miRagen Announces New Interim Data From Phase 1 Clinical Trial of Cobomarsen in Mycosis Fungoides

On May 9, 2018

miRagen Therapeutics announced new interim data from its Phase 1 clinical trial of cobomarsen in patients with the mycosis fungoides (MF) form of cutaneous T-cell lymphoma (CTCL). The data will be presented May 9, 2018, at the “2015… 2018 T-Cell Lymphomas: we are close to the finalization” medical meeting in Bologna, Italy, by Christiane Querfeld, M.D., Ph.D., Chief of the Division of Dermatology, and Director, Cutaneous Lymphoma Program at the City of Hope in Duarte, California.

“These additional cobomarsen Phase 1 data provide further evidence that microRNA-based therapeutics have the potential to improve the quality of life for patients living with mycosis fungoides,” said miRagen President and CEO William S. Marshall, Ph.D. “Cobomarsen continues to be safe and generally well tolerated at all doses tested in the Phase 1 trial, with multiple patients receiving more than a year of therapy with no serious adverse events attributed to the drug. In addition, the improvement in skin disease observed in the trial appears to be durable and is accompanied by improvements in metrics that measure the patients’ quality of life. These data support our plans to initiate a Phase 2 clinical trial for cobomarsen in patients with CTCL in the second half of 2018.”

The interim results being presented at the conference include safety observations from longer-term dosing of existing patients who have continued participation in the trial and quality of life assessments from 18 patients in the trial. As of April 30, 2018, highlights include the following:

Cobomarsen treatment resulted in durable improved quality of life, as measured by the Skindex-29 Total Score.
- 13 of 18 subjects showed an improvement over the first 100 days on cobomarsen.
- Improvement and stabilization remain durable in four subjects for up to one year, and one subject was stable after more than 400 days on cobomarsen.
Cobomarsen continued to be generally well tolerated at all dose levels evaluated.
- Multiple patients received more than a year of therapy (up to 39 grams cumulative dose) with no serious adverse events attributed to cobomarsen.
- 300 and 600 mg intravenous infusions had similar efficacy and tolerability, offering the most consistent response rate based on skin mSWAT scores, which is a measurement of the severity of skin disease over a patient’s entire body.

“These encouraging longer-term data demonstrated that cobomarsen treatment resulted in durable improved quality of life for patients with MF, a disease that in many cases causes painful, disfiguring tumors on the skin, and is in some cases deadly,” said Dr. Querfeld. “There is a critical need for a treatment for MF that is efficacious and can be tolerated with long-term dosing, and we believe cobomarsen presents a potential therapeutic option.”

Oral Presentation Details
Title: Cobomarsen in CTCL/MF

Session: CTCL: Conventional and Emerging Drugs
Date: May 9, 2018, 11:30 a.m. CET
Location: Royal Hotel Carlton

Cobomarsen is an inhibitor of microRNA-155. In CTCL, as well as certain other blood cancers, microRNA-155 is present at abnormally high levels and may play a role in the proliferation of blood and lymph cells. miRagen believes therapeutic inhibition of microRNA-155 may reduce aberrant cell proliferation and tumor growth characteristics of certain types of cancer.