Financial Data and Key Metrics Changes - As of September 30, 2022, cash, cash equivalents, and short-term investments were $431 million, compared to $197 million as of December 31, 2021, indicating a significant increase in liquidity [58] - The company believes that its current cash position will be sufficient to fund operations into the second half of 2025 [58] Business Line Data and Key Metrics Changes - The IV VRDN-001 program showed promising results, with a 75% proptosis responder rate compared to 56% for TEPEZZA, and a 4.0-point improvement in Clinical Activity Score (CAS) [22][35] - The 20 mg per kg cohort demonstrated a mean change in proptosis of 2.04 millimeters, which is higher than the mean changes reported for TEPEZZA [34] Market Data and Key Metrics Changes - The Thyroid Eye Disease (TED) market is currently valued at $2 billion in the U.S. and is expected to grow to over $4 billion globally [8] - The company aims to capture a significant share of this market with its differentiated product offerings [55] Company Strategy and Development Direction - The company is focused on delivering a complete portfolio of products for the treatment of TED, including both intravenous and subcutaneous options [7][55] - The Phase 3 program consists of two pivotal efficacy studies, THRIVE and THRIVE-2, expected to read out in mid and late 2024, respectively [45] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the efficacy of VRDN-001, highlighting its potential to offer a shorter treatment course and faster symptom relief compared to existing therapies [44] - The company is well-funded and positioned to advance its portfolio rapidly, with a strong foundation for future growth in the TED market [56] Other Important Information - The company reported that the 3 mg per kg cohorts for VRDN-001 are fully enrolled, with top-line data expected in early January 2023 [11] - VRDN-002 has shown a half-life of up to 43 days, significantly better than TEPEZZA, positioning it well for upcoming trials [15] Q&A Session Summary Question: Can you provide more color on the discrepancy in the percent of patients who achieve a CAS of zero or one between the 10 and 20 mg per kg cohorts? - Management explained that both cohorts had a higher percentage of patients achieving CAS of zero or one compared to TEPEZZA, with baseline characteristics influencing the observed differences [62] Question: Is there any scenario where you would consider adding a weekly dosing cohort for the subcutaneous TED proof-of-concept trial? - Management indicated confidence in the every-other-week dosing paradigm and is exploring the potential for even less frequent dosing based on upcoming data [74] Question: Can you clarify what the mean proptosis reduction was in the placebo patients with this updated data set? - The mean proptosis change in the placebo group was an improvement of 0.5 millimeters, consistent with TEPEZZA studies [80] Question: What can you say about the ongoing 3 mg/kg cohort at this point? Any color on safety or tolerability? - No serious adverse events have been reported in the 3 mg/kg cohort so far [84] Question: Could you offer any additional color on the hyperglycemia event in the 20 mg/kg cohort? - Management clarified that variability in glucose measures was consistent with the underlying diabetes condition of the patient and not drug-related [90] Question: Will you require a comparison to active drug in the Phase 3 study? - Management confirmed that the study design includes two active arms and one placebo arm, with no requirement for comparison to an active drug [92] Question: What gives you the confidence for selecting either VRDN-002 or VRDN-003 for Phase III development by early 2024? - Management stated that both candidates are expected to perform similarly in terms of efficacy, allowing for a robust choice based on pharmacokinetic data [100]

Viridian Therapeutics, Inc. (NASDAQ:VRDN) Q2 2022 Earnings Conference Call August 15, 2022 8:00 AM ET Company Participants John Jordan - VP of IR Jonathan Violin - President and CEO Barrett Katz - Chief Medical Officer, Raymond Douglas - Director of Thyroid Eye Disease Kristian Humer - CFO and Chief Business Officer Conference Call Participants Chris Howerton - Jefferies Thomas Smith - SVB Securities Gavin Clark-Gartner - Evercore ISI Rami Katkhuda - LifeSci Capital Laura Chico - Wedbush Securities Kalpit P ...

| --- | --- | |-----------------------------------------------------------------------------|-------| | | | | ENGINEERING MEDICINES | | | TO IMPROVE PATIENT CARE *VIRIDIAN VRDN-001 & VRDN-002 initial clinical data | | | August 15, 2022 | | 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, "anti ...

PART I. FINANCIAL INFORMATION [Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) The company reported a net loss of $55.2 million for the six months ended June 30, 2022, an increase from a $36.4 million loss in the same period of 2021, with total assets decreasing to $169.8 million from $203.7 million, primarily due to cash used in operations, ending the period with $161.2 million in cash, cash equivalents, and short-term investments [Condensed Consolidated Balance Sheets](index=5&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of June 30, 2022, total assets decreased to $169.8 million from $203.7 million, driven by reduced cash and investments, while total liabilities increased to $27.9 million from $16.0 million, and stockholders' equity decreased to $141.9 million from $187.7 million Condensed Consolidated Balance Sheet Data (in thousands) | Account | June 30, 2022 | December 31, 2021 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $31,293 | $42,299 | | Short-term investments | $129,914 | $154,666 | | Total current assets | $166,151 | $200,163 | | Total assets | $169,814 | $203,709 | | **Liabilities and Stockholders' Equity** | | | | Total current liabilities | $21,388 | $13,636 | | Long-term debt, net | $4,585 | $— | | Total liabilities | $27,916 | $15,993 | | Total stockholders' equity | $141,898 | $187,716 | | Total liabilities and stockholders' equity | $169,814 | $203,709 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) For the three and six months ended June 30, 2022, the company reported increased net losses of $29.5 million and $55.2 million, respectively, primarily driven by higher research and development expenses, which rose to $39.5 million for the six-month period Statement of Operations Highlights (in thousands) | Metric | Three Months Ended June 30, 2022 | Three Months Ended June 30, 2021 | Six Months Ended June 30, 2022 | Six Months Ended June 30, 2021 | | :--- | :--- | :--- | :--- | :--- | | Collaboration revenue - related party | $256 | $1,090 | $472 | $2,541 | | Research and development | $21,712 | $12,565 | $39,458 | $26,371 | | General and administrative | $8,108 | $6,523 | $16,467 | $12,683 | | Loss from operations | $(29,564) | $(17,998) | $(55,453) | $(36,513) | | Net loss | $(29,491) | $(17,964) | $(55,184) | $(36,424) | | Net loss per share, basic and diluted | $(1.06) | $(2.21) | $(2.05) | $(5.04) | [Condensed Consolidated Statements of Cash Flows](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) For the six months ended June 30, 2022, net cash used in operating activities was $39.1 million, offset by $22.8 million from investing and $5.3 million from financing, resulting in a net decrease of $11.0 million in cash and equivalents, ending at $31.3 million Cash Flow Summary (in thousands) | Activity | Six Months Ended June 30, 2022 | Six Months Ended June 30, 2021 | | :--- | :--- | :--- | | Net cash used in operating activities | $(39,128) | $(26,170) | | Net cash provided by (used in) investing activities | $22,826 | $(333) | | Net cash provided by financing activities | $5,296 | $8,311 | | **Net decrease in cash and cash equivalents** | **$(11,006)** | **$(18,192)** | | Cash and cash equivalents at end of period | $31,293 | $27,704 | [Notes to Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) The notes detail the company's business focus on developing treatments for Thyroid Eye Disease (TED) with its candidates VRDN-001, VRDN-002, and VRDN-003, confirming sufficient cash for at least the next twelve months while noting the need for additional capital, and outlining key accounting policies, collaboration agreements, debt facilities, and milestone payment commitments - The company is a biotechnology firm focused on treatments for Thyroid Eye Disease (TED), with **VRDN-001**, **VRDN-002**, and **VRDN-003** as its leading product candidates[28](index=28&type=chunk) - As of **June 30, 2022**, the company had an accumulated deficit of **$413.5 million** and expects to continue generating operating losses[29](index=29&type=chunk) - The company believes its current cash, cash equivalents, and short-term investments of approximately **$161.2 million** are sufficient to fund operations for at least the **next twelve months** from the financial statement issuance date[31](index=31&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 focus on developing Thyroid Eye Disease (TED) treatments with candidates VRDN-001, VRDN-002, and VRDN-003, noting positive VRDN-001 data in August 2022, attributing increased operating losses to higher R&D and G&A expenses, and confirming $161.2 million in cash as of June 30, 2022, sufficient to fund operations into 2024 [Overview and Recent Developments](index=35&type=section&id=Overview%20and%20Recent%20Developments) The company is advancing Thyroid Eye Disease (TED) treatments with VRDN-001, VRDN-002, and VRDN-003, having announced positive VRDN-001 Phase 1/2 data in August 2022, completed VRDN-002 Phase 1, and plans to advance a candidate to registrational trials by late 2023 - Announced positive initial clinical data from the first cohort of the VRDN-001 Phase 1/2 trial in TED patients, showing an **83% proptosis responder rate** and a **2.4mm mean reduction in proptosis at week 6**[154](index=154&type=chunk) - **VRDN-002**, designed for low-volume subcutaneous injection, completed a Phase 1 trial, with a subcutaneous proof of concept trial in TED patients planned to start in the **second half of 2023**[159](index=159&type=chunk) - The company plans to advance either **VRDN-002** or **VRDN-003** to registrational trials by the **end of 2023**, with a global Phase 3 program for a subcutaneous therapy expected to start in **early 2024**[161](index=161&type=chunk) [Results of Operations](index=39&type=section&id=Results%20of%20Operations) For the three and six months ended June 30, 2022, revenue decreased to $0.3 million and $0.5 million respectively, while R&D expenses significantly increased to $21.7 million and $39.5 million, and G&A expenses also rose, primarily due to higher clinical trial costs, personnel, and milestone fees Comparison of Operations for the Three Months Ended June 30 (in thousands) | Account | 2022 | 2021 | Change | | :--- | :--- | :--- | :--- | | Collaboration revenue | $256 | $1,090 | $(834) | | Research and development | $21,712 | $12,565 | $9,147 | | General and administrative | $8,108 | $6,523 | $1,585 | Comparison of Operations for the Six Months Ended June 30 (in thousands) | Account | 2022 | 2021 | Change | | :--- | :--- | :--- | :--- | | Collaboration revenue | $472 | $2,541 | $(2,069) | | Research and development | $39,458 | $26,371 | $13,087 | | General and administrative | $16,467 | $12,683 | $3,784 | - The increase in R&D expenses for the six months ended June 30, 2022 was driven by a **$5.1 million increase in milestone, license, and option fees** (including a **$3.0M payment to ImmunoGen** and a **$2.5M fee to Paragon**), a **$4.3 million increase in clinical trial expenses** for VRDN-001 and VRDN-002, and a **$3.4 million increase in personnel costs**[183](index=183&type=chunk) [Liquidity and Capital Resources](index=41&type=section&id=Liquidity%20and%20Capital%20Resources) As of June 30, 2022, the company held $161.2 million in cash and investments, sufficient to fund operations into 2024, having secured a $75.0 million loan agreement with Hercules Capital and an active $75.0 million ATM offering with Jefferies - The company had **$161.2 million** in cash, cash equivalents, and short-term investments as of **June 30, 2022**, which is expected to fund operations into **2024**[193](index=193&type=chunk) - In **April 2022**, the company entered into a loan and security agreement with Hercules Capital for up to **$75.0 million**, with an initial draw of **$5.0 million**[198](index=198&type=chunk) - The company has an active "at the market" (ATM) offering agreement with Jefferies to sell up to **$75.0 million** of common stock, but no shares had been sold under this agreement as of **June 30, 2022**[200](index=200&type=chunk) [Quantitative and Qualitative Disclosures about Market Risk](index=44&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) As a smaller reporting company, the company is exempt from providing quantitative and qualitative disclosures about market risk - As a smaller reporting company, Viridian is not required to provide quantitative and qualitative disclosures about market risk[207](index=207&type=chunk) [Controls and Procedures](index=44&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of the quarter-end, with no material changes to internal control over financial reporting during the most recent fiscal quarter - Management concluded that the company's disclosure controls and procedures were **effective** as of the end of the quarter covered by the report[210](index=210&type=chunk) - There were 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[211](index=211&type=chunk) PART II. OTHER INFORMATION [Legal Proceedings](index=45&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any legal proceedings expected to have a material adverse effect on its business, financial condition, or results of operations - The company reports that it is not currently involved in any **material legal proceedings**[213](index=213&type=chunk) [Risk Factors](index=45&type=section&id=Item%201A.%20Risk%20Factors) The company outlines significant financial, developmental, regulatory, and operational risks, including the need for additional capital, a history of losses, dependence on early-stage clinical candidates, uncertainties of clinical trials, reliance on third parties, competition, intellectual property, and healthcare reforms [Risks Related to Financial Condition and Capital Requirements](index=46&type=section&id=Risks%20Related%20to%20Our%20Financial%20Condition%20and%20Capital%20Requirements) This section details financial risks, including the critical need for additional capital to continue operations, a history of significant losses with an accumulated deficit of $413.5 million, and the potential for future capital raises to dilute ownership or impose restrictive covenants - The company will need to raise **additional capital** to fund operations and service obligations; failure to do so when needed would prevent it from continuing as a **going concern**[216](index=216&type=chunk) - The company has a history of net losses, with an **accumulated deficit of $413.5 million** as of **June 30, 2022**, and anticipates continued significant losses[221](index=221&type=chunk) - Raising additional capital may cause **dilution to stockholders**, restrict operations, or require relinquishing rights to technologies or product candidates[229](index=229&type=chunk) [Risks Related to Product Candidate Discovery and Development](index=50&type=section&id=Risks%20Related%20to%20the%20Discovery%20and%20Development%20of%20Our%20Product%20Candidates) This section covers inherent drug development risks, including costly and time-consuming clinical trials that may fail or cause side effects, heavy dependence on early-stage candidates where initial results may not predict future success, and potential delays from patient enrollment difficulties, especially for rare diseases - Clinical development is expensive and risky, with a high potential for **failure at any stage**, which could prevent or delay completion of trials[234](index=234&type=chunk) - The company is heavily dependent on the success of its product candidates, which are in **early stages of clinical development**, and cannot guarantee they will receive regulatory approval[241](index=241&type=chunk) - The company may face difficulties enrolling patients in clinical trials, particularly for **rare diseases**, which could delay or prevent the trials[247](index=247&type=chunk) [Risks Related to Regulatory Approval and Legal Compliance](index=56&type=section&id=Risks%20Related%20to%20Regulatory%20Approval%20of%20Our%20Product%20Candidates%20and%20Other%20Legal%20Compliance%20Matters) This section discusses regulatory hurdles, including potential biosimilar competition for biologics, no guarantee of expedited designations, ongoing regulatory requirements and healthcare reform impacts post-approval, and critical compliance with fraud, abuse, and data privacy laws to avoid substantial penalties - Product candidates regulated as biologics may face competition from **biosimilars** sooner than anticipated due to the BPCIA, which allows for an abbreviated approval pathway for such products[258](index=258&type=chunk) - The company may not receive expedited designations like **Orphan Drug**, **Breakthrough Therapy**, or **Fast Track**, and even if granted, these do not guarantee faster development or approval[261](index=261&type=chunk)[262](index=262&type=chunk)[265](index=265&type=chunk) - The company is subject to numerous foreign, federal, and state healthcare laws, including **anti-kickback**, **false claims**, and **data privacy regulations** (like GDPR), with non-compliance leading to significant penalties[273](index=273&type=chunk) [Risks Related to Reliance on Third Parties](index=60&type=section&id=Risks%20Related%20to%20Our%20Reliance%20on%20Third%20Parties) This section highlights dependencies on external partners, including vulnerability to health epidemics like COVID-19, reliance on CROs for clinical trials and CMOs for manufacturing, and the complex, difficult manufacturing process for biologics, given the company's lack of internal capabilities - The company relies on third-party **CROs** to conduct clinical trials and **CMOs** to manufacture product candidates; failure of these third parties to perform adequately could delay or halt development and commercialization[284](index=284&type=chunk)[286](index=286&type=chunk) - The manufacturing process for biologic product candidates is **complex** and susceptible to production loss, failure, or variation, which could delay or prevent supply for trials or commercialization[288](index=288&type=chunk) - Health epidemics like the **COVID-19 pandemic** could adversely affect the operations of the company or the third parties it relies on, potentially disrupting the supply chain and clinical trials[280](index=280&type=chunk)[281](index=281&type=chunk) [Risks Related to Intellectual Property](index=65&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) This section addresses intellectual property risks, including the uncertainty of obtaining and maintaining patent protection and trade secrets, potential challenges to patents, impact of U.S. patent law changes, risks of third-party infringement claims, and the need to comply with license agreement obligations to maintain product candidate rights - The company's success depends on its ability to obtain and maintain **patent protection and trade secrets**, but the patent process is uncertain and patents may be challenged or invalidated[301](index=301&type=chunk)[302](index=302&type=chunk) - **Third-party claims of intellectual property infringement** could prevent or delay development and commercialization efforts, leading to substantial litigation expense and potential damages[317](index=317&type=chunk) - The company relies on **licenses from third parties** for its product candidates and could lose these important rights if it fails to comply with its contractual obligations[327](index=327&type=chunk) [Risks Related to Commercialization](index=71&type=section&id=Risks%20Related%20to%20Commercialization%20of%20Our%20Product%20Candidates) This section details commercialization challenges, including the company's lack of experience in manufacturing, sales, and marketing, substantial competition from major pharmaceutical companies, and the dependence of commercial success on market acceptance by physicians, patients, and payors, along with adequate reimbursement - The company has **no experience in commercial manufacturing, sales, or marketing** and must either build these capabilities or rely on third-party collaborators to generate revenue[337](index=337&type=chunk) - The company faces substantial competition from well-established pharmaceutical companies with greater financial and technical resources, including **Horizon Therapeutics**, **Immunovant**, and **Novartis** in the TED space[342](index=342&type=chunk)[344](index=344&type=chunk) - Commercial success depends on **market acceptance** and **adequate reimbursement** from third-party payors, which is uncertain[348](index=348&type=chunk)[352](index=352&type=chunk) [Risks Related to Business Operations](index=75&type=section&id=Risks%20Related%20to%20Our%20Business%20Operations) This section covers operational risks, including high dependence on the CEO and key personnel, challenges in managing growth, restrictive covenants in the Hercules loan agreement, exposure to IT system failures and data breaches, and potential limitations on net operating loss (NOL) carryforwards due to ownership changes - The company is highly dependent on its **President and CEO, Dr. Jonathan Violin**, and faces intense competition for qualified personnel[355](index=355&type=chunk) - The **Hercules Loan and Security Agreement** contains covenants that could restrict operations and, in an event of default, could force accelerated repayment of debt[358](index=358&type=chunk)[359](index=359&type=chunk) - The ability to use **net operating loss (NOL) carryforwards** to offset future taxable income may be limited by **Section 382 of the Internal Revenue Code** if the company undergoes an "ownership change"[367](index=367&type=chunk)[368](index=368&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=82&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This item is not applicable for the reporting period - Not applicable[391](index=391&type=chunk) [Defaults Upon Senior Securities](index=82&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This item is not applicable for the reporting period - Not applicable[392](index=392&type=chunk) [Mine Safety Disclosures](index=82&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable for the reporting period - Not applicable[393](index=393&type=chunk) [Other Information](index=82&type=section&id=Item%205.%20Other%20Information) This item is not applicable for the reporting period - Not applicable[394](index=394&type=chunk) [Exhibits](index=83&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the quarterly report, including corporate governance documents, material contracts like the Hercules Loan and Security Agreement and lease amendments, CEO and CFO certifications, and XBRL data files - Filed exhibits include the **Second Amendment to the Massachusetts lease**, the **Amended & Restated 2016 Equity Incentive Plan**, and **officer certifications**[396](index=396&type=chunk) - The exhibit list incorporates by reference previously filed documents, such as the **Certificate of Incorporation**, **Bylaws**, and the **Hercules Loan and Security Agreement**[396](index=396&type=chunk)

Viridian Therapeutics May 2022 %VIRIDIAN Cautionary note regarding forward-looking statements This presentation contains forward-looking statements relating to Viridian Therapeutics, Inc., including statements about our plans to obtain funding, develop and commercialize our therapeutic candidates, our planned clinical trials, the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates, the clinical utility of our therapeutic candidates and our intellectual proper ...

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, 2022 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 jurisd ...

Financial Data and Key Metrics Changes - The company ended Q1 2022 with $175 million in cash, cash equivalents, and short-term investments, positioning it strongly for future program advancements [32] - Research and development expenses increased to $17.7 million in Q1 2022 from $13.8 million in the same period last year, driven by personnel-related costs and clinical trial expenses [35] - General and administrative expenses rose to $8.4 million in Q1 2022 compared to $6.2 million in the prior year, attributed to personnel-related costs and consulting expenses [36] - The net loss for Q1 2022 was $25.7 million, up from $18.5 million in the same quarter of 2021, primarily due to increased operating costs and lower collaboration revenue [37] Business Line Data and Key Metrics Changes - The company is advancing its lead candidates for Thyroid Eye Disease (TED), specifically VRDN-001 and VRDN-002, with significant progress in clinical trials [9][25] - VRDN-001 is positioned as a differentiated intravenous product with higher affinity and potency compared to TEPEZZA, the only FDA-approved therapy for TED [10][22] - VRDN-002 is designed for convenient subcutaneous administration and incorporates half-life extension technology, potentially allowing for less frequent dosing [25][26] Market Data and Key Metrics Changes - The TED market is rapidly growing, currently annualizing at $2 billion in the U.S. just two years post-launch of TEPEZZA [21][22] - The company aims to establish VRDN-001 and VRDN-002 as meaningful therapeutic advancements in treating TED, competing across all settings of care [29] Company Strategy and Development Direction - The company is preparing VRDN-001 for pivotal trials, assuming positive proof-of-concept data, and plans to assess multiple dosing regimens in parallel [23] - The strategy includes advancing both intravenous and subcutaneous options for TED, with a focus on reducing patient burden through less frequent dosing [25][29] - The company is also expanding its pipeline with next-generation antibodies for other indications, while maintaining a strong focus on the TED portfolio [30] Management's Comments on Operating Environment and Future Outlook - Management expressed excitement for upcoming data readouts that are expected to confirm the potential of their antibodies in treating TED [8] - The company is confident in its enrollment projections for ongoing trials and anticipates sharing top-line data in the third quarter [13][17] - Management highlighted the importance of demonstrating proof-of-concept data similar to TEPEZZA as a major value-creating event [22] Other Important Information - The company entered into a credit facility with Hercules for up to $75 million, enhancing its financial strength and operational flexibility [33][34] - The ongoing trials for VRDN-001 and VRDN-002 are designed to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics, with top-line data expected soon [27][28] Q&A Session Summary Question: Clarification on healthy volunteers data - Management clarified that the data presented is from an interim analysis and that the top dose cohort is still ongoing, affecting the data consistency observed [42] Question: Dosing comparison between VRDN-001 and VRDN-002 - Management indicated that while both molecules have similar receptor affinity, pharmacokinetics will differ, with VRDN-002 expected to have a longer half-life due to its design [44] Question: Impact of competitor's subcutaneous formulation - Management discussed the potential implications of a competitor's high concentration formulation but emphasized their focus on developing a low-volume, less frequent subcutaneous product [47][51] Question: Safety data on hypertension and hyperglycemia - Management noted that reported cases of hypertension and hyperglycemia were mild and deemed unrelated to the study drug [54] Question: Patient enrollment and study design - Management confirmed that enrollment is proceeding as expected, with a focus on enrolling patients likely to benefit from the treatment [56][60] Question: Plans for subcutaneous formulation of VRDN-001 - Management expressed optimism about advancing a subcutaneous formulation quickly, with no significant hurdles anticipated [61] Question: Timing for VRDN-002 data readout - Management clarified that the readout for VRDN-002 is now expected in the third quarter, with dose escalation completed [67] Question: Pharmacokinetic data for VRDN-001 - Management stated that pharmacokinetic data is still being collected, but initial results are promising and consistent with previous oncology data [68][70] Question: Dosing frequency for subcutaneous formulation - Management indicated that it is too early to determine the exact dosing frequency for a subcutaneous formulation, pending further data [84]

Viridian Therapeutics April 2022 %VIRIDIAN Cautionary note regarding forward-looking statements This presentation contains forward-looking statements relating to Viridian Therapeutics, Inc., including statements about our plans to obtain funding, develop and commercialize our therapeutic candidates, our planned clinical trials, the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates, the clinical utility of our therapeutic candidates and our intellectual prop ...

Viridian Therapeutics March 2022 %VIRIDIAN Cautionary note regarding forward-looking statements This presentation contains forward-looking statements relating to Viridian Therapeutics, Inc., including statements about our plans to obtain funding, develop and commercialize our therapeutic candidates, our planned clinical trials, the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates, the clinical utility of our therapeutic candidates and our intellectual prop ...

Part I [Item 1. Business](index=5&type=section&id=ITEM%201.%20BUSINESS) The company develops 'fast-follower' therapies for serious diseases, with a primary focus on Thyroid Eye Disease (TED) [Company Overview and Strategy](index=5&type=section&id=Company%20Overview%20and%20Strategy) The company's strategy is to develop 'fast-follower' and 'bio-superior' therapies for clinically validated targets - Viridian's business model is to develop 'fast-follower' and 'bio-superior' therapies for diseases with existing proof-of-concept, aiming to improve upon first-generation drugs in efficacy, safety, or convenience[18](index=18&type=chunk) - The company's primary focus is on **Thyroid Eye Disease (TED)**, a debilitating autoimmune disease[21](index=21&type=chunk)[28](index=28&type=chunk) - Key strategic elements include rapidly advancing VRDN-001 for quick market entry, developing the more convenient subcutaneous VRDN-002, and expanding the portfolio beyond TED[29](index=29&type=chunk) [Thyroid Eye Disease (TED) and Market Potential](index=7&type=section&id=Thyroid%20Eye%20Disease%20(TED)%20and%20Market%20Potential) TED is a sight-threatening autoimmune disorder with a significant market validated by the success of Tepezza® - TED is a sight-threatening autoimmune disorder causing inflammation, proptosis (bulging eyes), double vision, and potential blindness[31](index=31&type=chunk)[32](index=32&type=chunk) - The only FDA-approved therapy for TED is Horizon Therapeutics' Tepezza® (teprotumumab), an anti-IGF-1R antibody, which **validates the mechanism of action** for Viridian's candidates[21](index=21&type=chunk)[37](index=37&type=chunk) TED Market Potential | Metric | Value | | :--- | :--- | | Annual Incidence (U.S.) | Over 60,000 patients | | Patients Requiring IV Treatment (U.S.) | 20,000 - 25,000 annually | | Tepezza® 2021 Net Sales | $1.66 billion | [Product Candidates](index=8&type=section&id=Product%20Candidates) The company's pipeline is led by VRDN-001 (intravenous) and VRDN-002 (subcutaneous) for Thyroid Eye Disease - **VRDN-001:** The most advanced program, an intravenous (IV) anti-IGF-1R monoclonal antibody, with initial Phase 1/2 data expected in Q2 2022[23](index=23&type=chunk)[46](index=46&type=chunk) - **VRDN-002:** A next-generation anti-IGF-1R antibody designed for low-volume subcutaneous injection, with initial Phase 1 data expected mid-2022[24](index=24&type=chunk)[47](index=47&type=chunk) - **VRDN-003:** An exploratory program evaluating hypotheses that may yield a product profile differentiated beyond VRDN-002[48](index=48&type=chunk) [Intellectual Property, Competition, and Licensing](index=10&type=section&id=Intellectual%20Property%2C%20Competition%2C%20and%20Licensing) The company protects its candidates with patent applications and navigates a competitive landscape through strategic licensing - As of December 31, 2021, the company has multiple patent applications for VRDN-001 and VRDN-002, with potential **expirations no earlier than 2041**[49](index=49&type=chunk)[50](index=50&type=chunk) - Key competitors in the TED space include **Horizon Therapeutics plc (Tepezza®)**, Immunovant, Inc, Novartis International AG, Harbour BioMed, and Valenza Bio Inc[51](index=51&type=chunk) - The company has key licensing agreements with ImmunoGen for VRDN-001 technology and Xencor for IGF-1R antibody technology[52](index=52&type=chunk)[54](index=54&type=chunk)[55](index=55&type=chunk) [Government Regulation and Manufacturing](index=12&type=section&id=Government%20Regulation%20and%20Manufacturing) The company is subject to extensive government regulation and relies entirely on third-party contract manufacturing - The company is subject to extensive regulation by the FDA and comparable foreign authorities, governing all stages of product development[58](index=58&type=chunk)[83](index=83&type=chunk) - The company may seek expedited development pathways such as **Orphan Drug Designation, Fast Track, and Breakthrough Therapy Designation**[73](index=73&type=chunk)[76](index=76&type=chunk)[78](index=78&type=chunk) - Viridian does not own manufacturing facilities and is **entirely dependent on third-party CDMOs** for the production of its product candidates[124](index=124&type=chunk)[125](index=125&type=chunk) [Item 1A. Risk Factors](index=24&type=section&id=ITEM%201A.%20RISK%20FACTORS) The company faces significant financial, clinical development, third-party reliance, and competitive risks - **Financial Risk:** The company has a history of losses (**$79.4 million in 2021**) and an accumulated deficit of **$358.3 million**, requiring additional capital to fund operations beyond 2024[133](index=133&type=chunk)[137](index=137&type=chunk)[138](index=138&type=chunk) - **Clinical Development Risk:** The company is heavily dependent on the success of its early-stage product candidates, and clinical trial outcomes are uncertain[153](index=153&type=chunk)[159](index=159&type=chunk)[164](index=164&type=chunk) - **Third-Party Reliance Risk:** Viridian relies on CROs and CMOs to conduct clinical trials and manufacture its products, and any failure by these parties could harm development[194](index=194&type=chunk)[196](index=196&type=chunk) - **Commercial and Competitive Risk:** The company faces substantial competition from larger companies, and success depends on market acceptance, pricing, and reimbursement[251](index=251&type=chunk)[256](index=256&type=chunk) [Item 1B, 2, 3, 4](index=59&type=section&id=ITEM%201B%2C%202%2C%203%2C%204) The company reports no unresolved SEC comments, leases office space, and has no material legal proceedings - The company has no unresolved SEC staff comments[296](index=296&type=chunk) - Leased properties include approximately 4,371 sq ft of office space in Waltham, MA and 27,128 sq ft of office and lab space in Boulder, CO[297](index=297&type=chunk) - The company is not currently a party to any material legal proceedings[298](index=298&type=chunk) Part II [Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=60&type=section&id=ITEM%205.%20MARKET%20FOR%20REGISTRANT'S%20COMMON%20EQUITY%2C%20RELATED%20STOCKHOLDER%20MATTERS%20AND%20ISSUER%20PURCHASES%20OF%20EQUITY%20SECURITIES) The company's stock trades on Nasdaq, it does not pay dividends, and it recently issued shares in a private placement - Common stock is traded on The Nasdaq Capital Market under the symbol **"VRDN"**[302](index=302&type=chunk) - The company has never paid dividends and does not anticipate paying them in the foreseeable future, retaining earnings for business development[303](index=303&type=chunk) - On December 2, 2021, the company issued **394,737 shares** of common stock to Xencor in a private placement as an upfront payment for licensed rights[304](index=304&type=chunk) [Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=60&type=section&id=ITEM%207.%20MANAGEMENT'S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) The company's net loss decreased in 2021 due to a non-recurring R&D expense, while operating expenses increased [Results of Operations](index=65&type=section&id=Results%20of%20Operations) Operational results for 2021 show a lower net loss compared to 2020, driven by a non-recurring IPR&D expense Comparison of Years Ended December 31, 2021 and 2020 (in thousands) | Financial Metric | 2021 | 2020 | Change | | :--- | :--- | :--- | :--- | | Revenue | $2,963 | $1,050 | $1,913 | | Research and development expenses | $56,886 | $28,304 | $28,582 | | General and administrative expenses | $25,805 | $13,265 | $12,540 | | Acquired in-process R&D expense | $0 | $69,861 | ($69,861) | | Net loss | ($79,413) | ($110,715) | $31,302 | - The **$28.6 million increase in R&D expenses** in 2021 was primarily due to a $22.8 million increase in preclinical outsourcing and manufacturing for VRDN programs[343](index=343&type=chunk) - The **$12.5 million increase in G&A expenses** in 2021 was mainly driven by a $9.6 million increase in personnel-related costs due to higher headcount[346](index=346&type=chunk) - Acquired IPR&D expense of **$69.9 million** was recorded in 2020 related to the acquisition of Private Viridian, with no corresponding expense in 2021[345](index=345&type=chunk) [Liquidity and Capital Resources](index=66&type=section&id=Liquidity%20and%20Capital%20Resources) The company has sufficient cash to fund operations into 2024, supported by recent financing activities - As of December 31, 2021, the company had **$197.0 million in cash**, cash equivalents, and short-term investments, which is expected to fund planned operations into 2024[347](index=347&type=chunk) - The company has an accumulated deficit of **$358.3 million** as of December 31, 2021[348](index=348&type=chunk) Cash Flow Summary (in thousands) | Activity | Year Ended Dec 31, 2021 | Year Ended Dec 31, 2020 | | :--- | :--- | :--- | | Net cash used in operating activities | ($54,581) | ($29,779) | | Net cash used in investing activities | ($74,292) | ($50,481) | | Net cash provided by financing activities | $125,275 | $101,311 | - Financing activities in 2021 were primarily driven by net proceeds of **$107.3 million** from the sale of common stock and **$15.7 million** from the sale of Series B Preferred Stock[363](index=363&type=chunk) [Item 7A. Quantitative and Qualitative Disclosures About Market Risk](index=69&type=section&id=ITEM%207A.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURES%20ABOUT%20MARKET%20RISK) As a smaller reporting company, this disclosure is not required - The company is a smaller reporting company and is not required to provide quantitative and qualitative disclosures about market risk[365](index=365&type=chunk) [Item 8. Financial Statements and Supplementary Data](index=69&type=section&id=ITEM%208.%20FINANCIAL%20STATEMENTS%20AND%20SUPPLEMENTARY%20DATA) This section presents the company's audited consolidated financial statements and related notes for 2021 and 2020 [Report of Independent Registered Public Accounting Firm](index=77&type=section&id=Report%20of%20Independent%20Registered%20Public%20Accounting%20Firm) The auditor issued a clean opinion and identified accrued clinical trial costs as a critical audit matter - The auditor, KPMG LLP, issued an opinion that the consolidated financial statements **present fairly**, in all material respects, the financial position of the company[400](index=400&type=chunk) - A critical audit matter was identified related to the evaluation of **accrued outsourced clinical trials and preclinical studies** due to subjective judgment required[406](index=406&type=chunk)[407](index=407&type=chunk) [Consolidated Financial Statements](index=80&type=section&id=Consolidated%20Financial%20Statements) This section provides key data from the company's balance sheets and statements of operations Consolidated Balance Sheet Data (in thousands) | Metric | Dec 31, 2021 | Dec 31, 2020 | | :--- | :--- | :--- | | Cash, cash equivalents, and short-term investments | $196,965 | $127,639 | | Total Assets | $203,709 | $131,255 | | Total Liabilities | $15,993 | $11,218 | | Total Stockholders' Equity | $187,716 | $120,037 | Consolidated Statement of Operations Data (in thousands) | Metric | Year Ended Dec 31, 2021 | Year Ended Dec 31, 2020 | | :--- | :--- | :--- | | Total Revenue | $2,963 | $1,050 | | Research and Development | $56,886 | $28,304 | | General and Administrative | $25,805 | $13,265 | | Net Loss | ($79,413) | ($110,715) | | Net Loss Per Share | ($6.66) | ($31.13) | [Notes to Consolidated Financial Statements](index=87&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) This section details key accounting policies, including asset acquisition and revenue recognition - The October 2020 acquisition of Private Viridian was accounted for as an asset acquisition, resulting in a **$69.9 million charge for acquired IPR&D**[485](index=485&type=chunk)[486](index=486&type=chunk) - In 2021, the company recognized **$3.0 million in collaboration revenue** related to its license agreement with Zenas BioPharma[490](index=490&type=chunk) - As of December 31, 2021, the company had federal net operating loss carryforwards of approximately **$157.8 million**[555](index=555&type=chunk)[556](index=556&type=chunk) [Item 9. Changes in and Disagreements With Accountants on Accounting and Financial Disclosure](index=69&type=section&id=ITEM%209.%20CHANGES%20IN%20AND%20DISAGREEMENTS%20WITH%20ACCOUNTANTS%20ON%20ACCOUNTING%20AND%20FINANCIAL%20DISCLOSURE) The company reported no disagreements with its accountants on accounting and financial disclosure - None[367](index=367&type=chunk) [Item 9A. Controls and Procedures](index=69&type=section&id=ITEM%209A.%20CONTROLS%20AND%20PROCEDURES) Management concluded that disclosure controls and internal controls over financial reporting were effective - Management concluded that the company's disclosure controls and procedures were **effective** as of December 31, 2021[368](index=368&type=chunk) - Based on an evaluation using the COSO framework, management concluded that the company's internal control over financial reporting was **effective** as of December 31, 2021[371](index=371&type=chunk)[373](index=373&type=chunk) [Item 9B. Other Information](index=71&type=section&id=ITEM%209B.%20OTHER%20INFORMATION) The company updated its registered agent and office in Delaware - On March 9, 2022, the company changed its registered agent and registered office in Delaware[375](index=375&type=chunk) Part III [Items 10, 11, 12, 13, and 14](index=72&type=section&id=ITEMS%2010%2C%2011%2C%2012%2C%2013%2C%20and%2014) Required information on governance and compensation is incorporated by reference from the upcoming proxy statement - Information for Items 10, 11, 12, 13, and 14 is **incorporated by reference** from the company's 2022 Proxy Statement[380](index=380&type=chunk)[382](index=382&type=chunk)[383](index=383&type=chunk) Part IV [Item 15. Exhibits, Financial Statement Schedules](index=73&type=section&id=ITEM%2015.%20EXHIBITS%2C%20FINANCIAL%20STATEMENT%20SCHEDULES) This section lists the financial statements and exhibits filed with the annual report - This item contains the index to the consolidated financial statements and a list of all exhibits filed with the report[386](index=386&type=chunk)[387](index=387&type=chunk) [Item 16. Form 10-K Summary](index=75&type=section&id=ITEM%2016.%20FORM%2010-K%20SUMMARY) The company has not provided a Form 10-K summary - None[395](index=395&type=chunk)