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ACI-24 low dose

Phase 1

Down Syndrome | Monoclonal antibody | Other |AC Immune SA|Last Updated: Oct 15, 2021

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 Enrollment20
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02738450Safety, Tolerability and Immunogenicity of ACI-24 Vaccine in Adults With Down SyndromePHASE1 COMPLETED 20Mar 1, 2016Jun 1, 2020Oct 15, 20214 United States
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Study Endpoints
Primary Endpoints
Antibody Titer (Serum Anti-Aβ1-42 Free IgG) - Mean Absolute Value
Values at baseline (week 0) and week 50 are reported

All subjects who received at least 1 dose of the study treatment of either ACI-24 300 μg, ACI-24 1000 μg or placebo are considered. The measure is expressed in Arbitrary Units per mL (AU/mL). AU/mL in a sample is obtained by back-calculation towards the standard curve.

Secondary Endpoints
Amyloid Beta 1-40 in Blood - Mean Absolute Value
Values at baseline (week 0) and week 50 are reported
CANTAB - MOT Latency Score
Values at baseline (week 0) and week 50 are reported
CANTAB - PAL First Attempt Memory Score
Values at baseline (week 0) and week 50 are reported
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ACI-24 low doseACTIVE_COMPARATORVaccine formulation will be administrated s.c. 7 times.
ACI-24 high doseACTIVE_COMPARATORVaccine formulation will be administrated s.c. 7 times.
PlaceboPLACEBO_COMPARATORThe placebo is ready-to-use solution for injection, administrated s.c. 7 times.
Interventions
NameTypeDescription
ACI-24 low doseBIOLOGICALACI-24 administered as a sterile suspension in PBS via s.c. injection.
ACI-24 high doseBIOLOGICALACI-24 administered as a sterile suspension in PBS via s.c. injection.
PlaceboBIOLOGICALPlacebo is a standard PBS sterile solution administrated via s.c. injection.
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Eligibility Criteria
Age Range25 Years — 45 Years
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Males or females with Down Syndrome aged ≥25 to ≤45 years, with a cytogenetic diagnosis being either Trisomy 21 or Complete Unbalanced Translocation of the Chromosome 21. * Subjects and their study partner/legal representative in the opinion of the investigator able to underst...

Countries:United States
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