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.

Glutathione arsenoxide (GSAO) is a peptide trivalent arsenical that has potential anti-angiogenic capability, suggesting that it could be used for treatment in cancers where tumor metastasis relies on new blood vessel formation (angiogenesis). Endothelial cell proliferation drives new blood vessel formation. GSAO treatment causes a concentration-dependent increase in superoxide levels, ATP depletion, mitochondrial depolarization, and apoptosis in proliferating, but not endothelial cells. GSAO is able to block blood flow to solid tumors in mice, thereby inhibiting tumor growth in mice with no apparent toxicity at efficacious doses. Initial experiments have implicated GSAO in perturbing mitochondrial function. Other molecular effects of GSAO in human cells, for example on the phosphorylation of proteins, are still largely unknown. A phase I clinical trial has been terminated.

Rislenemdaz, also known as MK-0657 or CERC‐30, is an orally active, selective NMDA receptor subunit 2B (NR2B) antagonist which was a study for the treatment of Parkinson's disease and major depressive disorder (MDD). The data from the phase I clinical trials have shown that drug was not effective in improving motor symptoms in patients with Parkinson disease. In case of using this drug to treat MDD, in spite of the Missing Primary Endpoint in phase II clinical trials, it was shown, that MK-0657 had possessed a potential clinical meaningfulness, that is why it was suggested to continue studying it for MDD patients.

Iodohippuric acid I-125 (I-I25 Hippuran) is radioisotope that was used as a renogram probe in nefhrography in 1960s-1970s. It is an analog of I-131 labeled I(131) hippuran which has been recognized as the radiopharmaceutical standard for the measurement of effective renal plasma flow in subjects with renal failure but which use has been compromised by the suboptimal imaging characteristics of the 364-keV photon of 131I and the delivery of relatively high radiation doses to kidneys and thyroid in patients with impaired renal function. It was shown that radiation risk was rendered to a minimum with the use of the "cocktail" of 169U-EDTA and 125I-hippuran.

Pfizer was developing PF-04991532, a potent and selective hepatoselective glucokinase activator. PF-04991532 ameliorates hyperglycemia without causing hepatic steatosis in diabetic rats. F-04991532 reduced plasma glucose concentrations independent of changes in insulin concentrations in a dose-dependent manner both acutely and after 28 days of sub-chronic treatment. PF-04991532 may offer glycemic control without inducing hepatic steatosis supporting the evaluation of tissue specific activators in clinical trials. In 2012, Pfizer discontinued the development of the compound.

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).

Icometasone (CL09) is a synthetic glucocorticoid corticosteroid. It is a metabolite of Mometasone Furoate. The binding on human serum albumin was shown to be non saturable, suggesting that other proteins were involved in CL09 binding. This binding was demonstrated to be reversible. CL09 was extensively metabolized since no unchanged CL09 was recovered in bile or urine and at least nine metabolites have been detected. It was studied in Europe as an anti-asthmatic agent but investigation is discontinued.