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Genetic Engineering & Biotechnology News | GENengnews.com | AUGUST 2014 | 37 Clinical Trials Cancer > Agenus reported results from a sin- gle-arm, multi-institutional, open-label, Phase II study showing that patients with newly diagnosed glioblastoma multi- forme (GBM) who received its Prophage autologous cancer vaccine added to the standard-of-care treatment, lived nearly twice as long as expected. In the study, 50% of the patients lived for two years. Prophage patients demonstrated a me- dian overall survival (OS) of approximately 24 months, and 33% of patients remained alive at two years and continued to be fol- lowed for survival. Patients treated with the vaccine also had a median progression-free survival (PFS) of nearly 18 months; Agenus said this is around two to three times longer than patients treated with radiation and temozolomide alone. Also, 22% of pa- tients were alive and without progression at 24 months. Each patient received vaccine tailor- made for them from their own surgically resected tumor. The vaccine works by stimulating the patient's immune system to attack the tumor based on the spec- trum of mutant proteins their own tumor expresses. According to the company, the response to Prophage seems to be more pronounced in patients with less expres- sion of the checkpoint ligand PDL-1 on the white blood cells, suggesting that combi- nations of Prophage with checkpoint mod- ulators such as PD-1 antagonists may make Prophage even more efective in a greater percentage of patients with GBM. Median OS, the primary endpoint of the trial, is 23.8 months and remains du- rable in patients treated with Prophage. For the standard of care alone, median OS survival rate is 14.6 months. Median PFS, consistent with previous reports, is 17.8 months and remains durable. Nearly 22% of patients were alive without progression at 24 months. > Boehringer Ingelheim presented results from a Phase II study that enrolled patients with previously untreated acute myeloid leukemia (AML) aged 65 or older and ineligible for intensive remission in- duction therapy. The patients lived longer when treated with volasertib combined with low-dose cytarabine (LDAC). The overall survival data showed that volaser- tib, when used in combination with LDAC, increased the percentage of older AML patients who achieved remission. Patients treated with volasertib com- bined with LDAC had a median overall survival of 8 months versus 5.2 months in patients treated with LDAC alone. The response rate was more than doubled for patients receiving volasertib and LDAC versus LDAC alone (31% vs. 13.3%). Volasertib is an investigational com- pound that inhibits enzymes called Polo- like kinases (Plks). Plk1 is the best charac- terized kinase of the Plk family. Inhibition of Plk1 by volasertib ultimately results in apoptosis. when Plk1 activityis inhibited, the extremely high cell division that is characteristic of AML should be blocked, which may result in cancer regression, ac- cording to the company. Chronic Heart Failure > CardioCell began its Phase IIa trial for chronic heart failure patients with non- ischemic cardiomyopathy. More than 20 patients are being recruited at the study's three sites—Emory University, Northwest- ern University, and the University of Penn- sylvania. The treatment and placebo will be administered intravenously. Progress will be tracked at the base- line, at 90 days, and at 180 days and will be measured by MRI to observe improve- ments in ejection fraction (EF) and seg- mental changes in contractility. At 90 days after the frst injection, the control group will receive treatment, and the previous treatment group will become the control group receiving saline solution. The end- point includes EF measurements every three months to observe signs of efcacy via increase in EF. Herpes > Genocea Biosciences repor ted positive top-line data from a Phase I/IIa study of GEN-003, the company's immu- notherapy candidate against herpes sim- plex virus type 2 (HSV-2). At 12 months after the fnal dose, in an analysis of an exploratory endpoint, the mean reduc- tion in genital lesion rate was 42% be- low baseline for the best-performing 30 microgram dose group, indicating that GEN-003 may continue to provide a du- rable efect on genital lesions beyond six months. GEN-003 remained safe and well tolerated. Final analysis of results at six months after dosing showed that the 30 microgram dose group decreased the genital lesion rate by 65%, and the viral shedding rate by 40%, both versus baseline. At 12 months, the mean viral shedding rate reverted to base- line levels for all dose groups. Both antibody and T-cell responses to GEN-003 remained elevated over baseline at 12 months. Patients were sequentially enrolled into one of three dose cohorts (10, 30, or 100 micrograms of each protein) and random- ized within cohorts to receive either GEN- 003, vaccine antigens without adjuvant, or placebo. Patients received three injections of the assigned treatment into an arm mus- cle at 21-day intervals, and were followed for 12 months after the third injection. n TRANSLATIONAL MEDICINE