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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
Phase 1 data of first-in-class NBD1 stabilizers, SION-719 and SION-451, demonstrated they were generally well tolerated and exceeded desired pharmacokinetic
About this update from Sionna Therapeutics, Inc.
[{"type":"text","content":"Phase 1 data of first-in-class NBD1 stabilizers, SION-719 and SION-451, demonstrated they were generally well tolerated and exceeded desired pharmacokinetic targets New preclinical data show that NBD1 stabilizers restored the half-life of F508del-CFTR up to wild-type levels WALTHAM, Mass., Oct. 24, 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 data presented at the 2025 North American Cystic Fibrosis Conference (NACFC) being held in Seattle, Washington October 22-25. “We are focused on advancing therapies that have the potential to make a meaningful difference for people living with CF, a mission underscored by the data presented this week at NACFC,” said Mike Cloonan, President and Chief Executive Officer of Sionna. “With multiple nucleotide binding domain 1 (NBD1) programs in the clinic, and the recent initiation of our Phase 2a proof-of-concept study in CF patients, we are at an important stage for our company and for people living with CF. The Phase 1 data highlighted at NACFC provides tremendous momentum as we continue to advance our NBD1 stabilizers to the next phases of development, with read-outs from our ongoing trials expected in mid-2026.” SION-719 and SION-451 Phase 1 Data Jason H. Maley, M.D., MS, Senior Director of Clinical Development at Sionna, presented data from the two Phase 1 clinical trials of SION-719 and SION-451, the company’s first-in-class nucleotide-binding domain 1 (NBD1) stabilizers. The randomized, double-blind, placebo-controlled Phase 1 trials enrolled over 200 healthy volunteers and evaluated each compound’s safety, tolerability, and pharmacokinetics (PK) across single and multiple ascending dose cohorts. As previously disclosed, Sionna’s two Phase 1 trials demonstrated that both SION-719 and SION-451 were generally well tolerated and exceeded target exposure levels. Based on these Phase 1 data and its cystic fibrosis human bronchial epithelial (CFHBE) model, Sionna believes that its NBD1 stabilizers have the potential to deliver clinically meaningful benefit when SION-719 is added to the standard of car...