Ankündigung • Aug 11
Sionna Therapeutics Reports Topline Data from Two Development Programs in Cystic Fibrosis Sionna Therapeutics reported topline data from two nucleotide binding domain 1 (NBD1) stabilizer programs, the PreciSION CF Phase 2a proof-of-concept (POC) trial of SION-719 when added to Trikafta (elexacaftor/tezacaftor/ivacaftor) in CF participants, and the Phase 1 trial of SION-451-based proprietary dual combinations in healthy subjects. The PreciSION CF trial did not meet its key activity endpoint, with a -1.0 mmol/L mean placebo-adjusted sweat chloride change (p=0.7). Potential confounders were observed in the trial, including, for example, higher than anticipated levels of variability in individual sweat chloride levels and differences in Trikafta exposure levels between the SION-719 and placebo periods. The Company is evaluating the impact of these on the interpretation of the trial data. SION-719 was generally well tolerated when added to Trikafta for 14 days with most treatment emergent adverse events mild to moderate, no serious adverse events, and no meaningful trends in adverse events related to liver function tests. Based on these results, Sionna does not plan to advance SION-719 as an add-on to standard of care. The Company received the topline data on Friday, August 7, 2026, and is continuing to analyze the results of the PreciSION CF study. The Phase 1 trial of SION-451 in dual combinations with either SION-2222 or SION-109 in healthy subjects achieved the safety, tolerability, and pharmacokinetic (PK) objectives, including target exposures defined prior to the Phase 2a PreciSION CF results. Based on the totality of the data and target coverage observed, SION-451 + SION-2222 was identified as the preferred dual combination. All SION-451 twice-daily + SION-2222 once-daily doses were generally well tolerated. One participant discontinued due to rash. In the SION-451 twice-daily + SION-2222 twice-daily cohorts, most treatment emergent adverse events were mild to moderate, and no serious adverse events were reported. Two participants discontinued due to elevated liver function tests and flu-like symptoms. The SION-451 + SION-109 regimens were all generally well tolerated. Sionna is evaluating next steps for the SION-451 program. Sionna ended the Second Quarter with approximately $268.3 million in cash, cash equivalents, and marketable securities. The Company intends to take actions to preserve capital while evaluating next steps. The PreciSION CF Phase 2a trial (NCT07108153) was a randomized, double-blind, placebo-controlled, crossover trial that enrolled 15 adult participants with CF homozygous for F508del on a stable dose of physician-prescribed Trikafta. The primary objective was to evaluate the safety and tolerability of SION-719 when added to SOC. Secondary objectives were to assess the PK of SION-719 and the change in sweat chloride levels, an important measure of CFTR function, when SION-719 was added to SOC. The Phase 1 healthy volunteer trial (NCT07035990) evaluated the safety, tolerability, and PK of varying doses of SION-451 in dual combinations with SION-2222 (galicaftor), a transmembrane domain 1 (TMD1)-directed CFTR corrector, and with SION-109, an intracellular loop 4 (ICL4)-directed CFTR corrector. A total of 120 participants were dosed across both dual combinations. Live-News • Aug 11
Sionna Therapeutics Cystic Fibrosis Drug Misses Phase 2 Goal in Trikafta Combo Study Sionna Therapeutics reported that its lead cystic fibrosis candidate failed to deliver a significant additive effect in a Phase 2a trial when combined with Vertex’s Trikafta, using sweat chloride reduction as the main efficacy readout.
Management signaled this is a major setback for the current cystic fibrosis program and said Sionna will now shift its efforts toward other pipeline candidates, removing one potential future competitor to Vertex in this indication.
Sionna Therapeutics shares trade around $4.50 and are down about 91% over the past day, leaving the stock at a record low following the trial outcome.
This outcome leaves Sionna Therapeutics heavily reliant on earlier-stage assets, which introduces more development risk and timing uncertainty for any future revenue. Investors now need to focus on the depth and diversity of the remaining pipeline and the company’s ability to fund continued research after such a sharp move in the share price. Price Target Changed • Aug 10
Price target decreased by 19% to US$41.80 Down from US$51.60, the current price target is an average from 6 analysts. New target price is 829% above last closing price of US$4.50. Stock is down 78% over the past year. The company is forecast to post a net loss per share of US$2.52 next year compared to a net loss per share of US$1.88 last year. Recent Insider Transactions Derivative • Jul 08
Chief Legal Officer exercised options and sold US$384k worth of stock On the 6th of July, Jennifer Fitzpatrick exercised 10k options at a strike price of around US$6.11 and sold these shares for an average price of US$43.60 per share. This trade did not impact their existing holding. As of today, Jennifer currently holds no shares directly. Company insiders have collectively sold US$6.3m more than they bought, via options and on-market transactions in the last 12 months. Live-News • May 14
Sionna Therapeutics Eyes Key 2026 Trial Results With Strong Cash and Analyst Support Sionna Therapeutics completed enrollment in its Phase 2a PreciSION CF trial evaluating NBD1 stabilizer SION-719 as an add-on therapy for cystic fibrosis, with topline data from this study and a Phase 1 dual-combination trial with SION-451 expected in summer 2026.
The company reported a Q1 2026 net loss of $26.8 million, or $0.60 per share, and held cash and equivalents of $289.9 million, which management projects is sufficient to fund operations into 2028.
Director Peter Thompson and OrbiMed Advisors each sold roughly $13.3 million of Sionna stock under pre-arranged trading plans, while several research firms reiterated positive ratings and price targets up to $53 following the earnings update and trial progress.
The key swing factor for Sionna over the next two years is the paired readout from SION-719 and the SION-719 / SION-451 combination. These data could materially influence how the pipeline and funding runway are perceived.
Insider sales under trading plans do not automatically signal a change in company fundamentals. However, they are worth tracking alongside clinical milestones and cash usage as you assess risk and potential dilution over time. Ankündigung • Apr 30
Sionna Therapeutics Inc Completes Enrollment in Precision Cf Phase 2A Trial Evaluating Nbd1 Stabilizer Sion-719 Added to Standard of Care in Participants with Cystic Fibrosis Sionna Therapeutics, Inc. announced the completion of enrollment in the PreciSION CF Phase 2a proof-of-concept (POC) trial. This trial is evaluating SION-719, a first-in-class nucleotide binding domain 1 (NBD1) stabilizer, when added to Trikafta (elexacaftor/tezacaftor/ivacaftor), the current standard of care (SOC) in CF. Sionna anticipates data from the PreciSION CF trial in the summer of 2026. The PreciSION CF Phase 2a trial (NCT07108153) is a randomized, double-blind, placebo-controlled, crossover POC study that enrolled adult CF patients homozygous for F508del on a stable dose of physician-prescribed Trikafta. The objectives of the trial are to evaluate the safety, tolerability, and pharmacokinetics of SION-719 when administered with SOC and to assess change in sweat chloride levels, an important measure of CFTR function. The PreciSION CF trial is being conducted at multiple sites, including sites in the CF Foundation-supported Therapeutics Development Network (TDN), the largest CF clinical trials network in the world. Ankündigung • Apr 29
Sionna Therapeutics, Inc., Annual General Meeting, Jun 17, 2026 Sionna Therapeutics, Inc., Annual General Meeting, Jun 17, 2026. Ankündigung • Oct 25
Sionna Therapeutics Announces Presentation of SION-719 and SION-451 Phase 1 Data and Poster of New Preclinical Data on the Impact of NBD1 Stabilizer on F508del-CFTR Half-Life at the 2025 North American Cystic Fibrosis Conference Sionna Therapeutics, Inc. announced data presented at the 2025 North American Cystic Fibrosis Conference (NACFC) being held in Seattle, Washingt on October 22-25. Jason H. Maley, M.D., MS, Senior Director of Clinical Development at Sionna, presented data from the two Phase 1 clinical trials of SION-719 and SION-451, the company's first-in-class nucleotide-binding domain 1 (NBD1) stabilizers. The randomized, double-blind, placebo-controlled Phase 1 trials enrolled over 200 healthy volunteers and evaluated each compound's safety, tolerability, and pharmacokinetics (PK) across single and multiple ascending dose cohorts. As previously disclosed, Sionna's two Phase 1 trials demonstrated that both SION-719 andSION-451 were generally well tolerated and exceeded target exposure levels. Based on these Phase 1 data and its cystic fibrosis human bronchial epithelial (CFHBE) model, Sionna believes that its NBD1 stabilizers have the potential to deliver clinically meaningful benefit when SION-719 is added to the standard of care (SOC) or when SION-451 is used in proprietary dual combinations with one of Sionna's complementary modulators. The Phase 1 data also supported the use of a tablet formulation in future trials and indicated that both compounds can be dosed in a fed or fast state. In a poster presented by Greg Hurlbut, Ph.D., Co-Founder and Senior Vice President of Discovery Research at Sionna, new preclinical data from metabolic pulse-chase labeling studies show the impact of Sionna's NBD1 stabilizers on the half-life of F508del-CFTR protein, in the presence and absence of Sionna's complementary CFTR modulators. In these studies, NBD1 stabilizers SION-719 and NBD1 stabilizers Sionna-719 and SION -451 increased the half-life of mature F508del-CFTR proteins to levels seen with wild-type CFTR. This effect was apparent even when NBD1 stabilizers were used as single agents. The most common CFTR mutation that causes CF is F508del, which results in the instability of CFTR's NBD1 domain and impaired CFTR folding, trafficking, half-life, and function," said Dr. Hurlbut. The minimal amount of F508del-CF TR protein that does reach the apical cellular membrane exhibits a dramatically increased rate ofdegradation and decreased half-life relative to wild-type CFTR. The company have previously previously announced that the NBD1 stabilizers are currently being evaluated in the Phase 1 data in the clinical trial. Ankündigung • Sep 09
Sionna Therapeutics, Inc Appoints Caroline Stark Beer as Chief Business Officer Sionna Therapeutics, Inc. announced the appointment of Caroline Stark Beer, MBA, as its Chief Business Officer (CBO). Caroline brings over 20 years of experience in the life science industry to her role with Sionna. She was most recently CBO at Jnana Therapeutics until Jnanas sale to Otsuka Pharmaceutical. Caroline led the team responsible for corporate and business development, alliance management, legal contracts, new product planning, and communications. Before Jnana, she spent over a decade at Alnylam Pharmaceuticals; in her final role there, she served as VP of Business Development. During this time, she led the formation of a variety of high-impact partnerships including a 5-year collaboration with Regeneron Pharmaceuticals to discover and develop new RNA interference (RNAi) therapeutics. Prior to Alnylam, Caroline served in business development at Amicus Therapeutics and started her career as a strategy consultant at Bain &Company. Caroline received her MBA from the MIT Sloan School of Management and a Bachelor of Economics from Duke University. Ankündigung • Aug 25
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 Sionna Therapeutics, Inc. announced that the first subjects have been dosed in a Phase 1 trial evaluating SION-451, a first-in-class nucleotide binding domain 1 (NBD1) stabilizer, in proprietary dual combinations with SION-2222 (galicaftor), a transmembrane domain 1 (TMD1)-directed CFTR corrector, and with SION-109, an intrracellular loop 4 (ICL4)-directed CFTR corrector. This Phase 1 trial is a randomized, double-blind, placebo-controlled trial in healthy volunteers. It is designed to assess the safety, tolerability, and pharmacokinetics (PK) of varying doses of dual combinations of SION-2222 and SION-109 with SION-109. Topline data are anticipated in mid-2026, and will inform selection of a dual combination for a planned Phase 2b trial in people living with CF. CF is a progressive and life-threatening genetic disease that affects more than 100,000 people globally. While advances in the discovery and development of modulators have significantly improved the lives of people living with CF, at least two-thirds of patients on the current standard of care do not have normal CFTR function as measured by levels of sweat chloride. The NBD1 domain of the CFTR protein plays a critical role in the folding, stability and trafficking of CFTR to a cell's surface, but no approved CF therapies directly stabilize NBD1. Positive data from Phase 1 clinical trials and preclinical data support the mechanistic rationale and advancement of this dual combination trial. In its recent Phase 1 trial, SION-451 was generally well tolerated and exceeded PK concentration targets that Sionna believes, based on its preclinical CF human bronchialhelial (CFHBE) model, indicate the potential to provide clinically meaningful benefit, including the potential for wild-type levels of CFTR function, in proprietary dual combinations with complementary modulators. Ankündigung • Jun 18
Sling Therapeutics, Inc. Appoints Ken Lock as Chief Commercial Officer Sling Therapeutics, Inc. announced the appointment of Ken Lock as Chief Commercial Officer. Lock brings over 20 years of commercial leadership experience as Sling prepares for a confirmatory Phase 3 study of linsitinib. Most recently, Lock served as Chief Commercial Officer at ACELYRIN, where he led the commercial, medical affairs, and alliance management functions in the TED market. Lock also served as Chief Commercial Officer at Arcutis Biotherapeutics, where he built and led a team of over 130 people and was responsible for the launch and brand development of ZORYVE®. Prior to that, he held sales and marketing roles of increasing responsibility at Gilead Sciences, Amgen, and Wyeth (now Pfizer). Lock holds an M.B.A. from the Cornell Johnson Graduate School of Management and a dual B.S. and B.A. in Biochemistry/Cell Biology and Psychology from the University of California, San Diego. Ankündigung • Jun 07
Sionna Therapeutics, Inc. Announces Presentation of Preclinical Data That Demonstrate Proprietary Dual Combination Therapies Enable Full CFTR Correction in CFHBE Model Sionna Therapeutics, Inc. announced the presentation of preclinical data that demonstrate that the company's nucleotide-binding domain 1 (NBD1) stabilizers, in dual combinations with proprietary complementary modulators, enable full CFTR correction in CF models. The data are featured in an oral presentation and poster session at the European Cystic Fibrosis Society (ECFS) 48thEuropean Cystic Fibrosis Conference being held in Milan, Italy. Preclinical data suggest these compounds, when used in combination, have the potential to dramatically improve clinical outcomes and quality of life for people with CF." Sionna has completed Phase 1 clinical trials evaluating two potent first-in-class small molecule NBD1 stabilizers, SION-719 and SION-451. The company is also developing modulators with mechanisms of action that are complementary to NBD1, including SION-2222 (galicaftor), a transmembrane domain 1 (TMD1)-directed CFTR corrector, and SION-109, an intracellular loop 4 (ICL4)-directed CFTR corrector. In the CFHBE model, SION-719 & SION-451 dual combinations also show potential for clinically meaningful improvement including to wild-type levels at concentrations below Emax. Earlier this week, Sionna announced its plans to advance SION-719 andSION-451 to the next stage of clinical development. The company plans to evaluate SION-719 in a Phase 2a proof-of-concept (POC) trial in CF patients as an add-on to standard of care (SOC), and SION-451 in a Phase 1 healthy volunteer trial evaluating SION-451 in two dual combinations with SION-2222 and with SION-109. Both trials are expected to initiate in the second half of 2025, with data anticipated in mid-2026. Ankündigung • Jun 04
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 Sionna Therapeutics, Inc. announced positive data from its Phase 1 clinical trials of SION-719 and SION-451, the company's first-in-class nucleotide-binding domain 1 (NBD1) stabilizers, in healthy volunteers. Sionna plans to advance both NBD1 stabilizers to the next stage of clinical development -- SION-719 to a Phase 2a proof-of-concept (POC) trial in CF patients as an add-on to SOC, and SION-451 to a Phase 1 proprietary dual combination trial in healthy volunteers. The Phase 1 randomized, double-blind, placebo-controlled clinical trials evaluated the safety, tolerability, and PK profiles of single ascending doses (SAD) and multiple ascending doses (MAD) of SION-719, and SION-451 in healthy volunteers. SionNA is progressing SION-719 into a Phase 2a POC trial evaluating the compound as an add- on to SOC and SION-451 to a Phase 1 proprietary dual combination trial in healthy volunteers. Positive Phase 1 Results for SION-719 and SION-451: The Phase 1 randomized, double-blind, placebo-controlled clinical trials evaluated the safety, tolerability, and PK profiles of single ascending doses (SAD) and multiple ascending doses (MAD) of SION-719 and SION-451 in healthy volunteers. The effect of food on the PK and the bioequivalence of a tablet formulation was also evaluated in Part C of each trial. 100 subjects were dosed in the SION-719 trial and 110 subjects were dosed in the SION-451 trial. Both compounds were generally well tolerated in the Phase 1 trials. There were no serious adverse events, treatment emergent adverse events (TEAEs) that led to discontinuation of drug, or dose-limiting TEAEs observed. Most TEAEs were mild to moderate (Grade 1 or Grade 2). There was one Grade 1 TEAE related to liver function tests (LFTs) in a SION-451 treated subject who tested positive for influenza; no TEAEs related to LFTs were observed in the other cohorts of SION-451. There were no TEAEs related to LFTs in SION-719 treated subjects. The data from Part C of each trial support the use of a tablet formulation in future studies and indicate that both compounds can be dosed in a fed or fasted state. SION-719 and SION-451 each achieved desired target PK concentrations at multiple dose levels in a twice daily regimen. Both stabilizers met exposure thresholds that, based on Sionna’s preclinical CFHBE model, have the potential to provide clinically meaningful benefit if administered as an add-on to SOC or in a proprietary dual combination with complementary modulators. Next Phase of Clinical Development for NBD1 Stabilizers: SION-719 to Advance to Phase 2a Proof-of-concept in CF Patients as an Add-on to Standard of Care: Sionna is progressing SION-719 into a Phase 2a POC trial evaluating the compound as an add-on to SOC in CF patients. The trial objectives will be to show an improvement in CFTR function as defined by sweat chloride reduction and to demonstrate that NBD1 is mechanistically unique from, and synergistic with, the components of SOC. Sionna is on track to initiate this trial in the second half of 2025 with topline data anticipated in mid-2026. The U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for SION-719, and the first subject has been dosed in a midazolam drug-drug interaction (DDI) study to confirm SION-719 can be dosed in combination with the SOC according to its label. This study will be completed prior to initiation of the Phase 2a trial. SION-451 to Advance to Phase 1 Healthy Volunteer Dual Combination Trial with Complementary Modulators: Sionna plans to initiate a Phase 1 healthy volunteer dual combination trial evaluating SION-451 in combination with SION-2222 (galicaftor), a transmembrane domain 1 (TMD1)-directed CFTR corrector, and with SION-109, an intracellular loop 4 (ICL4)-directed CFTR corrector. The trial will assess the safety, tolerability, and PK of varying doses of the dual combinations, and will inform selection of a dual combination for a Phase 2b trial in CF patients. Sionna is on track to initiate the healthy volunteer trial in the second half of 2025 with topline data expected in mid-2026. Ankündigung • May 22
Sionna Therapeutics to Present Preclinical Data During Oral Session At the 48Th European Cystic Fibrosis Conference Sionna Therapeutics, Inc. announced that preclinical data assessing combinations of Sionna's nucleotide-binding domain 1 (NBD1) stabilizers, SION-451 and SION-719, with complementary Sionna CFTR modulators, galicaftor (SION-2222) and SION-109, will be featured in an oral presentation at the European Cystic Fibrosis Society's (ECFS) 48thEuropean Cystic Fibrosis Conference, being held June 4-7, 2025 in Milan, Italy. Ankündigung • Apr 30
Sionna Therapeutics, Inc., Annual General Meeting, Jun 12, 2025 Sionna Therapeutics, Inc., Annual General Meeting, Jun 12, 2025. Ankündigung • Feb 07
Sionna Therapeutics, Inc. has completed an IPO in the amount of $190.588194 million. Sionna Therapeutics, Inc. has completed an IPO in the amount of $190.588194 million.
Security Name: Common Stock
Security Type: Common Stock
Securities Offered: 10,588,233
Price\Range: $18
Transaction Features: Sponsor Backed Offering