miRagen Therapeutics announced a series of strategic changes across its business that are intended to reallocate its existing capital to deliver on milestones in 2020.
The changes include a revised development strategy aimed at delivering data in 2020 from a modified Phase 2 SOLAR clinical trial of cobomarsen in cutaneous T-cell lymphoma (CTCL), consulting with the U.S. Food and Drug Administration (FDA) to define a development path for cobomarsen in adult T-cell leukemia/lymphoma (ATLL), and focusing future pipeline development efforts primarily on MRG-229, a novel second generation miR-29 mimic and potential treatment in patients with idiopathic pulmonary fibrosis (IPF).
These changes will also result in a workforce reduction that will impact approximately 18 employees. After changes to the SOLAR clinical trial and the reduction in workforce, the Company believes its cash and cash equivalents will now be sufficient to fund its operations into the fourth quarter of 2020.
The reductions are primarily in positions relating to research and development and corresponding project, general and administrative support. miRagen estimates that it will incur approximately $0.7 million in restructuring charges primarily for severance and other related costs for the employees impacted by the reduction in force over the next seven months. In addition, the Company plans to enter into severance and retention bonus agreements with its remaining workforce.
“We are executing on a strategy to streamline our operations which we believe will allow us to focus our development efforts and deliver important milestones in 2020,” said William S. Marshall, Ph.D., President and Chief Executive Officer of miRagen. “Delivering controlled data in CTCL and gaining clarity on the development path for cobomarsen in ATLL are important aspects in support of our belief that cobomarsen has the potential to be a broad-based therapy for the treatment of cancer patients with elevated levels of miR-155.”
The Company also announced Paul Rubin, M.D., Executive Vice President of R&D, is leaving the Company effective December 31, 2019.
“Paul has made significant contributions to miRagen on multiple levels,” Dr. Marshall said. “He has led our research and development efforts for the last three years resulting in important advances in our clinical and preclinical pipeline with the goal of helping to serve patients around the world. We wish Paul the best in his future endeavors and appreciate the ability to continue to seek advice and guidance in the future.”
The Company has named Diana Escolar, M.D. as Chief Medical Officer. Dr. Escolar joined the Company in January 2018 and has served as miRagen’s Senior Vice President of Clinical Sciences. Dr. Escolar has helped to lead the clinical development strategies and implement the Company’s clinical programs over the last two years.
“We are pleased that Diana will continue to lead our clinical sciences efforts and expand her role as the Chief Medical Officer,” Dr. Marshall said. “She is a dynamic physician scientist with strong capabilities in clinical development, especially in the rare disease setting.”
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The Company will stop the enrollment of new patients in its Phase 2 clinical trial of cobomarsen, SOLAR, effective as of the end of the 2019. Patients enrolled in the trial at that time will continue to be evaluated for safety and clinical response. The Company initially planned to enroll up to 126 patients and now expects to enroll approximately 30 patients. Despite the reduction in patient enrollment, the Company believes that evaluation of data from this set of patients can provide important evidence regarding the safety and efficacy of cobomarsen for the treatment of CTCL in a shorter period of time and require fewer resources.
miRagen is also evaluating cobomarsen in a Phase 1 basket trial of other cancers where the disease process appears to be correlated with an increase in miR-155 levels, ATLL, diffuse large B-cell lymphoma, and chronic lymphocytic leukemia. In this clinical trial, the Company believes that cobomarsen has demonstrated promising interim results in several ATLL patients. Based on these interim results, the Company is announcing today that it is focusing its cobomarsen expansion indication efforts on ATLL and will request a meeting with the FDA to explore a potential expedited development pathway for cobomarsen in ATLL. The Company expects to have a meeting with the FDA in the second quarter of 2020.
The Company is developing miR-29 mimics or replacements for miR-29, a microRNA that is found at abnormally low levels in a number of pathological fibrotic conditions. The Company’s lead microRNA-29 mimics are remlarsen and MRG-229. Remlarsen is the Company’s most advanced product candidate in fibrosis, which is currently being evaluated in a Phase 2 clinical trial assessing its safety, tolerability, and activity in the potential prevention or reduction of keloid formation in patients with a history of keloid scars, a form of pathological scarring. The Company reported interim data from this clinical trial, which suggests that remlarsen was generally safe and well tolerated, treatment had no negative effect on healing reported and initial volume reductions in treated keloids compared to placebo in a subset of patients were observed. Based on this data, the Company has decided to continue its analysis of patient data at the one-year primary endpoint of the clinical trial. With this data, the Company may seek a collaboration partner for the future development of remlarsen.
In addition, based on preclinical data with MRG-229, the Company announced that its pipeline development efforts and allocation of future capital will be primarily focused on the development of MRG-229 for IPF. The Company believes that the efficacy and safety profile of MRG-229 positions it as a potentially differentiated approach to the treatment of IPF. miRagen expects to report additional preclinical-safety and -efficacy data during the first half of 2020. This program is supported by a grant in collaboration with the National Institutes of Health and Yale University.
“We have also been encouraged by the preclinical data we have generated with our next generation microRNA-29 mimic, MRG-229, and will primarily focus our pipeline development efforts and allocation of future capital on advancing MRG-229. We believe that the preclinical data we have collected demonstrate that MRG-229 has the potential for superior efficacy in treating patients with IPF, a fibrotic disease with high unmet medical need. If effective, MRG-229 possesses a mechanism of action that we believe could be particularly valuable for patients with this deadly disease,” Dr. Marshall said.