VBP-15 FREE ALCOHOL, also known as Vamorolone and VBP-15, is an anti-inflammatory compound used in the treatment of muscular dystrophy. Vamorolone is a dissociative steroid that retains high affinity binding and nuclear translocation of both glucocorticoid (agonist) and mineralocorticoid (antagonist) receptors, but does not show pharmacodynamic safety concerns of existing glucocorticoid drugs at up to 20 mg/kg/day. Vamorolone is a first-in-class steroidal multi-functional drug that shows potent inhibition of pro-inflammatory NFkB pathways via high-affinity binding to the glucocorticoid receptor, high affinity antagonism for the mineralocorticoid receptor, and membrane stabilization properties. Pre-clinical data in multiple mouse models of inflammation showed retention of anti-inflammatory efficacy, but loss of most or all side effects. Vamorolone has received Orphan Drug Designation in the US and Europe and is being developed for chronic treatment of boys with Duchenne Muscular Dystrophy (DMD).

Phenylacetic acid (abr. PAA and synonyms are: α-toluic acid, benzeneacetic acid, alpha tolylic acid, 2-phenylacetic acid, β-phenylacetic acid) is an organic compound containing a phenyl functional group and acarboxylic acid functional group. Because it is used in the illicit production of phenylacetone (used in the manufacture of substituted amphetamines), it is subject to controls in countries including the United States and China Phenylacetic acid is used in some perfumes, possessing a honey-like odor in low concentrations, and is also used in penicillin G production. It is also employed to treat type II hyperammonemia to help reduce the amounts of ammonia in a patient's bloodstream by forming phenylacetyl-CoA, which then reacts with nitrogen-rich glutamine to form phenylacetylglutamine. This compound is then secreted by the patient's body. In Phase 2 of clinical research it investigated in the treatment of Brain and Central Nervous System Tumors.

N-Methyl-D-aspartic acid is an amino acid derivative acting as a specific agonist at the NMDA receptor, and therefore mimics the action of the neurotransmitter glutamate on that receptor. Unlike glutamate, NMDA only binds to and regulates the NMDA receptor and has no effect on other glutamate receptors (such as those for AMPA and kainate). NMDA receptors are particularly important when they become overactive during withdrawal from alcohol as this causes symptoms such as agitation and, sometimes, epileptiform seizures. NMDA is a water-soluble synthetic substance that is not normally found in biological tissue.

PF-04447943 is a potent, selective brain penetrant PDE9 inhibitor (Ki of 2.8, 4.5 and 18 nM) for human, rhesus and rat recombinant PDE9 respectively and high selectivity for PDE9 versus PDEs1-8 and 10-11. PF-04447943 was being developed by Pfizer for the treatment of cognitive disorders. PF-04447943 attenuates a scopolamine-induced deficit in a novel rodent attention task. PF-04447943 enhances synaptic plasticity and cognitive function in rodents. PF-04447943 has completed Phase II clinical trials in subjects with mild to moderate AD in 2013 but this research was discontinued. Pfizer completes a phase I trial in Sickle cell anaemia.

MPEP (2-methyl-6-(phenylethynyl)pyridine) was one of the first compounds found to act a selective antagonist for the metabotropic glutamate receptor subtype mGluR5. It was under development by Novartis in the late 1990's. MPEP was found to produce neuroprotective effects following acute brain injury in animals. MPEP was also found to have positive effects on animal models of depression, anxiety and morphine withdrawl.

2-Amino-1-methyl-6-phenylimidazo[4,5-b]pyridine (PhIP) is a mutagenic and carcinogenic heterocyclic amine formed during ordinary cooking, and is subsequently metabolically activated by cytochrome P4501A2 (CYP1A2) and N-acetyltransferase 2 (NAT2). PhIP has been used in trials studying the basic science of Pancreas Cancer.