Rindopepimut shows impressive mid-stage results for brain cancer

The US biotech company Celldex announced that its investigational vaccine for glioblastoma, the most common type of brain cancer, met the main goal of extending survival time for patients without a progression of the disease in a phase II clinical study. Now the company is pushing forward into large-scale late-stage trials.  An international, placebo-controlled phase III trial will be initiated in the second half of 2011, according to the statement of the company.

In a phase II study, the company’s injectable vaccine Rindopepimut (CDX-110) showed impressive results with 66 percent of patients were progression-free at 8.5 months from diagnosis or 5.5 months from start of vaccination.

The data, consistent with previous studies, represent a statistically significant increase over a predetermined progression-free rate estimate of 53 percent.  The results are very encouraging for Celldex as the current standard of treatment (consisting of radiation and temozolomide) showed between 29% and 45% of patients progression-free at 8.5 months post-diagnosis.

“These data suggest that rindopepimut is extending survival well beyond what we have seen historically in this patient population,” said Rose Lai, M.D., assistant professor of neurology, Columbia University Medical Center, and lead investigator on the ACT III study. “The consistency of data from three separate studies is impressive and clearly supports the plan to conduct a controlled pivotal study in GBM.”

The immunotherapeutic vaccine Rindopepimut, developed by Celldex, targets epidermal growth factor receptor variant III (EGFRvIII),  a mutation leading to EGFR overexpression associated with glioblastoma multiforme.

Celldex had suffered a setback earlier this year when its partner Pfizer pulled out from a co-development deal, saying the vaccine no longer constituted a strategic priority to them. Celldex now holds all global rights for the development and commercialization of Rindopepimut.

The immunotherapeutic vaccine Rindopepimut is similar to Dendreon’s Provenge, a vaccine approved by FDA recently and indicated to treat advanced prostate cancer in men.