Viridian Therapeutics (VRDN) Evercore ISI 8th Annual HealthCONx Conference summary

Key clinical program updates

• Achieved strong results across proptosis, diplopia, and clinical activity score endpoints, leading to Breakthrough Therapy Designation submission.

• Rapid treatment effect observed after one infusion, with majority of patients responding within three weeks.

• Subcutaneous program uses the same antibody as IV, with half-life extension for at-home self-administration; top-line data expected in Q1 and Q2 for active and chronic populations.

• Both IV and subcutaneous programs target a $2B U.S. market, with plans for European MAA filing in Q1 and recent partnership in Japan.

• Placebo response rates have been consistently low in large studies, with robust operational consistency across trials.

Product differentiation and market strategy

• Subcutaneous formulation offers at-home administration via auto-injector, increasing patient convenience and access.

• Treatment regimen is shorter (three months, five infusions) and uses 70% less drug compared to the current standard.

• No need to switch patients from existing products; new option available at all stages post-diagnosis.

• Commercial strategy leverages pricing insights from existing products in Europe and Japan.

• Focus on validated targets and differentiated profiles in large markets, with additional FcRn programs in development.

Clinical trial design and expectations

• REVEAL-1 (active) and REVEAL-2 (chronic) studies mirror successful THRIVE designs, enrolling broad and representative patient populations.

• Subcutaneous 003 is de-risked, matching or exceeding IV exposure levels; efficacy bar set by TEPEZZA's 50%-70% placebo-adjusted proptosis response.

• Placebo arms expected to remain low based on prior trial consistency; operational rigor maintained across studies.

• Safety profile for IGF-1R is well known and manageable; subcutaneous formulation may offer improved safety due to lower Cmax and overall exposure.

• Launch of veligrotug anticipated next year, with subcutaneous version following closely; no specific launch guidance provided yet.