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Natalizumab

Phase 3

Multiple Sclerosis, Relapsing-Remitting | Small molecule | Immunology |Biogen Inc.|Last Updated: Jun 12, 2024

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 Trials4
Total Enrollment2,795
FDA Designations
No designations recorded
Clinical Trials (4)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03689972A Study to Evaluate Efficacy, Safety, and Tolerability of EID of Natalizumab (BG00002) in Participants With RRMS Switching From Treatment With Natalizumab SID in Relation to Continued SID Treatment- Followed by Extension Study Comprising SC and IV Natalizumab AdministrationPHASE3 COMPLETED 585Nov 27, 2018Jul 24, 2023Jun 12, 2024107 United States, Australia +9
NCT00030966Safety and Efficacy of Natalizumab in Combination With Avonex in the Treatment of Multiple SclerosisPHASE3 COMPLETED 1,200Jan 1, 2002Dec 1, 2005Jun 18, 200988 United States, Austria +5
NCT00027300Safety and Efficacy of Natalizumab in the Treatment of Multiple SclerosisPHASE3 COMPLETED 900Nov 1, 2001Jan 1, 2005Jan 9, 201753 United States, Belgium +6
NCT00097760Natalizumab in Combination With Glatiramer Acetate (GA) in Patients With Relapsing-Remitting Multiple SclerosisPHASE2 COMPLETED 110Jun 1, 2003Mar 1, 2004Jun 18, 2009 -
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Study Endpoints
Primary Endpoints
Part 1: Mean Number of New or Newly Enlarging T2 Hyperintense Lesions at Week 72
Week 72

T2 hyperintense lesions were analyzed by magnetic resonance imaging (MRI) scans of brain. New MRI scans were compared with the prior MRI scans to analyze the number of new or newly enlarging T2 hyperintense lesions at Week 72 relative to baseline.

Part 2: Percentage of Participants Indicating a Preference for Natalizumab SC Administration at the End of Crossover Period of Part 2
Week 150
Primary objectives of this study are to determine if natalizumab is effective in reducing the rate of clinical relapse at 1 year and in slowing the progression of disability at 2 years as measured by EDSS.
1 year and 2 years
The primary objectives of this study are to determine whether natalizumab, when compared with placebo, is effective in reducing the rate of clinical relapses at 1 year and, in slowing the progression of disability at 2 years.
1 year and 2 years
Rate of development of new active lesions on MRI scans.
Week 20
Secondary Endpoints
Part 1: Time to First Relapse as Adjudicated by an Independent Neurology Evaluation Committee (INEC)
Up to Week 72
Part 1: Annualized Relapse Rate at Week 72
Week 72
Part 1: Time to Expanded Disability Status Scale (EDSS) Worsening
Up to Week 72
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part 1: IV Q4WEXPERIMENTALParticipants received natalizumab 300 mg intravenous (IV) infusion once Q4W up to Week 72.
Part 1: IV Q6WEXPERIMENTALParticipants received natalizumab 300 mg IV infusion once Q6W up to Week 72.
Part 2: Run-in Period: IV Q6WEXPERIMENTALParticipants who completed Part 1 or were newly enrolled in Part 2 received natalizumab 300 mg IV infusion Q6W from Week 72 through Week 102.
Part 2: Crossover Period: IV Q6W, then SC Q6WEXPERIMENTALParticipants who completed run-in period of Part 2 were randomized to receive natalizumab 300 mg IV infusion Q6W from Week 108 through Week 126 followed by natalizumab 300 mg subcutaneous (SC) injection Q6W from Week 132 through Week 150 along with a single dose of natalizumab 300 mg SC injection or IV infusion as per participant's choice at Week 156.
Part 2: Crossover Period: SC Q6W, then IV Q6WEXPERIMENTALParticipants who completed run-in period of Part 2 were randomized to receive natalizumab 300 mg SC injection Q6W from Week 108 through Week 126 followed by natalizumab 300 mg IV infusion Q6W from Week 132 through Week 150 along with a single dose of natalizumab 300 mg SC injection or IV infusion as per participant's choice at Week 156.
Group 1EXPERIMENTALAdding natalizumab monthly infusion to Avonex weekly injection for up to 116 weeks.
Group 2PLACEBO_COMPARATORAdding placebo monthly infusion to Avonex weekly injection for up to 116 weeks.
Interventions
NameTypeDescription
NatalizumabDRUGNatalizumab 300 mg IV infusion.
PlaceboDRUGPlacebo monthly infusion for up to 116 weeks.
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Eligibility Criteria
Age Range18 Years — 60 Years
SexALL
Healthy VolunteersNo
Study Sites107

Key Inclusion Criteria: For Part 1: * Ability of the participant to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local participant privacy regulations. * Diagnosi...

Countries:United StatesAustraliaBelgiumCanadaFranceGermanyIsraelItalyNetherlandsSpainUnited KingdomAustriaCzechia
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