TAK-063 is a highly potent, selective, and orally active phosphodiesterase 10A (PDE10A) inhibitor with IC50 of 0.30 nM; >15000-fold selectivity over other PDEs. TAK-063 is currently being evaluated in clinical trials for the treatment of schizophrenia. Phosphodiesterase 10A (PDE10A) is a cAMP/cGMP phosphodiesterase highly expressed in medium spiny neurons (MSNs) in the striatum. TAK-063 represents a promising drug for the treatment of schizophrenia with potential for superior safety and tolerability profiles.

20-Hydroxyecdysone is a naturally occurring ecdysteroid hormone, which is marketed as dietary supplements that can increase strength and muscle mass during resistance training, particularly bodybuilding. It was found, that 20-hydroxyecdysone did not affect body composition or training adaptations nor did they influence the anabolic/catabolic hormone status or general markers of catabolism in resistance-trained males. Because is known, that ecdysteroids have been shown to prevent various changes in mammalian tissues after female sex hormone deprivation. 20-Hydroxyecdysone also was investigated on these properties. It was found in rats, that 20-Hydroxyecdysone had a beneficial effect on reducing blood pressure and consequently preventing dilated cardiac hypertrophy. Some in vitro experiments showed, that 20-hydroxyecdysone had effects on lymphocytes and neutrophils, and may act as an immunomodulator.

CMX-2043 is a is an α-lipoic acid analog developed by Ischemix LLC for reduction of cellular injury and organ damage due to ischemia-reperfusion injury (IRI). CMX-2043 was more effective than lipoic acid in antioxidant effect, activation of insulin receptor kinase, soluble tyrosine kinase, and Akt phosphorylation, and activated insulin-like growth factor 1 as effectively as lipoic acid. In a rat model of cardiac ischemia-reperfusion injury, treatment with CMX-2043 reduced myocardial IRI as measured by the myocardial infarction/area at risk ratio, and reduced the incidence of arrhythmia. In a 360-patient Phase 2 trial, CMX-2043 demonstrated safety but did not meet pre-specified endpoints regarding prevention of contrast-induced acute kidney injury or cardiac injury in cardiac catheterization lab subjects, and no further development of the drug was reported.

TAK-733 had been developed by Millennium Pharmaceuticals (subsidiary of Takeda) for the treatment of adult patients with advanced non-hematological malignancies. In 2015, Takeda discontinued this studies. TAK-733 is an inhibitor of MEK that exhibits anticancer chemotherapeutic activity. In tumor explant models, TAK-733 induces tumor regression. In colorectal cancer cells and melanoma cells, this compound suppresses cell growth. Additionally, TAK-733 decreases tumor growth and weight in animal models of lung cancer and melanoma.

Ipatasertib (LCL161) binds to inhibitors of apoptosis proteins (IAPs) with high affinity and initiates the destruction of cIAP1 and cIAP2, which further induces apoptosis via caspase activation. Ipatasertib is advancing in clinical development including five Phase 2 trials in patients with Breast cancer, Multiple myeloma, Myelofibrosis, Small cell lung cancer and Ovarian cancer. The most common LCL161-related adverse events were nausea and vomiting.

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.

TD139 is a highly potent, specific inhibitor of the galactoside binding pocket of galectin-3. TD139 is formulated for inhalation, which enables direct targeting the fibrotic tissue in the lungs, while minimizing systemic exposure. TD139 was initially developed by a team of scientists from Lund University, Sweden, and Edinburgh University, the UK. TD-139 is in phase I/II clinical trials for the treatment of idiopathic pulmonary fibrosis.

Q203 (6-chloro-2-ethyl-N-(4-(4-(4-(trifluoromethoxy)phenyl)piperidin-1-yl)benzyl)imidazo [1,2-a]pyridine-3-carboxamide) is an an imidazopyridine antitubercular compound. Q203 targets the cytochrome b subunit (QcrB) of the cytochrome bc1 complex. This complex is an essential component of the respiratory electron transport chain of ATP synthesis. Q203 inhibited the growth of multidrug-resistant (MDR) and extensively drug-resistant (XDR) M. tuberculosis clinical isolates in culture broth medium in the low nanomolar range and was efficacious in a mouse model of tuberculosis at a dose less than 1 mg per kg body weight, which highlights the potency of this compound. In addition, Q203 displays pharmacokinetic and safety profiles compatible with once-daily dosing. Q203 is a promising new clinical candidate for the treatment of tuberculosis.