RARE Daily

Roche Acquires Enterprise Therapeutics’ Cystic Fibrosis Program for $97 Million Upfront

October 7, 2020

Rare Daily Staff

Enterprise Therapeutics said Roche has acquired its novel cystic fibrosis therapeutic approach that could treat all patients independent of CFTR mutation genotype, a deal that could challenge Vertex Pharmaceuticals’ supremacy in the space.

Under the terms of the deal, Enterprise’s shareholders received an upfront payment of $97 million (£75 million) and are eligible to receive additional contingent payments, to be made based on the achievement of certain predetermined milestones.

“We have deep capabilities in this area and look forward to a robust program focused on helping cystic fibrosis patients and patients suffering from other muco-obstructive disorders as quickly as possible,” said James Sabry, global head of pharma partnering for Roche.

Enterprise’s novel TMEM16A potentiator portfolio, including phase 1 candidate ETD002, is focused toward treating all people with cystic fibrosis (CF), with potential to benefit people with other severe respiratory diseases characterized by excessive mucus congestion.

Cystic fibrosis (CF) is estimated to affect 75,000 people globally. CF is caused by loss of function mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene that normally produces an anion channel highly expressed by the airway epithelium. Increasing anion conductance via CFTR modulation is a clinically validated approach for treating CF, however it is not currently available or effective for all people with CF. Vertex’s drugs can only treat up to 90 percent of CF patients.

One of the main causes of difficulty in breathing and increased risk of infection is mucus congestion in the lungs. The ETD002 compound targets the underlying mechanisms of mucus congestion, and is expected to restore lung function, reduce the frequency of lung infections and improve patient quality of life.

In pre-clinical models, Enterprise has demonstrated that ETD002 enhances the activity of TMEM16A, an alternative anion channel present in airway epithelial cells, and by doing so increases anion and fluid flow into the airways, thinning the mucus and increasing its clearance. As TMEM16A potentiation is independent of the mutational status of CFTR, this makes the approach potentially applicable to all people with CF, and perhaps patients with non-CF muco-obstructive lung disease.

Photo: James Sabry, global head of pharma partnering for Roche


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