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ALN-HSD

Phase 2

Metabolic Dysfunction-Associated SteatoHepatitis (MASH) | Small molecule | Infectious Disease |Regeneron Pharmaceuticals, Inc.|Last Updated: May 19, 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 Enrollment120
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05519475A Precision Medicine Approach Using Gene Silencing to Treat a Chronic Liver Disease Called Metabolic Dysfunction-Associated Steatohepatitis (MASH) in Adult Participants at Increased Genetic Risk for This ConditionPHASE2 RECRUITING 120Feb 9, 2023Dec 7, 2028May 19, 202671 United States, Japan +2
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Study Endpoints
Primary Endpoints
Change in quantitative liver Fibrosis (qFibrosis)
Baseline to week 52

Change in the continuous qFibrosis score measured by second harmonic generation/two-photon excitation microscopy

Secondary Endpoints
Improvement of Non-Alcoholic Steatohepatitis Clinical Research Network (NASH-CRN) Fibrosis (F) stage by ≥1 stage without worsening of MASH on liver biopsy
Baseline to week 52
Resolution of MASH with no worsening of NASH-CRN fibrosis on liver biopsy
Baseline to week 52
Change in serum ALanine aminoTransferase (ALT)
Baseline to week 52
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-HSDEXPERIMENTALRandomized 1:1
PlaceboPLACEBO_COMPARATORRandomized 1:1
Interventions
NameTypeDescription
ALN-HSDDRUGAdministered per the protocol
PlaceboDRUGAdministered per the protocol
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites71

Key Inclusion Criteria: 1. Adult male or female ≥18 years (or country's legal age of adulthood) 2. A diagnosis of MASH with Fibrosis (F) stage 2 or 3, according to the NASH-CRN 3. NAS score ≥3, as defined in the protocol 4. Meets genotype criteria for study enrollment, as defined in the protocol 5....

Countries:United StatesJapanPuerto RicoSouth Korea
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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT05519475Enrollment: 90 → 120
LOWMay 24, 2026NCT05519475studyFirstPostDate: changed