Core Insights - Sionna Therapeutics has initiated the PreciSION CF Phase 2a proof-of-concept trial for SION-719, a first-in-class NBD1 stabilizer, in combination with the current standard of care, Trikafta, for cystic fibrosis treatment [2][3] - The trial aims to evaluate the safety, tolerability, pharmacokinetics, and efficacy of SION-719, with topline data expected in mid-2026 [3][4] Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel medicines to normalize CFTR protein function, specifically targeting the NBD1 domain [6] - The company aims to restore CFTR function to near-normal levels for cystic fibrosis patients, leveraging over a decade of research on NBD1 [6] Clinical Trial Details - The PreciSION CF Phase 2a trial is a randomized, double-blind, placebo-controlled study enrolling adult CF patients homozygous for F508del on a stable dose of Trikafta [3] - The trial will measure changes in CFTR function through sweat chloride levels, a key indicator of CFTR activity [3] Previous Trial Results - In a Phase 1 trial, SION-719 was well tolerated and exceeded pharmacokinetic concentration targets, indicating potential for clinically meaningful improvement over the standard of care [4] - A successful drug-drug interaction study confirmed that SION-719 can be safely administered with Trikafta [4]