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ALN-TTRSC02

Phase 1

Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) | Small molecule | Other |Alnylam Pharmaceuticals, Inc.|Last Updated: Sep 21, 2018

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
RandomizedPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment80
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02797847A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy SubjectsPHASE1 COMPLETED 80Jun 7, 2016Jan 12, 2018Sep 21, 20181 United Kingdom
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Study Endpoints
Primary Endpoints
Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs)
Day 1 through to Day 314
Secondary Endpoints
Profile of pharmacokinetics (PK) of ALN-TTRSC02
Day 1 through to Day 3
Effect of ALN-TTRSC02 on serum TTR levels as measured by reduction from baseline in serum TTR
Day 1 through to Day 314
Effect of ALN-TTRSC02 on serum Vitamin A levels as measured by reduction from baseline in serum Vitamin A
Screening through to Day 314
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-TTRSC02ACTIVE_COMPARATOR -
Sterile normal saline 0.9% for SC administrationPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
ALN-TTRSC02DRUGAscending doses of ALN-TTRSC02 by subcutaneous (SC) injection
Sterile Normal Saline (0.9% NaCl)DRUGCalculated volume to match active comparator
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Male and female subjects, age 18 to 65 years, inclusive. * Body mass index (BMI) ≥18.0 kg/m2 and ≤30 kg/m2 assessed at Screening. * No clinically significant health concerns, as determined by medical history and physical examination, in the opinion of the Investigator. * Women...

Countries:United Kingdom
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