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ALN-PNP

Phase 2

Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD) | Small molecule | Metabolic |Regeneron Pharmaceuticals, Inc.|Last Updated: Apr 14, 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_CONTROLLEDBiomarker
Total Trials1
Total Enrollment204
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07527910A Phase 2a Study of ALN-PNP With and Without a GLP1R Agonist in Adult Patients With Homozygous PNPLA3-Related MASLDPHASE2 NOT YET_RECRUITING 204Apr 20, 2026Nov 27, 2029Apr 14, 2026 -
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Study Endpoints
Primary Endpoints
Percent change in liver fat
From baseline at week 24

Part A

Secondary Endpoints
Achievement of liver fat <5%
At weeks 24 and 48
Occurence of Treatment-Emergent Adverse Events (TEAEs)
Through week 60
Severity of TEAEs
Through week 60
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part A: A1EXPERIMENTAL -
Part A: A2EXPERIMENTAL -
Part B: B1EXPERIMENTAL -
Part B: B2EXPERIMENTAL -
Part B: B3EXPERIMENTAL -
Part B: B4PLACEBO_COMPARATOR -
Interventions
NameTypeDescription
ALN-PNPDRUGAdministered per the protocol
TirzepatideDRUGAdministered per the protocol
PlaceboDRUGAdministered per the protocol Placebo matching ALN-PNP
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo

Key Inclusion Criteria: Part A and Part B: 1. Homozygous for the PNPLA3 p.I148M genotype 2. Liver fat by Magnetic Resonance Imaging-Proton Density Fat Fraction (MRI-PDFF) ≥15% at visit 3 3. Has a Body Mass Index (BMI) ≥30 to \<45 kg/m\^2 at visit 2 Part A: To be eligible for randomization on stud...

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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07527910primaryCompletionDate: changed
LOWMay 24, 2026NCT07527910studyFirstPostDate: changed
LOWMay 21, 2026NCT07527910NEW_TRIAL: changed
LOWMay 21, 2026NCT07527910NEW_TRIAL: changed