ABTL 0812 is a autophagy inducer that acts via PI3K/Akt/mTOR pathway and has a dual mechanism of action. ABTL-0812 is a first-in-class small molecule, orally administered that binds to the nuclear receptors PPARα/γ inducing TRIB3 overexpression which blocks Akt activation, the central kinase of the PI3K/Akt/mTOR pathway, and inducing PPAR-dependent Endoplasmic Reticular Stress (ER-stress). The combination of TRIB3-mediated inhibition of the PI3K/Akt/mTOR pathway and the ER-Stress induction results in an autophagy-mediated cancer cell death. In animal cancer models ABTL0812 is efficacious as single agent with an excellent safety profile in a broad spectrum of cancer types: lung, endometrial and pancreatic cancer and neuroblastoma. ABTL0812 is also active on cells resistant to other targeted therapies, on tumor stem cells and inhibits metastasis formation. Preliminary results show promising immunomodulatory effects. ABTL0812 is currently in phase 2 clinical trials in Europe in patients with endometrial cancer or squamous cell lung cancer, as a first-line treatment in combination with chemotherapy and as a maintenance treatment after the chemotherapy cycles. The study is being conducted in leading cancer hospitals in Spain and France. This same phase 2 study was also approved by the US FDA in December 2017. In addition, the FDA approved the protocol for a phase 2 study in pancreatic cancer in January 2018. ABTL-0812 has also received Orphan Drug Designations (ODD) for pancreatic cancer, biliary cancer and the pediatric cancer neuroblastoma by the FDA in the USA and by the EMA in Europe.

Odalasvir (previously known as ACH-3102) is a second-generation inhibitor of the nonstructural protein 5A (NS5A) of hepatitis C virus (HCV). It was reported that HCV NS5A is associated with interferon signaling related to HCV replication and hepatocarcinogenesis. HCV NS5A inhibitors efficiently inhibited HCV replication. It is known that HCV is a leading cause of hepatocellular carcinoma (HCC) in Japan and is one of the major causes of end-stage liver disease, HCC, and liver transplantation in the United States and Europe. Odalasvir completed phase II clinical trial, where was evaluated efficacy and safety of its combinations with AL-335, and simeprevir in the treatment of chronic hepatitis C Infection.

VX-970 (VE-822) is an ATR kinase inhibitor. VE-822 decreased maintenance of cell-cycle checkpoints, increased persistent DNA damage and decreased homologous recombination in irradiated cancer cells. Vertex Pharmaceuticals is developing VX 970 for the treatment of advanced solid tumours. Phase I/II development is underway in the US for small-cell lung cancer and in the UK for solid tumours. Phase II development of VX 970 as a combination therapy in urogenital cancer, ovarian, primary peritoneal and fallopian tube cancer indications is underway in the US.

ASP-543 (also known as YM-543), a selective inhibitor of the sodium-glucose cotransporter 2. This protein is specifically expressed in the kidney that plays an important role in renal glucose reabsorption, and its inhibition may present a novel therapeutic strategy for treating diabetes. ASP-543 participated in phase II clinical trials in Europe and in the USA for the treatment of Type 2 diabetes mellitus but these studies were discontinued.

Orantinib (SU-6668) is an orally bioavailable receptor tyrosine kinase inhibitor. Orantinib binds to and inhibits the autophosphorylation of vascular endothelial growth factor receptor 2 (VEGFR2), platelet-derived growth factor receptor (PDGFR), and fibroblast growth factor receptor (FGFR), thereby inhibiting angiogenesis and cell proliferation. Orantinib also inhibits the phosphorylation of the stem cell factor receptor tyrosine kinase c-kit, often expressed in acute myelogenous leukemia cells. Orantinib was in phase II clinical trials for the treatment of breast cancer. It was also in phase III clinical trials for the treatment of hepatocellular carcinoma. However, this research was terminated in 2014. The compound was originally developed by Sugen (subsidiary of Pfizer). In 1998, a co-development agreement took place between Sugen and Taiho for the compound.