Enterprise raises $33M to fund its Phase 2a trial of EDT001 for CF
Trial to enroll patients ineligible for, or not receiving, CFTR modulators
Enterprise Therapeutics has raised £26 million (nearly $33 million USD) in series B financing, which the company is planning to use to fund a Phase 2a clinical trial of EDT001, its experimental treatment for cystic fibrosis (CF).
The upcoming trial is expected to enroll people with CF who are either ineligible for or are not receiving CFTR modulators, according to the company.
The trial will use a crossover design — meaning patients will be split in two groups and then will be switched — and will assess if EDT001 treatment can improve lung function as measured by forced expiratory volume in one second, with the aim of providing a clinical proof-of-concept for EDT001. Enterprise did not specify when the trial is expected to begin but is targeting early 2024.
New round of investment funding led by Panakes Partners
The new round of investing to fund the trial was led by Panakes Partners. Rob Woodman, PhD, a partner at Panakes, will be joining Enterprise’s board of directors.
“Knowing the quality and track record of the Enterprise team, I am delighted to now be part of the next stage of its development,” Woodman said in a company press release. “We strongly believe that Enterprise’s approach offers the potential to impact the future of therapeutics for a range of respiratory diseases, including cystic fibrosis.”
CF is caused by mutations in the gene CFTR, which provides instructions for making a protein of the same name. The CFTR protein normally helps control the movement of chloride ions in and out of cells, which is critical for producing wet, slippery mucus. In CF, the CFTR protein is dysfunctional or entirely absent, leading to the production of thick, sticky mucus that causes most disease symptoms.
CFTR modulators are a recently developed class of therapy to improve the functionality of the CFTR protein in people with some specific mutations. While these treatments have revolutionized care for many patients, about 10% are ineligible for currently available modulators.
EDT001 is designed to target a protein called ENaC (epithelial sodium ion channel). Like CFTR, ENaC helps regulate the amount of fluid in the lungs. By targeting ENaC, EDT001 is expected to increase the amount of water in mucus, helping mucus be more slippery and thereby easing CF symptoms.
Notably, since it doesn’t target the CFTR protein itself, EDT001 is expected to be similarly effective regardless of a patient’s specific mutations.
According to its website, Enterprise seeks to discover disease-modifying therapies that target the underlying mechanisms of mucus congestion in respiratory diseases such as CF, asthma, and chronic obstructive pulmonary disease.