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Low-Dose REGN4461

Phase 2

Generalized Lipodystrophy | Small molecule | Metabolic |Regeneron Pharmaceuticals, Inc.|Last Updated: Jun 15, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials1
Total Enrollment16

FDA Designations

No designations recorded

Clinical trial landscape

Low-Dose REGN4461 · 1 trial · 1 indication

Phase 2 1
NCT04159415Study of REGN4461, a Leptin Receptor Agonist Antibody, in Patients With Generalized LipodystrophyGeneralized Lipodystrophy
COMPLETED16 Analytics
PHASE2COMPLETED
Study of REGN4461, a Leptin Receptor Agonist Antibody, in Patients With Generalized Lipodystrophy
Generalized LipodystrophyUnlock trial analytics

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 to 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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Recent Changes (Last 90 Days)

MEDIUMJul 16, 2026NCT04159415TRIAL_REMOVED: changed
MEDIUMJul 16, 2026NCT04159415TRIAL_REMOVED: changed
MEDIUMJul 16, 2026NCT04159415TRIAL_REMOVED: changed

Frequently asked questions about Low-Dose REGN4461

What is Low-Dose REGN4461 used for?

Low-Dose REGN4461 is an investigational drug being studied for generalized lipodystrophy, a rare metabolic condition, and has also been evaluated in healthy volunteers. It is being developed by Regeneron Pharmaceuticals, Inc. (REGN) and is currently in Phase 2 clinical development.

What does Low-Dose REGN4461 target?

Low-Dose REGN4461 is a leptin receptor agonist antibody, meaning it targets and activates the leptin receptor. This mechanism is relevant to its potential use in generalized lipodystrophy, a condition characterized by fat loss and metabolic abnormalities.

Who makes Low-Dose REGN4461?

Low-Dose REGN4461 is being developed by Regeneron Pharmaceuticals, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol REGN. The drug is currently in Phase 2 clinical trials for generalized lipodystrophy.

What phase is Low-Dose REGN4461 in?

Low-Dose REGN4461 is in Phase 2 clinical development for generalized lipodystrophy. It is an investigational drug and has not been approved by regulatory authorities. A Phase 1 study in healthy volunteers has been completed.

What clinical trials is Low-Dose REGN4461 in?

Low-Dose REGN4461 has been studied in two completed trials. NCT03530514 was a Phase 1 study in healthy volunteers with 113 participants in Belgium. NCT04159415 was a Phase 2 study in patients with generalized lipodystrophy, enrolling 16 participants across the United States, Peru, Russia, and Turkey.

Is Low-Dose REGN4461 the same as REGN4461?

Yes, Low-Dose REGN4461 is the same drug as REGN4461. The name 'Low-Dose' refers to the specific dosage being studied, but the active agent is REGN4461, a leptin receptor agonist antibody developed by Regeneron Pharmaceuticals.