Company: SION
Filing Date: 2025-01-17
Form Type: S-1
Source: 0001193125-25-008474
Chunk: 180

Company: Sionna Therapeutics, Inc.
Filing Date: 2025-01-17
Form: S-1
Chunk 180
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 conducted by AbbVie in Europe and the U.S. to evaluate the safety,                                                                                                                                                                                                   
 tolerability and efficacy of navocaftor alone and in combination with galicaftor in 76 CF patients that were homozygous for F508del mutation. The primary efficacy endpoint of this trial was absolute change in lung function (ppFEV1) from baseline (day 1) through day 29. The trial was completed in June 2022. |

| • |     | This trial included eight total arms: a single placebo arm, two arms with different doses of navocaftor in combination with 
 placebo for galicaftor, and five treatment arms across a range                                                              |

135

| of doses of galicaftor (doses 10 mg, 30 mg, 100 mg, 200 mg, 300 mg once daily (“QD”)) in combination with navocaftor 150 mg for 28 days. |

| • |     | The mean absolute change from baseline in ppFEV1 from baseline                                                                                                                                                                           
 through Day 29 ranged from -0.1 to 3.5 percentage points for the five dual combination dose groups (navocaftor 150mg and galicaftor across a range of doses). The least squares means (“LS means”) change from baseline in ppFEV1 was    
 statistically significant in the 200mg and 300mg galicaftor combination groups, 3.5 ppFEV1 (p<0.05) and 3.1 ppFEV1 (p<0.05),                                                                                                             
 respectively. These data are from the analysis in which all spirometry values were used, regardless of usage status of bronchodilator or airway clearance regimen before spirometry collection. LS means with standard errors (“SE”) and 
 p-values are from mixed-effect model repeat measures analysis.                                                                                                                                                                           |

| • |     | AbbVie also assessed sweat chloride improvement as a 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