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news.Boston Biomedical announced that its lead investigational compound, napabucasin, has been granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in the treatment of gastric cancer, including gastroesophageal junction (GEJ) cancer
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Napabucasin is an orally administered cancer stemness inhibitor designed to inhibit cancer stemness pathways by targeting STAT3, and is currently being investigated in a global Phase III clinical trial in combination with paclitaxel in patients with advanced gastric and GEJ cancer (the BRIGHTER study).
"This first Orphan Drug Designation for napabucasin is an important milestone for Boston Biomedical, as well as for patients with advanced gastric/GEJ cancer," 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.
"In the U.S., only about 1 in 3 patients with gastric cancer survive five years after diagnosis, so there remains a significant need for innovation in this tumor type. We hope that napabucasin, with a first-in-class mechanism of action, will help address this significant unmet medical need."
The FDA’s Orphan Drug Designation program provides special status and development incentives for drugs and biologics that 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 Each year, about 26,000 people in the U.S. are diagnosed with gastric cancer.ii
Phase Ib/II data for napabucasin in gastric/GEJ cancer were previously presented at the American Society of Clinical Oncology 2015 annual meeting; these data found that napabucasin and weekly paclitaxel can be combined in patients with advanced pre-treated gastric/GEJ cancer.
Lesion regression, objective responses and prolonged stable disease were observed in heavily pre-treated patients. Common adverse events identified through this clinical trial included grade 1 or 2 diarrhea, nausea, fatigue, abdominal pain, vomiting and anorexia.iii
Cancer Stem Cells (CSCs) exhibit 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 the 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.