Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07108153 | Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of SION-719 When Added to Trikafta | PHASE2 | ACTIVE NOT_RECRUITING | 16 | — | — | Nov 3, 2025 | Jul 1, 2026 | May 6, 2026 | 13 | United States, Australia |
Adverse events will be assessed by Common Terminology Criteria for Adverse Events (CTCAE), v. 5.0
| Arm | Type | Description |
|---|---|---|
| Crossover Treatment Sequence 1: SION-719 followed by placebo-to-match SION-719 | EXPERIMENTAL | Participants receive SION-719 for one treatment period, and placebo to match SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study. |
| Crossover Treatment Sequence 2: Placebo-to-match SION-719 followed by SION-719 | EXPERIMENTAL | Participants receive placebo to match SION-719 for one treatment period and SION-719 for the other treatment period. All participants will remain on their physician-prescribed Trikafta as background therapy throughout the study. |
| Name | Type | Description |
|---|---|---|
| SION-719 | DRUG | All participants receive SION-719, as specified by their treatment sequence assignment |
| Placebo-to-match SION-719 | DRUG | All participants receive placebo to match SION-719, as specified by their treatment sequence assignment |
Inclusion Criteria: * Diagnosis of CF with F508del homozygous genotype based on documented CFTR genotype laboratory report. * Adherent to physician-prescribed Trikafta, as assessed by the Investigator, for at least 3 months prior to the Screening visit, taken at the recommended dose without modific...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 9 | PHASE3 | VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, VNZ/TEZ/D-IVA, VX-522 mRNA therapy |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719, SION-451, SION-2222, SION-109 |
| BiomX Ltd | PHGE | 1 | PHASE2 | BX004 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Illumina, Inc. | ILMN | 1 | — | Undisclosed |