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PF-06865571

Phase 2

Nonalcoholic Fatty Liver Disease | Small molecule | Infectious Disease |Pfizer, Inc.|Last Updated: Mar 21, 2025

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 Enrollment256
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04321031Metabolic Interventions to Resolve Non-alcoholic Steatohepatitis (NASH) With Fibrosis (MIRNA)PHASE2 COMPLETED 256Jun 15, 2020Feb 21, 2024Mar 21, 2025291 United States, Bulgaria +10
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Study Endpoints
Primary Endpoints
Mean Proportion of Participants Achieving Resolution of NASH Without Worsening/Improvement of Fibrosis by >=1 Stage Without Worsening of NASH/Both Based on Assessment by Sponsor-Identified Central Pathologist at Week 48:Bayesian Dose Response Model (BDRM)
Week 48

NASH resolution: disappearance of ballooning (Nonalcoholic Fatty Liver Disease \[NAFLD\] Activity Score \[NAS\] ballooning score=0;0=no ballooning,1=few balloon cells,2=many cells with prominent ballooning; higher scores(HS)=more disease activity \[DA\]),residual/no lobular inflammation(NAS lobular inflammation score 0/1,0=no foci,1= \<2 foci, 2=2-4 foci,3= \>4 foci; HS=more DA),NAS steatosis score 0,1,2,3; 0= \<5% hepatocytes involved (HI),1=5-33% HI ,2= 34-66% HI, 3= \>66% HI; HS=more DA. No worsening of fibrosis: no change/decrease of at least 1 stage in Brunt-Kleiner scale (BKS) compared to baseline (CTB). Improvement in fibrosis by \>=1 stage: decrease of at least 1 stage in BKS CTB. No worsening of NASH: no change/increase in NAS for ballooning, inflammation, steatosis CTB. BDRM utilized to characterize dose response across BID treatment groups, estimate posterior mean proportion (\& 90% credible interval \[CI\], indicated as 'confidence interval' below) for placebo and each BID dose studied.

Number of Participants Achieving Resolution of NASH Without Worsening or Improvement in Fibrosis by >= 1 Stage Without Worsening of NASH or Both Based on Assessment by Sponsor-Identified Central Pathologist at Week 48: Logistic Regression Model
Week 48

Resolution of NASH: disappearance of ballooning (NAS ballooning score= 0; where 0= no ballooning, 1= few balloon cells, 2= many cells with prominent ballooning; higher scores= more disease activity), residual or no lobular inflammation (NAS lobular inflammation score 0 or 1, where 0= no foci, 1= \<2 foci, 2= 2-4 foci, 3= \>4 foci; higher scores= more disease activity), NAS steatosis score 0, 1, 2, or 3, where 0= \<5% hepatocytes involved, 1= 5-33% hepatocytes involved, 2= 34-66% hepatocytes involved, 3= \>66% hepatocytes involved; higher scores= more disease activity. No worsening of fibrosis: no change or decrease of at least 1 stage in BKS compared to baseline. Improvement in fibrosis by \>=1 stage: decrease of at least 1 stage in BKS compared to baseline. No worsening of NASH: no change or increase in NAS for ballooning, inflammation, steatosis compared to baseline.

Secondary Endpoints
Percent Change From Baseline in Liver Fat at Week 48: Bayesian Dose Response Model
Baseline, Week 48
Percent Change From Baseline in Liver Fat at Week 48: Pairwise Comparisons With Analysis of Covariance (ANCOVA)
Baseline, Week 48
Mean Proportion of Participants Achieving Resolution of NASH, Without Worsening of Fibrosis Based on Assessment by Sponsor-Identified Central Pathologist(s) at Week 48: Bayesian Dose Response Model
Week 48
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeBASIC_SCIENCE
Treatment Arms
ArmTypeDescription
PlaceboPLACEBO_COMPARATORparticipants will receive medication for 48 weeks
PF-06865571 25 milligrams (mg) twice daily (BID)EXPERIMENTALparticipants will receive medication for 48 weeks
PF-06865571 75 mg BIDEXPERIMENTALparticipants will receive medication for 48 weeks
PF-06865571 150 mg BIDEXPERIMENTALparticipants will receive medication for 48 weeks
PF-06865571 300 mg BIDEXPERIMENTALparticipants will receive medication for 48 weeks
PF-06865571 (150 mg BID) + PF-05221304 (5 mg BID)EXPERIMENTALparticipants will receive medication for 48 weeks
PF-06865771 (300 mg BID) + PF-05221304 (10 mg BID)EXPERIMENTALparticipants will receive medication for 48 weeks
Interventions
NameTypeDescription
PlaceboDRUGTablet
PF-06865571DRUGTablet
PF-05221304DRUGTablet
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites291

Inclusion Criteria: * Biopsy proven NASH with either F2 or F3 fibrosis, per NASH CRN definition * BMI \>/= 22.5kg/m2 Exclusion Criteria: * Evidence of other causes of liver disease such as Alcoholic steatohepatitis, (de)compensated cirrhosis, active viral hepatitis * Any condition possibly affect...

Countries:United StatesBulgariaCanadaChinaHong KongIndiaJapanPolandPuerto RicoSlovakiaSouth KoreaTaiwan
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