FoldRx Pharmaceuticals Inc, and Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), the nonprofit affiliate of the Cystic Fibrosis Foundation, announced today that FoldRx will receive up to $22 million over five years to develop treatment for 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.
Scientists believe that cystic fibrosis is caused by protein misfolding. An imperfectly folded protein can be rendered ineffective because its trafficking pathway is disrupted, leaving it unable to reach its target destination and leading to disease. An ability to repair this defect may offer a therapeutic strategy for treating a variety of protein-misfolding diseases, including CF, which affects 30,000 Americans. Ten million Americans are symptomless carriers of the disease.
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.
