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ALXN2220

Phase 3

Transthyretin Amyloid Cardiomyopathy | Small molecule | Cardiovascular |AstraZeneca PLC|Last Updated: Feb 23, 2026

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 Enrollment1,181
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06183931Study of ALXN2220 Versus Placebo in Adults With ATTR-CMPHASE3 ACTIVE NOT_RECRUITING 1,181Jan 11, 2024Oct 5, 2027Feb 23, 2026212 United States, Argentina +25
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Study Endpoints
Primary Endpoints
Total Occurrence of ACM and CV clinical events during the Blinded Treatment Period
Baseline up to the end study (up to Month 48)
Secondary Endpoints
Change From Baseline in Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS) at Month 24
Baseline, Month 24
Time to CV-related Mortality
Baseline up to the end of study (up to Month 48)
Change from Baseline in Six-minute Walk Test (6MWT) at Month 24
Baseline, Month 24
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALXN2220EXPERIMENTALParticipants will receive weight-based dose of ALXN2220 via intravenous (IV) infusion every 4 weeks (q4w) for at least 24 months up to a maximum of 48 months.
PlaceboPLACEBO_COMPARATORParticipants will receive placebo via IV infusion q4w for at least 24 months up to a maximum of 48 months.
Interventions
NameTypeDescription
ALXN2220DRUGParticipants will receive ALXN2220 via IV infusion.
PlaceboDRUGParticipants will receive placebo via IV infusion.
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Eligibility Criteria
Age Range18 Years — 90 Years
SexALL
Healthy VolunteersNo
Study Sites212

Inclusion Criteria: * Diagnosis of ATTR-CM with either wild-type or variant TTR genotype * End-diastolic interventricular septal wall thickness ≥ 12 mm on echocardiography measured at Screening * NT-proBNP \> 2000 pg/mL at Screening * Treatment with a loop diuretic for at least 30 days prior to Scr...

Countries:United StatesArgentinaAustraliaAustriaBelgiumBrazilCanadaChinaCzechiaDenmarkFranceGermanyGreeceIrelandIsraelItalyJapanNetherlandsNorwayPolandSouth KoreaSpainSwedenSwitzerlandTaiwanTurkey (Türkiye)United Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06183931primaryCompletionDate: changed
LOWMay 24, 2026NCT06183931studyFirstPostDate: changed