Viridian Therapeutics(VRDN) - 2025 Q1 - Quarterly Report

Product Development - The company is developing two product candidates for Thyroid Eye Disease (TED): veligrotug for intravenous administration and VRDN-003 for subcutaneous administration, with VRDN-003 designed for at-home self-administration[170][176]. - In phase 1/2 clinical trials, veligrotug showed that 71% of active TED patients were proptosis responders and 54% had complete resolution of diplopia after two infusions[171]. - The THRIVE study for veligrotug enrolled 113 patients, achieving all primary and secondary endpoints with statistical significance (p < 0.0001) and a rapid treatment effect observed in 53% of patients within three weeks[172]. - The company plans to submit a Biologics License Application (BLA) for veligrotug in the second half of 2025 and for VRDN-003 by the end of 2026[172][176]. - VRDN-003 has a prolonged half-life of 40 to 50 days, which is four to five times that of veligrotug, and is currently in pivotal clinical studies[174][176]. - The company is also developing engineered FcRn inhibitors, including VRDN-006 and VRDN-008, targeting a significant commercial market opportunity in autoimmune diseases[177]. - VRDN-006 demonstrated comparable potency to the current standard of care in FcRn inhibition, efgartigimod, while maintaining a similar safety profile[178]. - VRDN-008 showed three times the half-life of efgartigimod and deeper IgG reductions, with ongoing studies to generate additional data[179]. Financial Performance - Collaboration revenue for the three months ended March 31, 2025, was $72,000, unchanged from the same period in 2024[193]. - Research and development expenses increased by $35.9 million, reaching $76.8 million for the three months ended March 31, 2025, compared to $40.9 million in 2024[193]. - General and administrative expenses rose to $17.1 million for the three months ended March 31, 2025, up from $15.0 million in 2024, reflecting a $2.1 million increase[200]. - Other income, net decreased to $7.0 million for the three months ended March 31, 2025, compared to $7.4 million in 2024, primarily due to changes in interest income[200]. - The accumulated deficit reached $1,082.8 million as of March 31, 2025, primarily due to research and development expenses and general administrative costs[202]. - The company anticipates a $22.9 million increase in clinical trial costs and a $4.0 million increase in chemistry, manufacturing, and controls costs for ongoing global phase 3 clinical trials[199]. - Personnel-related costs increased by $3.1 million, driven by headcount growth to support research and development efforts[199]. - The company expects research and development expenses to continue to rise as clinical and nonclinical programs advance[198]. Cash Flow and Financing - The company had $636.6 million in cash, cash equivalents, and short-term investments as of March 31, 2025, expected to fund operations into the second half of 2027[201]. - Net cash used in operating activities was $92.7 million for the three months ended March 31, 2025, primarily due to a net loss of $86.9 million[219]. - Net cash provided by investing activities was $100.1 million during the three months ended March 31, 2025, mainly from net maturities of short-term investments[221]. - Net cash provided by financing activities was $9.2 million during the three months ended March 31, 2025, including proceeds from the September 2022 ATM Agreement[222]. - The Company sold 3,058,751 shares under the September 2022 ATM Agreement at a weighted average price of $22.86 per share, generating approximately $67.7 million in net proceeds[217]. - The January 2024 Public Offering raised approximately $150 million from the sale of 7,142,858 shares at $21.00 per share[216]. - The Company reported a net increase in cash and cash equivalents of $16.6 million for the three months ended March 31, 2025, compared to a decrease of $11.2 million in the same period of 2024[217]. - The Company is required to pay an end-of-term fee equal to 6% of the principal amount of funded Amended Term Loan advances at maturity[215]. - The Hercules Loan and Security Agreement provides access to a term loan of up to $75.0 million, with an initial tranche of $25.0 million drawn[210]. - The Company has access to an increased term loan of up to $150 million, structured in four tranches, with the first tranche of $50 million already partially drawn[212]. - The Amended Term Loan bears an interest rate of 8.95% as of March 31, 2025, with interest payable monthly[213]. Market Environment - The global macroeconomic environment remains uncertain, potentially impacting future operations due to various geopolitical and economic factors[180]. - There were no material changes to market risks in the three months ended March 31, 2025, compared to the previous disclosures[224].