First People Dosed With Investigational Therapy EDT001

The first participants have been dosed in the first clinical trial testing EDT001, an investigational therapy for cystic fibrosis (CF) being developed by Enterprise Therapeutics.

The trial is being conducted in healthy participants (without CF) to assess the investigational medicine’s safety profile.

“We are excited to be entering the clinic with ETD001,” John Ford, PhD, Enterprise’s CEO, said in a press release.

CF is caused by mutations in the CFTR gene that provides instructions for making the CFTR protein, which regulates the transport of water and salts in and out of cells. One of the hallmark consequences of CFTR dysfunction in CF is the formation of abnormally thick mucus, which impairs the function of the lungs and other organs, and provides a breeding ground for infectious bacteria.

ETD001 is designed to target a protein called epithelial sodium ion channel, or ENaC. Like the CFTR protein, ENaC helps to coordinate the amount of water and other substances present in mucus.

By targeting ENaC, EDT001 aims to increase the amount of water in the mucus, making it less thick. This could improve lung function and life quality, and reduce the frequency of lung infections, in people with CF.

According to Ford, EDT001 “has best-in-class potential due to its long duration of action in the lung and favourable pre-clinical safety profile.”

The investigational therapy “could significantly improve quality of life for people living with cystic fibrosis and other respiratory diseases linked to mucus obstruction,” Ford said.

Notably, because it targets the ENaC protein, EDT001 is expected to work equally well in all people with CF, regardless of which type of CFTR mutation they have. This sets EDT001 apart from other CF treatments, such as CF modulators — a therapy class that works to correct the function of the CFTR protein in people with specific mutations.

“ENaC inhibition via ETD001 shows great promise in providing a therapeutic option for the many people with cystic fibrosis who are not genetically suited to or fail to benefit from CFTR modulators,” said David Morris, MD, chief medical officer at Enterprise.

“We look forward to generating our first data in human volunteers under this program,” Morris said.