Neuroendocrine tumors (NETs) are a group of rare and heterogeneous malignancies, most commonly arise from neuroendocrine cells of the gastrointestinal tract (particularly pancreas and small bowel), followed by lung, liver, and thymus. While most NETs are more indolent than other epithelial malignancies, poorly differentiated NETs can become aggressive and resistant to therapy. The PI3K/AKT/mTOR signaling pathway plays a central role in the pathogenesis and progression of NETs [1] and clinical studies [RADIANT1-4] demonstrated safety and efficacy of mTOR inhibitors in patients with NET.

A phase 2 pilot study is currently investigating the utility of ABI-009 as single agent in in patients with metastatic, unresectable, low or intermediate grade neuroendocrine tumors of the lung or gastroenteropancreatic system (NCT03670030).

1. Chan et al., Therap Adv Gastroenterol 2017 10(1): 132-141.