Clonitazene is a synthetic opioid analgesic, structurally related to etonitazene. In the USA clonitazene is a schedule I narcotic controlled substance.

Forfenimex (also known as forphenicinol), a low molecular weight immunomodifier was studied as an antineoplastic agent. Preclinical experiments in vitro have shown that forfenimex enhanced the antitumor effects of cyclophosphamide in a model of squamous cell carcinoma. Experiments on rodents have revealed that this compound possessed protective effect against P. aeruginosa infection. This influence achieved due to macrophage activation followed by the enhancement of the bactericidal activity of polymorphonuclear cells (PMN).

Glutaurine is formed from the amino group of taurine with the gamma-carboxy group of L-glutamic acid. It was originally discovered in the parathyroid. Evidence has been found suggesting that glutaurine plays a role in peripheral thyroid hormonal regulation. Glutaurine increases triiodothyronine (T3) levels, but does not alter thyroxine (T4) levels. Glutaurine was also shown to prevent electroconvulsive shock-induced amnesia by counteracting the shock effect on the memory consolidation phase. Other roles that have been suggested for glutaurine include roles as a mouse metabolite, a mammalian metabolite, a human metabolite, an anticonvulsant, an anxiolytic drug and a hormone.

Lipoprotein lipase (LPL) is a rate-limiting enzyme that hydrolyzes circulating triglyceride-rich lipoproteins such as very low-density lipoproteins and chylomicrons. Otsuka Pharmaceutical Factory, Inc. synthesized the LPL activator NO-1886 (ibrolipim, [4-(4-bromo-2-cyano-phenylcarbamoyl)-benzyl]-phosphonic acid diethyl ester, CAS 133208-93-2). NO-1886 increased LPL mRNA and LPL activity in adipose tissue, myocardium and skeletal muscle, resulting in an elevation of postheparin plasma LPL activity and LPL mass. It was discovered, that this agent has a potentially beneficial for the treatment of hypertriglyceridemia, hypo-HDL cholesterolemia, and protection from atherosclerosis. In addition, this agent may be used in the treatment of obesity and obesity-linked health problems in postmenopausal women.

Peretinoin is an orally available, acyclic retinoid with potential antineoplastic and chemopreventive activities. Peretinoin binds to and activates nuclear retinoic acid receptors (RAR), which in turn recruit coactivator proteins and promote, with other transcriptional complexes, the transcription of target genes. As a result, this agent may modulate the expression of genes involved in the regulation of cell proliferation, cell differentiation, and apoptosis of both normal and tumor cells. Peretinoin inhibits post-therapeutic recurrence of hepatocellular carcinoma via unclear mechanisms. The European Commission granted Orphan Drug designation for Kowa's peretinoin to treat hepatocellular carcinoma (HCC).

Imexon (INN, trade name Amplimexon) is a substance that is being studied in the treatment of some types of cancer, including pancreatic, lung, breast, prostate, melanoma, and multiple myeloma. Imexon is a thiol-binding small molecule which induces mitochondrial oxidation, a loss of membrane potential and cytochrome C, leading to apoptosis.

Cicaprost is a prostacyclin receptor (IP) agonist and orally active prostacyclin analog with potent systemic and pulmonary vasodilatation and anti-inflammatory activity. In preclinical models, Cicaprost treatment largely prevented the hypercholesterolemia-related impairment of coronary vasodilation and nitric oxide release in Isolated Langendorff-hearts. Cicaprost inhibits proinflammatory chemokines production not only from lipopolysaccharides (LPS) or (tumor necrosis factor-alpha) induced primary human monocyte-derived macrophages but also from LPS-stimulated monocyte-derived dendritic cells. Besides that Cicapost strongly inhibits lymph node and organ metastases of spontaneously metastasizing mammary tumors with a mode of action different from cytostatic or antihormonal drugs. In animal models, Cicaprost prevents metastasis if given continuously from the day of tumor implantation, and is effective in reducing metastasis if treatment is begun following surgical removal of the primary tumor when micrometastases are already present. Clinical trials of Cicaprost in healthy male volunteers demonstrate significant anti-platelet and vasodilatory effects.

Indibulin is a novel synthetic compound that was identified in a cell-based screening assay to discover cytotoxic drugs. Indibulin destabilizes microtubules and blocks cell cycle transition specifically at the G2-M phase. Indibulin effectively induces apoptosis through Bcl-2 phosphorylation and Bax translocation in human malignant glioma cells in a p53-independent manner. This agent has been shown to be active against multidrug-resistant (MDR) and taxane-resistant tumour cell lines. Indibulin was used in phase I/II clinical trials of patients with advanced solid tumours (metastatic breast cancer). Pharmacokinetic analysis showed a better tolerability underfeeding condition. Dose-limiting toxicities were nausea and vomiting, which seemed to be related to solvent lactic acid.

Betulinic acid (BA) is a plant-derived pentacyclic triterpenoid that exerts potent anti-cancer effects in vitro and in vivo. It`s anticancer property is linked to its ability to induce apoptotic cell death in cancer cells by triggering the mitochondrial pathway of apoptosis. In contrast to the cytotoxicity of betulinic acid against a variety of cancer types, normal cells and tissue are relatively resistant to betulinic acid, pointing to a therapeutic window. Compounds that exert a direct action on mitochondria present promising experimental cancer therapeutics, since they may trigger cell death under circumstances in which standard chemotherapeutics fail. Thus, mitochondrion-targeted agents such as betulinic acid hold great promise as a novel therapeutic strategy in the treatment of human cancers. Betulinic acid has antiretroviral, antimalarial, and anti-inflammatory properties. Betulinic acid exerts its inhibitory effect by preventing topoisomerase I-DNA interaction as a result of which the 'cleavable complex' is not formed. In consequence, it also acts as an antagonist to camptothecin-mediated cleavage. The antitumor pharmacological effects of BA consist of triggering apoptosis via the mitochondrial pathway, regulating the cell cycle and the angiogenic pathway via factors, including specificity protein transcription factors, cyclin D1 and epidermal growth factor receptor, inhibiting the signal transducer and activator of transcription 3 and nuclear factor‑κB signaling pathways, preventing the invasion and metastasis of tumor cells, and affecting the expression of topoisomerase I, p53 and lamin B1. Betulinic Acid has also been used in trials studying the treatment of Dysplastic Nevus Syndrome. Betulinic acid acts as anti-melanoma agent through inhibiting aminopeptidase N activity with IC50 of 7.3 uM. Betulinic acid is an inhibitor of HIV-1 with EC50 of 1.4 uM.

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.