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Low-Dose REGN4461

Phase 2

Generalized Lipodystrophy | Small molecule | Other |Regeneron Pharmaceuticals, Inc.|Last Updated: Nov 17, 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 Enrollment16
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04159415Study of REGN4461, a Leptin Receptor Agonist Antibody, in Patients With Generalized LipodystrophyPHASE2 COMPLETED 16 — — Jan 7, 2020Sep 24, 2024Nov 17, 20258 United States, Peru +2
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Study Endpoints
Primary Endpoints
Absolute Change From Baseline in Participants With Elevated Baseline Hemoglobin A1c (HbA1c ≥7%) at Week 8
Baseline, Week 8

Absolute change from baseline in HbA1c for subgroup of participants with baseline HbA1c ≥7% reported

Absolute Change From Baseline in Fasting Glucose at Week 8
Baseline, Week 8

Absolute change from baseline in fasting glucose for subgroup of participants with baseline HbA1c ≥7% reported

Absolute Change From Baseline in Weighted Mean Glucose (WMG) at Week 8
Baseline, Week 8

Absolute change from baseline in WMG for subgroup of participants with baseline HbA1c ≥7% reported

Percent Change From Baseline in Fasting Triglycerides (TG) at Week 8
Baseline, Week 8

Percent change from baseline in participants with elevated baseline fasting TG (fasting TG ≥250 mg/dL) reported

Secondary Endpoints
Absolute Change From Baseline in HbA1c and Fasting TG Composite Endpoint at Week 8
Baseline, Week 8
Absolute Change From Baseline in Fasting Glucose
Baseline, Weeks 16, 24, 36, 52; OLTP 5 Weeks 4, 12, 24, 52
Absolute Change From Baseline in Fasting Glucose for Participants With Baseline HbA1c ≥7%
Baseline, Weeks 16, 24, 36, 52; OLTP 5 Weeks 4, 12, 24, 52
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Treatment AEXPERIMENTAL -
Treatment BEXPERIMENTAL -
Interventions
NameTypeDescription
PlaceboDRUGIntravenous (IV) infusion loading dose or subcutaneous (SC) injection weekly (QW).
Low-Dose REGN4461DRUGIV infusion loading dose or SC injection QW.
High-dose REGN4461DRUGIV infusion loading dose or SC injection QW.
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Eligibility Criteria
Age Range12 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites8

Key Inclusion Criteria: * Diagnosis of congenital or acquired generalized lipodystrophy (GLD), as defined in the protocol * Presence of one or both of the following metabolic abnormalities at screening: 1. HbA1c ≥ 7% OR 2. Fasting TG ≥250 mg/dL * Generally stable diet (based on patient's recal...

Countries:United StatesPeruRussiaTurkey (Türkiye)
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