Benafentrine (also known as AH 21-132), an inhibitor of the phosphodiesterase (PDE) III and IV isoenzymes, had an anti-allergic effect and was studied for the treatment of allergic bronchial asthma. However, the development of the drug was discontinued at phase I.

Batelapine (or CGS 13429), an antipsychotic drug, that was undergoing preclinical development, but the further studied were discontinued

Sardomozide (previously known as SAM486A or CGP-48664) was developed as an inhibitor of S-adenosyl-methionine-decarboxylase, a key enzyme for polyamine biosynthesis. Sardomozide possessed the broad-spectrum antiproliferative and antitumor activity. The drug participated in a phase II clinical trial as a monotherapy in patients with refractory or relapsed non-Hodgkin's lymphoma. In Phase II clinical trial in patients with metastatic melanoma, the drug didn’t have significant therapeutic potential. The further development of this drug was discontinued.

Dovitinib is an orally active small molecule that exhibits potent inhibitory activity against multiple receptor tyrosine kinases (RTK) involved in tumor growth and angiogenesis. Dovitinib strongly binds to fibroblast growth factor receptor 3 (FGFR3) and inhibits its phosphorylation, which may result in the inhibition of tumor cell proliferation and the induction of tumor cell death. In addition, this agent may inhibit other members of the RTK superfamily, including the vascular endothelial growth factor receptor; fibroblast growth factor receptor 1; platelet-derived growth factor receptor type 3; FMS-like tyrosine kinase 3; stem cell factor receptor (c-KIT); and colony-stimulating factor receptor 1; this may result in an additional reduction in cellular proliferation and angiogenesis, and the induction of tumor cell apoptosis. There are several ongoing Phase I/III clinical trials for dovitinib.

Acumapimod (BCT-197) is an oral p38 MAP kinase inhibitor and an anti-inflammatory agent developed by Mereo BioPharma that has completed Phase 2 development as first-line therapy for severe acute exacerbations of chronic obstructive pulmonary disease. Another study on acute kidney injury in patients undergoing cardiac surgery had been discontinued. Acumapimod is an inhibitor of p38a with an IC50 value < 1 uM. Previous studies undertaken by Novartis, showed the drug has the capacity to reduce the inflammatory marker TNFα and to increase FEV1.

CGM-097, a novel, highly optimized, and selective inhibitor of the p53-Mdm2 interaction. CGM-097 binds to human Mdm2 protein with a Ki value of 1.3 nM, activates p53 in human cells and induces robust p53-dependent cell cycle arrest and apoptosis in human p53 wild-type tumor cells. Its activity and selectivity has been tested and confirmed across a large panel of cancer cell lines from the Cancer Cell Line Encyclopedia. CGM-097 displays desirable pharmacokinetic and pharmacodynamic profiles in animals together with excellent oral bioavailability, which triggers rapid and sustained activation of p53-dependent pharmacodynamic biomarkers resulting in tumor regression in multiple xenografted models of p53 wild-type human cancer. The validation and understanding of its mechanism of action, the overall favorable drug-like properties and the characterization of its on-target toxicological profile in preclinical species strongly supported the initiation of Phase I clinical trials with CGM-097 in pre-selected patients with p53 wild-type tumors.

BLZ 945, an orally active antagonist of the colony-stimulating factor1 receptor (CSF1R), is being developed by Novartis and Celgene Corporation for the treatment of advanced solid tumors and tumor-induced osteolytic lesions in bone and skeletal-related events. Phase I/II development for solid tumors is underway in the US, Italy, Spain, and Singapore. Preclinical trials were ongoing for tumor-induced osteolysis in Europe and the US. However, no recent reports of development had been identified for this indication.

FGF-401 is an inhibitor of human fibroblast growth factor receptor 4 (FGFR4), with potential antineoplastic activity. Upon administration, FGF401 binds to and inhibits the activity of FGFR4, which leads to an inhibition of tumor cell proliferation in FGFR4-overexpressing cells. FGFR4 is a receptor tyrosine kinase upregulated in certain tumor cells and involved in tumor cell proliferation, differentiation, angiogenesis, and survival. FGF-401 is an FGFR4 inhibitor in phase I/II clinical studies at Novartis for the treatment of positive FGFR4 and KLB expression solid tumors and hepatocellular carcinoma.

LIK-066 (licogliflozin) is an inhibitor of the sodium-dependent glucose co-transporter (sodium-glucose transporter; sodium-glucose transport protein; sodium-glucose linked transporter; SGLT) family members 1 and 2 (SGLT1/2) with antihyperglycemic activity. By binding to and blocking SGLT1/2, licogliflozin suppresses the reabsorption of glucose in the proximal tubule within the kidneys and enhances urinary excretion of glucose. This normalizes blood glucose levels. LIK-066 is in phase II clinical trials by Novartis for the treatment of type 2 diabetes. Licogliflozin treatment (1-84 days) leads to significant weight loss and favorable changes in a variety of metabolic parameters and incretin hormones. Dual inhibition of SGLT1/2 with licogliflozin in the gut and kidneys is an attractive strategy for treating obesity and diabetes.

Cipargamin is an experimental synthetic antimalarial molecule belonging to the spiroindolone class. It possesses both the potency (average IC50 of 550 pM against asexual blood-stage P. falciparum) and favorable pharmacokinetics (elimination half-life of ~24 hours in humans) needed for a single-dose cure, a feature that could help slow the onset of parasite resistance and that is not shared by existing, approved antimalarial drugs. KAE609 is also unique in its ability to block transmission to mosquitoes. Cipargamin is a parasite P-type ATPase4 inhibitor. Cipargamin in phase II clinical trials for the treatment of acute, uncomplicated malaria due to plasmodium falciparum monoinfection. Nausea was the most common reported adverse effect. The adverse events were generally mild and did not lead to any discontinuations of the drug.