WALTHAM, Mass., Jan. 05, 2026 (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 44th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13th, 2026 at 9:45 a.m. PT/12:45 p.m. ET ...

Core Insights - The transaction involving Advisors LLC Orbimed's sale of 33,356 shares of Sionna Therapeutics was primarily driven by the remaining share capacity, as their indirect holdings have significantly decreased in 2025, limiting further large-scale disposals [1][5]. Company Overview - Sionna Therapeutics is a clinical-stage biotechnology company focused on developing innovative treatments for cystic fibrosis, utilizing a specialized approach to CFTR modulation to address unmet medical needs in this patient population [7]. Transaction Details - The sale of 33,356 shares was valued at $1,486,009.80 and was executed on December 24, 2025, through indirect holdings attributed to OrbiMed Private Investments VIII, LP, with no direct ownership involved [5][6]. - This transaction represented 0.93% of Advisors LLC Orbimed's indirect holdings, indicating a structured approach to selling as their holdings near exhaustion [4][5]. Market Context - The size of the sale aligns with the median size of recent open-market sales, suggesting a continued pattern of structured, incremental block sales [3][5]. - Despite the sale, Advisors LLC Orbimed retains over 3.5 million shares, indicating that this transaction does not reflect a lack of confidence in Sionna Therapeutics [9]. Clinical Progress - Sionna Therapeutics raised approximately $191 million in its initial public offering in February and has made progress with its experimental treatments, including positive phase 1 results for lead candidates SION-719 and SION-451 [9][10]. - The company has initiated a phase 2 trial with SION-719 involving cystic fibrosis patients, with top-line results expected around mid-year [10].

The most talked about and market moving research calls around Wall Street are now in one place. Here are today's research calls that investors need to know, as compiled by The Fly.Top Upgrades: More bullish on the name, CFRA upgraded Pool Corp. (POOL) to Buy from Hold with a $304 price target.Piper Sandler upgraded SEI Investments (SEIC) to Overweight from Neutral with a price target of $102, up from $93, as part of a Q4 preview for the asset management group. The firm believes SEI will be a "clear benefic ...

Core Insights - The company, Sionna, is focused on developing treatments for cystic fibrosis (CF) by targeting a differentiated and previously considered undruggable target known as NBD1 [3] Company Overview - Sionna aims to transform the standard of care in cystic fibrosis, with NBD1 as the central element of its strategy [3] - The company has recently initiated a Phase IIa proof-of-concept study for its NBD1 stabilizer, SION-719, which will be tested in conjunction with TRIKAFTA, the current standard of care [4]

Financial Data and Key Metrics Changes - The company raised $219 million in its IPO in February, providing financial flexibility to execute its strategy through 2028 [7] - The company entered Q3 with $325 million in cash, ensuring a cash runway into 2028 [47] Business Line Data and Key Metrics Changes - The company is focused on cystic fibrosis (CF) and is developing two NBD1 stabilizers: SION-719 for an add-on approach and SION-451 for a dual combination strategy [5][6] - The goal is to improve CFTR function in patients, with a target of achieving at least a 10 millimole per liter change in sweat chloride as a primary endpoint in clinical studies [30][69] Market Data and Key Metrics Changes - The CF market is currently valued at $12 billion and is expected to grow to $15 billion [12] - The company aims to create more treatment options for the approximately 85%-90% of CF patients with the F508del mutation, as two-thirds of patients on standard care do not achieve normal CFTR function [16][19] Company Strategy and Development Direction - The company is pursuing a differentiated approach to CF treatment by targeting the NBD1 protein, which has been considered undruggable [4][10] - The strategy includes a dual combination approach to potentially provide meaningful clinical benefits above the current standard of care, which is a triple combination therapy [6][12] - The company is open to exploring new therapeutic areas in the future but is currently focused solely on CF [50][51] Management's Comments on Operating Environment and Future Outlook - Management believes that the current modulators have not fully addressed the unmet needs in CF, as many patients still do not achieve normal CFTR function [16] - The company is confident in its ability to leverage its historical research and partnerships, particularly with the Cystic Fibrosis Foundation, to advance its clinical studies [65][66] - Management emphasized the importance of patient access in pricing strategies, aiming to ensure that new therapies are accessible to patients [60] Other Important Information - The company has licensed three compounds from AbbVie, enhancing its portfolio with clinical data and complementary mechanisms [42][44] - The company is conducting a healthy volunteer phase 1 study for its dual combination strategy, which is expected to inform future development [38][40] Q&A Session Summary Question: What is the company's approach to addressing unmet needs in CF? - The company aims to improve CFTR function and believes that NBD1 stabilization is key to moving more patients to normal CFTR function [16][19] Question: How does the company plan to differentiate its products in the market? - The company is focused on improving efficacy through its dual combination strategy and leveraging historical data to predict clinical outcomes [67][70] Question: What is the expected timeline for clinical data? - The company anticipates data from its phase 2a proof of concept study in mid-2026, which will be crucial for future development decisions [5][30]

Financial Data and Key Metrics Changes - The company raised $219 million in its IPO in February 2025, providing financial flexibility to execute its strategy through 2028 [7] - As of Q3, the company reported having $325 million in cash, ensuring a cash runway into 2028 [46] Business Line Data and Key Metrics Changes - The company is focused on developing two NBD1 stabilizers: SION-719 for an add-on approach to the standard of care and SION-451 for a dual combination strategy [5][6] - The company aims to demonstrate a clinically meaningful improvement of at least 10 millimoles per liter in sweat chloride, which is expected to correlate with a 3-point improvement in FEV1 [34][35] Market Data and Key Metrics Changes - The cystic fibrosis market is currently valued at $12 billion and is projected to grow to $15 billion [12] - The company targets the same patient population as the standard of care, focusing on the F508del mutation, which affects 85%-90% of cystic fibrosis patients [19] Company Strategy and Development Direction - The company aims to transform the standard of care in cystic fibrosis by focusing on the NBD1 target, which has been historically considered undruggable [4][10] - The dual combination strategy is prioritized, with the goal of raising the efficacy bar through innovative combinations of therapies [6][12] Management's Comments on Operating Environment and Future Outlook - Management believes that there is a significant unmet need in the cystic fibrosis community, as two-thirds of patients on standard care do not achieve normal CFTR function [16] - The company is optimistic about its clinical development strategy and the potential to improve patient outcomes significantly [70] Other Important Information - The company has licensed three compounds from AbbVie, enhancing its portfolio with clinical data and complementary mechanisms [41][42] - The Therapeutics Development Network (TDN) of the Cystic Fibrosis Foundation is a key partner, providing access to a well-organized research network [65][66] Q&A Session Summary Question: What is the company's strategy for addressing the unmet needs in cystic fibrosis? - The company aims to improve CFTR function in patients who are currently on standard care but do not achieve normal function, focusing on the NBD1 stabilization approach [16][19] Question: How does the company plan to differentiate its products in the market? - The company plans to leverage its unique dual combination strategy and the historical data from its assays to demonstrate improved efficacy compared to existing therapies [6][70] Question: What are the expected timelines for clinical data? - The company anticipates data from its phase 2a proof of concept study in mid-2026, which will be crucial for informing future development and capital raising decisions [5][29]

Summary of Sionna Therapeutics FY Conference Call Company Overview - **Company**: Sionna Therapeutics (NasdaqGM:SION) - **Date of Conference**: December 03, 2025 - **Key Speakers**: Mike, Elena, Charlotte, and Corey Casimov (Senior Biotech Analyst at Evercore) Key Accomplishments in 2025 - Successful completion of Phase 1 programs for two NBD1 stabilizers: SION-719 and SION-451 with positive data outcomes [2][4] - Initiation of Phase 2 proof of concept study for SION-719 in combination with Trikafta, aimed at demonstrating improved CFTR function [2][3] - Progression of SION-451 into a Phase 1 study with healthy volunteers, testing its combination with two other modulators [3][4] - Completion of IPO in February 2025, raising over $219 million, providing cash runway into 2028 [4][5] Unmet Needs in Cystic Fibrosis (CF) - Acknowledgment of Vertex's contributions to CF treatment but recognition of ongoing unmet needs, as only about one-third of patients achieve normal CFTR function with current therapies [6][7] - Focus on improving patient outcomes by lowering sweat chloride levels and enhancing overall CFTR function [6][8] Mechanism of Action and Clinical Strategy - Emphasis on the unique mechanism of NBD1 stabilizers, which may provide additional efficacy compared to existing treatments [12][15] - Data from preclinical studies indicating that NBD1 stabilizers can improve CFTR protein half-life to wild-type levels, supporting the rationale for the NBD1 anchored pipeline [13][15] Clinical Trials and Future Directions - Phase 2a precision proof of concept trial for SION-719 aims to show significant improvement in sweat chloride levels compared to Trikafta [22][24] - The dual combination approach is preferred, with expectations of better efficacy than current standard care [25][26] - Plans to evaluate safety and pharmacokinetics in the combination studies of SION-451 with other modulators [30] Assay Confidence and Predictive Power - High confidence in the CFHBE assay used to predict clinical outcomes, validated through comparisons with Vertex's compounds [31][32] - The assay is seen as a tool to support the development of new treatment options for CF patients [34] Strategic Options and Future Pathways - Potential to pursue multiple combinations if data supports it, with a focus on capital availability to fund both add-on and dual combination approaches [35][36] - Anticipation of significant developments in 2026 as data from ongoing studies becomes available [37] This summary encapsulates the key points discussed during the conference call, highlighting Sionna Therapeutics' progress, strategic focus, and future plans in the cystic fibrosis treatment landscape.

Core Insights - Sionna Therapeutics is focused on developing innovative treatments for cystic fibrosis (CF) by normalizing the function of the cystic fibrosis transmembrane conductance regulator (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 improving clinical outcomes for CF patients [2] Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company dedicated to revolutionizing CF treatment through novel medicines that stabilize CFTR's nucleotide binding domain 1 (NBD1) [2] - The company aims to deliver differentiated medicines that restore CFTR function to near-normal levels, potentially enhancing the quality of life for individuals with CF [2] - Sionna is also developing a portfolio of complementary CFTR modulators designed to work synergistically with its NBD1 stabilizers [2] Investor Engagement - Sionna will participate in upcoming investor events, including the Annual Evercore Healthcare Conference on December 3, 2025, and the Citi 2025 Global Healthcare Conference on December 4, 2025 [4] - Live webcasts of these presentations will be available on Sionna's Investor Relations website, with replays accessible after the events [1][3]

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.