Fluorocyclopentenylcytosine (RX-3117) is a novel small molecule nucleoside compound that is incorporated into DNA or RNA of cancer cells and inhibits both DNA and RNA synthesis which induces apoptotic cell death of tumor cells. Fluorocyclopentenylcytosine also mediates the down-regulation of DNA methyltransferase 1 (DNMT1), an enzyme responsible for the methylation of cytosine residues on newly synthesized DNA and also a target for anticancer therapies. Preclinical studies have shown Fluorocyclopentenylcytosine to be effective in both inhibiting the growth of various human cancer xenograft models, including colon, lung, renal and pancreas, as well as overcoming chemotherapeutic drug resistance. Fluorocyclopentenylcytosine has demonstrated a broad spectrum anti-tumor activity against 50 different human cancer cell lines and efficacy in 12 different mouse xenograft models. The efficacy in the mouse xenograft models was superior to that of gemcitabine. In addition, in human cancer cell lines made resistant to the anti-tumor effects of gemcitabine, Fluorocyclopentenylcytosine still retains its full anti-tumor activity. In August 2012, Rexahn reported the completion of an exploratory Phase I clinical trial of Fluorocyclopentenylcytosine in cancer patients conducted in Europe, to investigate the oral bioavailability, safety and tolerability of the compound. In this study, oral administration of Fluorocyclopentenylcytosine demonstrated an oral bioavailability of 34-58% and a plasma half-life (T1/2) of 14 hours. In addition, Fluorocyclopentenylcytosine was safe and well tolerated in all subjects throughout the dose range tested. Fluorocyclopentenylcytosine is in phase I/II clinical trials by Rexahn for the treatment of bladder cancer and pancreatic cancer. This compound was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pancreatic cancer in September 2014.

18F-THK-5351 is a novel radiotracer that demonstrates high binding selectivity and affinity for tau pathology and exhibits better pharmacokinetics in the living brain than previous THK tau probes. FluoroTau is in phase II clinical trials as a positron emission tomography (PET) imaging agent for the diagnosis and monitoring of the progression of Alzheimer's disease(AD) in South Korea. This compound was originally discovered by Tohoku University, and now is being developed by GE Healthcare, Samung Medical Centre and Asan Medical Center.

BMS-707035 is a potent, specific, and reversible HIV-I integrase (IN) inhibitor that blocks HIV IN strand transfer activity. Bristol-Myers Squibb was developing BMS-707035 for the treatment of HIV infections, however development was discontinued.

BLZ 945, an orally active antagonist of the colony-stimulating factor1 receptor (CSF1R), is being developed by Novartis and Celgene Corporation for the treatment of advanced solid tumors and tumor-induced osteolytic lesions in bone and skeletal-related events. Phase I/II development for solid tumors is underway in the US, Italy, Spain, and Singapore. Preclinical trials were ongoing for tumor-induced osteolysis in Europe and the US. However, no recent reports of development had been identified for this indication.

AMG-319 is an oral, small molecule inhibitor of PI3Kdelta which is now being tested in phase II for the treatment of head and neck squamous cell carcinoma and in phase I for lymphoid malignancy.

Mitoglitazone (previously known as MSDC-0160 or CAY-10415) is a mTOT (mitochondrial target of thiazolidinediones) modulator that targets the mitochondrial pyruvate carrier (MPC), which is a key controller of cellular metabolism. MSDC-0160 is modulated MPC and act as insulin sensitizers without activating PPAR gamma. (Mitoglitazone exhibits very low binding affinity and activity at PPARγ). Mitoglitazone has been used in trials phase II studying the treatment of Type 2 Diabetes and Alzheimer's disease; the treatment for diabetes was discontinued. In addition, MSDC-0160 has demonstrated significant neuroprotective effects in the En1+/- mouse model of Parkinson’s disease via modulation of the mTOR-autophagy signaling cascade.

CC-223 is an orally available inhibitor of the mammalian target of rapamycin (mTOR) with potential antineoplastic activity. mTOR kinase inhibitor CC-223 inhibits the activity of mTOR, which may result in the induction of tumor cell apoptosis and a decrease in tumor cell proliferation. CC-223 disrupted mitochondrial function, and induced mitochondrial permeability transition pore (mPTP) opening and reactive oxygen species (ROS) production. CC-223 is currently in phase II clinical trials for the treatment of Multiple myeloma; Non-Hodgkin's lymphoma; Solid tumours. The most common treatment-related adverse events were hyperglycemia, fatigue and rash.

CUDC-907 is a small molecule inhibitor of histone deacetylase and PI3 kinase developed by Curis. It is investigated in clinical trials for the treatment of relapsed or refractory lymphomas, thyroid cancer, multiple myeloma, breast cancer and other malignancies.

Merestinib (LY2801653) is a small molecule that has been shown in vitro to be a reversible type II ATP-competitive slowoff inhibitor of MET tyrosine kinase with a dissociation constant (Ki) of 2 nM, a pharmacodynamic residence time (Koff) of 0.00132 min−1 and a half-life (t1/2) of 525 min. Preclinical testing also has shown merestinib to inhibit several other receptor tyrosine oncokinases including MST1R, FLT3, AXL, MERTK, TEK, ROS1, NTRK1/2/3, and DDR1/2 and the serine/threonine kinases MKNK1/2. Merestinib is being investigated in a phase II clinical trials in patients with biliary tract cancer, non-small cell lung cancer and solid tumours.

R1479 (4′-azidocytidine) is a specific inhibitor of hepatitis C virus (HCV) replication. 4′-azidocytidine triphosphate is an inhibitor of native HCV replicase and recombinant HCV polymerase (NS5B). Balapiravir (R1626) is the tri-isobutyrate ester prodrug of R1479 under clinical development to improve exposure of R1479 upon oral administration.