ImmunoCellular Therapeutics, Ltd. ("ImmunoCellular") (NYSE MKT:IMUC) announced that ICT-107, its dendritic cell-based vaccine, demonstrated a statistically significant increase in progression-free survival (PFS) in patients with newly diagnosed glioblastoma multiforme (GBM) in its randomized, placebo-controlled phase II trial. A comparison of PFS between ICT-107 and placebo showed a statistically significant difference in the Kaplan-Meier (K-M) curves favoring ICT-107>
The differences in the overall survival (OS) K-M curves did not reach statistical significance in the intent-to-treat population (the primary endpoint) or the per-protocol population, with p-values and HRs of>
The OS analysis includes data on 67 events (patient deaths) out of a possible 124, whereas the PFS analysis includes data from 103 events. ImmunoCellular Therapeutics plans to continue following patients in this trial to collect more mature OS data. In the matured data from the open label, phase I trial, the Company observed a consistent benefit in both PFS and OS compared with historical controls, and on this basis thinks that it is possible that the primary OS benefit could be clarified as the phase II data mature.
In this phase II study, ICT-107 was generally safe and well tolerated, with no imbalance of adverse events between the active and placebo groups.
GBM is the most common and aggressive primary cancer of the brain. Patients with this disease have few therapeutic options; temozolomide is currently the only FDA-approved systemic chemotherapy for newly diagnosed GBM.
Patrick Wen, MD, Director of the Center for Neuro-Oncology at The Dana Farber Cancer Institute and Professor of Neurology at Harvard Medical School, and an investigator on this trial, said, "The progression-free survival data look promising in this study. To my knowledge, this is the first time a placebo-controlled immunotherapy trial in glioblastoma has demonstrated a statistically significant improvement in a clinically relevant measure, such as progression-free survival. We await additional data to evaluate the effect on overall survival."
John Yu, MD, Founder, Chairman and Chief Scientific Officer of ImmunoCellular Therapeutics, said, "We are quite pleased to see such a strongly statistically significant result in PFS in this exploratory trial, and believe that in conjunction with the indications of a survival benefit, these results provide a strong medical rationale for continued development of ICT-107 as a potential treatment for glioblastoma. We look forward to discussing this important clinical outcome, and what next steps, including a phase III trial, might entail, with the FDA in an end-of-phase-II meeting."
Andrew Gengos, ImmunoCellular Therapeutics Chief Executive Officer, said, "Although we missed the primary OS endpoint, it is encouraging that the OS and PFS results are consistent and that most of the predefined secondary endpoints in the OS subgroups numerically favor ICT-107 over placebo, although none has reached statistical significance. These phase II results, in conjunction with the phase I results which have indicated the potential for long-term survival, support our view that ICT-107 has a biological and clinically relevant effect in GBM, and potentially may provide a long-term survival benefit. We plan to analyze the results further in the coming weeks and learn more with the goal of informing our next development and regulatory steps. We want to thank the patients who participated in this trial, and our trial site collaborators and dedicated clinical team for their high quality work."
ImmunoCellular anticipates presenting the results of the ICT-107 phase II trial at an upcoming national scientific or medical forum.
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