Risk Factors Update Summary

• Cash, cash equivalents, and short-term investments increased from $424 million to $477 million.
• Added EMA to regulatory approvals list, increasing regulatory authority oversight. This change might result in more stringent approval processes.
• Failure to effectively compete against established treatments or new products could harm financial condition.
• Transition to large accelerated filer status and compliance with Sarbanes-Oxley Act of 2002 is time-consuming and costly.
• The company plans to initiate a pivotal program for VRDN-003 in mid-2024.
• Amgen intends to acquire Horizon Therapeutics plc, impacting competitive landscape for TED therapeutics.
• Added potential risks from global conflicts impacting capital markets and economic volatility.
• Potential product liability claims could result in up to $10 million per occurrence, up to an aggregate limit of $10 million.
• Increased competition in TED therapeutics market with more companies developing anti-FcRn therapeutics.
• Enhanced focus on cybersecurity threats and breaches, with increased sophistication and frequency.
• The company relies heavily on third-party manufacturers, which could impact product availability and costs.
• The company may not be the first to obtain marketing approval for orphan drug indications.
• Breakthrough Therapy designation may be sought for a product, with specific studies required post-approval.
• Uncertainties exist regarding the scope and protection of orphan drug exclusivity.