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Sionna Therapeutics Announces Positive Phase 1 Data for NBD1 Stabilizers SION-719 and SION-451 and Advances Both Programs in Clinical Development for Cystic Fibrosis
SION-719 and SION-451 were generally well tolerated and achieved desired pharmacokinetic targets that reinforce their potential as either an add-on to
About this update from Sionna Therapeutics, Inc.
[{"type":"text","content":"SION-719 and SION-451 were generally well tolerated and achieved desired pharmacokinetic targets that reinforce their potential as either an add-on to standard of care or in a Sionna dual combination Sionna plans to advance SION-719 to a Phase 2a proof-of-concept add-on to standard of care trial in cystic fibrosis patients, and SION-451 to a Phase 1 healthy volunteer dual combination trial Next trials to initiate in the second half of 2025 with topline data expected in mid-2026 Sionna to hold a conference call today at 8:00 a.m. ET WALTHAM, Mass., June 04, 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 positive data from its Phase 1 clinical trials of SION-719 and SION-451, the company’s first-in-class nucleotide-binding domain 1 (NBD1) stabilizers, in healthy volunteers. Both NBD1 stabilizers were generally well tolerated and achieved pharmacokinetic (PK) concentration targets established using Sionna’s preclinical CF human bronchial epithelial (CFHBE) model. These data reinforce the potential of both NBD1 stabilizers to provide clinically meaningful benefit for CF patients, including the potential for wild-type CFTR function, either as an add-on to standard of care (SOC), or in a proprietary dual combination with complementary modulators. Sionna plans to advance both NBD1 stabilizers to the next stage of clinical development — SION-719 to a Phase 2a proof-of-concept (POC) trial in CF patients as an add-on to SOC, and SION-451 to a Phase 1 proprietary dual combination trial in healthy volunteers. “Today’s announcement brings us one step closer to our vision of developing novel NBD1-led proprietary dual combinations to transform the treatment paradigm for CF patients. The successful completion of two NBD1 Phase 1 trials and being the first company to bring NBD1 compounds into the clinic are important milestones,” said Mike Cloonan, President and Chief Executive Officer of Sionna. “Based on these encouraging data, we are fortunate to have the option to advance the clinical development of both NBD1 stabilizers while maintaining our timeli...