Viridian Therapeutics (VRDN) Study result summary

Study design and patient population

• REVEAL-2 was a phase 3, randomized, controlled, double-masked trial enrolling 204 patients with chronic Thyroid Eye Disease (TED), the largest pivotal trial in TED to date.

• Patients were randomized to Q4 weekly, Q8 weekly, or placebo arms, with broad inclusion criteria for clinical activity score (CAS).

• Baseline characteristics were balanced across arms, with most patients being female and average age just over 50.

• The trial aimed to capture a wide spectrum of chronic TED, including low and high CAS subgroups.

• High completion rates were observed in all arms.

Efficacy results

• Both Q4W and Q8W regimens achieved highly statistically significant proptosis responder rates at week 24: 50% (Q4W) and 54% (Q8W) vs. 15% for placebo (p<0.0001).

• Q4W arm achieved a 61% diplopia response rate and 44% complete resolution, both statistically significant compared to placebo.

• Mean proptosis reduction was -1.9 mm (Q4W) and -2.1 mm (Q8W) compared to -0.5 mm for placebo.

• Efficacy was consistent across low and high CAS subgroups, supporting broad applicability.

• Separation from placebo was observed as early as week 4 and deepened through week 24.

Safety and tolerability

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

• Low rates of hearing impairment were observed: 4.1% (Q4W) and 8.8% (Q8W) placebo-adjusted, mostly tinnitus; mild hypoacusis and eustachian tube disorder were rare and resolved.

• Injection site reactions were mostly grade one and more frequent in placebo than treatment arms; none led to discontinuation.

• Most common AEs included muscle spasms, injection site reactions, and hyperglycemia.

• 91% of elegrobart-treated patients completed the full course; three discontinuations occurred due to manageable AEs.