CPP-115 is a gamma-aminobutyric acid aminotransferase activator, developed by Catalyst Pharmaceutical Partners, Inc. The compound does not exhibit other GABAergic or off-target activities and is rapidly and completely orally absorbed and eliminated. In rats, CPP-115 produced inhibition of cocaine-induced increases in extracellular dopamine and in synaptic dopamine in the nucleus accumbens. CPP-115 decreased spasms in the multiple-hit rat model of infantile spasms. CPP-115 was found to have high therapeutic potential for the treatment of cocaine addiction and for a variety of epilepsies, has successfully completed a Phase I safety clinical trial, and was found to be effective in the treatment of infantile spasms (West syndrome).

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.

Samatasvir (also known as IDX 719) was developed by Idenix Pharmaceuticals as a pan-genotypic inhibitor of the hepatitis C (HCV) non-structural protein 5A (NS5A). This drug was studied in phase II clinical trials for the treatment of Hepatitis C, and Chronic Hepatitis C Infection. However, the development of samatasvir was discontinued.

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.