The Cystic Fibrosis Foundation has invested more than $6.6 million in additional funding in Sionna Therapeutics to continue research into the development of potential new modulator therapies that could provide alternatives to others already on the market.

Sionna has been testing various compounds that could form a potential modulator therapy. Two are in Phase 1 clinical trials, and Sionna plans to move two more into Phase 1 trials in 2024. The Foundation’s funding will help Sionna advance these compounds through clinical trials so the company can identify a lead drug to advance into a Phase 2a trial.

These novel compounds target different places in the cystic fibrosis transmembrane conductance regulator (CFTR) protein, one of which, called NBD1, is the region of the protein where the F508del mutation is found. Previous attempts to address this particular area of the CFTR protein had been unsuccessful.  

“We are committed to developing additional modulator options that not only could be more effective than those on the market, but also could provide more options to people with CF — especially those who cannot tolerate current modulator therapies because of side effects,” said JP Clancy, MD, senior vice president of clinical research at the Cystic Fibrosis Foundation. “Simultaneously, we are working to develop treatment options for those who are not eligible for modulators, especially those with two copies of nonsense and rare mutations.”

The Foundation has been supporting the development of this type of modulator therapy for more than a decade by funding early-stage F508del corrector discovery work at Genzyme/Sanofi that would eventually lead to Sionna’s current pipeline. Since 2011, the Foundation has provided about $51 million to this research, including about $13 million directly to Sionna.