Sionna Therapeutics (SION) Corporate presentation summary

Leadership and expertise

• Executive team and board have extensive experience in biotech and rare disease drug development, with backgrounds at leading companies and institutions.

• Leadership includes expertise in CF research, clinical development, and commercialization.

Scientific foundation and pipeline

• Over 15 years of CFTR research led to a robust pipeline focused on NBD1 stabilizers and complementary modulators.

• NBD1 stabilizers SION-719 and SION-451 are advancing through clinical development, targeting the F508del mutation present in ~90% of CF patients.

• Pipeline includes dual combinations with TMD1- and ICL4-directed correctors, aiming for wild-type CFTR function.

Clinical progress and milestones

• Positive Phase 1 data for SION-719 and SION-451 showed favorable safety, tolerability, and pharmacokinetics.

• Ongoing Phase 2a POC trial for SION-719 as add-on to standard of care, and Phase 1 dual combo trial for SION-451, both with topline data expected mid-2026.

• Preclinical and clinical data suggest potential for clinically meaningful improvements in CFTR function, including to wild-type levels.