RARE Daily

Enterprise Therapeutics Raises $33.1 Million to Advance Lead Program for Cystic Fibrosis

January 30, 2024

Rare Daily Staff

Enterprise Therapeutics, a UK biotech focused on respiratory disease therapeutics, raised $33.1 million (£26 million) in a series B financing to advance its lead program to proof of concept to treat cystic fibrosis and expand its pipeline.

Panakes Partners led the financing, with participation by existing investors Versant Ventures, Novartis Venture Fund, Forbion, Epidarex Capital, and IP Group. Rob Woodman, partner at Panakes, joins Enterprise’s Board of Directors.

Cystic fibrosis is an inherited, progressive disease caused by mutations in the CFTR gene. Lung disease is the leading cause of morbidity and mortality in people with cystic fibrosis. Cystic fibrosis causes impaired lung function, inflammation and bronchiectasis and is commonly associated with persistent lung infections and repeated exacerbations due to the inability to clear thickened mucus from the lungs. People with cystic fibrosis require lifelong treatment with multiple daily medications. The complications of the disease result in progressive loss of lung function, increasing need for IV antibiotics and hospitalizations, ultimately leading to end-stage respiratory failure.

The investment will fund the phase 2a clinical trial of lead candidate ETD001, a novel low molecular weight compound with first-in-class potential, which targets the ENaC ion channel in the airway epithelium, increasing the hydration and clearance of mucus. ETD001 has been designed to be long acting, delivering durable target engagement, which is expected to drive substantial improvements in lung function. The trial will involve a cross-over design study assessing FEV1 lung function in people with cystic fibrosis who are either ineligible for or are not receiving CFTR modulators.

Enterprise’s pipeline of disease-modifying therapies targets the underlying mechanisms of mucus congestion, to enhance the clearance of mucus from the airways, restore lung function, and ultimately to reduce morbidity and mortality in chronic respiratory diseases of high unmet medical need, such as cystic fibrosis, chronic obstructive pulmonary disease, and severe asthma. Enterprise has established an Italian subsidiary to support R&D activities. This funding will enable the expansion of the company’s clinical activities into Italy via addition of clinical investigator sites and progression of its other preclinical programs targeting mucus congestion.

“We have made tremendous progress to date in developing novel therapeutics for patients suffering from chronic respiratory diseases,” said John Ford, CEO at Enterprise Therapeutics. “Such medicines are essential to reduce the frequency of lung infections and improve patient quality of life.”

Photo: John Ford, CEO at Enterprise Therapeutics

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