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May 13, 2013

Agenus released preliminary results from a phase II trial of Prophage G-100 (HSPPC-96) for glioblastoma multiforme (GBM). This single-arm study enrolled 46 patients with newly diagnosed GBM. Subjects received the HSPPC-96 vaccination, as well as radiation and temozolomide as the standard of care. Results showed patients treated with the HSPPC-96 arm showed a 146% increase in progression-free survival (PFS) over standard of care alone (17 months versus 6.9 months, respectively) and a 60% increase in overall survival (OS) over standard of care alone (23.3 months versus 14.6 months, respectively). In addition, 32 patients treated at UCSF underwent testing for expression of B7-H1 in blood samples taken prior to surgery, which showed patients with low expression of B7-H1 (53%) had better PFS (21.6 months) than those with high B7-H1 (47%) expression (11.4 months). This finding may have the potential to help identify a more responsive patient population for future trials.

April 22, 2013

Activartis reported preliminary results from a phase II trial of AV0113 for the treatment of glioblastoma multiforme (GBM). This multi-center, randomized, placebo-controlled study had enrolled 100 subjects with GBM at the time of analysis. Subjects received standard first-line therapy, plus AV0113 DC-CIT inoculated into inguinal lymph nodes: after six weeks of chemo and radiotherapy, four weekly applications; six more applications every four weeks; and then one boost immunization every three months. Preliminary results revealed a very promising trend suggesting an overall survival benefit of patients in the AV0113 treatment group compared to the randomized control group. At 12 months, 64% of patients in the treatment group and 48% of patients in the control group were still alive. At 18 months, 50% of patients in the treatment and 33% of patients in the control group were still alive. Patients receiving AV0113 cancer immunotherapy tended to experience signs of relapse earlier compared to control patients. AV0113-triggered inflammation in the tumor tissue may explain this observation, which was also made in other clinical trials studying cancer immunotherapy. AV0113 treatment was well tolerated. The most frequent adverse events were local swelling, redness and tenderness at the injection site. Activartis will continue the phase II trial to confirm the observed trend.

March 12, 2012

Apogenix GmbH released initial results from a phase II trial of APG101 for the second line treatment of Glioblastoma Multiforme. This open-label, randomized, controlled study enrolled 83 subjects with first or second relapse. The subjects received APG101 plus radiotherapy versus radiotherapy alone. The primary objective of the trial was to increase the percentage of subjects reaching a six month rate of progression free survival (PFS6) by 100%. This objective was reached. During treatment with APG101 for up to two years, no drug-related adverse effects were observed.

November 7, 2011

YM Biosciences reported preliminary results from a phase II trial of nimotuzumab for pediatrics with recurrent diffuse intrinsic pontine glioma. This open-label, single-arm, multi-center study enrolled 46 subjects aged three to 18 years of age. The subjects received nimotuzumab 150 mg/m2 administered intravenously once weekly from week one to seven and once every two weeks from week eight to 18. Treatment with nimotuzumab was well tolerated with most adverse events reported as mild or moderate in severity. No complete responses (CR) were observed. At week eight, a partial response (PR) was reported in two subjects, stable disease (SD) in six subjects and progressive disease (PD) in 11 subjects who were evaluable for response, resulting in a clinical benefit rate (CR+PR+SD) of 18.2%. At week 18, one subject continued to have a PR and three subjects continued with SD, for an overall response rate of 2.3%. The median duration of response, time to progression, and overall survival were 2.1 months, 1.7 months and 3.2 months respectively.

September 19, 2011

Immunocellular Therapeutics reported long term results from a phase I trial of ICT-107, their cancer vaccine candidate for the treatment of glioblastoma multiforme. The study enrolled 16 subjects with newly diagnosed glioblastoma who received three injections of ICT-107 in addition to standard treatment with surgery, radiation and chemotherapy. Three-year overall survival is 55% compared to 16% for historical standard of care (SOC). The data show that 38% of subjects who received ICT-107 continue to show no tumor recurrence after three years, compared to the historic disease-free survival rate of 6% with SOC. Out of these subjects, 19% remain disease-free after more than four years. The three-year median overall survival (OS) for subjects receiving ICT-107 is currently 38.4 months, and median progression-free survival (PFS) is 17 months. This historical median OS is 14.6 months and PFS is 6.9 months with SOC.

November 29, 2010

Celldex issued results from a phase II trial of rindopepimut, an immunotherapeutic vaccine under investigation for glioblastoma multiforme (GBM). This open label trial, ACT III, enrolled 65 subjects with newly-diagnosed EGFRvIII-expressing GBM who had undergone surgical resection followed by radiation therapy, temozolomide (TMZ) without tumor progression. Rindopepimut mixed with granulocyte-macrophage colony stimulating factor (GM-CSF) (~150 mcg) was administered intradermally bi-weekly for three doses prior to starting maintenance TMZ and monthly thereafter on day 21 of each TMZ cycle until disease progression. The results for the predefined primary endpoint (66% Progression Free Rate at approximately 8.5 months post-diagnosis) show a statistically significant improvement (p≡0.0168) over a predetermined estimate of 53%, which is beyond the range of expected progression-free survival for treatment with standard of care, consisting of radiation plus TMZ. In addition, 82% of the evaluable population developed a specific anti-EGFRvIII antibody response that was maintained at a significant level. Vaccination with rindopepimut plus TMZ was well tolerated.

September 13, 2010

Immunocellular Therapeutics reported positive long-term results from a phase I trial evaluating ICT-107, their dendritic cell-based vaccine for brain cancer. This trial enrolled 16 subjects with newly diagnosed glioblastoma multiforme who received three injections of ICT-107 in addition to standard treatment with surgery, radiation and chemotherapy. The data showed six out of the 16 subjects (37.6%) were disease-free after more than two years, with three of these subjects (18.8%) remaining disease-free for more than three years. One subject remains disease-free after almost four years. The historical two-year progression-free survival rate is approximately 11%, and the three-year progression-free survival rate is less than 5%. The median progression-free survival was 17.7 months compared to the historic median of 6.9 months. Eleven of the 16 subjects were still alive. No treatment-related serious adverse events have been observed to date.

November 9, 2009

YM BioSciences released results from a phase III trial of nimotuzumab for the treatment of diffuse intrinsic pontine glioma (DIPG). This open label, single arm study enrolled 42 pediatric subjects with inoperable DIPG, across sites in Germany, Italy, Belarus and Russia. The subjects received nimotuzumab in combination with radiotherapy. The primary endpoint was the probability of progression-free survival (PFS) at six months post-diagnosis or median PFS over six months. After 24 weeks, partial response and stable disease was reported in 76% of the subjects and the median survival of all subjects was 9.6 months, with responders having a median survival of 11.4 months. After one year of treatment, 14 of the 41 evaluable subjects (34%) were reported alive compared to a historical rate of 39.9% (± 4.3%) reported for aggressive chemotherapy and radiotherapy. Based on the results, YM BioSciences plans to pursue approval in the European Union.

October 26, 2009

NorthWest Biotherapeutics reported positive long-term follow-up from phase I/II trials of DCVax-Brain, a dendritic cell immunotherapy vaccine for Glioblastoma multiforme. Data are from 20 subjects who were administered three injections of DCVax-Brain intradermally two weeks apart. All subjects were also treated with current standard of care (surgical removal of the initial tumor, plus radiation and chemotherapy). At this time, 1 subject treated with DCVax-Brain in addition to standard of care had died. This subject had survived for approximately 7 years (80.5 months). Overall, 85% of the subjects treated with DCVax-Brain have lived longer than the median survival with the current standard of care treatment (14.6 months versus 36.4 months; p≡0.0004). In addition, 22% of the subjects treated with DCVax-Brain have reached or exceeded the 6-year survival mark. With standard of care treatment, less than 5% of patients with GBM are still alive at 5 years. Disease progression was also markedly improved with DCVax-Brain. With standard of care treatment alone, GBM brain tumors typically recur (progress) in 6.9 months while the median time to disease progression in the subjects treated with DCVax-Brain is 26.4 months (p≡0.00001). In the subjects treated with DCVax-Brain plus standard of care, 74% have been free of recurrence for 1 year, 45% recurrence-free for 2 years, 33% for 3 years, 28% for 4 years and 22% recurrence-free for 5 years.

May 25, 2009

Exelixis issued positive interim results from a phase II trial of XL184 for the treatment of glioblastoma multiforme. This open-label, uncontrolled trial planned to enroll 46 subjects who were to receive daily XL-184 (25 or 100 mg gelatin capsules). The primary endpoint was the 6-month progression-free survival rate. Data are from 26 subjects who were assessed at four weeks after treatment. Ten of the subjects (38%) had tumor shrinkage of at least 50 percent, including one who had a 100 percent reduction in tumor size. Nine subjects had tumor measurement changes ranging from plus 24% to minus 49% and seven had at least a 25 percent increase in tumor burden. Of 17 subjects who had not received prior anti-angiogenic treatment, nine had at least a 50 percent reduction in tumor burden.

November 10, 2008

YM Biosciences released positive results from two phase II trials of nimotuzumab for the treatment of brain metastases and brain cancer. The first trial was a randomized, open, controlled trial in 30 subjects diagnosed with advanced non-small cell lung cancer (NSCLC) and unresectable brain metastases. The subjects received nimotuzumab (200 mg administered as weekly intravenous infusions over weeks 1-6) plus palliative radiation (40 Gy in four weeks) or palliative radiation alone. The primary endpoint was disease control rate (DCR- Complete Response + Partial Response + Stable Disease). Data are from the first 21 subjects. DCR was 91.6% for the nimotuzumab plus radiation arm compared to 44.4% for the radiation alone arm. Subjects treated with the combination had a median survival of 7 months compared to the control group for whom the median survival was 2.47 months (p= 0.0039). The second trial was a randomized, double blind phase II/III trial in 80 subjects with newly diagnosed high-grade glioma. Following biopsy, partial or total resection of the tumor, the subjects received six weekly infusions of placebo or nimotuzumab (200 mg) while also receiving radiotherapy, followed by a maintenance dose of nimotuzumab or placebo every 21 days. The treatment duration was one year. Results are from the first 65 enrolled subjects. The median survival time for the subjects treated with nimotuzumab plus radiotherapy was 16.43 months, while the median survival time for the placebo arm was 10.49 months. Nimotuzumab was well tolerated in both studies.

November 26, 2007

Genentech reported positive results from a phase II trial of Avastin for the treatment of glioblastoma multiforme (GBM). This open-label, multicenter, randomized, non-comparative study enrolled 167 subjects with GBM whose cancer had relapsed after first- or second-line therapy, all of whom had received prior temozolimide. The subjects were randomized to receive Avastin alone or in combination with irinotecan every other week for up to 104 weeks. The primary endpoints were six-month progression free survival (PFS) and objective response rate. The PFS rates were 36% and 51% in the Avastin-alone and Avastin plus chemotherapy arms, respectively. Preliminary tumor responses were observed in 21% of the Avastin alone arm and in 34% of the Avastin plus chemotherapy arm. Treatment was generally well tolerated. Based on the data Genentech plans to meet with the FDA in order to determine the next step of action towards regulatory filing.

Keryx released positive interim results from a phase II trial of perifosine for the treatment of recurrent malignant glioma. This trial enrolled twenty-five subjects who had failed prior radiation therapy and had evidence of tumor progression. The subjects were treated with a loading dose of 600 mg (150mg x 4) followed by 100 mg daily dose of perifosine. Response was measured by the MacDonald Criteria (partial response greater than or equal to 50% decrease in bidirectional tumor area and stable disease = between 25% worse to 50% better). Out of twenty evaluable subjects, partial response was observed in two, stable disease was observed in four and overall response (partial + stable) was reported in six subjects. The median progression free survival and overall survival was nine weeks and forty-nine weeks, respectively. Treatment was generally well tolerated. Subjects with anaplastic gliomas showed the greatest response, hence the trial planned to continue enrolling only this population.

May 1, 2006

NeoPharm announced positive results of a phase I trial of cintredekin besudotox (IL13-PE38QQR), for the treatment of malignant glioma, at the 74th Annual Meeting of the American Association of Neurological Surgeons in San Francisco. This open-label study enrolled 19 patients with treatment-naïve malignant glioma, who received single 96-hour infusions of the drug (0.25 mcg/ml or 0.5 mcg/ml) via intraparenchymal catheter, followed by standard fractionated radiation therapy (5940-6100 cGy, 5 days per week for 6-7 weeks) with or without temozolomide (75 mg/m2 per day during radiation). Trial data yielded a positive safety profile for the drug: no dose limiting toxicities (DLT) were observed with either combination using the lower dose; 1 DLT was observed at the higher dose in combination with radiotherapy alone, but 5 additional subjects had received cintredekin plus radiotherapy and 3 had received cintredekin plus radiotherapy & temozolomide without incidence of DLT.

SuperGen has reported negative interim results of a phase II trial of Orathecin for the treatment of first-line pancreatic cancer. The trial had enrolled 39 subjects, who received 1000 mg/m2 gemcitabine every week for three out of four weeks with concurrent 1.5 mg/m2 Orathecin daily for five of seven days each week; treatment cycles were repeated every four weeks until evidence of disease progression was noted or patient withdrawal. Trial data failed to meet the pre-specified threshold for median survival, with median duration of 6.0 months and a 1 year survival rate of 27%. These results were insufficient to warrant phase III studies of the drug under the current development program; SuperGen announced plans to review the Orathecin development effort.

January 9, 2006

Allos Therapeutics announced positive results of a phase III study, dubbed REACH, of Efaproxyn (efaproxiral) for the treatment of brain metastases. Trial data indicated that the drug significantly decreased risk of death by 25% in combination with whole-brain radiation therapy (WBRT) and supplemental oxygen (SO), compared to WBRT plus SO alone (p=0.01). Subpopulation analysis of patients with brain metastases due to non-small cell lung cancer or breast cancer indicated a significant improvement in response rate (13%; p=0.01), and a trend towards improvement in median survival time in (38% improvement; p=0.07). Treatment with the drug was generally well tolerated, with most adverse events being mild to moderate (grade 1 or 2) in severity. This randomized, open-label study enrolled 538 patients, who received a regimen of WBRT plus SO alone (n=267), or in combination with Efaproxyn (n=271).

January 2, 2006

Pharmacyclics announced top-line results of a phase III trial of Xcytrin (motexafin), for the treatment of brain metastases in subjects with non-small cell lung cancer (NSCLC). Trial data failed to demonstrate significant improvement in time to neurological progression (15.4 months vs. 10.0 months for placebo; p=0.122). Further, no improvements were noted in overall survival or several other secondary endpoints, though reduction in steroid use trended positively. North American subjects did yield significant improvements (24.2 months vs. 8.8 months; p=0.004). This randomized, controlled study enrolled 554 patients across 94 site in North America, Europe and Australia.

November 29, 2004

Alteris Therapeutics has announced positive results of a phase I clinical trial of their EGFRvIII peptide therapeutic vaccine, for the treatment of malignant glioma. Results from the trial met their primary safety and tolerability endpoints, with no serious adverse events reported and a favorable tolerability profile. Furthermore, the drug demonstrated significant preliminary evidence of efficacy: 2 patients experienced near-complete remission after vaccination, more than 25% of subjects experienced stable disease state during the study, median overall time to disease progression was 314 days, compared to a historical baseline of I24 days, and median survival time was over 596 days, or about 20 months, compared to a historical baseline of 11-13 months. This open-label safety and efficacy trial enrolled a total of 16 patients with malignant glioma at 1 US site, who received a total of three doses of the trial once every two weeks.

Hybridon has issued positive interim results of a phase I trial of IMOxine, their investigational second-generation immunomodulatory oligonucleotide for the treatment of solid tumors. Results from the 19 subjects completing safety evaluations met primary safety and tolerability endpoints, with no dose-limiting toxicity, a tolerability profile consistent with immune system stimulation, and a manageable adverse event profile including transient hypoxia/dyspnea (n=1), abdominal pain with nausea/vomiting (n=1), and anemia requiring transfusion (n=2). There were 4 early withdrawals, all due to disease progression. Furthermore, results from the 17 subjects completing preliminary efficacy evaluations indicated that the drug produced stable disease state in 53% of patients (n=9) after 8 weeks of treatment, and 1 of these subjects maintained this state into the 11th month of treatment. This open-label, open-duration safety and immunpharmacology study enrolled 23 patients with assorted refractory solid tumors at 1 US site. Subjects received IMOxine via weekly subcutaneous injection at one of 5 dosing regimens (0.04, 0.16, 0.32, 0.48, or 0.64 mg/kg).

Pharmacyclics announced the combined results of two phase I trials of Xcytrin (motexafin gadolinium) in combination with cranial irradiation for the treatment of glioblastoma multiforme (GBM). Data from both trials indicated that the duration-ranging regimens met primary safety and tolerability endpoints, with no drug related interruptions of radiation therapy and a manageable adverse event profile which included numbness, tingling and rash of fingertips, nausea and mild diarrhea, and reversible hepatic chemistry abnormalities. Preliminary evidence indicates that the drug promoted improvements in survival. Specifically, patients receiving a cumulative dose >60 mg/kg of Xcytrin experienced a median survival time of 11.5 months, with 82% alive at 6 months and 46% at 12. Patients a cumulative dose <60mg/kg experienced median survival of 16.4 months, with 93% and 78% alive at 6 and 12 months. Overall median survival for the entire group of patients was 14.7 months, with 91% and 69% alive at 6 and 12 months. Both trials were open-label, cumulative-dose-escalation studies (40-117 mg/kg), which enrolled a combined total of 55 subjects with treatment-naïve GBM.

May 5, 2003

NeoPharm reported positive preliminary results from an ongoing phase I/II trial investigating IL13-PE38QQR, a tumor-targeting agent for the treatment of brain cancer. Results showed cytotoxic effects against malignant glioma tumor cells at a concentration range of .5-2.0 micrograms/mL when infused directly into the tumor. Data showed survival without tumor progression of three to over 90 weeks. In addition, overall survival from time of treatment was 12 to over 90 weeks. The most frequent adverse events reported were headache, sensory symptoms, motor symptoms, aphasia and seizure. The study was designed in three stages to assess the saftey, tolerability and biologic effect of IL13-PE38QQR in addition to surgical resection of recurrent malignant glioma.