Mibampator, also known as LY451395, is a potent and highly selective an AMPA receptor potentiator, which plays a role in the regulation of the glutamatergic system. The AMPA system also has important functions in the regulation of synapses, synaptic regeneration, and neuroprotection and is therefore a good therapeutic target for treatments aiming to improve cognition and function or alter disease progression. Mibampator was in the phase II clinical trial for the treatment of agitation and aggression in Alzheimer's disease (AD).

Moxaprindine is a antiarrhythmic drug for the treatment of ventricular arrhythmias patented by Manufacture de Produits Pharmaceutiques A. Christiaens, S. A. In clinical studies Moxaprindine shows high efficacy in suppressing ventricular arrhythmias occurring before, during and after maximal exercise stress testing. This effect was obtained both in subjects with clinically normal hearts and in a limited number of patients with ischemic heart disease.

Brostallicin is a DNA minor groove binder which has shown very promising preclinical activity against a variety of human tumors both in vitro and in vivo. Brostallicin shows potent in vitro cytotoxic activity against tumor cells with IC50 values in the low nanomolar range, circumvents resistance to alkylating agents and camptothecins. Brostallicin has broad antitumor activity in animal models. A clear therapeutic gain is observed in preclinical models when brostallicin is combined with anticancer agents such as cisplatin, doxorubicin, and taxotere.

8-chloroadenosine (8-Cl-Ado) is a ribonucleoside analog. The mechanism of its action remains poorly understood, however, it is known that the drug inhibits RNA synthesis. It has significant cytotoxic activity against lymphoid and myeloid malignant cells. The nucleoside analog 8-Cl-Ado is phosphorylated into its cytotoxic triphosphate 8-Cl-ATP. The accumulation of the cytotoxic metabolite results in a parallel decrease of the ATP cellular pools. 8-Cl-Ado gets incorporated into RNA during transcription, hindering this process. In addition, this triphosphate inhibits ATP-dependent poly(A) tail synthesis, and, as a consequence, mRNA processing is inhibited, resulting in vitro cytotoxicity in several solid and hematological malignancies. This agent is currently in clinical trials for the treatment of chronic lymphocytic leukemia and acute myeloid leukemia.

Allicin (diallylthiosulfinate) is a defence molecule from garlic (Allium sativum L.) with a broad range of biological activities. Allicin is produced upon tissue damage from the non-proteinogenic amino acid alliin (S-allylcysteine sulfoxide) in a reaction that is catalyzed by the enzyme alliinase. Allicin is known to be hydrophobic and can be readily absorbed through the cell membrane without inducing any damage to the phospholipid bilayer and then rapidly metabolized to exert pharmacological effects. The majority of allicin's effects are believed to be mediated via redox-dependent mechanisms. Allicin is physiologically active in microbial, plant and mammalian cells. In a dose-dependent manner allicin can inhibit the proliferation of both bacteria and fungi or kill cells outright, including antibiotic-resistant strains like methicillin-resistant Staphylococcus aureus (MRSA). In plants allicin inhibits seed germination and attenuates root-development. Furthermore, in mammalian cell lines, including cancer cells, allicin induces cell-death and inhibits cell proliferation. Allicin was found to provide cardio-protective effects by inducing vasorelaxation and alleviating various pathological conditions of CVD, including cardiac hypertrophy, angiogenesis, platelet aggregation, hyperlipidemia and hyperglycemia. Allicin was also discovered to further protect the cardiovascular system by enhancing the antioxidant status by lowering the level of reactive oxygen species and stimulating the production of glutathione.

Johnson and Johnson was developing usistapide, a microsomal triglyceride transfer protein (MTTP) inhibitor, for the treatment of obesity and type 2 diabetes. Usistapide, also known as JNJ-16269110 and R256918, has been used in trials studying the treatment of obesity, overweight, metabolic diseases, nutrition disorders, and nutritional and metabolic diseases. Usistapide was removed from the company pipeline and appears to have been discontinued.

LY404039 [(-)-(1R,4S,5S,6S)-4-amino-2-sulfonylbicyclo[3.1.0]-hexane-4,6-dicarboxylic acid] is an agonist of orthosteric metabotropic glutamate receptor (mGluR)2/3. In addition, it acts as an agonist at dopamine D2 receptors. LY404039 demonstrated broad antipsychotic and anxiolytic efficacy across multiple animal models. LY-2140023 is a methionine amide prodrug of LY-404039 being developed by Eli Lilly & Co for the potential oral treatment of schizophrenia.

Combretastatin is a phenol derivative isolated from the bark of Combretum caffrum, commonly known as South African Bush Willow. Combretastatin is an effective antimitotic agent, and like colchicine, inhibited tubulin polymerization and stimulated tubulin-dependent GTP hydrolysis.