CNDAC (TAK-109) is an analog of the nucleoside deoxycytidine with potential antineoplastic activity. CNDAC is incorporated into DNA and induces single-strand breaks, which are converted into double-strand breaks (DSBs) when cells go through a second S phase. This results in the cell cycle arrest in the S and G2/M phases, DNA fragmentation, and tumor cell apoptosis. Sapacitabine, a prodrug of CNDAC, is being developed by the US biotechnology company Cyclacel for the treatment of hemalogical cancers and solid tumors.

A diaminopyrimidine compound R547 is a small molecule selective ATP-competitive inhibitor of cyclin-dependent kinases CDK1, CDK2 and CDK4 and has excellent in vitro cellular potency, inhibiting the growth of various human tumor cell lines. In vivo, R547 showed antitumor activity in all of the models tested to date, including six human tumor xenografts and an orthotopic syngeneic rat model. R547 was being developed by Roche for the treatment of solid tumours. The compound was undergoing clinical development in the US. However, no recent development has been reported and it is assumed to have been discontinued.

MP-412 (AV-412) is a potent dual inhibitor of EGFR and ErbB2 tyrosine kinases, including the mutant EGFR (L858R,T790M), which is clinically resistant to the EGFR-specific kinase inhibitors erlotinib and gefitinib. AV-412 has potential as a therapeutic agent for the treatment of cancers expressing EGFR and ErbB2, especially those resistant to the first generation of small-molecule inhibitors.AVEO Pharmaceuticals was developing AV-412 for the treatment of cancer, however development has been discontinued.

PCI-27483 is a reversible small-molecule inhibitor of activated factor VII (factor VIIa) with potential antineoplastic and antithrombotic activities. FVII, a serine protease, becomes activated (FVIIa) upon binding with TF forming the FVIIa/TF complex, which induces intracellular signaling pathways by activating protease activated receptor 2 (PAR-2). Upon subcutaneous administration, factor VIIa inhibitor PCI-27483 selectively inhibits factor FVIIa in the VIIa/TF complex, which may prevent PAR-2 activation and PAR2-mediated signal transduction pathways, thereby inhibiting tumor cell proliferation, angiogenesis, and metastasis of TF-overexpressing tumor cells. A phase I study in healthy volunteers was conducted to assess the PD and PK profiles of PCI- 27483 following a single, SC injection. The halflife of PCI-27483 was 10-12 h. The International Normalized Ratio (INR) was strongly correlated with drug plasma concentration. PCI-27483 was well tolerated. This compound has being evaluated in a phase Ib/II trial in patients with pancreatic cancer receiving treatment with gemcitabine. However, no recent development has been reported. The compound was originally developed by Celera, then licensed to Pharmacyclics (acquired by Abbvie in 2015) later. In 2012, the product was licensed to Novo Nordisk by Pharmacyclics for disease outside of oncology.

Epetraborole (AN3365 or GSK2251052) is a novel boron-containing antibiotic that inhibits bacterial leucyl tRNA synthetase. It is active against Gram-negative bacteria, including Enterobacteriaceae bearing NDM-1 and KPC carbapenemases, as well as P. aeruginosa. Epetraborole was under development for the treatment of Gram-negative bacterial infections.

TAK-875 (Fasiglifam) is the potent, selective and orally bioavailable GPR40 agonist. The drug was in Phase III clinical trials for the treatment of type 2 diabetes mellitus. Termination phase III development of TAK-875 for the potential treatment of type-2 diabetes mellitus was announced in 2013 due to concerns about liver safety.

Fozivudine tidoxil is a thioether lipid–Zidovudine (ZDV) conjugate. After intake it is split intracellularly into the lipid moiety and ZDV-monophosphate, which is subsequently phosphorylated to the active metabolite ZDV-triphosphate. The rationale behind the development of fozivudine (FZD) was to take advantage of the high cleavage activity in mononuclear cells and other organs resulting in increased amounts of intracellular ZDV available for phosphorylation to the active metabolite, and a very low activity in red blood and stem cells, which should result in reduced haematologic toxicity. It is member of the family of nucleoside reverse transcriptase (RT) inhibitors. Fozivudine tidoxil has been in Phase II clinical trials for the treatment of HIV infection. There were three adverse events possibly related to fozivudine: urine abnormality, gastrointestinal pain and abnormal dreams.

Pterostilbene is a naturally derived compound found primarily in blueberries and Pterocarpus marsupium heartwood. The multiple benefits of pterostilbene in the treatment and prevention of human disease have been attributed to its antioxidant, anti-inflammatory, and anti-carcinogenic properties leading to improved function of normal cells and inhibition of malignant cells. The antioxidant activity of pterostilbene has been implicated in anti-carcinogenesis, modulation of neurological disease, anti-inflammation, attenuation of vascular disease, and amelioration of diabetes. Pterostilbene increases LDL and reduces blood pressure in adults. Low doses of pterostilbene seem to hold some benefit for cognition.

Rigosertib sodium (ON 01910.Na) is a small molecule inhibitor of critical pathways important in the growth and survival of cancer cells, being developed by Onconova Therapeutics ("Onconova") for the treatment of hematologic malignancies and solid tumors. Rigosertib (ON-01910) is a non-ATP-competitive inhibitor of PLK1 with IC50 of 9 nM in a cell-free assay. It shows 30-fold greater selectivity against Plk2 and no activity to Plk3. Extensive Phase I and Phase II studies with rigosertib have been conducted at leading institutions in the U.S. and abroad in more than 450 patients with solid tumors and hematological cancers, including MDS and AML. MDS and AML are blood disorders widely recognized as difficult to manage, with limited therapeutic options available for patients, especially those with drug-resistant disease. The multi-site Phase III ONTIME trial in MDS patients is under a Special Protocol Assessment (SPA) from the U.S. FDA and is being supported by an award from the Therapeutics Acceleration Program (TAP) of the Leukemia and Lymphoma Society (LLS). FDA has granted Orphan Drug Designation for the use of rigosertib in MDS. The clinical program in solid tumors is also advancing with initiation of the Phase II/III combination ONTRAC trial (ON 01910.Na TRial in Patients with Advanced Pancreatic Cancer) and Phase II single agent trial in ovarian cancer. In Japan, SymBio is developing rigosertib for the treatment of refractory/relapsed HR-MDS (IV form) and first-line LR-MDS (oral form).