Viridian Announces Positive Topline Results from Phase 3 Trial of Veligrotug for TED

Viridian Therapeutics announced positive topline data from its THRIVE phase 3 clinical trial of VRDN-001, now branded as veligrotug, in patients with active thyroid eye disease (TED). Veligrotug, an intravenously administered anti-insulin-like growth factor-1 receptor (IGF-1R) antibody, demonstrated promising results in addressing this autoimmune condition.

“We are thrilled to see these exciting THRIVE topline results, which met our high bars for both efficacy and safety,” Steve Mahoney, Viridian’s President and CEO, said in a company news release. “Veligrotug’s rapid onset of clinically meaningful responses and its superior safety profile exceeded our expectations. With a differentiated five-infusion dosing regimen, veligrotug offers the potential for a more convenient treatment with a lower IV burden compared to the current standard of care.”

Dr. Mahoney emphasized the importance of veligrotug and VRDN-003, another therapy in development for TED, which Viridian hopes will become the preferred treatments for TED patients. Viridian also initiated two phase 3 clinical trials for VRDN-003 last month, aiming for transformative subcutaneous injections that could further expand the market.

Phase 3 Clinical Results

The THRIVE trial enrolled 113 patients and met both primary and secondary endpoints, demonstrating significant improvements in TED-related symptoms. Veligrotug-treated patients showed improvements in proptosis (bulging eyes), clinical activity score (CAS), and diplopia (double vision).

• Proptosis Response Rate: 70% of veligrotug-treated patients achieved a proptosis response after 15 weeks, compared to 5% of placebo-treated patients.
• Diplopia Resolution: 54% of veligrotug-treated patients achieved complete resolution of diplopia, compared to 12% in the placebo group.
• CAS Improvement: 64% of veligrotug-treated patients saw a reduction in CAS to 0 or 1, indicating maximal or near-maximal therapeutic effect.

The trial also showed a rapid onset of action, with over half of the veligrotug-treated patients seeing a proptosis response after one infusion.

Veligrotug was generally well-tolerated, with most adverse events (AEs) reported as mild. There were no serious treatment-related AEs, and the rate of hearing impairment—closely monitored due to its known association with IGF-1R therapies—was low, with a placebo-adjusted rate of 5.5%, according to Viridian. 

Viridian said it remains on track to submit a biologics license application (BLA) for veligrotug in the second half of 2025. The ongoing THRIVE-2 trial, focused on patients with chronic TED, is set to deliver topline data by the end of 2024.

Viridian’s broader TED franchise also includes VRDN-003, a subcutaneously administered IGF-1R antibody currently in phase 3 trials. Viridian aims for a BLA submission for VRDN-003 by the end of 2026, about 1 year after veligrotug.