Sionna Therapeutics (SION) Earnings Call Presentation

Sionna Therapeutics Overview - Sionna's approach to stabilizing NBD1 has the potential to revolutionize CF treatment, addressing the unmet need for >66% of patients who lack normal CFTR function[7] - The CFTR modulator market is currently >$11 billion and is projected to reach $15 billion by 2029[7] - Sionna has a robust clinical-stage pipeline of NBD1 stabilizers and complementary modulators, offering multiple paths to transform CF patient care[7] Phase 1 Clinical Trial Results - Both NBD1 stabilizers, SION-719 and SION-451, exceeded desired PK targets and were generally well-tolerated in Phase 1 trials[13] - SION-719 is advancing to a Phase 2a proof-of-concept trial as an add-on to standard of care (SOC), based on its high potency at low doses[13] - SION-451 is advancing as an anchor in proprietary dual combinations, based on higher exposure achieved[13] Clinical Development Strategy - A Phase 2a proof-of-concept trial for SION-719 in CF patients is planned for the second half of 2025[14] - A Phase 1 healthy volunteer dual combination trial for SION-451 is also planned for the second half of 2025[14] - Sionna's cash runway extends into 2028, supporting the execution of its clinical development plans[14, 61] Market Opportunity - Approximately 106,000 patients worldwide have CF, with ~90% having at least one F508del-CFTR mutation[55] - >66% of patients on SOC do not have normal CFTR function, highlighting the significant unmet need[55, 59]