Viridian Therapeutics (VRDN) Study result summary

Study design and patient population

• REVEAL-1 was a phase 3, randomized, controlled, double-masked trial enrolling 132 patients with active thyroid eye disease, randomized equally to Q4 weekly, Q8 weekly, or placebo arms.

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

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

• Baseline characteristics were balanced, with most patients female, average age around 50, and similar disease severity across arms.

• The study included a 24-week primary endpoint and follow-up through week 52.

Efficacy results

• REVEAL-1 met its primary endpoint: Q4 weekly arm achieved a 54% proptosis responder rate at week 24 vs. 18% for placebo; Q8 weekly arm achieved 63%.

• Q4 weekly arm showed 51% complete diplopia resolution at week 24 vs. 16% for placebo; Q8 weekly arm achieved 28%.

• Both Q4 and Q8 arms showed rapid onset of effect, with significant proptosis response after one dose and deepening effect through week 24.

• MRI and exophthalmometer measurements confirmed robust and consistent proptosis responses at all measured time points.

• Significant improvements were seen in mean proptosis reduction and overall responder rates for both dosing regimens.

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% Q4, 2.3% Q8, placebo-adjusted); all events were tinnitus, none led to discontinuation or hearing loss.

• Injection site reactions and muscle spasms were mostly mild and occurred early; no dosing interruptions or discontinuations.

• Serious adverse events were rare and mostly unrelated to treatment; only two discontinuations due to TEAEs.