Viridian Therapeutics (VRDN) Leerink’s Global Healthcare Conference 2025 summary

Key clinical achievements and data

• Two large phase 3 studies (THRIVE and THRIVE-2) for TED showed robust, consistent efficacy and rapid onset of action, with significant improvements in proptosis and diplopia resolution across all endpoints and time points.

• Safety profile was favorable, with low rates of mild hearing impairment and very low dropout rates, supporting a strong risk-benefit balance.

• Chronic TED study included a broader, more representative patient population, aiming for label inclusion and payer discussions, unlike the current competitor.

• Durability of response is expected to be similar to existing therapies, with ongoing follow-up to 52 weeks and data to be included in the BLA or presented at medical congresses.

• STRIVE safety study completed in early January, supplementing the safety database for BLA submission.

Regulatory and development milestones

• On track for a BLA filing in the second half of 2024, driven by completion and data lock of the chronic study follow-up period.

• Two phase 3 subcutaneous (subQ) studies (REVEAL and REVEAL-2) are enrolling, with top-line data expected in the first half of 2026.

• SubQ program uses a half-life extension technology, enabling more convenient dosing and potentially improved safety.

• FcRn franchise in clinical development, with first human data expected in Q3 2024 and an IND for VRDN-008 planned by year-end.

Commercial strategy and market dynamics

• IV product offers rapid onset, fewer infusions, shorter infusion times, and 70% less drug compared to the current therapy, targeting new start patients and flares in chronic disease.

• SubQ product to launch in a 2 mL autoinjector pen, aiming for 70%+ market share over time due to convenience and home administration.

• Market penetration for current therapy remains low, with significant opportunity for growth, especially in chronic and ex-U.S. markets.

• Chronic data in the label is expected to provide a commercial advantage in payer and physician discussions.

• Infrastructure and execution from IV launch will support rapid subQ uptake.