SAR407899 is a potent, ATP-competitive Rho kinase inhibitor. It antihypertensive action in animals. Sanofi is developing SAR 407899 for the treatment of microvascular angina (Syndrome X). It was previously being developed in clinical trials for the treatment of diabetic neuropathies, diabetic nephropathies, erectile dysfunction, pulmonary hypertension, hypertension and kidney disorders, but development was discontinued for those indications.

I-BET-762 (GSK 525762) is a small molecule benzodiazepine, by 'mimicking' acetylated histones interferes with the recognition of acetylated histones by BET family of bromodomains (BRD2, BRD3, and BRD4), which disrupts chromatin remodeling and gene expression. Prevention of the expression of certain growth-promoting genes may lead to an inhibition of tumour cell growth. GlaxoSmithKline is developing GSK 525762 for the oral treatment of solid tumours and haematological malignancies.

LX 2931 (LX 3305) is an inhibitor of sphingosine 1-phosphate (S1P) lyase. S1P lyase is an enzyme identified as a promising new target on a pathway associated with regulation of the immune system. Lexicon Pharmaceuticals, Inc. was developing LX 2931 for the treatment of rheumatoid arthritis. LX 2931 has disappeared from the pipeline of Lexicon Pharmaceuticals, Inc. In preclinical studies LX 2931 was effective against experimental cerebral malaria, lung inflammation in a F508del CFTR murine cystic fibrosis model and osteoporosis.

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.

TIC10 (TIC10 isomer or ONC201 isomer) is a potent, orally active, and stable small molecule and is an efficacious antitumor therapeutic agent that acts on tumor cells and their microenvironment to enhance the concentrations of the endogenous tumor necrosis factor-related apoptosis-inducing ligand (TRAIL). The isomeric structure of TIC10/ONC201 is critical to its activity: anti-cancer activity is associated with the angular structure and not the linear TIC10 isomer. TIC10 transcriptionally induces a sustained up-regulation TRAIL in tumors and normal cells in a p53-independent manner. TIC10 inactivates kinases Akt and extracellular signal-regulated kinase (ERK), leading to the translocation of Foxo3a into the nucleus, where it binds to the TRAIL promoter to up-regulate gene transcription. TIC10 crosses the blood-brain barrier. TIC10 treatment caused tumor regression in the HCT116 p53−/− xenograft, RKO human colon cancer xenograft–bearing mice and human triple-negative breast cancer xenografts and significantly prolonged the survival of Eμ-myc transgenic mice, which spontaneously develop metastatic lymphoma from weeks 9 to 12 of age by 4 weeks.

Famitinib, an orally active, small molecule, is a tyrosine kinase inhibitor against multiple targets, including vascular endothelial growth factor receptor 2/3, platelet-derived growth factor receptor, and stem cell factor receptor (c-kit). Jiangsu Hengrui Medicine Co is developing famitinib against a wide variety of advanced-stage solid cancers. Famitinib is participating in phase III clinical trials to evaluate its safety and efficacy in patients with advanced colorectal adenocarcinoma. The other phase III clinical trial for patients with non-squamous non-small cell lung cancer was terminated because of the difficulty in recruitment. In addition, the drug is involved in phase II clinical trials for the treatment of cervical cancer, endometrial cancer, fallopian tube cancer, gastrointestinal stromal tumors, nasopharyngeal cancer, and neuroendocrine tumors.

Moxazocine is a benzomorphan derivative patented by Bristol-Myers Co. as potent opioid analgesic. Moxazocine acts as a partial agonist or mixed agonist/antagonist of the opioid receptors and binds preferentially to the κ-opioid receptor. In clinical studies, Moxazocine demonstrated superior efficacy compared morphine, but was never marketed.