Financial Data and Key Metrics Changes - The company raised $219 million from its IPO in February 2025, providing significant financial flexibility and a cash runway extending into 2028 [54][55] - The market for cystic fibrosis treatments is currently valued at $11 billion and is projected to grow to $15 billion in the near term [53] Business Line Data and Key Metrics Changes - The company has concluded Phase I trials for both CYON719 and CYON451R2, reporting positive data indicating both compounds were well tolerated and exceeded pharmacokinetic (PK) targets [8][9] - CYON719 will advance into Phase IIa as an add-on to standard care, while CYON451 will be used in a dual combination study [11][12] Market Data and Key Metrics Changes - The company aims to drive incremental clinical benefits above the standard of care, specifically targeting a 10 millimole improvement in sweat chloride levels compared to Trikafta [27][40] - The company is focused on stabilizing the NBD1 region of the CFTR protein, which is critical for correcting cystic fibrosis [18][22] Company Strategy and Development Direction - The company's mission is to transform the standard of care in cystic fibrosis through differentiated NBD1 stabilizers [5][6] - The strategy includes a dual combination approach with NPD-one as the anchor, aiming to achieve fully normal CFTR function for more patients [7][21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of NPD-one to deliver clinically meaningful benefits, emphasizing the need for further data to guide future decisions [44][52] - The company is optimistic about the upcoming Phase IIa data and its implications for both the add-on and dual combination strategies [42][45] Other Important Information - The company has prioritized two complementary mechanisms, Cyon2222 and Cyon109, for future studies based on their favorable profiles [46][48] - The company plans to conduct a drug-drug interaction (DDI) study to ensure that the addition of CYON719 does not affect the components of Trikafta [34][36] Q&A Session Summary Question: Can you provide an overview of Ciona's story and strategy? - Ciona was formed as a spinout from Sanofi in late 2019, with a mission to transform cystic fibrosis care through innovative therapies [3][4] Question: What are the key milestones for the company in the next 12-18 months? - Key milestones include the initiation of Phase IIa for CYON719 and a healthy volunteer study for CYON451, both expected to yield data by mid-2026 [14][16] Question: How does the company plan to ensure the efficacy of its treatments? - The company aims for a 10 millimole improvement in sweat chloride levels, which has been shown to correlate with meaningful improvements in FEV1 [27][40] Question: What is the rationale behind the dual combination strategy? - The dual combination strategy is expected to provide a differentiated treatment option that could lead to fully normal CFTR function, addressing the limitations of current therapies [21][52] Question: How does the company view the commercial opportunity in the cystic fibrosis market? - The company believes that its dual combination approach could become the new standard of care, with significant commercial potential given the size of the market [52][53]

Sionna Therapeutics (SION) Update / Briefing June 04, 2025 08:00 AM ET Speaker0 Good morning and welcome to the Ciena Therapeutics Conference Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be an opportunity to ask questions. Please note that this event is being recorded. I will now turn the call over to Juliet Lapdorf, Senior Director of Investor Relations. You may begin. Speaker1 Thank you. Good morning, and welcome to Ceona's conference call to ...

Sionna Therapeutics (SION) 2025 Conference May 07, 2025 12:30 PM ET Speaker0 This is life sciences. Welcome to day one of our conference for 2025. We're pleased to have Ciona Therapeutics and CEO Mike Clunan to run us through the story here. I think this is, you know, one of the more interesting stories, one of the recent IPOs in the sector, which we haven't seen many of. So I think a lot of high interest. I think a lot of interesting science and going after one of one of the more compelling opportunities t ...