Celldex
Therapeutics is developing
rindopepimut (CDX-110), a 14-mer
injectable peptide vaccine for the potential treatment of
glioblastoma multiforme (GBM).
Rindopepimut specifically targets a novel junctional
epitope of the EGFR deletion mutant
EGFRvIII, which is a constitutively active receptor that is expressed in approximately 60 to 70% of patients with GBM.
EGFRvIII expression is correlated with worse prognosis and reduced overall survival. Importantly,
EGFRvIII is not expressed in normal brain tissue, making it an excellent therapeutic target. Preclinical studies demonstrated lasting
tumor regression and increased survival times, as well as efficient generation of
EGFRvIII-specific humoral and cellular immune responses, in animals expressing
EGFRvIII and vaccinated with
rindopepimut. Phase I and II clinical trials in patients with GBM demonstrated significantly increased median time to progression and overall survival time in those vaccinated with
rindopepimut compared with matched historical controls. Only limited side effects have been observed in patients. Given these results,
rindopepimut is an extremely promising
therapy for patients with GBM. Phase I and II clinical trials in patients with GBM were ongoing at the time of publication. In the future, it may be beneficial to combine
rindopepimut with other treatment modalities to further prolong survival.