Flutafuranol F-18 (also known as NAV4694), a fluorine-18 labeled positron emission tomography (PET) imaging agent that was developed for diagnostic use. Flutafuranol F-18 binds to beta-amyloid deposits in the brain that could then be imaged in PET scans. It is known that amyloid plaque pathology is a required feature of Alzheimer’s disease (AD) and the presence of amyloid pathology is a supportive feature for the diagnosis of probable AD. Patients who are negative for amyloid pathology do not have AD. Thus, flutafuranol F-18 was studied in phase III clinical trial as an aid in the imaging for patients with Alzheimer’s disease and in phase II clinical trial for patients with mild cognitive impairment.

PF-04620110 is an orally active, selective and potent DGAT1 (Acyl-CoA:diacylglycerol acyltransferase 1) inhibitor that inhibits triacylglycerol synthesis in cells and in rodents. PF-04620110 inhibits DGAT-1 with an IC50 of 19 nM and shows high selectivity versus a broad panel of off-target pharmacologic end points. In vivo DGAT-1 inhibition has been demonstrated through reduction of plasma triglyceride levels in rodents at doses of ≥0.1 mg/kg following a lipid challenge. On the basis of this pharmacologic and pharmacokinetic profile, PF-04620110 has been advanced to human clinical studies. PF-4620110 had been in phase I clinical trials by Pfizer for the treatment of type 2 diabetes. But this research was discontinued in 2011.

I-BET-762 (GSK 525762) is a small molecule benzodiazepine, by 'mimicking' acetylated histones interferes with the recognition of acetylated histones by BET family of bromodomains (BRD2, BRD3, and BRD4), which disrupts chromatin remodeling and gene expression. Prevention of the expression of certain growth-promoting genes may lead to an inhibition of tumour cell growth. GlaxoSmithKline is developing GSK 525762 for the oral treatment of solid tumours and haematological malignancies.

Emixustat (formerly ACU-4429) is a nonretinoid compound with a unique mode of action in the retinal pigment epithelium, where it modulates the biosynthesis of visual chromophore through its effect on retinal pigment epithelium protein 65 (RPE65). Emixustat modulates the visual cycle by inhibiting a critical enzyme of this pathway RPE65. Acucela Inc., a Kubota Pharmaceutical company, is developing emixustat for the treatment of Stargardt disease and proliferative diabetic retinopathy.

AZD0328, a spirofuropyridine, is a selective alpha7 nicotinic receptor agonist. This drug participated in clinical trials phase II in patients with schizophrenia. However, studies were terminated because the drug didn’t meet the current target product profile. Besides AZD0328 has been studied in phase I for the treatment of Alzheimer's disease.

Defactinib is an oral, investigational drug candidate for the treatment of various solid tumors. Through dual inhibition of FAK and PYK2, defactinib targets key resistance mechanisms in the tumor microenvironment (TME), including limited local immune response, dense stroma, and resident cancer stem cells, that may limit the effectiveness of current and investigational treatments. Treatment-related adverse events are: unconjugated hyperbilirubinemia, fatigue and headache.

T-900607 is a pentafluorophenylsulphonamide derivative patented by Tularik Inc. as antiproliferative agent. Antitumor mechanism of T900607 is similar to the vinca alkaloids in terms of disruption of microtubule polymerization but uniquely causes a specific covalent modification of β-tubulin. In preclinical studies, T900607 was shown to bind irreversibly and specifically to the β1, β2, and β4 isotypes of β-tubulin is not a substrate for p-glycoprotein drug pump and has activity in the preclinical setting in MDR models. T900607 was evaluated in human tumor xenografts and showed activity in MX-1, MCF-7, and MCF-7/ADR mammary, C13 ovarian, HT 29 colon, and Caki-1 renal carcinoma as well as lymphoblastic leukemia, with equal or more efficacious effects compared to vinblastine, doxorubicin and paclitaxel. In a clinical trial, T-900607 shows significant toxicity, consisting of thrombocytopenia, nausea/vomiting, fatigue, and apparent cardiac toxicity.

GI-181771X is part of a class of organic compounds known as benzodiazepines. It is a cholecystokinin 1 receptor agonist investigated by GlaxoSmithKline for the treatment of obesity. In one study, GI181771X did not reduce body weight and had no effect on waist circumference or other cardiometabolic risk markers. This negative result was later assigned to an inadequate trial design rather than compound activity. Gastrointestinal side effects were more common with GI181771X than with placebo treatment. Also, in rodents, morphological changes in the pancreas were observed after administration of GI-181771X, including necrotizing pancreatitis, acinar cell hypertrophy/atrophy, zymogen degranulation, focal acinar cell hyperplasia, and interstitial inflammation. In contrast however, pancreatic changes were not observed in monkeys or in human obese patients (in a clinical trial).