Financial Data and Key Metrics Changes - The company ended Q1 2020 with $36.1 million in cash and cash equivalents, an increase from $26.8 million at the end of 2019 [29] - Revenue for the quarter was $0.8 million, up from $0.4 million in Q1 2019, primarily due to increases in grant revenue and collaboration agreements [30] - Net loss for Q1 2020 was $8 million, or $0.18 per share, compared to a net loss of $11.6 million, or $0.38 per share, in Q1 2019 [31] Business Line Data and Key Metrics Changes - Research and development expenses decreased to $6.1 million in Q1 2020 from $8.8 million in Q1 2019, attributed to reduced personnel costs and clinical activities [30] - General and administrative expenses were $2.7 million for the quarter, down from $3.4 million in the same period last year, mainly due to decreased personnel costs [30] Market Data and Key Metrics Changes - The company is facing challenges in clinical activities due to the COVID-19 pandemic, impacting patient visits and data collection for trials [11][14] - The SOLAR Phase II clinical trial for cobomarsen has completed enrollment of 37 patients, but the pandemic has affected data collection and patient dosing [12][14] Company Strategy and Development Direction - The company has streamlined its development strategy to focus on long-term shareholder value, adapting to the challenges posed by the COVID-19 pandemic [10] - There is an ongoing evaluation of cobomarsen for treating miR-155 elevated blood cancers, with plans to request a meeting with the FDA regarding expedited development for ATLL [20] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the uncertainty caused by COVID-19 on clinical trials and the inability to predict milestone achievements [11][26] - The company remains confident in its ability to deliver important results from its programs despite the pandemic's impact [26] Other Important Information - The company is developing two distinct microRNA-29 mimics for treating various forms of pathological fibrosis, with a focus on MRG-229 for idiopathic pulmonary fibrosis [21][22] - The company plans to report additional preclinical safety and efficacy data for MRG-229 in Q2 2020 [23] Q&A Session Summary Question: How should we think about operating expenses in 2Q and the rest of the year versus Q1 given the COVID delays? - The CFO indicated that operating expenses are expected to decrease on a quarterly basis due to lower workforce expenses and reduced costs associated with the SOLAR trial [34] Question: What types of results could we see in the MRG-229 preclinical data, and could there be potential use for COVID-19? - Management discussed ongoing exploratory toxicology and efficacy studies, expressing interest in the antifibrotic activity of MRG-229 and cobomarsen in the context of COVID-19 [36][38] Question: Can you clarify the delays in the SOLAR trial and the FDA meeting timeline? - Management confirmed that while patient dosing continues, data collection has been impacted, and they cannot guarantee the timing of the FDA meeting due to COVID-19 prioritization [45][49]

Revenue and Financial Performance - Revenue for the three months ended March 31, 2020, was $0.8 million, an increase from $0.4 million in the same period of 2019, primarily due to a $0.1 million increase in grant revenue and a $0.3 million increase in reimbursable research and development activities under the Servier Collaboration Agreement[188][189] - The net loss for the three months ended March 31, 2020, was $8.0 million, compared to a net loss of $11.6 million for the same period in 2019[188] - Net cash used in operating activities was $8.5 million for the three months ended March 31, 2020, a decrease of $3.3 million compared to $11.8 million in the same period of 2019[204] - Net cash provided by investing activities was $2.0 million for the three months ended March 31, 2020, compared to a net cash outflow of $2.8 million in the same period of 2019[205] - Net cash provided by financing activities was $17.7 million for the three months ended March 31, 2020, significantly higher than $0.2 million in the same period of 2019[206] - As of March 31, 2020, the company had $36.1 million in cash and cash equivalents, which is expected to fund operations into the third quarter of 2021[192] - The company generated an accumulated deficit of $176.2 million through March 31, 2020, with substantial operating losses primarily from research and development expenses[198] Expenses - Research and development expenses for the three months ended March 31, 2020, were $6.1 million, a decrease from $8.8 million in the same period of 2019[188] - General and administrative expenses for the three months ended March 31, 2020, were $2.7 million, down from $3.4 million in the same period of 2019[188] - Research and development expenses decreased to $6.1 million for the three months ended March 31, 2020, down from $8.8 million in the same period of 2019, a reduction of approximately 30.7%[190] - General and administrative expenses were $2.7 million for the three months ended March 31, 2020, compared to $3.4 million in the same period of 2019, reflecting a decrease of about 20.6%[191] Clinical Development and Research - Cobomarsen is being developed for the treatment of patients with certain cancers, including cutaneous T-cell lymphoma and adult T-cell leukemia/lymphoma, with ongoing clinical trials impacted by the COVID-19 pandemic[171] - MRG-110 has shown potential in increasing angiogenesis and is being evaluated for heart failure and wound healing applications, with data from Phase 1 trials indicating safety and tolerability[175] - Remlarsen is being evaluated for ocular fibrotic indications, with preclinical studies showing faster healing of corneal injuries[174] - MRG-229 is focused on idiopathic pulmonary fibrosis, with ongoing preclinical studies supported by a grant from the NIH and Yale University[178] - The company anticipates an increase in research and development expenses as it continues clinical trials and preclinical research programs[182] - The company expects to incur significant expenses and increased operating losses for at least the next several years as it continues clinical development and seeks regulatory approval for its product candidates[200] Impact of COVID-19 - The COVID-19 pandemic may adversely affect the company's liquidity and results of operations, with potential delays in clinical trials and financing[197] Cost Restructuring - The company began implementing a cost restructuring plan in August 2019, identifying approximately 44 positions for elimination, or about 50% of its workforce[202] Future Revenue Expectations - Future revenue generation is expected to fluctuate based on the timing of achieving clinical and regulatory milestones, as well as the success of product commercialization[179]

Part I [Business](index=5&type=section&id=ITEM%201.%20BUSINESS) Miragen Therapeutics is a clinical-stage biopharmaceutical company developing RNA-targeted therapies for high unmet medical needs, with a pipeline of three clinical-stage candidates and a strategy focused on microRNA expertise, expedited regulatory pathways, and strategic collaborations [Company Overview and Product Pipeline](index=5&type=section&id=Company%20Overview%20and%20Product%20Pipeline) Miragen is a clinical-stage biopharmaceutical company developing proprietary RNA-targeted microRNA therapies, with three clinical-stage product candidates for cancers, pathological fibrosis, and heart failure - The company's core focus is on RNA-targeted therapies, specifically microRNAs, for diseases with high unmet medical needs[20](index=20&type=chunk) Clinical Stage Product Candidates | Product Candidate | Target MicroRNA | Indication(s) | | :--- | :--- | :--- | | **Cobomarsen** | miR-155 (Inhibitor) | Cutaneous T-cell lymphoma (CTCL), Adult T-cell leukemia/lymphoma (ATLL) | | **Remlarsen / MRG-229** | miR-29 (Replacement) | Pathological fibrosis (e.g., Idiopathic Pulmonary Fibrosis - IPF) | | **MRG-110** | miR-92 (Inhibitor) | Heart failure, wound healing, ischemic disease | [Our Strategy](index=6&type=section&id=Our%20Strategy) The company's strategy leverages microRNA expertise to advance lead candidates, utilize FDA rare disease pathways, and seek collaborations for pipeline expansion and new target identification - A core part of the strategy is to utilize FDA's rare disease development pathways (orphan drug, fast track, breakthrough therapy) to potentially streamline clinical development and reduce time to market for its product candidates[23](index=23&type=chunk) - The company plans to continue developing its lead candidates: cobomarsen for blood cancers like CTCL and ATLL, and remlarsen/MRG-229 for pathological fibrosis, with a specific focus on MRG-229 for Idiopathic Pulmonary Fibrosis (IPF)[23](index=23&type=chunk) - Miragen intends to seek collaborations with other biotechnology and pharmaceutical companies to develop additional product candidates, particularly for disease areas where development costs would exceed its resources[23](index=23&type=chunk) [Our Product Candidates](index=6&type=section&id=Our%20Product%20Candidates) The company's clinical pipeline includes cobomarsen for CTCL/ATLL, remlarsen/MRG-229 for pathological fibrosis, and MRG-110 for heart failure, with key milestones anticipated for 2020 Anticipated Milestones for Product Candidates | Milestone | Product Candidate | Indication | Expected Timing | | :--- | :--- | :--- | :--- | | Report preclinical safety & efficacy data | MRG-229 | IPF | Q2-2020 | | Meet with FDA for expedited path | Cobomarsen | ATLL | Q3-2020 | | Report topline data from Phase 2 trial | Cobomarsen | CTCL | Q3-2020 | | Report primary endpoint data from Phase 2 trial | Remlarsen | Cutaneous Fibrosis | 2H-2020 | - Enrollment in the Phase 2 SOLAR trial for cobomarsen in CTCL was stopped to allow for an interim analysis of 37 patients, with topline data expected in Q3 2020[36](index=36&type=chunk)[37](index=37&type=chunk) - Following positive interim Phase 1 data in ATLL, the company is focusing its expansion efforts for cobomarsen on this indication and plans to meet with the FDA to discuss an expedited development pathway[26](index=26&type=chunk)[27](index=27&type=chunk) - The collaboration with Servier for MRG-110 was terminated in February 2020, resulting in Miragen regaining full global rights. The company is exploring potential new collaborations for its future development[52](index=52&type=chunk) [Strategic Collaborations and License Agreements](index=13&type=section&id=Strategic%20Collaborations%20and%20License%20Agreements) The company relies on key collaborations and license agreements, including a terminated Servier agreement, and ongoing agreements with the University of Texas, RICC, and Yale, involving milestone and royalty payments - The collaboration agreement with Servier for MRG-110 was terminated in February 2020. This agreement was a significant source of revenue, generating **$4.3 million** in 2019 and **$7.4 million** in 2018[72](index=72&type=chunk) - The company has an exclusive patent license with the University of Texas, requiring potential future milestone payments up to ~**$0.6 million** for clinical trial initiations and **$2.5 million** upon regulatory approvals in the U.S. and other regions, plus low-single-digit royalties[76](index=76&type=chunk) - Under a license agreement with Roche Innovation Center Copenhagen (RICC), Miragen is obligated to make milestone payments up to **$5.2 million** per licensed product and pay mid-single-digit royalties on net sales[80](index=80&type=chunk)[82](index=82&type=chunk) - A subcontract with Yale University, funded by an NIH grant, supports the development of a miR-29 mimic for IPF. Miragen retains rights to its solely developed IP and has an exclusive option to license IP developed by Yale under the agreement[86](index=86&type=chunk)[87](index=87&type=chunk) [Manufacturing, Sales, and Marketing](index=16&type=section&id=Manufacturing%2C%20Sales%2C%20and%20Marketing) Miragen fully outsources manufacturing and has not yet established a commercial strategy, considering future partnerships or an in-house sales force - The company fully outsources its manufacturing, relying on third-party contract manufacturers for all required materials and finished product candidates for clinical trials[97](index=97&type=chunk) - No commercial strategy for sales, marketing, or distribution has been defined, as all product candidates are still in development. Future options include strategic partnerships or establishing an in-house commercial force[98](index=98&type=chunk) [Intellectual Property](index=16&type=section&id=Intellectual%20Property) As of December 31, 2019, Miragen's IP portfolio includes 298 patents and applications, with key U.S. patents for lead candidates expiring between 2033 and 2036 - As of year-end 2019, the company's patent portfolio consists of 298 patents and applications, of which 216 are issued or allowed[99](index=99&type=chunk) Key U.S. Patent Expiration Outlook | Product Candidate | U.S. Patent(s) | Projected Expiration Year | | :--- | :--- | :--- | | **Cobomarsen** | 9,771,585; 9,994,852 | 2036 | | **MRG-110** | 9,803,202 | 2033 | | **Remlarsen** | 9,376,681; 9,994,847 | 2035 | [Competition](index=17&type=section&id=Competition) Miragen faces intense competition from other microRNA and oligonucleotide developers, as well as major pharmaceutical companies treating the same diseases - Competition is categorized into three groups: other microRNA-targeted developers, other oligonucleotide therapeutic developers, and companies treating the same diseases with different modalities[108](index=108&type=chunk)[112](index=112&type=chunk) - Major pharmaceutical companies with marketed or developing therapies for CTCL include Bristol-Myers Squibb, Merck, Takeda, and Novartis[108](index=108&type=chunk) - Key competitors with marketed therapeutics for pulmonary fibrosis include Boehringer Ingelheim and F. Hoffmann-La Roche[109](index=109&type=chunk) [Government Regulation](index=18&type=section&id=Government%20Regulation) The company's operations are extensively regulated by the FDA and other authorities, covering drug development, approval, and post-market compliance, including various expedited programs and healthcare laws - The FDA drug approval process requires extensive preclinical studies, an effective IND application, and adequate and well-controlled human clinical trials (typically Phases 1, 2, and 3) to establish safety and efficacy before an NDA can be submitted and approved[112](index=112&type=chunk)[113](index=113&type=chunk)[120](index=120&type=chunk) - The company may seek Orphan Drug Designation for products treating rare diseases (affecting <**200,000** people in the U.S.), which can provide benefits like seven years of market exclusivity upon approval[127](index=127&type=chunk)[129](index=129&type=chunk) - Expedited FDA programs such as Fast Track, priority review, accelerated approval, and Breakthrough Therapy Designation are available for drugs that treat serious conditions and address unmet medical needs, potentially speeding up the development and review process[131](index=131&type=chunk)[132](index=132&type=chunk)[133](index=133&type=chunk) - The business is subject to numerous healthcare regulations, including the federal Anti-Kickback Statute, False Claims Act, HIPAA for patient data privacy, and the Physician Payments Sunshine Act for reporting payments to healthcare professionals[143](index=143&type=chunk)[144](index=144&type=chunk)[145](index=145&type=chunk)[150](index=150&type=chunk) [Risk Factors](index=27&type=section&id=ITEM%201A.%20RISK%20FACTORS) The company faces significant risks related to its financial viability, product development uncertainties, regulatory hurdles, intellectual property, reliance on third parties, and potential impacts from health epidemics like COVID-19 - The company needs to raise additional capital to continue as a going concern. Current resources are projected to fund operations only into the third quarter of 2021[169](index=169&type=chunk) - The company has a history of significant net losses (**$41.9 million** in 2019) and an accumulated deficit of **$168.2 million** as of December 31, 2019, with no products approved for sale[175](index=175&type=chunk) - The therapeutic approach using microRNAs is novel and unproven, with no such products yet approved by the FDA, making the development timeline, cost, and likelihood of approval highly uncertain[197](index=197&type=chunk)[199](index=199&type=chunk) - The business could be adversely affected by health epidemics like the COVID-19 outbreak, which could disrupt clinical trial sites, patient enrollment, and third-party manufacturing operations[314](index=314&type=chunk)[317](index=317&type=chunk)[318](index=318&type=chunk) [Unresolved Staff Comments](index=66&type=section&id=ITEM%201B.%20UNRESOLVED%20STAFF%20COMMENTS) The company reports that it has no unresolved staff comments from the SEC - None[401](index=401&type=chunk) [Properties](index=66&type=section&id=ITEM%202.%20PROPERTIES) The company leases 27,128 square feet of office and laboratory space in Boulder, Colorado, under a lease expiring in December 2020 - The company leases **27,128** sq. ft. of office and lab space in Boulder, CO, with the lease expiring in December 2020[402](index=402&type=chunk) [Legal Proceedings](index=66&type=section&id=ITEM%203.%20LEGAL%20PROCEEDINGS) The company is not currently a party to any legal proceedings that would materially adversely affect its business or financial condition - The company is not currently a party to any material legal proceedings[403](index=403&type=chunk) [Mine Safety Disclosures](index=66&type=section&id=ITEM%204.%20MINE%20SAFETY%20DISCLOSURES) This item is not applicable to the company - Not applicable[404](index=404&type=chunk) Part II [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=67&type=section&id=ITEM%205.%20MARKET%20FOR%20REGISTRANT%27S%20COMMON%20EQUITY%2C%20RELATED%20STOCKHOLDER%20MATTERS%20AND%20ISSUER%20PURCHASES%20OF%20EQUITY%20SECURITIES) The company's common stock trades on The Nasdaq Capital Market under 'MGEN', with no dividends paid or anticipated, as earnings are retained for business growth - Common stock is traded on The Nasdaq Capital Market under the symbol MGEN[407](index=407&type=chunk) - The company has not paid and does not anticipate paying dividends, intending to retain all future earnings for business development[409](index=409&type=chunk) [Selected Financial Data](index=67&type=section&id=ITEM%206.%20SELECTED%20FINANCIAL%20DATA) This item is not applicable to the company - Not applicable[410](index=410&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=67&type=section&id=ITEM%207.%20MANAGEMENT%27S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) The company reported an increased net loss of **$41.9 million** in 2019, with decreased revenue and higher R&D expenses, and projects current capital to fund operations into Q3 2021 [Results of Operations](index=71&type=section&id=Results%20of%20Operations) For 2019, revenue decreased to **$4.5 million** due to a non-recurring milestone, while R&D expenses increased to **$34.8 million**, resulting in a net loss of **$41.9 million** Comparison of Operations (2019 vs. 2018) | Financial Metric | 2019 (in thousands) | 2018 (in thousands) | Change | | :--- | :--- | :--- | :--- | | **Revenue** | $4,461 | $8,386 | ($3,925) | | **Research and development expenses** | $34,794 | $30,421 | $4,373 | | **General and administrative expenses** | $11,646 | $11,049 | $597 | | **Net loss** | ($41,873) | ($32,703) | ($9,170) | - The decrease in revenue was primarily due to a €3.0 million (**$3.7 million**) development milestone payment from the Servier Collaboration Agreement in 2018 that did not recur in 2019[433](index=433&type=chunk) - The increase in R&D expenses was driven by a **$3.4 million** rise in clinical development and manufacturing costs for cobomarsen and a **$2.5 million** increase in personnel-related costs, including restructuring charges[434](index=434&type=chunk) [Liquidity and Capital Resources](index=71&type=section&id=Liquidity%20and%20Capital%20Resources) As of December 31, 2019, the company held **$26.8 million** in cash, projecting funds to last into Q3 2021, and has utilized various equity financing vehicles - As of December 31, 2019, the company held **$26.8 million** in cash, cash equivalents, and short-term investments[435](index=435&type=chunk) - The company projects its current cash resources, supplemented by recent stock sales, will be sufficient to fund operations into the third quarter of 2021[435](index=435&type=chunk) - In February 2020, the company raised approximately **$14.0 million** in net proceeds from a public offering of common stock and warrants[439](index=439&type=chunk) - In December 2019, the company entered into a common stock purchase agreement with Aspire Capital for up to **$20.0 million**, of which **$5.1 million** had been raised as of January 2020[438](index=438&type=chunk) [Cash Flows](index=73&type=section&id=Cash%20Flows) In 2019, net cash used in operating activities increased to **$36.1 million**, while investing activities provided **$28.2 million**, and financing activities provided only **$0.1 million** Summarized Cash Flows (2019 vs. 2018) | Activity | 2019 (in thousands) | 2018 (in thousands) | | :--- | :--- | :--- | | **Net cash used in operating activities** | ($36,056) | ($26,844) | | **Net cash provided by (used in) investing activities** | $28,226 | ($29,907) | | **Net cash provided by financing activities** | $70 | $41,916 | [Quantitative and Qualitative Disclosures About Market Risk](index=74&type=section&id=ITEM%207A.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURES%20ABOUT%20MARKET%20RISK) This item is not applicable to the company - Not applicable[452](index=452&type=chunk) [Financial Statements and Supplementary Data](index=74&type=section&id=ITEM%208.%20FINANCIAL%20STATEMENTS%20AND%20SUPPLEMENTARY%20DATA) The required financial statements and supplementary data are provided elsewhere in the report, starting on page F-1 - The financial statements are located in Part IV, Item 15(a)(1) of the Annual Report[453](index=453&type=chunk) [Changes in and Disagreements with Accountants on Accounting and Financial Disclosure](index=74&type=section&id=ITEM%209.%20CHANGES%20IN%20AND%20DISAGREEMENTS%20WITH%20ACCOUNTANTS%20ON%20ACCOUNTING%20AND%20FINANCIAL%20DISCLOSURE) This item is not applicable to the company - Not applicable[454](index=454&type=chunk) [Controls and Procedures](index=74&type=section&id=ITEM%209A.%20CONTROLS%20AND%20PROCEDURES) Management concluded that disclosure controls and internal control over financial reporting were effective as of December 31, 2019, with an unqualified opinion from KPMG LLP - Management concluded that the company's disclosure controls and procedures were effective as of December 31, 2019[455](index=455&type=chunk) - Management concluded that the company's internal control over financial reporting was effective as of December 31, 2019[458](index=458&type=chunk) - The independent registered public accounting firm, KPMG LLP, issued an unqualified opinion on the effectiveness of the company's internal control over financial reporting as of December 31, 2019[459](index=459&type=chunk) [Other Information](index=75&type=section&id=ITEM%209B.%20OTHER%20INFORMATION) This item is not applicable to the company - Not applicable[461](index=461&type=chunk) Part III [Directors, Executive Officers, and Corporate Governance](index=76&type=section&id=ITEM%2010.%20DIRECTORS%2C%20EXECUTIVE%20OFFICERS%2C%20AND%20CORPORATE%20GOVERNANCE) Information for this item is incorporated by reference from the definitive proxy statement for the 2020 annual meeting of stockholders - Information is incorporated by reference from the definitive proxy statement for the 2020 annual meeting of stockholders[464](index=464&type=chunk) [Executive Compensation](index=76&type=section&id=ITEM%2011.%20EXECUTIVE%20COMPENSATION) Information for this item is incorporated by reference from the definitive proxy statement - Information is incorporated by reference from the definitive proxy statement[466](index=466&type=chunk) [Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=76&type=section&id=ITEM%2012.%20SECURITY%20OWNERSHIP%20OF%20CERTAIN%20BENEFICIAL%20OWNERS%20AND%20MANAGEMENT%20AND%20RELATED%20STOCKHOLDER%20MATTERS) Information for this item is incorporated by reference from the definitive proxy statement - Information is incorporated by reference from the definitive proxy statement[467](index=467&type=chunk) [Certain Relationships and Related Transactions and Director Independence](index=76&type=section&id=ITEM%2013.%20CERTAIN%20RELATIONSHIPS%20AND%20RELATED%20TRANSACTIONS%20AND%20DIRECTOR%20INDEPENDENCE) Information for this item is incorporated by reference from the definitive proxy statement - Information is incorporated by reference from the definitive proxy statement[468](index=468&type=chunk) [Principal Accounting Fees and Services](index=76&type=section&id=ITEM%2014.%20PRINCIPAL%20ACCOUNTING%20FEES%20AND%20SERVICES) Information for this item is incorporated by reference from the definitive proxy statement - Information is incorporated by reference from the definitive proxy statement[469](index=469&type=chunk) Part IV [Exhibits, Financial Statement Schedules](index=77&type=section&id=ITEM%2015.%20EXHIBITS%2C%20FINANCIAL%20STATEMENT%20SCHEDULES) The financial statements are submitted separately, financial statement schedules are omitted, and a list of exhibits is referenced - The financial statements are submitted in a separate section beginning on page F-1[472](index=472&type=chunk) - Financial statement schedules have been omitted[473](index=473&type=chunk) [Form 10-K Summary](index=77&type=section&id=ITEM%2016.%20FORM%2010-K%20SUMMARY) The company reports that there is no Form 10-K summary - None[475](index=475&type=chunk) Financial Statements [Report of Independent Registered Public Accounting Firm](index=82&type=section&id=Report%20of%20Independent%20Registered%20Public%20Accounting%20Firm) KPMG LLP issued unqualified opinions on the consolidated financial statements and the effectiveness of internal control over financial reporting for 2019, noting the adoption of ASC Topic 606 - KPMG LLP issued an unqualified opinion on the consolidated financial statements[486](index=486&type=chunk) - KPMG LLP issued an unqualified opinion on the effectiveness of the company's internal control over financial reporting as of December 31, 2019[487](index=487&type=chunk)[494](index=494&type=chunk) [Consolidated Financial Statements](index=84&type=section&id=Consolidated%20Financial%20Statements) The consolidated financial statements present the company's financial position and results for 2019 and 2018, showing a net loss of **$41.9 million** in 2019 and decreased cash and equivalents Consolidated Balance Sheet Data (as of Dec 31) | Account | 2019 (in thousands) | 2018 (in thousands) | | :--- | :--- | :--- | | **Total Assets** | $30,262 | $66,147 | | **Total Liabilities** | $14,508 | $14,803 | | **Total Stockholders' Equity** | $15,754 | $51,344 | Consolidated Statement of Operations Data (Year Ended Dec 31) | Account | 2019 (in thousands) | 2018 (in thousands) | | :--- | :--- | :--- | | **Total Revenue** | $4,461 | $8,386 | | **Total Operating Expenses** | $46,440 | $41,470 | | **Net Loss** | ($41,873) | ($32,703) | | **Net Loss Per Share** | ($1.34) | ($1.10) | [Notes to Consolidated Financial Statements](index=89&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) The notes detail the company's liquidity, accounting policy changes, a 2019 restructuring, the termination of the Servier collaboration, loan agreements, and capital stock transactions

miRagen Therapeutics, Inc. (MGEN) Q4 2019 Earnings Conference Call March 11, 2020 4:30 PM ET Company Participants Dan Ferry - Managing Director of LifeSci Advisors Bill Marshall - President and CEO Diana Escolar - Chief Medical Officer Jason Leverone - CFO Conference Call Participants Kambiz Yazdi - Wedbush Securities Jonathan Miller - Evercore ISI Operator Greetings. Welcome to miRagen Therapeutics Fourth Quarter 2019 Earnings Call. [Operator Instructions] Please note this conference is being recorded. I w ...

miRagen Therapeutics, Inc. (MGEN) Q3 2019 Earnings Conference Call November 7, 2019 4:30 PM ET Company Participants Dan Ferry – Managing Director of LifeSci Advisors Bill Marshall – President and Chief Executive Officer Jason Leverone – Chief Financial Officer Conference Call Participants Jonathan Miller – Evercore ISI Leland Gershell – Oppenheimer Madhu Kumar – R.W. Baird Liana Moussatos – Wedbush Securities Operator Thank you for standing by. This is the conference operator. Welcome to the miRagen Therape ...

[PART I. FINANCIAL INFORMATION](index=3&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) This section provides the unaudited condensed consolidated financial statements and management's discussion and analysis for Miragen Therapeutics, Inc [ITEM 1. FINANCIAL STATEMENTS](index=3&type=section&id=ITEM%201.%20FINANCIAL%20STATEMENTS) This section presents the unaudited condensed consolidated financial statements, including balance sheets, statements of operations, equity changes, and cash flows, with explanatory notes [Condensed Consolidated Balance Sheets](index=3&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) This section provides a snapshot of the company's assets, liabilities, and stockholders' equity at specific reporting dates Condensed Consolidated Balance Sheets (in thousands) | Item | Sep 30, 2019 | Dec 31, 2018 | | :-------------------------------- | :----------- | :----------- | | **Assets** | | | | Cash and cash equivalents | $24,857 | $32,606 | | Short-term investments | $8,981 | $29,875 | | Total current assets | $38,217 | $65,370 | | Total assets | $38,813 | $66,147 | | **Liabilities & Stockholders' Equity** | | | | Total current liabilities | $10,385 | $6,733 | | Total liabilities | $15,644 | $14,803 | | Total stockholders' equity | $23,169 | $51,344 | | Total liabilities and stockholders' equity | $38,813 | $66,147 | - Total current assets decreased by approximately **$27.15 million (41.5%)** from December 31, 2018, to September 30, 2019, primarily due to reductions in cash, cash equivalents, and short-term investments[9](index=9&type=chunk) - Total stockholders' equity decreased by approximately **$28.18 million (54.9%)** from December 31, 2018, to September 30, 2019, largely driven by accumulated deficit[9](index=9&type=chunk) [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=4&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) This section details the company's revenue, expenses, and net loss over specified interim periods Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands) | Item | 3 Months Ended Sep 30, 2019 | 3 Months Ended Sep 30, 2018 | 9 Months Ended Sep 30, 2019 | 9 Months Ended Sep 30, 2018 | | :-------------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Total revenue | $695 | $944 | $3,581 | $7,910 | | Research and development | $9,027 | $7,399 | $26,377 | $22,187 | | General and administrative | $2,898 | $2,696 | $9,112 | $8,354 | | Total operating expenses | $11,925 | $10,095 | $35,489 | $30,541 | | Net loss | $(11,230) | $(9,011) | $(31,755) | $(22,386) | | Net loss per share, basic and diluted | $(0.36) | $(0.29) | $(1.02) | $(0.77) | - Total revenue decreased by **$0.25 million (26.4%)** for the three months ended September 30, 2019, and by **$4.33 million (54.7%)** for the nine months ended September 30, 2019, compared to the respective prior periods[10](index=10&type=chunk) - Research and development expenses increased by **$1.63 million (22.0%)** for the three months and **$4.19 million (18.9%)** for the nine months ended September 30, 2019, driven by increased clinical trial and personnel costs[10](index=10&type=chunk) - Net loss increased by **$2.22 million (24.6%)** for the three months and **$9.37 million (41.8%)** for the nine months ended September 30, 2019, reflecting higher operating expenses and lower revenue[10](index=10&type=chunk) [Condensed Consolidated Statements of Changes in Stockholders' Equity](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Changes%20in%20Stockholders'%20Equity) This section outlines the changes in the company's equity components, including common stock and accumulated deficit, over the reporting period Changes in Stockholders' Equity (in thousands) | Item | Dec 31, 2018 | Sep 30, 2019 | | :-------------------------------- | :----------- | :----------- | | Common Stock (shares) | 30,839,463 | 31,181,749 | | Common Stock (amount) | $308 | $312 | | Additional Paid-in Capital | $177,335 | $180,905 | | Accumulated Other Comprehensive Gain (Loss) | $(3) | $3 | | Accumulated Deficit | $(126,296) | $(158,051) | | Total Stockholders' Equity | $51,344 | $23,169 | - Total stockholders' equity decreased from **$51.34 million** at December 31, 2018, to **$23.17 million** at September 30, 2019, primarily due to a significant increase in accumulated deficit from net losses[12](index=12&type=chunk) - Additional paid-in capital increased by **$3.57 million**, reflecting issuances of common stock and share-based compensation expense during the nine-month period[12](index=12&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) This section presents the cash inflows and outflows from operating, investing, and financing activities for the reporting periods Condensed Consolidated Statements of Cash Flows (in thousands) | Cash Flow Activity | 9 Months Ended Sep 30, 2019 | 9 Months Ended Sep 30, 2018 | | :-------------------------------- | :-------------------------- | :-------------------------- | | Net cash used in operating activities | $(28,250) | $(18,669) | | Net cash provided by (used in) investing activities | $21,226 | $(38,998) | | Net cash provided by (used in) financing activities | $(725) | $41,926 | | Net decrease in cash and cash equivalents | $(7,749) | $(15,741) | | Cash and cash equivalents at end of period | $24,857 | $31,700 | - Net cash used in operating activities increased by **$9.58 million**, from **$18.67 million** in 2018 to **$28.25 million** in 2019, primarily due to a higher net loss[16](index=16&type=chunk)[198](index=198&type=chunk) - Investing activities shifted from a net cash outflow of **$39.00 million** in 2018 to a net cash inflow of **$21.23 million** in 2019, driven by **$54.00 million** in maturities of short-term investments[16](index=16&type=chunk)[199](index=199&type=chunk) - Financing activities shifted from a net cash inflow of **$41.93 million** in 2018 to a net cash outflow of **$0.73 million** in 2019, mainly due to a public offering in 2018 and higher principal payments on notes payable in 2019[16](index=16&type=chunk)[200](index=200&type=chunk) [Notes to Condensed Consolidated Financial Statements](index=8&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) This section provides detailed explanations and disclosures supporting the condensed consolidated financial statements [1. DESCRIPTION OF BUSINESS](index=8&type=section&id=1.%20DESCRIPTION%20OF%20BUSINESS) This note describes Miragen Therapeutics, Inc.'s core business, product candidates, and financial outlook - Miragen Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on discovering and developing RNA-targeted therapies, specifically microRNAs, for diseases with high unmet medical needs[18](index=18&type=chunk) - The company has three product candidates in clinical development: cobomarsen, remlarsen, and MRG-110[18](index=18&type=chunk) - As of September 30, 2019, the company had **$33.8 million** in cash, cash equivalents, and short-term investments, which are expected to fund operations through Q2 2020[20](index=20&type=chunk) - The Servier Collaboration Agreement was terminated effective February 1, 2020, meaning the company does not expect future revenue from this agreement[19](index=19&type=chunk) - The company has an accumulated deficit of **$158.1 million** as of September 30, 2019, and expects significant operating losses to continue[22](index=22&type=chunk) [2. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES](index=9&type=section&id=2.%20SUMMARY%20OF%20SIGNIFICANT%20ACCOUNTING%20POLICIES) This note outlines the key accounting principles and methods used in preparing the financial statements - The company adopted ASC 606 (Revenue from Contracts with Customers) and ASU 2018-07 (Share-Based Payment Accounting) effective January 1, 2019, with no material impact on financial statements[29](index=29&type=chunk)[68](index=68&type=chunk)[69](index=69&type=chunk) - Revenue recognition follows a five-step model, recognizing revenue when control of goods or services is transferred to the customer[32](index=32&type=chunk) - Research and development costs are expensed as incurred, including employee-related expenses, CRO fees, manufacturing costs, and license fees where future economic benefit is uncertain[43](index=43&type=chunk)[44](index=44&type=chunk) - Investments are classified as available-for-sale securities and measured at fair value, with unrealized gains/losses reported in stockholders' equity[47](index=47&type=chunk)[48](index=48&type=chunk) Fair Value Measurements (in thousands) | Item | Sep 30, 2019 (Level 1) | Dec 31, 2018 (Level 1) | | :-------------------------------- | :--------------------- | :--------------------- | | Money market funds | $24,917 | $32,936 | | U.S. treasury securities | $8,981 | $29,875 | | Total assets | $33,898 | $62,811 | | Common Stock warrants (Level 3) | $82 | $82 | [3. COST RESTRUCTURING PLAN](index=14&type=section&id=3.%20COST%20RESTRUCTURING%20PLAN) This note details the company's cost reduction initiatives, including workforce reductions and associated charges - In August 2019, the company announced a cost restructuring plan to reduce costs and reallocate resources to cobomarsen and microRNA-29 mimics, eliminating approximately **26 positions**[72](index=72&type=chunk)[165](index=165&type=chunk) Restructuring Expense (in thousands) | Item | 2019 | | :---------------------- | :--- | | Retention | $787 | | Severance | $288 | | Other | $17 | | Total restructuring charges | $1,092 | - Restructuring charges of **$1.1 million** were recorded in Q3 2019, with **$0.9 million** in R&D and **$0.2 million** in G&A, with an additional **$0.4 million** expected in Q4 2019[73](index=73&type=chunk)[166](index=166&type=chunk) [4. STRATEGIC ALLIANCE AND COLLABORATION WITH SERVIER](index=15&type=section&id=4.%20STRATEGIC%20ALLIANCE%20AND%20COLLABORATION%20WITH%20SERVIER) This note describes the collaboration agreement with Servier, its termination, and the financial impact on revenue - The Servier Collaboration Agreement, initiated in October 2011 for RNA-targeting therapeutics in cardiovascular disease, was terminated by Servier effective February 1, 2020[75](index=75&type=chunk)[76](index=76&type=chunk)[168](index=168&type=chunk) - Upon termination, Miragen will regain global rights to MRG-110 in all indications and territories[76](index=76&type=chunk)[169](index=169&type=chunk) Collaboration Revenue from Servier (in thousands) | Item | 3 Months Ended Sep 30, 2019 | 3 Months Ended Sep 30, 2018 | 9 Months Ended Sep 30, 2019 | 9 Months Ended Sep 30, 2018 | | :-------------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Milestone payments | $0 | $0 | $0 | $3,690 | | Research and development reimbursable costs | $625 | $814 | $3,471 | $3,248 | | Total collaboration revenue | $625 | $814 | $3,471 | $6,938 | - Collaboration revenue decreased significantly in 2019, primarily due to a **€3.0 million ($3.7 million)** development milestone payment received in 2018 that did not recur in 2019[84](index=84&type=chunk)[185](index=185&type=chunk) [5. PROPERTY AND EQUIPMENT](index=16&type=section&id=5.%20PROPERTY%20AND%20EQUIPMENT) This note provides details on the company's property and equipment, including depreciation and net book value Property and Equipment, Net (in thousands) | Item | Sep 30, 2019 | Dec 31, 2018 | | :-------------------------------- | :----------- | :----------- | | Lab equipment | $2,519 | $2,489 | | Leasehold improvements | $741 | $741 | | Computer hardware and software | $461 | $428 | | Furniture and fixtures | $166 | $159 | | Property and equipment, gross | $3,887 | $3,817 | | Less: accumulated depreciation and amortization | $(3,291) | $(3,090) | | Property and equipment, net | $596 | $727 | - Net property and equipment decreased from **$727 thousand** at December 31, 2018, to **$596 thousand** at September 30, 2019, primarily due to accumulated depreciation[85](index=85&type=chunk) - Depreciation and amortization expense was **$0.1 million** for the three months and **$0.2 million** for the nine months ended September 30, 2019 and 2018, recorded primarily in R&D[85](index=85&type=chunk) [6. ACCRUED LIABILITIES](index=17&type=section&id=6.%20ACCRUED%20LIABILITIES) This note presents a breakdown of the company's accrued liabilities, including clinical trial costs and restructuring liabilities Accrued Liabilities (in thousands) | Item | Sep 30, 2019 | Dec 31, 2018 | | :-------------------------------- | :----------- | :----------- | | Accrued outsourced clinical trials and preclinical studies | $2,607 | $1,129 | | Restructuring liability | $804 | $0 | | Accrued employee compensation and related taxes | $652 | $1,704 | | Total accrued liabilities | $4,857 | $3,868 | - Total accrued liabilities increased by **$989 thousand (25.6%)** from December 31, 2018, to September 30, 2019[86](index=86&type=chunk) - The increase was primarily driven by a **$1.48 million** increase in accrued outsourced clinical trials and preclinical studies and the recognition of an **$804 thousand** restructuring liability[86](index=86&type=chunk) - Accrued employee compensation and related taxes decreased by **$1.05 million**[86](index=86&type=chunk) [7. NOTES PAYABLE](index=17&type=section&id=7.%20NOTES%20PAYABLE) This note details the company's outstanding loan agreements and future principal payment obligations - The company has a **$10.0 million** loan agreement with Silicon Valley Bank (2017 SVB Loan Agreement) from November 2017, with a 30-month payment period following an 18-month interest-only period[87](index=87&type=chunk) Notes Payable (in thousands) | Item | Sep 30, 2019 | Dec 31, 2018 | | :-------------------------------- | :----------- | :----------- | | Principal amount outstanding | $8,667 | $10,000 | | Total note payable | $9,231 | $10,298 | | Less: current maturities | $(3,972) | $(2,294) | | Note payable, net of current portion | $5,259 | $8,004 | - Principal amount outstanding decreased by **$1.33 million** from December 31, 2018, to September 30, 2019[91](index=91&type=chunk) Future Annual Minimum Principal Payments (in thousands) | Year | Amount | | :--- | :----- | | 2019 | $1,000 | | 2020 | $4,000 | | 2021 | $3,667 | | Total | $8,667 | [8. COMMITMENTS AND CONTINGENCIES](index=18&type=section&id=8.%20COMMITMENTS%20AND%20CONTINGENCIES) This note outlines the company's various contractual obligations, including indemnifications, employment agreements, license fees, and lease commitments - The company has indemnification agreements with directors and officers, with unlimited potential future payments[92](index=92&type=chunk) - Employment agreements include severance packages for termination without cause or resignation for good reason, with enhanced benefits for change of control events[94](index=94&type=chunk)[95](index=95&type=chunk) - License agreements with the University of Texas, Roche Innovation Center Copenhagen A/S (RICC), t2cure GmbH, and The Brigham and Women's Hospital involve milestone payments, royalties, and annual fees for intellectual property rights[96](index=96&type=chunk)[101](index=101&type=chunk)[112](index=112&type=chunk)[117](index=117&type=chunk) - The RICC License Agreement requires milestone payments up to **$5.2 million** per licensed product, with **$0.1 million** incurred in 2019 and **$0.7 million** in 2018[102](index=102&type=chunk)[104](index=104&type=chunk) - A multi-year facility lease for office and lab space extends through August 2020, with future minimum payments of **$379 thousand**[120](index=120&type=chunk)[122](index=122&type=chunk) [9. CAPITAL STOCK](index=22&type=section&id=9.%20CAPITAL%20STOCK) This note provides information on the company's authorized and outstanding common stock, along with recent equity financing activities - The company is authorized to issue **100,000,000 shares** of Common Stock (**$0.01** par value) and **5,000,000 shares** of preferred stock[123](index=123&type=chunk) - As of September 30, 2019, **31,181,749 shares** of Common Stock were issued and outstanding[9](index=9&type=chunk) - Under the LLS Stock Purchase Agreement, the company expects to raise up to **$5.0 million** from LLS and its affiliates, with **$1.4 million** in net proceeds received as of October 31, 2019[124](index=124&type=chunk)[189](index=189&type=chunk) - Under the ATM Agreement with Cowen, the company may sell up to **$50.0 million** in Common Stock, with **$0.4 million** net proceeds received in 2019 and **$10.6 million** cumulatively since March 2017[125](index=125&type=chunk)[128](index=128&type=chunk)[188](index=188&type=chunk) [10. WARRANTS](index=23&type=section&id=10.%20WARRANTS) This note details the company's outstanding common stock warrants, including exercise prices and expiration dates Outstanding Common Stock Warrants as of September 30, 2019 | Number of Underlying Shares | Exercise Price | Expiration Date | | :-------------------------- | :------------- | :-------------- | | 10,707 | $52.50 | 2020 | | 11,718 | $8.53 | 2025 | | 24,097 | $7.15 | 2024 | | Total: 46,522 | Weighted Average: $17.93 | | - The company had **46,522** Common Stock warrants outstanding as of September 30, 2019, with a weighted average exercise price of **$17.93**[131](index=131&type=chunk) [11. SHARE-BASED COMPENSATION](index=24&type=section&id=11.%20SHARE-BASED%20COMPENSATION) This note describes the company's equity incentive plan, option activity, and share-based compensation expense recognized - The 2016 Equity Incentive Plan allows for the grant of various equity awards, with **1,290,930 shares** available for issuance as of September 30, 2019[133](index=133&type=chunk)[134](index=134&type=chunk) Common Stock Option Activity (in thousands) | Item | Number of Options | Weighted Average Exercise Price | | :-------------------------------- | :---------------- | :------------------------------ | | Outstanding at December 31, 2018 | 3,527 | $5.76 | | Granted | 1,078 | $2.89 | | Exercised | (142) | $0.60 | | Forfeited or expired | (429) | $5.49 | | Outstanding at September 30, 2019 | 4,034 | $5.20 | - The weighted-average grant-date fair value of options granted to employees and directors was **$2.31** for the three months and **$5.51** for the nine months ended September 30, 2019[139](index=139&type=chunk) Share-Based Compensation Expense (in thousands) | Item | 9 Months Ended Sep 30, 2019 | 9 Months Ended Sep 30, 2018 | | :-------------------------------- | :-------------------------- | :-------------------------- | | Research and development | $1,175 | $933 | | General and administrative | $1,792 | $1,809 | | Total share-based compensation expense | $2,967 | $2,742 | - Total unrecognized share-based compensation costs were **$6.4 million** as of September 30, 2019, to be recognized over a weighted-average period of **2.25 years**[142](index=142&type=chunk) [12. NET LOSS PER SHARE](index=26&type=section&id=12.%20NET%20LOSS%20PER%20SHARE) This note explains the calculation of net loss per share and the impact of potentially dilutive securities - Basic and diluted net loss per share are the same for all periods presented due to the company being in a loss position, making all potential common shares antidilutive[60](index=60&type=chunk)[143](index=143&type=chunk) Potentially Dilutive Securities (in thousands) | Item | Sep 30, 2019 | Sep 30, 2018 | | :-------------------------------- | :----------- | :----------- | | Options to purchase Common Stock | 4,034 | 3,517 | | Warrants to purchase Common Stock | 47 | 49 | | Total | 4,081 | 3,566 | [ITEM 2. MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS](index=28&type=section&id=ITEM%202.%20MANAGEMENT'S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) This section provides management's perspective on Miragen Therapeutics, Inc.'s financial performance, liquidity, and operational developments [Overview](index=28&type=section&id=Overview) This section introduces Miragen Therapeutics, Inc.'s business model, product candidates, and strategic direction - Miragen Therapeutics is a clinical-stage biopharmaceutical company developing RNA-targeted therapies, specifically microRNAs, for diseases with high unmet medical needs[151](index=151&type=chunk) - The company's clinical-stage product candidates include cobomarsen (miR-155 inhibitor for blood cancers), remlarsen (miR-29 replacement for fibrotic conditions), and MRG-110 (miR-92 inhibitor for heart failure and healing)[151](index=151&type=chunk)[152](index=152&type=chunk)[153](index=153&type=chunk)[154](index=154&type=chunk) - The Servier Collaboration Agreement for MRG-110 was terminated effective February 1, 2020, leading Miragen to regain global rights to MRG-110 and evaluate new development strategies[154](index=154&type=chunk) [Recent Developments and Anticipated Milestones](index=29&type=section&id=Recent%20Developments%20and%20Anticipated%20Milestones) This section highlights key operational achievements, clinical trial progress, and future strategic objectives - For cobomarsen, the SOLAR Phase 2 clinical trial for CTCL began dosing in April 2019, with primary endpoint data now expected in the second half of 2021 due to site activation delays[157](index=157&type=chunk) - The SOLAR trial is supported by a collaboration with LLS, which invested an additional **$0.5 million** in October 2019 upon achieving an enrollment milestone[158](index=158&type=chunk) - Remlarsen's Phase 2 clinical trial for keloid formation has completed enrollment, with interim data expected by year-end 2019[160](index=160&type=chunk) - MRG-110 Phase 1 clinical trials showed increased angiogenesis and reduced alpha-smooth muscle actin expression, supporting its advancement to Phase 2, subject to capital availability[163](index=163&type=chunk)[164](index=164&type=chunk) - A cost restructuring plan was announced in August 2019, eliminating **26 positions** and incurring **$1.1 million** in charges in Q3 2019, with an additional **$0.4 million** expected in Q4 2019[165](index=165&type=chunk)[166](index=166&type=chunk) [Financial Operations Overview](index=30&type=section&id=Financial%20Operations%20Overview) This section provides a general discussion of the company's revenue sources, operating expenses, and their expected trends - Revenue primarily consists of license fees, milestone payments, and R&D service reimbursements from collaboration agreements and grants[167](index=167&type=chunk) - The termination of the Servier Collaboration Agreement means no future revenue is expected from this source, and future revenue will fluctuate based on milestone achievements and product commercialization[168](index=168&type=chunk)[170](index=170&type=chunk) - Research and development expenses are expected to increase as clinical trials advance and new programs are initiated, with costs including employee-related expenses, CRO fees, manufacturing, and license fees[171](index=171&type=chunk)[175](index=175&type=chunk) - General and administrative expenses cover salaries, benefits, professional fees, and other support functions[177](index=177&type=chunk) [Critical Accounting Policies and Estimates](index=31&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) This section describes the significant accounting policies and judgments that materially affect the financial statements - The company's financial statements are prepared under U.S. GAAP, requiring estimates and assumptions, particularly for accrued expenses in clinical trials and preclinical studies[179](index=179&type=chunk)[180](index=180&type=chunk) - Accrued expenses for clinical trials are based on estimates of costs incurred for services from CROs and other providers, with adjustments possible in future periods[180](index=180&type=chunk) [Results of Operations](index=32&type=section&id=Results%20of%20Operations) This section analyzes the company's financial performance, comparing revenue and expenses across different reporting periods Comparison of Three Months Ended September 30 (in thousands) | Item | 2019 | 2018 | Change ($) | Change (%) | | :-------------------------- | :--- | :--- | :--------- | :--------- | | Revenue | $695 | $944 | $(249) | -26.4% | | Research and development | $9,027 | $7,399 | $1,628 | 22.0% | | General and administrative | $2,898 | $2,696 | $202 | 7.5% | | Net loss | $(11,230) | $(9,011) | $(2,219) | 24.6% | Comparison of Nine Months Ended September 30 (in thousands) | Item | 2019 | 2018 | Change ($) | Change (%) | | :-------------------------- | :--- | :--- | :--------- | :--------- | | Revenue | $3,581 | $7,910 | $(4,329) | -54.7% | | Research and development | $26,377 | $22,187 | $4,190 | 18.9% | | General and administrative | $9,112 | $8,354 | $758 | 9.1% | | Net loss | $(31,755) | $(22,386) | $(9,369) | 41.9% | - The decrease in nine-month revenue was primarily due to a **$3.7 million** development milestone payment in 2018 that did not recur in 2019 and a **$0.9 million** decrease in grant revenue[185](index=185&type=chunk) - Nine-month R&D expenses increased by **$4.2 million**, driven by **$2.9 million** in clinical development and manufacturing costs for cobomarsen and **$2.3 million** in personnel-related costs, partially offset by decreased technology license fees[186](index=186&type=chunk) [Liquidity and Capital Resources](index=33&type=section&id=Liquidity%20and%20Capital%20Resources) This section assesses the company's ability to meet its short-term and long-term financial obligations and its need for future capital - As of September 30, 2019, the company had **$33.8 million** in cash, cash equivalents, and short-term investments, expected to fund operations through Q2 2020[187](index=187&type=chunk) - The company has an accumulated deficit of **$158.1 million** as of September 30, 2019, and has not generated revenue from product sales[190](index=190&type=chunk) - The termination of the Servier Collaboration Agreement for MRG-110 necessitates seeking new development and licensing partners, with future development subject to capital availability[191](index=191&type=chunk)[192](index=192&type=chunk) - The cost restructuring plan in August 2019 aimed to reduce costs and focus resources on cobomarsen and microRNA-29 mimics, incurring **$1.1 million** in charges in Q3 2019[193](index=193&type=chunk) - The company will require substantial additional capital through equity/debt financings or collaborations to fund ongoing clinical development and operations[194](index=194&type=chunk) Summarized Cash Flows (in thousands) | Cash Flow Activity | 9 Months Ended Sep 30, 2019 | 9 Months Ended Sep 30, 2018 | | :-------------------------------- | :-------------------------- | :-------------------------- | | Operating activities | $(28,250) | $(18,669) | | Investing activities | $21,226 | $(38,998) | | Financing activities | $(725) | $41,926 | | Total | $(7,749) | $(15,741) | [ITEM 3. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK](index=36&type=section&id=ITEM%203.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURES%20ABOUT%20MARKET%20RISK) This section addresses the company's exposure to market risks and how they are managed - The company has no applicable quantitative and qualitative disclosures about market risk[207](index=207&type=chunk) [ITEM 4. CONTROLS AND PROCEDURES](index=36&type=section&id=ITEM%204.%20CONTROLS%20AND%20PROCEDURES) This section reports on the effectiveness of the company's disclosure controls and internal control over financial reporting - Management, including the CEO and CFO, concluded that the company's disclosure controls and procedures were effective at a reasonable level of assurance as of September 30, 2019[209](index=209&type=chunk) - No changes in internal control over financial reporting occurred during the most recent fiscal quarter that materially affected, or are reasonably likely to materially affect, internal control over financial reporting[210](index=210&type=chunk) [PART II. OTHER INFORMATION](index=37&type=section&id=PART%20II.%20OTHER%20INFORMATION) This section provides additional legal, risk, and administrative information not covered in the financial statements [ITEM 1. LEGAL PROCEEDINGS](index=37&type=section&id=ITEM%201.%20LEGAL%20PROCEEDINGS) This section discloses any material legal actions or proceedings involving the company - The company is not currently involved in any legal proceedings that are expected to have a material adverse effect on its business, financial condition, or results of operations[212](index=212&type=chunk) [ITEM 1A. RISK FACTORS](index=37&type=section&id=ITEM%201A.%20RISK%20FACTORS) This section details the significant risks and uncertainties that could materially impact the company's business, financial condition, and future prospects [Risks Related to Our Financial Condition and Capital Requirements](index=37&type=section&id=Risks%20Related%20to%20Our%20Financial%20Condition%20and%20Capital%20Requirements) This section outlines risks associated with the company's liquidity, funding needs, and ability to continue operations - The company's current liquidity position and recurring losses raise substantial doubt about its ability to continue as a going concern, requiring additional capital beyond Q2 2020[214](index=214&type=chunk)[219](index=219&type=chunk) - Failure to raise additional capital could force the company to delay or discontinue product development, reduce headcount, seek unfavorable strategic alliances, or cease operations[216](index=216&type=chunk)[217](index=217&type=chunk)[218](index=218&type=chunk) - The company has an accumulated deficit of **$158.1 million** as of September 30, 2019, and expects significant losses to continue as product candidates advance through clinical trials[221](index=221&type=chunk)[225](index=225&type=chunk) - Raising additional capital through equity sales will dilute existing stockholders, and debt financing may impose restrictive covenants[233](index=233&type=chunk) [Risks Related to the Development of Our Product Candidates](index=41&type=section&id=Risks%20Related%20to%20the%20Development%20of%20Our%20Product%20Candidates) This section addresses the inherent risks in clinical trials, regulatory approvals, and the unproven nature of microRNA-targeted therapies - Clinical trials are costly, time-consuming, and inherently risky, with potential for delays or failures due to various factors including patient enrollment difficulties, adverse events, or regulatory changes[235](index=235&type=chunk)[236](index=236&type=chunk)[258](index=258&type=chunk)[259](index=259&type=chunk) - The microRNA-targeted therapeutic approach is unproven, with no such therapeutics yet approved, increasing development complexity and uncertainty[239](index=239&type=chunk)[241](index=241&type=chunk)[242](index=242&type=chunk) - Product candidates may cause undesirable side effects, leading to clinical trial delays, regulatory approval limitations, or significant negative consequences post-approval[244](index=244&type=chunk)[245](index=245&type=chunk)[246](index=246&type=chunk) - Negative public opinion or increased regulatory scrutiny of microRNA therapies could damage public perception and hinder development or approval[249](index=249&type=chunk) - The company is heavily dependent on the success of its early-stage clinical candidates, and earlier preclinical/clinical results may not predict future success[250](index=250&type=chunk)[256](index=256&type=chunk) [Risks Related to Regulatory Approval of Our Product Candidates and Other Legal Compliance Matters](index=47&type=section&id=Risks%20Related%20to%20Regulatory%20Approval%20of%20Our%20Product%20Candidates%20and%20Other%20Legal%20Compliance%20Matters) This section covers risks associated with obtaining and maintaining regulatory approvals, as well as compliance with healthcare laws and government funding requirements - Breakthrough Therapy or Fast Track designations may not lead to faster development or approval, nor do they guarantee marketing approval[270](index=270&type=chunk)[271](index=271&type=chunk)[272](index=272&type=chunk) - Accelerated approval for product candidates, including cobomarsen, may require confirmatory trials, and failure to verify clinical benefit or comply with post-approval requirements could lead to withdrawal of approval[274](index=274&type=chunk)[275](index=275&type=chunk)[276](index=276&type=chunk) - Approved products will be subject to ongoing regulatory requirements (manufacturing, labeling, promotion, safety reporting), and non-compliance could result in fines, withdrawal of approval, or other penalties[278](index=278&type=chunk)[279](index=279&type=chunk)[282](index=282&type=chunk) - Healthcare legislative reforms (e.g., ACA challenges, drug pricing measures) could adversely affect the business by limiting reimbursement or increasing pricing pressures[286](index=286&type=chunk)[287](index=287&type=chunk)[289](index=289&type=chunk) - Non-compliance with healthcare fraud and abuse laws (Anti-Kickback, False Claims, HIPAA, GDPR) could lead to substantial penalties, exclusion from government programs, and reputational harm[290](index=290&type=chunk)[291](index=291&type=chunk)[293](index=293&type=chunk)[294](index=294&type=chunk)[309](index=309&type=chunk) - Reliance on government funding may impose requirements that limit actions, increase costs, and subject the company to potential financial penalties[295](index=295&type=chunk)[296](index=296&type=chunk)[301](index=301&type=chunk) [Risks Related to Our Intellectual Property](index=54&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) This section discusses challenges in obtaining, maintaining, and enforcing intellectual property rights, including patent protection and potential infringement claims - The company may not successfully obtain or maintain necessary intellectual property rights for microRNA targets, product compounds, and processes through acquisitions and in-licenses, hindering business growth[312](index=312&type=chunk)[314](index=314&type=chunk) - Reliance on patents, trade secrets, and confidentiality agreements carries risks, including failure to obtain broad patent claims, challenges to validity, and difficulty in protecting trade secrets[315](index=315&type=chunk)[317](index=317&type=chunk)[329](index=329&type=chunk)[330](index=330&type=chunk) - Changes in U.S. patent law, such as the Leahy-Smith America Invents Act and Supreme Court rulings, could diminish patent value and increase prosecution/enforcement costs[323](index=323&type=chunk)[324](index=324&type=chunk)[326](index=326&type=chunk)[328](index=328&type=chunk) - Third-party claims of intellectual property infringement are a significant risk in the biotechnology industry, potentially leading to substantial litigation expenses, damages, or injunctions[331](index=331&type=chunk)[334](index=334&type=chunk)[335](index=335&type=chunk) - Failure to comply with license agreement obligations or disruptions with licensors could result in loss of critical license rights[342](index=342&type=chunk) - Protecting intellectual property rights globally is expensive and challenging, as foreign laws may offer less protection, and enforcement actions can be costly and divert resources[347](index=347&type=chunk)[349](index=349&type=chunk)[350](index=350&type=chunk) [Risks Related to Our Reliance on Third Parties](index=59&type=section&id=Risks%20Related%20to%20Our%20Reliance%20on%20Third%20Parties) This section highlights risks associated with dependence on third-party contractors for clinical trials, manufacturing, and potential collaborations - The company relies heavily on third-party CROs to conduct clinical trials and manufacturers for product candidates, posing risks if these parties fail to perform or comply with regulations[351](index=351&type=chunk)[353](index=353&type=chunk)[355](index=355&type=chunk) - Disruptions in the supply of product candidates or active ingredients from third-party manufacturers could delay clinical trials and regulatory approval[354](index=354&type=chunk) - Commercial manufacturing costs for product candidates are uncertain and may not be commercially feasible, impacting viability[357](index=357&type=chunk) - Collaborations, if formed, carry risks such as insufficient resource commitment, disagreements, and potential termination, as seen with the Servier Collaboration Agreement[361](index=361&type=chunk)[362](index=362&type=chunk) - Indemnification agreements with third parties could lead to substantial liabilities if obligations exceed insurance coverage or collaborators fail to indemnify[363](index=363&type=chunk)[365](index=365&type=chunk) [Risks Related to Commercialization of Our Product Candidates](index=62&type=section&id=Risks%20Related%20to%20Commercialization%20of%20Our%20Product%20Candidates) This section outlines challenges in marketing, sales, competition, market acceptance, and reimbursement for the company's product candidates - Limited marketing and sales experience means the company must establish capabilities or find collaborators, with potential for delays or failure to generate revenue[366](index=366&type=chunk)[367](index=367&type=chunk) - Forming strategic collaborations is competitive and complex; failure to do so could alter development plans and require significant internal investment[368](index=368&type=chunk)[369](index=369&type=chunk)[370](index=370&type=chunk) - Market opportunities for product candidates may be smaller than anticipated, requiring significant market share to achieve profitability, especially for rare diseases like MF[372](index=372&type=chunk) - The company faces substantial competition from major pharmaceutical and biotechnology companies with greater resources, potentially leading to faster product development or market dominance by competitors[373](index=373&type=chunk)[374](index=374&type=chunk)[376](index=376&type=chunk) - Commercial success depends on market acceptance by physicians, patients, and third-party payors, influenced by efficacy, safety, cost, and reimbursement[378](index=378&type=chunk)[379](index=379&type=chunk) - Failure to obtain or maintain adequate reimbursement or insurance coverage for products could limit marketability and revenue generation[385](index=385&type=chunk)[387](index=387&type=chunk)[388](index=388&type=chunk) [Risks Related to Our Business Operations](index=66&type=section&id=Risks%20Related%20to%20Our%20Business%20Operations) This section covers operational risks, including key personnel retention, organizational growth, IT systems, and tax implications - Future success depends on retaining key personnel, including the CEO, and attracting/motivating other qualified scientific and technical staff in a competitive industry[391](index=391&type=chunk) - Expanding the organization will require additional managerial and operational resources, and difficulties in managing this growth could disrupt operations[392](index=392&type=chunk) - Failure to effectively manage operational changes, including past and potential future restructuring charges (e.g., August 2019 plan), could harm business and financial performance[393](index=393&type=chunk)[394](index=394&type=chunk)[395](index=395&type=chunk) - Failure in IT and storage systems could disrupt business operations, lead to unauthorized data access, and result in fines or litigation[397](index=397&type=chunk) - The ability to use net operating losses (NOLs) to offset future taxable income may be limited due to expiration periods and ownership change rules (Section 382 of the Code), potentially increasing future tax liabilities[398](index=398&type=chunk)[399](index=399&type=chunk)[400](index=400&type=chunk) - New or future changes to tax laws, such as the Tax Cuts and Jobs Act of 2017, could adversely affect the company's effective tax rate and financial condition[401](index=401&type=chunk)[403](index=403&type=chunk) [Risks Related to Ownership of our Common Stock](index=68&type=section&id=Risks%20Related%20to%20Ownership%20of%20our%20Common%20Stock) This section addresses risks concerning stock price volatility, listing requirements, corporate governance, and dividend policy - The market price of the company's common stock is expected to be volatile due to various factors, including regulatory approvals, clinical trial results, competition, and macroeconomic conditions[404](index=404&type=chunk)[405](index=405&type=chunk)[407](index=407&type=chunk) - Failure to meet Nasdaq Capital Market listing requirements, such as the minimum bid price, could result in delisting, negatively impacting stock price and liquidity[409](index=409&type=chunk)[410](index=410&type=chunk)[411](index=411&type=chunk)[413](index=413&type=chunk)[414](index=414&type=chunk) - Compliance with public company laws and regulations (e.g., Sarbanes-Oxley Act) incurs significant costs and demands on management, which will increase after ceasing to be an 'emerging growth company' on December 31, 2019[415](index=415&type=chunk)[429](index=429&type=chunk) - Anti-takeover provisions in charter documents and Delaware law could make acquisitions more difficult and prevent stockholders from replacing management[416](index=416&type=chunk) - The company does not anticipate paying cash dividends in the foreseeable future, making capital appreciation the sole source of gain for stockholders[418](index=418&type=chunk) - Failure to maintain proper and effective internal controls could impair the ability to produce accurate financial statements, leading to loss of investor confidence and a negative impact on stock price[424](index=424&type=chunk)[426](index=426&type=chunk)[427](index=427&type=chunk)[428](index=428&type=chunk) [ITEM 2. UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS](index=72&type=section&id=ITEM%202.%20UNREGISTERED%20SALES%20OF%20EQUITY%20SECURITIES%20AND%20USE%20OF%20PROCEEDS) This section reports on any unregistered sales of equity securities and the application of their proceeds - Not applicable for this reporting period[430](index=430&type=chunk) [ITEM 3. DEFAULTS UPON SENIOR SECURITIES](index=72&type=section&id=ITEM%203.%20DEFAULTS%20UPON%20SENIOR%20SECURITIES) This section discloses any defaults on senior securities by the company - Not applicable for this reporting period[431](index=431&type=chunk) [ITEM 4. MINE SAFETY DISCLOSURES](index=72&type=section&id=ITEM%204.%20MINE%20SAFETY%20DISCLOSURES) This section provides disclosures related to mine safety, if applicable to the company's operations - Not applicable for this reporting period[432](index=432&type=chunk) [ITEM 5. OTHER INFORMATION](index=72&type=section&id=ITEM%205.%20OTHER%20INFORMATION) This section includes any other material information not covered in previous items - Not applicable for this reporting period[433](index=433&type=chunk) [ITEM 6. EXHIBITS](index=73&type=section&id=ITEM%206.%20EXHIBITS) This section lists all documents filed as exhibits to the report, including agreements and certifications - The exhibits include corporate governance documents (Certificate of Incorporation, Bylaws), key agreements (Amendment to Research Subaward Agreement with Yale University, Assignment and Assumption Agreement with LLS TAP Miragen, LLC, Amendment to Amended and Restated License Agreement with Roche Innovation Center Copenhagen A/S), and certifications (Rule 13a-14(a), 18 U.S.C. 1350)[436](index=436&type=chunk) [FORWARD-LOOKING STATEMENTS](index=27&type=section&id=FORWARD-LOOKING%20STATEMENTS) This section provides a cautionary note regarding forward-looking statements, highlighting inherent risks and uncertainties - The report contains forward-looking statements based on current expectations, involving substantial risks and uncertainties, which may cause actual results to differ materially[146](index=146&type=chunk)[147](index=147&type=chunk) - Readers are cautioned not to place undue reliance on forward-looking statements and are advised to review the 'Risk Factors' section for a comprehensive understanding of business risks[147](index=147&type=chunk)[148](index=148&type=chunk) [SIGNATURES](index=75&type=section&id=SIGNATURES) This section contains the official certifications and signatures of the company's principal executive and financial officers - The report is duly signed by William S. Marshall, Ph.D., Chief Executive Officer, and Jason A. Leverone, Chief Financial Officer, on November 8, 2019[442](index=442&type=chunk)

miRagen Therapeutics, Inc. (MGEN) Q2 2019 Earnings Conference Call August 7, 2019 4:30 PM ET Company Participants Dan Ferry – Managing Director-LifeSci Advisors, LLC Bill Marshall – President and Chief Executive Officer Jason Leverone – Chief Financial Officer Conference Call Participants Liana Moussatos – Wedbush Securities Jonathan Miller – Evercore ISI Jennifer Grayson – R. W. Baird Leland Gershell – Oppenheimer Suji Jeong – Jefferies Operator Greetings and welcome to the miRagen Therapeutics’ Q2 2019 Ea ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2019 or o 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 MIRAGEN THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware (State or other j ...

Restoring Biological Harmony for Patients with Debilitating Disease Pg. 1 miRagen Therapeutics NASDAQ: MGEN Jefferies 2019 Healthcare Conference June 2019 Cautionary Note Regarding Forward-Looking Statements Pg. 2 This presentation contains forward-looking statements relating to Miragen 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 a ...

miRagen Therapeutics, Inc. (MGEN) Q1 2019 Results Earnings Conference Call May 8, 2019 4:30 PM ET Company Participants Dan Ferry - LifeSci Advisors Bill Marshall - President and Chief Executive Officer Jason Leverone - Chief Financial Officer Paul Rubin - Executive Vice President of Research and Development Adam Levy - Chief Business Officer Conference Call Participants Jonathan Miller - Evercore ISI Liana Moussatos - Wedbush Securities Eun Yang - Jefferies Madhu Kumar - Robert W. Baird George Zavoico - B. ...