Viridian Therapeutics (VRDN) Study result summary

Study design and patient population

• REVEAL-1 was a phase 3, randomized, controlled, double-masked trial evaluating subcutaneous elegrobart in active thyroid eye disease (TED), enrolling 132 patients across Q4W, Q8W, and placebo arms, with balanced baseline characteristics and a 24-week primary endpoint.

• Key inclusion criteria included CAS ≥3, proptosis ≥3 mm, and TED symptom onset within 15 months.

• Patients received a loading dose followed by either five (Q4) or two (Q8) single injection doses; placebo matched accordingly.

• Baseline characteristics were well balanced between arms, with most patients being female and average age around 50.

• The study included follow-up through week 52.

Efficacy results

• REVEAL-1 met its primary endpoint: Q4W and Q8W arms achieved proptosis responder rates of 54% and 63% at week 24, versus 18% for placebo, both highly statistically significant.

• Q4W arm showed 51% complete diplopia resolution at week 24 vs. 16% for placebo; Q8W arm showed 28% complete resolution.

• Rapid onset of effect was observed, with significant proptosis response as early as week 4 after one dose.

• MRI-based proptosis endpoints were consistent with exophthalmometer results and significant at all measured time points.

• Early and sustained separation from placebo was observed for both proptosis and diplopia endpoints.

Safety and tolerability

• Elegrobart was generally well tolerated, with most adverse events mild and consistent with the anti-IGF-1R class.

• Hearing impairment rates were low (11.3% Q4W, 2.3% Q8W placebo-adjusted), consisting only of tinnitus without hearing loss.

• Injection site reactions were mostly grade one and did not lead to discontinuations.

• Serious adverse events were rare and mostly unrelated to treatment.

• Muscle spasms, injection site reactions, and headache were among the most common adverse events.