Patients with newly diagnosed glioblastoma do not experience any prolongation in survival if treated with rindopepimut, according to a study published in The Lancet Oncology.1

In a randomized phase 3 ACT IV trial ( Identifier: NCT01480479), 405 patients with minimal residue disease (MRD), 338 patients with significant residual disease (SRD), and 2 unevaluable patients, were assigned to receive temozolomide 150 mg/m2  to 200 mg/m2 with rindopepimut 500μg or with control (100μg keyhole limpet haemocyanin).

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At the preplanned interim analysis, the study was discontinued for futility.

No differences were observed for overall survival (OS) at time of final analysis for patients with MRD; median OS was 20.1 months (95% CI, 18.5-22.1) in the study group compared with 20.0 months (95% CI, 18.1-21.9) in the control group.

Nine patients in the rindopepimut group and 7 patients in the control group died due to adverse events (AE), of which only 1 was potentially associated with rindopepimut.

The most frequently reported grade 3 to 4 AEs in the rindopepimut arm and the control group, respectively, were thrombocytopenia (9% vs 6%), fatigue (2% vs 5%), brain edema (2% vs 3%), seizure (2% vs 2%), and headache (2% vs 3%). Reported serious AEs include seizure (5% vs 6%), and brain edema (2% vs 3%).

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Despite the results, authors concluded by saying “combination approaches potentially including rindopepimut might be required to show efficacy of immunotherapy in glioblastoma.”


  1. Weller M, Butowski N, Tran DD, et al. Rindopepimut with temozolomide for patients with newly diagnosed, EGFRvIII-expressing glioblastoma (ACT IV): a randomised, double-blind, international phase 3 trial. Lancet Oncol. 2017 Aug 22. doi: 10.1016/S1470-2045(17)30517-X