Viridian Therapeutics (VRDN) investor relations material

Portfolio overview and strategy

• Focus on de-risked science targeting large, established markets, including IGF-1R for thyroid eye disease (TED) and FcRn for multiple indications.

• TED market in the U.S. is valued at $2 billion annually, with significant growth potential through two programs: IV (Veligrotug) and subcutaneous (subQ).

• Veligrotug IV program completed phase 3 trials, received breakthrough therapy designation, and BLA was filed in October, aiming for mid-2026 commercial launch pending priority review.

• SubQ program completed pivotal study enrollment, offers at-home administration via autoinjector, and is expected to follow IV launch by about a year.

• Robust clinical enrollment with over 500 patients in TED trials this year, exceeding timelines and U.S. representation.

Market expansion and competitive positioning

• TED is a new start market with 20,000 incident cases annually in the U.S. and 200,000 moderate to severe prevalent cases targeted.

• Current market penetration is in single digits, leaving room for significant expansion.

• Veligrotug offers a shorter 12-week treatment, lower dosage, faster infusion, and strong efficacy and safety data from the largest TED studies to date.

• SubQ program expected to further expand the market by enabling self-administration.

Regulatory and launch preparation

• FDA division of ophthalmology has remained stable and supportive, facilitating productive interactions and BLA submission.

• Breakthrough therapy designation supports eligibility for priority review; decision expected by year-end, potentially enabling a mid-2026 launch.

• Launch strategy benefits from prior market education and infrastructure established by the first approved product, with improved insurance coverage and reduced access barriers.