BERKELEY, Calif. – Aduro Biotech, Inc. today announced that the Office of Orphan Product Development of the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CRS-207, a novel immuno-oncology product candidate, for the treatment of mesothelioma. This designation potentially allows Aduro seven years of limited marketing exclusivity in the United States if it is the first to obtain FDA marketing approval for mesothelioma, and qualifies the company for grant funding to offset the cost of clinical testing as well as tax credits for certain research and a waiver of the Biologics License Application user fee. The FDA previously granted orphan designation to CRS-207 and GVAX Pancreas for the treatment of pancreatic cancer.
“This is an important step for Aduro as we continue to develop CRS-207 for this very difficult to treat cancer,” said Dirk Brockstedt, Ph.D., senior vice president of research and development at Aduro. “We believe the combination of CRS-207 together with chemotherapy may offer the promise of a potential new therapeutic regimen for patients suffering from mesothelioma. Importantly, we plan to report additional data from the ongoing Phase 1b study later this year.”
Aduro is developing CRS-207 for patients with malignant pleural mesothelioma who have not received prior therapy and are not eligible for surgical resection. Patients are currently being enrolled in a single-arm Phase 1b clinical trial of CRS-207 in combination with standard-of-care chemotherapy. Interim results presented at the International Mesothelioma Interest Group Conference in October 2014 demonstrated a 94% rate of disease control (partial response and stable disease) for the 16 treated, evaluable patients with response data. Specifically, 75% (12/16) had confirmed partial responses and 19% (3/16) experienced stable disease. At the time of the presentation, estimated progression-free survival was 7.5 months, with one patient on study for more than 19 months. That patient is currently receiving maintenance therapy with CRS-207 alone following the combination treatment with chemotherapy. Based on these encouraging results, Aduro opened an expansion cohort of up to a total of 40 patients and expects to finish enrollment in the study in 2015, with top-line results presented in 2016.
About Malignant Pleural Mesothelioma
Mesothelioma is a form of cancer that affects the smooth layer of mesothelial cells that surround the chest, lungs, heart and abdomen. Malignant pleural mesothelioma (MPM), which affects the thin balloon-shaped lining of the lungs, is the most common form of this disease and accounts for approximately 3,000 cases a year in the United States. MPM is an aggressive disease with a poor prognosis. Most MPM patients are not candidates for surgical resection. Based on prior studies, expected median progression-free survival is 5.7 months and overall survival is 12.1 months with combination pemetrexed and cisplatin chemotherapy. The tumor-associated antigen mesothelin is overexpressed in virtually all cases of mesothelioma.
CRS-207 is one of a family of product candidates based on Aduro’s live-attenuated, double-deleted (LADD) Listeria monocytogenes immunotherapy platform that induce a potent innate and T cell-mediated adaptive immune response. CRS-207 has been engineered to express the tumor-associated antigen mesothelin, which is over-expressed in many cancers including mesothelioma and pancreatic, non-small cell lung, ovarian, endometrial and gastric cancers.
Aduro Biotech, Inc. is a private, clinical-stage immuno-oncology company focused on the development of technology platforms to stimulate an immune response against cancer. Aduro’s lead platform is based on proprietary strains of live-attenuated, double-deleted (LADD) Listeria monocytogenes that induce a potent innate immune response and have been engineered to express tumor-associated antigens to induce tumor-specific T cell-mediated immunity. Aduro has received Breakthrough Therapy designation from the FDA for its lead LADD regimen, CRS-207 in combination with GVAX Pancreas in pancreatic cancer. The company is evaluating the proprietary immuno-oncology combination in the ongoing Phase 2b ECLIPSE clinical trial and has additional ongoing clinical trials with its LADD platform in mesothelioma and glioblastoma. The company is also developing clinical candidates using cyclic dinucleotide (CDN) synthetic small molecule immune modulators that are designed to activate the intracellular STING receptor, a central mediator of the innate immune response. For more information, please visit www.aduro.com.