Approval Probability
TA Base Rate
Adjusted LOA
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SION-719 · 1 trial · 1 indication
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 | 8 | PHASE3 | VX-121/TEZ/D-IVA |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719 |
| BiomX Inc. | 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 |
SION-719 is an investigational small molecule being developed by Sionna Therapeutics for the treatment of cystic fibrosis. It is designed to be used as an add-on therapy in people with CF and is currently in Phase 2 clinical development. It has not been approved by the FDA.
SION-719 is being studied for cystic fibrosis, a respiratory disease. The Phase 2 proof-of-concept trial evaluated SION-719 when added to Trikafta in adults with CF. It is an investigational therapy and is not approved for any indication.
SION-719 targets NBD1, the nucleotide-binding domain 1 of the cystic fibrosis transmembrane conductance regulator protein. NBD1 is a protein target, and SION-719 is a small molecule designed to act on it. This mechanism is being explored as an add-on approach in cystic fibrosis.
SION-719 is being developed by Sionna Therapeutics, Inc., which trades on the Nasdaq under the ticker SION. The company is advancing the compound for cystic fibrosis.
SION-719 is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA. The Phase 2 proof-of-concept study has been completed, and no Phase 3 trials for SION-719 have been registered.
SION-719 has been studied in one clinical trial, NCT07108153, a Phase 2 proof-of-concept study evaluating its safety, tolerability, pharmacodynamics, and pharmacokinetics when added to Trikafta. The trial enrolled 15 participants across the United States and Australia and has been completed.