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ACT-128800 Dose 1

Phase 2

Multiple Sclerosis | Small molecule | Immunology |Johnson & Johnson|Last Updated: Mar 30, 2025

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_CONTROLLEDDMC
Total Trials1
Total Enrollment464
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01006265Clinical Study to Evaluate the Efficacy, Safety, and Tolerability of ACT-128800 in Patients With Relapsing-remitting Multiple SclerosisPHASE2 COMPLETED 464Oct 1, 2009Jul 1, 2011Mar 30, 2025115 United States, Australia +21
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Study Endpoints
Primary Endpoints
Cumulative Number of New T1 Gadolinium-Enhancing (Gd+) Lesions on Magnetic Resonance Imaging (MRI) Scan From Week 12 to Week 24
From Week 12 to 24

Cumulative Number of new T1 gadolinium-enhancing (Gd+) lesions per year on magnetic resonance imaging (MRI) scan from Week 12 to Week 24 were reported. Negative binomial (NB) regression analysis on Per protocol analysis set and imputation was applied for the missing data. Here, MS signifies multiple sclerosis.

Secondary Endpoints
Annualized Confirmed Relapse Rate
Up to 24 weeks
Number of Participants With First Confirmed Relapse as Assessed by Kaplan-Meier Estimate From Baseline to Week 24
Baseline to Week 24
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ACT-128800 Dose 1EXPERIMENTALACT-128800 Dose 1
ACT-128800 Dose 2EXPERIMENTALACT-128800 Dose 2
ACT-128800 Dose 3EXPERIMENTALACT-128800 Dose 3
PlaceboPLACEBO_COMPARATORMatching placebo
Interventions
NameTypeDescription
ACT-128800 Dose 1DRUGACT-128800 (Dose 1) administered orally once daily
PlaceboDRUGMatching placebo administered orally once daily
ACT-128800 Dose 2DRUGACT-128800 (Dose 2) administered orally once daily
ACT-128800 Dose 3DRUGACT-128800 (Dose 3) administered orally once daily
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Eligibility Criteria
Age Range18 Years — 55 Years
SexALL
Healthy VolunteersNo
Study Sites115

Inclusion Criteria: * Adult males and females * Diagnosis of RRMS as defined by the revised (2005) McDonald Diagnostic Criteria for Multiple Sclerosis (MS). * Signed inform consent prior to initiation of any study-mandated procedure. Exclusion Criteria: * A diagnosis of MS categorized as primary ...

Countries:United StatesAustraliaAustriaBelgiumBulgariaCanadaCzechiaFinlandFranceGermanyHungaryIsraelItalyNetherlandsPolandRomaniaRussiaSerbiaSpainSwedenSwitzerlandUkraineUnited Kingdom
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