NEW YORK – miRagen Therapeutics said on Thursday that it received guidance from the US Food and Drug Administration on registration-enabling development plans for cobomarsen as a treatment for T-cell leukemia/lymphomas with microRNA-155 overexpression.
Boulder, Colorado-based miRagen said in a statement that the "now defined regulatory pathway for potential approval of cobomarsen for both CTCL and ATLL is an important incremental step" in the broader program strategy to study cobomarsen's efficacy in a variety of miR-155 elevated blood cancers.
"We received clear guidance from the FDA on a potential path to registration in this ultra-rare indication based upon a small randomized controlled trial with a primary endpoint of progression free survival that is appropriate for a maintenance therapy," miRagen President and CEO William Marshall said in a statement.
The company is currently awaiting additional cobomarsen data from the ongoing Phase II SOLAR trial in cutaneous T-cell lymphoma patients, which they expect will be available by the end of the year. After evaluating the activity of cobomarsen in both adult T-cell leukemia/lymphoma and in cutaneous T-cell lymphoma, miRagen will finalize further development plans for the cobomarsen program, which "may include seeking a partner for the late-stage development and potential commercialization of cobomarsen in miR-155 elevated cancers including CTCL and ATLL," Marshall added.
MicroRNA-155 overexpression has been linked to poor prognosis in patients across a variety of hematological malignancies and solid tumors. Cobomarsen is a miR-155 inhibitor that appeared to be active and well-tolerated in a Phase I clinical trial, which included patients with CTCL, ATLL, DLBCL, and CLL. In July, cobomarsen was granted orphan drug designation in the US for T-cell lymphomas such as CTCL and ATLL.