Financial Performance - Viridian Therapeutics reported a quarterly loss of $1 per share, which aligns with the Zacks Consensus Estimate, compared to a loss of $1.02 per share a year ago [1] - The company posted revenues of $0.08 million for the quarter ended June 2025, missing the Zacks Consensus Estimate by 48.63%, and showing a slight increase from $0.07 million year-over-year [2] - Over the last four quarters, the company has surpassed consensus EPS estimates two times and topped consensus revenue estimates just once [2][3] Stock Performance - Viridian Therapeutics shares have declined approximately 9% since the beginning of the year, while the S&P 500 has gained 7.1% [3] - The current status of estimate revisions translates into a Zacks Rank 3 (Hold) for the stock, indicating expected performance in line with the market in the near future [6] Future Outlook - The current consensus EPS estimate for the coming quarter is -$0.96 on revenues of $0.15 million, and for the current fiscal year, it is -$3.96 on revenues of $0.33 million [7] - The outlook for the industry, specifically the Medical - Biomedical and Genetics sector, is currently in the bottom 42% of over 250 Zacks industries, which may impact the stock's performance [8]

[PART I. FINANCIAL INFORMATION](index=6&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Financial Statements](index=6&type=section&id=Item%201.%20Financial%20Statements) This section presents the unaudited condensed consolidated financial statements for the quarter ended June 30, 2025, including balance sheets, statements of operations, stockholders' equity, and cash flows, with a net loss of $187.6 million and an accumulated deficit of $1.18 billion as of June 30, 2025 Condensed Consolidated Balance Sheet Highlights (in thousands) | | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $117,324 | $99,594 | | Short-term investments | $446,032 | $617,990 | | Total assets | $582,324 | $742,403 | | **Liabilities & Equity** | | | | Total liabilities | $67,155 | $70,764 | | Total stockholders' equity | $515,169 | $671,639 | | Accumulated deficit | $(1,183,504) | $(995,857) | Condensed Consolidated Statements of Operations (in thousands, except per share data) | | Three Months Ended June 30, | Six Months Ended June 30, | | :--- | :--- | :--- | | | **2025** | **2024** | **2025** | **2024** | | Collaboration revenue - related party | $75 | $72 | $147 | $144 | | Research and development | $86,626 | $56,193 | $163,461 | $97,136 | | General and administrative | $20,216 | $16,066 | $37,319 | $31,091 | | **Loss from operations** | **$(106,767)** | **$(72,187)** | **$(200,633)** | **$(128,083)** | | **Net loss** | **$(100,735)** | **$(64,993)** | **$(187,647)** | **$(113,535)** | | Net loss per share, basic and diluted | $(1.00) | $(0.77) | $(1.87) | $(1.37) | Condensed Consolidated Statements of Cash Flows Highlights (in thousands) | | Six Months Ended June 30, | | :--- | :--- | | | **2025** | **2024** | | Net cash used in operating activities | $(168,026) | $(91,281) | | Net cash provided by (used in) investing activities | $175,780 | $(81,069) | | Net cash provided by financing activities | $9,976 | $177,190 | | **Net increase in cash and cash equivalents** | **$17,730** | **$4,840** | [Note 1: Description of Business and Liquidity](index=11&type=section&id=1.%20DESCRIPTION%20OF%20BUSINESS) This note details the company's focus on developing medicines for rare diseases, its accumulated deficit of $1.18 billion, and its $563.4 million in cash and investments, sufficient to fund operations for at least the next twelve months - The company's most advanced program is veligrotug, a monoclonal antibody targeting IGF-1R for the treatment of Thyroid Eye Disease (TED), also developing VRDN-003 and a portfolio of FcRn inhibitors (VRDN-006, VRDN-008) for autoimmune diseases[27](index=27&type=chunk)[28](index=28&type=chunk) - As of June 30, 2025, the company had an accumulated deficit of **$1.18 billion** and expects to continue generating operating losses for the foreseeable future[29](index=29&type=chunk) - The company had approximately **$563.4 million** in cash, cash equivalents, and short-term investments as of June 30, 2025, and believes these resources are sufficient to fund operations and capital expenditures for at least the next twelve months[31](index=31&type=chunk) [Note 5: Debt](index=19&type=section&id=5.%20DEBT) This note outlines the company's amended loan agreement with Hercules Capital, providing access to $150.0 million, with $20.0 million drawn and an interest-only period extended to April 2026 - The amended term loan with Hercules Capital provides access to up to **$150.0 million**, with **$20.0 million** drawn as of the report date[85](index=85&type=chunk)[86](index=86&type=chunk) - The interest-only payment period was extended to **April 1, 2026**, due to the achievement of development milestones related to positive topline results from the Phase 3 THRIVE and THRIVE-2 trials[88](index=88&type=chunk) Debt Summary (as of June 30, 2025, in thousands) | Component | Amount | | :--- | :--- | | Gross term loan proceeds | $20,000 | | Accrued end-of-term fee | $788 | | **Total debt** | **$20,788** | | Less: current portion | $(8,435) | | **Long-term debt, net** | **$12,353** | [Note 6: Collaboration Agreements](index=21&type=section&id=6.%20COLLABORATION%20AGREEMENTS) This note details collaboration agreements, including a license with Zenas BioPharma generating minor revenue and related-party agreements with Paragon Therapeutics resulting in $4.5 million in R&D costs in H1 2025 - The license agreement with Zenas BioPharma for IGF-1R antibody products in China was sublicensed to Zai Lab in January 2025, with the company recognizing **$0.1 million** in related collaboration revenue in H1 2025[92](index=92&type=chunk)[95](index=95&type=chunk) - The agreements with Paragon Therapeutics are considered related-party transactions, with the company recording **$4.5 million** in R&D costs in H1 2025, including a **$3.5 million** payment for achieving R&D objectives and a **$1.0 million** clinical milestone payment[103](index=103&type=chunk)[104](index=104&type=chunk)[105](index=105&type=chunk) [Note 7: Commitments and Contingencies](index=23&type=section&id=7.%20COMMITMENTS%20AND%20CONTINGENCIES) This note describes significant financial commitments, including potential milestone payments of up to $48.0 million to ImmunoGen and $45.0 million to Enable Injections, plus $2.7 million in future minimum lease obligations - The company is obligated to make potential future development milestone payments of up to **$48.0 million** to ImmunoGen (acquired by AbbVie) and up to **$45.0 million** to Enable Injections upon achieving specified milestones[107](index=107&type=chunk)[110](index=110&type=chunk) Future Lease Payments (as of June 30, 2025, in thousands) | Period | Amount | | :--- | :--- | | 2025 (remainder) | $367 | | 2026 | $748 | | 2027 | $613 | | 2028 | $627 | | 2029 | $370 | | **Total future minimum lease payments** | **$2,725** | [Note 8: Capital Stock](index=27&type=section&id=8.%20CAPITAL%20STOCK) This note details the company's capital structure, including a new $300.0 million ATM sales agreement established in March 2025 and a $150.0 million public offering completed in January 2024 - In March 2025, the company entered into a new Open Market Sale Agreement (ATM) with Jefferies for up to **$300.0 million** of its common stock, terminating the prior September 2022 ATM agreement, with no shares sold under the new agreement during the quarter[124](index=124&type=chunk) - In January 2024, the company raised approximately **$150.0 million** in gross proceeds from a public offering of 7,142,858 shares of common stock[125](index=125&type=chunk) [Note 10: Share-Based Compensation](index=29&type=section&id=10.%20SHARE-BASED%20COMPENSATION) This note reports $10.8 million in share-based compensation expense for Q2 2025 and $21.1 million for H1 2025, alongside stockholder approval for an 8,000,000 share increase to the 2016 Equity Incentive Plan Share-Based Compensation Expense (in thousands) | | Three Months Ended June 30, 2025 | Six Months Ended June 30, 2025 | | :--- | :--- | :--- | | Research and development | $5,173 | $10,921 | | General and administrative | $5,671 | $10,143 | | **Total** | **$10,844** | **$21,064** | - In June 2025, stockholders approved an **8,000,000 share** increase to the 2016 Equity Incentive Plan[138](index=138&type=chunk) - As of June 30, 2025, there was **$106.3 million** of total unrecognized share-based compensation cost related to stock options and **$14.3 million** related to unvested RSUs[149](index=149&type=chunk) [Note 13: Subsequent Events](index=34&type=section&id=13.%20SUBSEQUENT%20EVENTS) This note discloses a July 2025 collaboration and license agreement with Kissei Pharmaceutical for veligrotug and VRDN-003 in Japan, including a $70.0 million upfront payment and potential milestones - The agreement with Kissei includes a **$70.0 million** upfront payment to Viridian[159](index=159&type=chunk) - Viridian is eligible to receive up to an additional **$315.0 million** in development, regulatory, and sales milestones, plus tiered royalties ranging from the twenties to the mid-thirties on future net sales in Japan[159](index=159&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=35&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's financial condition, clinical program progress for TED and FcRn inhibitors, increased R&D and G&A expenses, and a projected cash runway into the second half of 2027 [Overview and Recent Developments](index=35&type=section&id=Overview%20and%20Recent%20Developments) The company is advancing its pipeline for TED and autoimmune diseases, with veligrotug nearing BLA submission, VRDN-003 in pivotal trials, and FcRn inhibitors progressing towards proof-of-concept data and IND submission - Veligrotug (TED Program): Received FDA Breakthrough Therapy designation in May 2025, with a BLA submission anticipated in H2 2025 and an MAA submission to the EMA in H1 2026[170](index=170&type=chunk) - VRDN-003 (Subcutaneous TED Program): Pivotal Phase 3 trials (REVEAL-1 and REVEAL-2) are ongoing, with topline data expected in H1 2026 and a BLA submission anticipated by the end of 2026[173](index=173&type=chunk) - FcRn Inhibitor Program: Proof-of-concept IgG reduction data for VRDN-006 is expected in Q3 2025, and an IND for the half-life extended VRDN-008 is planned for submission by the end of 2025[175](index=175&type=chunk)[176](index=176&type=chunk) [Results of Operations](index=39&type=section&id=Results%20of%20Operations) This section compares financial results, highlighting significant increases in R&D expenses due to advancing clinical trials and manufacturing, and higher G&A costs driven by personnel and pre-commercialization activities Comparison of Operating Expenses (in thousands) | | Three Months Ended June 30, | Six Months Ended June 30, | | :--- | :--- | :--- | | | **2025** | **2024** | **2025** | **2024** | | Research and development | $86,626 | $56,193 | $163,461 | $97,136 | | General and administrative | $20,216 | $16,066 | $37,319 | $31,091 | - R&D expenses for the six months ended June 30, 2025, increased by **$66.3 million** compared to the same period in 2024, driven by a **$41.2 million** increase in TED portfolio costs and a **$19.7 million** increase in FcRn portfolio costs[199](index=199&type=chunk)[201](index=201&type=chunk)[204](index=204&type=chunk) - G&A expenses for the six months ended June 30, 2025, increased by **$6.2 million** compared to the prior year period, primarily due to a **$4.4 million** increase in personnel costs, a **$3.3 million** increase in market research for veligrotug, and a **$2.5 million** increase in professional fees[205](index=205&type=chunk)[209](index=209&type=chunk) [Liquidity and Capital Resources](index=43&type=section&id=Liquidity%20and%20Capital%20Resources) As of June 30, 2025, the company had $563.4 million in cash and investments, expected to fund operations into H2 2027, with net cash used in operations increasing to $168.0 million in H1 2025 - The company's cash, cash equivalents, and short-term investments totaled **$563.4 million** as of June 30, 2025[206](index=206&type=chunk) - The current cash position is expected to fund planned operations into the **second half of 2027**[206](index=206&type=chunk) Summarized Cash Flows (in thousands) | | Six Months Ended June 30, | | :--- | :--- | | | **2025** | **2024** | | Net cash used in operating activities | $(168,026) | $(91,281) | | Net cash provided by (used in) investing activities | $175,780 | $(81,069) | | Net cash provided by financing activities | $9,976 | $177,190 | [Quantitative and Qualitative Disclosures about Market Risk](index=46&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) The company reports no material changes to its market risks during the six months ended June 30, 2025, compared to its prior annual report disclosures - No material changes to market risks were reported for the period[227](index=227&type=chunk) [Controls and Procedures](index=46&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of quarter-end, with no material changes to internal control over financial reporting during the period - Based on an evaluation as of the end of the quarter, the principal executive officer and principal financial officer concluded that the company's disclosure controls and procedures were effective[230](index=230&type=chunk) - There have been no changes in internal control over financial reporting during the most recent fiscal quarter that have materially affected, or are reasonably likely to materially affect, internal controls[231](index=231&type=chunk) [PART II. OTHER INFORMATION](index=47&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Legal Proceedings](index=47&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any legal proceedings that would have a material adverse effect on its business, financial condition, or results of operations - The company is not currently a party to any material legal proceedings[233](index=233&type=chunk) [Risk Factors](index=47&type=section&id=Item%201A.%20Risk%20Factors) This section outlines key risks including significant financial losses, the high cost and uncertainty of clinical trials, reliance on third-party manufacturers like WuXi Biologics, intense competition, intellectual property challenges, and evolving regulatory landscapes - Financial Risk: The company has a history of significant losses, with a **$1.18 billion** accumulated deficit, and will require substantial additional capital to fund operations beyond its current cash runway, which is projected into the **second half of 2027**[235](index=235&type=chunk)[236](index=236&type=chunk) - Third-Party and Geopolitical Risk: The company relies heavily on third-party manufacturers, including the Chinese CDMO WuXi Biologics, and proposed U.S. legislation like the BIOSECURE Act could disrupt this relationship, potentially forcing a costly and delayed transition to alternative suppliers[335](index=335&type=chunk)[344](index=344&type=chunk) - Clinical and Regulatory Risk: The company's success is dependent on costly and inherently risky clinical trials, and failure to demonstrate safety and efficacy or to obtain regulatory approval would materially harm the business, as the regulatory process is lengthy, unpredictable, and subject to changes in policy[258](index=258&type=chunk)[269](index=269&type=chunk) - Commercialization Risk: The company faces substantial competition from large, well-funded companies like Amgen in the TED market and must build its own commercial capabilities to launch products successfully, if approved[305](index=305&type=chunk)[311](index=311&type=chunk) - Intellectual Property Risk: The business depends on protecting licensed and owned intellectual property, facing risks of infringement claims from third parties and challenges to the validity of its patents, which could be expensive and time-consuming to defend[354](index=354&type=chunk)[373](index=373&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=87&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities during the period - None[468](index=468&type=chunk) [Other Information](index=88&type=section&id=Item%205.%20Other%20Information) The company states that no directors or officers adopted or terminated any Rule 10b5-1 or non-Rule 10b5-1 trading arrangements during the quarter - No directors or officers adopted or terminated a Rule 10b5-1 trading arrangement during the quarter[471](index=471&type=chunk) [Exhibits](index=88&type=section&id=Item%206.%20Exhibits) This section provides an index of exhibits filed with the quarterly report, including corporate governance documents and officer certifications

[Business Highlights and Pipeline Update](index=1&type=section&id=Business%20Highlights%20and%20Pipeline%20Update) Viridian highlights progress on its lead TED drug candidates, FcRn inhibitor pipeline, and a Japan licensing deal [Veligrotug Program for Thyroid Eye Disease (TED)](index=1&type=section&id=Veligrotug%20Program%20for%20Thyroid%20Eye%20Disease%20%28TED%29) Veligrotug, Viridian's lead candidate for TED, received FDA **Breakthrough Therapy Designation (BTD)** and met all Phase 3 trial endpoints - The FDA granted **Breakthrough Therapy Designation (BTD)** to veligrotug, a status for drug candidates that may offer substantial improvement over existing therapies. This was based on its robust improvement in diplopia and rapid proptosis response[1](index=1&type=chunk)[7](index=7&type=chunk) - Veligrotug successfully met all primary and secondary endpoints in its two pivotal Phase 3 trials, THRIVE (active TED) and THRIVE-2 (chronic TED)[4](index=4&type=chunk) - Long-term data from the THRIVE trial showed strong durability, with **70% of patients** who responded to proptosis treatment at 15 weeks maintaining that response at 52 weeks, 40 weeks after the last dose[7](index=7&type=chunk) - A Biologics License Application (BLA) submission to the U.S. FDA is on track for the second half of 2025, with a potential U.S. commercial launch in 2026[1](index=1&type=chunk)[7](index=7&type=chunk) [VRDN-003 Program for Thyroid Eye Disease (TED)](index=2&type=section&id=VRDN-003%20Program%20for%20Thyroid%20Eye%20Disease%20%28TED%29) VRDN-003, a subcutaneously delivered TED antibody, aims for infrequent self-administration with pivotal data expected 1H 2026 - Topline data from the pivotal clinical trials, REVEAL-1 and REVEAL-2, are expected in the first half of 2026[7](index=7&type=chunk) - The company plans to submit a BLA for VRDN-003 by the end of 2026[7](index=7&type=chunk) - VRDN-003 is designed for infrequent self-administration at home using a low-volume autoinjector[7](index=7&type=chunk) - VRDN-003 has a half-life of **40-50 days**, which is **4-5 times longer** than veligrotug, potentially allowing for dosing every 4 or 8 weeks (Q4W/Q8W)[14](index=14&type=chunk) [Japan Licensing Agreement with Kissei Pharmaceutical](index=1&type=section&id=Japan%20Licensing%20Agreement%20with%20Kissei%20Pharmaceutical) Viridian entered an exclusive licensing agreement with Kissei Pharmaceutical for veligrotug and VRDN-003 in Japan Kissei Pharmaceutical Licensing Deal Terms | Term | Value | | :--- | :--- | | Upfront Cash Payment | $70 million | | Potential Future Milestones | Up to $315 million | | Royalties on Net Sales | Tiered, ranging from 20s to mid-30s | - Kissei will be responsible for all development, regulatory, and commercialization activities and costs in Japan[7](index=7&type=chunk) [FcRn Inhibitor Portfolio](index=2&type=section&id=FcRn%20Inhibitor%20Portfolio) Viridian advances its FcRn inhibitor portfolio, with key data and IND submissions anticipated in 2025 - VRDN-006: Data from the Phase 1 clinical trial in healthy volunteers, including proof-of-concept IgG reduction, is expected in **Q3 2025**[7](index=7&type=chunk)[11](index=11&type=chunk) - VRDN-008: An Investigational New Drug (IND) submission is on track for **year-end 2025**[7](index=7&type=chunk)[11](index=11&type=chunk) - In non-human primate studies, VRDN-008 demonstrated a **longer half-life** and **more sustained IgG reduction** compared to efgartigimod[7](index=7&type=chunk)[16](index=16&type=chunk) [Expected Upcoming Milestones](index=2&type=section&id=Expected%20Upcoming%20Milestones) Viridian outlines key upcoming milestones for its portfolio, including BLA submissions and clinical data readouts - **Veligrotug:** - BLA submission in **2H 2025** - U.S. commercial launch in **2026** (if approved) - MAA submission in **1H 2026**[7](index=7&type=chunk) - **VRDN-003:** - Topline data in **1H 2026**[7](index=7&type=chunk) - **VRDN-006:** - Healthy volunteer clinical data in **Q3 2025**[11](index=11&type=chunk) - **VRDN-008:** - IND submission by **year-end 2025**[11](index=11&type=chunk) [Second Quarter 2025 Financial Results](index=1&type=section&id=Second%20Quarter%202025%20Financial%20Results) Viridian reports Q2 2025 financial results, detailing cash position, operating expenses, and balance sheet [Financial Summary](index=1&type=section&id=Financial%20Summary) Viridian ended Q2 2025 with **$563.4 million** cash, extending its runway into **2H 2027** Key Financial & Operational Metrics (Q2 2025) | Metric | Value | | :--- | :--- | | Cash, Cash Equivalents & Short-Term Investments | **$563.4 million** (as of June 30, 2025) | | Cash Runway | Into **2H 2027** | | Q2 2025 R&D Expenses | **$86.6 million** | | Q2 2025 G&A Expenses | **$20.2 million** | | Common Stock Outstanding | **100,320,386 shares** (as-converted basis) | - The increase in R&D expenses was driven by costs for multiple ongoing Phase 3 trials (veligrotug, VRDN-003) and a Phase 1 trial (VRDN-006)[11](index=11&type=chunk) - The increase in G&A expenses was primarily due to preparatory commercial activities for veligrotug and increased professional service and personnel costs[11](index=11&type=chunk) [Condensed Consolidated Statements of Operations](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Viridian reported a Q2 2025 net loss of **$100.7 million**, reflecting increased operating expenses year-over-year Statement of Operations Highlights (Three Months Ended June 30) | Metric (in thousands USD) | 2025 | 2024 | | :--- | :--- | :--- | | Total revenue | $75 | $72 | | Research and development | $86,626 | $56,193 | | General and administrative | $20,216 | $16,066 | | **Loss from operations** | **($106,767)** | **($72,187)** | | **Net loss** | **($100,735)** | **($64,993)** | | Net loss per share, common | ($1.00) | ($0.77) | [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of June 30, 2025, Viridian's total assets decreased to **$582.3 million**, primarily due to cash utilization Balance Sheet Highlights (in thousands USD) | Metric | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents and short-term investments | $563,356 | $717,584 | | **Total assets** | **$582,324** | **$742,403** | | Total liabilities | $67,155 | $70,764 | | **Total stockholders' equity** | **$515,169** | **$671,639** |

Thyroid Eye Disease (TED) Portfolio - Veligrotug (Intravenous) - Positive THRIVE and THRIVE-2 topline data in active and chronic TED showed a robust clinical profile[12] - In the THRIVE trial, 70% of patients receiving veligrotug achieved a proptosis response at 15 weeks, compared to 5% on placebo[57] - In the THRIVE trial, 53% of patients receiving veligrotug achieved a proptosis response at 3 weeks, after just 1 infusion[59] - In the THRIVE-2 trial (chronic TED), 56% of patients achieved a proptosis response compared to 8% in the placebo group[78] - Viridian plans to submit the BLA for veligrotug in the second half of 2025 and the EU MAA in the first half of 2026[3, 12] Thyroid Eye Disease (TED) Portfolio - VRDN-003 (Subcutaneous) - REVEAL-1 and REVEAL-2 trials are enrolling and dosing patients, with topline data expected in the first half of 2026[12] - The BLA submission for VRDN-003 is anticipated by year-end 2026[3, 12] - Phase 1 HV Study showed VRDN-003 has an extended half-life of 40–50 days and increases IGF-1 levels ~4-fold[102, 104] FcRn Inhibitor Portfolio - VRDN-006 proof-of-concept Phase 1 clinical trial data is expected in Q3 2025[3, 12] - VRDN-008 is on track for IND submission by year-end 2025[3, 12] - A single dose of VRDN-008 demonstrated a longer half-life and deeper IgG reduction compared to efgartigimod in NHPs[148, 149] Financial - Viridian had $637 million in cash as of March 31, 2025, providing a runway into the second half of 2027[12]

Group 1 - The discussion in the biotech community has focused on profitable buy-write or covered call strategies for selected biotech stocks over recent months [1] - Viridian Therapeutics, Inc. (NASDAQ: VRDN) has been highlighted as a promising clinical-stage biotech company, despite a significant stock price drop of one-third since the last analysis [2] - The investing group, The Biotech Forum, offers a model portfolio featuring 12-20 high upside biotech stocks, along with live chat discussions and weekly market commentary [2] Group 2 - The article emphasizes that the analyst holds a beneficial long position in VRDN shares through various financial instruments [3] - There is a disclaimer regarding past performance not guaranteeing future results, and no specific investment advice is provided [4]

Summary of Viridian Therapeutics (VRDN) 2025 Conference Call Company Overview - **Company**: Viridian Therapeutics (VRDN) - **Event**: Jefferies Global Healthcare Conference - **Date**: June 04, 2025 Key Points Industry and Product Development - Viridian is focused on the treatment of Thyroid Eye Disease (TED) with its lead program involving both intravenous (IV) and subcutaneous (SUBQ) administration methods [2][23] - The company is completing phase three studies and preparing to file with the FDA [2][18] Clinical Trial Results - The main endpoints for the phase three trials include proptosis response (eye bulging), clinical activity score (pain and inflammation), and diplopia (double vision) [4][5] - The results showed significant improvement in both diplopia and proptosis, with a rapid treatment effect observed after just one infusion [6][7] - 70% of patients maintained their response at 40 weeks post-infusion, compared to Amgen's TEPEZZA, which reported a 53% response rate [8][9] Breakthrough Therapy Designation - The FDA granted breakthrough therapy designation based on rapid treatment onset and superior diplopia resolution rates compared to TEPEZZA [3][14] - Viridian's treatment showed a 20% placebo-adjusted complete resolution of diplopia, significantly higher than TEPEZZA's 3% [14][16] Regulatory and Filing Strategy - The company is awaiting the completion of the chronic study follow-up period before filing for approval [18][23] - A priority review could lead to a PDUFA date in 2026 [23][24] Subcutaneous (SUBQ) Program - The SUBQ program is currently enrolling in two phase three studies, with data expected in the first half of 2026 and a BLA filing anticipated by the end of 2026 [27][28] - The SUBQ formulation is designed for self-administration, which could significantly increase patient access and convenience [41][51] Market Dynamics and Competitive Landscape - TEPEZZA, currently the only approved treatment for TED, has seen declining sales due to initial reimbursement challenges and a lengthy treatment regimen [60][64] - Viridian aims to capture market share by offering a more convenient treatment option with fewer infusions (5 vs. 8 for TEPEZZA) and a shorter treatment duration [63][64] Future Opportunities - The company is optimistic about its potential to become a commercial entity in 2026, leveraging its differentiated data and the convenience of its SUBQ administration [73][74] - Viridian is also developing an FcRn program, targeting multiple indications with a focus on IgG suppression, which could further expand its market opportunities [78][79] Conclusion - Viridian Therapeutics is positioned for significant growth with its innovative treatment for TED and ongoing development of its SUBQ program, alongside a promising FcRn initiative. The company is set to capitalize on the current market dynamics and the shortcomings of existing therapies.

Core Viewpoint - Viridian Therapeutics reported a quarterly loss of $0.87 per share, which was better than the Zacks Consensus Estimate of a loss of $0.95, indicating an earnings surprise of 8.42% [1] Financial Performance - The company posted revenues of $0.07 million for the quarter ended March 2025, missing the Zacks Consensus Estimate by 55.83%, with no year-over-year revenue growth [2] - Over the last four quarters, Viridian has surpassed consensus EPS estimates two times and topped consensus revenue estimates only once [2] Stock Performance - Viridian Therapeutics shares have declined approximately 31.6% since the beginning of the year, contrasting with the S&P 500's decline of 3.9% [3] - The current Zacks Rank for the stock is 3 (Hold), suggesting it is expected to perform in line with the market in the near future [6] Earnings Outlook - The current consensus EPS estimate for the upcoming quarter is -$0.98 on revenues of $0.16 million, and for the current fiscal year, it is -$4.01 on revenues of $0.55 million [7] - The estimate revisions trend for Viridian is mixed, which could change following the recent earnings report [6] Industry Context - The Medical - Biomedical and Genetics industry, to which Viridian belongs, is currently in the top 31% of over 250 Zacks industries, indicating a favorable outlook compared to lower-ranked industries [8]

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].

Financial Performance - Total revenue for Q1 2025 remained flat at $72,000 compared to Q1 2024[18] - Net loss for Q1 2025 was $86,912,000, compared to a net loss of $48,542,000 in Q1 2024, representing an increase of 79.2%[18] - Net loss per share for common stock was $0.87 in Q1 2025, up from $0.59 in Q1 2024[18] - The company reported a comprehensive loss of $86,657,000 for Q1 2025, compared to a comprehensive loss of $49,247,000 in Q1 2024[18] Expenses - R&D expenses increased to $76.8 million in Q1 2025, up from $40.9 million in Q1 2024, due to more clinical trials[12] - General and administrative expenses rose to $17.1 million in Q1 2025, compared to $15.0 million in Q1 2024, driven by preparatory commercial activities[12] - Research and development expenses increased by 87.7% to $76,835,000 in Q1 2025 from $40,944,000 in Q1 2024[18] - Total operating expenses rose by 67.8% to $93,938,000 in Q1 2025 from $55,969,000 in Q1 2024[18] Cash and Assets - Strong cash position of $636.6 million as of March 31, 2025, supporting operations into the second half of 2027[2] - Cash, cash equivalents, and short-term investments decreased to $636,633,000 as of March 31, 2025, down from $717,584,000 as of December 31, 2024[20] - Total assets decreased to $660,981,000 as of March 31, 2025, from $742,403,000 as of December 31, 2024[20] - Total stockholders' equity declined to $604,473,000 as of March 31, 2025, from $671,639,000 as of December 31, 2024[20] - Total liabilities decreased to $56,508,000 as of March 31, 2025, from $70,764,000 as of December 31, 2024[20] Clinical Development - Veligrotug BLA submission is on track for the second half of 2025, with a potential U.S. launch in 2026[4] - Anticipated topline data from phase 3 trials REVEAL-1 and REVEAL-2 for VRDN-003 expected in the first half of 2026[5] - VRDN-008 IND submission is on track for year-end 2025, showing longer half-life than efgartigimod[7] - VRDN-006 proof-of-concept data expected in Q3 2025, focusing on IgG reduction[7] Market and Leadership - The market size for myasthenia gravis and chronic inflammatory demyelinating polyneuropathy is projected to approach $10 billion by 2030[6] - Jeff Ajer appointed to the Board of Directors, bringing over 25 years of experience in commercialization for rare diseases[8] Shares - Shares outstanding as of March 31, 2025, totaled 100,258,627, including common and preferred stock[12]

Financial Performance - The company reported a net loss of $269.9 million for the year ended December 31, 2024, compared to a net loss of $237.7 million for 2023, resulting in an accumulated deficit of $995.9 million as of December 31, 2024[220]. - The company anticipates significant ongoing expenses and increasing operating losses as it continues the development of its product candidates and advances them into large clinical trials[225]. - The company has never generated revenue from product sales and does not expect to do so for the foreseeable future, relying primarily on equity and debt financings for funding[229][232]. - The company may need to raise substantial additional capital to fund operations, and failure to do so could negatively impact its financial condition and product development efforts[222][238]. - The company expects research and development expenses to increase as it advances product candidates through clinical trials and pre-commercial activities[228]. - Significant costs associated with clinical trials and manufacturing activities could impact financial performance[246]. - The company may need to establish commercial manufacturing and sales capabilities or enter into agreements with third parties to generate revenue[280]. - The company may face challenges in forming strategic collaborations for product candidates, which could alter development and commercialization plans[282]. - The company may need to allocate resources effectively to avoid missing out on more profitable opportunities in its research programs[272]. - The company is hiring and training a commercial team to support future product launches, which is critical for success[296]. - The company expects to need significant additional capital for operations, which may involve selling common stock or other equity securities, potentially causing dilution for stockholders[413]. Cash and Investments - As of December 31, 2024, the company had $717.6 million in cash, cash equivalents, and short-term investments, which are expected to fund operations into the second half of 2027[221][227]. Regulatory and Compliance Risks - Regulatory authorities may withdraw approval if compliance with standards is not maintained, leading to potential product recalls or marketing restrictions[244]. - Failure to obtain regulatory approval for product candidates could materially affect business prospects[257]. - Regulatory approval processes are lengthy and unpredictable, potentially delaying commercialization[250]. - Undesirable side effects from product candidates may result in delayed regulatory approval or restrictive labeling[258]. - Regulatory authorities may require additional clinical trials or data, impacting development timelines[255]. - Changes in regulatory requirements could lead to increased costs and delays in product development[247]. - The company must comply with ongoing regulatory requirements post-approval, which may include costly post-marketing studies[247]. - Regulatory authorities have substantial discretion in the approval process, which may lead to unexpected delays or refusals[251]. - The company is developing combination products such as VRDN-003, VRDN-006, and VRDN-008, which may face delays in marketing authorizations due to regulatory complexities[262]. - The company is subject to various healthcare fraud and abuse laws, which could result in substantial penalties if not complied with[373]. - Non-compliance with environmental, health, and safety laws could lead to fines or penalties that materially affect the company's financial condition[374]. - The company faces significant costs related to compliance with evolving data privacy and security laws, including the GDPR and CCPA[376][378]. - Non-compliance with data protection laws could result in government investigations, civil penalties, and reputational harm[379]. Competition and Market Risks - The company faces substantial competition in the treatment of TED from major pharmaceutical and biotechnology companies, including Amgen and Roche Holdings AG[285]. - Competitors may develop more effective or less costly products, potentially rendering the company's candidates obsolete[286]. - The company faces significant competition from larger firms with greater resources, impacting its market position[289]. - The approval timeline for competitors' products could allow them to establish a strong market presence before the company can enter[288]. - The commercial success of the company's products in the U.S. depends on market adoption by patients, payors, and healthcare providers[290]. - Pricing pressures are expected due to managed healthcare trends and governmental price controls, affecting profitability[295][294]. - Regulatory approval processes for product candidates may face competition from biosimilars, impacting market exclusivity and pricing[363][364]. - Seeking orphan drug designation may not guarantee exclusivity or protection from competition, affecting financial performance[366][368]. Intellectual Property Risks - The company relies on patent rights and trade secret protections, and any failure to maintain these could impair its competitive position[323]. - The patent position is uncertain, and challenges to patents could result in reduced exclusivity for product candidates[326]. - The company may not have sufficient patent term protections, as patents generally expire 20 years after filing, potentially exposing it to competition[329]. - Collaborations with third parties may not yield expected results, and disagreements could lead to delays or termination of development efforts[321]. - Patent term extensions (PTEs) may be relied upon to extend exclusivity for product candidates, but there are no assurances that these will be obtained, potentially impacting business and financial condition[330]. - Changes in U.S. patent laws, including the Leahy-Smith America Invents Act, have increased uncertainties and costs related to patent applications and enforcement, which could adversely affect business operations[331]. - The U.S. Patent and Trademark Office (USPTO) has issued guidance that may hinder the ability to obtain patent claims for natural products, affecting the patent portfolio's validity[332]. - The transition to a "first-to-file" system under the Leahy-Smith Act requires timely patent application filings, increasing the risk of losing patent rights[334]. - Geopolitical actions, such as those related to Russia's invasion of Ukraine, may limit patent application processes and enforcement, leading to potential loss of patent rights in affected regions[337]. - The European Unified Patent Court (UPC) became effective on June 1, 2023, allowing for centralized patent revocation proceedings, which could negatively impact the ability to enforce European patents[338]. - The company relies on trade secret protection and confidentiality agreements to safeguard proprietary information, but these measures may not be foolproof against breaches[339]. - Third-party intellectual property claims could delay or prevent the commercialization of product candidates, leading to potential litigation costs and resource diversion[341]. - The company is dependent on third-party licenses for certain intellectual property rights, and any failure to maintain these licenses could adversely affect business prospects[346]. - The patent protection for some product candidates is contingent on third parties, and any mismanagement in patent prosecution by licensors could hinder the ability to commercialize those candidates[350]. - Patent protection is contingent on compliance with various procedural and fee requirements, with potential loss of rights due to non-compliance[351]. - Failure to comply with licensing agreements could result in loss of important license rights, adversely affecting business operations[352]. - Legal proceedings to defend patents may be costly and time-consuming, with unpredictable outcomes impacting business stability[353][354]. - Interference proceedings may be necessary to determine patent rights, with unfavorable outcomes potentially harming business operations[355]. - Intellectual property litigation risks include potential disclosure of confidential information, which could negatively affect stock prices[356]. - Claims regarding wrongful use of confidential information by employees could lead to costly litigation and loss of intellectual property rights[357]. - Challenges to inventorship of patents may require litigation, risking valuable intellectual property rights and incurring substantial costs[358]. - Global protection of intellectual property rights is limited, with some jurisdictions offering less extensive protections than the U.S.[359][360]. Operational Risks - The company faces risks related to business disruptions that could harm operations and financial condition, including natural disasters and pandemics[239]. - The company relies on third-party manufacturers for clinical supplies, which poses risks related to compliance and supply chain disruptions[303][308]. - Manufacturing processes are complex and may lead to delays or product shortages, impacting clinical trial timelines[305][306]. - The company does not currently have the capability to manufacture its product candidates internally, relying on external vendors[309]. - The actual cost of manufacturing may not be commercially feasible, affecting the viability of product candidates[309]. - The company is facing challenges in attracting and retaining qualified personnel, which is critical for product development in the competitive biotechnology and pharmaceutical industries[385]. - There is a shortage of highly qualified personnel in the Massachusetts region, where the company primarily operates, leading to intense competition for talent[387]. - The company may need to expand its organization, which could disrupt operations and require significant capital expenditures[390]. - Macroeconomic conditions, including inflation and geopolitical events, may adversely affect the company's business and financial condition[391]. - The company is vulnerable to cybersecurity threats, which could disrupt clinical trials and harm its reputation and financial results[398]. - The company may not have adequate insurance coverage for security incidents, which could lead to significant financial losses[400]. Tax and Financial Structure Risks - The company's net operating loss carryforwards may be limited due to U.S. tax law restrictions, potentially affecting future tax liabilities[402]. - Changes in equity ownership could limit the company's ability to utilize pre-change net operating loss carryforwards[403]. - Changes in tax laws could adversely affect the company's business operations and financial performance, potentially impacting cash flow and results of operations[404]. - The effective tax rate may fluctuate due to various factors, including audits and changes in tax laws, which could lead to tax obligations exceeding accrued amounts[405]. - The company does not anticipate paying any cash dividends in the foreseeable future, with expectations to retain future earnings for business development[411]. - Future sales of shares by existing stockholders could lead to a decline in the stock price, particularly after legal restrictions on resale lapse[412]. - The principal stockholders own a significant percentage of the company's stock, allowing them to exert substantial control over matters requiring stockholder approval[415]. - The market price of the company's common stock has historically been volatile and may continue to experience significant fluctuations due to various factors[416]. - General market volatility, often unrelated to the company's operating performance, may adversely affect the trading price of its common stock[417]. - Class action securities litigation may arise following periods of market volatility, potentially resulting in substantial costs and diverting management attention[418].