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 ...

Exhibit 99.1 Viridian Therapeutics Highlights Recent Progress and Reports Second Quarter 2024 Financial Results - THRIVE VRDN-001 global phase 3 clinical trial in active thyroid eye disease (TED) remains on track for topline readout in September 2024 - - THRIVE-2 VRDN-001 global phase 3 clinical trial in chronic TED topline readout expected year-end 2024; enrollment completed in July and exceeded its target - - REVEAL-1 and REVEAL-2, global phase 3 clinical trials for subcutaneous VRDN-003 in patients with ...

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 June 30, 2024 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 No. 001-36483 VIRIDIAN THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 47-1187261 (State or other jurisdi ...

The sudden soaring of the stock price can be attributed to positive investor mindset regarding the progress of Viridian's clinical-stage pipeline. The company is evaluating its lead candidate, VRDN-001 in active and chronic thyroid eye disease (TED) in two separate late-stage studies, namely THRIVE and THRIVE-2, respectively. While top-line data from the THRIVE study is expected in September, data from the THRIVE-2 study is anticipated before this year's end. If the outcomes from both studies are positive, ...

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]

| --- | --- | |-------|-----------------------| | | | | | E N G I N E E R I N G | Cautionary note regarding forward-looking statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as, but not limited to, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "will, ...