Parafluorofentanyl is a selective mu-opioid agonist, an analog of fentanyl, developed by Janssen. The drug was not developed for human use but is produced and abused illegally.

GSK-1614235 (mizagliflozin) is a sodium glucose co-transporter type 1 (SGLT1) inhibitor that has been investigated for treatment in type 2 diabetes. It is thought to suppress glucose absorption from the intestine in a way that is different from conventional type 2 diabetes drugs, thereby improving postprandial hyperglycemia. Phase 1 studies have been completed.

Buparlisib (NVP-BKM12), a dimorpholino pyrimidine derivative, is a selective pan class I phosphatidylinositol-3 kinase (PI3K) inhibitor for treating cancer. It penetrates the blood-brain barrier and has a potential as a glioma treatment. NVP-BKM120 inhibits PI3K activity by binding to the ATP binding cleft of this enzymes and was tested against class I PI3K and other kinases using an ATP depletion (Kinase-Glo) assay. The compound was shown to be active against P110 α, β, γ and δ. The inhibition of the PI3K signaling pathways leads to different forms of cell death on the basis of p53 statuses. Buparlisib demonstrated its activity in human glioblastoma (GBM) cells in vitro and in vivo and is in clinical trials for solid tumors including GBM.

Defoslimod is an analog of lipopolysaccharide endotoxin-derived lipid A obtained from E. coli, developed as an immunomodulatory adjuvant and an immunotherapeutic agent for the treatment of cancer. Defoslimod acts as an agonist of Toll-like receptor (TLR) 2/4. In a phase 1 clinical study in patients with solid tumors, conducted in 2007, defoslimod was administered as an intravenous infusion. The therapy was well tolerated at biologically active concentrations, with 3 patients of 17 exhibited disease stabilization with a mean duration of 4 months. No further development of the drug was reported.

MPEP (2-methyl-6-(phenylethynyl)pyridine) was one of the first compounds found to act a selective antagonist for the metabotropic glutamate receptor subtype mGluR5. It was under development by Novartis in the late 1990's. MPEP was found to produce neuroprotective effects following acute brain injury in animals. MPEP was also found to have positive effects on animal models of depression, anxiety and morphine withdrawl.