Viridian Therapeutics, Inc. Q2 FY2022 earnings call

• VRDN-001 trial: First 10mg/kg cohort had 83% overall responders, 83% proptosis responders, 83% achieved CAS 0 or 1, 75% with baseline diplopia resolved; favorable safety profile with no serious adverse events. - Mechanism: VRDN-001 is full antagonist of IGF-1R, unlike Tepezza and others as partial antagonists. - VRDN-002: Initial data shows half-life 30-40 days in healthy volunteers, favorable safety profile. - Phase 3: THRIVE program to initiate by year end, evaluating VRDN-001 in active and chronic TED with 5-infusion course vs Tepezza's 8-infusion. - Subcutaneous: VRDN-002 and VRDN-003 in development for subcutaneous formulations.

No specific product segment financial performance with revenue contribution % provided. Focus is on clinical data of VRDN-001 and VRDN-002. VRDN-001 trial results showed 83% overall responders, 83% proptosis responders, etc. VRDN-002 initial data shows extended half-life and favorable safety profile.

• Accelerate VRDN-001 to Phase 3, THRIVE program initiated by year end. - VRDN-002 proof-of-concept study to initiate by year end, top-line data in second half of next year. - Plans to develop best-in-class IV and subcutaneous therapies for TED.

• Transient adverse events like tinnitus, muscle spasms (reversible). - Uncertainties in retreatment and pricing. - Regulatory and competitive risks.

Q: Chris Howerton asked about hearing loss observation and subcutaneous strategy.

A: Barrett Katz and Raymond Douglas commented on transient tinnitus resolving; Jonathan Violin discussed subcutaneous strategy with VRDN-002 and 003 in parallel.

Q: Thomas Smith asked about FDA engagement, enrollment, and cost of Phase 3 program.

A: Jonathan Violin said they'll meet with FDA, enrollment on track with robust sites, costs not detailed but well capitalized.

Q: Gavin Clark-Gartner asked about 20mg/kg 001 safety data and retreatment.

A: Jonathan Violin said 20mg/kg safety data ongoing, retreatment important but early.

Q: Rami Katkhuda asked about MRI vs exophthalmometer and IGF-1 levels.

A: Barrett Katz and Jonathan Violin discussed MRI supplementing exophthalmometry, more complete antagonism of 001 leading to higher IGF-1 levels.

Q: Laura Chico asked about proptosis evaluation and ex-U.S. commercialization.

A: Raymond Douglas talked about MRI use, Jonathan Violin said too early for ex-U.S. commercial details.

Q: Kalpit Patel asked about registrational study design and 001 dose.

A: Jonathan Violin explained 5-infusion design based on data, 10mg/kg in THRIVE program.

Q: Jason Butler asked about chronic trial and retreatment.

A: Jonathan Violin said chronic trial has specific baseline characteristics, no prior Tepezza treatment allowed.

Q: Trevor Allred asked about safety profile of full antagonist 001.

A: Jonathan Violin said safety profile great with over 20 people exposed showing no issues.

Q: Michael Higgins asked about methotrexate use.

A: Barrett Katz said inclusion criteria disallow recent methotrexate use.