Viridian Therapeutics (VRDN) Jefferies 2024 Global Healthcare Conference summary

Lead program and phase III outlook

• Top-line phase III data for the IV program (VRDN-001) is expected in September, with high confidence based on prior phase II results and targeting a clinically validated pathway (IGF-1R).

• Phase II data showed robust efficacy, including an 83% proptosis response rate after two infusions, and improvements in pain and double vision, suggesting a strong quality-of-life impact.

• The IV regimen uses a lower dose and fewer infusions (10 mg/kg, five infusions, 30-minute duration) compared to the current standard, potentially reducing patient burden.

• Efficacy is expected to be similar to or better than the current standard, with most benefit achieved by the fifth infusion.

• Safety profile in phase II was favorable, with minimal hearing loss and adverse events resolving without intervention.

Subcutaneous (Sub-Q) program development

• Plans are underway to start a pivotal phase III Sub-Q program, following positive PK/PD data showing extended half-life and strong target engagement.

• Sub-Q dosing regimens under consideration include every two, four, or eight weeks, with trial design details to be announced soon.

• The Sub-Q program aims to match IV exposure levels, offering flexibility and potential for greater patient convenience.

• Two well-controlled studies are planned for Sub-Q, with at least one active arm per study to optimize dosing strategies.

• Overlapping PK/PD profiles between IV and Sub-Q are expected to de-risk the Sub-Q program.

Market dynamics and economic considerations

• The market is driven by new patient starts, with treatment based on symptom presentation rather than diagnosis timing.

• Ophthalmologists and oculoplastic surgeons typically refer patients to external infusion centers, so there is no financial disincentive for adopting Sub-Q formulations.

• The lack of in-house infusion incentives differentiates this market from others like oncology or rheumatology.

• Sub-Q formulations could enhance patient convenience and market adoption due to these dynamics.

• Upcoming data and program updates are expected later in the summer.