Company: SION
Filing Date: 2025-03-20
Form Type: 10-K
Source: 0002036042-25-000005
Chunk: 28

Company: Sionna Therapeutics, Inc.
Filing Date: 2025-03-20
Form: 10-K
Item: Item 1
Chunk 28
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 secondary efficacy endpoint. Mean SwCl improvement occurred in all groups with galicaftor 30 mg or higher, with improvement of 18.6 mmol/L in the 200 mg group (p<0.001) and 19.9 mmol/L in the 300 mg group (p<0.001).

•Galicaftor was generally well-tolerated in combination with navocaftor in this trial. Most reported AEs were mild to moderate in severity. Two serious adverse events (“SAEs”) occurred in two subjects receiving galicaftor in combination with navocaftor (ileus and cholecystitis acute); both were considered unrelated to the trial drugs by the investigator.

•Trial GLPG-2222-CL-202 was a Phase 2 dose-ranging trial conducted by Galapagos in Europe and the U.S. to evaluate the safety and tolerability and the effect on CFTR function (as assessed by sweat chloride), pulmonary function and the Cystic Fibrosis Questionnaire— Revised (“CFQ-R”), which measures health-related quality of life, of galicaftor in 59 CF patients that were homozygous for F508del mutation. The trial was completed in October 2017.

•Four doses of galicaftor were tested in this trial (50 mg, 100 mg, 200 mg, 400 mg QD) over 29 days.

•Galicaftor was generally well-tolerated in this trial. The majority of reported treatment-emergent AEs were mild or moderate in severity. A total of four SAEs were reported (two after galicaftor, two after placebo) in two subjects in the pooled placebo and one subject in the galicaftor 100 mg QD treatment group, respectively. The three subjects experienced one or two events of infective pulmonary exacerbation of CF, all of which were considered not related to trial drug.

•Sweat chloride levels, lung function (ppFEV1) and CFQ-R were also assessed as secondary endpoints. Mean sweat chloride concentrations decreased dose-dependently with increasing doses of galicaftor, with a maximum decrease observed in the 200 mg QD treatment group on days 15 and 29, with statistically significant LS means differences compared to placebo of -11.2 (95% CI, -19.1; -3.3; p=0.0062) and -15.8 (95% CI, -23