CFTR ICL4

- SION-109 is a small molecule targeting the interface between the ICL4 region and the NBD1 domain of the CFTR protein; designed to complement the company's NBD1 modulator portfolio - - First subject dosed in Phase 1 clinical trial - BOSTON, Jan. 24, 2024 /PRNewswire/ -- Sionna Therapeutics, a life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF), today announced that the first healthy subject has been dosed in a Phase 1 clinical trial of SION-109 following clearance of its Investigational New Drug application (IND) by the U.S. Food and Drug Administration (FDA). SION-109 is a small molecule designed to target the interface between the intracellular loop 4 (ICL4) region and the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. It is a complementary mechanism to the company's portfolio of compounds directly targeting NBD1, including SION-638, the first-in-class NBD1 stabilizer now in Phase 1. "Our ability to target NBD1 correction is truly novel, and with SION-109 now entering clinical development, we have the opportunity to develop proprietary combination treatments that have the potential to achieve full CFTR correction," said Mike Cloonan, President and Chief Executive Officer of Sionna. "Advancing our second investigational drug into Phase 1 is an achievement made possible by the tireless work of our research and development teams, who have deep experience in CF and a sharp focus on advancing these programs rapidly to deliver new options for people living with CF and their families." CF is caused by genetic mutations in the CFTR protein, an epithelial ion channel that is essential for producing healthy, freely flowing mucus in the airways, digestive system, and other organs. The most common mutation in CFTR, ΔF508, causes NBD1 to unfold at body temperature and severely impairs CFTR function. Complementary targets including ICL4 may enable combination treatments with the potential for full CFTR correction. It is estimated that 90 percent of people living with CF have the genetic mutation ΔF508. About Sionna Therapeutics Sionna Therapeutics is a clinical-stage life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF) by normalizing the function of CFTR, the key protein associated with disease progression in CF. Building on over a decade of extensive research on the genetic mutations associated with CF and founded in 2019, Sionna is advancing a pipeline of small molecules engineered to correct the protein defects caused by ΔF508, the most common mutation that affects the CFTR protein. The company has a first-in-class portfolio of programs directly targeting correction of NBD1, the key and unique mechanism to enable full restoration of ΔF508-CFTR function, and complementary programs targeting ICL4 and TMD1. Sionna's pipeline has the potential to deliver best-in-class efficacy and reach previously unachievable levels of long-term benefit for people with CF. For information about Sionna visit . Media Contact Adam Daley Berry & Company Public Relations 212.253.8881 [email protected] Investor Contact [email protected] SOURCE Sionna Therapeutics