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VK0214

Phase 1

Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD) | Small molecule | Neurology |Viking Therapeutics, Inc.|Last Updated: Feb 17, 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 Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04973657A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMNPHASE1 COMPLETED 24Jun 22, 2021Nov 8, 2024Feb 17, 20259 United States, France +3
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Study Endpoints
Primary Endpoints
Incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious AEs (TESAEs)
28 days

Evaluate the safety and tolerability of multiple oral doses of VK0214 in subjects with AMN

Secondary Endpoints
Evaluate the Pharmacokinetics of VK0214
28 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeOTHER
Treatment Arms
ArmTypeDescription
VK0214 Active 20mgEXPERIMENTAL20mg QD
VK0214 Active 40mgEXPERIMENTAL40mg QD
PlaceboPLACEBO_COMPARATORPlacebo QD
Interventions
NameTypeDescription
VK0214DRUGAPI in capsule
PlaceboOTHERAPI in capsule
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria: * Provide a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study and is willing and able to participate. * Have diagnosis of X-linked adrenoleukodystrophy (X-ALD) by genetic testing and elevated...

Countries:United StatesFranceGermanyItalyUnited Kingdom
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