Roche has bought a phase 1 pan-genotype cystic fibrosis program from Enterprise Therapeutics. The deal, which is worth £75 million ($97 million) upfront to Enterprise’s shareholders, gives Roche a TMEM16A potentiator designed to restore lung function in all cystic fibrosis patients.
A group of ex-Novartis researchers, later led by ex-Dezima executive John Ford, developed TMEM16A potentiator ETD002 as part of a broader push to create therapeutics that work in all patients with cystic fibrosis, a disease in which the big breakthroughs so far have benefited people with particular genotypes. The researchers identified TMEM16A as a good target in light of evidence its role in the transport of chloride could mitigate the effect of loss of function in the chloride channel CFTR.
In January, Enterprise researchers co-authored a paper on their work to realize that vision, in which they shared evidence that enhancing the activity of TMEM16A boosts epithelial fluid secretion and mucus clearance, regardless of whether CFTR is fully functional. Having hired Novartis Venture Fund managing director David Morris as chief medical officer, Enterprise moved ETD002 into the clinic in August.
The work has attracted the attention of the other big Swiss pharmaceutical company. Roche has bought ETD002 and the rest of Enterprise’s portfolio of TMEM16A potentiators and tasked its Genentech unit with developing the assets.
Genentech broke new ground in 1993 when it became the first company to win FDA approval for a cystic fibrosis drug in 30 years. The drug, Pulmozyme, remains part of the portfolio of products sold by Genentech’s parent company Roche, but other biotechs have taken over the cystic fibrosis market over the past decade.
Vertex won approval for its first cystic fibrosis drug Kalydeco in 2012 and followed up with a series of other assets that brought the power of that product to more patients. Last year, the FDA approved Trikafta, positioning Vertex to treat the 90% of patients who have at least one F508del mutation in the CFTR gene.
Work is underway at Vertex and other companies to raise the bar still higher. In ETD002, Roche has gained a drug that positions it to join the companies trying to rise to that significant challenge.
Enterprise will also continue working on the challenge. Roche’s acquisition of ETD002 leaves Enterprise focused on ETD001, which blocks the ENaC ion channel that is involved in the transport of sodium. Acting on ENaC could rehydrate airway surfaces and improve mucociliary clearance.