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National Brain Tumor Society Statement on Discontinuation of RINTEGA Clinical Trial For Newly Diagnosed Glioblastoma Patients

2016-03-07

On Monday, March 7, 2016, the biotechnology company Celldex Therapeutics announced that based on a preplanned interim analysis, its phase 3 clinical trial of the investigational immunotherapy RINTEGA (rindopepimut) failed to reach statistical significance for its primary endpoint of overall survival (OS) in newly diagnosed, EGFRvIII-positive glioblastoma patients. As a result of this analysis, Celldex is discontinuing this clinical trial, known as ACT IV.


David F. Arons, JD, Chief Executive Officer of the National Brain Tumor Society, today issued the following statement regarding the discontinuation of the ACT IV study in newly diagnosed, EGFRvIII-positive GBM patients:

National Brain Tumor Society (NBTS) is disappointed to learn that RINTEGA was unable to demonstrate superior effectiveness to current standard of care treatment for newly diagnosed glioblastoma (GBM) patients with EGFRvIII mutations. EGFRvIII mutations are present in nearly a third of all newly diagnosed GBM patients, and this population has been shown to have an especially poor prognosis, making it an area of high-unmet medical need that would benefit greatly from new and more effective treatment options.

NBTS, however, is pleased that Celldex will allow patients from the phase 2 ACT III and phase 3 ACT IV trials, as well as compassionate use recipients, who have been treated with RINTEGA to continue access to the treatment should they and their doctors believe it beneficial.

In regards to the ACT IV data, NBTS believes that it is important to note that the ACT IV data on survival in the RINTEGA arm were consistent with previous encouraging results in earlier phase 2 trials, and the impetus for discontinuing this trial was that the OS of patients in the RINTEGA and the control arms were not significantly different from each other — because the control arm showed an unexpectedly better OS than historical data predicted. Thus, there is still much to learn, particularly when the full ACT IV trial data – now unblinded – is more thoroughly reviewed and evaluated. It is our hope that what is eventually learned from the ACT IV trial will help improve the clinical trial design and process for future immunotherapy trials in brain cancer.

While the ACT IV results are disappointing for the brain tumor community, NBTS commends the sponsors and patients who advance treatment development by their investment and participation in brain tumor clinical trials. Clinical trials are designed specifically for patients and still remain one of the best avenues to receive the most cutting-edge treatments, as well as the sole vehicle for new, safe and effective therapies to reach the market.

Finally, NBTS would like to emphasize that this result does not shake its confidence in the overall promise and potential of immunotherapy as a treatment strategy in neuro-oncology. There are a host of different types of immunotherapeutic agents currently under investigation for the treatment of brain tumors. These not only include vaccines like RINTEGA – which is also still being evaluated in an ongoing phase 2 trial, called ReACT, in combination with the anti-cancer drug Avastin in recurrent GBM patients – but also those in the categories of oncolytic viruses, checkpoint inhibitors, and adoptive cell transfer techniques. As we work together to continue to advance our understanding, we should remain hopeful that any number of these investigational agents will provide more, and better, treatment options for brain tumor patients in the near future.

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