Rafigrelide is an imidazoquinazoline derivative patented by pharmaceutical company Shire LLC as platelet-lowering agent for the treatment of myeloproliferative diseases. Rafigrelide is a chemical analog of anagrelide, which is used to reduce platelet counts in myeloproliferative disorders. Compared with anagrelide, Rafigrelide has reduced potency against phosphodiesterase III, which may help to reduce potential side effects. The concentration of Rafigrelide that produces 50% of the maximum inhibition (IC50) of PDE III is 164 nM, making it an approximately 200-fold less potent inhibitor of phosphodiesterase III than 3-hydroxy anagrelide. In the clinical trial, Rafigrelide showed antithrombotic properties during a two-week treatment period in healthy male volunteers. The reductions in thrombus formation were seen in surrogate models of both high and low shear rates suggesting drug efficacy in different arterial conditions.

Alisporivir (DEBIO-025) is a first-in-class synthetic cyclophilin inhibitor, in development with Debiopharm as an oral treatment for hepatitis C virus (HCV) infections. Alisporivir has the potential to be used for the treatment of additional diseases such as other viral infections, certain muscular dystrophies, and myocardial infarction. Alisporivir has potent anti-hepatitis C virus (HCV) activity both in vitro and in vivo. It is currently being evaluated in phase II clinical trials.

Danusertib is a serine/threonine kinase inhibitor of multiple kinases, including aurora-A, B, and C. It also inhibits several cancer related tyrosine kinases as well as Abl, Trk-a, fibroblast growth receptor-1 and Ret. Danusertib is in phase II trials for the treatment of solid tumours, prostate cancer and chronic myeloid leukemia (CML). The most frequently reported side effects were neutropenia, nausea, anorexia, fatigue, and diarrhea.

LADOSTIGIL, a rasagiline derivative, is a reversible acetylcholinesterase and butyrylcholinesterase inhibitor with neuroprotective properties. It also acts as an irreversible brain monoamine oxidases inhibitor. It is under development for the treatment of neurodegenerative disorders like dementia and Alzheimer's disease.

Inecalcitol is a calcitriol analog with potential antineoplastic activity patented by a global pharmaceutical company Laboratoire Theramex. Inecalcitol is a potent agonist of vitamin D receptor (VDR). Inecalcitol was shown to be more potent than calcitriol in decreasing tumor cell growth and inducing apoptosis in a number of different model systems including models of breast cancer, prostate cancer, and squamous cell cancer. Importantly, at the doses shown to induce tumor regression in the animal models investigated, Inecalcitol had no major effect on blood calcium levels. In this phase I study, Inecalcitol was found to be well tolerated. Currently, Inecalcitol in combination with the cytotoxic drug, decitabine is undergoing a phase II clinical trial in patients with acute myeloid leukemia who are unfit to receive standard chemotherapy.

Tozasertib, originally developed as VX-680 by Vertex (Cambridge, MA) and later renamed MK-0457 by Merck (Whitehouse Station, NY), was the first aurora kinase inhibitor to be tested in clinical trials. The drug, a pyrimidine derivative, has affinity for all aurora family members at nanomolar concentrations with inhibitory constant values (Ki(app)) of 0.6, 18, and 4.6 nM for aurora A, aurora B, and aurora C, respectively. Preclinical studies confirmed that tozasertib inhibited both aurora A and aurora B kinase activity, and activity has been reported against prostate, thyroid, ovarian, and oral squamous cancer cell lines. Upon treatment with tozasertib, cells accumulate with a 4N DNA content due to a failure of cytokinesis. This ultimately leads to apoptosis, preferentially in cells with a compromised p53 function. Tozasertib is an anticancer chemotherapeutic pan-aurora kinase (AurK) inhibitor that also inhibits FMS-like tyrosine kinase 3 (FLT3) and Abl. Tozasertib is currently in clinical trials as a potential treatment for acute lymphoblastic leukemia (ALL). In cellular models of cancer, tozasertib activates caspase-3 and PARP and decreases expression of HDAC, increasing apoptosis and inhibiting cell growth. In other cellular models, tozasertib inhibits cell proliferation and metastasis by blocking downstream ERK signaling and downregulating cdc25c and cyclin B. This compound also decreases tumor growth in an in vivo model of prostate cancer.

Sunepitron (CP-93,393) is an anxiolytic drug with highly selective serotonin 5-hydroxytryptamine 1A autoreceptor agonist, alpha2-adrenergic antagonist, and dopamine D2 agonist properties. Sunepitron hydrochloride had been in Phase III clinical trials by Pfizer for the treatment of anxiety disorder and depression. However, this research has been discontinued.

Oglufanide, an angiogenesis inhibitor, an immunomodulator, that originally was developed and registered in Russia under the brand name timogen. Oglufanide inhibits vascular endothelial growth factor (VEGF), which may inhibit angiogenesis. This agent has also been reported to stimulate the immune response to hepatitis C virus and intracellular bacterial infections. Oglufanide was studied in the USA for the treatment of cancer, and in September 2001, it was granted Orphan Drug designation for the treatment of ovarian cancer. In addition, in Australia this drug was involved in phase II clinical trial for the treatment of hepatitis C. Oglufanide is also participated in phase III trials for patients with Kaposi's sarcoma, however, this study was discontinued.