Galena Biopharma has acquired Mills Pharmaceuticals, gaining worldwide rights to GALE-401 (Anagrelide CR), a controlled release formulation of anagrelide. Galena plans to pursue the expedited 505(b)(2) regulatory pathway for approval of GALE-401 for the treatment of Essential Thrombocythemia (ET), as well as orphan drug status. GALE-401 has an estimated market value of approximately $200 million in the U.S., according to the company.
Mills Pharmaceuticals received an upfront payment and is eligible to receive as many as 4,000,000 shares (valued at approx. $28 million) based on certain regulatory milestones, as well as $3 million upon FDA approval.
ET is a bone marrow disease characterized by highly elevated platelet counts, and is associated with vascular complications. Anagrelide immediate release (IR) is one of two generic drugs approved to treat ET. GALE-401 is expected to greatly decrease the adverse events profile relative to the approved product, which includes nausea, diarrhea, abdominal pain, palpitations, tachycardia, and headache.
The Phase I program successfully achieved the desired PK/PD (pharmacokinetic/pharmacodynamic) profile to allow initiation of Phase II studies. The FDA has also indicated that only one Phase III trial is required for approval. The company expects expand the market to treat younger and elderly patient populations with ET based on dosing and tolerability advantages of GALE-401.
"This acquisition is an excellent fit for Galena's focused business strategy, adding another novel product to our pipeline which strengthens the depth and breadth of our hematology-oncology portfolio," said Mark J. Ahn, Ph.D., president and chief executive officer of Galena Biopharma. "ET is a serious condition in which current agents often have very debilitating side effects. We believe GALE-401 can enhance the therapeutic index for ET patients—reducing the side effects of anagrelide while maintaining efficacy for these patients. With established guidance from the FDA on the development process, we are excited to initiate a Phase II study in mid-2014."