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ALXN2220

Phase 3

Transthyretin Amyloid Cardiomyopathy | Small molecule | Cardiovascular |AstraZeneca PLC|Last Updated: Aug 28, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment1,181

FDA Designations

No designations recorded

Clinical trial landscape

ALXN2220 · 2 trials · 2 indications

Phase 3 2
NCT07789483An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid CardiomyopathyTransthyretin Amyloid Cardiomyopathy (ATTR-CM)
NOT YET_RECRUITING1,180 Analytics
NCT06183931Study of ALXN2220 Versus Placebo in Adults With ATTR-CMTransthyretin Amyloid Cardiomyopathy
ACTIVE NOT_RECRUITING1,181 Analytics
PHASE3NOT YET_RECRUITING
An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid Cardiomyopathy
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)Unlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
Transthyretin Amyloid CardiomyopathyUnlock trial analytics

Study Endpoints

Primary Endpoints

Percentage of participants with TEAEs
Up to 24 months

To evaluate the long-term safety and tolerability of ALXN2220 in participants with ATTR-CM

Total Occurrence of ACM and CV clinical events during the Blinded Treatment Period
Baseline up to the end of study (up to Month 48)

Secondary Endpoints

Total occurrence of ACM and CV clinical events
Up to 24 months
Total occurrence of CV clinical events
Up to 24 months
Time to ACM
Up to 24 months
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ALXN2220EXPERIMENTALEligible participants will receive ALXN2220 administered IV q4w for a minimum of 12 months and up to 24 months or until participant has access to commercially available ALXN2220 for ATTR-CM, whichever occurs first.
PlaceboPLACEBO_COMPARATORParticipants will receive placebo via IV infusion q4w for at least 30 months up to a maximum of 48 months.

Interventions

NameTypeDescription
ALXN2220DRUGTo be administered as an IV infusion
PlaceboDRUGParticipants will receive placebo via IV infusion.
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Eligibility Criteria

Age Range18 Years to 90 Years
SexALL
Healthy VolunteersNo
Study Sites195

* Inclusion Criteria * Type of Participant: Participants who have completed the Blinded Treatment Period (including the PC visit) of Study 301. * Sex and Contraceptive/Barrier Requirements: Male and/or female (according to their reproductive organs and functions assigned by chromosomal complement). ...

Countries:United StatesArgentinaAustraliaAustriaBelgiumBrazilCanadaChinaCzechiaDenmarkFranceGermanyGreeceIrelandIsraelItalyJapanNetherlandsNorwayPolandSouth KoreaSpainSwedenSwitzerlandTaiwanTurkey (Türkiye)United Kingdom
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Recent Changes (Last 90 Days)

LOWAug 31, 2026NCT07789483lastUpdatePostDate: changed
LOWAug 31, 2026NCT07789483lastUpdatePostDate: changed
LOWAug 28, 2026NCT07789483NEW_TRIAL: changed
LOWAug 28, 2026NCT07789483NEW_TRIAL: changed
MEDIUMAug 11, 2026NCT06183931primaryCompletionDate: changed
MEDIUMAug 11, 2026NCT06183931primaryCompletionDate: changed
MEDIUMAug 11, 2026NCT06183931primaryCompletionDate: changed

Frequently asked questions about ALXN2220

What is ALXN2220 used for in transthyretin amyloid cardiomyopathy?

ALXN2220 is an investigational small molecule being developed for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM), a condition in which abnormal protein deposits accumulate in the heart. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.

Who makes ALXN2220?

ALXN2220 is being developed by AstraZeneca PLC, a biopharmaceutical company listed on the stock exchange under the ticker symbol AZN. The drug is currently in Phase 3 clinical trials for transthyretin amyloid cardiomyopathy.

What phase is ALXN2220 in?

ALXN2220 is in Phase 3 clinical development. It is being studied in two Phase 3 trials for transthyretin amyloid cardiomyopathy, including a placebo-controlled study and an open-label long-term safety and efficacy study. The drug is investigational and not yet approved.

What clinical trials is ALXN2220 in?

ALXN2220 is being evaluated in two Phase 3 trials. NCT06183931 is a randomized, double-blind, placebo-controlled study in adults with ATTR-CM, enrolling 1181 participants. NCT07789483 is an open-label study assessing long-term safety and efficacy, with a planned enrollment of 1180 participants.

Is ALXN2220 the same as any other drug?

ALXN2220 is the primary name for this investigational drug. No alternative names have been reported for this compound, which is being developed by AstraZeneca for transthyretin amyloid cardiomyopathy.