for CFFT — calls for FoldRx to use its novel yeast-based, high throughput screening platform to detect new compounds that could improve the function of a misfolded protein associated with cystic fibrosis, thus helping treat the disease.

Cystic fibrosis is a fatal genetic disease that affects approximately 30,000 children and adults in the United States. It causes life-threatening lung infections and serious digestive complications. More than 10 million Americans are symptomless carriers of the CF gene.

FoldRx will retain full worldwide commercialization rights and receive CFFT payments upon successful completion of specific research and development milestones, including development of two clinical candidates to the point of Phase 1 clinical trials. The company will also assume part of the preclinical development costs and own all new intellectual property generated during the collaboration. CFFT will be eligible to receive royalties from FoldRx on net sales of any approved products.

Scientists believe that many diseases such as cystic fibrosis, Parkinson's and Alzheimer's are caused by protein misfolding. An imperfectly folded protein, sometimes resulting from major gene mutations, can be rendered ineffective because its trafficking pathway is disrupted, leaving it unable to reach its target destination and leading to disease. Recent studies suggest that the ability to repair this defect may offer a therapeutic strategy for treating a variety of protein-misfolding diseases, including CF.

FoldRx has developed a powerful yeast-based, high throughput screening platform designed to study the mechanism of protein misfolding and its relation to disease, and to identify small-molecule modulators of protein misfolding. Yeast is a well-recognized model system, as many of the proteins and cellular mechanisms in yeast have similar counterparts in humans. This collaboration with CFFT could eventually lead to the design of new drugs that will improve biological function of the mutated protein involved in CF, and thus treat the disease.