Nacubactam (FPI-1459) was developed as an antibacterial drug. Nacubactam successfully has completed phase I clinical trials for the treatment of serious gram-negative bacterial infections. The drug is currently being developed for the treatment of complicated urinary tract infection, hospital-acquired bacterial pneumonia, ventilator-associated bacterial pneumonia, and complicated intra-abdominal infections. FPI-1459 works through several mechanisms of action, inhibiting a number of beta-lactamase enzymes as well as certain bacterial cell wall enzymes. In January 2019, FPI-1459 received Fast Track and Qualified Infectious Disease Product designations from the U.S. Food and Drug Administration (FDA).

Sepetoprost (also known as ONO-9054), prostaglandin analog was developed as a dual agonist of prostaglandin E3 (EP3) and prostaglandin F (FP) receptors. It is known, that the use of a dual prostaglandin EP3 and prostaglandin F receptor agonists is a novel approach for the reduction of intraocular pressure (IOP) in open-angle glaucoma and ocular hypertension. Sepetoprost was successfully studied in clinical trials phase II for the treatment of open-angle glaucoma, ocular hypertension, and mild open angle-glaucoma.

ST-101 (also known as ZSET1446) is an azaindolizinone derivative patented by Zenyaku Kogyo Kabushiki Kaisha for the treatment of Alzheimer's disease and improvement of cerebral function. In preclinical models, ST-101 stimulates acetylcholine release and improves methamphetamine-induced impairment of recognition memory in mice by activating extracellular signal-regulated kinase 1/2. Impaired neurogenesis observed in olfactory bulbectomized mice was significantly improved by chronic administration with ST-101. We confirmed that administration with mecamylamine, a nicotinic acetylcholine receptor antagonist, inhibits ST-101-enhanced neurogenesis in the dentate gyrus. ST-101 administration also restored decreased phosphorylation of Akt and extracellular signal-regulated kinase in the dentate gyrus of olfactory bulbectomized mice.

RRX-001, also known as ABDNAZ, is a dinitroazetidine derivative with potential radiosensitizing activity. Upon administration, RRx-001 is able to dilate blood vessels, thereby increasing tumor blood flow and thus improving oxygenation to the tumor site. By increasing oxygen levels, these tumor cells may be more susceptible to radiation therapy. Tumor hypoxia is correlated with tumor aggressiveness, metastasis and resistance to radiotherapy. In mouse models, RRx-001 administered intravenously as a single agent was equipotent to cisplatin while better tolerated. RRx-001 also showed activity as a radiosensitizer in both in vitro and in vivo models. The activity of RRx-001 is thought to be associated with a nucleophilic substitution by circulating thiol compounds and covalent binding of RRx-001 to cysteinyl residues in Hb, followed by the generation of nitrogen oxides. During 2014-2015 EpicentRx has launched Phase 2 trials in brain, colorectal, non-small cell lung, small cell lung and cholangiocarcinoma both alone and in combination. The anti-proliferative effects of RRx-001 are not explainable via a single mechanism. RRx-001 exerts its anti-proliferative effect, at least partially, through interference with glucose 6 phosphate dehydrogenase (G6PD), a key enzyme in the pentose phosphate pathway, responsible for maintaining adequate levels of the major cellular reductant, NADPH.

MIP-1095 I-123 is under investigation in clinical trial phase II to evaluate the safety and efficacy in combination with enzalutamide in patients with prostate-specific membrane antigen (PSMA)-avid metastatic castration-resistant prostate cancer who have progressed on abiraterone. It is known that MIP-1095 potently inhibited the glutamate carboxypeptidase activity of PSMA and when radiolabeled with 123I exhibited high affinity for prostate-specific membrane antigen (PSMA) on human prostate cancer LNCaP cells.

Gemilukast (ONO-6950) is an orally active compound that has been evaluated for the treatment of asthma. It is an CysLT1 and CysLT2 antagonist. Activation of CysLT2 receptors has been suggested to contribute to antigen-induced bronchoconstriction (constriction of the airways in the lungs) in certain asthma patients. In addition to its CysLT1 and CysLT2 antagonist activity, Gemilukast was shown to dose-dependently reduce LTC4-induced bronchoconstriction in asthmatic models, and reduce antigen-induced constriction of isolated human bronchi. A phase II trial with Gemilukast was discontinued in 2018.