miRagen Therapeutics has announced new provisional data from its phase 1 clinical trial of cobomarsen in patients with mycosis fungoides (MF). The data were presented in May at “2015 … 2018 T-cell lymphomas: we are close to finalization”, meeting doctor held in Bologna. Additional data obtained on cobomarsen provide further evidence that microRNA therapies have the potential to improve quality of life for patients living with mycosis fungoides. Cobomarsen continues to be safe and generally well tolerated at all doses tested in the Phase 1 study, with more patients receiving more than one year of therapy without serious adverse events attributed to the drug. Furthermore, the improvement in skin disease observed in the study appears to be lasting and is accompanied by improvements in metrics that measure patients’ quality of life. These data support the company’s plans to initiate a phase 2 clinical trial in patients with CTCL in the second half of 2018. Intermediate results presented at the conference include safety observations from long-term administration to 18 patients. The highlights presented are:
- cobomarsen treatment has produced a lasting improvement in quality of life, as measured by the total Skindex-29 score.
- 13 out of 18 subjects showed improvement in the first 100 days with cobomarsen.
- Improvement and stabilization remain durable in four subjects up to one year after treatment and one subject was stable after more than 400 days of cobomarsen.
- Cobomarsen continued to be generally well tolerated at all assessed dose levels.
- More patients received more than one year of therapy (up to 39 grams of cumulative dose) without serious adverse events attributed to cobomarsen.
- 300 and 600 mg intravenous infusions have similar efficacy and tolerability, offering the highest response rate based on mSWAT skin scores.