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

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

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) VIRIDIAN THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 47-1187261 (State or other jurisdiction of incorporation or organization) (I.R.S. Employer Identification No.) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXC ...

[PART I. FINANCIAL INFORMATION](index=5&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) For the six months ended June 30, 2023, Viridian Therapeutics reported a net loss of $123.2 million, a significant increase from the $55.2 million loss in the same period of 2022, driven by substantial rises in R&D and G&A expenses [Condensed Consolidated Balance Sheets](index=5&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of June 30, 2023, total assets were $351.5 million, a decrease from $435.1 million at December 31, 2022, primarily due to a reduction in cash, cash equivalents, and short-term investments Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2023 | December 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $87,082 | $155,579 | | Short-term investments | $247,209 | $268,971 | | **Total Assets** | **$351,467** | **$435,091** | | **Liabilities & Equity** | | | | Total Liabilities | $35,610 | $40,027 | | Total Stockholders' Equity | $315,857 | $395,064 | | **Total Liabilities & Stockholders' Equity** | **$351,467** | **$435,091** | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) The company reported a net loss of $55.1 million for the three months and $123.2 million for the six months ended June 30, 2023, representing a significant increase in losses compared to the same periods in 2022, driven by higher research and development and general and administrative expenses Statement of Operations Highlights (in thousands, except per share data) | Metric | Q2 2023 | Q2 2022 | 6 Months 2023 | 6 Months 2022 | | :--- | :--- | :--- | :--- | :--- | | Collaboration Revenue | $72 | $256 | $170 | $472 | | Research and Development | $40,083 | $21,712 | $90,823 | $39,458 | | General and Administrative | $19,264 | $8,108 | $41,095 | $16,467 | | Loss from Operations | $(59,275) | $(29,564) | $(131,748) | $(55,453) | | Net Loss | $(55,063) | $(29,491) | $(123,214) | $(55,184) | | Net Loss Per Share | $(1.27) | $(1.06) | $(2.88) | $(2.05) | [Condensed Consolidated Statements of Cash Flows](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) For the six months ended June 30, 2023, net cash used in operating activities was $107.2 million, with net cash provided by investing activities of $27.4 million and financing activities of $11.3 million, resulting in a net decrease in cash and cash equivalents of $68.5 million Cash Flow Summary for Six Months Ended June 30 (in thousands) | Activity | 2023 | 2022 | | :--- | :--- | :--- | | Net Cash Used in Operating Activities | $(107,187) | $(39,128) | | Net Cash Provided by Investing Activities | $27,418 | $22,826 | | Net Cash Provided by Financing Activities | $11,272 | $5,296 | | **Net Decrease in Cash** | **$(68,497)** | **$(11,006)** | [Notes to Condensed Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) The notes detail the company's rare disease focus, particularly VRDN-001 for TED, confirm sufficient cash to fund operations for at least the next twelve months, and outline key agreements including collaborations, license obligations, and an upsized term loan facility - The company is a biopharmaceutical firm focused on rare diseases, with its most advanced program, VRDN-001, targeting Thyroid Eye Disease (TED) - As of June 30, 2023, the company had an accumulated deficit of **$611.4 million** and expects to continue generating operating losses. However, with approximately **$334.3 million** in cash, cash equivalents, and short-term investments, management expects to fund operations for at least the **next twelve months**[26](index=26&type=chunk)[28](index=28&type=chunk) - In April 2022, the company entered into a loan agreement with Hercules Capital for up to **$75.0 million**, drawing an initial **$5.0 million**. In August 2023, this was amended to increase the available principal to **$150.0 million**, with an additional **$15.0 million** drawn[79](index=79&type=chunk)[82](index=82&type=chunk)[85](index=85&type=chunk) - The company has several key license agreements with potential future milestone payments, including up to **$48.0 million** to ImmunoGen, and up to **$45.0 million** to Enable Injections, plus potential commercial milestones and royalties for both[97](index=97&type=chunk)[102](index=102&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=34&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management highlights significant progress in clinical programs for Thyroid Eye Disease (TED), with a net loss increase to $123.2 million for the first six months of 2023, and expects current cash of $334.3 million to fund operations into the second half of 2025 [Overview and Recent Developments](index=34&type=section&id=Overview%20and%20Recent%20Developments) The company focuses on developing best-in-class medicines for rare diseases, with its lead program VRDN-001 targeting Thyroid Eye Disease (TED), showing positive clinical proof-of-concept data and advancing into Phase 3 trials, while also progressing subcutaneous candidates - VRDN-001 has established clinical proof-of-concept in both active and chronic Thyroid Eye Disease (TED)[140](index=140&type=chunk) - Key upcoming milestones include topline results from the THRIVE Phase 3 trial (active TED) in **mid-2024** and the THRIVE-2 Phase 3 trial (chronic TED) by **year-end 2024**[140](index=140&type=chunk) - The company is advancing **three subcutaneous (SC) candidates** and plans to select a lead program by **year-end 2023** to move into a pivotal trial in **mid-2024**[141](index=141&type=chunk)[144](index=144&type=chunk) [Results of Operations](index=38&type=section&id=Results%20of%20Operations) For the three and six months ended June 30, 2023, the company experienced a significant increase in operating expenses, primarily driven by higher R&D costs due to milestone/license fees, manufacturing, and personnel, alongside increased G&A expenses from personnel costs Comparison of Operating Expenses - Three Months Ended June 30 (in thousands) | Expense Category | 2023 | 2022 | Increase | | :--- | :--- | :--- | :--- | | Research and development | $40,083 | $21,712 | $18,371 | | General and administrative | $19,264 | $8,108 | $11,156 | - The Q2 2023 increase in R&D expenses was driven by higher costs for manufacturing (**$4.5M**), clinical trials (**$5.0M**), personnel (**$5.0M**), and milestone fees (**$2.0M**)[163](index=163&type=chunk) Comparison of Operating Expenses - Six Months Ended June 30 (in thousands) | Expense Category | 2023 | 2022 | Increase | | :--- | :--- | :--- | :--- | | Research and development | $90,823 | $39,458 | $51,365 | | General and administrative | $41,095 | $16,467 | $24,628 | - The six-month increase in R&D expenses was primarily due to a **$14.5M** net increase in milestone/license fees, a **$12.3M** increase in manufacturing costs, and a **$9.7M** increase in personnel costs[169](index=169&type=chunk) [Liquidity and Capital Resources](index=40&type=section&id=Liquidity%20and%20Capital%20Resources) As of June 30, 2023, the company had $334.3 million in cash, cash equivalents, and short-term investments, which management believes is sufficient to fund operations into the second half of 2025, despite an accumulated deficit of $611.4 million and the need for substantial additional capital - The company has **$334.3 million** in cash, cash equivalents, and short-term investments as of June 30, 2023, which is expected to fund operations into the **second half of 2025**[173](index=173&type=chunk) - The company has an accumulated deficit of **$611.4 million** as of June 30, 2023, and has not generated any revenue from product sales[174](index=174&type=chunk) - In August 2023, the company amended its loan agreement with Hercules Capital, increasing the total available principal from **$75.0 million** to **$150.0 million** and drew an additional **$15.0 million**[181](index=181&type=chunk) - The company has a **$175.0 million** At-The-Market (ATM) agreement with Jefferies, under which no shares were sold in the first six months of 2023[182](index=182&type=chunk) [Quantitative and Qualitative Disclosures about Market Risk](index=43&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) As a smaller reporting company, Viridian Therapeutics is not required to provide the information for this item - The company is a smaller reporting company and is not required to provide quantitative and qualitative disclosures about market risk[192](index=192&type=chunk) [Controls and Procedures](index=43&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of June 30, 2023, with no material changes in internal control over financial reporting during the most recent fiscal quarter - Management concluded that the company's disclosure controls and procedures were effective at a reasonable level of assurance as of the end of the quarter[194](index=194&type=chunk) - No changes in internal control over financial reporting occurred during the quarter that have materially affected, or are reasonably likely to materially affect, these controls[195](index=195&type=chunk) [PART II. OTHER INFORMATION](index=43&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Legal Proceedings](index=43&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any legal proceedings that it believes would have a material adverse effect on its business, financial condition, or results of operations - The company is not currently involved in any material legal proceedings[197](index=197&type=chunk) [Risk Factors](index=43&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks related to its financial condition, the uncertain outcomes of product development and clinical trials, regulatory approvals, reliance on third parties, and intellectual property protection [Risks Related to Financial Condition and Capital Requirements](index=44&type=section&id=Risks%20Related%20to%20Our%20Financial%20Condition%20and%20Capital%20Requirements) The company has a history of significant losses, with an accumulated deficit of $611.4 million as of June 30, 2023, and will need to raise substantial additional capital, with potential future financing causing dilution or operational restrictions - The company will need to raise additional capital to fund operations and service obligations; failure to do so when needed could prevent it from continuing as a going concern[199](index=199&type=chunk) - The company has a limited operating history, has historically incurred significant losses (**$611.4M** accumulated deficit as of June 30, 2023), and anticipates continued losses[203](index=203&type=chunk) - Raising additional capital may cause dilution to stockholders, involve restrictive debt covenants, or require relinquishing valuable rights to technologies or product candidates on unfavorable terms[213](index=213&type=chunk)[214](index=214&type=chunk)[215](index=215&type=chunk) [Risks Related to Product Candidate Discovery and Development](index=49&type=section&id=Risks%20Related%20to%20the%20Discovery%20and%20Development%20of%20Our%20Product%20Candidates) The company's success is heavily dependent on its early-stage product candidates, with clinical trials being expensive, time-consuming, and uncertain, carrying a high risk of failure, potential for undesirable side effects, and challenges in patient enrollment for rare diseases - Clinical development is expensive, time-consuming, and involves significant risk; a failure of one or more clinical trials can occur at any stage[219](index=219&type=chunk) - The company is heavily dependent on the success of its product candidates, which are in early stages of development, and cannot guarantee that data will be sufficient for regulatory approval[226](index=226&type=chunk) - Early positive results from preclinical studies and clinical trials are not predictive of future results in larger, later-stage trials[230](index=230&type=chunk) - Difficulty enrolling and maintaining patients in clinical trials for rare diseases could delay or prevent the completion of these trials[232](index=232&type=chunk) [Risks Related to Regulatory Approval and Legal Compliance](index=54&type=section&id=Risks%20Related%20to%20Regulatory%20Approval%20of%20Our%20Product%20Candidates%20and%20Other%20Legal%20Compliance%20Matters) The company's biologic product candidates may face competition from biosimilars, may not receive special regulatory designations, and will be subject to extensive ongoing regulatory requirements and healthcare reform measures, alongside stringent fraud, abuse, and data privacy laws - Product candidates regulated as biologics may face competition from biosimilars sooner than anticipated under the BPCIA, which allows for an abbreviated approval pathway for competing products[241](index=241&type=chunk) - The company may not receive or maintain expedited regulatory designations such as Orphan Drug, Breakthrough Therapy, or Fast Track, which could hinder development timelines[244](index=244&type=chunk)[245](index=245&type=chunk)[246](index=246&type=chunk) - Even if a product is approved, the company will remain subject to ongoing and extensive regulatory requirements for manufacturing, labeling, and marketing, and failure to comply can lead to significant penalties[250](index=250&type=chunk) - The company is subject to various federal and state healthcare laws, including anti-kickback, false claims, and data privacy regulations (e.g., GDPR), with non-compliance carrying substantial penalties[255](index=255&type=chunk) [Risks Related to Reliance on Third Parties](index=59&type=section&id=Risks%20Related%20to%20Our%20Reliance%20on%20Third%20Parties) Viridian heavily relies on third-party CROs for clinical trials and CMOs for manufacturing, lacking internal capabilities, making it vulnerable to supply chain disruptions, regulatory non-compliance, and complex biologic manufacturing issues that could harm development and commercialization - The company relies on third-party CROs for clinical trials and CMOs for manufacturing, and is responsible for their compliance with regulatory standards like GCP and cGMP[262](index=262&type=chunk) - The company lacks internal manufacturing capabilities and relies on a limited number of third-party suppliers, making it vulnerable to supply chain disruptions which could delay clinical trials[266](index=266&type=chunk)[271](index=271&type=chunk) - The manufacturing process for biologic product candidates is complex and prone to issues like contamination or variability, which could lead to product loss, delays, and harm the company's reputation[267](index=267&type=chunk)[268](index=268&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) In May and July 2023, the company issued a total of 243,902 shares of common stock to two accredited investors as partial consideration for certain licenses and rights, relying on the exemption from registration provided by Section 4(a)(2) of the Securities Act - On May 22, 2023, and July 14, 2023, the company issued a combined **243,902 shares** of common stock to two accredited investors as partial consideration for licenses and rights granted to the company[377](index=377&type=chunk) [Defaults Upon Senior Securities](index=82&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) Not applicable - There were no defaults upon senior securities during the period[378](index=378&type=chunk) [Mine Safety Disclosures](index=82&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) Not applicable - The company has no mine safety disclosures to report[379](index=379&type=chunk) [Other Information](index=83&type=section&id=Item%205.%20Other%20Information) On August 7, 2023, the company amended its Loan and Security Agreement with Hercules Capital, Inc., increasing the total term loan facility from $75 million to $150 million, secured by substantially all of the company's assets excluding intellectual property - On August 7, 2023, the company amended its loan agreement with Hercules Capital, increasing the aggregate term loan facility available from **$75 million** to **$150 million**[380](index=380&type=chunk) [Exhibits](index=83&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the quarterly report, including corporate governance documents, material contracts such as the amended loan agreement, and certifications from the principal executive and financial officers - The report includes several exhibits, notably the First Amendment to the Loan and Security Agreement with Hercules Capital, Inc., and required officer certifications[383](index=383&type=chunk)

Viridian Therapeutics, Inc. (NASDAQ:VRDN) Q1 2023 Results Conference Call May 9, 2023 4:30 PM ET Company Participants Louisa Stone - Manager, IR Scott Myers - President and CEO Kristian Humer - Chief Financial and Business Officer Dr. Thomas Ciulla - Chief Development Officer Todd James - SVP, Corporate Affairs and IR Conference Call Participants Derek Archila - Wells Fargo Gavin Clark-Gartner - Evercore ISI Thomas Smith - SVB Securities Laura Chico - Wedbush Securities Kalpit Patel - B. Riley Securities Ja ...

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, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File No. 001-36483 VIRIDIAN THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 47-1187261 (State or other jurisd ...

Financial Position - As of December 31, 2022, the company had $424.6 million in cash, cash equivalents, and short-term investments, which are expected to fund operations into the second half of 2025 [166]. - The company reported a net loss of $129.9 million for the year ended December 31, 2022, compared to a net loss of $79.4 million for 2021, resulting in an accumulated deficit of $488.2 million [170]. - The company has never generated any revenue from product sales and anticipates continued significant losses for the foreseeable future [176]. - The company expects research and development expenses to increase as product candidates advance through clinical trials, necessitating additional capital [167]. - Future funding requirements will depend on the pace, results, and costs of clinical development efforts, as well as macroeconomic conditions [171]. - The company may need to raise additional capital through equity or debt financing, which could dilute existing stockholders' ownership [180]. - If unable to raise necessary funds, the company may have to delay or discontinue product development or commercialization efforts [181]. Clinical Development Risks - Clinical trials are costly and inherently risky, with no guarantee of successful completion or regulatory approval [185]. - The company anticipates incurring significant costs associated with the commercialization of any approved product candidates, which may affect future profitability [179]. - The company currently generates no revenue from sales of any products and may never be able to develop or commercialize a product candidate [192]. - The product development program may not uncover all possible adverse events, and rare side effects may only be detected with a significantly larger patient population [190]. - The company may face difficulties in enrolling and maintaining patients in clinical trials, which could delay or prevent clinical trials of product candidates [197]. - The company is heavily dependent on the success of its product candidates, and any inability to complete clinical development could result in additional costs or impair revenue generation [187]. - The company may need to conduct additional nonclinical studies if manufacturing or formulation changes are made to product candidates [187]. Regulatory and Compliance Challenges - Regulatory authorities may withdraw approvals or require additional warnings on drug labels, impacting market acceptance [191]. - The Biologics Price Competition and Innovation Act may subject the company's product candidates to competition sooner than anticipated, affecting commercial prospects [206]. - The company is seeking Orphan drug designation for VRDN-001 from the FDA, but there is no guarantee of receiving such designation [209]. - The company may seek Breakthrough Therapy designation for its product candidates, but this designation does not ensure a faster development or approval process [210]. - The company may apply for Fast Track designation for its product candidates, which does not guarantee marketing approval in a specific timeframe [211]. - The company may pursue accelerated approval for its product candidates, but there is no assurance that the FDA will accept the application or grant approval [212]. - If accelerated approval is obtained, the company will be subject to rigorous post-approval requirements, including confirmatory studies [213]. - Legislative reforms, such as the Inflation Reduction Act of 2022, may adversely affect the company's ability to obtain marketing approval and impact pricing [216]. - The company may face penalties for non-compliance with healthcare fraud and abuse laws, which could adversely affect operations and financial results [217]. - Failure to comply with environmental, health, and safety laws could result in significant fines or penalties, impacting the company's financial condition [219]. - Non-compliance with data privacy laws could lead to government enforcement actions and negatively affect the company's operating results [220]. - The company may face reputational harm and financial penalties if it fails to comply with its published privacy policies and documentation [222]. Manufacturing and Supply Chain Risks - The company does not have the capability to manufacture product candidates internally and relies on third-party manufacturers, which could lead to delays or disruptions in clinical trials if these manufacturers fail to meet quality or quantity requirements [231]. - The company faces potential shortages and increased costs for essential items due to disruptions from the COVID-19 pandemic, impacting research and development activities [226]. - Manufacturing processes are complex and subject to numerous risks, including contamination and production failures, which could adversely affect patient outcomes and regulatory approvals [228]. - The company may encounter difficulties in scaling up manufacturing processes, which could delay clinical trials and increase costs [235]. - There is uncertainty regarding the commercial viability of product candidates due to the inability to reliably estimate manufacturing costs [232]. - Collaborations with third-party manufacturers may not result in successful development or commercialization of product candidates, posing additional risks [236]. Intellectual Property Concerns - The company relies on patent rights and trade secret protections to safeguard intellectual property, but there is no guarantee of exclusivity or successful patent issuance [241]. - The patent position of the company is uncertain, and challenges to patent validity could impair competitive advantages [243]. - Indemnification provisions in contracts could materially affect the company's financial condition if obligations exceed insurance coverage [239]. - The company has filed patent applications covering various aspects of its product candidates, but cannot assure which patents will issue or their enforceability [246]. - The company may not have sufficient patent term protections, as patents generally expire 20 years after filing, potentially exposing it to competition from generic medications [248]. - Patent term extensions may be available under the Hatch-Waxman Act in the U.S. and supplementary protection certificates in Europe, but the company cannot guarantee these will be obtained [249]. - Changes in U.S. patent law, including the Leahy-Smith America Invents Act, have increased uncertainties and costs surrounding patent prosecution and enforcement [250]. - The company relies on trade secret protection and confidentiality agreements to safeguard proprietary know-how, but these can be difficult to enforce [256]. - Third-party claims of intellectual property infringement could delay the company's development and commercialization efforts [258]. - The company may face challenges in acquiring or in-licensing necessary third-party intellectual property rights due to competition from more established companies [265]. - If the company fails to comply with obligations in its license agreements, it risks losing important license rights [269]. - The company may incur significant expenses and time in lawsuits to protect or enforce its patents, with unpredictable outcomes [270]. - The company’s ability to develop and commercialize product candidates may be adversely affected if licensors do not follow appropriate patent prosecution procedures [268]. - The company may face significant costs and distractions due to potential litigation or interference proceedings related to its patents, which could adversely affect its ability to raise funds for clinical trials and research programs [271]. Market and Competitive Landscape - The company lacks experience in establishing commercial manufacturing and sales capabilities, which may hinder its ability to generate revenue from product candidates [277]. - The company is competing against major pharmaceutical and biotechnology companies with more extensive resources, which may impact its market position and ability to commercialize products [281]. - The company may struggle to secure adequate reimbursement and insurance coverage for its products, limiting its ability to market them effectively [293]. - The company anticipates pricing pressures due to managed healthcare trends and governmental price controls, which could affect profitability [294]. - The company may not be able to protect its intellectual property rights globally, leading to potential competition from unlicensed products in jurisdictions without patent protection [274]. - The company faces risks in forming strategic collaborations for product development, which may alter its commercialization plans if unsuccessful [279]. - The company may not achieve market acceptance for its products, which is crucial for generating sufficient revenue [287]. - The company is at risk of losing valuable intellectual property rights if it fails to defend against claims of wrongful use or disclosure of confidential information [273]. Operational and Management Challenges - The company may not be able to attract and retain qualified personnel, which is critical for its research and development efforts [295]. - The company anticipates needing additional managerial, operational, and financial resources to manage its expected growth, which may lead to operational mistakes and reduced productivity [296]. - Macroeconomic conditions, including inflation and geopolitical events, may adversely affect the company's business and financial condition [297]. - The company has experienced extreme volatility in credit and financial markets, which could impact its ability to secure necessary financing and affect its growth strategy [298]. - Disruptions in clinical trials and manufacturing due to macroeconomic conditions could materially affect the company's financial results [299]. - The Hercules Loan and Security Agreement imposes covenants that may limit the company's operational flexibility and could require early repayment of debt under certain conditions [300]. - The company faces risks related to information technology failures and cyber-attacks, which could disrupt operations and adversely impact financial results [301]. - Supply chain vulnerabilities may expose the company to risks from natural disasters or unforeseen events, potentially affecting business operations [302]. - Security incidents could lead to unauthorized access to sensitive information, resulting in legal liabilities and increased costs [303]. Tax and Financial Reporting - The company may face limitations on its net operating loss carryforwards due to changes in ownership or tax law, which could adversely affect cash flow [307]. - Changes in tax laws could materially impact the company's financial performance and effective tax rate, leading to increased tax obligations [310]. - The company does not anticipate paying any cash dividends in the foreseeable future, expecting to retain future earnings to fund business development and growth [316]. - Future sales of shares by existing stockholders could lead to a decline in the stock price, particularly after legal restrictions on resale lapse [317]. - The company expects to need significant additional capital for current and future operations, which may involve selling common stock or other equity securities, potentially causing dilution for existing stockholders [318]. - The principal stockholders, including directors and officers, own a substantial portion of the voting stock, allowing them to exert significant control over matters requiring stockholder approval [320]. - The market price of the company's common stock has historically been volatile, influenced by various factors including market conditions and the performance of similar companies [322]. - The company incurs significant legal, accounting, and compliance costs associated with public company reporting requirements, which may increase over time [324]. - If equity research analysts do not publish favorable reports or cease coverage, it could negatively impact the stock price and trading volume [325]. - The company is subject to the reporting requirements of the Sarbanes-Oxley Act, necessitating effective internal controls over financial reporting, which may incur substantial costs [326]. - Weaknesses in internal financial controls could result in material misstatements of financial statements, potentially leading to restatements and loss of investor confidence [328]. - As a smaller reporting company, the company is not required to provide certain market risk disclosures, which may limit transparency [383].

Viridian Therapeutics, Inc. (NASDAQ:VRDN) Q4 2022 Earnings Conference Call March 8, 2023 8:00 AM ET Company Participants Louisa Stone - Manager, Investor Relations Scott Myers - President and Chief Executive Officer Kristian Humer - Chief Financial Officer Deepa Rajagopalan - Chief Product and Strategy Officer Todd James - Senior Vice President, Corporate Affairs and Investor Relations Conference Call Participants Gavin Clark-Gartner - Evercore ISI Laura Chico - Wedbush Securities Jason Butler - JMP Securit ...

%VIRIDIAN J.P. Morgan 41st Annual Healthcare Conference January 9, 2023 Cautionary note regarding forward-looking statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as, but not limited to, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "will," or "woul ...

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]