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Aztreonam for

Phase 3

Cystic Fibrosis | Small molecule | Respiratory |Gilead Sciences, Inc.|Last Updated: Jul 17, 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 Enrollment484
FDA Designations
No designations recorded
Clinical Trials (3)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00757237Aztreonam for Inhalation Solution vs Tobramycin Inhalation Solution in Patients With Cystic Fibrosis & Pseudomonas AeruginosaPHASE3 COMPLETED 274 — — Aug 1, 2008Nov 1, 2010Jul 4, 201192 United States, Austria +11
NCT01375049Aztreonam Lysine for Pseudomonas Infection Eradication StudyPHASE2 COMPLETED 105 — — Aug 1, 2011May 1, 2013Jul 17, 201458 United States, Austria +7
NCT01055847Aztreonam for Inhalation (AI) in Patients With Cystic Fibrosis & P. Aeruginosa InfectionPHASE2 COMPLETED 105 — — Jun 1, 2003Sep 1, 2004Jan 26, 201020 United States
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Study Endpoints
Primary Endpoints
Relative Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Percent Predicted at Day 28
Baseline and end of treatment Course 1 (Day 28)

Spirometry was performed according to American Thoracic Society (ATS) guidelines at each visit. FEV1 percent predicted is a normalized value of FEV1 calculated using the Knudson equation and based upon participant age, gender, and height. Treatment effect on the relative change from baseline in FEV1 percent predicted at Day 28 (Visit 4) was tested using an analysis of covariance (ANCOVA) model-based method.

Mean Actual Change From Baseline in FEV1 Percent Predicted Across 3 Treatment Courses
Baseline, and end of treatment Courses 1 (Week 4), 2 (Week 12), and 3 (Week 20)

Spirometry was performed according to ATS guidelines at each visit. FEV1 percent predicted is a normalized value of FEV1 calculated using the Knudson equation and based upon participant age, gender, and height. Treatment effect on the average adjusted means for the actual change in FEV1 percent predicted at Visits 4, 6, and 8 (Weeks 4, 12, and 20) was tested by mixed-effect model repeated measures (MMRM) analysis using the ITT population analysis set.

Percentage of Participants With PA-negative Cultures at All Time Points After Cessation of Active Treatment (Evaluable Analysis Set)
Day 28 to Day 196

The percentage of participants with PA-negative cultures at all time points after cessation of active treatment at Day 28 (assessed at Days 56, 112, and 196) was summarized for the Evaluable Analysis Set.

Percentage of Participants With PA-negative Cultures at All Time Points After Cessation of Active Treatment (Sensitivity Analysis Set)
Day 28 to Day 196

The percentage of participants with PA-negative cultures at all time points after cessation of active treatment at Day 28 (assessed at Days 56, 112, and 196) was summarized for the Sensitivity Analysis Set.

Change in FEV1 from Baseline to Day 14
14 Days
Secondary Endpoints
Relative Change From Baseline in FEV1 Percent Predicted at Day 28 in Subjects Who Received Inhaled Tobramycin for >= 84 Days in the 12 Months Prior to Randomization
Baseline and end of treatment Course 1 (Day 28)
Mean Actual Change From Baseline in FEV1 Percent Predicted Across 3 Treatment Courses in Subjects Who Received Inhaled Tobramycin for >= 84 Days in the 12 Months Prior to Randomization
Baseline and end of treatment Courses 1 (Week 4), 2 (Week 12), and 3 (Week 20)
Time to Need for Intravenous (IV) Antipseudomonal Antibiotics for Respiratory Events
Day 0 to Day 168 (end of study)
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AZLI 75 mg 3 times a day (TID)EXPERIMENTAL -
TIS 300 mg 2 times a day (BID)ACTIVE_COMPARATOR -
Aztreonam for Inhalation Solution (AZLI)EXPERIMENTALParticipants will receive one 28-day course of AZLI, then will be followed for a 24-week period (through Day 196).
AI 75 mgEXPERIMENTALAztreonam for Inhalation 75 mg twice daily
AI 225 mgEXPERIMENTALAztreonam for Inhalation 225 mg twice daily
PlaceboPLACEBO_COMPARATORPlacebo
Interventions
NameTypeDescription
Aztreonam for Inhalation Solution (AZLI)DRUGAztreonam for inhalation solution (75 mg) was administered 3 times a day (TID) for 28 days for each treatment cycle via the PARI eFlow electronic nebulizer.
Tobramycin Inhalation Solution (TIS)DRUGTobramycin inhalation solution (300 mg) was administered 2 times a day (BID) for 28 days for each treatment cycle via the PARI LC Plus nebulizer with compressor or via another nebulizer compatible with country-specified labeling.
Aztreonam for Inhalation (AI)DRUGAztreonam for Inhalation
PlaceboDRUGSaline Placebo
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Eligibility Criteria
Age Range6 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites92

Inclusion Criteria: * Males or females aged 6 years and older * Subjects with CF as diagnosed by one of the following: documented sweat chloride \>= 60 mEq/L by quantitative pilocarpine iontophoresis test, or documented sweat sodium \>= 60 mmol/L, or 2 well characterized genetic mutations in the Cy...

Countries:United StatesAustriaBelgiumDenmarkFranceGermanyIrelandItalyNetherlandsPortugalSpainSwitzerlandUnited KingdomPoland
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Competitive Landscape -Cystic Fibrosis 18 trials
CompanyTickerTrialsLead PhaseDrugs
Vertex Pharmaceuticals IncorporatedVRTX9PHASE3VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, VNZ/TEZ/D-IVA, VX-522 mRNA therapy
Sionna Therapeutics, Inc.SION2PHASE2SION-719, SION-451, SION-2222, SION-109
BiomX LtdPHGE1PHASE2BX004
4D Molecular Therapeutics, Inc.FDMT1PHASE24D-710
Arcturus Therapeutics Holdings, Inc.ARCT1PHASE2ARCT-032
Krystal Biotech, Inc.KRYS1PHASE1KB407
Illumina, Inc.ILMN1 — Undisclosed
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