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AZLI

Phase 3

Bronchiectasis | Small molecule | Respiratory |Gilead Sciences, Inc.|Last Updated: Apr 16, 2014

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials3
Total Enrollment629
FDA Designations
No designations recorded
Clinical Trials (3)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01313624Safety and Effectiveness of AZLI (an Inhaled Antibiotic) in Adults With Non-Cystic Fibrosis BronchiectasisPHASE3 COMPLETED 266Apr 1, 2011Jun 1, 2013Apr 16, 201460 United States, Australia +1
NCT01314716Safety and Effectiveness of AZLI (an Inhaled Antibiotic) in Adults With Non-Cystic Fibrosis BronchiectasisPHASE3 COMPLETED 274Apr 1, 2011Jul 1, 2013Apr 16, 201485 United States, Australia +8
NCT00805025Evaluation of the Quality of Life Questionnaire-Bronchiectasis (QOL-B) in Patients With BronchiectasisPHASE2 COMPLETED 89Dec 1, 2008Oct 1, 2009Mar 20, 201424 United States
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Study Endpoints
Primary Endpoints
Change in QOL-B Respiratory Symptoms Score at Day 28
Baseline to Day 28

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.

Reliability of the Respiratory Domain of the Quality of Life Questionnaire-Bronchiectasis (QOL-B)
Day -14 to Day 0

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 of the Respiratory Domain of the QOL-B
Day -14

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.

Secondary Endpoints
Change in QOL-B Respiratory Symptoms Score at Day 84
Baseline to Day 84
Time to Protocol-Defined Exacerbation (PDE)
Baseline to Day 112
Responsiveness of the Respiratory Domain of the QOL-B as Assessed by the Anchor-based Minimal Clinically Important Difference (MCID) Following Categorization of Level of Change Using the Global Rating of Change Questionnaire (GRCQ)
Day 0 to Day 28
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AZLI-AZLIEXPERIMENTALParticipants 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-AZLIPLACEBO_COMPARATORParticipants 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.
AZLIEXPERIMENTALParticipants were evaluated beginning 14 days prior to starting a 28-day course of AZLI (Day 0 to Day 28), followed by post-treatment assessments every 14 days through Day 56, for a total of 70 days of participation in the study.
Interventions
NameTypeDescription
AZLIDRUGAZLI 75 mg reconstituted with diluent and administered via nebulizer three times daily
PlaceboDRUGPlacebo to match AZLI administered via nebulizer three times daily
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites60

Inclusion Criteria: * Male/Female 18 years or older with non-CF bronchiectasis * Chronic sputum production on most days * Positive sputum culture for gram-negative organisms * Must have met lung function requirements Exclusion Criteria: * History of CF * Hospitalized within 14 days prior to joini...

Countries:United StatesAustraliaCanadaBelgiumFranceGermanyItalyNetherlandsSpainUnited Kingdom
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Competitive Landscape -Bronchiectasis 8 trials