The American Association for Cancer Research (AACR) Annual Meeting is taking place in Atlanta. Here’s a look at just a few stories coming out of the meeting.
The American Association for Cancer Research (AACR) Annual Meeting is taking place in Atlanta, with biopharma companies and academic institutions presenting cutting-edge science. Here’s a look at just a few stories coming out of the meeting.
Clovis Oncology’s Interim Data From Phase II Trial of Rubraca in Pancreatic Cancer
Boulder, Colo.-based Clovis Oncology gave multiple presentations at the AACR meeting. Today’s was interim results from the Phase II trial of Rubraca (rucaparib) in platinum-sensitive patients with advanced pancreatic cancer. The data suggested that the drug for first-line maintenance therapy after treatment with platinum-based chemotherapy provides disease control in patients with BRCA1, BRCA2 or PALB2 mutations.
“PARP inhibitors have demonstrated activity in multiple cancers that are associated with BRCA mutations,” stated Kim A. Reiss Binder, Assistant Professor of Medicine in the Perelman School of Medicine at the University of Pennsylvania and primary investigator of the study. “Given the seemingly intractable challenge presented by pancreatic cancer, we are very pleased that early results from this study support the mounting evidence suggesting PARP inhibitors may have a beneficial role in this disease.”
MiNA Therapeutics Provides Preclinical Data of the Effects of MTL-CEBPA
MiNA Therapeutics, headquartered in London, UK, presented preclinical data in support of the immunological effects of MTL-CEBPA in combination with sorafenib, checkpoint inhibitors and radiofrequency ablation. MTL-CEBPA is a double-stranded RNA formulation in a liposomal nanoparticle designed to activate the CEBPA gene. The CEBPA gene encodes CCAAT/enhancer binding protein alpha, a transcription factor that is a master regulator of various cell lines in myeloid cells, liver cells and adipose tissue. C/EBP-alpha in cancer is important in regulating tumor growth and the tumor immune microenvironment.
“With these additional preclinical studies, we continue to elucidate the benefits that our lead candidate MTL-CEBPA can provide not only alone but also in combination to improve standard of care treatments,” stated Robert Habib, chief executive officer of MiNA Therapeutics. “We look forward to sharing these results with the scientific community at AACR which support our ongoing clinical investigation of MTL-CEBPA in severe liver cancer patients.”
Noxxon Pharma’s NOX-A12 in Phase I/II in Pancreatic and Colorectal Cancer
NOXXON Pharma, located in Berlin, Germany, presented results from its Phase I/II trial of NOX-A12 in combination with Keytruda (pembrolizumab) in patients with microsatellite-stable, metastatic pancreatic and colorectal cancer. The study found the drug was safe and well-tolerated. The combination of NOX-A12 and Keytruda created an immune response and stable disease in 25% of patients and prolonged the time on treatment versus prior therapy for 35% of patients. Overall survival was 48% at six months and 33% at 12 months.
“Based on the fact that these indications have remained unaffected by checkpoint inhibitors, the outcome of this trial emphasizes the real potential of NOX-A12 has on targeting the tumor microenvironment and enabling the intended mode of action of pembrolizumab,” stated Aram Mangasarian, chief executive officer of Noxxon. “These results provide a strong rationale for moving this program forward into a larger scale randomized trial with a less-advanced patient population, an opportunity we intend to pursue with a partner.”
MD Anderson’s Umbralisib in Lymphoma
Nathan Fowler, associate professor in the Department of Lymphoma & Myeloma at The University of Texas MD Anderson Cancer Center presented data from a Phase II trial of umbralisib in relapsed marginal zone lymphoma (MZL). Fowler and his group reported that 55% of patients who had at least six months of follow-up had a partial or complete response after receiving the drug. Umbralisib is a small-molecule inhibitor targeting a signaling pathway linked to MZL cell growth and expansion. The trial enrolled 69 patients, with 38 patients responding positively. Progression-free survival was 71% after one year.
“Umbralisib is part of a new class of drugs that are quite active in low-grade lymphomas,” Fowler stated. “These PI3K inhibitors have shown activity across a spectrum of low-grade lymphomas and are effective in shutting down some of the key signaling that is occurring with MZL.”
Baylor College of Medicine Presents CAR-T and Chemo in Sarcoma
Shoba Navai, Assistant Professor of Pediatrics at the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children’s Hospital, and Houston Methodist Hospital presented results from a Phase I trial of HER-2-targeted CAR-T therapy. In the trial, Navai and colleagues treated 10 patients from the ages of 4 to 54, with refractory/metastatic HER2-positive sarcoma. Five had osteosarcoma, three rhabdomyosarcoma and one each with Ewing sarcoma and synovial sarcoma. They had received up to five previous salvage therapies. They received up to three infusions of HER2-targeted CAR-T cells after lymphodepletion with either fludarabine or fludarabine and cyclophosphamide. In eight of the patients, CAR-T cells expanded with a median peak expansion on the seventh day. And CAR-T cells were found in all patients at six weeks.
Navai stated, “Our study shows that HER2-targeted CAR-T-cell therapy given in combination with lymphodepletion chemotherapy is safe, and though very early, it shows promising antitumor activity in some patients with advanced HER2-positive sarcomas.”
