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Sionna Therapeutics Announces Initiation of PreciSION CF Phase 2a Trial Evaluating NBD1 Stabilizer, SION-719, When Added to Standard of Care in Participants with Cystic Fibrosis
SION-719 is the first NBD1 stabilizer being evaluated in people living with cystic fibrosis Topline data expected in mid-2026 WALTHAM, Mass., Oct. 21, 2025
About this update from Sionna Therapeutics, Inc.
[{"type":"text","content":"SION-719 is the first NBD1 stabilizer being evaluated in people living with cystic fibrosis Topline data expected in mid-2026 WALTHAM, Mass., Oct. 21, 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 initiation of the PreciSION CF Phase 2a proof-of-concept (POC) trial evaluating SION-719, a first-in-class nucleotide binding domain 1 (NBD1) stabilizer, when added to Trikafta® (elexacaftor/tezacaftor/ivacaftor), the current standard of care (SOC) in CF. “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 none of the currently approved CF therapies directly stabilize NBD1. We are thrilled to initiate the first clinical trial to evaluate an NBD1 stabilizer in people living with CF,” said Charlotte McKee, M.D., Chief Medical Officer of Sionna. “We are confident in the potential of NBD1 and believe that adding SION-719 to the standard of care could demonstrate clinically meaningful improvement in CFTR function. If the trial is successful, we believe it would represent an important step in validating the NBD1 mechanism and would provide evidence that NBD1 stabilization is mechanistically unique from, and synergistic with, the components of standard of care.” The PreciSION CF Phase 2a trial is a randomized, double-blind, placebo-controlled, crossover POC study that is enrolling adult CF patients homozygous for F508del on a stable dose of physician-prescribed Trikafta. The objectives of the trial are to evaluate the safety, tolerability, and pharmacokinetics (PK) of SION-719 when administered with SOC and to assess change in CFTR function as measured by sweat chloride levels, an important measure of CFTR function. The PreciSION CF trial is being conducted at multiple sites, including sites in the CF Foundation-supported Therapeutics Development Network (TDN), the largest CF clinical trials network in the world. Topline data are anticipated in mid-2026. In its recent Phase 1 trial in healthy volunteers, SION-719 was generally well tolerated and exceeded PK concentrati...