On November 14, 2014 ImmunoCellular Therapeutics reported the presentation of updated efficacy data from the Phase II trial of dendritic cell-based immunotherapeutic vaccine ICT-107 at the 19th Annual Scientific Meeting and Education Day of the Society for Neuro-Oncology, being held in Miami, FL (Press release ImmunoCellular Therapeutics, NOV 14, 2014, View Source [SID:1234500964]). Patrick Y. Wen, MD, Director of the Center for Neuro-Oncology at Dana Farber Cancer Institute and Professor of Neurology at Harvard Medical School, and principal investigator on the trial, will present the maturing data set in patients with newly diagnosed glioblastoma multiforme (GBM) in an oral presentation.

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Consistent with prior data presentations in December 2013 and June 2014, the results demonstrate a statistically significant progression-free survival (PFS) benefit, and a numeric overall survival (OS) benefit in ICT-107 treated patients compared to the control group. The ICT-107 treatment effect continues to be strongest in the pre-defined HLA-A2 subgroup of patients in which the MGMT methylated patients showed the largest treatment effect, with a significant PFS advantage over the control group, and continued potential for the OS advantage to move toward significance as more events occur. There were no differences in adverse events between the ICT-107 treated group and the control group.

"ICT-107 continues to hold promise for patients with newly diagnosed glioblastoma, as no other immunotherapy has shown statistical benefit for a clinical outcome in a controlled trial in this patient population," said Dr. Wen. "I think that the data from the phase II trial strongly support advancing to a registrational program."

"With this second update of the original trial results, we remain confident that there is a meaningful treatment benefit in HLA-A2 patients. In the per-protocol population, OS hazard ratios are in the 0.6-0.7 range for all HLA-A2 patients as a group as well as for each of the MGMT subgroups. If our upcoming phase III program generates statistically significant results in this range, ICT-107 could represent a clinically meaningful advance for GBM patients," said Andrew Gengos, ImmunoCellular’s Chief Executive Officer. "The US FDA and three national European regulators have indicated support for phase III testing. We anticipate hearing shortly from the EMA, and then expect to be in position to finalize our phase III design and move into trial execution in 2015."

Updated ICT-107 Phase II OS and PFS Results

As of October 2014, a total of 88 events (patient deaths) had been recorded from the 124 randomized patients, representing 9 additional events since these data from the phase II trial were last updated in June 2014. There were 25 active and 11 control patients alive for a total of 36 patients available for additional follow-up.
Median PFS for the HLA-A2 methylated MGMT per-protocol (PP) population was 24.1 months for the ICT-107 treated group and 8.5 months for control, representing a statistically significant 15.6-month PFS benefit for the ICT-107 treated group (age stratified HR = 0.257 [0.095-0.697], p = 0.004).
Median OS for the HLA-A2 methylated MGMT PP population was 23.9 months for the control group, and the median has not yet been reached for the ICT-107 treated group. At the time of the analysis, 65% of ICT-107 patients and 50% of the control patients were alive (age stratified HR = 0.631 [0.212-1.880], p = 0.404), suggesting the potential for long-term survival with ICT-107 treatment.
Median PFS for the HLA-A2 unmethylated MGMT PP population was 10.5 months for the ICT-107 treated group and 6.0 months for the control group, representing a 4.5-month median PFS benefit for the ICT-107 treated group (age stratified HR = 0.720 [0.351-1.474], p = 0.364).
Median OS for the HLA-A2 unmethylated MGMT PP population, was 15.8 months for ICT-107 patients, and 11.8 months for the control group, representing a 4-month median OS benefit for the ICT-107 treated group (age stratified HR = 0.652 [0.320-1.325], p = 0.233).
Median PFS in the intent-to-treat (ITT) population (all phase II patients) was 11.4 months for the ICT-107 treated group and 10.1 months for the control group, representing a statistically significant benefit in the ICT-107 treated group (age stratified HR = 0.640 [0.423-0.968], p = 0.033).
Median OS in the ITT population was 18.3 months for the ICT-107 treated group and 16.7 for the control group, representing a numeric, but not statistically significant, advantage for the treatment group (age stratified HR = 0.854 [0.547-1.334], p = 0.487).

The Company is utilizing all available information from the controlled phase II trial to design phase III testing in order to increase its probability of success, including the timing of randomization within the standard-of-care treatment these patients receive, in an attempt to limit the number of patients who are "early progressors" and unlikely to respond to therapy.