Galena Biopharma Inc., a biopharmaceutical company developing and commercializing innovative, targeted oncology treatments that address major medical needs across the full spectrum of cancer care, announced the first patient has been dosed in the GALE-401, or Anagrelide Controlled Release (CR), Phase 2 Clinical Trial. The Phase 2 study will treat patients with elevated platelet counts in myeloproliferative neoplasms (MPNs) including essential thrombocythemia (ET). Based on discussions with the U.S. Food and Drug Administration (FDA) and pending a successful development program, Galena would pursue approval via the 505(b)(2) regulatory pathway.
The Phase 2 trial is an open-label, single-arm, multicenter study of GALE-401 in 20 patients with MPN-related thrombocytosis. The goals of the study are to confirm the platelet-lowering activity of GALE-401 in patients with MPNs, to assess safety and tolerability, and to measure blood levels of the drug. The primary efficacy endpoint 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.
“Moving GALE-401 into the clinic is an important advancement in the treatment of MPN diseases, as well as for Galena’s overall product pipeline,” said Mark Schwartz, president and chief executive officer. “The initiation of this Phase 2 trial officially signals Galena’s expansion into hematology following our acquisition of the compound earlier this year. With GALE-401, we hope to offer an alternative treatment option for patients suffering from high platelet counts resulting from MPN diseases. I am proud of our clinical development team’s effort to advance this program and we look forward to top line data from the study in 2015.”
MPNs encompass a family of diseases including essential thrombocythemia, polycythemia vera, primary myelofibrosis, and chronic myelogenous leukemia. The active ingredient immediate release version of anagrelide has been shown to reduce excessive platelet counts and has been approved by the FDA for treating high platelet counts in patients with MPNs. Anagrelide CR is Galena’s new formulation of anagrelide that releases the active ingredient more slowly over time than currently marketed versions of this drug, and is therefore absorbed more slowly into the bloodstream. This slower release of drug could help to reduce adverse events that might be caused by high blood concentrations when the drug is absorbed rapidly, as it is with currently marketed products.
“While current treatment options for high platelet counts are effective, they sometimes cause significant side-effects that may prevent patients from taking an adequate dose of their medications. I look forward to working with anagrelide CR to explore this formulation to slowly release the drug into the patient’s system and potentially provide better outcomes,” added Srdan Verstovsek, professor, department of leukemia, division of cancer medicine, The University of Texas MD Anderson Cancer Center, and principal investigator for the Phase 2 trial.
Date: September 9, 2014
Source: Galena Biopharma