Viridian Therapeutics (VRDN) investor relations material

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 this indication 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; disease duration and severity were similar.

• 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

• REVEAL-2 met its primary endpoint: Q4 weekly arm achieved a 50% proptosis responder rate and Q8 weekly arm 54%, versus 15% for placebo at week 24 (p<0.0001).

• Statistically significant mean proptosis reductions were observed: 1.88 mm (Q4), 2.08 mm (Q8), vs. 0.52 mm (placebo).

• Q4 weekly arm achieved a 61% diplopia response rate and 44% complete resolution, both statistically significant compared to placebo.

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

• Significant and rapid proptosis reduction was observed from week 4 onward in both dosing arms.

Safety and tolerability

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

• Low rates of hearing impairment were observed: 4.1% (Q4) and 8.8% (Q8) placebo-adjusted; most cases were tinnitus or mild hypoacusis, with no lasting hearing loss.

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

• Three discontinuations occurred due to manageable AEs: hyperglycemia, tinnitus, and muscle spasm.

• 91% of elegrobart-treated patients completed the full course; no treatment-related serious adverse events occurred.