WALTHAM, Mass., Nov. 24, 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 participate in fireside chats at the following upcoming investor events: 8th Annual Evercore Healthcare Conference on Wed ...

Summary of Sionna Therapeutics Conference Call Company Overview - **Company**: Sionna Therapeutics (NasdaqGM:SION) - **Focus**: Development of treatments for cystic fibrosis (CF) targeting a differentiated mechanism involving NBD1 - **Market Size**: Current CF market valued at $11 billion, projected to grow to $15 billion, dominated by one player [2][2] Core Points and Arguments - **Unmet Need**: Despite advancements in CF treatment, a significant unmet need remains, particularly in achieving normal CFTR function for patients [1][1] - **Current Treatment Efficacy**: Only one-third of patients on standard care achieve normal CFTR function; Sionna aims to improve this with their combination strategy [2][2] - **Clinical Development**: - Two NBD1 stabilizers (719 and 451) are in development, with 719 advancing to a Phase 2a proof of concept study (PreciSION CF) [3][3] - The study aims to demonstrate the efficacy of NBD1 in improving CFTR function when added to standard care [6][6] - Data from the study is expected by mid-2026 [3][3] Study Design and Expectations - **Phase 2a Study**: - A two-way crossover design to assess safety, tolerability, and efficacy based on sweat chloride levels [5][5] - Targeting a minimum reduction of 10 millimoles in sweat chloride, which is expected to correlate with a 3% improvement in FEV1 [10][10] - **Patient Selection**: Focus on patients who do not achieve normal CFTR function on Trikafta but are not outliers in sweat chloride levels [19][19] Mechanism and Assay Validation - **Assay Confidence**: - The assay used for predicting clinical outcomes has been validated through historical data and comparisons with Vertex's methodologies [33][33] - Sionna's assay has shown predictive power, replicating clinical results of Vertex compounds [37][37] - **NBD1 Mechanism**: NBD1 is expected to provide significant improvements in CFTR function, potentially achieving wild-type levels when combined with other correctors [41][41] Future Development Path - **Regulatory Engagement**: Anticipated discussions with regulators regarding the development path, with FEV1 expected to be the primary registration endpoint [42][42] - **Combination Strategy**: The dual combination of 451 with either Sion-2222 or Sion-109 is prioritized for its potential to normalize CFTR function [31][31] Financial Position - **Cash Reserves**: As of Q3, Sionna has $325 million in cash, extending its runway into 2028, allowing for continued development of its clinical programs [51][51] Additional Insights - **Community Needs**: There is a strong demand for more treatment options in the CF community, emphasizing the importance of differentiated mechanisms [47][47] - **Capital Considerations**: Future advancements will depend on the ability to raise capital to support multiple development paths [27][27] This summary encapsulates the key points discussed during the Sionna Therapeutics conference call, highlighting the company's strategic focus, clinical development plans, and financial outlook.

Summary of Sionna Therapeutics FY Conference Call Company Overview - **Company**: Sionna Therapeutics (NasdaqGM:SION) - **Focus**: Development of treatments for Cystic Fibrosis (CF) targeting the NBD1 protein domain Industry Context - **Market Size**: The CF treatment market is currently valued at approximately $11 billion, predominantly controlled by Vertex Pharmaceuticals [3][39] - **Unmet Need**: About two-thirds of CF patients on standard care do not achieve normal CFTR function, indicating a significant opportunity for new treatments [1][3] Core Scientific Insights - **NBD1 Target**: NBD1 has historically been considered "undruggable" due to challenging chemistry and shallow binding pockets [6][7] - **F508 del Mutation**: This mutation affects approximately 90% of CF patients and causes instability in the NBD1 protein, leading to impaired functionality [7][8] - **Efficacy of NBD1 Stabilizers**: Preclinical data suggests that stabilizing NBD1 can achieve efficacy levels comparable to the current triple combination therapy, Trikafta [8][9] Clinical Development - **Current Studies**: - Phase 2A proof of concept study for NBD1 stabilizer 719 and a dual combination study with stabilizer 451 [4][5] - Both studies are expected to yield results in mid-2026 [5][36] - **Study Design**: The precision study will involve a two-way crossover design to assess the efficacy of 719 added to Trikafta, focusing on sweat chloride levels as a primary endpoint [19][21] Financial Health - **Cash Position**: As of Q3, the company reported $325 million in cash, providing a runway into 2028, which supports ongoing clinical trials and development milestones [39] Strategic Insights - **Dual Combination Strategy**: The company aims to combine NBD1 stabilizers with either an ICL4 corrector or a TMD1 corrector to enhance efficacy beyond current treatments [9][36] - **Selection of Compounds**: The decision to use 719 as an add-on and 451 for dual combination is based on their unique profiles and potency at different doses [16][18] Key Milestones - **Upcoming Data**: Results from both ongoing studies are anticipated in mid-2026, which will be critical for determining future strategies [5][36] Additional Considerations - **Assay Confidence**: The CFHBE assay used for testing has shown high predictive validity for clinical outcomes, enhancing confidence in the results [10][12] - **Market Research**: The threshold for clinically meaningful improvement has been set at a 10 millimole per liter change in sweat chloride, based on historical data and expert consensus [25][33] This summary encapsulates the key points discussed during the conference call, highlighting Sionna Therapeutics' strategic focus, clinical development plans, and financial health within the context of the CF treatment landscape.

Financial Performance - The company reported net losses of $54.8 million and $45.8 million for the nine months ended September 30, 2025, and 2024, respectively, with an accumulated deficit of $235.9 million as of September 30, 2025[85]. - For the three months ended September 30, 2025, total operating expenses decreased by $4.2 million to $23.8 million from $27.9 million in the same period of 2024[106]. - Research and development expenses decreased by $8.5 million to $16.0 million for the three months ended September 30, 2025, primarily due to a decrease in unallocated research and development expenses[107]. - For the nine months ended September 30, 2025, total operating expenses increased by $12.9 million to $65.4 million from $52.4 million in the same period of 2024[111]. - General and administrative expenses increased by $10.9 million to $20.3 million for the nine months ended September 30, 2025, primarily due to increased personnel-related expenses[114]. - Net cash used in operating activities was $49.7 million for the nine months ended September 30, 2025, primarily due to a net loss of $54.8 million[122]. - Interest income increased by $1.1 million to $3.5 million for the three months ended September 30, 2025, driven by increased investment in debt securities[109]. - Interest income increased by $4.0 million to $10.1 million for the nine months ended September 30, 2025, primarily due to increased investment in debt securities[117]. Funding and Capital - The company has raised aggregate net proceeds of $330.4 million from the sale and issuance of preferred stock since its inception in 2019, and $199.6 million from its IPO in February 2025[84]. - Net cash provided by financing activities was $204.1 million during the nine months ended September 30, 2025, primarily due to net proceeds from the IPO[126]. - The company expects existing cash and marketable securities to fund operations into 2028, but will need to raise additional capital in the future[127]. - As of September 30, 2025, the company had $325.0 million in cash, cash equivalents, and marketable securities[120]. Research and Development - The company anticipates that its research and development expenses will increase substantially as it advances its product candidates into later-stage development efforts[96]. - The company has initiated a Phase 2a trial for SION-719 in CF patients, with topline data expected in mid-2026[81]. - The company in-licensed three clinical-stage compounds from AbbVie to expand its portfolio, including galicaftor (SION-2222) which has completed Phase 2 trials[82]. - The company is currently engaged in discovery, preclinical studies, and clinical trials for its product candidates, with ongoing assessments of costs and timelines[129]. Regulatory and Compliance - The company has not generated any revenue to date and does not expect to do so for several years, if ever, pending regulatory approvals for its product candidates[88]. - The company expects general and administrative expenses to increase to support research and development activities and compliance with public company reporting[101]. - The company is classified as an "emerging growth company" and may utilize certain exemptions from reporting requirements until it no longer meets the criteria[136]. - The company will remain an emerging growth company until it achieves total annual gross revenue of at least $1.235 billion or meets other specified conditions[136]. - The company is also classified as a "smaller reporting company," allowing it to take advantage of scaled disclosures until certain revenue and market capitalization thresholds are met[138]. Risks and Uncertainties - The company may face negative impacts on its financial condition if it fails to raise additional funds or enter into strategic agreements[130]. - The company has the potential to delay or limit product development if it is unable to secure additional funding[131]. - The company is required to make estimates and assumptions that affect the reported amounts in its financial statements, which may differ from actual results[133]. - The company has elected to use an extended transition period for complying with new accounting standards, which may affect comparability with other public companies[136]. - There were no material changes to the company's critical accounting estimates during the nine months ended September 30, 2025[134]. - There were no material changes to the company's contractual obligations and commitments during the nine months ended September 30, 2025[132].

Financial Performance - Net loss for Q3 2025 was $20.3 million, compared to a net loss of $25.4 million in Q3 2024[8] - Total operating expenses for Q3 2025 were $23.8 million, compared to $28.0 million in Q3 2024[15] Research and Development - Research and development expenses for Q3 2025 were $16.0 million, down from $24.6 million in Q3 2024, primarily due to one-time charges in the prior year[7] - Initiated PreciSION CF Phase 2a trial for SION-719 in October 2025, with topline data anticipated in mid-2026[4] - Initiated Phase 1 trial for SION-451 in dual combinations in August 2025, with topline data also expected in mid-2026[4] - The FDA cleared an Investigational New Drug (IND) application for SION-451, supporting its advancement to later-stage development[4] - SION-719 and SION-451 were generally well tolerated in Phase 1 trials, exceeding target exposure levels[5] Administrative Expenses - General and administrative expenses increased to $7.8 million in Q3 2025 from $3.4 million in Q3 2024, mainly due to personnel-related costs and professional fees[8] Cash and Assets - Cash, cash equivalents, and marketable securities totaled $325.0 million as of September 30, 2025, expected to fund operations into 2028[9] - Total assets as of September 30, 2025, were $339.3 million, up from $185.8 million at the end of 2024[17]

Core Insights - Sionna Therapeutics has initiated the PreciSION CF Phase 2a proof-of-concept trial for NBD1 stabilizer SION-719 as an add-on to standard care for cystic fibrosis patients, with topline data expected in mid-2026 [1][3] - The company has also started a Phase 1 trial for SION-451 in dual combinations with other modulators, with results anticipated in mid-2026 [1][3] - Sionna maintains a strong cash position of approximately $325 million, which is expected to fund operations into 2028 [1][8] Pipeline Updates - The PreciSION CF Phase 2a trial will assess the safety, tolerability, and pharmacokinetics of SION-719, measuring CFTR function through sweat chloride levels [3] - A successful drug-drug interaction study confirmed that SION-719 can be safely combined with Trikafta, the current standard of care [3] - The Phase 1 trial for SION-451 will evaluate its efficacy in combination with SION-2222 and SION-109 in healthy volunteers [3] Business Highlights - In September 2025, Sionna appointed Caroline Stark Beer as Chief Business Officer, bringing over 20 years of life science industry experience [5] Financial Results - Research and development expenses for Q3 2025 were $16 million, down from $24.6 million in Q3 2024, primarily due to one-time charges from the previous year [6] - General and administrative expenses increased to $7.8 million in Q3 2025 from $3.4 million in Q3 2024, attributed to personnel costs and professional fees [7] - The net loss for Q3 2025 was $20.3 million, an improvement from a net loss of $25.4 million in Q3 2024 [7][14]

Core Insights - Sionna Therapeutics is focused on developing novel medicines to improve the treatment of cystic fibrosis by normalizing the function of the CFTR protein [2][4] - The company is advancing a pipeline of small molecules aimed at correcting defects caused by the F508del genetic mutation, which is significant for cystic fibrosis patients [2] Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company dedicated to revolutionizing cystic fibrosis treatment [2] - The company's approach involves stabilizing the nucleotide binding domain 1 (NBD1) of the CFTR protein, which is believed to be crucial for enhancing clinical outcomes and quality of life for cystic fibrosis patients [2] - Sionna is also developing complementary CFTR modulators to work synergistically with its NBD1 stabilizers [2] Investor Engagement - Sionna will participate in upcoming investor events, including the Guggenheim 2nd Annual Healthcare Innovation Conference and the Stifel 2025 Healthcare Conference [4] - Live webcasts of these presentations will be available on Sionna's Investor Relations website, with replays accessible after the events [1][4]

Core Insights - Sionna Therapeutics has presented promising Phase 1 data for its first-in-class NBD1 stabilizers, SION-719 and SION-451, indicating they are well tolerated and meet pharmacokinetic targets [1][4] - The company aims to transform cystic fibrosis treatment by developing therapies that normalize CFTR protein function, with ongoing clinical trials and a focus on advancing NBD1 stabilizers [2][8] Phase 1 Data - Phase 1 trials for SION-719 and SION-451 involved over 200 healthy volunteers, assessing safety, tolerability, and pharmacokinetics in a randomized, double-blind, placebo-controlled design [3] - Both compounds were found to be generally well tolerated and exceeded target exposure levels, supporting their potential clinical benefits when combined with standard care or other modulators [4] Preclinical Data - New preclinical studies demonstrated that NBD1 stabilizers significantly increased the half-life of F508del-CFTR protein to levels comparable to wild-type CFTR, even when used as single agents [5][6] - The F508del mutation destabilizes CFTR's NBD1 domain, leading to impaired function, and Sionna's NBD1 stabilizers may restore CFTR function effectively [6] Company Mission and Future Directions - Sionna Therapeutics is focused on developing differentiated medicines that can restore CFTR function for cystic fibrosis patients, leveraging over a decade of research on NBD1 [8] - The company has initiated a Phase 2a proof-of-concept study in CF patients, with further trial read-outs expected in mid-2026 [2]

Core Insights - Sionna Therapeutics has initiated the PreciSION CF Phase 2a proof-of-concept trial for SION-719, a first-in-class NBD1 stabilizer, in combination with the current standard of care, Trikafta, for cystic fibrosis treatment [2][3] - The trial aims to evaluate the safety, tolerability, pharmacokinetics, and efficacy of SION-719, with topline data expected in mid-2026 [3][4] Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel medicines to normalize CFTR protein function, specifically targeting the NBD1 domain [6] - The company aims to restore CFTR function to near-normal levels for cystic fibrosis patients, leveraging over a decade of research on NBD1 [6] Clinical Trial Details - The PreciSION CF Phase 2a trial is a randomized, double-blind, placebo-controlled study enrolling adult CF patients homozygous for F508del on a stable dose of Trikafta [3] - The trial will measure changes in CFTR function through sweat chloride levels, a key indicator of CFTR activity [3] Previous Trial Results - In a Phase 1 trial, SION-719 was well tolerated and exceeded pharmacokinetic concentration targets, indicating potential for clinically meaningful improvement over the standard of care [4] - A successful drug-drug interaction study confirmed that SION-719 can be safely administered with Trikafta [4]

Core Insights - Sionna Therapeutics is focused on developing novel medicines to improve the treatment of cystic fibrosis (CF) by normalizing the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein [1][3] Group 1: Upcoming Presentation - Data will be presented at the 2025 North American Cystic Fibrosis Conference (NACFC) in Seattle from October 22-25, 2025 [1] - The presentation will cover the safety, tolerability, and pharmacokinetics of novel NBD1 stabilizers SION-719 and SION-451 from two phase 1 first-in-human studies [2] - The session will take place on October 24, 2025, from 10:15 a.m. to 12:15 p.m. PT [2] Group 2: Research Focus - Sionna Therapeutics aims to restore CFTR function to near-normal levels by stabilizing CFTR's nucleotide binding domain 1 (NBD1) [3] - The company is advancing a pipeline of small molecules designed to correct defects caused by the F508del genetic mutation, which affects NBD1 [3] - Sionna is also developing complementary CFTR modulators to work synergistically with its NBD1 stabilizers to enhance CFTR function [3]