Nicanartine [MRZ 3124] is an antioxidant and cholesterol-lowering agent that was under development with Merz + Co. (later Merz Pharma) in Germany. Development of nicanartine for the treatment of atherosclerosis and reperfusion injury was discontinued at the phase I stage.

Silydianin is a flavonolignan from Silymarin, which is the major constituent of milk thistle extract. Silydianin, an active constituent of Silybium marianum, have inhibitory properties against the P2Y12 receptor and block ADP-induced blood platelet activation. Silydianin has antiinflammatory activity, it regulates caspase-3 activation, affects cell membranes and acts as a free radical scavenger. Silydianin non-competitively inhibits the lipoxygenase from soybeans in vitro. It also inhibits the formation of prostaglandins in vitro.

CPI-613 is a lipoate derivative synthesized to be catalytically inert but to potentially mimic lipoate catalytic intermediates. The drug is in phase II of clinical trials for the treatment of Myelodysplastic syndromes; Pancreatic cancer; Small cell lung cancer; Solid tumors; Bile duct cancer; Acute Myeloid leukemia. The mechanism of CPI-613 action can be explained by (I) inhibition of tumor cell pyruvate dehydrogenase complex (PDC) through activation of pyruvate dehydrogenase kinases leading to inactivating phosphorylation of the E1alpha-subunit of PDC; and (II) inhibition of alpha-ketoglutarate dehydrogenase.

Arimoclomol (INN; originally codenamed BRX-345, which is a citrate salt formulation of BRX-220) is an experimental drug developed by a biopharmaceutical company CytRx Corporation. In 2011 the worldwide rights to arimoclomol were bought by Danish biotech company Orphazyme ApS. The European Medicines Agency (EMA) and U.S. Food & Drug Administration (FDA) granted orphan drug designation to arimoclomol as a potential treatment for Niemann-Pick type C in 2014 and 2015 respectively. Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones. Since damaged proteins, called aggregates, are thought to play a role in many diseases, CytRx believes that arimoclomol could treat a broad range of diseases.

Limazocic is a cyclic disulfide compound newly synthesized as a hepatoprotective agent. It has a protective effect against both chronic liver injuries induced by carbon tetrachloride (CCL) and swine serum and several chemically induced acute liver injuries. The inhibitory effects of limazocic on immunologically induced liver injuries in mice have also been demonstrated and furthermore, limazocic has been shown to have immunomodulatory effects such as induction of cytotoxic cell activities and modulation of interleukin-2 and interferon-gamma production. Limazocic had a markedly inhibitory effect on ANIT-induced intrahepatic cholestasis in rats. Limazocic suppresses VEGF production and reduces vascular leakage and the growth of mouse experimental choroidal neovascularization.

Ciapilome is pyrimidinone derivative patented by German pharmaceutical company Byk Gulden Lomberg Chemical Factory GmbH as xanthine oxidase inhibitor for the treatment of gout, arrhythmia, and heart insufficiencies. Ciapilome causes a pronounced decrease in uric acid blood levels in rats on peroral administration.

Cebaracetam is piperazinylpyrrolidinone derivative patented by pharmaceutical company Ciba-Geigy A.-G. for treatment of amnesia and retention defects.

Vindeburnol, a derivative of the plant alkaloid vincamine that that bears neuroprotective properties. Animal model for Alzheimer's disease has shown that vindeburnol reduced neuroinflammation and amyloid burden. In addition, the treatment with this drug can be of benefit in multiple sclerosis patients.

Aloracetam [CAS 493] is a nootropic agent with potential in the treatment of Alzheimer's disease. Aloracetam was undergoing clinical evaluation with Hoechst for the treatment of Alzheimer's disease, but this research was discontinued later.

Cipralisant (GT-2331) is an H3-receptor ligand developed by Gliatech in the late 1990s. The drug exhibits complex pharmacology: it behaves as an antagonist in a guinea pig jejunum contraction assay and mouse disogenia model, as a partial agonist in a rat brain synaptosome model, and as a full agonist at recombinant H3 receptors. In preclinical models, cipralisant enhanced wakefulness and improved learning in developmental rat models. In 2000 cipralisant was investigated in the clinical trials for the treatment of attention-deficit hyperactivity disorder, but due to the bankruptcy of Gliatech the development of the drug was discontinued.