Approval Probability
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Label indication CAYSTON ® is indicated to improve respiratory symptoms in cystic fibrosis (CF) patients with Pseudomonas aeruginosa . Safety and effectiveness have not been established in pediatric patients below the age of 7 years, patients with FEV 1 <25% or >75% predicted, or patients colonized with Burkholderia cepacia [see Clinical Studies (14) ]. FDA label
Also known as Cayston®, Aztreonam for Inhalation Solution (AZLI), aztreonam, inhaled antibiotic
AZLI · 6 trials · 4 indications
PDEs were characterized by a change or worsening from baseline of 1 or more documented signs or symptoms (decreased exercise tolerance, increased cough, increased sputum or chest congestion, decreased appetite, or other signs or symptoms) associated with the use of non-study IV or inhaled antibiotics and be verified by a blinded independent adjudication committee.
Participants who discontinued study drug due to safety or tolerability reasons were defined as those with "Adverse Event (AE)/Safety or Tolerability" on the Study Drug Completion electronic case report form as the reason for early discontinuation. The 95% confidence interval (CI) was calculated using the exact binomial method.
The mean (SD) change in the Respiratory Symptoms score on the Quality of Life Questionnaire-Bronchiectasis (QOL-B) was measured from baseline to the end of Course 1 (Day 28). The QOL-B respiratory symptoms score was transformed onto a scale of 0-100, with higher scores representing a better quality of life.
The relative change (AUCave) in FEV1 % predicted from baseline to Week 24 was analyzed. FEV1 % predicted is defined as FEV1 % of the patient divided by the average FEV1 % in the population for any person of similar age, sex and body composition. AUCave is the calculated area under the curve corrected for baseline and adjusted by the number of days on study through Week 24.
Test-retest reliability is a measure of the stability or reproducibility of a measure over a period of time during which status on the underlying construct has not changed, and is measured by the intraclass correlation of scores obtained at 2 time points within that period. Test-retest reliability of respiratory symptoms was calculated for response at Day -14 and Day 0. Reliability of the participants' QOL-B responses was assessed from an Intraclass Correlation Coefficient (ICC). A score of ≥ 0.70 would indicate strong reliability. The QOL-B respiratory symptoms score was transformed onto a scale of 0-100, with higher scores representing a better quality of life.
Convergent validity was assessed at Day -14 by examining the correlations between relevant QOL-B domains and other indicators of health status: a bronchiectasis severity score based on high-resolution computerised tomography (HRCT) scan results, forced expiratory volume in 1 second (FEV1) percent predicted, 6-minute walk test (6MWT) results, and St. George's Respiratory Questionnaire (SGRQ) symptoms scores. Correlations with absolute values of 0.30 to 0.50 indicated moderate evidence of convergent validity.
| Arm | Type | Description |
|---|---|---|
| AZLI | ACTIVE_COMPARATOR | Participants will be randomized to receive 3 cycles of treatment, each cycle consisting alternating regimens: AZLI for 28 days followed by TIS for 28 days. |
| Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive 3 cycles of treatment, each cycle consisting alternating regimens: placebo to match AZLI for 28 days followed by TIS for 28 days. |
| Open-label AZLI | EXPERIMENTAL | Participants received three 28-day courses of AZLI, each followed by 28 days off-treatment. |
| AZLI-AZLI | EXPERIMENTAL | Participants were randomized to receive blinded AZLI for 2 cycles of 28 days on treatment with each cycle followed by 28 days off treatment, followed by open-label AZLI for 28 days plus 56 days of treatment-free follow-up. |
| Placebo-AZLI | PLACEBO_COMPARATOR | Participants were randomized to receive blinded placebo to match AZLI for 2 cycles of 28 days on treatment with each cycle followed by 28 days off treatment, followed by open-label AZLI for 28 days plus 56 days of treatment-free follow-up. |
| Name | Type | Description |
|---|---|---|
| AZLI | DRUG | Aztreonam for Inhalation Solution (AZLI) 75 mg 3 times daily combined with diluent administered using an eFlow nebulizer |
| Placebo to match AZLI | DRUG | Placebo to match AZLI 3 times daily combined with diluent administered using an eFlow nebulizer |
| Tobramycin inhalation solution | DRUG | Tobramycin inhalation solution (TIS) 300 mg 2 times daily using a PARI® LC Plus nebulizer and DeVilbiss Pulmo-Aide® air compressor |
| Placebo | DRUG | Placebo to match AZLI administered via nebulizer three times daily |
Inclusion Criteria: * Confirmed diagnosis of CF * Presence of PA in 2 lower respiratory tract cultures in the 12 months prior to screening * Forced expiratory volume (FEV)1 ≥ 25 and ≤ 75% predicted * History of 1 hospitalization or 1 course of IV antibiotics for an acute respiratory exacerbation in...
Top 4 of 6 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| AstraZeneca PLC | AZN | 3 | PHASE2 | AZD0292 |
| GSK plc Sponsored ADR | GSK | 1 | PHASE2 | GSK3862995B |
| Merck & Co., Inc. | MRK | 1 | PHASE2 | Ensifentrine |
| Genentech, Inc. | RHHBY | 1 | PHASE1 | GDC-6988 |
AZLI is an investigational inhaled antibiotic being studied for use in cystic fibrosis and non-cystic fibrosis bronchiectasis. It is developed by Gilead Sciences, Inc. (NASDAQ: GILD). AZLI is a small molecule delivered via inhalation to target chronic Pseudomonas aeruginosa infections in the lower airways of patients with these conditions.
AZLI targets Pseudomonas aeruginosa, a bacterium that chronically infects the lower airways of patients with cystic fibrosis and bronchiectasis. By delivering an antibiotic directly to the lungs via inhalation, AZLI aims to reduce bacterial load and improve respiratory outcomes in these patient populations.
AZLI is developed by Gilead Sciences, Inc., a biopharmaceutical company traded on NASDAQ under the ticker GILD. Gilead is conducting clinical trials to evaluate the safety and effectiveness of AZLI in adults with non-cystic fibrosis bronchiectasis and in children with cystic fibrosis and chronic Pseudomonas aeruginosa infection.
AZLI is in Phase 3 clinical development. Multiple Phase 3 trials have been completed, including studies in adults with non-cystic fibrosis bronchiectasis and in children with cystic fibrosis. AZLI is investigational and has not been approved by the FDA for any indication.
AZLI has been studied in several completed clinical trials. NCT01313624 and NCT01314716 are Phase 3 trials evaluating safety and effectiveness in adults with non-cystic fibrosis bronchiectasis. NCT01404234 is a Phase 3 trial assessing safety in children with cystic fibrosis and chronic Pseudomonas aeruginosa. NCT00805025 is a Phase 2 trial of a quality of life questionnaire in bronchiectasis patients.
AZLI is a distinct inhaled antibiotic formulation developed by Gilead Sciences. It is not the same as other marketed inhaled antibiotics, as it is a proprietary small molecule designed for aerosolized delivery to the lungs. No alternative names for AZLI have been disclosed in clinical trial records.