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ALXN1720

Phase 3

Generalized Myasthenia Gravis | Small molecule | Neurology |AstraZeneca PLC|Last Updated: Apr 21, 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_CONTROLLEDDMC
Total Trials1
Total Enrollment261
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05556096Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia GravisPHASE3 ACTIVE NOT_RECRUITING 261Nov 21, 2022Aug 31, 2027Apr 21, 2026132 United States, Argentina +20
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Study Endpoints
Primary Endpoints
Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 26
Baseline, Week 26
Secondary Endpoints
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Week 26
Baseline, Week 26
Percentage of Responders Based on Reduction of the MG-ADL Total Score at Week 26
Baseline up to Week 26
Percentage of Responders based on Reduction of the QMG Total Score at Week 26
Baseline up to Week 26
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALXN1720EXPERIMENTALParticipants will receive a weight-based initial (loading) dose of ALXN1720 on Day 1, followed by weight-based maintenance treatment with ALXN1720 on Day 8 and once every week (Q1W) thereafter for a total of 26 weeks. Following this randomized controlled treatment (RCT) period, all participants will receive ALXN1720 in an open-label extension (OLE) period of 105 weeks.
PlaceboPLACEBO_COMPARATORParticipants will receive placebo during the 26-week RCT period, after which they will enter the OLE period of the study and receive ALXN1720.
Interventions
NameTypeDescription
ALXN1720COMBINATION_PRODUCTCombination product consisting of syringe prefilled with ALXN1720.
PlaceboCOMBINATION_PRODUCTCombination product consisting of syringe prefilled with placebo.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites132

Inclusion Criteria: * Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV * Positive serological test for autoantibodies against AChR Exclusion Criteria: * History of thymectomy, or any other ...

Countries:United StatesArgentinaAustriaBrazilCanadaChinaDenmarkFranceGermanyIsraelItalyJapanNetherlandsPolandPortugalSerbiaSouth KoreaSpainSwitzerlandTaiwanTurkey (Türkiye)United Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05556096primaryCompletionDate: changed
LOWMay 24, 2026NCT05556096studyFirstPostDate: changed