 Progress on next generation microRNA-29 mimic for use in pulmonary fibrosis MRG-229 includes new preclinical data; additional NIH grant funding triggered Exploratory preclinical safety and pharmacokinetic data for MRG-229 expected before year end Plan to receive guidance from the FDA on proposed regulatory pathway for cobomarsen in ATLL before year end A majority of patients in the Phase 2 SOLAR trial of cobomarsen in CTCL have continued to receive uninterrupted treatment and are being evaluated for clinical response Reported 30.6 million in cash and cash equivalents as of June 30, 2020; anticipate cash runway into Q3 2021BOULDER, Colo., Aug. 05, 2020 GLOBE NEWSWIRE -- miRagen Therapeutics, Inc. NASDAQ: MGEN, a clinical-stage biopharmaceutical company focused on the discovery and development of proprietary RNA-targeted therapies with a specific focus on microRNAs, today reported financial results for the second quarter ended June 30, 2020 and provided business updates.The miRagen team continues to execute on our strategy across multiple product opportunities, by pursuing guidance from the FDA on a development pathway for cobomarsen in ATLL, delivering preclinical data for MRG-229 in pulmonary fibrosis models that triggered NIH funding to support large animal safety and pharmacokinetic studies, and working closely with our clinical centers to ensure our ability to deliver topline data from the planned interim analysis in the Phase 2 SOLAR trial, said William S. Marshall, Ph.D., miRagens President and Chief Executive Officer.We are encouraged by the preclinical and clinical safety and efficacy data we have generated across all of our programs and energized about the potential of microRNA-based therapies to bring benefit to patients in need.Summary of Program Highlights this QuarterCobomarsen: Cobomarsen is currently being evaluated for the potential treatment of patients with miR-155 elevated hematological malignancies, including Cutaneous T-Cell Lymphoma CTCL and Adult T-Cell LeukemiaLymphoma ATLL. CTCL: As a result of the COVID-19 pandemic, the Company has seen an impact on clinical activities at some sites where the SOLAR trial is being conducted. However, with a majority of patients in the SOLAR trial continuing to receive uninterrupted treatment and evaluation for clinical response, the impact has been limited to a small number of patients. As of July 17, 2020, 34 of the 37 CTCL patients are being followed for a response, and 32 have continued to receive uninterrupted treatment. ATLL: Based on the clinical data released earlier this year, during the second quarter of 2020, the Company requested a meeting with the FDA to discuss the development path for cobomarsen in ATLL and plans to meet with the FDA and receive guidance on the Companys proposed clinical path in this indication before the end of 2020.MRG-229: MRG-229 is a miR-29 mimic, or replacement for miR-29, a microRNA that is found at abnormally low levels in a number of pathologic fibrotic conditions. Idiopathic Pulmonary Fibrosis IPF: In June 2020, the Company hosted a key opinion leader KOL webcast to discuss the current and increasing unmet medical need in treating patients with IPF and reported additional preclinical rodent safety and in vitro human efficacy data for MRG-229. In summary, the latest preclinical observations were: - Next-generation targeted miR-29 mimics demonstrated down-regulation of targeted genetic pathways and anti-fibrotic effects including decreased collagen secretion in diseased lung fibroblasts in vitro, highlighting functional activity in addition to molecular readouts. - Anti-fibrotic activity of next-generation miR-29 mimics in mouse bleomycin-induced pulmonary fibrosis was observed for both intravenous and subcutaneous routes of administration. - Next-generation targeted miR-29 mimics demonstrated efficacy in ex vivo profibrotic-induced human precision cut lung slices. - Exploratory toxicity studies in rodents showed that MRG-229 treatment resulted in no adverse effects on organ histology, hematology, clinical chemistries, coagulation, or urinalysis.Based on these results, the Company has initiated a non-human primate toxicology study and expects to report additional preclinical safety and efficacy data before the end of 2020.Financial Results Cash Position and Runway: Cash and cash equivalents were 30.6 million as of June 30, 2020, compared to cash, cash equivalents, and short-term investments of 26.8 million as of December 31, 2019. Net cash used in operating activities was 6.8 million for the second quarter of 2020 as compared to 7.2 million during the second quarter of 2019. miRagen believes that its current cash and cash equivalents will be sufficient to fund the Companys operations into the third quarter of 2021. Revenue: Revenue was 0.2 million for the second quarter of 2020, compared to 2.5 million for the second quarter of 2019. The decrease in revenue was primarily due to a decrease in research and development activities reimbursable to the Company under a prior license and collaboration agreement. Research and Development Expenses: Research and development expenses were 3.8 million for the second quarter of 2020, compared to 8.6 million for the second quarter of 2019. The 4.8 million decrease in research and development expenses was primarily attributable to a decrease in clinical and related manufacturing development activities associated with the Phase 2 SOLAR clinical trial of cobomarsen, personnel-related costs, and other miscellaneous expenses during the second quarter of 2020. General and Administrative Expenses: General and administrative expenses were 2.7 million for the second quarter of 2020, compared to 2.9 million for the second quarter of 2019. The decrease in general and administrative expenses was due primarily to decreased personnel-related costs, which were partially offset by increases in legal costs during the second quarter of 2020. Net Loss: The Companys net loss was 6.4 million, or 0.12 per share, for the second quarter of 2020, compared to 8.9 million, or 0.29 per share, for the second quarter of 2019.Conference Call Information The miRagen Therapeutics management team will host a conference call and webcast today at 4:30 p.m. ET to provide and discuss its financial results for the second quarter 2020. To access the call, please dial 877-407-0789 domestic or 201-689-8562 international and provide the passcode 13706539. A live webcast of the call will be available on the Investors section of the miRagen Therapeutics website at and a replay of this conference call will be available approximately one hour after its completion.About miRagen Therapeutics, Inc.Miragen Therapeutics, Inc. is a clinical-stage biopharmaceutical company discovering and developing proprietary RNA-targeted therapies with a specific focus on microRNAs and their role in diseases where there is a high unmet medical need. miRagen has three clinical stage product candidates, cobomarsen, remlarsen, and MRG-110. miRagens clinical product candidate for the treatment of certain cancers, cobomarsen, is an inhibitor of miR-155, which is found at abnormally high levels in malignant cells of several blood cancers. miRagens clinical product candidate for the treatment of pathological fibrosis, remlarsen, is a replacement for miR-29, which is found at abnormally low levels in a number of pathological fibrotic conditions, including cutaneous, cardiac, renal, hepatic, pulmonary and ocular fibrosis, as well as in systemic sclerosis. MRG-110, an inhibitor of miR-92, is miRagens product candidate for the treatment of heart failure and other ischemic disease. In addition to these programs, miRagen is developing a pipeline of preclinical product candidates. The goal of miRagens translational medicine strategy is to progress rapidly to first-in-human studies once it has established the pharmacokinetics, pharmacodynamic, safety, and manufacturability of the product candidate in preclinical studies. For more information, please visit For information on clinical trials please visit Regarding Forward-Looking StatementsThis press release may contain forward-looking statements that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements contained in this press release other than statements of historical fact, including statements regarding miRagens strategy, anticipated clinical development milestones, future operations, future financial position, future revenue, projected expenses, prospects, plans and objectives of management or the expected features of or potential indications for miRagens product candidates are forward-looking statements. The words believe, may, will, estimate, continue, anticipate, intend, plan, expect, predict, potential, opportunity, goals, or should, and similar expressions are intended to identify forward-looking statements. Such statements are based on managements current expectations and involve risks and uncertainties. 