Company: SION
Filing Date: 2025-03-20
Form Type: 10-K
Source: 0002036042-25-000005
Chunk: 29

Company: Sionna Therapeutics, Inc.
Filing Date: 2025-03-20
Form: 10-K
Item: Item 1
Chunk 29
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.2; -8.3; p<0.0001) mmol/L, respectively. After termination of the trial drug, mean sweat chloride concentrations returned to baseline values in all treatment groups. Percent predicted FEV1 and CFQ-R did not significantly improve in any group.

•Trial GLPG-2222-CL-201 was a Phase 2 trial conducted by Galapagos in Europe and Australia that evaluated the safety and tolerability and the effect on CFTR function (sweat chloride), lung function (ppFEV1) and CFQ-R of galicaftor in 37 CF patients that were heterozygous for F508del and a gating mutation, receiving ivacaftor. The trial was completed in August 2017.

•Two doses of galicaftor were tested in this trial (150 mg or 300 mg QD) over 29 days.

•Galicaftor was well-tolerated in this trial. Most treatment-emergent AEs were mild in severity, and there were no SAEs.

•Changes from baseline in sweat chloride, ppFEV1 and CFQ-R at day 29 were also assessed as secondary endpoints. The galicaftor 300 mg QD treatment group had a statistically significant LS means with CI difference compared to placebo of -11.7 (95% CI, -21.1; -2.2) mmol/L (p=0.0170). There were no statistically significant changes in sweat chloride in the 150 mg and 300 mg treatment groups. Lung function and CFQ-R did not significantly improve in any group. All results are described for the modified intent to treat population, which excluded one subject who received an incorrect trial kit.

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These Phase 2 results demonstrated that galicaftor as a single agent increased CFTR activity in patients with the F508del mutation. The activity of galicaftor in combination with navocaftor in Trial M19-530 was similar to the activity of approved dual combination modulators, as seen via indirect, cross-trial comparisons and as predicted based on our CFHBE model (Figure 25). These data supported the selection of a 200 mg dose QD of galicaftor for subsequent trials in combination with navocaftor.

Figure 25. Galicaftor + Navocaftor Combination Showed Similar Activity to Symdeko in Phase 2 Cross-Trial Comparison

In addition to galicaftor