Vaccine: Rindopepimut
Company: Celldex Therapeutics
Target: Brain cancer

Celldex Therapeutics' ($CLDX) brain cancer vaccine, rindopepimut, also known as CDX-110, targets EGFRvIII, an activated mutation of the epidermal growth factor receptor (EGFR). This mutation is found in about 31% of cases of glioblastoma multiforme, a form of fast-growing brain cancer and the most common type of primary brain tumor. It can contribute to tumor growth, and is linked with poor long-term survival. It is not seen in normal tissue.

In the ACT III Phase II trial, which involved people with newly diagnosed EGFRvIII-positive glioblastoma, 65 patients were given rindopepimut in combination with standard-of-care treatment (temozolomide), after having undergone surgery and standard chemotherapy and radiation therapy.

Rindopepimut improved survival, with a median survival of 24.6 months, compared with 15.2 months for matched patients receiving standard treatment in published studies. Overall survival at 24 months was 52%, compared with 6% in the historical group. The vaccine was well-tolerated and the patients had robust immune responses.

In September 2010, Pfizer ($PFE) returned its rights to the vaccine because it was not a strategic priority. Rindopepimut is in a Phase III registration trial called ACT IV for the first-line treatment of glioblastoma, with trials under way in the U.S. and planned in additional centers elsewhere. The vaccine is also under assessment in a Phase II trial in the U.S. in patients with recurrent glioblastoma (the ReACT trial).