Aderbasib, also known as INCB007839, is an orally bioavailable low nanomolar hydroxamate-based inhibitor of the ADAM (A Disintegrin And Metalloprotease) family of multifunctional membrane-bound proteins with potential antineoplastic activity. Aderbasib represses the metalloproteinase "sheddase" activities of ADAM10 and ADAM17, which may result in the inhibition of tumor cell proliferation. In preclinical studies, Aderbasib with lapatinib prevented the growth of HER-2/neu–positive BT474-SC1 human breast cancer xenografts in vivo. Aderbasib was being developed by Incyte as a potential adjunctive treatment for metastatic breast cancer. Aderbasib in combination with trastuzumab increased the response rate in HER2 positive metastatic breast cancer patients with advanced disease, relative to historical controls (50% versus 15–35%). INC7839 also improved progression-free survival in a subset of the patients expressing the p95 fragment of HER2. Aderbasib development was halted in 2011 after positive findings from Phase II trials were contradicted by further research. Aderbasib is currently being tested in combination with rituximab for diffuse large B cell non-Hodgkin lymphoma.

Thiambutosine is a carbanilide derivative patented by Imperial Chemical Industries Ltd. as the antituberculous agent. In vitro, the resistance of Mycobacterium tuberculosis H37Rv to Thiambutosine develops more slowly than resistance to streptomycin, p-aminosalicylic acid, or isoniazid. A sub-inhibiting concentration of Thiambutosine with either streptomycin or isoniazid considerably retards the emergence of strains resistant to these two agents.

AZD1775 selectively targets and inhibits WEE1, a tyrosine kinase that phosphorylates cyclin-dependent kinase 1 (CDK1, CDC2) to inactivate the CDC2/cyclin B complex. Inhibition of WEE1 activity prevents the phosphorylation of CDC2 and impairs the G2 DNA damage checkpoint. This may lead to apoptosis upon treatment with DNA damaging chemotherapeutic agents. Current ongoing trials of AZD1775 include monotherapy and combination therapy with certain DNA damaging agents in solid tumors, ovarian tumors, gynaecological cancer, non-small cell lung cancer. AZD1775 is genotoxic, which is considered to be a result of its mechanism of action. Common serious adverse events (with chemotherapy) include: febrile neutropenia, neutropenia, thrombocytopenia.

Mafosfamide is a synthetic oxazaphosphorine derivative with antineoplastic properties. Mafosfamide alkylates DNA, forming DNA cross-links and inhibiting DNA synthesis. The effects of mafosfamide on various types of cancer cells were determined during preclinical investigations and clinical trials. Its development has been discontinued.

Rubitecan [Orathecin™] is a topoisomerase I inhibitor extracted from the bark and leaves of the Camptotheca acuminata tree, which is native to China. Rubitecan is an oral compound being developed for the treatment of pancreatic cancer and other solid tumours by SuperGen. Rubitecan binds to and inhibits the enzyme topoisomerase I and induces protein-linked DNA single-strand breaks, thereby blocking DNA and RNA synthesis in dividing cells; this agent also prevents repair of reversible single-strand DNA breaks.

Atizoram (CP-80633) is a phosphodiesterase IV inhibitor with bronchodilatory and antiinflammatory properties. It was in phase II trials with Pfizer in the US for the treatment of asthma, atopic dermatitis and psoriasis. Development of atizoram has been discontinued in the asthma indication and no recent development has been reported for the other indications. CP-80633 inhibits PDE4 isozymes (human lung IC50 = 1.27 uM) in the absence of effects on PDE1, PDE2, PDE3 and PDE5 isozymes (IC50 > 100 uM). It exhibits no significant selectivity for any single cloned PDE4A, B, C or D isoform.

Cetylamine is an aliphatic primary amine that possesses surface-active properties and widely used in water treatment. Cetylamine shows moderate anti-tuberculosis activity. Cetylamine also may be used as a source of fluoride in the prevention of dental caries.

Diflomotecan is an E-ring modified camptothecin analogue, which possesses greater lactone stability in plasma compared with other topoisomerase I inhibitors. Diflomotecan was the first homocamptothecin to enter clinical studies. Phase I data are summarized for both the intravenous and oral schedules. The toxicity is primarily haematological while no severe gastrointestinal toxicity has been observed in contrast to other topoisomerase I inhibitors. Diflomotecan has a high oral bioavailability (72 - 95%) and the oral day 1 - 5 every 3 weeks regimen is recommended for Phase II testing because it is relatively well tolerated. Diflomotecan had been in Phase II clinical trials for the treatment of small cell lung cancer. However, this research has been discontinued.

Halopemide is a new psychotropic agent, a structural analog of the neuroleptics of the butyrophenone type but with different pharmacological and clinical properties. Preliminary clinical findings indicate that halopemide lacks the ability to induce parkinsonism and may be an effective drug in the treatment of psychosis characterized by autism, emotional withdrawal or apathy. However, the results from of the double-blind study showed no significant difference between the placebo phase and halopemide phase. Halopemide is phospholipase D2 inhibitor: PLD1 and PLD2. Halopemide may be used as a screen to identify inhibitors of human PLD2 using an in vitro biochemical assay. It is also inhibitory at benzodiazepine binding sites.