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lonigutamab

Phase 1

Thyroid Eye Disease | Small molecule | Endocrine |ACELYRIN, INC.|Last Updated: Jul 1, 2025

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-BlindCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment31
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05683496Efficacy and Safety of Lonigutamab in Subjects With Thyroid Eye Disease (TED)PHASE1 COMPLETED 31Feb 14, 2023May 26, 2025Jul 1, 202511 United States, Australia
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Study Endpoints
Primary Endpoints
Incidence and characterization of nonserious treatment emergent adverse events (TEAEs)
Day 1 to Day 169

Safety and Tolerability

Incidence and characterization of serious treatment emergent adverse events (TEAEs)
Day 1 to Day 169

Safety and Tolerability

Secondary Endpoints
PK profile of lonigutamab
Day 1 to Day 169
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1EXPERIMENTALSingle SC injection of dose 1 of lonigutamab or placebo at Day 1 and Day 21
Cohort 2EXPERIMENTALmultiple doses of dose 2 of lonigutamab administered SC injection weekly
Cohort 3EXPERIMENTALmultiple doses of lonigutamab dose 3 administered SC injection every 4 weeks.
Cohort 4EXPERIMENTALmultiple doses of lonigutamab dose 4 administered SC injection every 4 or 3 weeks.
Interventions
NameTypeDescription
lonigutamabDRUGsubcutaneous injection
PlaceboDRUGsubcutaneous injection
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites11

Key Inclusion Criteria: * Male or female, ≥18 and ≤75 years of age. * Proptosis defined in the study eye as ≥3 mm above normal. * Clinical Activity Score (CAS) ≥4 (using a 7-item scale) for the most severely affected eye * Onset of active TED symptoms prior to baseline * Must agree to use highly ef...

Countries:United StatesAustralia
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