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PF-05221304

Phase 2

Nonalcoholic Fatty Liver Disease | Small molecule | Infectious Disease |Pfizer, Inc.|Last Updated: Dec 9, 2020

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 Enrollment305
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03248882Phase 2a, Dose-ranging Study With PF-05221304 in Nonalcoholic Fatty Liver Disease (NAFLD)PHASE2 COMPLETED 305Aug 22, 2017Mar 27, 2019Dec 9, 2020140 United States, Australia +4
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Study Endpoints
Primary Endpoints
Percent Change From Baseline in Liver Fat by Magnetic Resonance Imaging-Proton Density Fat Fraction (MRI- PDFF) at Week 16
Baseline (between Day -14 and Day 1), Week 16

MRI-PDFF utilized a gradient echo sequence with low flip angle (FA) to minimize T1 bias, corrected T2\* decay (due to iron overload) via modeling of the fat signal as a superposition of multiple frequency components from 5 different lipid types, and was applied in each of the 9 Couinaud segments. This technique improved fat quantification accuracy for the entire liver permitting quantification of small differences/changes following pharmacological intervention.

Secondary Endpoints
Percent Change From Baseline in Alanine Aminotransferase at Week 16
Baseline (Day 1 pre-dose), Week 16
Number of Participants With Treatment-Emergent Adverse Events
From first dose of study treatment (Day 1) up to Week 20
Number of Participants With Laboratory Abnormalities
From first dose of study treatment (Day 1) up to Week 20
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
PlaceboPLACEBO_COMPARATORDouble-Blind, PF-05221304-matching Placebo
PF-05221304 - 2 mgACTIVE_COMPARATORPF-05221304 - 2 mg, once-daily
PF-05221304 - 10 mgACTIVE_COMPARATORPF-05221304 - 10 mg, once-daily
PF-05221304 - 25 mgACTIVE_COMPARATORPF-05221304 - 25 mg, once-daily
PF-05221304 - 50 mgACTIVE_COMPARATORPF-05221304 - 50 mg, once-daily
Interventions
NameTypeDescription
PlaceboDRUGPlacebo
PF-05221304DRUGPF-05221304, Experimental Drug
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites140

Inclusion Criteria: * Body Mass Index \>= 25 kg/m2 * Body Weight \> 50 kg * Liver fat (assessed via MRI-PDFF) \>= 8% * Biopsy-proven NASH - diagnosed in previous 24-months * Presumed NASH - per Sponsor's definition * NAFLD with minimal inflammation/fibrosis * Features of Metabolic Syndrome Exclusi...

Countries:United StatesAustraliaCanadaIsraelPolandTaiwan
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