Clinical Trials and Drug Development - Reported positive topline phase 3 data for veligrotug in patients with active thyroid eye disease (TED), achieving all primary and secondary endpoints with high statistical significance (p < 0.0001) after five infusions[4] - THRIVE-2 trial completed enrollment with 188 patients, exceeding the target of 159, and topline data expected in December 2024[4] - VRDN-003, a next-generation subcutaneous IGF-1R inhibitor, has two global phase 3 trials (REVEAL-1 and REVEAL-2) initiated in August 2024, with topline data expected in the first half of 2026[6] - VRDN-006 is on track for IND submission by year-end 2024, with proof-of-concept IgG reduction data anticipated in the second half of 2025[7] - VRDN-008 demonstrated a threefold longer half-life compared to efgartigimod in non-human primate studies, with deeper IgG reductions[8] - Biologics License Application (BLA) for veligrotug is anticipated in the second half of 2025, pending data[5] Financial Performance - Net loss for Q3 2024 was $76.7 million, compared to $47.7 million for the same period last year[10] - Total revenue for the three months ended September 30, 2024, was $86,000, compared to $72,000 for the same period in 2023, representing a 19.4% increase[15] - Total operating expenses for the three months ended September 30, 2024, were $83,566,000, up from $51,296,000 in the prior year, reflecting a 62.8% increase[15] - The net loss for the three months ended September 30, 2024, was $76,689,000, compared to a net loss of $47,660,000 for the same period in 2023, indicating a 60.9% increase in losses[15] - The company reported a comprehensive loss of $75,214,000 for the three months ended September 30, 2024, compared to a comprehensive loss of $47,551,000 for the same period in 2023, reflecting a 58.4% increase[15] - The net loss per share, basic and diluted, was $(1.15) for the three months ended September 30, 2024, compared to $(1.09) for the same period in 2023[15] Expenses and Investments - Research and development expenses increased to $69.2 million for Q3 2024, compared to $30.4 million for Q3 2023, driven by higher clinical trial costs[10] - General and administrative expenses decreased to $14.4 million for Q3 2024, down from $20.9 million for Q3 2023, due to reduced personnel-related costs[10] - Research and development expenses for the three months ended September 30, 2024, were $69,158,000, significantly higher than $30,385,000 in the prior year, indicating a 128.3% increase[15] Cash and Assets - Cash, cash equivalents, and short-term investments totaled $753.2 million as of September 30, 2024, up from $571.4 million as of June 30, 2024, following a public offering that generated net proceeds of $243.2 million[10] - Cash, cash equivalents, and short-term investments increased to $753,240,000 as of September 30, 2024, from $477,370,000 at the end of 2023, showing a 57.6% growth[16] - Total assets rose to $771,900,000 as of September 30, 2024, compared to $490,424,000 at the end of 2023, marking a 57.4% increase[16] - Total stockholders' equity increased to $707,496,000 as of September 30, 2024, up from $442,022,000 at the end of 2023, representing a 60.1% rise[16] Shareholder Information - The weighted-average shares used to compute basic and diluted loss per share increased to 66,420,063 for the three months ended September 30, 2024, from 43,654,577 in the same period of 2023[15]

Core Viewpoint - Viridian Therapeutics, Inc. announced positive results from the phase III THRIVE study for its lead drug veligrotug, leading to a 32.1% surge in share price on September 10 [1]. Study Results - The THRIVE study met all primary and secondary endpoints, showing statistically significant improvements in thyroid eye disease (TED) symptoms after 15 weeks of treatment with veligrotug [2]. - Patients receiving veligrotug had a 70% proptosis responder rate compared to 5% for the placebo group, with a mean reduction in proptosis of 2.9 mm versus 0.5 mm [3]. - Complete resolution of diplopia was observed in 54% of patients treated with veligrotug, compared to 12% in the placebo group [4]. - An overall response rate of 67% was achieved in the veligrotug group versus 5% in the placebo group, indicating the treatment was generally safe and well tolerated [5]. Future Plans - Viridian plans to file a biologics license application (BLA) for veligrotug in the second half of 2025 and is also evaluating another candidate, VRDN-003, in late-stage studies for TED [6][7]. - Top-line data from the phase III studies for VRDN-003 is expected in the first half of 2026, with a BLA filing anticipated by the end of 2026 [8]. Competitive Landscape - If approved, veligrotug and VRDN-003 will face competition from Amgen's Tepezza, the first approved drug for TED, which is administered intravenously [9]. - Amgen is also developing a subcutaneous formulation of Tepezza, which may offer a more convenient administration method compared to intravenous drugs [9][10].

Core Insights - Viridian Therapeutics has released positive data from its phase 3 THRIVE study for veligrotug, which treats active thyroid eye disease (TED), achieving a 70% proptosis responder rate compared to 5% for placebo [2][3] - The company plans to file a Biologics License Application (BLA) for veligrotug for both active and chronic TED by the second half of 2025, with additional data from the THRIVE-2 study expected by the end of 2024 [1][8] - Viridian is also developing other candidates, including VRDN-006 for autoimmune disorders and VRDN-008 with extended half-life technology, which may provide competitive advantages in a crowded market [4][8] Company Developments - The THRIVE study involved 154 patients, randomized to receive either 10 mg/kg of veligrotug or placebo over 15 weeks, with a primary endpoint of proptosis responder rate [3] - The primary endpoint was met with statistical significance (p<0.0001), showing a mean reduction in proptosis of 2.9 mm for the drug group versus 0.5 mm for placebo [3] - Viridian's cash position as of June 30, 2024, was $571.4 million, providing a cash runway into the second half of 2026, allowing the company to fund upcoming milestones [5] Market Opportunity - The market for thyroid eye disease is projected to reach $4.13 billion by 2034, indicating significant potential for new treatments [3] - Competition exists with Amgen's Tepezza, which has been on the market since 2020, making it challenging for new entrants like veligrotug to capture market share [3][8] - The anti-FcRn market is becoming crowded, with several players developing similar treatments, necessitating differentiation strategies for Viridian's products [4] Future Catalysts - Results from the THRIVE-2 study are anticipated by the end of 2024, which will be crucial for the company's regulatory strategy [1][8] - Two phase 3 studies for VRDN-003, an anti-IGF-1R monoclonal antibody, were initiated in August 2024, with topline data expected in the first half of 2026 [3][4] - The company aims to initiate a phase 1 study for VRDN-006 by the end of 2024, targeting autoimmune disorders [4]

Group 1 - Viridian Therapeutics Inc (NASDAQ:VRDN) stock increased by 11.1% to $15.75, reaching a high of $16.35 after a late-stage study for its eye disorder treatment met its main goal [1] - Despite the recent increase, VRDN shares are still down 28% year to date, and momentum has not reached the 200-day moving average [2] - A short squeeze may support the stock's upward movement, as short interest decreased by 7.3% in the last two reporting periods, with 8 million shares sold short, representing 12.9% of the total float [2] Group 2 - There has been significant put trading activity for Viridian, with 6,000 puts bought to open in the last 10 days compared to only 460 calls [3]

Investment Rating - The investment rating for Viridian Therapeutics Inc. (VRDN) is "Buy" with a 12-month price target of $25.00, indicating an upside potential of 70.1% from the current price of $14.70 [9]. Core Insights - Viridian Therapeutics Inc. (VRDN) is well-positioned with its VRDN-003 product, which is expected to have best-in-class potential in the subcutaneous (SC) treatment of thyroid eye disease (TED) due to its dosing regimens and autoinjector properties [5][8]. - SLRN has refocused its pipeline strategy to prioritize lonigutamab for TED, moving directly to Phase 3 development by Q1 2025, which increases competitive pressure on VRDN-003 [2][3]. - VRDN plans to initiate Phase 3 trials for VRDN-003 in August 2024, with topline data expected in the first half of 2026, ahead of SLRN's timelines [3][5]. Summary by Sections Pipeline Strategy - SLRN has discontinued investments in HS/PsA and reduced its workforce by 33% to extend its cash runway to mid-2027, focusing solely on lonigutamab for TED [2]. - Early Phase 1/2 data for lonigutamab showed promising results, but VRDN-003's half-life extension and autoinjector features are seen as significant commercial advantages [2][5]. Clinical Development - VRDN-003's Phase 3 REVEAL-1/REVEAL-2 trials are set to begin in August 2024, with topline data expected in 1H26 and a BLA filing by the end of 2026 [3][5]. - SLRN's decision to skip Phase 2b/3 studies and move directly to Phase 3 could intensify competition, but VRDN-003 is anticipated to deliver data from its primary endpoints before SLRN [3][5]. Safety and Efficacy - Updates on tinnitus effects from SLRN's trials indicate that hearing adverse events are mostly transient and manageable, aligning with VRDN's observations [4][5]. - The Phase 3 studies for both VRDN and SLRN may reveal different occurrences of hearing adverse events due to their varying study durations [4].

Viridian Therapeutics, Inc. (VRDN) came out with a quarterly loss of $1.02 per share versus the Zacks Consensus Estimate of a loss of $0.87. This compares to loss of $1.27 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of -17.24%. A quarter ago, it was expected that this company would post a loss of $1.07 per share when it actually produced a loss of $0.79, delivering a surprise of 26.17%. Over the last four quarters, the compa ...

Financial Data and Key Metrics - The company ended the quarter with $613 million in cash, cash equivalents, and short-term investments, maintaining a cash runway into the second half of 2026 [11][41] Business Line Data and Key Metrics - The THRIVE Phase 3 trial for VRDN-001 in active thyroid eye disease (TED) completed enrollment in March 2024 with 113 patients, exceeding the target of 90 due to strong patient demand [9][24] - The company anticipates filing a Biologics License Application (BLA) for VRDN-001 in the second half of 2025 [10] - The subcutaneous VRDN-003 program completed a positive Type C meeting with the FDA and is on track to initiate pivotal clinical trials mid-year 2024 [10][35] - The FcRn portfolio is progressing as planned, with an IND submission for VRDN-006 expected by the end of 2024 and non-human primate data for VRDN-008 expected in the second half of 2024 [11][39] Market Data and Key Metrics - The U.S. market for moderate to severe TED is estimated at 190,000 patients, with the current standard of care generating approximately $1.8 billion in U.S. sales in 2023 [13][14] - The TED market is considered a "new start market," meaning patients can choose from available treatments during each flare-up without displacing chronic therapies [15][16] Company Strategy and Industry Competition - The company aims to develop differentiated therapies for unmet medical needs, focusing on both IV and subcutaneous delivery options for TED and FcRn inhibitors for autoimmune diseases [7][36] - VRDN-001 is positioned as a potential best-in-class IV therapy with fewer doses and shorter infusion times compared to the current standard of care [27][28] - VRDN-003 is designed to offer a best-in-class subcutaneous dosing profile with the potential for at-home self-administration, which could significantly expand the market [29][31] Management Commentary on Operating Environment and Future Outlook - Management highlighted strong execution across the pipeline, with key catalysts including THRIVE top-line results in September 2024 and THRIVE-2 top-line results at the end of 2024 [40][41] - The company remains well-capitalized and is focused on delivering on upcoming milestones [41] Other Important Information - The company is conducting the STRIVE study to complete the safety database for the BLA submission of VRDN-001, with a data cut expected once the requisite number of patients is reached [26][72] - VRDN-003 demonstrated an extended half-life of 40-50 days in healthy volunteers, supporting its potential as a best-in-class subcutaneous therapy [32][34] Q&A Session Summary Question: What defines success for the THRIVE trial? - Success for THRIVE would be a safety and efficacy profile similar to TEPEZZA, with potential improvements in hearing impairment due to lower drug exposure [44][45] Question: How is the company addressing safety concerns in THRIVE? - The company is using standard methodologies for adverse event reporting and audiometry to monitor hearing, consistent with FDA guidance and current clinical practice [51][58] Question: What are the plans for VRDN-003 pivotal trials? - The company had a positive Type C meeting with the FDA and is on track to initiate pivotal trials mid-2024, with more details to be provided after receiving meeting minutes [56][62] Question: How does the company view the competitive landscape in TED? - The company sees strong patient demand for IGF-1R therapies, as evidenced by the over-enrollment in THRIVE, and believes its subcutaneous VRDN-003 could be a game-changer in the market [79][90] Question: What is the timeline for FcRn data? - Non-human primate data for VRDN-008 is expected in the second half of 2024, with healthy volunteer data for VRDN-006 anticipated in the second half of 2025 [91][92] Question: How does the company plan to expand internationally? - The company is considering ex-U.S. markets, particularly Europe, where the epidemiology of TED is similar to the U.S., but specific plans will be shared at a later time [66]

Commission File No. 001-36483 VIRIDIAN THERAPEUTICS, INC. UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to | Title of each class | Trading Symbol(s) | Name of each exchange on which registered | | --- | - ...

Exhibit 99.1 Viridian Therapeutics Highlights Recent Progress and Reports First Quarter 2024 Financial Results - THRIVE VRDN-001 global phase 3 clinical trial in active thyroid eye disease (TED) completed and exceeded its target for enrollment in March 2024; topline readout expected in September 2024 - - THRIVE-2 VRDN-001 global phase 3 clinical trial for patients with chronic TED remains on track for topline readout by year-end 2024 - Thyroid Eye Disease Portfolio VRDN-001, an intravenously delivered anti- ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-36483 VIRIDIAN THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) (State or other jurisdiction of incorporatio ...