Viridian Therapeutics, Inc. Q4 FY2022 earnings call

• Scott Myers joined as CEO about a month prior and discussed his background in pharma. He highlighted progress in 2022, including positive data from VRDN-001 Phase I and II trials. - VRDN-001 IV program had positive top-line data from 3 dose cohorts, with initial results from chronic TED proof-of-concept study expected in Q2 2023. - Phase III THRIVE trial initiated in Dec 2022 with first patient enrolled, top-line results expected mid-2024. THRIVE-2 planned to start mid-2023 with results end-2024. - Subcutaneous programs: VRDN-001 Phase I in healthy volunteers expected Q4 2023, VRDN-002 proof-of-concept trial in active TED expected by end-2023, VRDN-003 advancing to IND in Q2 2023 with Phase I in healthy volunteers expected Q4 2023. - Plan to select lead subcu program by end-2023, with pivotal trial planned mid-2024. - Early-stage preclinical pipeline to expand into rare and autoimmune diseases with multiple programs like VRDN-004, 005, 006.

As of the end of the fourth quarter and full year 2022, Viridian had approximately $424.6 million in cash, cash equivalents and short-term investments compared with $431.3 million as of September 30, 2022. Research and development expenses were $39.3 million during the fourth quarter of 2022 compared with $22.4 million for the same period last year. For the 12 months ended December 31, 2022, research and development expenses were $100.9 million compared with $56.9 million for the same period last year. As of March 1, 2023, Viridian had approximately 57.7 million shares of common stock outstanding on an as-converted basis.

• Expect initial results from chronic TED proof-of-concept study in Q2 2023. - Phase III THRIVE trial top-line results expected mid-2024. - THRIVE-2 planned to start mid-2023 with results end-2024. - VRDN-001 Phase I subcu in healthy volunteers expected Q4 2023. - VRDN-002 proof-of-concept trial in active TED expected by end-2023. - VRDN-003 advancing to IND in Q2 2023 with Phase I in healthy volunteers expected Q4 2023. - Plan to select lead subcu program by end-2023, with pivotal trial mid-2024.

• Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ, as described in recent Form 10-Q and 10-K with the SEC. - Uncertainties related to clinical trial outcomes, regulatory approvals, and market acceptance of products.

Q: What's the reason to test 001 subcu in healthy volunteers? And details on 002 subcutaneous proof-of-concept trial?

A: On 001 subcu in healthy volunteers, it's to look at immunogenicity and bioavailability like with 003. On 002, no more details spoken yet.

Q: Expectations for chronic TED proof-of-concept study results and how efficacy vs active TED? And impact of competitor data?

A: In chronic TED, expecting some impact on proptosis but lower than active TED. Horizon's data midyear may help shape clinical development strategy.

Q: Rationale for dose selection for second chronic type cohort at 3 mg and how to select subcu assets?

A: Selected 3 mg dose as saw good results in 3 mg per kg cohort in Jan. Will use multiple data like healthy volunteer data to select subcu program.

Q: Attractive markets for non-TED assets?

A: Look for great markets with limited competition, differentiate based on efficacy, safety, or delivery convenience, then bring to market with commercialization efforts.

Q: Excitement about Viridian's opportunity and most compelling program? Cash runway guidance?

A: Excited about multiple programs, pipeline, team, and being well-capitalized. Cash runway is baked into second half of 2025, funds THRIVE, THRIVE-2, subcu programs, and non-TED program unveiling. Operating expenses to increase as moving closer to commercialization.

Q: Update on subcu pen device?

A: Looking at auto-injector devices, early stages of assessment, to select and integrate into subcu program following subcu bake off later this year.

Q: Inclusion criteria of chronic TED study and comparison to TEPEZZA Phase IV?

A: Chronic study inclusion: CAS 0-7, proptosis ≥3 mm, onset >12 months. TEPEZZA Phase IV: >15 months, CAS 0-1.

Q: Dosing frequency and number of infusions for 001 subcu formulation? Inclusion of CAS scores in THRIVE-2?

A: Subcu dosing regimens not disclosed. THRIVE-2 for 001 will mirror proof-of-concept Phase II design, inclusive of CAS but chronic patients tend to have lower CAS.

Q: Strategic value of advancing 2 assets for chronic vs active TED?

A: Difficult to have 2 assets in same market; shorter course of therapy for active TED beneficial. Subcu strategy in chronic allows home administration, beneficial for patients.

Q: Initial proof-of-concept results for 001 in chronic TED patients, number of patients, etc.?

A: 16 patients over 2 cohorts, 10 mg per kg, q3 x 2, placebo arm in 10 mg and 3 mg per kg. Expect to see effect in proptosis, diplopia, CAS scores.

Q: Phase III THRIVE site openings, target mix, effect of TEPEZZA on enrollment? Week-12 data for 001 proof-of-concept trial?

A: Target 50 clinical sites in NA and Europe, 31 sites opened, majority in US. No enrollment guidance. No week-12 data expected for other cohorts as patients not dosed longer after week 21.