Sintropium [VAL 480] is a gastrointestinal, biliary and urinary spasmolytic agent that was under development with Valeas in Italy. It is a muscarinic receptor antagonist. Sintropium bromide has a prompt anticholinergic action and for this reason may be used in the treatment of painful conditions of the bile, gastro-enteric and renal tracts, and also during the course of endoscopic examinations.

F-351 (Hydronidone), a pyridine derivative, is a non-steroidal antiinflammatory drug. It inhibits hepatic stellate cell proliferation and also the TGF-β signaling pathway. Shanghai Genomics is developing F-351 for the treatment of liver fibrosis induced by HBV chronic hepatitis (HBV). For additional indications, F-351 has the potential to be further developed for Non-alcoholic steatohepatitis (NASH) liver disease, Non-alcoholic fatty liver disease (NAFLD) and chronic kidney failure.

MedImmune is developing MEDI 9197 (formerly 3M-052), a toll-like receptor 7/8 dual agonist, for the treatment of cancer. MEDI9197 has been designed to activate a broad range of innate immune cells through targeting of both TLR 7 and 8, leading to a more robust adaptive immune response. A TLR7 and 8 dual agonist can additionally convert immune suppressive cells in the tumor to those with anti-tumor properties, allowing the generation of an effective anti-tumor response. MEDI9197 is uniquely designed for intratumoral injection, allowing the compound to be retained in the tumor and provide specific immune activation, enhancing its safety and tolerability profile.

Osaterone acetate (previously known as TZP-4238), a synthetic steroidal anti-androgen agent. Osaterone acetate is used in veterinary in Europe in the treatment of benign prostatic hyperplasia (BPH) in male dogs. Osaterone acetate inhibits the effects of an excess of male hormone (testosterone) through various mechanisms. It competitively prevents the binding of androgens to their prostatic receptors and blocks the transport of testosterone into the prostate. Osaterone acetate was also investigated in Japan in the treatment of prostate cancer and BPH and, in addition, was studied in postmenopausal osteoporosis in humans. However, these studies were discontinued.

NM-404 I-124 ([124I]CLR-1404, LIGHT ) is a phospholipid ether analog labeled with iodine I 124, with a potential imaging property upon positron emission tomography (PET). Upon administration, NM-404 I-124 selectively accumulates in and is retained within tumor cells for a prolonged period of time due to the decreased activity of a phospholipase D (PLD), most likely isoform 1 of PLD, in tumor cells compared to normal cells. As tumor cells are unable to metabolize and eliminate MN404, tumor cells can be visualized upon PET imaging. In addition, NM-404 I-124 may provide a more accurate image of the tumor than imaging with the current standard. PLD is an enzyme found in the cell membrane of normal cells that degrades phospholipids. NM-404 I-124 enters cells predominately via lipid rafts, specialized microdomains of plasma membranes enriched in cholesterol and glycosphingolipids, which are found to be overexpressed 6-10 fold in malignant cells compared to normal cells. Although blood pool uptake in major vascular structures demonstrates high uptake initially, there is no significant CLR1404 uptake in normal brain tissue. Therefore, avid brain tumor uptake results in high tumor to back-ground signal and thus clear identification of viable tumor cells on PET scans. NM-404 I-124 selectively illuminated malignant tumors in 52 of 54 animal models of cancer, demonstrating evidence of broad-spectrum, cancer-selective uptake and retention. FDA granted orphan drug designation in the United States for NM-404 I-124 as a diagnostic for the management of glioma.

IOWH032 is a synthetic extracellular CFTR inhibitor, originally developed to treat diarrhea, that entered Phase II clinical trials in 2013 but did not progress further in clinical development. iOWH032 has low CFTR inhibition potency (IC50 > 5 uM) and hence rapid (seconds or less) dissociation from CFTR. iOWH032 inhibited secretion by nearly 70% as measured on a semi-quantitative fecal output scale.

Artefenomel, a novel trioxolane, is a lead candidate for inclusion in a new antimalarial combination, specifically formulated for children. Artefenomel has been demonstrated curative in as little as one dose.

Vesatolimod, also known as GS-9620, is being developed in clinical studies for the treatment of chronic hepatitis B viral (HBV) infection, with the goal of inducing a liver-targeted antiviral effect without inducing the adverse effects associated with current systemic interferon-α (IFN-α) therapies. It is demonstrate interferon-stimulated gene induction without detectable serum interferon at low oral doses. GS-9620 is a potent and oral agonist of Toll-like receptor-7, a pattern-recognition receptor whose activation results in innate and adaptive immune stimulation