Galena doses first patient in Phase II trial of GALE-401 to treat MPN-related thrombocytosis

The company would pursue approval via the 505(b)(2) regulatory pathway, based on discussions with the US Food and Drug Administration (FDA) and pending a successful development program.

The open-label, single-arm, multicenter Phase II trial of GALE-401 will enroll 20 patients with MPN-related thrombocytosis.

The trial is designed to confirm the platelet-lowering activity of GALE-401 in patients with MPNs, to evaluate safety and tolerability as well as to measure blood levels of the drug.

The primary efficacy endpoint of the trial will be the proportion of subjects who achieve a complete or partial platelet response for at least four weeks during the first six months of treatment.

Galena Biopharma president and chief executive officer Mark Schwartz said moving GALE-401 into the clinic is an important advancement in the treatment of MPN diseases, as well as for the company’s overall product pipeline.

"The initiation of this Phase 2 trial officially signals Galena’s expansion into hematology following our acquisition of the compound earlier this year," Schwartz said.

"With GALE-401, we hope to offer an alternative treatment option for patients suffering from high platelet counts resulting from MPN diseases."

The company intends to report top line data from the Phase II trial of GALE-401 in 2015.

The existing immediate release formulation (anagrelide IR) is approved by the FDA to treat high platelet counts in patients with myeloproliferative neoplasms (MPNs), including polycythemia vera (PV), chronic myelogenous leukemia (CML), primary myelofibrosis (PMF) and essential thrombocythemia (ET).

Image: Galena’s Phase II trial of GALE-401 will enroll 20 patients with MPN-related thrombocytosis. Photo: courtesy of Michelle Meiklejohn/ freedigitalphotos.net