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ALN-AT3SC

Phase 1

Hemophilia A | Small molecule | Hematology |Sanofi|Last Updated: Dec 19, 2020

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_CONTROLLED
Total Trials1
Total Enrollment51
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02035605A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B PatientsPHASE1 COMPLETED 51Jan 20, 2014Jul 20, 2017Dec 19, 202014 United States, Bulgaria +3
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Study Endpoints
Primary Endpoints
The safety of ALN-AT3SC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), dose-limiting toxicities (DLTs), and AEs leading to study drug discontinuation.
Part A (SAD phase): through day 56; Part B (MAD) phase: through Day 70; Part C (MD Phase) through Day 112; Part D (MD Phase in patients with inhibitors) through Day 112
Secondary Endpoints
The pharmacokinetics (PK) of ALN-AT3SC as characterized by plasma PK profiles and urine samples.
Part A (SAD) phase: through day 56; Part B (MAD) phase: through Day 70; Part C (MD Phase) through Day 112; Part D (MD Phase in patients with inhibitors) through Day 112
The pharmacodynamic (PD) effect of ALN-AT3SC, evaluated by Plasma AT levels.
Part A (SAD) phase: through day 56; Part B (MAD) phase: through Day 70; Part C (MD Phase) through Day 112; Part D (MD Phase in patients with inhibitors) through Day 112
The pharmacodynamic (PD) effect of ALN-AT3SC, evaluated by Plasma TG.
Part A (SAD) phase: through day 56; Part B (MAD) phase: through Day 70; Part C (MD Phase) through Day 112; Part D (MD Phase in patients with inhibitors) through Day 112
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-AT3SCACTIVE_COMPARATOR -
Sterile Normal Saline (0.9% NaCl)PLACEBO_COMPARATOR -
Interventions
NameTypeDescription
ALN-AT3SCDRUGAscending doses of ALN-AT3SC 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
SexMALE
Healthy VolunteersYes
Study Sites14

Inclusion Criteria: Part A (SAD phase) inclusion: * Healthy adult males aged 18 to 40 years inclusive at Screening. * Subjects with adequate complete blood counts and liver function tests. * Willing to provide written informed consent and willing to comply with study requirements. Part B \& C (MA...

Countries:United StatesBulgariaRussiaSwitzerlandUnited Kingdom
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