Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
AZLI three times daily · 1 trial · 3 indications
The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for children and adults with CF. The CFQ-R contains both general and CF-specific scales. The CFQ-R was administered at Days 0, 14, 28, and 42. The endpoint was change in respiratory symptoms (e.g., coughing, congestion, wheezing) from Day 0 (baseline), assessed with the CFQ-R RSS (score range: 0-100; higher scores indicating fewer symptoms, higher health-related quality of life, or better functioning). Baseline CFQ-R RSS and age group (\<18 vs. \>=18 years) were included as covariates in the analysis.
| Arm | Type | Description |
|---|---|---|
| Placebo three times daily (TID) | PLACEBO_COMPARATOR | - |
| AZLI 75 mg three times daily (TID) | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| AZLI 75 mg three times daily (TID) | DRUG | - |
| Placebo three times daily (TID) | DRUG | - |
Inclusion Criteria: * Participants ≥ 6 years of age * Documentation of CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria: * Sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis test * Two well ch...
| 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 |
AZLI three times daily is an investigational small molecule being studied for the treatment of Cystic Fibrosis, specifically in patients with mild lung disease and Pseudomonas aeruginosa infection. It is administered as an inhalation solution. The drug is currently in Phase 3 clinical development and has not been approved by regulatory authorities.
AZLI three times daily is being developed by Gilead Sciences, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol GILD. The company is conducting clinical trials to evaluate the safety and efficacy of this inhaled aztreonam formulation for patients with Cystic Fibrosis and Pseudomonas aeruginosa lung infection.
AZLI three times daily is in Phase 3 clinical development. It is an investigational drug, meaning it has not yet received regulatory approval. The Phase 3 trial has been completed, and the drug remains under investigation for the treatment of Cystic Fibrosis with mild lung disease and Pseudomonas aeruginosa infection.
AZLI three times daily has one completed Phase 3 clinical trial registered as NCT00712166. This randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of aztreonam for inhalation solution in 160 patients with Cystic Fibrosis, mild lung disease, and Pseudomonas aeruginosa. The trial was conducted in the United States, Australia, and Canada.
AZLI three times daily is not FDA approved. It is an investigational drug currently in Phase 3 clinical development. The completed Phase 3 trial (NCT00712166) evaluated its safety and efficacy, but regulatory approval has not been granted. The drug remains under investigation for Cystic Fibrosis with Pseudomonas aeruginosa infection.