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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...
Top 6 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | Alyftrek, Trikafta, Ivacaftor, VX-272, Tezacaftor |
| 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 |
| Roivant Sciences Ltd. | ROIV | 1 | PHASE1 | SP-101 |
SION-719 is an investigational small molecule developed by Sionna Therapeutics, Inc. for cystic fibrosis. It is designed to be added to Trikafta, an existing CF treatment regimen. SION-719 has completed a Phase 2 proof-of-concept study and is not approved for any use.
SION-719 is being developed for cystic fibrosis, a respiratory disease. The Phase 2 study evaluated SION-719 when added to Trikafta, so the intended use is as an add-on to existing CF modulator therapy rather than as a standalone treatment. It remains investigational.
SION-719 targets NBD1, a protein domain of the cystic fibrosis transmembrane conductance regulator. NBD1 is a protein target, and SION-719 is a small molecule designed to act on it. This is distinct from the mechanism of Trikafta, which SION-719 is studied alongside.
Sionna Therapeutics, Inc. is developing SION-719. The company trades on the Nasdaq under the ticker SION. Sionna is the sponsor of the clinical program for SION-719 in cystic fibrosis.
SION-719 is in Phase 2 development. Its single Phase 2 proof-of-concept trial has been completed, and no trials are currently active. SION-719 is investigational and has not been approved by the FDA for any indication.
SION-719 has been studied in one Phase 2 trial, NCT07108153, titled a proof-of-concept study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of SION-719 when added to Trikafta. That trial is completed and enrolled 15 participants across the United States and Australia.