BN-82451 belongs to a family of small molecules designated as multitargeting or hybrid molecules. BN-82451 is orally active, has good central nervous system penetration, and elicits potent neuronal protection and antiinflammatory properties. BN-82451 acts via three major pathways involved in neuronal death: excito-toxicity, oxidative stress, and inflammation, and is also a mitochondrial protective agent. Because BN-82451 is a multitargeting agent, each of its specific sites of action has been extensively evaluated, namely, neuronal excitotoxicity (sodium channel blocker), oxida-tive stress (antioxidant), neuroinflammation (cyclooxygenase inhibitor), and mitochondrialdysfunction (mitochondria-protective properties). BN-82451 was found to exert a significant protection in experimental animal models mimicking aspects of cerebral ischemia, Parkinson disease, Huntington disease, and more particularly amyotrophic lateral sclerosis. BN-82451 is in phase II clinical trials by Ipsen Pharma for the treatment of Huntington’s disease. In 2015, orphan drug designation was assigned in the U.S. to the compound for the indication.

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.

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.

L-aminocarnityl-succinyl-leucyl-argininal-diethylacetal (Myodur or C101) is a compound that comprise a carrier residue that is an analog of carnitine, a linker group, and a residue of a protease inhibitor comprising an acetal derivative of the aldehyde group. Myodur is a muscle cell targeted product that inhibits calpain in an effort to preserve muscle tissue. In 2006 CepTor Corporation (OTCBB:CEPO), a development-stage biopharmaceutical company focusing on cell-targeted therapeutic products for neuromuscular and neurodegenerative diseases announced that the Office of Orphan Products Development of the Food and Drug Administration (FDA) granted orphan drug designation for Myodur for both Duchenne and Becker muscular dystrophies. Myodur development has been discontinued.