CAMBRIDGE, Mass., Nov. 14, 2016 /PRNewswire/ -- Boston Biomedical, an industry leader in the development of novel compounds designed to target cancer stemness pathways, announced that its lead investigational compound, napabucasin, has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in the treatment of pancreatic cancer. This is the second Orphan Drug Designation for napabucasin, an orally administered agent designed to inhibit cancer stemness pathways by targeting STAT3; the first designation was for gastric cancer including gastroesophageal junction (GEJ) cancer.
"Receiving another Orphan Drug Designation for napabucasin is an important regulatory milestone achieved by Boston Biomedical and an exciting step towards the clinical advancement of this first-in-class therapy," said Chiang J. Li, M.D. FACP, President, CEO and Chief Medical Officer of Boston Biomedical, and the Head of Global Oncology for Sumitomo Dainippon Pharma Group. "Pancreatic cancer has a five year survival rate of 7% and few viable treatment options. This designation represents our determination to address an unmet need and potentially bring a new treatment to those with this difficult-to-treat cancer." The FDA's Orphan Drug Designation program provides special status and development incentives for drugs and biologics which are intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. i In 2016, it is estimated that over 53,000 people in the U.S. will be diagnosed with pancreatic cancer. ii Phase Ib data for napabucasin in metastatic pancreatic cancer (NCT02231723) were previously presented at the American Society of Clinical Oncology 2016 annual meeting. These data showed that napabucasin may be combined with gemcitabine and nab-PTX and showed signs of anti-tumor activity in patients with metastatic pancreatic cancer. Of the 37 patients enrolled in the study, fifty-seven percent of patients (17 of 30 evaluable patients) had prolonged disease control (=24 weeks). iii Common adverse events (AEs) identified in this clinical trial were grade 1 diarrhea, nausea, fatigue and neuropathy, which were reversible and manageable with symptom medications. iiiAbout Cancer Stem CellsCancer stem cells (CSCs) possess the property of stemness - the ability to self-renew and differentiate into heterogeneous cancer cells. This allows the CSCs to act like seeds, causing a patient's cancer to relapse or spread within their body. iv,v Evidence suggests that these cells possess resistance to conventional chemotherapy and radiation, so while such treatments can successfully shrink tumors, a population of CSCs may still survive. v,vi Boston Biomedical is leading the biopharmaceutical industry in the development of novel compounds designed to target cancer stemness pathways, with the goal of addressing ongoing challenge in cancer treatment.