Recent Updates
Recently added Catalysts

P-1037

Phase 2

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: Jul 26, 2021

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment142
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02343445Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF)PHASE2 COMPLETED 142Apr 1, 2015Feb 1, 2016Jul 26, 202133 United States
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Safety (number of adverse events of P-1037 in treatment groups)
Day 15

number of adverse events of P-1037 in treatment groups

Safety [change in forced expiratory volume in one second (FEV1) from predosing to 1 hour post dosing]
Day 1

change in FEV1 from predosing to 1 hour post dosing with P-1037 in 4.2 % hypertonic saline and P-1037 in 0.17% saline

Secondary Endpoints
Absolute change in FEV1
Day 15
Effect of P-1037 on other forced vital capacity (FVC) measures (change in FVC from baseline (immediately before first dose) to same time of day on Day 15)
Day 15
Effect of P-1037 on Cystic Fibrosis Questionnaire - Revised (CFQ-R)
Day 15
Unlock Study Endpoints
Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
P-1037 in Hypertonic Saline (HS)EXPERIMENTALP-1037 Solution for Inhalation, 85 μg twice daily (BID) (28.3 μg/mL) in hypertonic saline (4.2% saline) BID
P-1037 in SalineEXPERIMENTALP-1037 Solution for Inhalation, 85 μg BID (28.3 μg/mL) in 0.17% saline BID
SalinePLACEBO_COMPARATORPlacebo (0.17% saline) BID
Hypertonic SalineSHAM_COMPARATORHypertonic saline (4.2% saline) BID
Interventions
NameTypeDescription
P-1037DRUGP-1037 is a novel ENaC inhibitor
Hypertonic SalineDRUG4.2% saline solution
SalineDRUG0.17% saline solution
Unlock Study Design Details
Eligibility Criteria
Age Range12 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites33

Inclusion Criteria: * Male or female, 12 years of age or older. * Diagnosis of cystic fibrosis as determined by the 1997 CF Consensus criteria (NIH Consensus Statement, 1997) * Non-smoker * FEV1 at Screening Visit 1 between 40% and 90% * Stable regimen of CF medications and chest physiotherapy for ...

Countries:United States
Unlock Eligibility Criteria