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.