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Bis-Choline Tetrathiomolybdate

Phase 2

Wilson Disease | Small molecule | Rare Disease |AstraZeneca PLC|Last Updated: Oct 18, 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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment31
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04422431Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840PHASE2 COMPLETED 31 — — Dec 2, 2020May 17, 2023Oct 18, 202412 United States, Canada +6
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Study Endpoints
Primary Endpoints
Change From Baseline in Liver Cu Concentration at Week 48 (Treatment Period)
Baseline, Week 48 (Treatment Period)

Liver biopsy samples were taken for the assessment of liver Cu concentration. Multiple imputation was used to impute missing data at Week 48 due to any reason based on Baseline values.

Secondary Endpoints
Number of Participants With Change From Baseline in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Stage at Week 48 (Treatment Period)
Baseline, Week 48 (Treatment Period)
Number of Participants With Change From Baseline in Metavir Fibrosis Score at Week 48 (Treatment Period)
Baseline, Week 48 (Treatment Period)
Number of Participants With Change From Baseline in Ishak Fibrosis Score at Week 48 (Treatment Period)
Baseline, Week 48 (Treatment Period)
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALXN1840EXPERIMENTALParticipants will receive ALXN1840.
Interventions
NameTypeDescription
Bis-Choline TetrathiomolybdateDRUGParticipants will be initiated at 15 milligrams once daily, then the dose will be increased to 30 milligrams once daily at Week 6.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites12

Inclusion Criteria: 1. Diagnosis of WD by Leipzig Criteria ≥ 4 or by historical test results. 2. Continuous treatment for WD with penicillamine, trientine or zinc for at least 1 year prior to screening. 3. Body mass index \< 30 kilograms/meter squared. 4. Able to cooperate with a percutaneous liver...

Countries:United StatesCanadaDenmarkNew ZealandRussiaSingaporeSpainUnited Kingdom
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