Full Press Release Details
Brain Cancer Vaccine Shows Extended Survival in Phase 2 Final Data
Overall Survival Nearly Double than Expected with Standard of Care
LEXINGTON, Mass.--(BUSINESS WIRE)--July 1, 2014--Agenus Inc. (NASDAQ:
AGEN), announced final results from a single-arm, multi-institutional,
open-label, Phase 2 study showing that patients with newly diagnosed
glioblastoma multiforme (GBM) who received Agenus' Prophage autologous
cancer vaccine added to the standard of care treatment, lived nearly
twice as long as expected. In this Phase 2 study, 50% of the patients
lived for two years, an encouraging result for a cancer that often kills
patients within one year 1-7. Prophage patients demonstrated
a median overall survival of approximately 24 months and 33% of patients
remain alive at 2 years and continue to be followed for survival.
"These data suggest that Prophage is generating an effective immune
response which is translating into an extension in survival far beyond
what is historically seen in patients with GBM. These data provide the
impetus for a definitive, randomized clinical trial," said Andrew Parsa,
MD, PhD,Principal Investigator of the study and the Michael J. Marchese
Professor and Chair of the Department of Neurological Surgery at the
Feinberg School of Medicine at Northwestern University. "Glioblastoma
tumors are often resistant to standard therapies and the extended
progression-free survival and proportion of long-term survivors is very
In addition to the long-term survival data, vaccine treated patients had
a median progression-free survival (PFS) of nearly 18 months,
approximately two to three-times longer than patients treated with
radiation and temozolomide alone1. Importantly, 22% of
patients were alive and without progression at 24 months and continue to
be followed for survival.
Interestingly, the response to Prophage seems to be more pronounced in
those patients with less expression of the checkpoint ligand PDL-1 on
the white blood cells, suggesting that combinations of Prophage with
checkpoint modulators like PD-1 antagonists might make Prophage even
more effective in a greater percentage of patients with GBM.
"We believe that Prophage may play an important role in changing the
treatment paradigm for patients with GBM," said Garo Armen, PhD, CEO and
chairman of Agenus Inc. "We are exploring partnerships for Phase 3
studies of Prophage in GBM. Additionally, we are excited about the
potential combinations of Prophage with PD-1 antagonists and other
checkpoint modulators in GBM."
Prophage is an autologous cancer vaccine, and each patient receives
vaccine prepared from their own surgically resected tumor. As a result,
the vaccine appears to help stimulate the patient's immune system to
attack the tumor based on the spectrum of mutant proteins expressed by
their own tumor. Since most cancers result from an accumulation of
random mutations, which produce different mutant proteins in each
patient, this approach is intended to individually tailor each patient's
vaccine to optimally target the immune attack to that patient's actual
Phase 2 Prophage Study in Newly Diagnosed GBM
single-arm trial of Prophage in patients with newly diagnosed GBM
undergoing gross total resection includes 46 patients treated at eight
centers (UCSF, Columbia, UPENN, Miami, Valley Hospital, Northern
Westchester Hospital, Oklahoma, Johns Hopkins, and Northwestern) across
the US. Patients were treated with surgical resection, radiation and
temozolomide as the standard of care in addition to Prophage
vaccination. The cohort was comparable to patients with surgically
resectable newly diagnosed GBM on prognostic factors such as age,
Karnofsky Performance Score, and MGMT methylation status. Analyses of
data collected to date show more than 50% of the patients were alive at
two years and patients continued to be followed. These results indicate
considerable improvement when compared to expectations for patients
treated with the standard of care (gross total resection plus radiation
and temozolomide), which is 26% of patients alive at 24 months.1
Median overall survival (OS), the primary endpoint of the trial, is 23.8
months and remains durable in patients treated with Prophage. For the
standard of care alone, median OS survival rate is 14.6 months.1 PFS
data remains durable with previous reports with a median PFS of 17.8
months and nearly 22% of patients alive without progression at 24 months.
The Phase 2 recurrent and newly diagnosed trials are being sponsored by
Dr. Parsa and primarily have been supported through funding from the
American Brain Tumor Association, Accelerated Brain Cancer Cure,
National Brain Tumor Society, and National Cancer Institute Special
Programs of Research Excellence. Dr. Parsa has not received any
financial support or expense reimbursement for this work or for
consulting activities on behalf of Agenus. He does not have an equity
interest in Agenus or a financial relationship with the company.
About Glioblastoma Multiforme (GBM)
The incidence rates of
primary malignant brain and central nervous system cancers have
increased over the last three decades.8 The American Cancer
Society estimates that more than 23,000 malignant tumors of the brain or
spinal cord will be diagnosed during 2013 in the US, and that more than
14,000 people will die from these tumors. 9 GBM is the most
common primary malignant brain tumor and accounts for the majority of
diagnoses. It has been associated with a particularly poor prognosis,
with survival rates at one and five years equaling 33.7% and 4.5%,
respectively.10 The current standard of care for patients
with newly diagnosed GBM is surgical resection followed by fractionated
external beam radiotherapy and systemic temozolomide11
resulting in a median OS of 14.6 months12 based on data from
a randomized Phase 3 trial. Although this treatment can prolong
survival, it is not curative and the vast majority of patients with GBM
experience recurrent disease, with a median time to recurrence of seven
months.13 From the time of recurrence, the median survival is
three to nine months.1-7 Current treatment options for
patients with recurrent GBM, include surgery, chemotherapy (i.e.,
CCNU, temozolomide), bevacizumab, and radiotherapy.
About Prophage Series Vaccines
Prophage Series vaccines are
individualized cancer vaccines derived from each patient's own tumor. As
a result of its individualized nature, each Prophage Series vaccine
contains the precise signals (antigenic fingerprint) of the patient's