Viridian Therapeutics (VRDN) Goldman Sachs 45th Annual Global Healthcare Conference summary

Key announcements and program updates

• Announced positive Type C meeting and finalized Phase 3 program design for VRDN-003 (REVEAL), targeting both active and chronic thyroid eye disease (TED), with simultaneous trial initiation in August and top-line data expected in early 2026, followed by a BLA filing at the end of 2026.

• VRDN-003 is a half-life extended, subcutaneous IGF-1R antibody, with two dosing regimens (every 4 and 8 weeks) and an at-home auto-injector planned for launch, aiming for best-in-class convenience and efficacy.

• Phase 3 studies are powered to address placebo effect noise seen in earlier trials, and both MRI and EXO measurements will be used for proptosis response.

• The company is also advancing an FcRn portfolio, with VRDN-006 (Fc fragment) IND expected by year-end and VRDN-008 (half-life extension) NHP data in the second half of the year.

• Commercial preparations are ahead of schedule, with a strong internal team and flexibility regarding potential partnerships.

Market landscape and differentiation

• U.S. TED market remains robust at $1.8–2 billion, with low current penetration and significant room for growth, especially with subcutaneous options improving access and convenience.

• VRDN-001 IV is positioned to compete with TEPEZZA by offering a shorter, five-dose regimen versus eight, with similar or better efficacy and safety, and a faster infusion time.

• Subcutaneous VRDN-003 is expected to capture over 70% of the market due to its convenience, but IV will remain relevant for certain patients and providers.

• The company expects to file in Europe and Japan, mirroring competitors' global expansion strategies.

• Both active and chronic TED populations are seen as high unmet need, with chronic patients particularly motivated to treat symptom flares.

Clinical development strategy and future outlook

• VRDN-003 moves directly to Phase 3 based on shared binding domain and PK/PD modeling with VRDN-001, bypassing traditional Phase 2 in patients.

• The auto-injector device is commercially available and will be integrated into the program through standard bridging studies, with launch planned in the auto-injector format.

• The company is building a broad autoimmune portfolio, aiming for multiple approved and commercial products over the next decade.

• Long-term, the company envisions a leading position in autoimmune therapeutics, leveraging clinically validated targets and a differentiated product suite.

• Ongoing commercial readiness and flexible partnership approach position the company for strong market entry and growth.