Galena Biopharma Inc., a biotechnology company focused on developing innovative, targeted oncology treatments, has initiated the phase III PRESENT trial for NeuVax (E75 peptide plus GM-CSF) vaccine in HER2 1+ and 2+ breast cancer patients (often referred to as HER2 negative) in the adjuvant setting to prevent recurrence.

“Approximately fifty percent of breast cancer patients have tumours that are HER2 1+ or 2+. These patients are successfully rendered disease-free following treatment with current standards of care, and then are sent home to watch and wait with the hope their cancer never returns. Our encouraging findings from the previous phase I/II clinical trial show that NeuVax may provide a significant benefit to maintain disease-free survival in these patients who have no other [adjuvant therapy/treatment] options,” added Beth Mittendorf, MD, assistant professor, Department of Surgical Oncology, University of Texas MD Anderson Cancer Centre and principal investigator for the present phase III study.

The PRESENT (Prevention of Recurrence in Early-Stage, Node-Positive Breast Cancer with Low to Intermediate HER2 Expression with NeuVax Treatment) study is a randomized, multi-centre, multinational clinical trial that will enroll approximately 700 breast cancer patients. The trial design has been updated to include current National Comprehensive Cancer Network guidelines and recently received Special Protocol Assessment (SPA) concurrence from the US Food and Drug Administration (FDA). Based on a successful phase II trial, which achieved its primary endpoint of disease-free survival (DFS), the FDA has agreed that the design and planned analysis of the phase III study adequately address the objectives necessary to support an acceptable regulatory submission for marketing approval.

“NeuVax addresses a critical unmet medical need, well-recognized by investigators, and the interest from potential patients is very high,” said Rosemary Mazanet, MD, PhD, chief medical officer. “We are grateful to our investigators and our clinical trial sites for their ongoing feedback and support in reaching this crucial first step of enrolling patients ahead of schedule.”

The NeuVax phase III trial will be conducted in adjuvant breast cancer patients who are node positive, have an HLA status of A2/A3+, and have low or intermediate HER2 expression (IHC 1+, 2+, sometimes referred to as HER2 negative). These patients are not eligible to receive Herceptin (trastuzumab, marketed by Roche-Genentech) therapy that is currently approved only for patients with high HER2, or 3+ expression.

Once qualified patients have achieved a complete response from current standard-of-care treatment (surgery, radiation and/or chemotherapy), they will be randomized and dosed with either NeuVax or control (placebo plus GM-CSF). Patients will receive one intradermal injection every month for six months, followed by a booster inoculation every six months thereafter. The primary endpoint is disease-free survival at three years or 139 events (recurrence of cancer). A data safety monitoring board will conduct an interim analysis for safety and futility after 70 events. Galena is currently on track for the initiation of approximately 100 investigator sites in the U.S. and abroad.

NeuVax consists of the E75 peptide derived from human epidermal growth factor receptor 2 (HER2) combined with the immune adjuvant granulocyte macrophage colony-stimulating factor (GM-CSF). Treatment with NeuVax stimulates cytotoxic (CD8+) T cells in a highly specific manner to target cells expressing any level of HER2.

According to the National Cancer Institute, over 200,000 women in the US are diagnosed with breast cancer annually. Of these women, about 75 percent test positive for HER2 (IHC 1+, 2+ or 3+). Only 25 percent of all breast cancer patients, those with HER2 3+ disease, are eligible for Herceptin (trastuzumab; marketed by Roche-Genentech), which had revenues of over $5 billion in 2010. NeuVax targets the remaining 50 percent of HER2-positive patients (HER2 1+ and 2+) who achieve remission with current standard of care, but have no available HER2-targeted adjuvant treatment options to maintain their disease-free status.

Galena Biopharma, Inc. develops innovative, targeted oncology treatments that address major unmet medical needs to advance cancer care.