Veligrotug Shows Durability for Thyroid Eye Disease in Phase 3 Trial Results

Long-term data from the Phase 3 THRIVE trial for veligrotug, an anti-insulin-like growth factor-1 receptor (IGF-1R) intravenous antibody targeted to treat active thyroid eye disease (TED), indicated positive long-term durability.1

Announced on May 20, 2025, by Viridian Therapeutics, Inc., these results come soon after the US Food and Drug Administration (FDA) granted veligrotug Breakthrough Therapy Designation. The designation supports veligrotug’s eligibility for priority review, as well as all Fast Track designation features.2

“We view the strength of today’s durability and safety resolution data as reinforcing [veligrotug’s] strong and consistently robust clinical profile,” said Steve Mahoney, president and chief executive officer of Viridian Therapeutics. “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.”1

Veligrotug is an intravenously delivered anti-IGF-1R antibody with the potential to be the IV treatment-of-choice for active and chronic TED patients. IGF-1R is a clinically validated target for TED; compared to currently available IGF-1R inhibitors, veligrotug has the potential to improve patient experience through a differentiated dosing regimen featuring shorter infusion time and fewer infusions.1

Veligrotug achieved all primary and secondary endpoints in the Phase 3 clinical trials THRIVE and THRIVE-2, as announced by Viridian Therapeutics in 2024. It demonstrated rapid onset of treatment effect and statistically significant reduction and resolution of diplopia in both trials. THRIVE-2 was the first data set from a global Phase 3 trial in patients with chronic TED to exhibit significant diplopia response and resolution.1

The THRIVE trial evaluated 5 infusions of either veligrotug or placebo every three weeks over 52 weeks; primary topline analysis occurred at week 15 and followed patients through week 52. Across 30 patients, 21 (70%) were proptosis responders at week 15 and maintained their response at the end of the study follow-up. Investigators defined responders at week 15 who still had ≥2-millimeter reduction in proptosis compared to baseline by the end of the study without worsening in the fellow eye.1

The THRIVE-2 trial enrolled 188 patients who were randomized to either veligrotug (n = 125) and placebo (n = 63). More than half (56%) of veligrotug patients exhibited an overall response, compared to 7% of placebo patients. Overall response rate was defined as achieving proptosis response without worsening of CAS from baseline and without worsening in the fellow eye. Additionally, veligrotug was generally well-tolerated with a safety profile consistent with prior trials.3

In the release, Viridian Therapeutics indicated the Biologics License Application (BLA) submission for veligrotug remained on track for the second half of 2025.

“We believe these data, together with a streamlined dosing regimen of five infusions, position [veligrotug] to become a market-leading TED therapeutic, if approved,” Mahoney said. “We continue to make great progress towards submitting the BLA in the second half of this year and preparing for a potential launch in 2026.”1

References

1. Viridian Therapeutics. Viridian Therapeutics Announces Positive Long-Term Durability Data from the Veligrotug Phase 3 THRIVE Clinical Trial in Patients with Active Thyroid Eye Disease (TED). 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. Viridian Therapeutics. Viridian Therapeutics Receives FDA Breakthrough Therapy Designation for Veligrotug for the Treatment of Thyroid Eye Disease (TED). 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

3. Viridian Therapeutics. Viridian Therapeutics Announces Positive Topline Results from Veligrotug Phase 3 THRIVE-2 Clinical Trial in Patients with Chronic Thyroid Eye Disease. December 16, 2024. Accessed May 20, 2025. https://investors.viridiantherapeutics.com/news/news-details/2024/Viridian-Therapeutics-Announces-Positive-Topline-Results-from-Veligrotug-Phase-3-THRIVE-2-Clinical-Trial-in-Patients-with-Chronic-Thyroid-Eye-Disease/default.aspx