Sionna Therapeutics Announces Presentation of SION-719 and SION-451 Phase 1 Data and Poster of New Preclinical Data on the Impact of NBD1 Stabilizers on F508del-CFTR Half-Life at the 2025 North American Cystic Fibrosis Conference

Core Insights - Sionna Therapeutics has presented promising Phase 1 data for its first-in-class NBD1 stabilizers, SION-719 and SION-451, indicating they are well tolerated and meet pharmacokinetic targets [1][4] - The company aims to transform cystic fibrosis treatment by developing therapies that normalize CFTR protein function, with ongoing clinical trials and a focus on advancing NBD1 stabilizers [2][8] Phase 1 Data - Phase 1 trials for SION-719 and SION-451 involved over 200 healthy volunteers, assessing safety, tolerability, and pharmacokinetics in a randomized, double-blind, placebo-controlled design [3] - Both compounds were found to be generally well tolerated and exceeded target exposure levels, supporting their potential clinical benefits when combined with standard care or other modulators [4] Preclinical Data - New preclinical studies demonstrated that NBD1 stabilizers significantly increased the half-life of F508del-CFTR protein to levels comparable to wild-type CFTR, even when used as single agents [5][6] - The F508del mutation destabilizes CFTR's NBD1 domain, leading to impaired function, and Sionna's NBD1 stabilizers may restore CFTR function effectively [6] Company Mission and Future Directions - Sionna Therapeutics is focused on developing differentiated medicines that can restore CFTR function for cystic fibrosis patients, leveraging over a decade of research on NBD1 [8] - The company has initiated a Phase 2a proof-of-concept study in CF patients, with further trial read-outs expected in mid-2026 [2]