Enterprise Therapeutics: Exclusive: UK biotech raises $33.1M to test CF drug that could work on top of standard of care

Enterprise Therapeutics closed a £26 million Series B round to fund a Phase IIa trial of its cystic fibrosis drug, which aims to significantly improve hydration and clear away the mucus that makes breathing difficult for these patients. Panakes Partners led the round, along with participation from big-name existing investors like Versant Ventures, Novartis Venture Fund and Forbion. The UK-based biotech plans to dose the first patient with ETD001, its lead drug, in a Phase IIa trial around Easter, CEO John Ford told Endpoints News, with the first batch of data set to come out in mid-2025. The trial, which will be conducted in the UK and Europe, has already secured regulatory approval. The trial will assess lung function in people with cystic fibrosis who are either ineligible for or are not receiving CFTR modulators like Vertex's Trikafta and Symdeko. Only some cystic fibrosis patients have certain CFTR mutations that make them eligible for these drugs. "What we wanted to do is bring other mechanisms to the field that weren't dependent on CFTR repair therapy, which Vertex has done a fantastic job," Ford said. He added that there's still 10% or 15% of patients whose genotypes don't respond to Vertex's CFTR modulators, so other mechanisms are needed. ETD001 targets the ENaC ion channel in the airway. The molecule blocks the channel in order to draw water into the mucus, hydrating it and clearing it quickly from the lung. That can lead to fewer infections, an easier time breathing and improved lung function. Investor interest stemmed in part from positive Phase I data in healthy volunteers that showed the molecule stayed in the lungs for a long period of time, according to Ford. He added that Enterprise's drug could potentially work on top of the standard of care in cystic fibrosis, which includes Vertex's CFTR modulators, as well as other respiratory diseases linked to mucus-obstruction such as COPD. "Mucus is pretty important in other diseases of the lung, so we've been very conscious of COPD and other indications such as bronchiectasis," Ford said. "There's a bigger play on this, but the unmet need, which we're absolutely working so hard for with this funding, is to get to those patients that don't have any therapy or have limited treatment options in CF."