Viridian Therapeutics announces positive long-term durability data for veligrotug for the treatment of active thyroid eye disease

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Viridian Therapeutics has announced positive long-term durability data from its THRIVE, phase 3 clinical trial of veligrotug for the treatment of active thyroid eye disease (TED).

Veligrotug is an intravenously (IV) delivered, anti-insulin-like growth factor-1 receptor (IGF-1R) antibody. The company also notes that veligrotug has the potential to “improve patient experience with a differentiated dosing regimen featuring a shorter infusion time and fewer infusions compared to the currently approved and marketed IGF-1R inhibitor.”

The THRIVE phase 3 clinical trial in active TED evaluated 5 infusions of veligrotug or placebo every 3 weeks with primary topline analysis at week 15 and followed patients through week 52.

At 52 weeks, 70% of veligrotug patients (21/30) in THRIVE, who were proptosis responders at week 15 and continued follow-up to the end of the study maintained their proptosis response. The company defined maintenance of response as responders at week 15 who still had at least a 2-millimeter (mm) reduction in proptosis compared to baseline at week 52, without worsening in the fellow eye (≥2 mm increase).

Additionally, no changes to the safety profile in the follow-up period were seen, with the vast majority of adverse events reported at the week 15 primary analysis resolved by week 52.

Steve Mahoney, Viridian’s president and CEO commented on the data in a press release from the company.¹

“We view the strength of today’s durability and safety resolution data as reinforcing veli’s strong and consistently robust clinical profile,” said Mahoney. “We believe that the totality of veligrotug’s clinical data continues to demonstrate its potential to be the treatment-of-choice for patients living with TED. We believe these data, together with a streamlined dosing regimen of five infusions, position veli to become a market leading TED therapeutic, if approved.”

As previously reported,² the THRIVE phase 3 met both the primary and all secondary endpoints at 15 weeks after 5 infusions of veligrotug. Additionally, the THRIVE-2 trial³ met all primary and secondary endpoints at the 15-week primary analysis timepoint after 5 infusions of veligrotug.

Recently,⁴ veligrotug was granted Breakthrough Therapy Designation from the US Food and Drug Administration (FDA). Breakthrough Therapy Designation is reserved for drug candidates intended to treat serious or life-threatening conditions that address an unmet need where clinical evidence has indicated they may demonstrate substantial improvement on a clinically significant endpoint over existing therapies.

The company noted that it intends to submit a Biologics License Application for veligrotug for the treatment of TED in the second half of 2025, with a potential launch in 2026 pending approval.

Looking forward, the company is currently dosing patients in 2 global phase 3 clinical trials for VRDN-003, REVEAL-1 and REVEAL-2, in active and chronic TED, respectively. The company expects topline data from the trials in the first half of 2026 and plans to submit a BLA for VRDN-003 by the end of 2026.

References:

1. Viridian Therapeutics announces positive long-term durability data from the veligrotug phase 3 THRIVE clinical trial in patients with active Thyroid Eye Disease (TED). Published May 20, 2025. Accessed May 20, 2025. https://investors.viridiantherapeutics.com/news/news-details/2025/Viridian-Therapeutics-Announces-Positive-Long-Term-Durability-Data-from-the-Veligrotug-Phase-3-THRIVE-Clinical-Trial-in-Patients-with-Active-Thyroid-Eye-Disease-TED/default.aspx

2. Harp M. Viridian announces positive topline data from phase 3 THRIVE trial evaluating veligrotug in patients with active TED. Ophthalmology Times. Published September 12, 2024. Accessed May 20, 2025. https://www.ophthalmologytimes.com/view/viridian-announces-positive-topline-data-from-phase-3-thrive-trial-evaluating-veligrotug-in-patients-with-active-ted

3. Harp M. Viridian announces topline data from THRIVE-2 phase 3 clinical trial of veligrotug for treatment of thyroid eye disease. Ophthalmology Times. Published December 18, 2024. Accessed May 20, 2025. https://www.ophthalmologytimes.com/view/viridian-announces-topline-data-from-thrive-2-phase-3-clinical-trial-of-veligrotug-for-treatment-of-thyroid-eye-disease

4. Viridian Therapeutics receives FDA Breakthrough Therapy Designation for veligrotug for the treatment of thyroid eye disease (TED). Published May 7, 2025. Accessed May 20, 2025. https://investors.viridiantherapeutics.com/news/news-details/2025/Viridian-Therapeutics-Receives-FDA-Breakthrough-Therapy-Designation-for-Veligrotug-for-the-Treatment-of-Thyroid-Eye-Disease-TED/default.aspx