Viridian announces positive topline data from Phase 3 THRIVE trial assessing veligrotug in patients with active TED

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Viridian Therapeutics announced positive topline data from its phase 3 THRIVE clinical trial (NCT05176639) of veligrotug, previously known as VRDN-001, in patients with active thyroid eye disease (TED).

Results showed that patients who received veligrotug, an intravenously delivered anti-insulin-like growth factor-1 receptor (IGF-1R) antibody, had “statistically significant and clinically meaningful improvements across [multiple] key disease endpoints at the primary efficacy analysis timepoint of 15 weeks.”^1,2

Furthermore, THRIVE met both the primary and all secondary endpoints at 15 weeks after 5 infusions of veligrotug. Additionally, veligrotug showed a rapid onset of action, with the majority (53%) of veligrotug-treated patients achieving a proptosis response after just 1 infusion, or 3 weeks after the start of therapy.¹

THRIVE enrolled 113 patients, with 75 patients treated with veligrotug and 38 treated with placebo.

Results showed a 70% proptosis responder rate (PRR) in patients receiving veligrotug, compared with 5% of patients receiving placebo (64% placebo-adjusted) (p < 0.0001), as well as a 2.9 mm mean reduction in proptosis from baseline in patients receiving veligrotug, compared with 0.5mm reduction in patients receiving placebo.¹

Complete diplopia resolution was seen in 54% patients receiving veligrotug, compared with 12% of patients receiving placebo, with a 63% diplopia response in patients receiving veligrotug, compared with 20% of patients receiving placebo.¹

The overall responder rate for those treated with veligrotug was 67%, while those treated with placebo was 5%. Veligrotug was also generally well-tolerated, with majority of adverse events (AEs) being mild. There was a low rate (4%) of discontinuations in the veligrotug arm, and no treatment-related serious AEs were reported.¹

Michael Yen, MD, an investigator in THRIVE and Professor of Oculoplastic Surgery and Ophthalmology, at Baylor College of Medicine, commented on the safety profile of veligrotug in a press release from Viridin.

“Veligrotug showed a favorable safety profile in a large phase 3 clinical trial that closely monitored safety, including the potential for hearing impairment,” said Yen. “As a THRIVE investigator, I am excited to see these results and look forward to the REVEAL clinical trials for VRDN-003 to help bring additional potential treatment options for patients living with TED.”

The company noted that the phase 3 clinical trial arm of THRIVE-2, evaluating veligrotug in patients with chronic TED is ongoing, with top-line data readout expected at the end of 2024. Furthermore, the company stated it plans on submitting a Biologics License Application for veligrotug for the treatment of TED in the second half of 2025.¹

References:

1. Viridian Therapeutics Announces Positive Topline Results from Veligrotug (VRDN-001) Phase 3 THRIVE Clinical Trial in Patients with Active Thyroid Eye Disease. Press Release; September 10, 2024. Accessed September 12, 2024. https://investors.viridiantherapeutics.com/news/news-details/2024/Viridian-Therapeutics-Announces-Positive-Topline-Results-from-Veligrotug-VRDN-001-Phase-3-THRIVE-Clinical-Trial-in-Patients-with-Active-Thyroid-Eye-Disease/default.aspx

2. A Safety, Tolerability and Efficacy Study of VRDN 001 in Healthy Volunteers and Persons With Thyroid Eye Disease (TED) (THRIVE). NCT05176639. https://www.clinicaltrials.gov/study/NCT05176639?spons=Viridian%20Therapeutics,%20Inc.&rank=2&page=1&limit=10