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Veligrotug

Phase 3

Thyroid Eye Disease | Small molecule | Endocrine |Viridian Therapeutics, Inc.|Last Updated: Aug 14, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials4
Total Enrollment671

FDA Designations

BREAKTHROUGH_THERAPYPRIORITY_REVIEW

Clinical trial landscape

Veligrotug · 4 trials · 1 indication

Phase 3 4
NCT06384547A Randomized, Controlled, Safety and Tolerability Study of VRDN-001 in Participants With Thyroid Eye Disease (TED)Thyroid Eye Disease
COMPLETED231 Analytics
NCT06179875An Open-label Study (OLE) for Non-responders of VRDN-001-101 and VRDN-001-301Thyroid Eye Disease
COMPLETED139 Analytics
NCT06021054An Efficacy, Safety, and Tolerability Study of Veligrotug (VRDN-001), in Participants With Chronic Thyroid Eye Disease (TED)Thyroid Eye Disease
COMPLETED188 Analytics
NCT05176639A Safety, Tolerability, and Efficacy Study of Veligrotug (VRDN 001) in Participants With Thyroid Eye Disease (TED)Thyroid Eye Disease
COMPLETED113 Analytics
PHASE3COMPLETED
A Randomized, Controlled, Safety and Tolerability Study of VRDN-001 in Participants With Thyroid Eye Disease (TED)
Thyroid Eye DiseaseUnlock trial analytics
PHASE3COMPLETED
An Open-label Study (OLE) for Non-responders of VRDN-001-101 and VRDN-001-301
Thyroid Eye DiseaseUnlock trial analytics
PHASE3COMPLETED
An Efficacy, Safety, and Tolerability Study of Veligrotug (VRDN-001), in Participants With Chronic Thyroid Eye Disease (TED)
Thyroid Eye DiseaseUnlock trial analytics
PHASE3COMPLETED
A Safety, Tolerability, and Efficacy Study of Veligrotug (VRDN 001) in Participants With Thyroid Eye Disease (TED)
Thyroid Eye DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Treatment-emergent Adverse Events (TEAEs) Through Week 15
Baseline through Week 15

An adverse event (AE) was defined as any untoward medical occurrence that developed or worsened in severity during the conduct of a clinical study and did not necessarily have a causal relationship to study drug. TEAE was defined as an AE occurring on or after the day of the first dose of study medication through the end of the study. A summary of all Serious Adverse Events (SAEs) and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Proptosis Responder Rate (PRR) in the Most Proptotic Eye as Measured by Exophthalmometer
Baseline to Week 15

Proptosis response in the most proptotic eye was defined as a reduction of proptosis of ≥2 millimeters (mm) from baseline in the most proptotic eye (without a corresponding increase of ≥2 mm in the other eye) as measured by exophthalmometer. Missing data were not imputed.

Proptosis Responder Rate (PRR) in the Study Eye As Measured by Exophthalmometer
Baseline to Week 15

Proptosis responder in the study eye was defined as a reduction of proptosis of ≥2 millimeters (mm) from baseline in the study eye (without a corresponding increase of ≥2 mm in the fellow eye) as measured by exophthalmometer. Missing data were imputed with the Multiple Imputation method.

Secondary Endpoints

Change From Baseline in Proptosis in the Study Eye as Measured by Exophthalmometer
Baseline, Week 15
Number of Participants With TEAEs Through Week 52
Baseline through Week 52
Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by Exophthalmometer
Baseline, Week 15
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Veligrotug 10 mg/kgEXPERIMENTALParticipants will receive 5 intravenous (IV) infusions of veligrotug 10 milligrams (mg)/kilogram (kg) at 3-week intervals for 12 weeks.
Veligrotug 3 mg/kgEXPERIMENTALParticipants will receive 5 IV infusions of veligrotug 3 mg/kg at 3-week intervals for 12 weeks.
Veligrotug/VeligrotugEXPERIMENTALNon-responder participants who received veligrotug in the parent Study VRDN-001-101 or VRDN-001-301 will receive veligrotug 10 milligrams (mg)/kilogram (kg) every 3 weeks (Q3W) for 12 weeks.
Placebo/VeligrotugEXPERIMENTALNon-responder participants who received placebo in the parent Study VRDN-001-101 or VRDN-001-301 will receive veligrotug 10 mg/kg Q3W for 12 weeks.
VeligrotugEXPERIMENTALParticipants will receive veligrotug 10 mg/kg as an intravenous (IV) infusion every 3 weeks (Q3W) for 12 weeks.
PlaceboEXPERIMENTALParticipants will receive placebo matched to veligrotug as an IV infusion Q3W for 12 weeks.

Interventions

NameTypeDescription
VeligrotugDRUG5 infusions of veligrotug
PlaceboDRUG5 IV Infusions of veligrotug matched placebo
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Eligibility Criteria

Age Range18 Years to 75 Years
SexALL
Healthy VolunteersNo
Study Sites50

Inclusion Criteria: * Have a clinical diagnosis of TED with or without proptosis and with any clinical activity score (CAS) (0 - 7) and in the opinion of the investigator may benefit from treatment * Must agree to use highly effective contraception method as specified in the protocol * Female TED p...

Countries:United StatesAustraliaFranceGermanyPolandSpainUnited KingdomNetherlandsTurkey (Türkiye)Hungary
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Frequently asked questions about Veligrotug

What is Veligrotug used for?

Veligrotug is an investigational small molecule being developed for the treatment of Thyroid Eye Disease (TED), a condition affecting the tissues around the eyes. It is currently in Phase 3 clinical development and has not yet been approved by regulatory authorities. The drug is being studied in patients aged 18 years and older.

Who makes Veligrotug?

Veligrotug is being developed by Viridian Therapeutics, Inc., a biopharmaceutical company listed on the NASDAQ under the ticker symbol VRDN. The company is conducting Phase 3 clinical trials to evaluate the drug's safety and efficacy in patients with Thyroid Eye Disease.

What phase is Veligrotug in?

Veligrotug is currently in Phase 3 clinical development for Thyroid Eye Disease. It has received Breakthrough Therapy designation and Priority Review designation from the FDA, indicating the agency's recognition of its potential to address a serious condition. However, it remains investigational and is not yet approved for commercial use.

What clinical trials is Veligrotug in?

Veligrotug has been studied in four completed Phase 3 clinical trials. These include NCT05176639, NCT06021054, NCT06179875, and NCT06384547, all conducted in patients with Thyroid Eye Disease. The trials were randomized, double-blind, and placebo-controlled, with a total enrollment of 671 participants across multiple countries.

Is Veligrotug the same as VRDN-001?

Yes, Veligrotug is also known as VRDN-001. Clinical trial records refer to the drug by both names, with studies such as NCT05176639 and NCT06021054 using the VRDN-001 designation. This naming convention is common during drug development before a brand name is established.

What does Veligrotug target?

Veligrotug is a small molecule designed to target the underlying mechanisms of Thyroid Eye Disease. While the specific molecular target is not disclosed in the available information, the drug is being developed to address the inflammatory and tissue changes associated with the condition, potentially offering a new treatment option for patients.