Vatalanib a potent oral tyrosine kinase inhibitor with a selective range of molecular targets, has been extensively investigated and has shown promising results in patients with solid tumors in early trials. Vatalanib selectively inhibits the tyrosine kinase domains of vascular endothelial growth factor (VEGF) receptor tyrosine kinases (important enzymes in the formation of new blood vessels that contribute to tumor growth and metastasis), platelet-derived growth factor (PDGF) receptor, and c-KIT. The adverse effects of vatalanib appear similar to those of other VEGF inhibitors. In the CONFIRM trials, the most common side effects were high blood pressure, gastrointestinal upset (diarrhea, nausea, and vomiting), fatigue, and dizziness.

Begacestat (GSI-953 or PF-5212362), a gamma-secretase inhibitor (GSI) that selectively inhibits cleavage of APP over Notch, was discovered for the treatment of Alzheimer's disease. The drug has shown promise results in phase I clinical trials, however further studies were discontinued.

Ditiocarb, the sodium salt of diethyldithiocarbamate, is a drug with strong antioxidant capacity and chelating activities. It improves the depressed immune responses of newborn and aged mice and mice that are treated with chemotherapy or irradiation. Ditiocarb prevents cisplatin nephrotoxicity in animals without reducing the drug's antitumor activity. Ditiocarb has therapeutic activity in the LP-BM5 murine retrovirus-induced immunodeficiency disease. In that AIDS model, it reduces lymphadenopathy and hypergammaglobulinemia, restores immunocompetence, and prolongs survival. Ditiocarb was safe and reduced the incidence of opportunistic infections in patients with symptomatic HIV infection but ditiocarb had no positive effect on HIV patients. The administration of ditiocarb did not induce any major adverse clinical or biological reactions. Sixty-four patients with nonmetastatic high-risk breast cancer were randomized in a double-blind trial of adjuvant immunotherapy with sodium ditiocarb (DDC) versus placebo. At 6 years, overall survival was 81% in DDC group versus 55%.

IRDABISANT is a histamine H3 receptor inverse agonist with potential therapeutic utility in cognition enhancement.

Cethromycin is a ketolide antibiotic derived from erythromycin A being investigated for use in community-acquired pneumonia and other respiratory tract infections. Cethromycin possesses reliable activity against the bacteria most commonly associated with community-acquired pneumonia including S. pneumoniae, H. influenzae, M. catarrhalis, M. pneumoniae, C. pneumoniae, and L. pneumophila. Unlike fluoroquinolones, cethromycin has a narrower spectrum of activity against gram-negative bacteria, which may reduce the risk of collateral damage and the incidence of Clostridium difficile infection. It offers an advantage over telithromycin in that hepatotoxicity does not seem to be a concern. The FDA denied approval of cethromycin for the treatment of CAP in 2009, requesting more efficacy data.

CI 1040 is an inhibitor of the mitogen-activated protein (MAP) kinase signal transduction pathway and has been shown to specifically inhibit MAP kinase kinase (MEK). CI 1040 was being developed by Parke-Davis (formerly a division of WarnerLambert, Now Pfizer) as an anticancer agent. It was the initial MEK inhibitor to undergo clinical evaluation based on promising preclinical activity. However, its development has been discontinued.

Lomeguatrib is a O6-methylguanine-DNA-methyl-transferase inhibitor which was developed by AstraZeneca for the treatment of cancer. It was tested in phase I and II of clinical trials for the treatment of colorectal cancer, melanoma and other solid tumors.

Dotarizine was developed as antimigraineur. Dotarizine inhibited the 5-HT2A response in a concentration-dependent manner. The mechanism of blockade by dotarizine of cerebral vessels contractility has three components: (i) presynaptic inhibition of noradrenaline release; (ii) blockade of postsynaptic vascular 5-HT receptors; (iii) blockade of Ca(2+)entry into the vascular smooth muscle cell cytosol. The compound does not affect the vascular receptors for noradrenaline, angiotensin II or prostaglandin F(2alpha). Dotarizine had a pronounced protective effect against electric seizures.

Safingol, the synthetic L-threo-stereoisomer of endogenous (D-erythro-) sphinganine, is an inhibitor of protein kinase C and sphingosine kinase in vitro, and in some cell types has been implicated in ceramide generation and induction of apoptosis. Safingol inhibits enzymatic activity and 3H-phorbol dibutyrate binding of purified rat brain PKC (IC50 = 37.5 uM and 31uM, respectively). Inhibits human PKCα, the major overexpressed isoenzyme in MCF-7 DOXR cells (IC50 = 40 uM). Safingol enhances the cytotoxic effect of the chemotherapeutic agent Mitomycin C (MMC) in gastric cancer cells by promoting drug-induced apoptosis. Safingol is an inhibitor of SphK (Sphingosine kinase). Safingol has been shown to act synergistically with other chemotherapeutic agents and may potentiate chemotherapy drug-induced apoptosis in vitro and in vivo.

LECOZOTAN, a benzodioxanylpiperazine derivative, is a selective serotonin 1A receptor antagonist. It was in development for the symptomatic treatment of cognitive deficits in Alzheimer's disease.