Why This Mostly-Unheard of Colorado Biotech Could Hit $1B in Revenue by 2025
miRagen is focused on RNA-based therapeutics, with an even more specific focus on microRNAs. microRNAs are about 20 to 25 nucleotides long and are single-stranded RNA molecules that regulate gene expression. They are involved in the pathways responsible for many diseases, including cardiovascular disease and fibrosis. They are transcribed from genes, but, unlike messenger RNA (mRNA), don’t encode for proteins.
microRNAs are believed to play a subtle but important function in fine-tuning gene expression which becomes more pronounced during stress or disease conditions. Over 1000 microRNAs have been identified in humans.
The company’s pipeline includes MRG-106 for cutaneous T-cell lymphoma, adult T-cell lymphoma/leukemia and other MIR-155 elevated non-Hodgkin’s lymphoma; MRG-201 for cutaneous fibrosis, idiopathic pulmonary fibrosis; MRG-107 for ALS; and MRG-110, which it has partnered with Servier for heart failure and other ischemic diseases. The programs further along are MRG-106 for cutaneous T-cell lymphoma and MRG-201 for cutaneous fibrosis, both of which are wrapping up Phase I trials.
On Dec. 11, 2017, the company presented new interim clinical data on MRG-106 on mycosis fungoides form of cutaneous T-cell lymphoma. It was presented at the 2017 American Society of Hematology (ASH) Annual Meeting. The data showed that nine out of 14 patients treated for more than a month showed a 50 percent or greater improvement in total skin disease, and seven patients had a 50 percent improvement that lasted at least four months. The company also initiated dosing in three additional oncology indications, including chronic lymphocytic leukemia, diffuse large B-cell lymphoma and adult T-cell leukemia/lymphoma.
“We believe the new MRG-106 Phase I data further validate the utility of micro-RNA based therapeutics and provide important insights that may help guide potential Phase II clinical trial designs and expanded indication trials,” said William Marshall, the company’s president and chief executive officer, in a statement. “We believe that these findings suggest that, if approved, MRG-106 has the potential to offer profound benefits to patients with CTCL. We are currently evaluating MRG-106 in additional hematologic indications within the current Phase I trial, and plan to release interim data from these expansion indications in 2018.”
Much of Cann’s optimistic analysis of the company’s future is based on MRG-106 being approved in about 2022 and MRG-201 in cutaneous fibrosis in 2025, neither of which is right around the corner. As Benzinga reports, “The biotech company will have its first product revenues of $414.4 million in 2022, the analyst said. This could potentially grow to $1.2 billion in 2025 if both product candidates are successful in their planned indications, she said.”
It should be noted that there are plenty of opportunities for failure for drugs between Phase I and Phase III trials, as well as between 2018 and 2025.
“We estimate that miRagen’s operating losses will continue until the launch of its first compound and that the company will turn profitable in 2022 as revenue starts to meaningfully offset losses,” Cann wrote.
In order to launch those two programs and carry them from their current development stage to commercial products, Oppenheimer indicates miRage will need to raise $600 million.