Viridian Therapeutics (VRDN) Goldman Sachs 46th Annual Global Healthcare Conference summary

Portfolio progress and clinical milestones

• Advancing portfolio with positive phase 3 data in both active and chronic patient populations; BLA filing targeted for second half of the year, pending final THRIVE-2 chronic study follow-up data.

• Long-term follow-up data for the active study showed durable response; breakthrough therapy designation received, increasing odds for priority review and potentially accelerating launch timing.

• Subcutaneous (sub-Q) program in phase 3 (Reveal 1 and 2) is on track, with top-line readout expected in the first half of next year.

• FCRN portfolio progressing, with healthy volunteer data expected in Q3 and IND filing for half-life extended version by year-end.

Safety, efficacy, and differentiation

• 52-week safety data for the active study showed a favorable profile, with most adverse events mild and resolved.

• Data indicate lower hearing impairment rates compared to Tepezza, with most hearing-related events being mild and reversible.

• Five infusions and lower drug dose (70% less than Tepezza) offer a differentiated treatment profile, potentially increasing patient access.

• Durability data show 70% of patients maintained proptosis response at week 52, compared to 53% in Tepezza's label.

Market landscape and commercial strategy

• U.S. commercial launch preparations are advanced, with a full senior team and launch plans in place; priority review could accelerate launch.

• Market remains robust, with over 400 patients enrolled in trials last year and strong demand from both U.S. and international sites.

• Sub-Q formulation expected to drive further market penetration, offering at-home administration via auto-injector.

• Commercial strategy targets 2,000 core prescribers in the U.S., leveraging existing disease awareness efforts by competitors.