NeuVax™ (nelipepimut-S)

NeuVax consists of the E75 peptide derived from 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.  NeuVax is given as an intradermal injection once a month for six months, followed by a booster injection once every six months.  Based on a successful Phase II trial, which achieved its primary endpoint of disease free survival (DFS), the Food and Drug Administration (FDA) granted NeuVax a Special Protocol Assessment (SPA) for a Phase III clinical trial in adjuvant therapy of women with low-to-intermediate (also known as HER2-Negative, not eligible for Herceptin) status.

According to the National Cancer Institute, over 230,000 women in the U.S. are diagnosed with breast cancer annually.  Of these women, about 75% test positive for Human Epidermal growth factor Receptor 2 (IHC 1+, 2+ or 3+).  Only 25% of all breast cancer patients, those with HER2 3+ disease are eligible for Herceptin® (trastuzumab; Roche-Genentech) which had revenues of over $5 billion in 2010.  NeuVax targets the remaining 50% of low-to-intermediate (also known as HER2-Negative, not eligible for Herceptin) who achieve remission with current standard of care, but have no available HER2 targeted adjuvant treatment options to maintain their disease free status.

Folate Binding Protein-E39 (FBP)

Folate Binding Protein-E39 (FBP) is a targeted vaccine aimed at preventing the recurrence of ovarian, endometrial, and breast cancers. The FBP vaccine consists of the E39 peptide derived from the folate binding protein combined with the immune adjuvant granulocyte macrophage colony stimulatingfactor (GM-CSF).   FBP is over-expressed (20-80 fold) in more than 90% of ovarian and endometrial cancers, as well as 20–50% of breast, lung, colorectal, and renal cell carcinomas.  FBP has very limited tissue distribution and expression in non-malignant tissue making it an ideal immunotherapy target.

Ovarian cancer occurs in over 22,000 patients per year in the U.S. and is the most lethal gynecologic cancer.  Despite the incidence of ovarian cancer being only approximately 20% that of breast cancer, the number of patients that die from ovarian cancer is nearly 50% that of breast cancer.  Due to the lack of specific symptoms, the majority of ovarian cancer patients are diagnosed at later stages of the disease.  These patients are routinely surgically debulked to minimal residual disease, and then treated with platinum- and/or taxane-based chemotherapy.  While most patients respond to this treatment regime and become clinically free of disease, the majority of these patients will relapse, and once the disease recurs, the treatment options and successes drop dramatically.

Endometrial cancer is the most common gynecologic cancer and occurs in over 46,000 women, with over 8,000 deaths, in the US annually.  There are two basic types of endometrial cancer: endometriod and papillary serous.  The latter has a much more aggressive clinical course and the majority of these patients will die of this form of the disease.