Viridian Therapeutics Reports Positive Long-Term Data from Veligrotug Phase 3 Trial in TED Patients

Viridian Therapeutics, Inc., a biopharmaceutical entity centered around developing groundbreaking treatments for rare and serious diseases, has divulged promising long-term results from its THRIVE phase 3 clinical trial concerning veligrotug. This intravenously administered anti-insulin-like growth factor-1 receptor (IGF-1R) antibody is targeted at active thyroid eye disease (TED), an autoimmune disorder that affects the tissues surrounding the eye, inducing inflammation, expansion, and deterioration.

The THRIVE phase 3 trial assessed the efficacy of veligrotug in active TED patients through a series of five infusions administered every three weeks, juxtaposed against a placebo group. Initial analysis was conducted at week 15, with continued observation extending to week 52. Notably, 70% of patients treated with veligrotug who exhibited a proptosis response at the 15-week mark retained their improvement by the 52nd week. The criteria for sustained response necessitated a minimum 2-millimeter reduction in proptosis from the baseline at week 52, without detrimental changes in the fellow eye, as gauged by exophthalmometry. Importantly, the safety profile of veligrotug remained unchanged through the follow-up period, with adverse events mostly resolving by week 52.

Steve Mahoney, President and CEO of Viridian, emphasized the significance of the durability and safety data, which further underlines veligrotug's robust clinical profile. Mahoney expressed confidence in veligrotug's potential to emerge as the preferred treatment for TED patients, citing the streamlined regimen of five infusions. Viridian is progressing towards a Biologics License Application (BLA) submission, scheduled for the latter half of the year, with preparations underway for a potential U.S. market launch in 2026.

Previously, veligrotug demonstrated impressive results in both the THRIVE and THRIVE-2 clinical trials, addressing active and chronic TED respectively. The trials highlighted veligrotug's rapid treatment effect and significant reduction in diplopia, a common symptom in TED patients. Particularly, THRIVE-2 marked a milestone as the first global phase 3 trial showcasing a significant response and resolution of diplopia in chronic TED patients. Combined, THRIVE and THRIVE-2 represent the most comprehensive pivotal program ever undertaken for TED.

Veligrotug, an IV-administered anti-IGF-1R antibody, is progressing through phase 3 development with the potential to become the leading IV treatment for TED patients, both active and chronic. The IGF-1R target is validated clinically and commercially, with revenues in the U.S. reaching approximately $2 billion in 2024. Veligrotug may enhance patient experience by providing a differentiated dosing regimen, featuring shorter infusion times and fewer sessions compared to existing IGF-1R inhibitors.

Viridian Therapeutics, headquartered in Waltham, Massachusetts, focuses on discovering and advancing potential best-in-class medicines for serious and rare diseases. The company leverages its expertise in antibody discovery and protein engineering to develop unique therapeutic candidates targeting validated drug markets. Apart from veligrotug, Viridian is advancing VRDN-003, a subcutaneous IGF-1R antibody for TED, through ongoing global phase 3 trials, REVEAL-1 and REVEAL-2.

Besides its TED portfolio, Viridian is developing a novel range of neonatal Fc receptor inhibitors, including VRDN-006 and VRDN-008, aimed at treating multiple autoimmune diseases. With its strategic advancements in drug development, Viridian seeks to cement its position in the TED commercial market, contingent upon regulatory approval.