Intricate structure of the CFTR protein.
The CF Foundation recently announced two exciting developments in its ongoing effort to expand its pipeline of potential drugs to treat cystic fibrosis. In August, Cystic Fibrosis Foundation Therapeutics (CFFT) expanded its collaboration with Gilead Sciences, Inc. and launched a new collaboration with FoldRx Pharmaceuticals, Inc. Both Gilead Sciences and FoldRx may create significant opportunities to attack the basic defect for the majority of people living with CF.
Gilead Sciences, one of the world’s largest pharmaceutical companies, in collaboration with Parion Sciences, a development-stage pharmaceutical company, will develop a promising compound known as Parion 680. The agreement, which the Foundation helped broker, allows the two companies to pool their talent and advance critical research. The compound targets the overactive reabsorption of sodium into epithelial cells that leads to the development of thick mucus, which can lead to severe lung infections and irreversible lung damage. Parion 680 builds on very similar compounds developed through a $1.7 million grant to Parion from CFFT. Following further research, Parion plans to transition development responsibilities to Gilead during Phase 1 clinical trials.
“Gilead’s experience and resources will enable us to develop and test this compound much faster, perhaps years faster than we would have otherwise,” said Preston W. Campbell, III, M.D., the Foundation’s executive vice president of medical affairs.
By collaborating with Gilead and FoldRx, we are very optimistic about the chances to make real progress against CF."
Robert J. Beall, Ph.D.,
President and CEO
Increasing its chances to find new treatments and a cure for CF, CFFT has also partnered with FoldRx, a Massachusetts-based biotech firm that specializes in protein misfolding. As the body makes new proteins, they are shaped or “folded” into 3-D structures. Like a key that fits into a lock, the shape of the protein determines the function it will perform. A perfectly formed protein will work exactly as the correct key does to open a lock, whereas a misshaped protein, like the wrong key, will not function properly.
FoldRx aims to determine which new compounds could improve the function of the misfolded CFTR protein—the main protein associated with CF.
“If FoldRx were successful, it could lead to the design of drugs that effectively treat the disease, but we need to be cautiously optimistic. There’s still a long road of testing ahead of us before we can really find out the impact this technology could have,” said Campbell.
FoldRx will receive up to $22 million over five years upon successful completion of specific research and development milestones, including the planned development of two clinical candidates to begin testing in humans through clinical trials.
“We are a small disease, but as we have proven again and again, making the right investment in the early stages has an impact on how drugs are developed for cystic fibrosis,” said Robert J. Beall, Ph.D., the Foundation’s president and CEO. “By collaborating with Gilead and FoldRx, we are very optimistic about the chances to make real progress against CF.”