Viridian Therapeutics (VRDN) Study update summary

Phase 1 findings

• Two 3,000 mg SC doses at Weeks 0 and 4 in healthy volunteers (n=6) produced 74% peak IgG reduction, sustained through 8 weeks after dosing.

• VRDN-008 had a 15–18-day half-life, up to 6x longer than first-generation Fc fragments.

• Generally well tolerated, with albumin- and LDL-sparing profile; no serious adverse events or dose-limiting toxicities.

• Clinical results and modeling support potential Q8W dosing and a differentiated FcRn-inhibitor profile.

Planned registrational development

• An MG registrational trial is planned to start in mid-2027, pending regulatory feedback; at least one registrational trial is planned for 2027.

• Proposed placebo-controlled, 1:1:1 trial will compare SC Q8W with a Week 4 loading dose, SC Q4W and placebo; primary endpoint MG-ADL.

• Trial is designed to support potential approval as the first Q8W FcRn inhibitor in MG and a direct-to-registrational path.

• Development is planned across MG, Graves’ disease, CIDP, myositis and rheumatoid arthritis.

Product profile, delivery and opportunity

• VRDN-008 is a bifunctional, extended half-life FcRn inhibitor with potential Q8W SC dosing and at-home self-administration via the enFuse device.

• Exclusive global rights cover enFuse use with FcRn-targeted therapies; the hands-free wearable on-body injector is designed for at-home use and has 20 mL capacity.

• enFuse is in use with multiple FDA-approved medicines and in development as a delivery system for other drugs.

• Potential addressable market across multiple autoimmune diseases was estimated at $50bn; FcRn inhibitors in MG had 40% LTM growth, with FcRn inhibition clinically validated in MG.