Company: SION
Filing Date: 2025-05-12
Form Type: 10-Q
Source: 0002036042-25-000016
Chunk: 219

Company: Sionna Therapeutics, Inc.
Filing Date: 2025-05-12
Form: 10-Q
Item: Part I, Item 1
Chunk 219
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 results that may be expected for any period in the future. For convenience of presentation, some of the numbers have been rounded in the text below.

Overview

We are a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (“CF”) patients by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (“CFTR”) protein to deliver clinically meaningful benefit to CF patients. Our goal is to deliver differentiated medicines for people living with CF that can restore their CFTR function to as close to normal as possible by directly stabilizing CFTR’s nucleotide-binding domain 1 (“NBD1”). We believe stabilizing NBD1 is central to unlocking dramatic improvements in clinical outcomes and quality of life for CF patients. 

We believe our robust pipeline of NBD1 stabilizers and complementary CFTR modulators provide multiple potential pathways to achieving our goal, either in combination with each other to produce a proprietary combination CF therapy, or an NBD1 stabilizer in combination with the current standard of care for CF. We plan to evaluate multiple NBD1 stabilizer candidates and complementary modulator candidates and select the most promising candidates to advance into later-stage development.

In February 2025, we disclosed interim data from our two randomized, double-blinded, placebo-controlled Phase 1 clinical trials of our highly potent NBD1 stabilizers—SION-719 and SION-451—evaluating the safety, tolerability and pharmacokinetic ("PK") profile of single ascending doses ("SAD") and multiple ascending doses ("MAD") of each product candidate in healthy subjects. As of the interim data cutoff date of January 14, 2025, five SAD cohorts and three MAD cohorts of SION-719 had been completed, with over 60 healthy subjects dosed, and six SAD cohorts and three MAD cohorts of SION-451 had been completed, with over 70 healthy subjects dosed. Both SION-719 and SION-451 were generally well tolerated based on interim Phase 1 clinical data as of the interim data cutoff date. As of the interim data cutoff, at both single and multiple doses, SION-719 and SION-451 exposures were achieved that have the potential, based on our preclinical cystic fibrosis human bronchial epithelial (“CFHBE”) model, to provide clinically meaningful benefit if SION-719 or SION-451 were administered as part of a dual combination or as an