Viridian Therapeutics (VRDN) investor relations material

Phase I findings

• Two 3,000 mg SC doses at Weeks 0 and 4 in healthy volunteers (n=6) produced a 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.

• Modeling showed 3,000 mg Q8W dosing could sustain IgG reductions above first-generation Fc-fragment peak reductions; Q4W dosing modeled deeper reductions.

• Generally well tolerated, with albumin- and LDL-sparing effects and no serious adverse events or dose-limiting toxicities; nearly all events were grade 1, and one grade 3 CPK/AST elevation was deemed unrelated.

Registrational development

• At least one registrational trial is planned for 2027, starting with MG in mid-2027, pending regulatory feedback.

• Proposed MG trial: 1:1:1 randomization to SC Q8W with a Week 4 loading dose, SC Q4W, or placebo; MG-ADL is the primary endpoint.

• The 3,000 mg dose is planned for registrational studies; the MG program will use continuous dosing, with direct progression from Phase I subject to regulatory feedback.

• VRDN-008 is prioritized over VRDN-006; funding is expected to support at least one 2027 registrational trial alongside ongoing programs.

• Planned development includes Graves’ disease, CIDP, myositis and rheumatoid arthritis, in addition to MG.

Subcutaneous delivery and opportunity

• Pivotal development will use the enFuse device; the 3,000 mg dose is 20 mL at 150 mg/mL, with expected administration time of ~30 minutes.

• enFuse is designed for hands-free, at-home self-administration; Q8W dosing would mean seven SC administrations per year, and the device is used with multiple FDA-approved medicines.

• Exclusive global enFuse rights for FcRn-targeted therapies have been held since January 2023; Paragon and Enable agreements include development and commercial milestones and single-digit royalties.

• FcRn inhibitors were projected to generate over $7B in 2026 revenue, up 40% over the prior 12 months, with the market projected to approach $12B by 2030; the potential addressable market across autoimmune diseases was estimated at $50bn.