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AP01

Phase 2

Pulmonary Fibrosis | Small molecule | Immunology |Avalyn Pharma Inc.|Last Updated: Apr 6, 2026

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 Trials1
Total Enrollment375
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06329401A Study Evaluating the Safety and Efficacy of Inhaled AP01 in Participants With Progressive Pulmonary FibrosisPHASE2 RECRUITING 375Apr 3, 2024Jun 1, 2027Apr 6, 2026152 United States, Argentina +12
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Study Endpoints
Primary Endpoints
To evaluate the effect of AP01 high dose twice a day (BID) or AP01 low dose twice a day (BID) compared to placebo twice a day (BID)
Week 52

Change from baseline in forced vital capacity (FVC) (mL)

Secondary Endpoints
To evaluate the effect of AP01 high dose and AP01 low dose compared to placebo on disease progression (defined as absolute FVC percent predicted decline of ≥10% prior to Week 52)
52 weeks
To evaluate the effect of AP01 high dose, AP01 low dose compared to placebo on quality of life (QoL)
52 weeks
To evaluate the change from baseline in quantitative lung fibrosis score.
52 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AP01 High Dose BIDEXPERIMENTALPirfenidone Solution for Inhalation
AP01 Low Dose BIDEXPERIMENTALPirfenidone Solution for Inhalation
Placebo BIDPLACEBO_COMPARATORPlacebo solution for inhalation
Interventions
NameTypeDescription
AP01DRUGOral inhalation solution
PlaceboOTHERPlacebo oral inhalation solution
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites152

Inclusion Criteria: * Participant meets criteria for PPF, as follows: * In subjects with interstitial lung disease (ILD) of known or unknown etiology other than idiopathic pulmonary fibrosis (IPF) who have radiological evidence of pulmonary fibrosis, PPF is defined as: Physiological evidence of di...

Countries:United StatesArgentinaAustraliaBelgiumCanadaFranceGermanyItalyNetherlandsNew ZealandPolandSpainTurkey (Türkiye)United Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06329401primaryCompletionDate: changed
LOWMay 24, 2026NCT06329401studyFirstPostDate: changed