Business
Sionna Therapeutics Announces First Subjects Dosed in Phase 1 Trial Evaluating NBD1 Stabilizer, SION-451, in Proprietary Dual Combinations in Development for the Treatment of Cystic Fibrosis
Dosing initiated for SION-451 combined with SION-2222 and SION-451 combined with SION-109 in healthy volunteers; topline data anticipated in mid-2026 WALTHAM,
About this update from Sionna Therapeutics, Inc.
[{"type":"text","content":"Dosing initiated for SION-451 combined with SION-2222 and SION-451 combined with SION-109 in healthy volunteers; topline data anticipated in mid-2026\nWALTHAM, Mass., Aug. 25, 2025 (GLOBE NEWSWIRE) -- Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (CF) by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, today announced that the first subjects have been dosed in a Phase 1 trial evaluating SION-451, a first-in-class nucleotide binding domain 1 (NBD1) stabilizer, in proprietary dual combinations with SION-2222 (galicaftor), a transmembrane domain 1 (TMD1)-directed CFTR corrector, and with SION-109, an intracellular loop 4 (ICL4)-directed CFTR corrector. This Phase 1 trial is a randomized, double-blind, placebo-controlled trial in healthy volunteers. It is designed to assess the safety, tolerability, and pharmacokinetics (PK) of varying doses of dual combinations of SION-451 with SION-2222 and SION-451 with SION-109. Topline data are anticipated in mid-2026, and will inform selection of a dual combination for a planned Phase 2b trial in people living with CF. “I’ve spent many years as a pulmonary and critical care specialist caring for CF patients in the clinic, and I’ve been in a unique position to experience the evolution of treatment firsthand,” said Charlotte McKee, M.D., Chief Medical Officer of Sionna. “At Sionna, our research and understanding of the CFTR protein and the underlying causes of CF have led us to this exciting point, where we are taking the first ever NBD1-anchored dual combinations forward in clinical trials with the goal of revolutionizing the treatment paradigm for CF patients.” CF is a progressive and life-threatening genetic disease that affects more than 100,000 people globally. While advances in the discovery and development of modulators have significantly improved the lives of people living with CF, at least two-thirds of patients on the current standard of care do not have normal CFTR function as measured by levels of sweat chloride. The NBD1 domain of the CFTR protein plays a critical role in the folding, stability and trafficking of CFTR to a cell’s surface, but no approved CF therapies directly stabilize...