Viridian Therapeutics(VRDN) - 2025 Q3 - 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 veligrotug showing a 71% proptosis response rate in active TED patients [184]. - Veligrotug's phase 3 clinical trials, THRIVE and THRIVE-2, achieved all primary and secondary endpoints with statistical significance (p < 0.0001) and demonstrated a 70% durability of response at 52 weeks [185]. - 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 for TED [187]. - The company completed enrollment in the STRIVE clinical trial with 231 patients, exceeding the target of 212 due to high demand, to meet safety database requirements for veligrotug [185]. - The REVEAL-1 and REVEAL-2 trials for VRDN-003 enrolled 132 and 204 patients, respectively, surpassing their target enrollments due to demand [188]. - VRDN-006, an engineered FcRn inhibitor, demonstrated comparable potency to the current standard of care, efgartigimod, while sparing albumin and LDL levels [190]. - VRDN-008, another FcRn inhibitor, showed three times the half-life of efgartigimod and deeper IgG reductions, with a potential IND submission anticipated by the end of 2025 [191]. - The company anticipates submitting a Biologics License Application (BLA) for VRDN-003 by the end of 2026 [188]. Financial Performance - Revenue primarily consists of up-front payments for licenses, milestone payments, and research and development services under collaboration agreements [193]. - License revenue for the three months ended September 30, 2025 was $70.0 million, attributed to the collaboration and license agreement with Kissei [205]. - Research and development expenses for the three months ended September 30, 2025 increased by $17.1 million to $86.3 million compared to $69.2 million in 2024 [205]. - General and administrative expenses for the three months ended September 30, 2025 were $24.3 million, an increase of $9.9 million from $14.4 million in 2024 [210]. - For the nine months ended September 30, 2025, total research and development expenses were $249.7 million, up $83.4 million from $166.3 million in 2024 [211]. - Direct costs related to the TED portfolio increased by $60.4 million during the nine months ended September 30, 2025, primarily due to a $52.2 million increase in clinical trial costs [214]. - The collaboration revenue for the three months ended September 30, 2025 was $570,000, compared to $86,000 in 2024, reflecting an increase of $484,000 [205]. - The company received a $1.0 million milestone payment from Zenas BioPharma under the license agreement [194]. - The company is eligible to receive up to $315.0 million in non-refundable milestone payments from Kissei upon achieving specific milestones [195]. - Increase in chemistry, manufacturing, and controls costs by $8.4 million to support IND-enabling activities for the FcRn inhibitor portfolio [216]. - General and administrative expenses rose to $61.6 million for the nine months ended September 30, 2025, up from $45.5 million in the same period of 2024, marking a $16.1 million increase [217]. - Accumulated deficit reached $1,218.1 million since inception, primarily due to research and development expenses and general administrative costs [218]. - Net cash used in operating activities was $252.6 million for the nine months ended September 30, 2025, compared to $159.0 million for the same period in 2024, reflecting an increase of $93.6 million [234][235]. - Net cash provided by investing activities was $301.9 million for the nine months ended September 30, 2025, primarily from net maturities of short-term investments [236]. - Net cash provided by financing activities was $20.8 million during the nine months ended September 30, 2025, compared to $422.2 million in the same period of 2024, a decrease of $401.4 million [237][238]. - Company expects existing cash and short-term investments of $490.9 million as of September 30, 2025, to fund operations for at least twelve months [220]. - The Hercules Loan and Security Agreement was amended to increase the term loan to up to $150 million, with a maturity date of October 1, 2026 [226][227]. - In October 2025, the Company entered into a Purchase and Sale Agreement with DRI for up to $300 million in consideration [230]. - The Company sold 11,425,000 shares in a public offering at $22.00 per share in October 2025, raising significant capital [232]. Market Environment - The global macroeconomic environment remains uncertain, with potential impacts from trade disputes, geopolitical instability, and inflationary pressures [192].