Data featured in oral presentation and poster session at 48th European Cystic Fibrosis Conference WALTHAM, Mass., June 06, 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 the presentation of preclinical data that de ...

Sionna Therapeutics Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or other events to be materially different from any future results, performance or other events expressed or implied by the forward-looking statements. Given these uncertainties, you should not place undue reliance on forward-looking statements. Our actual future results, performance or other events may be materially different from what we expect. Exc ...

WALTHAM, Mass., May 22, 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 preclinical data assessing combinations of Sionna’s nucleotide-binding domain 1 (NBD1) stabilizers, SION-451 and SION-719, with complement ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q _________________________ (Mark One) x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____ to _____ Commission file number 001-42504 _________________________ SIONNA THERAPEUTICS, INC. (Exact name of ...

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 Exhibit 99.1 Sionna Therapeutics Reports First Quarter 2025 Financial Results 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 Strong cash position following completed upsized IPO, with approximately $354.7 million in cash ...

纪要涉及的行业或者公司 - 行业：生命科学、囊性纤维化治疗行业 - 公司：Sionna Therapeutics（SION）、Vertex、Sanofi、Genzyme、AbbVie 纪要提到的核心观点和论据 公司概况与背景 - 公司成立于2019年，从Sanofi分拆出来，相关科学研究可追溯到Genzyme，已对相关项目研究超十年 [5] - 2月完成首次公开募股，筹集2.19亿美元资金，资金可支撑到2028年，为公司提供财务灵活性 [20] 行业未满足需求 - 尽管Vertex在囊性纤维化治疗上取得成功，但未满足需求仍然很高，使用标准疗法TRIKAFTA的患者中，只有三分之一达到正常CFTR功能，三分之二未达到，这为公司提供机会 [6] - 囊性纤维化治疗市场规模达110亿美元且在增长，公司若能推动更多患者达到正常CFTR功能，将有巨大商业机会 [7] NPD - 1靶点的重要性 - NPD - 1虽不是新靶点，但多年来被认为不可成药，因其周围结合口袋浅；公司认为它是实现更多CFTR校正、推动更多患者达到正常CFTR功能的关键，且已获批的调节剂均未直接稳定NPD - 1，具有独特性和差异化 [7][8] - 约90%囊性纤维化患者的主要基因突变F508del位于CFTR蛋白的Nbd1区域，该突变使Nbd1在体温下不可逆展开，破坏蛋白折叠、转运和功能；Trikafta组合疗法是在NPD - 1周围进行校正，未直接稳定NPD - 1；若能稳定NPD - 1，单药效果几乎相当于Trikafta三联组合，若再校正蛋白其他部分，有潜力使CFTR功能完全正常化 [11][12][13] 研发管线与进展 - 管线有两个维度，Nbd1方面有两个化合物处于一期，组合选项方面有四个选项 [14] - 719和451两个NPD - 1项目一期接近完成，预计本季度公布顶线数据，两个化合物均达到一期设定的目标产品概况，即双药组合有潜力在标准疗法基础上增加临床显著益处 [14][15] - 优先选择AbbVie的Galacaftor和公司自己的ICL4校正剂109与NVD1组合，它们是优秀的组合资产 [16] 战略与市场策略 - 因NPD - 1的差异化，公司有多种进入市场的方式，最低产品概况是在标准疗法基础上添加NPD - 1；目标产品概况是Nbd1与另一种校正剂的双药组合，能在标准疗法基础上带来显著临床益处 [17][18] 临床前数据与一期数据 - CFHB测定法是囊性纤维化体外测定的金标准，具有高度临床预测性，公司用其筛选化合物和确定一期临床的暴露目标 [8][9] - 临床前数据显示，719和451单药在测定中效果几乎相当于Trikafta三联组合，与其他校正剂的双药组合有潜力使CFTR功能达到野生型水平 [23][24][26] - 一期数据显示，719和451耐受性良好，无严重不良事件，大多为轻度至中度，无导致停药的不良事件，肝功能测试除一例患流感受试者出现一级不良事件外无其他问题；支持每日两次给药 [27] - PK数据显示，719和451的所有测试剂量均高于作为附加疗法的目标，部分剂量高于双药组合目标，有多种剂量可推进 [32][35] 临床开发计划 - 2025年上半年完成一期，本季度公布顶线数据；下一步在囊性纤维化患者中进行2a期概念验证研究，将NPD - 1添加到标准疗法Trikafta上，进行为期两周的双臂研究，比较添加NPD - 1和安慰剂的汗液氯化物改善情况，预计2026年年中获得数据 [40][41][44] - 同时进行健康志愿者组合研究，研究Nbd1与2222、109的组合耐受性、PK和暴露情况，以确定最佳组合推进到2b期及以后，预计2026年年中获得数据 [45] 其他重要但是可能被忽略的内容 - 公司与AbbVie交易获得三个互补机制，Galacaftor有二期疗效和耐受性数据，与Navacaptor组合在FEV1和汗液氯化物方面有有意义的变化 [10][36][37] - 109刚完成一期，耐受性和PK情况令人鼓舞，给药方案为每日两次，与NPD - 1组合能达到设定的PK指标 [39] - 汗液氯化物与FEV1有强相关性，CFHB数据显示氯化物转运与FEV1相关性约为0.96，当汗液氯化物改善10毫摩尔或更高时，相关性更强 [53] - 公司的CFHB测定法与Vertex类似，关键是在体外测定中加入20%体积的人血清，以更接近体内环境 [55]

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 ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K __________________________ (Mark One) x ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2024 OR o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM____ to Commission file number 001-42504 _________________________________________________ SIONNA THERAPEUTICS, INC. (E ...

Exhibit 99.1 Sionna Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results Phase 1 MAD dosing completed for SION-451 and final MAD cohort of SION-719 planned; Interim data in healthy volunteers show potential to provide clinically meaningful benefit to CF patients. Topline data anticipated in first half of 2025 On track to initiate NBD1 Phase 2a proof-of-concept trial in combination with SOC in CF patients and combination MAD trials of an NBD1 stabilizer with a complementary modulator in h ...