Krystal Biotech (NasdaqGS:KRYS) Update / Briefing January 08, 2026 04:30 PM ET Company ParticipantsKrish Krishnan - CEODavid Sweet - Director of Clinical DevelopmentJorge Lascano - Professor of MedicineSuma Krishnan - President of Research and DevelopmentTrevor Parry - VP of Product DevelopmentConference Call ParticipantsGautam Chukka - Research AnalystJoe Pantginis - Director of Research and Senior Healthcare AnalystRitu Baral - Managing Director and Senior Biotechnology AnalystRoger Song - Senior AnalystA ...

Hardman Johnston Global Advisors, an investment management company, released its “Hardman Johnston Global Equity Strategy” investor letter for the third quarter of 2025. A copy of the letter can be downloaded here. The portfolio underperformed in the quarter due to stock selection. The composite returned 5.02%, net of fees, compared to 7.62% for the MSCI AC World Net Index. In addition, please check the fund’s top five holdings to know its best picks in 2025. In its third-quarter 2025 investor letter, Ha ...

PresentationWith that, I will hand the call over to Julian Pei, Head of Investor Relations, who will make introductory comments.Hello, ladies and gentlemen. Thank you for standing by, and welcome to our investor webcast today. As a reminder, today's call is being recorded.Julian Pei Good morning, and thank you for joining our webcast to discuss interim Phase I data from our AEROW trial in cystic fibrosis. Before we begin, I encourage everyone to visit our Investor Relations website, where you can find a pre ...

4D Molecular Therapeutics (NasdaqGS:FDMT) Update / Briefing December 17, 2025 08:00 AM ET Company ParticipantsJulian Pei - Head of Investor RelationsDaniel Giraldo - VP of Equity ResearchFelix Ratjen - Paediatric RespirologistJonathan Miller - Managing Director of Biotech and Pharma Equity ResearchJennifer Taylor Cousar - Professor of Internal Medicine and PediatricsGena Wang - Managing Director of Biotech Equity ResearchRyan McElroy - Equity Research AssociateDavid Kirn - Founder and CEOConference Call Par ...

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]

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 Viewpoint - Sionna Therapeutics is focused on transforming the treatment of cystic fibrosis (CF) by developing innovative medicines that normalize the function of the CFTR protein [2]. Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company dedicated to revolutionizing CF treatment through novel medicines aimed at restoring CFTR function [2]. - The company aims to deliver differentiated therapies that stabilize the nucleotide binding domain 1 (NBD1) of the CFTR protein, which is believed to be crucial for improving clinical outcomes and quality of life for CF patients [2]. - Sionna is advancing a pipeline of small molecules designed to correct defects caused by the F508del genetic mutation in NBD1 and is also developing complementary CFTR modulators to enhance CFTR function [2]. Investor Engagement - Sionna will participate in investor events, including the Cantor Global Healthcare Conference on September 3, 2025, and the Morgan Stanley 23rd Annual Global Healthcare Conference on September 8, 2025 [4]. - Live webcasts of these presentations will be available on the company's Investor Relations website, with replays accessible after the events [1][3].

Core Insights - Sionna Therapeutics is focused on developing novel medicines to normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, aiming to revolutionize the treatment paradigm for cystic fibrosis (CF) [1][4] Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company dedicated to creating therapies that restore CFTR function, particularly targeting the nucleotide-binding domain 1 (NBD1) [4] - The company has a pipeline of small molecules designed to correct defects caused by the F508del genetic mutation, which is prevalent in CF patients [4] Research and Development - Sionna has developed NBD1 stabilizers, SION-719 and SION-451, which have shown the ability to increase the stability of isolated ΔF508-NBD1 by 16°C, surpassing wild-type NBD1 levels [5] - Preclinical data indicate that SION-719 and SION-451 can correct F508del-CFTR maturation to wild-type levels when combined with other modulators, SION-2222 and SION-109 [5] - The company plans to advance SION-719 into a Phase 2a proof-of-concept trial and SION-451 into a Phase 1 trial, both expected to start in the second half of 2025 [3] Conference Presentation - Sionna presented preclinical data at the 48th European Cystic Fibrosis Conference, showcasing the effectiveness of dual combinations of NBD1 stabilizers with proprietary modulators [1][3]

Sionna Therapeutics Overview - Sionna's approach to stabilizing NBD1 has the potential to revolutionize CF treatment, addressing the unmet need for >66% of patients who lack normal CFTR function[7] - The CFTR modulator market is currently >$11 billion and is projected to reach $15 billion by 2029[7] - Sionna has a robust clinical-stage pipeline of NBD1 stabilizers and complementary modulators, offering multiple paths to transform CF patient care[7] Phase 1 Clinical Trial Results - Both NBD1 stabilizers, SION-719 and SION-451, exceeded desired PK targets and were generally well-tolerated in Phase 1 trials[13] - SION-719 is advancing to a Phase 2a proof-of-concept trial as an add-on to standard of care (SOC), based on its high potency at low doses[13] - SION-451 is advancing as an anchor in proprietary dual combinations, based on higher exposure achieved[13] Clinical Development Strategy - A Phase 2a proof-of-concept trial for SION-719 in CF patients is planned for the second half of 2025[14] - A Phase 1 healthy volunteer dual combination trial for SION-451 is also planned for the second half of 2025[14] - Sionna's cash runway extends into 2028, supporting the execution of its clinical development plans[14, 61] Market Opportunity - Approximately 106,000 patients worldwide have CF, with ~90% having at least one F508del-CFTR mutation[55] - >66% of patients on SOC do not have normal CFTR function, highlighting the significant unmet need[55, 59]

Company Overview - Sionna Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel medicines to normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein [3] - The company's mission is to revolutionize the treatment paradigm for cystic fibrosis (CF) by delivering differentiated medicines that restore CFTR function as close to normal as possible [3] - Sionna is advancing a pipeline of small molecules engineered to correct defects caused by the F508del genetic mutation, which is located in the nucleotide-binding domain 1 (NBD1) of the CFTR protein [3] Upcoming Events - Management will participate in a fireside chat at the Goldman Sachs 46th Annual Healthcare Conference in Miami, Florida on June 11, 2025, at 8:00 a.m. ET [1] - A live webcast of the presentation will be available on the "Events" page within the Investors section of Sionna's website, with a replay accessible after the event [2] Investor Relations - Sionna intends to use its Investor Relations website to disclose material nonpublic information and comply with disclosure obligations under Regulation FD [4] - Investors are encouraged to monitor the company's Investor Relations website, press releases, SEC filings, public conference calls, presentations, and webcasts for updates [4]