Welcome to 2025 and Syenza News, where the first two weeks of January are dedicated to Thyroid Awareness. This period focuses on highlighting advancements in thyroid care, especially chronic thyroid eye disease (TED) and the experimental treatment veligrotug (for thyroid eye disease therapy). Discussions will cover new technologies, their economic impact, outcomes research, and access throughout these two weeks.

Analysis of Viridian Therapeutics’ Announcement on Veligrotug

Viridian Therapeutics recently announced positive results from their Phase 3 THRIVE-2 clinical trial of veligrotug for chronic thyroid eye disease.

Key Findings of the THRIVE-2 Trial

Trial Design and Enrollment: The THRIVE-2 trial is a randomized, double-masked, placebo-controlled study with 188 patients. 125 received veligrotug and 63 received a placebo, with treatments administered three weeks apart.

Efficacy Outcomes: The trial successfully met all primary and secondary endpoints at the 15-week primary analysis timepoint. Veligrotug demonstrated statistically significant improvements in key disease endpoints, including:
– Proptosis: Patients treated with veligrotug achieved a Proptosis Responder Rate (PRR) of 56%. Just 8% of patients on placebo achieved the outcome. The mean reduction in proptosis was 2.34mm from baseline in the veligrotug group, versus 0.46mm in the placebo group.
– Diplopia: Among patients treated with veligrotug, 56% showed a response in diplopia. 32% achieving complete resolution compared to 25% and 14% in the placebo group, respectively.
– Clinical Activity Score (CAS): 54% of veligrotug patients achieved a maximal or near-maximal therapeutic effect on CAS. Researchers defined a maximal or near-maximal therapeutic effect on CAS as reaching a score of 0 or 1, contrasting it with 24% of patients in the placebo group. The mean reduction in CAS was 2.9 from baseline for veligrotug patients, compared to 1.3 for placebo patients.

Safety Profile: Researchers found that veligrotug was well-tolerated and matched the safety profile of earlier studies. There was a low incidence of hearing impairment – 12.8% vs. 3.2% in the placebo group.

Implications and Future Plans

Treatment Potential: The promising results from the THRIVE trials suggest veligrotug could become the preferred treatment for all types of active and chronic TED.

Future Clinical Trials: Viridian Therapeutics is running two global Phase 3 trials, REVEAL-1 and REVEAL-2, for VRDN-003, a new veligrotug formulation. Viridian Therapeutics expects topline results from the Phase 3 trials by mid-2026 and plans to submit a Biologics License Application by late 2026.

Conclusion

THRIVE-2 trial results show significant progress in treating chronic thyroid eye disease, offering new hope and emphasizing the importance of continued research in thyroid care.