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ALN-TTRSC04

Phase 1

Transthyretin-Mediated Amyloidosis | Small molecule | Other |Alnylam Pharmaceuticals, Inc.|Last Updated: Feb 17, 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 Enrollment96
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05661916A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy SubjectsPHASE1 COMPLETED 96Jan 16, 2023Sep 30, 2025Feb 17, 20261 United Kingdom
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Study Endpoints
Primary Endpoints
Frequency of Adverse Events
Up to 20 months
Secondary Endpoints
Change from Baseline in Serum Transthyretin (TTR) Levels Over Time
Up to 18 months
Maximum Observed Plasma Concentration (Cmax) of ALN-TTRSC04
Day 1 up to Day 4
Time to Maximum Observed Plasma Concentration (Tmax) of ALN-TTRSC04
Day 1 up to Day 4
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-TTRSC04EXPERIMENTALParticipants will be administered a single dose of ALN-TTRSC04.
PlaceboPLACEBO_COMPARATORParticipants will be administered a single dose of placebo.
Interventions
NameTypeDescription
ALN-TTRSC04DRUGALN-TTRSC04 will be administered by subcutaneous (SC) injection.
PlaceboDRUGPlacebo will be administered by SC injection.
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Body mass index (BMI) ≥18.0 kg/m\^2 and ≤30 kg/m\^2 assessed at Screening. Exclusion Criteria: * Has an estimated glomerular filtration rate (eGFR) \<90 mL/min/1.73m\^2 at screening. * Has alanine aminotransferase (ALT), aspartate aminotransferase (AST) or direct bilirubin a...

Countries:United Kingdom
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