Puma Biotechnology, Inc. (NYSE: PBYI), a development stage biopharmaceutical company, today announced that it has reached agreement with the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the planned Phase III clinical trial of the Company's lead drug candidate PB272 (neratinib) in patients with HER2-positive metastatic breast cancer who have failed two or more prior treatments (third-line disease). The SPA is a written agreement between the Company, as the trial's sponsor, and the FDA regarding the design, endpoints and planned statistical analysis approach of the Phase III trial to be used in support of a New Drug Application (NDA) for PB272. The European Medicines Agency (EMA) has also provided follow-on scientific advice (SA) consistent with that of the FDA regarding the Company's Phase III trial design and endpoints to be used and the ability of such design to support the submission of a European Union (EU) Market Authorization Application (MAA).
Pursuant to the SPA and SA, the Phase III trial will be a randomized trial of PB272 plus Xeloda versus Tykerb plus Xeloda in patients with third-line HER2-positive metastatic breast cancer. The trial is expected to enroll approximately 600 patients who will be randomized (1:1) to receive either PB272 plus Xeloda or Tykerb plus Xeloda. The trial will be conducted at approximately 150 sites in North America, Europe and Asia-Pacific. The agreed upon co-primary endpoints of the trial are progression-free survival and overall survival. The Company plans to use the progression-free survival data from the trial as the basis for submission of an NDA/MAA for Accelerated/Conditional Approval for PB272 from the regulatory agencies. Puma anticipates that it will begin patient enrollment in this Phase III trial in March or April of this year.
Alan H. Auerbach, Chief Executive Officer and President of Puma Biotechnology, said, "Obtaining FDA and EMA agreement on the overall Phase III trial design, and more specifically patient population and primary endpoints, represents an important milestone in the global development of PB272 and for Puma as a company. We look forward to initiating patient enrollment in the Phase III trial shortly."