Sionna Therapeutics (SION) Corporate presentation summary

Mission and leadership

• Aims to revolutionize cystic fibrosis (CF) treatment by targeting the underlying protein defect.

• Leadership team and board have extensive experience in biotech and rare disease drug development.

Scientific foundation and pipeline

• Over 15 years of CFTR research, with origins in Genzyme and Sanofi, underpin the approach.

• Focuses on stabilizing NBD1, a critical domain in CFTR, to restore function in F508del mutation carriers (~90% of CF patients).

• Pipeline includes NBD1 stabilizers (SION-719, SION-451), complementary modulators (SION-2222, SION-109), and proprietary dual combinations.

Clinical progress and milestones

• Positive Phase 1 data for SION-719 and SION-451 as single agents, showing favorable safety and pharmacokinetics.

• SION-719 Phase 2a proof-of-concept trial as add-on to standard of care (SOC) completed enrollment; topline data expected summer 2026.

• SION-451 dual combination Phase 1 trial ongoing; topline data also expected summer 2026.