miRagen Restructures to Focus on Fibrosis, Inks $20M Stock Deal

Xconomy Boulder/Denver — 

MicroRNA therapies developer miRagen Therapeutics is scaling back work on its most advanced cancer drug candidate and shifting resources to a compound being developed to treat fibrosis in the lungs.

The pipeline shuffle will be accompanied by a corporate shakeup. Boulder, CO-based miRagen (NASDAQ: MGEN) is also laying off some of its staff. The company said late Wednesday that those changes will leave the company with enough cash to keep going until the fourth quarter of next year.

MiRagen is developing drugs based on microRNA, which are RNA molecules that regulate gene expression and can influence pathways associated with some diseases. The company’s experimental microRNA cancer drug, cobomarsen, is in Phase 2 testing for cutaneous T cell lymphoma, a rare blood cancer of the T cells.

MiRagen says it will stop enrolling new patients in the cobomarsen Phase 2 study at the end of this year. That means the study, which was expected to enroll 126 patients, will have about 30. Those patients will continue to be evaluated to assess safety and response to the treatment. Despite cutting study enrollment, miRagen says it believes this smaller patient group can provide enough safety and efficacy data to decide its next steps for winning FDA approval. Preliminary data from this amended clinical trial is expected in the third quarter of 2020.

The strategy shift means that about 18 people will be laid off, miRagen says. According to the company’s annual report, miRagen had 77 full-time employees at the end of last year. As work on cobomarsen scales back, miRagen will turn its focus to its experimental treatments for fibrosis, the formation of scar tissue in organs such as the heart, lungs, liver, and kidneys. The company’s fibrosis drugs are intended to replace a microRNA called miR-29, whose levels are abnormally low in patients with a number of fibrotic conditions.

MiRagen says its research efforts and remaining cash will be directed to MRG-229, a preclinical compound being developed to treat idiopathic pulmonary fibrosis (IPF), which affects the lungs. The company believes its drug has the potential to offer a different approach to treating the disorder, which has few FDA-approved treatments. Additional preclinical data are expected in the first half of next year.

A fibrosis focus puts miRagen in company with several other biotechs with drugs in various stages of clinical development. Companies developing treatments for IPF and other forms of fibrosis include Austin, TX-based Lung Therapeutics; Scholar Rock (NASDAQ: [[ticker:SRRK) of Cambridge, MA, which has a partnership with Gilead Sciences (NASDAQ: GILD); and Pliant Therapeutics of South San Francisco.

The miRagen MRG-229 program is currently financially supported by grant funding from the National Institutes of Health and Yale University. The company now has a stock deal that provides more cash for that work. On Wednesday, it signed an agreement with Aspire Capital Fund, which purchased $1 million worth of mirRagen shares and will buy up to $19 million worth of stock over the next 30 months. MiRagen says the cash from these stock sales will support MRG-229 research.

The most advanced miRagen fibrosis compound, remlarsen, is in a Phase 2 study assessing its effect on patients who have keloid scars, a form of skin scarring. Based on results so far, miRagen plans to analyze patient data at the one-year primary endpoint of the clinical trial. Then, if the results support additional development, miRagen may seek to  collaborate with another company to further advance the experimental drug.

Photo by Caitlin Bigelow