Roche Acquires TMEM16A Portfolio for CF Treatment

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by Sara Guariglia |

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Roche has acquired Enterprise Therapeutics’s TMEM16A potentiator portfolio, which includes ETD002, a therapeutic candidate being evaluated in Phase 1 clinical trials for the treatment of cystic fibrosis (CF).

The TMEM16A portfolio is focused on treatments for CF and other severe respiratory disorders characterized by excessive mucus congestion. Genentech, a member of the Roche Group, now will lead the development of the TMEM16A portfolio.

“We are delighted that Roche … has decided to acquire Enterprise’s TMEM16A potentiator program, adding yet another exciting program to their respiratory portfolio,” Geert-Jan Mulder, MD, managing partner and co-founder at Forbion, said in a press release. “We expect to see further good progress of the company’s remaining program, a potential best in class ENaC-blocker that targets to restore mucus hydration making it easier for the lungs to be cleared.”

Forbion is a life sciences venture capital firm that had Enterprise Therapeutics in its company portfolio.

EDT002 is designed to increase the activity of TMEM16A — a chloride channel found on the epithelium (cells lining the respiratory tract). TMEM16A regulates the amount of salts and fluids in tissues, a balance that is needed for appropriate fluid flow and mucus clearance.

Excessive mucus buildup in the airways and an inability to expel mucus are common problems in CF, causing breathing difficulties and increasing the likelihood of severe lung infections.

A Phase 1 trial (NCT04488705) is currently investigating EDT002 as an inhaled treatment for CF. Recruitment for the study is ongoing in the U.K., and enrollment information can be found here. The trial seeks to enroll 88 healthy participants.

In preclinical studies, EDT002 successfully increased the activity of TMEM16A. This allowed for increased fluid balance in the respiratory pathways, resulting in lower mucus viscosity and an enhanced ability to clear mucus.

TMEM16A-targeted therapies can be used alone or in conjunction with other CF therapies, namely those that target the cystic fibrosis transmembrane conductance regulator (CTFR) gene —the defective gene in CF patients.

Importantly, TMEM16A potentiation works regardless of a patient’s CFTR mutational status, making this approach applicable to all patients with CF.

TMEM16A modulation also is expected to help those with other lung diseases characterized by excessive mucus buildup.

“Roche and Genentech have a proven track record of bringing new medicines to people with respiratory diseases, and have recognised the opportunity that our TMEM16A potentiator portfolio presents,” said John Ford, PhD, CEO of Enterprise Therapeutics.

“I am very proud of the team at Enterprise for identifying and developing this innovative approach to treat patients, with ETD002, the first of our compounds to reach the clinical stage. TMEM16A potentiation has the potential to significantly increase the quality of life for people living with cystic fibrosis, for many of whom existing therapies are not effective,” Ford added.

As part of the agreement, Enterprise’s shareholders received an upfront payment and are eligible to receive additional payments contingent on certain predetermined milestones.