Roche Pharmaceuticals has acquired a set of potentiator compounds from Enterprise Therapeutics, that it intends to develop into medicines for people with cystic fibrosis. The compounds will be developed by Genentech, a member of the Roche Group. Unlike other potentiators under development, the potential medicines target TMEM16A, an alternative chloride channel to CFTR. Though studies are in very early stages, researchers believe these therapies could potentially benefit all people with CF, regardless of their CFTR mutations. The Cystic Fibrosis Foundation previously awarded up to $7 million for development of one of the compounds, ETD002, which recently entered Phase 1 trials.
TMEM16A is a calcium activated chloride channel (CaCC) which opens in response to increases in calcium inside lung cells. The potentiator compounds are small-molecule drugs that make the CaCC channel more sensitive to small increases in calcium within the cells, thereby activating the channel more frequently and allowing more chloride to flow across the cell membrane. The increased flow of chloride is expected to help hydrate mucus within the airways, making it easier to expel and decreasing the likelihood of infections.
William Skach, M.D., Executive Vice President and Chief Scientific Officer for the Foundation, said he is delighted that companies continue to recognize the ongoing needs of people with CF and are joining the fight to raise the bar for new CF therapies.
“Our vision is to bring transformative, disease-modifying therapies for cystic fibrosis to every person with the disease. Roche has a long history in CF, beginning with Pulmozyme, and a reputation for delivering important medicines to patients with serious illnesses. We are excited to see them continue this track record of bringing forward novel treatments.”
Despite extraordinary progress in helping people with CF live longer and healthier lives, there is still critical work to be done to help all people living with this disease. Learn more about the potential treatments the Foundation is exploring for people with nonsense and other rare mutations that do not respond to CFTR modulators through our Path to a Cure.
For more information on this announcement, please see the Enterprise release.