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ALXN1720

Phase 3

Generalized Myasthenia Gravis | Small molecule | Neurology |AstraZeneca PLC|Last Updated: Jun 17, 2026

Success Probability

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment260

FDA Designations

No designations recorded

Clinical trial landscape

ALXN1720 · 3 trials · 3 indications

Phase 3 1Phase 1 2
NCT05556096Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia GravisGeneralized Myasthenia Gravis
ACTIVE NOT_RECRUITING260 Analytics
PHASE3ACTIVE NOT_RECRUITING
Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis
Generalized Myasthenia GravisUnlock trial analytics

Study Endpoints

Primary Endpoints

Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 26
Baseline, Week 26
Serum Concentration of ALXN1720
Day 1 (0.5 hours predose and postdose) and postdose on Days 2, 3, 8, 15, 29, 43, and 57
Incidence of Treatment-emergent and Serious Adverse Events (TEAEs, SAEs)
Up to 176 days following the first day of dosing

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.
ALXN1720 Single Dose SCEXPERIMENTALParticipants will receive a single dose of ALXN1720 SC.
ALXN1720 Multiple Dose SCEXPERIMENTALParticipants will receive multiple doses of ALXN1720 SC.
ALXN1720 Single Dose SC + rHuPH20EXPERIMENTALParticipants will receive a single dose of ALXN1720 SC in combination with rHuPH20.
ALXN1720 Single Dose IVEXPERIMENTALParticipants will receive a single dose of ALXN1720 IV.

Interventions

NameTypeDescription
ALXN1720COMBINATION_PRODUCTCombination product consisting of syringe prefilled with ALXN1720.
PlaceboCOMBINATION_PRODUCTCombination product consisting of syringe prefilled with placebo.
ALXN1720 SCDRUGALXN1720 will be administered via SC route.
ALXN1720 IVDRUGALXN1720 will be administered via IV route.
rHuPH20DRUGrHuPH20 will be administered via SC route.
Placebo SCDRUGPlacebo will be administered via SC route.
Placebo IVDRUGPlacebo will be administered via IV route.
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Eligibility Criteria

Age Range18 Years to 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)

LOWJun 18, 2026NCT05556096Enrollment: 261 → 260
LOWJun 18, 2026NCT05556096Enrollment: 261 → 260
LOWJun 18, 2026NCT05556096Enrollment: 261 → 260
LOWJun 18, 2026NCT05556096Enrollment: 261 → 260

Frequently asked questions about ALXN1720

What is ALXN1720 used for in generalized myasthenia gravis?

ALXN1720 is an investigational small molecule being studied for the treatment of generalized myasthenia gravis (gMG). It is currently in Phase 3 clinical development for this condition. The drug is administered subcutaneously and is being evaluated in a randomized, double-blind, placebo-controlled trial.

Who is developing ALXN1720?

ALXN1720 is being developed by AstraZeneca PLC, a biopharmaceutical company traded on the stock exchange under the ticker symbol AZN. The company is conducting clinical trials to evaluate the safety and efficacy of ALXN1720 in multiple patient populations.

What phase is ALXN1720 in?

ALXN1720 is in Phase 3 clinical development for generalized myasthenia gravis. The Phase 3 trial is active but not recruiting participants. Earlier Phase 1 studies of ALXN1720 have been completed in healthy volunteers and in participants with proteinuria.

What clinical trials is ALXN1720 in?

ALXN1720 has been studied in several clinical trials. The Phase 3 trial, NCT05556096, is evaluating the drug in adults with generalized myasthenia gravis. Completed Phase 1 trials include NCT04920370 in healthy subjects and NCT05314231 in participants with proteinuria.

What does ALXN1720 target?

ALXN1720 is a small molecule therapeutic agent. Its specific molecular target has not been disclosed in the available clinical trial information. The drug is being investigated for its potential effects in neurological and renal conditions.

Is ALXN1720 the same as other drugs for myasthenia gravis?

ALXN1720 is a distinct investigational drug being developed by AstraZeneca. It is not known to be the same as any other approved or investigational medication. Its unique mechanism and chemical structure differentiate it from existing therapies for generalized myasthenia gravis.