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Sionna Therapeutics to Present Data at the 2025 North American Cystic Fibrosis Conference
Globenewswire· 2025-10-01 20:05
WALTHAM, Mass., Oct. 01, 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 data will be presented at the 2025 North American Cystic Fibrosis Conference (NACFC) being held in Seattle, Washington on October 22-25, ...
1 Reason Wall Street Is Obsessed With Vertex Pharmaceuticals
Yahoo Finance· 2025-09-15 11:30
Key Points Vertex Pharmaceuticals has a lot of buy and hold ratings, but there aren't any sell ratings from Wall Street analysts. Around 75,000 people with cystic fibrosis rely on Vertex Pharmaceuticals to keep their lungs functioning. There are no other drugmakers marketing treatments that correct the misformed proteins responsible for cystic fibrosis. 10 stocks we like better than Vertex Pharmaceuticals › Shares of Vertex Pharmaceuticals (NASDAQ: VRTX) have risen more than 2,000% over the past ...
Sionna Therapeutics Announces First Subjects Dosed in Phase 1 Trial Evaluating NBD1 Stabilizer, SION-451, in Proprietary Dual Combinations in Development for the Treatment of Cystic Fibrosis
Globenewswire· 2025-08-25 11:00
Core Viewpoint - Sionna Therapeutics has initiated dosing for SION-451 in combination with SION-2222 and SION-109 in a Phase 1 trial, aiming to transform cystic fibrosis treatment with novel CFTR modulators, with topline data expected in mid-2026 [1][2][3] Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company focused on developing medicines that normalize CFTR protein function, specifically targeting the NBD1 domain to improve clinical outcomes for cystic fibrosis patients [5] - The company aims to deliver differentiated therapies that restore CFTR function to near-normal levels, leveraging over a decade of research on NBD1 and developing small molecules to correct defects caused by the F508del mutation [5] Clinical Trial Details - The Phase 1 trial is randomized, double-blind, and placebo-controlled, assessing the safety, tolerability, and pharmacokinetics of SION-451 in dual combinations with SION-2222 and SION-109 [2] - Topline data from this trial is anticipated in mid-2026, which will guide the selection of a dual combination for a planned Phase 2b trial in cystic fibrosis patients [2] Scientific Rationale - Positive data from previous Phase 1 trials and preclinical studies support the advancement of the dual combination trial, with SION-451 showing good tolerability and exceeding pharmacokinetic concentration targets [4] - The NBD1 domain is critical for CFTR protein stability and function, and no approved therapies currently stabilize this domain directly, highlighting the innovative approach of Sionna's research [3][4]
Sionna Therapeutics Announces Positive Phase 1 Data for NBD1 Stabilizers SION-719 and SION-451 and Advances Both Programs in Clinical Development for Cystic Fibrosis
GlobeNewswire News Room· 2025-06-04 11:00
Core Insights - Sionna Therapeutics announced positive Phase 1 clinical trial results for SION-719 and SION-451, which are first-in-class NBD1 stabilizers aimed at treating cystic fibrosis (CF) [2][3] - Both compounds were well tolerated in trials, achieving pharmacokinetic targets that suggest potential clinical benefits for CF patients [4][5][6] - The company plans to advance SION-719 to a Phase 2a proof-of-concept trial and SION-451 to a Phase 1 dual combination trial, with both trials expected to start in the second half of 2025 and topline data anticipated in mid-2026 [8][9] Company Development Plans - Sionna plans to initiate a Phase 2a trial for SION-719 as an add-on to standard of care in CF patients, focusing on improving CFTR function and demonstrating its unique mechanism [8] - SION-451 will enter a Phase 1 trial evaluating its combination with other CFTR modulators, aiming to inform future Phase 2b trials [9] Clinical Trial Details - The Phase 1 trials were randomized, double-blind, and placebo-controlled, involving 100 subjects for SION-719 and 110 subjects for SION-451 [4] - No serious adverse events were reported, and most treatment-emergent adverse events were mild to moderate [5] Market Context - The development of NBD1 stabilizers addresses a significant unmet need in CF treatment, as many patients on existing therapies do not achieve normal CFTR function [7] - Sionna's approach aims to restore CFTR function, potentially improving clinical outcomes and quality of life for CF patients [11]
Sionna Therapeutics to Present Preclinical Data During Oral Session at the 48th European Cystic Fibrosis Conference
Globenewswire· 2025-05-22 11:00
Core Insights - Sionna Therapeutics is focused on developing novel medicines to treat cystic fibrosis (CF) by normalizing the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein [1][3] - The company will present preclinical data on its NBD1 stabilizers, SION-451 and SION-719, in combination with CFTR modulators, galicaftor (SION-2222) and SION-109, at the European Cystic Fibrosis Conference [1][2] Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company aiming to revolutionize CF treatment through innovative medicines that stabilize CFTR's nucleotide-binding domain 1 (NBD1) [3] - The company is developing a pipeline of small molecules targeting the F508del genetic mutation and complementary CFTR modulators to enhance CFTR function [3] Presentation Details - The oral presentation titled "Stabilizers of CFTR NBD1 synergize with galicaftor (SION-2222) or SION-109 to enable full correction of ΔF508-CFTR" will take place on June 6, 2025 [2] - The presentation will be available on Sionna's website under the "Scientific Presentations" section [2]
Sionna Therapeutics Reports First Quarter 2025 Financial Results
Globenewswire· 2025-05-12 11:00
Phase 1 dosing completed for NBD1 stabilizers SION-719 & SION-451; both compounds continue to be generally well tolerated Phase 1 topline data anticipated this quarter On track to initiate Phase 2a proof-of-concept trial and at least one dual combination MAD trial in the second half of 2025; topline data for both anticipated in mid-2026 Business Highlights Completion of Upsized IPO: In February 2025, Sionna completed its upsized initial public offering (IPO), raising approximately $219 million gross proceed ...
VRTX Secures Nod for Expanded Use of CF Drug Kaftrio in Europe
ZACKS· 2025-04-08 11:55
Core Viewpoint - Vertex Pharmaceuticals has received an expanded label approval from the European Commission for its cystic fibrosis drug Kaftrio, allowing treatment for all patients aged two years and older with at least one non-class I mutation in the CFTR gene [1][2]. Company Overview - Vertex Pharmaceuticals holds a dominant position in the cystic fibrosis market, being the first company to develop a drug that addresses the underlying cause of the disease [6]. - The company markets multiple cystic fibrosis medications, including Kaftrio (Trikafta in the U.S.), Alyftrek, Symdeko/Symkevi, Orkambi, and Kalydeco [6][3]. Market Impact - The expanded label for Kaftrio will make nearly 4,000 additional cystic fibrosis patients eligible for treatment in the European region [2]. - Vertex's cystic fibrosis sales have shown significant growth, with Kaftrio contributing $10.24 billion in product sales in 2024, reflecting a year-over-year increase of approximately 15% [7]. Future Developments - Vertex is developing an mRNA therapeutic, VX-522, in collaboration with Moderna for approximately 5,000 cystic fibrosis patients who do not produce CFTR protein. The single ascending dose portion of the phase I/II clinical study is complete, with data from the ongoing multiple ascending dose portion expected in the first half of 2025 [8]. Stock Performance - Year to date, Vertex's stock has gained 18%, outperforming the industry, which has seen a 7% decline [4].