3-Pyridinecarboxylic acid, 1-(4-methylphenyl)ethyl ester is an choleretic agent.

D-Fagomine (1,2-dideoxynojirimycin) is a six-membered ring iminocyclitol that was first isolated from seeds of buckwheat (Fagopyrum sculentum Moench, Polygonaceae) and is also present in other plant sources such as mulberry (Morus Alba, Moraceae) leaves and gogi (Lycium chinense) roots. D-fagomine is present in common buckwheat-based foodstuffs in amounts ranging from 1 to 25 mg/kg or mg/L, it is stable during boiling, baking, frying and fermentation, and it is biosynthesised upon sprouting. The estimated total intake of D-fagomine resulting from a diet that includes such foodstuffs would be between 3 and 17 mg per day (mean for both genders; range from P5 to P95). In animal studies D-Fagomine lowers postprandial blood glucose. D-fagomine agglutinated Enterobacteriaceae (Escherichia coli, Salmonella enterica serovar Typhimurium), while it did not show this effect on Bifidobacterium spp. or Lactobacillus spp. D-fagomine significantly inhibited the adhesion of Enterobacteriaceae and promoted the adhesion of Lactobacillus acidophilus to intestinal mucosa. D-Fagomine did not show any effect on bacterial cell viability. D-fagomine may be used as a dietary ingredient or functional food component to reduce the health risks associated with an excessive intake of fast-digestible carbohydrates, or an excess of potentially pathogenic bacteria.

CYR-101 (MIN-101) is a cyclic amide derivative that has high equipotent affinities for 5-HT2A and sigma-2 receptors (Ki of 7.53 nM and 8.19 nM for 5-HT2A and sigma-2, respectively). MIN-101 also shows binding affinity for a1-adrenergic receptors but low or no affinity for muscarinic, cholinergic, and histaminergic receptors. MIN-101 demonstrated statistically significant efficacy in reducing negative symptoms and good tolerability in stable schizophrenia patients. The drug is in phase II clinical trials for the treatment of Schizophrenia.

Buthionine sulfoximine (BSO) is a selective inhibitor of γ-glutamylcysteine synthetase (γ-GCS), the rate-limiting enzyme in glutathione (GSH) synthesis. In cancer cells, glutathione depletion significantly increased cytotoxicity via oxidative stress. In addition, in neuroblastoma cells susceptible to Buthionine sulfoximine treatment, DNA damage and cell apoptosis occurred via ROS production. Buthionine sulfoximine plus melphalan was effective in treatment for patients with recurrent/refractory neuroblastoma. Buthionine sulfoximine may also be used to increase the sensitivity of parasites to oxidative antiparasitic drugs. Buthionine sulfoximine has been shown to increase the efficacy of nifurtimox against T. cruzi and has also been shown to be an effective modulator of GSH-mediated chemoresistance by increasing the in vitro cytotoxicity of alkylating agents and radiation. Buthionine sulfoximine has been tested in animal studies and in human phase I trials for adults with solid tumors, with documented clinical responses in patients with melanoma, ovarian carcinoma and small cell carcinoma of the lung treated with the combination of Buthionine sulfoximine and melphalan.

Veledimex is an oral activator ligand for a proprietary gene therapy promoter system, and a moderate inhibitor of and substrate for CYP3A4/5. Veledimex controls the expression of the target gene. The amount of gene product produced by the system and the duration of the effect is dependent on veledimex dose level and duration of dosing. Nonclinical studies demonstrated that intratumoral administration of Ad-RTS-IL12 along with oral administration of veledimex elicited dose-dependent anti-tumor effects in murine melanoma, breast cancer and glioma models which correlated with increased plasma exposure of veledimex. The FDA granted Fast Track designation for Ad-RTS-hIL-12 plus veledimex for the treatment of recurrent or progressive glioblastoma multiforme in adults. Ad-RTS-hIL-12 is an inducible adenoviral vector encoding human pro-inflammatory cytokine interleukin-12 (IL-12), which is under the transcriptional control of the RheoSwitch Therapeutic System. Veledixmex is an oral activator ligand. Data previously presented suggest that Ad-RTS-hIL-12 with 20 mg veledimex improves the median overall survival from 6 to 9 months seen with available therapies to 12.7 months, with further improvement in median overall survival to 17.8 months in a subset of subjects with reduced cumulative steroid exposure during the active dosing period of veledimex. Veledimex has been used in trials studying the treatment of glioblastoma multiforme, metastatic breast cancer, and anaplastic oligoastrocytoma.

Evodenoson (ATL-313) is an agonist of adenosine A2A receptor. It exerts anti-inflammatory activity through activation of adenosine A2A receptors on CD4+ T cells and neutrophils. Evodenoson demonstrates efficacy in animal models of sepsis, myocardial infarction, allograft rejection and enteritis.

Esuberaprost (314d) is an active enantiomer of beraprost, a prostacyclin analog. The pharmacologic action of esuberaprost is mediated by specifically binding to PGI2 receptors in smooth muscle cells (the blood vessel’s endothelium) and platelets. Upon binding, the compound widened blood vessels, preventing the formation of blood clots, and lowering blood pressure in the lungs. As a result, it was expected to improve the symptoms of pulmonary arterial hypertension. United Therapeutics announced it has ended its Phase 3 BEAT trial testing esuberaprost as an add-on therapy to its product Tyvaso (inhaled treprostinil) for clinically symptomatic patients with pulmonary arterial hypertension (PAH). According to a company press release, the study failed to reach its main endpoint of delaying the time to first clinical worsening events.