Core Viewpoint - Sionna Therapeutics is focused on transforming the treatment of cystic fibrosis (CF) by developing innovative medicines that normalize the function of the CFTR protein [2]. Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company dedicated to revolutionizing CF treatment through novel medicines aimed at restoring CFTR function [2]. - The company aims to deliver differentiated therapies that stabilize the nucleotide binding domain 1 (NBD1) of the CFTR protein, which is believed to be crucial for improving clinical outcomes and quality of life for CF patients [2]. - Sionna is advancing a pipeline of small molecules designed to correct defects caused by the F508del genetic mutation in NBD1 and is also developing complementary CFTR modulators to enhance CFTR function [2]. Investor Engagement - Sionna will participate in investor events, including the Cantor Global Healthcare Conference on September 3, 2025, and the Morgan Stanley 23rd Annual Global Healthcare Conference on September 8, 2025 [4]. - Live webcasts of these presentations will be available on the company's Investor Relations website, with replays accessible after the events [1][3].

Core Insights - Sionna Therapeutics is focused on developing novel medicines to normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, aiming to revolutionize the treatment paradigm for cystic fibrosis (CF) [1][4] Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company dedicated to creating therapies that restore CFTR function, particularly targeting the nucleotide-binding domain 1 (NBD1) [4] - The company has a pipeline of small molecules designed to correct defects caused by the F508del genetic mutation, which is prevalent in CF patients [4] Research and Development - Sionna has developed NBD1 stabilizers, SION-719 and SION-451, which have shown the ability to increase the stability of isolated ΔF508-NBD1 by 16°C, surpassing wild-type NBD1 levels [5] - Preclinical data indicate that SION-719 and SION-451 can correct F508del-CFTR maturation to wild-type levels when combined with other modulators, SION-2222 and SION-109 [5] - The company plans to advance SION-719 into a Phase 2a proof-of-concept trial and SION-451 into a Phase 1 trial, both expected to start in the second half of 2025 [3] Conference Presentation - Sionna presented preclinical data at the 48th European Cystic Fibrosis Conference, showcasing the effectiveness of dual combinations of NBD1 stabilizers with proprietary modulators [1][3]

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]

Company Overview - Sionna Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on revolutionizing the treatment of cystic fibrosis (CF) by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein [3] - The company aims to deliver differentiated medicines that restore CFTR function to near-normal levels by directly stabilizing CFTR's nucleotide-binding domain 1 (NBD1), which is believed to be crucial for improving clinical outcomes and quality of life for CF patients [3] Pipeline and Research Focus - Sionna is advancing a pipeline of small molecules engineered to correct defects caused by the F508del genetic mutation located in NBD1 [3] - The company is also developing a portfolio of complementary CFTR modulators designed to work synergistically with its NBD1 stabilizers to enhance CFTR function [3] Upcoming Events - Management will present at The Citizens Life Sciences Conference on May 7, 2025, at 12:30 p.m. ET [1] - A live webcast of the presentation will be available on the company's Investor Relations website, with a replay accessible for 90 days post-event [2]

WALTHAM, Mass., April 30, 2025 (GLOBE NEWSWIRE) -- Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (CF) by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, today announced that management will present at The Citizens Life Sciences Conference on Wednesday, May 7, 2025 at 12:30 p.m. ET. A live webcast of the prese ...