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ALXN2220

Phase 3

Amyloid Transthyretin Cardiomyopathy | Small molecule | Cardiovascular |AstraZeneca PLC|Trials Updated: Oct 7, 2026

ALXN2220 development status

Highest phase Phase 3
Registered trials 4 across 3 sponsors since Dec 2023

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials3
Total Enrollment2,396

FDA Designations

No designations recorded

ALXN2220 clinical trials

ALXN2220 · 3 trials · 3 indications

Phase 3 2Phase 2 1
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)
Change from Baseline to Week 48 in ECV measured by cMRI
Baseline, Week 48
Change from Baseline to Week 48 in Heart Retention/Whole Body Retention measured by scintigraphy
Baseline, Week 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?

ALXN2220 is being developed for the treatment of amyloid transthyretin cardiomyopathy, also called transthyretin amyloid cardiomyopathy or ATTR-CM. It is an investigational small molecule therapeutic being studied in adults with this cardiovascular condition. ALXN2220 has not been approved for any indication and remains in clinical development.

Who is developing ALXN2220?

ALXN2220 is being developed by AstraZeneca PLC, which trades on the Nasdaq under the ticker AZN. AstraZeneca is the sponsor of the clinical program evaluating ALXN2220 in adults with transthyretin amyloid cardiomyopathy.

What phase is ALXN2220 in?

ALXN2220 is in Phase 3 clinical development for transthyretin amyloid cardiomyopathy. The program includes two Phase 3 studies and one Phase 2 study, with a total planned enrollment of approximately 2,396 participants across the trials. ALXN2220 is investigational and has not been approved by the FDA.

What clinical trials is ALXN2220 in?

ALXN2220 is being evaluated in three registered trials. NCT06183931 is a Phase 3 placebo-controlled study of ALXN2220 versus placebo in adults with ATTR-CM. NCT07789483 is a Phase 3 open-label long-term safety and efficacy study. NCT07213583 is a Phase 2 re-treatment study in patients with ATTR-CM.

Is ALXN2220 placebo controlled?

Yes. The Phase 3 study NCT06183931 is a randomized, double-blind, placebo-controlled trial of ALXN2220 in adults with transthyretin amyloid cardiomyopathy. That trial has a planned enrollment of 1,181 participants and is currently active but not recruiting. The other Phase 3 trial, NCT07789483, is an open-label long-term safety and efficacy study.

Where is ALXN2220 being studied?

ALXN2220 trials are running across many countries, including the United States, Canada, Brazil, Argentina, the United Kingdom, France, Germany, Spain, Italy, the Netherlands, Japan, China, South Korea, Australia, and others. The Phase 3 studies enroll adults with transthyretin amyloid cardiomyopathy, and the Phase 2 re-treatment study runs in France, Germany, the Netherlands, and Spain.