Viridian Therapeutics, Inc. Q1 FY2022 earnings call

Clinical Progress

• VRDN-001: Phase I/II proof-of-concept trial for Thyroid Eye Disease (TED) ongoing; healthy volunteer data shows promising safety and robust IGF-1 plasma level increases; expects top line proof-of-concept data from TED patient cohorts in Q3 and data from 3 mg per kg cohort in Q4.
• VRDN-002: First-in-human Phase I trial initiated in March, dose escalation complete; top line data expected in Q3, aiming to show pharmacokinetic benefits of half-life extension for subcutaneous dosing.
• Pipeline Beyond TED: Expansion into discovery-stage programs, but laser-focused on TED portfolio.

Financials

• Ended Q1 2022 with $175 million in cash, cash equivalents, and short-term investments.
• R&D expenses were $17.7 million in Q1 2022 (up from $13.8 million in Q1 2021) due to personnel, license fees, and clinical trial costs for VRDN-001 and VRDN-002.
• General and administrative expenses were $8.4 million in Q1 2022 (up from $6.2 million in Q1 2021) due to personnel-related costs.
• Net loss was $25.7 million in Q1 2022 (wider than $18.5 million in Q1 2021) due to increased operating costs and lower collaboration revenue.

No specific product segment financial performance breakdown provided; focus is on clinical programs and financials.

Forward-Looking

• VRDN-001 Phase I/II proof-of-concept trial for TED patients to deliver top line data in Q3, with data from the 3 mg per kg cohort expected in Q4.
• VRDN-002 Phase I first-in-human trial to announce top line data in Q3.
• Plan to prepare VRDN-001 for pivotal trials assuming positive proof-of-concept data.

Risks

• Forward-looking statements subject to risks and uncertainties in clinical trials, regulatory approvals, and market competition. Factors discussed in the company's most recent SEC filings could cause actual results to differ materially from forward-looking statements.

Q: Chris Howerton with Jefferies asked about clarification on healthy volunteers data error bars and dosing equivalence between 001 and 002.

A: Jonathan Violin responded that the interim analysis for IGF-1 is ongoing with the top dose cohort, and VRDN-002 has half-life extension for better pharmacokinetics differing from 001's similar pharmacokinetics to teprotumumab.

Q: Thomas Smith with SVB Leerink inquired about safety of healthy volunteers data and enrollment in TED cohorts.

A: Jonathan Violin stated safety was clean with mild events deemed unrelated, and enrollment is progressing with 11 active sites mimicking TEPEZZA Phase III trial sites.

Q: Laura Chico with Wedbush asked about timing of 002 data and PK of 001.

A: Jonathan Violin clarified 002 data is expected in Q3, and 001 PK is consistent with oncology data with no target-mediated clearance.

Q: Rami Katkhuda with LifeSci Capital asked about correlation of IGF-1 biomarker with TED efficacy and dosing frequency for subcu.

A: Jonathan Violin explained IGF-1 biomarker is a target engagement marker, and dosing frequency for subcu depends on future data with a trade-off between volume and frequency.

Q: Jason Butler with JMP Securities inquired about timing and formulation of 001 subcu.

A: Jonathan Violin stated formulation to 150 mg/ml achieved with no hurdles, and 3 mg per kg cohort data will guide subcu development.