A-TRISACCHARIDE (or Blood group A trisaccharide), a core antigen fragment in ABO blood group system. It was found to be a major urinary carbohydrate depending on diet and blood type.

HPA-23 (ammonium-21-tungsto-9- antimoniate) inhibits retrovirus replication by inhibiting RNA-dependent DNA polymerases and thus could be effective in the treatment of HIV-1 infections and AIDS. HPA-23 inhibits nucleic acid polymerases in vitro and has antiviral activity both in vitro and in vivo. Furthermore, HPA-23 inhibits HIV-1 reverse transcriptase in vitro. Inhibition occurred when treatment with HPA-23 was started 18 to 24 h after infection in the plaque assay but no effect was seen when HPA 23 was added 48 h after virus inoculation. HPA-23 was concentrated in the lysosomes and localized in the macrophages of different tissues (thymus, spleen and bone marrow). By 1986, the National Academy of Science had concluded that no therapeutic benefits for persons infected with HIV could be attributed to HPA-23. It was subsequently abandoned as a treatment option.

Plevitrexed is an orally bioavailable, small molecule, non-polyglutamatable, antifolate quinazoline derivative thymidine synthetase inhibitor with potential antineoplastic activity. This compound belongs to the class of organic compounds known as hippuric acids, which consist of a benzoyl group linked to the N-terminal of a glycine. Plevitrexed is transported into the cell via the physiological reduced folate carrier (RFC) system. Intracellularly, this agent selectively binds to the folate binding site of thymidylate synthase and inhibits thymidine synthesis, which may result in DNA synthesis inhibition and apoptosis. Plevitrexed has been investigated for use/treatment in pancreatic cancer, solid tumors, gastric cancer, lung cancer, and colorectal cancer.

RG2833 is a compound originally developed by the Scripps Insitute for the treatment of Friedrick's Ataxia. RG2833 is a potent and selective inhibitor of neuronal histone deacetylase. RG2833 has been granted orphan drug status has been investigated in phase one clinical trials. There has also been proof of concept studies conducted for the treatment of Parkinson's disease.

Epitalon (amino acid sequence Ala-Glu-Asp-Gly) is a synthetic peptide that has been suggested to induce the activation of ribosomal genes, decondensation of pericentromeric structural heterochromatin and the release of genes repressed due to the age-related condensation of euchromatic chromosome regions. The researchers who generated Epitalon also reported an inhibitory effect on the development of spontaneous tumors in mice, and claimed that it increases neuronal activity and can be used in the treatment of Retinitis Pigmentosa. The peptide is still under pre‐clinical and clinical development and thus has not been approved for any therapeutic and/or prophylactic use by any government health authority in the USA or Europe.

RG3039, also known as PF-06687859, is the first small molecule developed specifically for treatment of Spinal muscular atrophy (SMA). This drug is used in SMA patients that have reached early stage clinical trials. It was shown that RG3039 improved survival, function and motor unit pathologies in SMA mouse models. It is known, that RG3039 is a potent inhibitor of the mRNA decapping scavenger enzyme (DcpS). DcpS is a nuclear shuttling protein that binds and hydrolyzes the m(7)GpppN mRNA cap structure and a modulator of RNA metabolism. Therefore, DcpS represents an intrigueing therapeutic target for modulating gene expression by a small molecule. The exact therapeutic mechanism remains unknown.

Diethylnorspermine is the structural analog of polyamines. Polyamines were shown to downregulate polyamine synthesis and uptake, upregulate polyamine catabolism, and result in a profound depletion of polyamines. Diethylnorspermine is adenosylmethionine decarboxylase, ornithine decarboxylase inhibitor and spermidine-spermine N1-acetyltransferase stimulant. Diethylnorspermine is under investigation as a novel anticancer drug for a variety of neoplasms. It has sustained inhibitory effects on the growth of human prostate carcinoma cells in vitro as well in vivo. This polyamine analog may provide a new tool in the chemotherapy of prostate cancers with various phenotypes. Diethylnorspermine was relatively well tolerated in patients with advanced hepatocellular carcinoma (HCC) including those with mild-to-moderate liver dysfunction but further evaluation of diethylnorspermine monotherapy for advanced HCC does not appear to be justified because of insufficient evidence of clinical benefit in the patients evaluated in this study. Also, no evidence of clinical activity was detected in metastatic breast cancer treatment (phase II clinical study).

INCB-8761 (PF-4136309) is an orally available human chemokine receptor 2 (CCR2) antagonist with potential immunomodulating and antineoplastic activities. Upon oral administration, CCR2 antagonist INCB-8761 specifically binds to CCR2 and prevents binding of the endothelium-derived chemokine ligand CLL2 (monocyte chemoattractant protein-1 or MCP1) to its receptor CCR2, which may result in inhibition of CCR2 activation and signal transduction. This may inhibit inflammatory processes as well as angiogenesis, tumor cell migration, and tumor cell proliferation. INCB-8761 is a potent CCR2 antagonist with high selectivity, weak hERG activity, high free fraction in protein binding, and an excellent in vitro and in vivo ADMET (ADME and toxicology) profile. INCB-8761 entered human clinical trials. It is in phase I/II clinical trials for pancreatic cancer.