Miragen Therapeutics (MGEN) To Present At Oppenheimer 29th Annual Healthcare Conference - Slideshow

Cobomarsen (miR-155 inhibitor) in Hematological Malignancies - Cobomarsen achieved human Phase 1 clinical proof-of-concept in Cutaneous T-Cell Lymphoma (CTCL)[6] - A controlled Phase 2 clinical trial in CTCL was recently initiated, with data potentially allowing for accelerated approval[6] - Promising clinical results were obtained in the treatment of Adult T-cell Leukemia/Lymphoma (ATLL)[6] - In a Phase 1 trial, 92% of MF subjects in the systemic administration cohorts showed improvement in tumor burden as assessed by mSWAT score[21] - 63% of subjects treated with cobomarsen at a 300 mg IV-infusion achieved a partial response (PR)[21,26] - The estimated potential worldwide market opportunity for Cobomarsen in CTCL is over $1 billion[18] Remlarsen (miR-29 replacement) in Pathological Fibrosis - Mechanistic proof-of-concept in dermal scarring was obtained from a Phase 1 clinical trial[6] - A Phase 2 clinical trial in pathological cutaneous fibrosis is expected to have a readout in the second half of 2019[6] - Blinded histology analysis showed a statistically significant (p=00086) reduction of fibroplasia in a human Phase 1 clinical trial[56,58] MRG-110 (miR-92 inhibitor) in Cardiovascular Disease - Two Phase 1 clinical trials for systemic and local administration are expected to have readouts in 2019[6] - Development is funded by Servier, with miRagen retaining commercial rights in the United States and Japan[6]